Pediatric Endocrine Society Provides Guidance for Growth Hormone Use in Pediatric Patients

Posted on June 3, 2017 by MaryO

HealthDay News—Use of growth hormone in children and adolescents should be considered carefully, with assessment of the risks and benefits necessary for each patient, according to guidelines published in the January issue of Hormone Research in Paediatrics.

Adda Grimberg, MD, from the Perelman School of Medicine at the University of Pennsylvania in Philadelphia, and colleagues updated guidelines on the use of growth hormone, focusing on idiopathic short stature (ISS), GH deficiency (GHD), and primary insulin-like growth factor-I (IGF-I) deficiency (PIGFD). The guidelines were written on behalf of the Pediatric Endocrine Society.

The researchers recommend use of growth hormone for children and adolescents with GHD. Prospective recipients of growth hormone treatment should receive guidance regarding potential adverse effects and should be monitored for these effects. Parents and clinicians should take a shared decision-making approach to treating patients with ISS, and assess the physical and physiological burdens for the child, while considering the risks and benefits of treatment.Follow-up assessment of benefit and psychosocial impact should be conducted at 12 months after initiation and dose optimization of GH. IGF-I therapy is recommended for patients with severe PIGFD. Diagnosis of PIGFD/GH insensitivity syndrome should be based on a combination of factors that fall into four stages.

Physicians with expertise in managing endocrine disorders in children should provide consultation for evaluation of GHD-ISS-PIGFD and manage treatment.

“The taskforce suggests that the recommendations be applied in clinical practice with consideration of the evolving literature and the risks and benefits to each individual patient,” the authors write. “In many instances, careful review highlights areas that need further research.”

Several authors disclosed financial ties to the pharmaceutical industry.

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Reference

Grimberg A, DiVall SA, Polychronakos C, et al; on behalf of the Drug and Therapeutics Committee of the Pediatric Endocrine Society. Guidelines for growth hormone and insulin-like growth factor-I treatment in children and adolescents: growth hormone deficiency, idiopathic short stature, and primary insulin-like growth factor-I deficiency. Horm Res Paediatr. 2016;86(6):361-397. doi: 10.1159/000452150

 From http://www.endocrinologyadvisor.com/adrenal/growth-hormone-use-in-pediatric-patients/article/634909/

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Cushing’s Testing at NIH

Posted on May 21, 2017 by MaryO
Rank Status Study
1 Recruiting Study to Evaluate CORT125134 in Patients With Cushing’s Syndrome

Condition: Cushing’s Syndrome
Intervention: Drug: CORT125134
2 Recruiting Cushing’s Disease Complications

Condition: Cushing’s Disease
Intervention: Other: Exams and questionnaires
3 Recruiting The Accuracy of Late Night Urinary Free Cortisol/Creatinine and Hair Cortisol in Cushing’s Syndrome Diagnosis

Condition: Cushing Syndrome
Intervention:
4 Recruiting Treatment for Endogenous Cushing’s Syndrome

Condition: Endogenous Cushing’s Syndrome
Intervention: Drug: COR-003
5 Recruiting Saliva Cortisol Measurement as a Screening Test for Suspicious Cushings Syndrome in Children.

Condition: Cushings Syndrome
Intervention: Other: Children refered to the obesity clinic
6 Recruiting Safety and Efficacy of LCI699 for the Treatment of Patients With Cushing’s Disease

Condition: Cushing’s Disease
Intervention: Drug: LCI699
7 Recruiting Treatment of Cushing’s Disease With R-roscovitine

Condition: Cushings Disease
Intervention: Drug: R-roscovitine
8 Recruiting A Study of ATR-101 for the Treatment of Endogenous Cushing’s Syndrome

Condition: Cushing Syndrome
Interventions: Drug: ATR-101;   Drug: Placebos
9 Recruiting Evaluation of 68Ga-DOTATATE PET/CT, Octreotide and F-DOPA PET Imaging in Patients With Ectopic Cushing Syndrome

Condition: Cushing Syndrome
Interventions: Drug: F-DOPA PET Scan;   Drug: Mifepristone;   Drug: Ga-DOTATATE;   Drug: Octreoscan;   Other: CT, MRI
10 Not yet recruiting Endocrine Cardiomyopathy in Cushing Syndrome: Response to Cyclic GMP PDE5 inhibitOrs

Condition: Cushing’s Syndrome Cardiomyopathy
Intervention: Drug: Tadalafil
11 Recruiting Long-term Beneficial Metabolic Effects of Adrenalectomy in Subclinical Cushing’s Syndrome of Adrenal Incidentaloma

Condition: Cushing Syndrome
Intervention: Procedure: surgery
12 Recruiting Long Term Safety and Efficacy of Pasireotide s.c. in Patients With Cushing’s Disease

Condition: Cushings Disease
Intervention: Drug: SOM230
13 Recruiting New Imaging Techniques in the Evaluation of Patients With Ectopic Cushing Syndrome

Condition: Cushing Syndrome
Interventions: Drug: Pentetreotide;   Drug: 18-F-fluorodeoxyglucose;   Drug: (18F)-L-3,4-dihydroxyophenylalanine (18F-DOPA)
14 Not yet recruiting Targeting Iatrogenic Cushing’s Syndrome With 11β-hydroxysteroid Dehydrogenase Type 1 Inhibition

Condition: Iatrogenic Cushing’s Disease
Interventions: Drug: AZD4017 and prednisolone;   Drug: Placebo Oral Tablet and prednisolone
15 Not yet recruiting Assessment of Persistent Cognitive Impairment After Cure of Cushing’s Disease

Condition: Cushing’s Disease
Intervention: Device: Virtual radial task in 3D
16 Recruiting Biomarker Expression in Patients With ACTH-Dependent Cushing’s Syndrome Before and After Surgery

Condition: Cushing’s Syndrome
Intervention:
17 Recruiting Efficacy and Safety Evaluation of Osilodrostat in Cushing’s Disease

Condition: Cushing’s Disease
Interventions: Drug: osilodrostat;   Drug: osilodrostat Placebo
18 Recruiting Effects of Metyrapone in Patients With Endogenous Cushing’s Syndrome

Condition: Cushing’s Syndrome
Intervention: Drug: metyrapone
19 Recruiting Adrenal Venous Sampling in Patients With Overt or Subclinical Cushings Syndrome, and Bilateral Adrenal Tumors

Condition: Cushing Syndrome
Intervention: Radiation: Adrenal venous sampling
20 Recruiting Glycemic Fluctuations in Newly Diagnosed Growth Hormone-Secreting Pituitary Adenoma and Cushing Syndrome Subjects

Condition: Pituitary Adenoma
Intervention: Device: continuous glucose monitoring
Rank Status Study
21 Recruiting Targeted Therapy With Gefitinib in Patients With USP8-mutated Cushing’s Disease

Conditions: Cushing’s Disease;   Corticotrophin Adenoma
Intervention: Drug: Gefitinib
22 Recruiting Cardiac Steatosis in Cushing’s Syndrome

Conditions: Endocrine System Disease;   Cardiovascular Imaging
Intervention: Other: 1H magnetic resonance spectroscopy and CMRI
23 Recruiting Study of Management of Pasireotide-induced Hyperglycemia in Adult Patients With Cushing’s Disease or Acromegaly

Conditions: Cushing’s Disease;   Acromegaly
Interventions: Drug: Pasireotide s.c.;   Drug: Sitagliptin;   Drug: Liraglutide;   Drug: Insulin;   Drug: Pasireotide LAR;   Drug: Metformin
24 Recruiting Study of Efficacy and Safety of Osilodrostat in Cushing’s Syndrome

Conditions: Cushing’s Syndrome;   Ectopic Corticotropin Syndrome;   Adrenal Adenoma;   Adrenal Carcinoma;   AIMAH;   PPNAD
Intervention: Drug: Osilodrostat
25 Recruiting Effects of Hormone Stimulation on Brain Scans for Cushing s Disease

Condition: Pituitary Neoplasm
Intervention: Drug: Acthrel
26 Recruiting Does Serum-DXM Increase Diagnostic Accuracy of the Overnight DXM Suppression Test in the Work-up of Cushing’s Syndrome?

Conditions: Cushing’s Syndrome;   Adrenal Incidentalomas;   Alcoholism;   Obesity
Intervention:
27 Recruiting Adrenalectomy Versus Follow-up in Patients With Subclinical Cushings Syndrome

Condition: Adrenal Tumour With Mild Hypercortisolism
Intervention: Procedure: Adrenalectomy
28 Recruiting Study of Adrenalectomy Versus Observation for Subclinical Hypercortisolism

Conditions: Hypercortisolism;   Cushing Syndrome
Interventions: Procedure: Adrenalectomy;   Other: Observation
29 Not yet recruiting Dynamic Hormone Diagnostics in Endocrine Disease

Conditions: Adrenal Insufficiency;   Congenital Adrenal Hyperplasia;   Cushing Syndrome;   Growth Hormone Deficiency;   Acromegaly;   Primary Hyperaldosteronism
Intervention: Other: 27 hour subcutaneous fluid sampling
30 Recruiting An Investigation of Pituitary Tumors and Related Hypothalmic Disorders

Conditions: Abnormalities;   Craniopharyngioma;   Cushing’s Syndrome;   Endocrine Disease;   Pituitary Neoplasm
Intervention:
31 Recruiting Ga-68-DOTATOC -PET in the Management of Pituitary Tumours

Condition: Pituitary Tumours
Intervention: Procedure: Gallium-68 DOTATOC PET
32 Recruiting Efficacy of Mifepristone in Males With Type 2 Diabetes Mellitus

Conditions: Type 2 Diabetes Mellitus;   Insulin Resistance
Interventions: Drug: Mifepristone 600 mg daily;   Drug: Placebo
33 Recruiting Targeted Therapy With Lapatinib in Patients With Recurrent Pituitary Tumors Resistant to Standard Therapy

Conditions: Pituitary Adenomas;   Prolactinomas
Intervention: Drug: Lapatinib
34 Recruiting Mutations of Glucocorticoid Receptor in Bilateral Adrenal Hyperplasia

Condition: General Glucocorticoid Resistance
Intervention: Genetic: blood collection for mutation characterization
35 Recruiting Defining the Genetic Basis for the Development of Primary Pigmented Nodular Adrenocortical Disease (PPNAD) and the Carney Complex

Conditions: Cushing’s Syndrome;   Hereditary Neoplastic Syndrome;   Lentigo;   Neoplasm;   Testicular Neoplasm
Intervention:
36 Not yet recruiting Reduction by Pasireotide of the Effluent Volume in High-output Enterostomy in Patients Refractory to Usual Medical Treatment

Condition: Enterostomy
Interventions: Drug: Pasireotide;   Drug: Placebo
37 Recruiting Mifepristone for Breast Cancer Patients With Higher Levels of Progesterone Receptor Isoform A Than Isoform B.

Condition: Breast Cancer
Intervention: Drug: Mifepristone
38 Recruiting SOM230 Ectopic ACTH-producing Tumors

Condition: Ectopic ACTH Syndrome
Intervention: Drug: Pasireotide
39 Recruiting Decreasing Rates of Intraurethral Catheterization Postoperatively in Spine Surgery

Condition: Post-operative Urinary Retention
Interventions: Drug: Tamsulosin;   Drug: Placebo
40 Recruiting Adrenal Tumors – Pathogenesis and Therapy

Conditions: Adrenal Tumors;   Adrenocortical Carcinoma;   Cushing Syndrome;   Conn Syndrome;   Pheochromocytoma
Intervention:

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Rare Disease Day 2017

Posted on February 28, 2017 by MaryO

Today is #RareDiseaseDay 2017! Today, with events taking place in over 90 countries all around the world, we hope to raise more awareness than ever for rare diseases!

With the theme of research, and the slogan, ‘With research, the possibilities are limitless’, #RareDiseaseDay 2017 is an opportunity to call on all researchers, universities, students, companies, policymakers and clinicians to do more research and to make them aware of the importance of research for the rare disease community.

This year’s Rare Disease Day video, which has been viewed over a hundred thousand times and translated into over 30 languages,  draws a parallel with a routine that many of us go through multiple times a day – searching for an answer on the internet. The video highlights how isolating it is when you search on the internet but receive the response ‘your search had no results’. It also highlights the hope and promise that comes with additional research into rare diseases, something that must be continuously strived for.

You are still able to participate in raising awareness of the day and be part of the change, by sharing the video, the poster, or any Rare Disease Day material on your Facebook, Twitter or other social media platforms.

This year, on the tenth edition of the day, Rare Disease Day events will be held for the first time in four African nations, Botswana, Nigeria, Senegal and Sudan. Events will also be held for the first time in Saint Pierre and Miquelon.

Learn more at http://www.rarediseaseday.org/page/news/today-is-rare-disease-day-2017

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What I’m doing for Rare Disease Day

Posted on February 28, 2017 by MaryO

rare disease day

 

Each and every day since 1897,  I tell anyone who will listen about Cushing’s.  I pass out a LOT Cushing’s business cards.

Adding to websites, blogs and more that I have maintained continuously since 2000 – at mostly my own expense.

Posting on the Cushing’s Help message boards about Rare Disease Day.  I post there most every day.

Tweeting/retweeting info about Cushing’s and Rare Disease Day today.

Adding info to one of my blogs about Cushing’s and Rare Disease Day.

Adding new and Golden Oldies bios to another blog, again most every day.

Thinking about getting the next Cushing’s Awareness Blogging Challenge set up for April…and will anyone else participate?

And updating https://www.facebook.com/CushingsInfo with a bunch of info today (and every day!)

~~~

Today is Rare Disease Day.

I had Cushing’s Disease due to a pituitary tumor. I was told to diet, told to take antidepressants and told that it was all my fault that I was so fat. My pituitary surgery in 1987 was a “success” but I still deal with the aftereffects of Cushing’s and of the surgery itself.

I also had another Rare Disease – Kidney Cancer, rare in younger, non-smoking women.

And then, there’s the adrenal insufficiency…

If you’re interested, you can read my bio here https://cushingsbios.com/2013/04/29/maryo-pituitary-bio/

 

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What You Should Know About Pituitary Tumors

Posted on February 15, 2017 by MaryO

Ask the Experts

Igor Kravets, MD
Endocrinologist, Assistant Professor Division of Endocrinology,
Diabetes and Metabolism
Stony Brook Medicine
Raphael Davis, MD
Neurosurgeon, Professor and Chair Department of Neurosurgery
Co-Director, Stony Brook University Neurosciences Institute

 

Where do pituitary tumors form?
Dr. Kravets: A pituitary tumor is an abnormal growth of cells in the pituitary gland, which is a small, pea-sized organ located in the center of the brain, behind the nose and eyes. The pituitary is a “master gland” of the body; it produces many hormones that control other endocrine glands and certain functions of the body.
Are they mostly benign or malignant?
Dr. Davis: Most pituitary tumors are benign (non-cancerous). However, because of the location of the pituitary gland at the base of the skull, pituitary tumors can cause problems since they grow upward. Eventually some will press against the area where the optic nerves intersect, causing vision problems. They can also cause hormonal imbalance.What causes pituitary tumors?
Dr. Kravets: No one knows for sure what causes pituitary tumors. About one to five percent of pituitary tumors occur within families. Most are not inherited, however there are certain, rare, inherited conditions such as multiple endocrine neoplasia type 1 (MEN 1), that carry a higher risk of pituitary tumors.

What are the different types of pituitary tumors?
Dr. Davis: Adenomas are benign tumors that develop on the pituitary gland behind the eyes. These tumors can change levels in hormone production or cause vision loss. Craniopharyngiomas are benign tumors that develop at the base of the brain where it meets the pituitary gland. They commonly affect children 5 to 10 years of age, but adults can sometimes be affected in their 50s and 60s.

What are the symptoms?
Dr. Kravets: Symptoms vary depending on the type and size of a pituitary tumor but not all pituitary tumors cause symptoms. Many pituitary tumors are not diagnosed until symptoms appear. Some pituitary tumors are found incidentally on brain imaging obtained for a reason unrelated to the pituitary. Certain symptoms may develop when pituitary tumors grow so large that they exert pressure on surrounding structures.

Such symptoms include:
• Changes in vision (particularly loss of peripheral/outer edge vision)
• Headache

Other symptoms are related to either deficiency or excessive production of certain hormones. Common symptoms caused by such hormonal disturbances include:
• Menstrual cycle changes (irregular or lack of menstrual periods
• Erectile dysfunction or loss of sex drive
• Weight changes
• Production of breast milk by a woman who has not given birth
• Accelerated or stunted growth in a child or teenager
• Growth of the hands, feet, forehead and jaw in adults
• Development of a round face, a hump between the shoulders or both

How is a pituitary tumor diagnosed?
Dr. Kravets: An endocrinologist will ask you about the symptoms you are experiencing, and about your personal and family health history. He or she will perform a physical exam and order tests of your blood and urine. A magnetic resonance imaging (MRI) scan or computerized tomography (CT or CAT) scan may also be ordered to obtain detailed images of the brain and the pituitary gland. In rare instances, a biopsy (surgical procedure to remove a small sample of the tumor for examination) is required.

What treatments are available?
Dr. Davis: Treatments may include surgery, radiation therapy or medication. Transsphenoidal surgery is surgery performed through the nose and sphenoid sinus (located in the very back part of the nose, just beneath the base of the brain) to remove a pituitary tumor. It can be performed with an endoscope, microscope or both and is a team effort between neurosurgeons and ear, nose and throat (otolaryngology/ENT) surgeons. Radiation therapy uses high-energy x-rays to kill the tumor cells and is recommended when surgery is not an option, if the pituitary tumor remains, or if the tumor causes symptoms that are not relieved by medicine.

Why choose Stony Brook?
Dr. Kravets: Our Pituitary Care Center provides access to all of the coordinated expert care you need in one location, close to home — which can make the course of your treatment easier. Our team includes specialists from endocrinology, neurosurgery, otolaryngology (ENT), radiation oncology, neuropathology, neuroradiology, neuro-ophthalmology, and patient education and support.

To make an appointment with one of our Pituitary Care Center endocrinologists, call
(631) 444-0580. To make an appointment with one of our Pituitary Care Center neurosurgeons,
call (631) 444-1213. To learn more, visit stonybrookmedicine.edu/pituitary.

All health and health-related information contained in this article is intended to be general and/or educational in nature and should not be used as a substitute for a visit with a healthcare professional for help, diagnosis, guidance, and treatment. The information is intended to offer only general information for individuals to discuss with their healthcare provider. It is not intended to constitute a medical diagnosis or treatment or endorsement of any particular test, treatment, procedure, service, etc. Reliance on information provided is at the user’s risk. Your healthcare provider should be consulted regarding matters concerning the medical condition, treatment, and needs of you and your family. Stony Brook University/SUNY is an affirmative action, equal opportunity educator and employer.

From https://www.stonybrookmedicine.edu/patientcare/pituitarytumors

Filed under: Cushing's, Diagnostic Testing, pituitary, Rare Diseases, symptoms, Treatments | Tagged: , , , , , | Leave a comment »

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