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  1. Friday, October 8, 2021 7:45 am - 4:00 pm OVERVIEW This conference will present the newest approaches and techniques in the diagnosis and treatment of pituitary adenomas, including acromegaly and Cushing’s disease. Diagnosis and treatment will be covered from the interdisciplinary and interprofessional perspective of endocrinology, radiology, neuro-ophthalmology, neurosurgery, and radiation oncology. Didactic presentations will include case discussions. The conference format, although virtual will provide a significant opportunity for interaction with expert faculty. A simulcast of transsphenoidal surgery will occur throughout the conference with real-time discussion and case review of the progress on the day of surgery, post-op management, surveillance and follow-up care. Participants will leave with up-to-date, practical information and written resources including: DDAVP stimulation protocol for Cushing’s disease localization, perioperative glucocorticoid and salt-water monitoring protocol, clinic note templates, laboratory testing panels, "Sick Day Rules" letter for patients with adrenal insufficiency. These materials will have immediate clinical application and help streamline care of pituitary patients at the office and during hospitalizations. LEARNING OBJECTIVES – CME Upon completion of this conference, participants should be able to: Evaluate a sellar mass to determine if it is a pituitary adenoma or other lesion Identify the value and limits of MRI in evaluating a sellar mass List the potential and limits of endoscopic transsphenoidal surgery for pituitary adenoma Manage, medically, a patient following endonasal surgery List the different types of radiation, including linear accelerator (IMRT, Cyberknife), gamma radiation, (Gamma Knife) and proton beam Treat, medically, patients who have acromegaly and Cushing’s disease Apply multidisciplinary, interprofessional and interdisciplinary approach in the management of pituitary disease LEARNING OBJECTIVES – PATIENTS Upon completion of this course patients, families and advocates will be able to: Identify the latest advances in pituitary tumor treatment Demonstrate familiarity with the terminology and technical aspects of pituitary tumor care Demonstrate patient-active behavior in working with the healthcare team to make ongoing treatment decisions WHO SHOULD ATTEND This activity has been designed for endocrinologists, neurosurgeons, ophthalmologists, gynecologists, general radiologists, nurse practitioners, nurses, residents and fellows. Additionally, patients and their caregivers, family members, advocates and members of the public who may benefit from understanding current innovative approaches to pituitary tumor care are invited. For additional information please contact Hyacen Putmon. Register Now
  2. Urine Tests: These involve collecting urine, usually for periods of twenty-four hours at a time. Twenty-four Hour Urine: The doctor will give you a gallon collection jug, usually with boric acid in it. The instructions are usually printed on the side. Generally, you urinate first thing in the morning, as usual. after that, you collect the rest of the urine for the next 24 hours in the jug. The directions usually tell you to refrigerate the jug. Directions for the Twenty-four Hour Urine Test Physicians have always relied upon analysis of urine specimens in order to diagnosis and treat many disease processes. Twenty-four hour urine collections are often employed to estimate the production rates of various hormones. The accuracy of test results depends entirely on the accuracy of the urine collection technique. These instructions are provided as a guide to ensure that your 24-hour urine collection is obtained in a manner that will permit reliance upon the test results. Urine samples should be collected in a large cup, urine collection hat or other container and then poured into the large bottle. Do not try to urinate directly into the bottle. Void urine prior to bowel movements in order to avoid losing urine that might normally be passed during a bowel movement. Urine collection hats can usually be purchased at medical supply stores if not provided by your physician or lab. If you should have a bowel movement while urinating the urine collection hat should keep the urine clean if used correctly. Urine samples should be collected in a large cup or other container and then poured into the large bottle. Do not try to urinate directly into the bottle. Void urine prior to bowel movements in order to avoid losing urine that might normally be passed during a bowel movement. Some patients are asked to collect more than one consecutive 24-hour urine sample. If that is the case, you should complete the first collection as instructed. Then, begin the second collection by adding any urine made in the next 24-hours to the second bottle. You should not discard any urine when starting the second or any subsequent collections. Simply change bottles at the stop and start times after adding that last sample required to complete the previous collection. The bottles for some tests contain a weak acid as a preservative. Do not discard the acid. If you accidentally get acid or urine from the bottle on your skin or clothing, rinse the effected area immediately with plenty of cold water. Collection bottles must be refrigerated. This is best accomplished by using an ice chest, cooler, or if so inclined, your refrigerator. If you forget to collect all of the urine or perform the test improperly, discard the specimen and start again on another day. If the bottle contained an acid preservative, you will need to obtain a new bottle from the laboratory or your physician's office. Otherwise, you may reuse the bottle after rinsing it with distilled water. Finally, please remember to call your physician, medical provider or nurse if you have any questions about the proper collection of a 24-hour urine sample. This Topic on the Message Boards.
  3. Cushing’s syndrome is a rare disorder that occurs when the body is exposed to too much cortisol. Cortisol is produced by the body and is also used in corticosteroid drugs. Cushing's syndrome can occur either because cortisol is being overproduced by the body or from the use of drugs that contain cortisol (like prednisone). Cortisol is the body’s main stress hormone. Cortisol is secreted by the adrenal glands in response to the secretion of adrenocorticotropic hormone (ACTH) by the pituitary. One form of Cushing’s syndrome may be caused by an oversecretion of ACTH by the pituitary leading to an excess of cortisol. Cortisol has several functions, including the regulation of inflammation and controlling how the body uses carbohydrates, fats, and proteins. Corticosteroids such as prednisone, which are often used to treat inflammatory conditions, mimic the effects of cortisol. Stay tuned for more basic info...
  4. Ieva Lase, Malin Grönberg, Olov Norlén, Peter Stålberg, Staffan Welin, Eva Tiensuu Janson First published: 13 August 2021 https://doi.org/10.1111/jne.13030 Abstract Neuroendocrine neoplasms (NENs) causing ectopic Cushing's syndrome (ECS) are rare and challenging to treat. In this retrospective cohort study, we aimed to evaluate different approaches for bilateral adrenalectomy (BA) as a treatment option in ECS. Fifty-three patients with ECS caused by a NEN (35 females/18 men; mean ± SD age: 53 ± 15 years) were identified from medical records. Epidemiological and clinical parameters, survival, indications for surgery and timing, as well as duration of surgery, complications and surgical techniques, were collected and further analysed. The primary tumour location was thorax (n = 30), pancreas (n = 14) or unknown (n = 9). BA was performed in 37 patients. Median time from diagnosis of ECS to BA was 2 months (range 1–10 months). Thirty-two patients received different steroidogenesis inhibitors before BA to control hypercortisolaemia. ECS resolved completely after surgery in 33 patients and severe peri- or postoperative complications were detected in 12 patients. There were fewer severe complications in the endoscopic group compared to open surgery (p = .030). Posterior retroperitoneoscopic BA performed simultaneously by a two surgeon approach had the shortest operating time (p = .001). Despite the frequent use of adrenolytic treatment, BA was necessary in a majority of patients to gain control over ECS. Complication rate was high, probably as a result of the combination of metastatic disease and metabolic disorders caused by high cortisol levels. The two surgeon approach BA may be considered as the method of choice in ECS compared to other BA approaches as a result of fewer complications and a shorter operating time. 1 INTRODUCTION Endogenous Cushing's syndrome (CS) has an estimated incidence of 0.2–5.0 per million people per year.1 In 5–10% of these, it is caused by ectopic secretion of adrenocorticotrophic hormone (ACTH) or, in extremely rare cases, corticotrophin-releasing hormone, from a non-pituitary tumour.1, 2 The treatment of neuroendocrine neoplasms (NENs) with ectopic secretion of ACTH is challenging. Because of the rarity and heterogeneity of this condition, there is no established evidence-based recommendation.3 Most patients with ectopic Cushing's syndrome (ECS) have severe hypercortisolaemia leading to disrupted electrolyte and glucose levels, metabolic alkalosis, thrombosis and life-threatening infections, amongst many other manifestations. Initiation of oncological treatment is often delayed as a result of the consequences of high cortisol levels. A reduction of the cortisol level is crucial for survival and hypercortisolaemia and hypokalaemia are negative prognostic factors.4, 5 If radical surgery of the tumour is not possible because of metastatic disease, normo-cortisolaemia can be achieved either by medical treatment with steroidogenesis inhibitors (SI) or bilateral adrenalectomy (BA),6 and BA has also been considered a treatment option for patients with occult or cyclic ECS. In patients with metastatic neuroendocrine carcinomas, platinum-based chemotherapy may be applied as first-line action, combined by SI and/or followed by BA. Computed tomography-guided percutaneous adrenal ablation has been reported in several case reports as a possible therapeutic alternative for patients in whom medical treatment has failed and BA is not feasible,7-10 althhough more data is needed to recommend this method in daily practice. In the 1930s, transabdominal open access BA was introduced as a treatment option for uncontrolled cortisol secretion.11 Sixty years later, in the 1990s, laparoscopic methods were established12, 13 and are now considered as the gold standard for BA (except for adrenal carcinomas) because they result in less postoperative pain, a shorter hospitalisation time and faster recovery.14 Laparoscopic transperitoneal adrenalectomy (LTA) is the most frequently applied surgical method. However, posterior retroperitoneoscopic adrenalectomy (PRA), introduced in 1995 by Walz et al,15 is gaining popularity.16 Using PRA compared to LTA offers a more direct approach to the adrenal glands, a shorter operating time (no need for reposition of the patient), less blood loss and faster recovery, and it aso has advantages for patients with obesity or a history of previous abdominal surgery.16 There are centres where PRA is performed by a two surgeon approach; thus, a simultaneous bilateral approach offers the possibility of decreasing the surgical time by up to 50% and reducing operative stress.17-19 The present study aimed to evaluate BA as a treatment option for ECS, as well as the effects of different approaches on morbidity and mortality. We hypothesised that endoscopic surgery methods could be superior regarding complication rate, operating and hospitalisation time compared to open access surgery and also influence overall survival. 2 MATERIALS AND METHODS 2.1 Patients and data A cohort of 59 patients with ECS was identified retrospectively from medical records of 894 patients with NENs, referred to the Department of Endocrine Oncology, Uppsala University Hospital between 1984 and 2019. None of the patients had a small-cell lung cancer (SCLC) because these tumours are not treated at our centre and possibly have a different mechanism behind ACTH production compared to that of NENs. Furthermore, SCLCs have a much more severe course of disease compared to well differentiated NENs and including them in the present study could mask any results important for NEN clinical management. Six patients were from outside Sweden and were excluded from further analysis because of a lack of follow-up data; thus, in total, 53 patients were available for analysis. Diagnosis of ECS was confirmed by histopathological examination of tumour specimen (n = 48) together with the clinical and biochemical picture of ACTH-dependent Cushing’s syndrome (elevated serum and urinary cortisol, high ACTH and pathological functional tests). In five patients where neither primary tumor, nor metastatic disease was found despite several PET examinations, including 68 Ga- DOTATOC-PET, 11C-5HTP-PET and 18FDG-PET in four of the five patients, ECS was confirmed on the basis of the clinical/biochemical picture and exclusion of pituitary origin by magnetic resonance imaging, as well as inferior sinus petrosus sampling. Epidemiological data, data on clinical parameters, survival, indication and duration of surgery, complications and surgical technique were extracted and further analysed. 2.2 Surgery BA was performed either by an open access approach, LTA or PRA. PRA was performed either by one surgeon (PRA-1S) or by two surgeons operating on both sides simultaneously (PRA-2S). Some patients were operated twice (one adrenal at the time) and, for those patients, operating time was pooled from both surgeries, if both sessions were performed within 1 week. Cases where conversion from an endoscopic to an open access approach was made peroperatively were grouped as open access surgery in further analysis. Patients who died during the postoperative stage (within 30 days) were excluded from calculation of hospitalisation time. Postoperative complications were graded using the Clavien–Dindo classification where complications of Grade 1 are defined as “any deviation from the normal postoperative course without the need for pharmacological treatment or surgical, endoscopic and radiological interventions. Allowed therapeutic regimens are drugs as antiemetics, antipyretics, analgesics, diuretics and electrolytes and physiotherapy”.20 Because almost all patients had mild, Grade 1 postoperative complications (metabolic disturbances caused by hypercortisolaemia), this variable is not described. We defined complications up to Grade 2 as mild and Grade 3–5 as severe. 2.3 Statistical analysis All parameters were analysed by descriptive statistics: normally distributed data as the mean ± SD, and data with skewed distribution and/or outliers were described as medians, accompanied by the 25th to 75th percentile ranges (Q1-Q3) or minimum-maximum (min-max). The defined event was death from any cause. Overall survival (OS) was defined as time from diagnosis of ECS or time of BA until date of death or, if the event was not found, censored at date of last observation, 31 December 2019. Kaplan-Meier plots were used for survival analysis and the log-rank test was used for comparison. Chi-squared was used for testing relationships between categorical variables. p < .05 was considered statistically significant. All statistical analyses were performed using IBM, version 27 (IBM Corp., Armonk, USA). 3 RESULTS 3.1 Studied patients ECS represented six% (n = 59) of NENs in our cohort. Six patients were excluded from further analysis, resulting in 53 ECS patients who were analysed; there were 35 females and 18 males with a mean ± SD age of 53 ± 15 years. The localisation of the primary NEN was thorax (n = 30), pancreas (n = 14) or unknown (n = 9). Histopathological staining for Ki-67 was available in 38 patients and Ki-67 was < 2% in five patients, 3–20% in 22 patients and > 20% in 11 patients. Patient characteristics are shown in Tables 1 and 2. Twenty-two patients (41.5%) in this cohort had concomitant hypersecretion of hormones other than ACTH from their tumour (5-HIAA, n = 10; calcitonin, n = 3; 5-HIAA + calcitonin, n = 2; glucagon, n = 3, gastrin, n = 2; growth hormone, n = 1; insulin + gastrin + vasointestinal peptide, n = 1). 3.2 Surgery Adrenalectomy was performed in 37 patients (70%); 24 patients were operated at Uppsala University Hospital, nine at Karolinska University Hospital in Stockholm and four at Umeå University Hospital. Median time from diagnosis of ECS to BA was 2 months (range 1–10 months). Median Ki-67 in patients who were operated within 2 months after ECS diagnosis was higher (Ki-67 18.5%) compared to those with BA performed later in the course of disease (Ki-67 9.5%), although the difference was not statistically significant (p = .085). Thirty-two (86%) patients received different SI prior to BA to control hypercortisolaemia. Eight of those were treated with chemotherapy as well in an attempt to reduce cortisol levels. The majority of patients was treated with ketoconazole, often in combination with other drugs (Table 3). Indications for BA in our cohort included (1) persistent hypercortisolaemia despite use of SI (n = 30); (2) BA as first choice of treatment to reduce cortisol levels (n = 5); and (3) no effect combined with severe side effects from SI including liver toxicity and severe leukopenia (n = 2). In 16 patients, BA was not performed as a result of (1) good control of ECS with SI (n = 4); (2) radical surgery of the primary tumour (n = 3); (3) good control of ECS with SI followed by radical surgery of the primary tumour (n = 5) and (4) the bad condition of the patient (n = 4). 3.3 Survival analysis There was no operative mortality in this cohort. Four patients died within 1 month after adrenalectomy (on day 5, 16, 22 and 30, respectively) as a result of multiple organ dysfunction syndrome and progression of NEN. At the end of the follow-up period, 14 patients were still alive and 39 had died. Median survival after BA was 24 months (95% confidence interval [CI] = 7–41, min-max: 0–428) with a 5-year survival of 22%. Median follow-up time for all patients from time of ECS diagnosis was 26 (range 6–62) months and after BA was 19 (range 3–50) months. OS was longer in patients where ECS was treated by radical surgery of the primary tumour or where good biochemical control was achieved by SI compared to patients who underwent BA, 96 months (95% CI 0–206) vs 29 months (95% CI 7–51), respectively. However, this difference was not statistically significant (p = .086), most likely as a result of the small sample size. Multiple hormone secretion correlated with shorter OS after BA (p = .009; hazard ratio = 2.9; 95% CI= 1.3–6.7). There was no significant difference in OS after BA depending on localisation of primary tumour (thoracic NENs 24 months [95% CI = 8–40, min-max: 0–428], pancreatic NENs 19 months [95% CI = 0–43, min-max: 0–60], p = .319) or surgical approach (open access approach 24 months [95% CI = 1–47], endoscopic approach 19 months [95% CI = 1–37], p = .720). Median time from ECS diagnosis to BA was 2 months (range 1–10). Patients who underwent BA within 2 months after ECS diagnosis had shorter OS compared to those who were operated at a later stage: 6 months (95% CI = 0–18) and 45 months (95% CI = 3–86) respectively (p = .007). The former group had a slightly higher median Ki-67 level (18% vs 9%), lower potassium (2.7 mmol L-1 vs 3.0 mmol L-1) and higher hormone levels (ACTH 217 vs 120 ng mL-1, morning cortisol 1448 vs 1181 nmol L-1 and UFC 5716 vs 4234 nmol per 24 h) at diagnosis compared to those who were operated later in the course of disease. 4 DISCUSSION The present study highlights new aspects of the advantages of an endoscopic approach of BA compared to open access surgery, regarding the incidence of severe complications graded using the Clavien-Dindo classification, as well as operation- and hospitalisation time. Our results indicate that PRA performed by two surgeons simultaneously is the method of choice for patients with ECS. However, despite these advantages, the endoscopic approach did not appear to improve overall survival. Recent Endocrine Society guidelines recommend SI as primary treatment for ECS in patients with occult or metastatic ECS followed by BA.6 Although the toxicity of SI in our cohort was low (n = 2; 6%), 32 patients (73%) had persistent hypercortisolaemia despite medical treatment and proceeded to BA. BA, especially with an endoscopic approach, with a short operating time and low complication risk, appears to play a major role in the appropriate management of hypercortisolaemia in ECS, where rapid reduction of cortisol levels is very important. Prolonged exposure to high cortisol levels, in combination with high risk for hepatotoxic and nephrotoxic SI side effects, increases morbidity and risk for severe complications, and often delays the start of oncological treatment. However, the trauma caused by surgery can also postpone initiation of chemotherapy.21 Therefore, a fast and minimally invasive surgical procedure appears to be a crucial factor for the better survival in ECS. The endoscopic approach is now considered as the gold standard for BA. Our study presents fewer severe complications, as well as shorter operating and hospitalisation times, when the endoscopic approach is compared with open surgery. In line with previous studies,19, 22 we observed a significantly shorter operating time when applying PRA compared to LTA because there is no need for repositioning of the patient during PRA. PRA-2S had the shortest operating time and should be considered as the best choice of surgical approach in ECS. This result ties well with previous studies reporting the median operating time to be between 43 and 157 min in PRA-2S, which is significantly shorter compared to LTA and PRA-1S.17-19 The median time from diagnosis to BA was 2 months, which is consistent with a previous study.23 However, OS was significantly shorter in patients who were operated within 2 months after diagnosis of ECS in our cohort compared to those operated at a later stage. These early operated patients probably had a more aggressive clinical course of disease, as indicated by slightly higher median Ki-67, lower potassium and higher hormone levels at diagnosis, and they were operated as a result of more acute indications (without time to proper pre-treatment with SI) than the other group. In our previous report on patients with ACTH-producing NENs, multiple hormone secretion was identified as the strongest indicator of a worse prognosis.4 A similar pattern of results was observed in this cohort, showing that patients with NENs, with concomitant hypersecretion of other hormones than ACTH from their tumour, had a shorter OS after BA compared to those with ACTH hypersecretion only. As a result of the extremely high preoperative cortisol levels in ECS, the substitution therapy needed after successful BA may be challenging.21 Over-replacement of glucocorticoids may lead to higher morbidity24 and mortality, especially in patients with metastatic NENs, who often have impaired immune function because of oncological treatment. Many patients suffer from glucocorticoid withdrawal syndrome, despite adequate replacement therapy, and it can take ≥ 1 year to gain control over these symptoms.6 This frequently leads to high dosage of glucocorticoids. The Endocrine Society guidelines recommend glucocorticoid replacement with hydrocortisone, 10–12 mg m-2 day-1 in divided doses.6 If we assume that most of our patients have body surface area around 2 m2 or less, the daily hydrocortisone dose should not exceed 25 mg. However, 1 year after BA, only one patient received 25 mg of hydrocortisone daily, with the majority receiving 30 mg or more. One-third of the patients had residual arterial hypertension and diabetes 3 months after BA, probably partially depending on too high a dose of glucocorticoids. There was a higher complication rate in our cohort compared to other studies19, 25, 26 and five patients needed conversion from an endoscopic approach to open surgery. In particular, the outcome of BA in ECS has not previously been systematically evaluated27 because most of the reports include patients with various aetiologies of CS.19, 22, 23, 28, 29 In a systematic review of the literature published between 1980 and 2012 on BA in CS, Reincke et al23 identified 37 studies and ECS was present in 13% of the patients. There are only few papers focused on BA in ECS solely21, 25, 26, 30, 31 and only one has a cohort with > 50 patients (n = 54).26 Patients with ECS have almost always a more aggressive course and more severe metabolic disturbance than patients with other types of Cushing’s syndrome, which probably leads to higher risk for postoperative complications. Furthermore, multiple liver metastases, fibrotic processes in the abdomen as a result of previous surgery or large primary tumour in pancreas could be some of the factors influencing surgical outcome in ECS. The present study has several limitations. First, all data were collected retrospectively from patient records and not all the preferred parameters were available for all patients. Second, even if our cohort is one of the largest regarding studies on BA in ECS, the number of patients is too low for reliable statistical analysis. Finally, our study covered more than three decades, BAs were performed at different clinics and by different surgeons. Therefore, the data should be interpreted carefully. In conclusion, the present study is one of few reports focusing on BA in specifically NEN patients with ECS and includes one of the largest patient cohorts analysed in the field. PRA-2S can be considered as method of choice in ECS compared to other BA approaches. The aim is to avoid administrating too high a hydrocortisone replacement dosage postoperatively because this can worsen the metabolic disturbance. As a result of the rarity of the condition, multicentre studies are needed with large, prospective cohorts and standardised inclusion criteria, aiming to further improve our knowledge about the management of ECS. ACKNOWLEDGEMENTS This study was funded by the Swedish Cancer Society (grant number CAN 18 0576), the Lions Foundation for Cancer Research at Uppsala University Hospital, Selanders Foundation and Söderbergs foundation at Uppsala University. CONFLICT OF INTERESTS The authors declare that they have no conflicts of interest. AUTHOR CONTRIBUTIONS Ieva Lase: Conceptualisation; Data curation; Formal analysis; Investigation; Methodology; Visualisation; Writing – original draft; Writing – review & editing. Malin Grönberg: Formal analysis; Supervision; Visualisation; Writing – review & editing. Olov Norlén: Conceptualisation; Writing – review & editing. Peter Stålberg: Conceptualisation; Writing – review & editing. Staffan Welin: Conceptualisation; Supervision; Writing – review & editing. Eva Tiensuu Janson: Conceptualisation; Funding acquisition; Methodology; Supervision; Writing – review & editing. ETHICAL APPROVAL The need for informed consent was waived by the local ethics committee. All procedures performed in this study were in accordance with the ethical standards of the institutional and/or national research committee and with the 1964 Helsinki declaration and its later amendments or comparable ethical standards. The study was approved by the local ethics committee, Regionala etikprövningsnämnden (EPN), in Uppsala, Sweden. PEER REVIEW The peer review history for this article is available at https://publons.com/publon/10.1111/jne.13030. The entire article, PDF, supporting tables and more can be found at https://onlinelibrary.wiley.com/doi/full/10.1111/jne.13030
  5. This article was originally published here J Clin Endocrinol Metab. 2021 Sep 3:dgab659. doi: 10.1210/clinem/dgab659. Online ahead of print. ABSTRACT CONTEXT: Confirming a diagnosis of Cushing’s disease (CD) remains challenging yet is critically important before recommending transsphenoidal surgery for adenoma resection. OBJECTIVE: To describe predictive performance of preoperative biochemical and imaging data relative to post-operative remission and clinical characteristics in patients with presumed CD. DESIGN, SETTING, PATIENTS, INTERVENTIONS: Patients (n=105; 86% female) who underwent surgery from 2007-2020 were classified into 3 groups: Group A (n=84) pathology-proven ACTH adenoma; Group B (n=6) pathology-unproven but with postoperative hypocortisolemia consistent with CD, and Group C (n=15) pathology-unproven, without postoperative hypocortisolemia. Group A+B were combined as Confirmed CD and Group C as Unconfirmed CD. MAIN OUTCOMES: Group A+B was compared to Group C regarding predictive performance of preoperative 24-hour urinary free cortisol (UFC), late night salivary cortisol (LNSC), 1mg dexamethasone suppression test (DST), plasma ACTH, and pituitary MRI. RESULTS: All groups had a similar clinical phenotype. Compared to Group C, Group A+B had higher mean UFC (p<0.001), LNSC(p=0.003), DST(p=0.06), ACTH(p=0.03) and larger MRI-defined lesions (p<0.001). The highest accuracy thresholds were: UFC 72 µg/24hrs; LNSC 0.122 µg/dl, DST 2.70 µg/dl, and ACTH 39.1 pg/ml. Early (3-month) biochemical remission was achieved in 76/105 (72%) patients: 76/90(84%) and 0/15(0%) of Group A+B versus Group C, respectively, p<0.0001. In Group A+B non-remission was strongly associated with adenoma cavernous sinus invasion. CONCLUSIONS: Use of strict biochemical thresholds may help avoid offering transsphenoidal surgery to presumed CD patients with equivocal data and improve surgical remission rates. Patients with Cushingoid phenotype but equivocal biochemical data warrant additional rigorous testing. PMID:34478542 | DOI:10.1210/clinem/dgab659
  6. As of September 1, 2021, the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), which is the virus responsible for the coronavirus disease 2019 (COVID-19), has infected over 219 million and caused the deaths of over 4.5 million worldwide. Although COVID-19 has been traditionally associated with its ability to cause varied symptoms resembling acute respiratory distress syndrome (ARDS), emerging scientific evidence has demonstrated that SARS-CoV-2 causes much more damage beyond its effects on the upper respiratory tract. To this end, in a recent study published in Reviews in Endocrine and Metabolic Disorders, the researchers discuss the extra-pulmonary manifestations of COVID-19. Risk factors for severe COVID-19 It is now a well-known fact that the likelihood of people falling severely ill or dying from COVID-19 is increases if these individuals are obese, or have certain comorbidities like diabetes mellitus (DM), vitamin D deficiency, and vertebral fractures (VFs). Any abnormality in the pituitary gland may lead to metabolic disorders, impaired immunity, and a host of other conditions that also make the body susceptible to infections. Since such conditions are common in patients with COVID-19 as well, it has been hypothesized that there might be a relationship between COVID-19 and pituitary gland disorders. On the other hand, researchers have also observed that COVID-19 causes increased severity of pituitary-related disorders, and even pituitary apoplexy, which is a condition defined as internal bleeding or impaired blood supply in the pituitary gland. A group of Italian researchers has reviewed this bidirectional relationship between the pituitary gland abnormalities and COVID-19 in their study recently published in Reviews in Endocrine and Metabolic Disorders. The link between pituitary gland abnormalities and COVID19 The pituitary gland releases hormones that regulate and control some of the most important functions of the body like growth, metabolism, energy levels, bone health, mood swings, vision, reproduction, and immunity, to name a few. The inability of the pituitary gland to release one or more of these hormones is known as ‘hypopituitarism.’ Factors responsible for hypopituitarism include traumatic brain injury, pituitary adenomas (tumors), genetic mutations, as well as infiltrative and infectious diseases. Hypopituitarism can lead to severe cases of DM, growth hormone deficiency (GHD), abnormal lipid profile, obesity, arterial hypertension, and immune dysfunctions. Interestingly, similar consequences of COVID-19 have also been reported. SARS-CoV-2 infects the human body by binding to a special class of receptors known as the angiotensin-converting enzyme 2 (ACE2) receptors. These receptors are located in the endothelial linings of most organs like the brain, heart, lungs, kidneys, intestine, liver, and pancreas, among others. The main function of the ACE2 receptors is binding to specific target molecules to maintain the renin-angiotensin system that is crucial for regulating dilation of blood vessels, as well as maintain blood glucose levels, the immune system, and homeostasis. Therefore, SARS-CoV-2 binding to these ACE2 receptors facilitates the entry of this virus into all the organs that have these receptors, thus leading to the ability of SARS-CoV-2 to cause widespread damage in the body. Upon entry into the pancreas, for example, SARS-CoV-2 can inhibit ß-cells function, which worsens hyperglycemia and increases the risk for acute diabetic complications. Similarly, the presence of ACE2 receptors in brain tissues may cause invasion into the pituitary gland and lead to pituitary apoplexy. The entry of SARS-CoV-2 into the brain can also cause neurological damage in infected patients, which may account for some of the common neurological complaints of COVID-19 including headaches, confusion, dysgeusia, anosmia, nausea, and vomiting. Study findings Hypopituitarism leading to metabolic syndrome has been scientifically linked to higher mortality in COVID-19 patients. In fact, the presence of a single metabolic syndrome component has been observed to double the risk of death by COVID-19. This risk was even higher among patients with DM and hypertension. There was also an increased incidence of VFs in COVID-19 patients with hypopituitarism. Hence, patients with DM, obesity, hypertension, and chronic inflammatory disease, are all at an increased risk of poor outcomes and death in COVID-19. Arterial hypertension is a common finding in adults with GHD, which is another consequence of hypopituitarism. Hypopituitarism also causes adrenal insufficiency, a condition that is primarily managed with glucocorticoids and hormonal replacement therapies. Notably, patients with COVID-19 are often treated for prolonged periods with high-dose exogenous glucocorticoids, which is a class of steroids that suppress some activities of the immune system. This treatment approach may result in suppression of the hypothalamic-pituitary–adrenal axis that can lead to adrenal insufficiency. Hypogonadism is another aspect of pituitary insufficiency that predisposes patients, especially males, to COVID-19. Evidence shows that males with hypogonadism were more frequently affected by metabolic syndrome. Pituitary apoplexy, albeit rare, has also been linked to COVID-19, especially in patients with pituitary adenomas and those who are being treated with anticoagulant therapy. This may be because the pituitary gland becomes overstimulated during an infectious disease, which may increase pituitary blood demand and lead to sudden infarction precipitating acute apoplexy. This phenomenon has also been shown in patients suffering from infectious diseases that cause hemorrhagic fevers. Taken together, pituitary apoplexy complicates treatment and management procedures in COVID-19 patients. Despite the use of steroids in COVID-19 patients, there have been no contraindications for vaccination in such patients. However, those on extensive hormonal therapies need constant monitoring for best results. Implications The pituitary gland acts like a double-edged sword for COVID-19. On one end, hypopituitarism predisposes patients to metabolic disorders like DM, obesity, and VFs, all of which are known risk factors for COVID-19. On the other hand, COVID-19 may cause direct or indirect damage to the pituitary glands by entering the brain and inducing unfavorable vascular events – though evidence on this remains lesser in comparison to that of hypopituitarism. Ultimately, the researchers of the current study conclude that managing patients with hormonal insufficiencies optimally with steroids is likely to improve outcomes in severe COVID-19. Journal reference: Frara, S., Loli, P., Allora, A., et al. (2021). COVID-19 and hypopituitarism. Reviews in Endocrine and Metabolic Disorders. doi:10.1007/s11154-021-09672-y. https://rd.springer.com/article/10.1007/s11154-021-09672-y#citeas. From https://www.news-medical.net/news/20210905/Hypopituitarism-and-COVID-19-e28093-exploring-a-possible-bidirectional-relationship.aspx
  7. Christina Tatsi, Maria E. Bompou, Chelsi Flippo, Meg Keil, Prashant Chittiboina, Constantine A. Stratakis First published: 25 August 2021 https://doi.org/10.1111/cen.14560 Abstract Objective Diagnostic workup of Cushing disease (CD) involves imaging evaluation of the pituitary gland, but in many patients no tumour is visualised. The aim of this study is to describe the association of magnetic resonance imaging (MRI) findings with the postoperative course of paediatric and adolescent patients with CD. Patients Patients with a diagnosis of CD at less than 21 years of age with MRI evaluation of the pituitary before first transsphenoidal surgery were included. Measurements Clinical, imaging and biochemical data were analysed. Results One hundred and eighty-six patients with paediatric or adolescent-onset CD were included in the study. Of all patients, 127 (68.3%) had MRI findings consistent with pituitary adenoma, while the remaining had negative or inconclusive MRI. Patients with negative MRI were younger in age and had lower morning cortisol and adrenocorticotropin levels. Of 181 patients with data on postoperative course, patients with negative MRI had higher odds of not achieving remission after the first surgery (odds ratio = 2.6, 95% confidence intervals [CIs] = 1.1–6.0) compared to those with positive MRI. In patients with remission after first transsphenoidal surgery, long-term recurrence risk was not associated with the detection of a pituitary adenoma in the preoperative MRI (hazard risk = 2.1, 95% CI = 0.7–5.8). Conclusions Up to one-third of paediatric and adolescent patients with CD do not have a pituitary tumour visualised in MRI. A negative MRI is associated with higher odds of nonremission after surgery; however, if remission is achieved, long-term risk for recurrence is not associated with the preoperative MRI findings. Full text at https://onlinelibrary.wiley.com/doi/full/10.1111/cen.14560
  8. This article was originally published here J Clin Endocrinol Metab. 2021 Jul 29:dgab557. doi: 10.1210/clinem/dgab557. Online ahead of print. ABSTRACT CONTEXT: Coronavirus disease 2019 (COVID-19) is a proinflammatory and prothrombotic condition, but its impact on adrenal function has not been adequately evaluated. CASE REPORT: A 46-year-old woman presented with abdominal pain, hypotension, and skin hyperpigmentation after COVID-19 infection. The patient had hyponatremia, serum cortisol <1.0 µg/dL, adrenocorticotropin (ACTH) of 807 pg/mL, and aldosterone ❤️ ng/dL. Computed tomography (CT) findings of adrenal enlargement with no parenchymal and minimal peripheral capsular enhancement after contrast were consistent with bilateral adrenal infarction. The patient had autoimmune hepatitis and positive antiphospholipid antibodies, but no previous thrombotic events. The patient was treated with intravenous hydrocortisone, followed by oral hydrocortisone and fludrocortisone. DISCUSSION: We identified 9 articles, including case reports, of new-onset adrenal insufficiency and/or adrenal hemorrhage/infarction on CT in COVID-19. Adrenal insufficiency was hormonally diagnosed in 5 cases, but ACTH levels were measured in only 3 cases (high in 1 case and normal/low in other 2 cases). Bilateral adrenal nonhemorrhagic or hemorrhagic infarction was identified in 5 reports (2 had adrenal insufficiency, 2 had normal cortisol levels, and 1 case had no data). Interestingly, the only case with well-characterized new-onset acute primary adrenal insufficiency after COVID-19 had a previous diagnosis of antiphospholipid syndrome. In our case, antiphospholipid syndrome diagnosis was established only after the adrenal infarction triggered by COVID-19. CONCLUSION: Our findings support the association between bilateral adrenal infarction and antiphospholipid syndrome triggered by COVID-19. Therefore, patients with positive antiphospholipid antibodies should be closely monitored for symptoms or signs of acute adrenal insufficiency during COVID-19. PMID:34463766 | DOI:10.1210/clinem/dgab557
  9. Dr. Friedman is getting a lot of emails on booster shots versus third shots. Third shots are for immuno-compromised patients that the FDA is recommending for a small group of patients The FDA also has the intention to soon make booster doses widely available to all healthy individuals. I am writing to clarify the difference between booster shots and third doses. Third Doses for Immuno-Compromised Patients The purpose of a third dose of mRNA vaccine is to give immuno-compromised patients the same level of protection that two doses provide someone who has a normal immune system. It is recommended that the following people get a third dose Been receiving cancer treatment for tumors or cancers of the blood Received an organ transplant and are taking medicine to suppress the immune system Received a stem cell transplant within the last two years or are taking medicine to suppress the immune system Been diagnosed with moderate or severe immunodeficiency conditions (such as DiGeorge syndrome, Wiskott-Aldrich syndrome) An advanced or untreated HIV infection Been under active treatment with high-dose corticosteroids (> 20 mg of prednisone or 100 mg of hydrocortisone) or other drugs that may suppress immune response Dr. Friedman thinks it is unlikely that any of his patients have these conditions. Patients with Cushing’s syndrome, Addison’s, diabetes or thyroid disorders do not qualify. In contrast, a Booster Dose is for Patients With Healthy Immune Systems A booster dose—which is different from a third dose for immuno-compromised patients—is for healthy patients and is meant to enhance immunity and may protect against new variants of the virus. The Biden administration has announced that it intends to make booster doses available for people with healthy immune systems in September 2021, after they are authorized or approved by the FDA. This has not happened yet, but when it happens, Dr. Friedman would encourage his patients to get it. Dr. Friedman is expecting a booster shot against the Delta variant to be released in the fall of 2021 and would recommend that for his patients. Dr. Friedman wishes everyone to stay healthy.
  10. Cushing disease is caused by tumour in the pituitary gland which leads to excessive secretion of a hormone called adrenocorticotrophic (ACTH), which in turn leads to increasing levels of cortisol in the body. Cortisol is a steroid hormone released by the adrenal glands and helps the body to deal with injury or infection. Increasing levels of cortisol increases the blood sugar and can even cause diabetes mellitus. However the disease is also caused due to excess production of hypothalamus corticotropin releasing hormone (CRH) which stimulates the synthesis of cortisol by the adrenal glands. The condition is named after Harvey Cushing, the doctor who first identified the disease in 1912. Cushing disease results in Cushing syndrome. Cushing syndrome is a group of signs and symptoms developed due to prolonged exposure to cortisol. Signs and symptoms of Cushing syndrome includes hypertension, abdominal obesity, muscle weakness, headache, fragile skin, acne, thin arms and legs, red stretch marks on stomach, fluid retention or swelling, excess body and facial hair, weight gain, acne, buffalo hump, tiredness, fatigue, brittle bones, low back pain, moon shaped face etc. Symptoms vary from individual to individual depending upon the disease duration, age and gender of the patient. Get Sample Copy of this Report @ https://www.persistencemarketresearch.com/samples/14155 Disease diagnosis is done by measuring levels of cortisol in patient’s urine, saliva or blood. For confirming the diagnosis, a blood test for ACTH is performed. The first-line treatment of the disease is through surgical resection of ACTH-secreting pituitary adenoma, however disease management is also done through medications, Cushing disease treatment market comprises of the drugs designed for lowering the level of cortisol in the body. Thus patients suffering from Cushing disease are prescribed medications such as ketoconazole, mitotane, aminoglutethimide metyrapone, mifepristone, etomidate and pasireotide. Cushing’s disease treatment market revenue is growing with a stable growth rate, this is attributed to increasing number of pipeline drugs. Also increasing interest of pharmaceutical companies to develop Cushing disease drugs is a major factor contributing to the revenue growth of Cushing disease treatment market over the forecast period. Current and emerging players’ focuses on physician education and awareness regarding availability of different drugs for curing Cushing disease, thus increasing the referral speeds, time to diagnosis and volume of diagnosed Cushing disease individuals. Growing healthcare expenditure and increasing awareness regarding Cushing syndrome aids in the revenue growth of Cushing’s disease treatment market. Increasing number of new product launches also drives the market for Cushing’s disease Treatment devices. However availability of alternative therapies for curing Cushing syndrome is expected to hamper the growth of the Cushing’s disease treatment market over the forecast period. For entire list of market players, request for Table of content here @ https://www.persistencemarketresearch.com/toc/14155 The Cushing’s disease Treatment market is segment based on the product type, technology type and end user Cushing’s disease Treatment market is segmented into following types: By Drug Type Ketoconazole Mitotane Aminoglutethimide Metyrapone Mifepristone Etomidate Pasireotide By End User Hospital Pharmacies Retail Pharmacies Drug Stores Clinics e-Commerce/Online Pharmacies Cushing’s disease treatment market revenue is expected to grow at a good growth rate, over the forecast period. The market is anticipated to perform well in the near future due to increasing awareness regarding the condition. Also the market is anticipated to grow with a fastest CAGR over the forecast period, attributed to increasing investment in R&D and increasing number of new product launches which is estimated to drive the revenue growth of Cushing’s disease treatment market over the forecast period. Depending on geographic region, the Cushing’s disease treatment market is segmented into five key regions: North America, Latin America, Europe, Asia Pacific (APAC) and Middle East & Africa (MEA). North America is occupying the largest regional market share in the global Cushing’s disease treatment market owing to the presence of more number of market players, high awareness levels regarding Cushing syndrome. Healthcare expenditure and relatively larger number of R&D exercises pertaining to drug manufacturing and marketing activities in the region. Also Europe is expected to perform well in the near future due to increasing prevalence of the condition in the region. Asia Pacific is expected to grow at the fastest CAGR because of increase in the number of people showing the symptoms of Cushing syndrome, thus boosting the market growth of Cushing’s disease treatment market throughout the forecast period. Some players of Cushing’s disease Treatment market includes CORCEPT THERAPEUTICS, HRA Pharma, Strongbridge Biopharma plc, Novartis AG, etc. However there are numerous companies producing branded generics for Cushing disease. The companies in Cushing’s disease treatment market are increasingly engaged in strategic partnerships, collaborations and promotional activities to capture a greater pie of market share. The research report presents a comprehensive assessment of the market and contains thoughtful insights, facts, historical data, and statistically supported and industry-validated market data. It also contains projections using a suitable set of assumptions and methodologies. The research report provides analysis and information according to categories such as market segments, geographies, types, technology and applications.
  11. 1) Visit RareVoiceAwards.org 2) Review the 2021 RareVoice categories 3) Nominate an advocate who gave rare disease patients a voice on Capitol Hill and in state government in 2020 and 2021. 4) Submit! The RareVoice Awards recipients are chosen by a committee from nominations received from the rare disease community. Nominations close August 27th, 2021 Federal Advocacy – Congressional Staff Honors congressional staffers who have worked to create and enact policies for the rare disease community Federal Advocacy – Patient/Organization Honors advocates or organizations that have worked to create and pass federal legislation State Advocacy – State Legislator Honors state legislators who have worked to create and enact policies for the rare disease community State Advocacy – Patient/Organization Honors advocates or organizations that have worked to create and pass state legislation Federal or State Advocacy by a Teenager Honors teen advocates that have advocated for state or federal legislation Diversity Empowerment - Patient/Organization Honors advocates or organizations that empowered diverse voices in advocacy Artist-to-Advocate Honors individuals who have utilized their artwork to advocate for federal or state legislation For information about sponsorship, please contact Elissa Taylor, etaylor@everylifefoundation.org EveryLife Foundation For Rare Diseases 1012 14th Street, NW, Suite 500 | Washington, District of Columbia 20005 202-697-7273 | info@everylifefoundation.org
  12. until
    Each year, Global Genes convenes one of the world’s largest gatherings of rare disease patients, caregivers, advocates, healthcare professionals, researchers, partners and allies. Join us for a variety of interactive and educational events, meet-ups, workshops and networking opportunities. Here you’ll have the opportunity to connect and engage with others in the rare disease community, while experiencing the sense of community and belonging we’ve missed so much. Gain insights about the latest in rare disease innovations, best practices for advocating on an individual and organizational level, and actionable strategies you can implement immediately to accelerate change. The 2021 RARE Patient Advocacy Summit is a virtual event, happening Monday, September 27, 2021 through Wednesday, September 29, 2021. 2021 RARE Patient Advocacy Summit virtual registration is now open! Register here: https://web.cvent.com/event/289bd182-abf4-49b0-90be-c2320566c276/regProcessStep1 Speakers: https://globalgenes.org/summit-featured-speakers/ Interested in becoming a speaker? Fill out the speaker inquiry form here.
  13. SAN DIEGO, CA, USA I August 10, 2021 I Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, today announced positive preliminary findings from the single ascending dose (SAD) portion of a first-in-human Phase 1 clinical study with CRN04894 demonstrating pharmacologic proof-of-concept for this first-in-class, investigational, oral, nonpeptide adrenocorticotropic hormone (ACTH) antagonist that is being developed for the treatment of conditions of ACTH excess, including Cushing’s disease and congenital adrenal hyperplasia. “ACTH is the central hormone of the endocrine stress response. Even though we’ve known about its clinical significance for more than 100 years, there has never been an ACTH antagonist available to intervene in diseases of excess stress hormones. This is an important milestone for the field of endocrinology and for our company,” said Scott Struthers, Ph.D., founder and chief executive officer of Crinetics. “I am extremely proud of our team that conceived, discovered and developed CRN04894 this far. This is the second molecule to emerge from our in-house discovery efforts and demonstrate pharmacologic proof of concept. I am very excited to see what it can do in upcoming clinical studies.” The 39 healthy volunteers who enrolled in the SAD cohorts were administered oral doses of CRN04894 (10 mg to 80 mg, or placebo) two hours prior to a challenge with synthetic ACTH. Analyses of basal cortisol levels (before ACTH challenge) showed that CRN04894 produced a rapid and dose-dependent reduction of cortisol by 25-56%. After challenge with a supra-pathophysiologic dose of ACTH (250 mcg), CRN04894 suppressed cortisol (as measured by AUC) up to 41%. After challenge with a disease-relevant dose of ACTH (1 mcg), CRN04894 showed a clinically meaningful reduction in cortisol AUC of 48%. These reductions in cortisol suggest that CRN04894 is bound with high affinity to its target receptor on the adrenal gland and blocking the activity of ACTH. CRN04894 was well tolerated in the healthy volunteers who enrolled in these SAD cohorts and all adverse events were considered mild. “We are very encouraged by these single ascending dose data which clearly demonstrate proof of ACTH antagonism with CRN04894 exposure in healthy volunteers,” stated Alan Krasner, M.D., chief medical officer of Crinetics. “We look forward to completing this study and assessing results from the multiple ascending dose cohorts. As a clinical endocrinologist, I recognize the pioneering nature of this work and eagerly look forward to further understanding the potential of CRN04894 for the treatment of diseases of ACTH excess.” Data Review Conference Call Crinetics will hold a conference call and live audio webcast today, August 10, 2021 at 4:30 p.m. Eastern Time to discuss the results of the CRN04894 SAD cohorts. To participate, please dial 800-772-3714 (domestic) or 212-271-4615 (international) and refer to conference ID 21996541. To access the webcast, please visit the Events page on the Crinetics website. The archived webcast will be available for 90 days. About the CRN04894-01 Phase 1 Study Crinetics is enrolling healthy volunteers in this double-blind, randomized, placebo-controlled Phase 1 study of CRN04894. Participants will be divided into multiple cohorts in the single ascending dose (SAD) and multiple ascending dose (MAD) phases of the study. In the SAD phase, safety and pharmacokinetics are assessed. In addition, pharmacodynamic responses are evaluated before and after challenges with injected synthetic ACTH to assess pharmacologic effects resulting from exposure to CRN04894. In the MAD phase, participants will be administered placebo or ascending doses of study drug daily for 10 days. Assessments of safety, pharmacokinetics and pharmacodynamics will also be performed after repeat dosing. About CRN04894 Adrenocorticotropic hormone (ACTH) is synthesized and secreted by the pituitary gland and binds to melanocortin type 2 receptor (MC2R), which is selectively expressed in the adrenal gland. This interaction of ACTH with MCR2 stimulates the adrenal production of cortisol, a stress hormone that is involved in the regulation of many systems. Cortisol is involved for example in the regulation of blood sugar levels, metabolism, inflammation, blood pressure, and memory formulation, and excess adrenal androgen production can result in hirsutism, menstrual dysfunction, infertility in men and women, acne, cardiometabolic comorbidities and insulin resistance. Diseases associated with excess of ACTH, therefore, can have significant impact on physical and mental health. Crinetics’ ACTH antagonist, CRN04894, has exhibited strong binding affinity for MC2R in preclinical models and demonstrated suppression of adrenally derived glucocorticoids and androgens that are under the control of ACTH, while maintaining mineralocorticoid production. About Cushing’s Disease and Congenital Adrenal Hyperplasia Cushing’s disease is a rare disease with a prevalence of approximately 10,000 patients in the United States. It is more common in women, between 30 and 50 years of age. Cushing’s disease often takes many years to diagnose and may well be under-diagnosed in the general population as many of its symptoms such as lethargy, depression, obesity, hypertension, hirsutism, and menstrual irregularity can be incorrectly attributed to other more common disorders. Congenital adrenal hyperplasia (CAH) encompasses a set of disorders that are caused by genetic mutations that result in impaired cortisol synthesis with a prevalence of approximately 27,000 patients in the United States. This lack of cortisol leads to a loss of feedback mechanisms and results in persistently high levels of ACTH, which in turn causes overstimulation of the adrenal cortex. The resulting adrenal hyperplasia and over-secretion of other steroids (particularly androgens) and steroid precursors can lead to a variety of effects from improper gonadal development to life-threatening adrenal crisis. About Crinetics Pharmaceuticals Crinetics Pharmaceuticals is a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors. The company’s lead product candidate, paltusotine, is an investigational, oral, selective nonpeptide somatostatin receptor type 2 agonist for the treatment of acromegaly, an orphan disease affecting more than 26,000 people in the United States. A Phase 3 program to evaluate safety and efficacy of paltusotine for the treatment of acromegaly is underway. Crinetics also plans to advance paltusotine into a Phase 2 trial for the treatment of carcinoid syndrome associated with neuroendocrine tumors. The company is also developing CRN04777, an investigational, oral, nonpeptide somatostatin receptor type 5 (SST5) agonist for congenital hyperinsulinism, as well as CRN04894, an investigational, oral, nonpeptide ACTH antagonist for the treatment of Cushing’s disease, congenital adrenal hyperplasia, and other diseases of excess ACTH. All of the company’s drug candidates are new chemical entities resulting from in-house drug discovery efforts and are wholly owned by the company. SOURCE: Crinetics Pharmaceuticals From https://pipelinereview.com/index.php/2021081178950/Small-Molecules/Crinetics-Pharmaceuticals-Oral-ACTH-Antagonist-CRN04894-Demonstrates-Pharmacologic-Proof-of-Concept-with-Dose-Dependent-Cortisol-Suppression-in-Single-Ascending-Dose-Port.html
  14. An international panel reached consensus for pre- and postoperative endocrine testing to manage adults undergoing transsphenoidal surgery, including measurement of prolactin and insulin-like growth factor I levels for all pituitary tumors. In adults and children, transsphenoidal surgery represents the cornerstone of management for most large or functioning sellar lesions with the exception of prolactinomas, Maria Fleseriu, MD, FACE, an Endocrine Today Editorial Board Member, professor of medicine and neurological surgery and director of the Pituitary Center at Oregon Health & Science University in Portland, and colleagues wrote in Pituitary. Endocrine evaluation and management are an essential part of perioperative care; however, the details of endocrine assessment and care are not universally agreed on. “Perioperative management of patients undergoing pituitary surgery is fascinating, as it involves many specialties — endocrinology, neurosurgery and ENT — and patients also get discharged very quickly in some countries, such as the United States,” Fleseriu told Healio. “At the start of the COVID-19 pandemic, the Physician Education Committee of the Pituitary Society, comprised of members from four continents, met to discuss a more streamlined process for workup before and after surgery for patients undergoing pituitary surgery. We have noticed big differences in management, but also some common themes, and decided to have a formal evaluation using a Delphi consensus and a much larger representation, with members from five continents.” Building consensus The task force behind the project, co-led by Nicholas A. Tritos, MD, DSc, associate professor of medicine at Harvard Medical School, and Pouneh K. Fazeli, MD, MPH, director of the neuroendocrinology unit and associate professor of medicine at University of Pittsburgh School of Medicine, created 35 questions and invited 55 pituitary endocrinologists to answer the questions in two Delphi rounds. Participants rated their extent of agreement with statements pertaining to perioperative endocrine evaluation and management, using a Likert-type scale. Strong consensus, defined as at least 80% of panelists rating their agreement as 6 to 7 on a scale from 1 to 7, was achieved for 24 of 35 items. Less strict agreement, defined as ratings of 5 to 7, was reached for 31 of 35 items. There were several significant findings, Fleseriu said. Despite uncertainty in previous guidelines, panelists reached consensus to measure serum IGF-I for all patients with pituitary tumors preoperatively to ensure proper diagnosis of growth hormone excess, Fleseriu said. “This is important because patients with GH-secreting adenomas do not always present with classic manifestations of acromegaly, require additional evaluation for comorbidities and postoperatively may benefit from further medical therapy or other adjuvant treatment,” Fleseriu said. Panelists also expressed agreement on preoperative administration of glucocorticoid and thyroid hormone replacement for patients with diagnosed deficiencies, as well as perioperative use of stress-dose glucocorticoid coverage for patients with known or suspected hypoadrenalism, but not for all patients undergoing transsphenoidal surgery. Panelists also agreed on postoperative monitoring of serum sodium and cortisol and the use of desmopressin on-demand, required to control hypernatremia and/or polyuria, for patients with central diabetes insipidus. “Agreement was achieved on postoperative monitoring of endocrine function, including morning serum cortisol in patients with Cushing’s disease, as well as serum IGF-I in patients with acromegaly,” Fleseriu said. More research needed Panelists did not reach consensus for a minority of items, representing areas where further research is needed, including measuring serum prolactin in dilution for all patients with large macroadenomas, Fleseriu said. “Prolactin immunoassays can be susceptible to the ‘hook effect’ artifact, which may lead to substantial underreporting of prolactin values in sera containing very high prolactin concentrations, thus having important implications for patient management,” Fleseriu said. “Newer automated immunoassay platforms are likely to detect the hook effect; however, this may not be the case in older assays, which are still in use in many countries or laboratories. Therefore, especially when surgery is performed at an institution where automated assays are available to detect hook effect, yet patient workup has been carried out at an outside laboratory, additional lab workup might be needed. We envision this scenario can occur more often with the widespread use of telemedicine and endocrine testing being carried out at a distant laboratory.” Additionally, there was a lack of consensus regarding preoperative testing for hypercortisolism in all patients with an apparently nonfunctioning pituitary adenoma. “This might reflect concern about false-positive results of endocrine testing in some individuals,” Fleseriu said. “On the other hand, published data suggest that some patients with Cushing’s disease may lack typical symptoms and signs and can present with an incidentally found sellar mass.” Panelists did not reach consensus on items concerning preoperative medical therapy for patients with acromegaly or Cushing’s disease, potentially reflecting differences in practice among international centers, the clinical heterogeneity of patient populations, and ongoing uncertainties regarding the benefits of preoperative medical therapy. “Single-center clinical experience suggests that preoperative medical therapy may be helpful in patients with Cushing’s disease and severe acute psychiatric illness or sepsis,” Fleseriu said. “Studies on acromegaly have very discordant results. “With this study — the largest international Delphi consensus on perioperative management of patients undergoing pituitary surgery — we identified key steps in protocols which are ready to be implemented in most centers, especially for preoperative evaluation, sodium abnormalities and glucocorticoids administration postop,” Fleseriu said. “We have also highlighted several areas where need for more research is needed to optimize patients’ outcomes.” For more information: Maria Fleseriu, MD, FACE, can be reached at fleseriu@ohsu.edu; Twitter: @MariaFleseriu. From https://www.healio.com/news/endocrinology/20210810/experts-offer-recommendations-for-management-of-pituitary-tumors
  15. Ahmed Saeed Mubarak Mohamed1, Ahmed Iqbal2, Suveera Prasad3, Nigel Hoggard4, Daniel Blackburn1 Correspondence to Dr Daniel Blackburn, Sheffield Teaching Hospitals NHS Foundation Trust Department of Clinical Neurology, Sheffield S10 2JF, UK; d.blackburn@sheffield.ac.uk Abstract Cushing’s disease is a rare endocrine condition in which a pituitary corticotroph adenoma drives excess adrenal cortisol production, and is one cause of endogenous Cushing’s syndrome. We present a young woman with 3 weeks of headaches and cognitive disturbance who subsequently developed florid psychosis requiring multiple admissions under neurology and psychiatry. Her clinical stigmata of hypercortisolism and biochemical abnormalities prompted an MR scan of the pituitary, which confirmed a pituitary microadenoma. Treatment with metyrapone and subsequent surgery led to complete recovery within 2 months. Cushing’s disease commonly causes neuropsychiatric symptoms and can present with psychosis. Diagnosing Cushing’s disease can be challenging, but with early diagnosis and treatment it has an excellent prognosis. http://dx.doi.org/10.1136/practneurol-2021-002974 Get the full text
  16. All of our country is very encouraged by the declining rates in both COVID-19 infections and death, due mostly to President Trump’s vaccine production and trial effort called Operation Warp Speed and President Biden’s vaccine distribution efforts. As of July 2021, The United States has administered 334,600,770 doses of COVID-19 vaccines, 184,132,768 people had received at least one dose while 159,266,536 people are fully vaccinated. The pandemic is by no means over, as people are still getting infected with COVID-19 with the emergence of the Delta Variant. In fact, recently cases, hospitalizations and deaths due to COVID-19 have gone up. In Los Angeles, the increased infection rate has led to indoor mask requirements. The main reason that COVID-19 has not been eliminated is because of vaccine hesitancy, which is often due to misinformation propagated on websites and social media. One of Dr. Friedman's patients gave him a link of an alternative doctor who gave multiple episodes of misinformation subtitled “Evidence suggests people who have received the COVID “vaccine” may have a reduced lifespan” about the COVID-19 vaccine that Dr. Friedman wants to address. Almost 30% of American say they will not get the vaccine, up from 20% a few months ago. Statistics are that people who are vaccinated have a 1:1,000,000 chance of dying from COVID, while people who are unvaccinated have a 1:500 chance of dying from COVID. I think most people would take the 1:1,000,000 risk. Dr. Friedman has always been a proponent of the COVID-19 vaccine because he is a scientist and bases his decisions on peer-reviewed literature and not social media posts. As we are getting to the stage where the COVID-19 pandemic could end if vaccination rates increase, he feels that it is even more important for people to get correct information about the COVID-19 vaccine. MYTH: People are dying at high rates from the COVID-19 vaccine and the rates of complications and deaths are underreported. FACT: The rates of complications and deaths from the vaccine are overreported. It is a fact that when 200 million people get a vaccine, some of them will get blood clots, some of them will have a heart attack, some of them will have strokes, some of them will have optic neuritis and some will have Guillain-Barré syndrome. These complications may not be due to the vaccine, but people remember that they got the vaccine recently. Anti-vaccine websites seem to play up on this and give false information that COVID-19 complications are underreported and fail to note that there is no control group, so we do not know how many people would have gotten blood clots, strokes, and heart attacks if they did not get the vaccine. For example, one anti-vaccine website highlighted a Tamil (Indian) actor Vivek, who died of a massive heart attack 5 days after getting the COVID-19 vaccine and tried to make a case that the vaccine caused that. Of course, the massive heart attack was due to years of buildup of cholesterol in his coronary arteries and had nothing to do with the COVID-19 vaccine. In fact, the complications attributed to the COVID-19 vaccine occur less frequently in those vaccinated than unvaccinated. The only complication that seems to possibly be more common in people who get vaccinated is blood clots, and the rate of that is still quite low. Overwhelmingly, the COVID-19 vaccine is effective and safe. MYTH: I had COVID-19 before. I don't need a vaccine. Natural immunity is better than a vaccine immunity. FACT: Most studies have shown that the COVID-19 vaccines are more effective, with longer-lasting immunity, than only having the COVID-19 infection. The immunity after natural infection varies and may be quite minimal in patients who had mild COVID-19 and likely declines within a couple of months of infection. In contrast, those who got the vaccine seem to have high levels of immunity even months after getting the vaccine. The vaccine also protects against the COVID-19 variants. If someone had one variant, it is unlikely that their natural immunity would protect them against other variants. MYTH: The COVID-19 vaccine leads to spike proteins circulating in your body for months after the vaccine. FACT: The mRNA from the vaccine, the spike protein that it generates, and all of the products of the COVID-19 vaccine are gone within hours, if not days, and do not hang around the body. MYTH: There is likely to be long-term effects, including infertility effects, of the COVID-19 vaccine. FACT: As the viral particles and proteins are gone within a couple hours to days and the vaccine only enters the cytoplasm and does not enter the DNA, it is very unlikely that there will be long-term effects. So far, the clinical trials of the COVID-19 vaccine have not resulted in any detrimental effects, and it has been a year since the trials started. Other vaccines have been used safely and do not give long-term side effects. There is no reason to think that this vaccine would give long-term side effects, and we have not seen any evidence of long-term side effects currently. Pregnant women who received COVID-19 vaccines have similar rates adverse pregnancy and neonatal outcomes (e.g., fetal loss, preterm birth, small size for gestational age, congenital anomalies, and neonatal death) as with pregnant women who did not receive vaccines. MYTH: People with autoimmune disease should not get the vaccine. FACT: Persons with autoimmune disease are likely more susceptible to COVID-19, and they should especially get the vaccine. People with preexisting conditions, including autoimmune diseases, have been shown to be give generally excellent immune responses to the vaccine, and it should especially be given to patients with Addison’s disease or Cushing's disease who may have higher rates of getting more severe COVID-19. In fact, the CDC as well Dr. Friedman recommends EVERYONE getting the vaccine, except 1) those under 12, 2) those who had an anaphylactic reaction to their first COVID-19 vaccine. Patients with AIDS, and those on immunosuppressive therapy for cancers, organ transplants and rheumatological conditions, may not be fully protected from vaccines and should be cautious (including wearing masks and social distancing), but still should get vaccinated. MYTH: Patients with autoimmune diseases, and other conditions do not mount an adequate immune response to the vaccine and may even should get a booster shot. FACT: The only patients that have been found not to have a good immune response to the vaccine is those with AIDS or on immunosuppressive drugs that are used in people with rheumatological diseases or transplants. With these exception, patients appear to mount a good immune response to the vaccine regardless of their preexisting condition and do not need a booster shot. MYTH: Why should I bother with the vaccine if it is going to require a booster shot? FACT: It is unclear whether booster shots will be required or not. Currently, the CDC and FDA do not recommend a booster shot, but Pfizer has petitioned the FDA to consider it and is starting more studies on whether a booster shot is effective. It is currently believed that the vaccine retains effectiveness for months to years after it is given. MYTH: We are almost at herd immunity now. Why bother getting a vaccine? FACT: We are not at herd immunity as people are still getting sick and dying from COVID-19. Dr. Friedman recently lost to COVID-19 his 43-year old patient with obesity and diabetes at MLK Outpatient Center. There are pockets in the United States with low vaccine rates, especially in the South. The vaccine is spreading among unvaccinated people, while the rate of spread among vaccinated people is quite low. Approximately 98% of those hospitalized with COVID-19 are unvaccinated. It is important from a public health viewpoint for all Americans to get vaccinated. MYTH: There is nothing to be concerned with about the variants. FACT: Especially the delta variant appears to be more contagious and aggressive than the other variants currently. The vaccines do appear to be effective against the delta variant but possibly a little less so. Variants multiply and can generate new variants only if they are infected into patients who are unvaccinated. To end the emergence of new variants, it is important for all Americans to get vaccinated. MYTH: I could just be careful, and I will not get the COVID-19 vaccine. FACT: Thousands of people who were careful and got COVID-19 and either died from it or became extremely sick. The best prevention against getting COVID-19 is to get vaccinated. MYTH: I am young. I do not have to worry about getting COVID. FACT: Many young people have gotten sick and died of COVID-19 and also, they are contagious and can spread COVID-19 if they are not vaccinated. Everyone, regardless of their age, as long as they are over 12, should get vaccinated. MYTH: If children under 12 are not vaccinated, the virus will still spread. FACT: The FDA and CDC do not recommend the vaccine for those under 12. They are very unlikely to get COVID-19 and are very unlikely to transmit it to others. They are the one group that does not need to get vaccinated. MYTH: COVID-19 vaccines are an experimental vaccine. FACT: While it is true that the FDA approved COVID-19 vaccines were granted emergency use authorization in December 2020 (Pfizer and Moderna) and Johnson and Johnson in February 2021. Both Pfizer and Moderna have petitioned the FDA for full approval, but by no means are these vaccines experimental. As mentioned, over 180 million Americans and many more worldwide have received the vaccine. This is more than any other FDA approved medication. Clinical trials are still ongoing and have enrolled thousands of people and Israel has monitored the effect of COVID-19 vaccines in 7 million Israelis. MYTH: The COVID-19 vaccine is a government plot to kill or injure people or a war against G-d. FACT: Yeah right If you want the pandemic to end, please get vaccinated and encourage your friends and colleagues to get vaccinated. For more information or to schedule an appointment with Dr. Friedman, go to goodhormonehealth.com
  17. Rachel Acree, Caitlin M Miller, Brent S Abel, Nicola M Neary, Karen Campbell, Lynnette K Nieman Journal of the Endocrine Society, Volume 5, Issue 8, August 2021, bvab109, https://doi.org/10.1210/jendso/bvab109 Abstract Context Cushing syndrome (CS) is associated with impaired health-related quality of life (HRQOL) even after surgical cure. Objective To characterize patient and provider perspectives on recovery from CS, drivers of decreased HRQOL during recovery, and ways to improve HRQOL. Design Cross-sectional observational survey. Participants Patients (n = 341) had undergone surgery for CS and were members of the Cushing’s Support and Research Foundation. Physicians (n = 54) were Pituitary Society physician members and academicians who treated patients with CS. Results Compared with patients, physicians underestimated the time to complete recovery after surgery (12 months vs 18 months, P = 0.0104). Time to recovery did not differ by CS etiology, but patients with adrenal etiologies of CS reported a longer duration of cortisol replacement medication compared with patients with Cushing disease (12 months vs 6 months, P = 0.0025). Physicians overestimated the benefits of work (26.9% vs 65.3%, P < 0.0001), exercise (40.9% vs 77.6%, P = 0.0001), and activities (44.8% vs 75.5%, P = 0.0016) as useful coping mechanisms in the postsurgical period. Most patients considered family/friends (83.4%) and rest (74.7%) to be helpful. All physicians endorsed educating patients on recovery, but 32.4% (95% CI, 27.3-38.0) of patients denied receiving sufficient information. Some patients did not feel prepared for the postsurgical experience (32.9%; 95% CI, 27.6-38.6) and considered physicians not familiar enough with CS (16.1%; 95% CI, 12.2-20.8). Conclusion Poor communication between physicians and CS patients may contribute to dissatisfaction with the postsurgical experience. Increased information on recovery, including helpful coping mechanisms, and improved provider-physician communication may improve HRQOL during recovery. Read the entire article in the enclosed PDF. bvab109.pdf
  18. With the goal of reducing false positives for adrenal insufficiency (AI), scientists are recommending a new, more precise diagnostic cutoff of 14-15 μg/dL of serum cortisol, rather than the current 18 μg/dL. The new data were published in the Journal of the Endocrine Society. Among the 110 patients evaluated in the retrospective analysis, new cortisol cutoffs after adrenocorticotropic hormone (ACTH) stimulation were identified when using several of the newer, more widely used diagnostic assays currently available, including Elecsys II (14.6 μg/dL), Access (14.8 μg/dL), and liquid chromatography-tandem mass spectrometry (LC-MS/MS) (14.5 μg/dL). Bradley Javorsky, MD, an endocrinologist and researcher at the Medical College of Wisconsin, served as the study's first author. He recently discussed the findings with MedPage Today. The exchange has been edited for length and clarity. What was the key knowledge gap your study was designed to address? Javorsky: It is safe to say that most clinicians, including many endocrinologists -- not to mention practice guidelines and clinical information resources -- still regard 18 μg/dL as the cutoff for making the biochemical diagnosis of AI after ACTH stimulation testing. However, this cutoff was derived from older polyclonal immunoassays that are no longer being used in many institutions. Newer, more specific monoclonal immunoassays and LC-MS/MS are being used instead. With these more specific assays, one might expect the cutoffs to be lower. What was your finding? Javorsky: After ACTH stimulation, the cutoff values for the newer, more specific cortisol assays were indeed lower at 14-15 μg/dL. Although there was excellent correlation between the new and older assays, the results from the new assays were 22-39% lower than those found by the older and less-specific Elecsys I assay, hence the lowered threshold. Did anything surprise you about the study results? Javorsky: Baseline cortisol had to be very low (approximately <2 μg/dL) in order to be predictive of subnormal cortisol values. This underscores the observation that ACTH stimulation testing is not perfectly sensitive. What are the clinical takeaways from these results? Javorsky: To avoid false-positive ACTH stimulation testing results -- and by extension avoid over-treating patients with glucocorticoids -- clinicians should be aware of the cortisol assay used in their institution and the new cortisol cutoff when evaluating patients for adrenal insufficiency. It should also be reinforced that careful interpretation in the context of clinical history is still essential to making the correct diagnosis. Discordant results among different assays underscore the importance of clinical judgment from an experienced physician when diagnosing AI. What are the takeaways? Javorsky: I think it is important that laboratories make the type of cortisol assay used in their institution easily accessible to clinicians and strongly consider posting the new cortisol cutoff after ACTH stimulation testing when reporting results. Read the study here and expert commentary on the clinical implications here. Disclosures Javorsky reported being a consultant for Clarus Therapeutics and a research investigator for Novartis Pharmaceuticals. Primary Source Journal of the Endocrine Society Source Reference: Javorsky BR, et al "New cutoffs for the biochemical diagnosis of adrenal insufficiency after ACTH stimulation using specific cortisol assays" J Endocrine Soc 2021; 5(4): bvab022. From https://www.medpagetoday.com/endocrine-society/adrenal-disorders/93188
  19. DEER PARK, Ill., June 15, 2021 (GLOBE NEWSWIRE) -- Eton Pharmaceuticals, Inc (Nasdaq: ETON), the U.S. marketer of ALKINDI SPRINKLE®, a treatment for adrenocortical insufficiency in pediatric patients, today announced that it has acquired U.S. and Canadian rights to Crossject’s ZENEO® hydrocortisone needleless autoinjector, which is under development as a rescue treatment for adrenal crisis. “The ZENEO autoinjector is a revolutionary delivery system, and this product is a terrific strategic fit with our current adrenal insufficiency business. Patients, advocacy groups, and physicians in the adrenal insufficiency community have repeatedly expressed to us the need for a hydrocortisone autoinjector, so we are excited to be partnering with Crossject to bring this product to patients in need,” said Sean Brynjelsen, CEO of Eton Pharmaceuticals. Patrick Alexandre, CEO of Crossject, added: ‘‘We are proud to announce a sound commercial agreement for ZENEO® Hydrocortisone in the US and Canada with an American leader in adrenal insufficiency. ETON has successfully established strong relations with the patient communities and medical specialists that are its core focus. ZENEO® Hydrocortisone answers a medical need. This strong partnership will contribute to saving lives by bringing to patients and their families a modern autoinjection possibility.’’ “We are delighted about Eton Pharmaceuticals' plans to partner with Crossject to bring this incredibly needed product to patients in the U.S.”, said Dina Matos, Executive Director of CARES Foundation, a leading North American advocacy foundation for patients with congenital adrenal hyperplasia, the most common presentation of adrenal insufficiencies in children. “The challenge for patients and caregivers facing an adrenal crisis is serious; an easy-to-use needleless autoinjector of hydrocortisone will be a game changer for our patients. We welcome this advancement.” ZENEO® is a proprietary needleless device developed and manufactured by Crossject. The pre-filled, single-use device propels medication through the skin in less than a tenth of a second. The device’s compact form factor, simple two-step administration, and needle-free technology make it an ideal delivery system for emergency medications that need to be administered in stressful situations by non-healthcare professionals. Crossject holds more than 400 global patents on the device, including 24 issued in the United States that extend as far as 2037, and has successfully completed bioequivalence and human factor studies with the ZENEO device using various medications. Crossject has developed a proprietary, room-temperature stable liquid formulation of hydrocortisone to be delivered via the ZENEO device. ZENEO hydrocortisone is expected to be the first and only hydrocortisone autoinjector available for patients that require a rescue dose of hydrocortisone. Currently, injectable hydrocortisone is only available in the United States in a lyophilized powder formulation that must be reconstituted and manually delivered via a traditional syringe. Eton expects to submit a New Drug Application for the product to the U.S. Food and Drug Administration in 2023 and plans to request Orphan Drug Designation. In the United States, it is estimated that approximately 100,000 patients currently suffer from adrenocortical insufficiency and are at risk for adrenal crisis. Under the terms of the agreement, Crossject will receive development and regulatory milestone payments from Eton of up to $5.0 million, commercial milestones of up to $6.0 million, and a 10% royalty on net sales of the product. Crossject will be responsible for the management and expense of development, clinical, and manufacturing activities. Eton will be responsible for all regulatory and commercial activities. About Adrenal Crisis Patients with adrenal insufficiency can go into adrenal crisis if their cortisol levels are too low. Adrenal crisis is typically caused by missed doses of maintenance hydrocortisone, trauma, surgery, illness, fever, or major psychological distress. Signs of adrenal crisis include hyperpigmentation, severe weakness, nausea, abdominal pain, and confusion. It is estimated that approximately 8% of adrenal insufficiency patients will report an adrenal crisis in any given year and more than 6% of cases result in death. About Crossject Crossject (ISIN: FR0011716265; Ticker: ALCJ; LEI: 969500W1VTFNL2D85A65) is developing and is soon to market a portfolio of drugs dedicated to emergency situations: epilepsy, overdose, allergic shock, severe migraine and asthma attack. The company’s portfolio currently contains eight products in advanced stages of development, including 7 emergency treatments, 5 of which are intended for life-threatening situations. Thanks to its patented needle-free self-injection system, Crossject aims to become the world leader in self-administered emergency drugs. The company has been listed on the Euronext Growth market in Paris since 2014, and benefits from Bpifrance funding. About Eton Pharmaceuticals Eton Pharmaceuticals, Inc. is an innovative pharmaceutical company focused on developing and commercializing treatments for rare diseases. The company currently owns or receives royalties from three FDA-approved products, including ALKINDI® SPRINKLE, Biorphen®, and Alaway Preservative Free®, and has six additional products that have been submitted to the FDA. Company Contact: David Krempa dkrempa@etonpharma.com 612-387-3740 From https://www.globenewswire.com/news-release/2021/06/15/2247745/0/en/Eton-Pharmaceuticals-Acquires-U-S-and-Canadian-Rights-to-ZENEO-Hydrocortisone-Autoinjector.html
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  21. Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients, families, healthcare professionals, and other supporters for learning, sharing, and connecting. Due to the ongoing COVID-19 pandemic, the general sessions, breakout workshops, and networking will again be virtual. The sessions, which will offer perspectives from patients, caregivers, and the medical community, will air live and be recorded for later viewing. Throughout the forum, participants will be able to visit the exhibit hall and have peer meetings with other attendees. Also this year, the Rare Impact Awards will return as part of the program. That presentation, on June 28, honors individuals, organizations, and industry innovators for exceptional work benefitting the rare disease community. “The health and well-being of people living with rare diseases, their loved ones and those working to improve their lives continues to remain a top priority for all of us here at NORD,” the organization stated in its forum announcement. “The COVID-19 pandemic brought us new ways to engage with our community and our 2020 virtual program was the most successful forum to date! In 2021 we will continue to work hard to keep our community healthy and safe while engaging in this impactful program,” NORD said. Registration for the “patient-centric” event is $39 for patients, caregivers, students, and NORD patient organization representatives. The cost is $75 for professional advocates, people from academia, physicians, and government representatives, and $500 for NORD corporate council members. For pharmaceutical, insurance, or other representatives, registration is $650. As for the agenda, the opening discussion will be on “The Patient-Professional Partnership” and will include three stories on the close bond between patients and their care professionals. Breakout sessions for Saturday, June 26 will include “Coping with Grief and Anticipatory Grief,” “Shared Decision-Making with Your Care Team,” and “Working While Rare” as first offerings, followed by “Getting Involved in Clinical Research: Finding and Preparing for Clinical Trials,” “Navigating Insurance, Social Security Disability and Patient Assistance Programs,” and “The ABCs of Advocating for Your Child’s Education” in the second group of workshops. Those will be followed by a plenary discussion on the topic “Building Resilience in a Time of Unknowns.” The speakers will explore how patients coped while waiting for a diagnosis, how they are faring while waiting for new treatments, and how they have kept it together during the pandemic. June 27 will start with an opening plenary discussion titled “The Rare Sibling Experience.” Here, three siblings of rare disease patients will share their experiences, including how they became advocates. Breakout sessions on this day will include “Fighting Back and Fighting Forward Through Advocacy,” “Palliative Care: Debunking the Myths,” “Rare in the Family: Navigating the Roles of Patient, Parent, and Caregiver” in the first set of discussion groups. Later offerings that Sunday will include “Aging with a Rare Condition,” “Finding Your Community and Building Your Support Network,” and “The Intersection of Race, Ethnicity, and Equity with Diagnosis and Treatment Access.” The closing plenary discussion, titled “Rare Breakthroughs Now and on the Horizon,” will cover the latest advances in the diagnosis, treatment, and care of rare diseases. Early this year, NORD put out a call out for individuals who were willing to share their real-life experiences with rare diseases at the conference. In all, including physicians, nurses, and other healthcare professionals, the conference will feature some 55 speakers. Access to the virtual program will be provided via email the week of the event.
  22. Wow, that's tough but thank you for being a concerned mom. Does she know your family history? Maybe talking about that would be a way in to discus symptoms and so on. Is she aware/concerned about any of the symptoms she's dealing with? If she doesn't live at home, perhaps she's already seeking medical attention but hasn't shared that with you to keep you from worrying. Please keep us posted on how this goes. Best of luck!
  23. Mayela, I'm so sorry you went through COVID but glad you're on the other side of it now. And a relapse doesn't sound like any fun Thanks for the update on The GRACE trial, though. Please keep us updated on your recovery from COVID and your relapse.
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