Tuesday, April 8, 2014

Cushing's Awareness Day 2014

It’s Here!

Dr. Cushing was born in Cleveland Ohio. The fourth generation in his family to become a physician, he showed great promise at Harvard Medical School and in his residency at Johns Hopkins Hospital (1896 to 1900), where he learned cerebral surgery under William S. Halsted.

After studying a year in Europe, he introduced the blood pressure sphygmomanometer to the U.S.A. He began a surgical practice in Baltimore while teaching at Johns Hopkins Hospital (1901 to 1911), and gained a national reputation for operations such as the removal of brain tumors. From 1912 until 1932 he was a professor of surgery at Harvard Medical School and surgeon in chief at Peter Bent Brigham Hospital in Boston, with time off during World War I to perform surgery for the U.S. forces in France; out of this experience came his major paper on wartime brain injuries (1918). In addition to his pioneering work in performing and teaching brain surgery, he was the reigning expert on the pituitary gland since his 1912 publication on the subject; later he discovered the condition of the pituitary now known as “Cushing’s disease“.


Today, April 8th, is Cushing’s Awareness Day. Please wear your Cushing’s ribbons, t-shirts, awareness bracelets or Cushing’s colors (blue and yellow) and hand out Robin’s wonderful Awareness Cards to get a discussion going with anyone who will listen.

And don’t just raise awareness on April 8.  Any day is a good day to raise awareness.

robin-harvey


MaryO

Friday, April 4, 2014

Cushing’s Awareness Challenge, Day 4

Another idea I borrowed from Robin - using a Wordle as inspiration for today's post.

Even though I'm "in remission" since 1987, I'm still way too fatigued, napping every afternoon for several hours.  People think I should be normal since my pituitary surgery was so long ago.  Well, no.


me-tired

Just a few days ago, I posted this abstract on Severe fatigue in patients with adrenal insufficiency.  I don't think that they needed to do this study at all.  Just ask any Cushie!

Abstract

BACKGROUND:

Fatigue is a frequently experienced complaint in patients with adrenal insufficiency (AI) and may be influenced by cortisol levels.

AIM:

The objective of this study was to determine the prevalence of severe fatigue in adrenal insufficiency (AI) patients, to assess which dimensions contribute to fatigue severity and to determine the association between salivary cortisol levels and momentary fatigue.

SUBJECTS AND METHODS:

We performed a cross-sectional study in the outpatient department of a university hospital. Included were 27 patients with congenital adrenal hyperplasia (CAH), 26 patients with primary AI (PAI), 24 patients with secondary AI (SAI) and 31 patients with adrenal insufficiency after treatment for Cushing's syndrome (Cush-AI). Measurements included computerised questionnaires to determine fatigue severity and physical and psychosocial contributors. Patients took four saliva samples at home, in which cortisol levels were measured.

RESULTS:

Severe fatigue was experienced by 41 % of the CAH patients, 42 % of the PAI patients, 50 % of the SAI patients and 42 % of the Cush-AI patients. Psychological distress, functional impairment, sleep disturbance, physical activity, concentration problems and social functioning contributed to the subjective experience of fatigue. Salivary cortisol levels were not correlated with momentary fatigue.

CONCLUSIONS:

A considerable proportion of AI patients experience severe fatigue. Salivary cortisol level is not a significant predictor for momentary fatigue in AI patients.
PMID:
24615365
[PubMed - in process]
From http://www.ncbi.nlm.nih.gov/pubmed/24615365

Back to my nap now...
maryo colorful zebra

Thursday, April 3, 2014

Cushing's Awareness Month, Day 3: Symptoms

robin-symptoms

Robin has made another excellent graphic of some of the symptoms of Cushing's.  There are far too many to be listed in any image, as shown by the list at http://www.cushings-help.com/toc.htm#symptoms

Just to be silly, a few years ago, I did my own version of Cushing's symptoms:

The Seven Dwarves of Cushing's

Men Get Cushing's, Too!


Robin (staticnrg) writes:
“If you don’t have a blog or don’t have the time to blog, you can still share for 30 days. MoxieMelissaTx and I are trying to make it easy for you. Share others’ blog posts. Share the info-graphics we are putting up each day. Tell your own story. Do a Wordle. Do something. It can be one day, or all 30 days. The more we talk about Cushing’s, the more folks will find out about it and/or realize they have it.”
I wanted to share Robin’s graphic.  It’s rare for men to have Cushing’s, even rarer than women.

Some men do get Cushing’s, though.  Some of their bios are here: http://www.cushie.info/index.php/cushing-s/news-items/14-bios-of-male-patients

Cushing's Awareness Month, April 2014


Thanks to Robin for this wonderful logo!  I’ve participated in these 30 days for Cushing’s Awareness several times so I’m not quite sure what is left to say this year but I always want to get the word out when I can.

 As I see it, there’s not a whole lot of change in the diagnosis or treatment of Cushing’s since last year.  (Apparently, Dr Harvey Cushing would have agreed with me: http://cushieblog.com/2014/04/03/what-would-harvey-cushing-say-about-cushings-disease-today/ )

 Maybe this is the year…?

Tuesday, December 31, 2013

Cushing’s Awareness Patient Day

Saturday, February 1st, 2014

San Francisco, California

Hosted by Kate Tully, R.N. and Katherine Waidner, R.N.
Cushing’s Patient Advocates – Corcept Therapeutics

Agenda and details to follow

The day will focus on endogenous Cushing’s, a condition caused by high cortisol in your body.

The day will not cover exogenous Cushing’s caused by steroids taken for various health conditions including asthma, arthritis or lupus.

Saturday, November 30, 2013

ARMC5 Mutations in Macronodular Adrenal Hyperplasia with Cushing's Syndrome

adrenal-hyperplasia

Guillaume Assié, M.D., Ph.D., Rossella Libé, M.D., Stéphanie Espiard, M.D., Marthe Rizk-Rabin, Ph.D., Anne Guimier, M.D., Windy Luscap, M.Sc., Olivia Barreau, M.D., Lucile Lefèvre, M.Sc., Mathilde Sibony, M.D., Laurence Guignat, M.D., Stéphanie Rodriguez, M.Sc., Karine Perlemoine, B.S., Fernande René-Corail, B.S., Franck Letourneur, Ph.D., Bilal Trabulsi, M.D., Alix Poussier, M.D., Nathalie Chabbert-Buffet, M.D., Ph.D., Françoise Borson-Chazot, M.D., Ph.D., Lionel Groussin, M.D., Ph.D., Xavier Bertagna, M.D., Constantine A. Stratakis, M.D., Ph.D., Bruno Ragazzon, Ph.D., and Jérôme Bertherat, M.D., Ph.D.
N Engl J Med 2013; 369:2105-2114 November 28, 2013 DOI: 10.1056/NEJMoa1304603

BACKGROUND

Corticotropin-independent macronodular adrenal hyperplasia may be an incidental finding or it may be identified during evaluation for Cushing’s syndrome. Reports of familial cases and the involvement of both adrenal glands suggest a genetic origin of this condition.

METHODS

We genotyped blood and tumor DNA obtained from 33 patients with corticotropin-independent macronodular adrenal hyperplasia (12 men and 21 women who were 30 to 73 years of age), using single-nucleotide polymorphism arrays, microsatellite markers, and whole-genome and Sanger sequencing. The effects of armadillo repeat containing 5 (ARMC5) inactivation and overexpression were tested in cell-culture models.

RESULTS

The most frequent somatic chromosome alteration was loss of heterozygosity at 16p (in 8 of 33 patients for whom data were available [24%]). The most frequent mutation identified by means of whole-genome sequencing was in ARMC5, located at 16p11.2. ARMC5 mutations were detected in tumors obtained from 18 of 33 patients (55%). In all cases, both alleles of ARMC5 carried mutations: one germline and the other somatic. In 4 patients with a germline ARMC5 mutation, different nodules from the affected adrenals harbored different secondary ARMC5 alterations. Transcriptome-based classification of corticotropin-independent macronodular adrenal hyperplasia indicated that ARMC5 mutations influenced gene expression, since all cases with mutations clustered together. ARMC5 inactivation decreased steroidogenesis in vitro, and its overexpression altered cell survival.

CONCLUSIONS

Some cases of corticotropin-independent macronodular adrenal hyperplasia appear to be genetic, most often with inactivating mutations of ARMC5, a putative tumor-suppressor gene. Genetic testing for this condition, which often has a long and insidious prediagnostic course, might result in earlier identification and better management. (Funded by Agence Nationale de la Recherche and others.)
Supported in part by grants from Agence Nationale de la Recherche (ANR-10-Blan-1136), Corticomedullosurrénale Tumeur Endocrine Network (Programme Hospitalier de Recherche Clinique grant AOM95201), Assistance Publique–Hôpitaux de Paris (Clinical Research Center Grant Genhyper P061006), Institut National du Cancer (Recherche Translationelle 2009-RT-02), the Seventh Framework Program of the European Commission (F2-2010-259735), INSERM (Contrat d’Interface, to Dr. Assié), the Conny-Maeva Charitable Foundation, and the intramural program of the Eunice Kennedy Shriver National Institute of Child Health and Human Development.
Disclosure forms provided by the authors are available with the full text of this article at NEJM.org.
Drs. Assié, Libé, Espiard, Rizk-Rabin, Ragazzon, and Bertherat contributed equally to this article.
We thank Drs. J. Chelly and M. Delpech of the cell bank of Cochin Hospital and Dr. B. Terris of the tumor bank of Cochin Hospital for their help in sample collection; Dr. E. Clauser of the oncogenetic unit of Cochin Hospital for help in microsatellite analysis; Drs. J. Guibourdenche and E. Clauser of the hormone biology unit of Cochin Hospital for cortisol assays; Drs. F. Tissier and Pierre Colin for pathological analysis; Anne Audebourg for technical assistance; J. Metral and A. de Reynies of the Cartes d’Identité des Tumeurs program of Ligue Nationale contre le Cancer for help in genomics studies and fruitful discussions; Dr. P. Nietschke of the bioinformatics platforms of Paris Descartes University for helpful discussions; all the members of the Genomics and Signaling of Endocrine Tumors team and of the genomic platform of Cochin Institute for their help in these studies; and the patients and their families, as well as the physicians and staff involved in patient care, for their active participation.

SOURCE INFORMATION

From INSERM Unité 1016, Centre National de la Recherche Scientifique Unité Mixte de Recherche 8104, Institut Cochin (G.A., R.L., S.E., M.R.-R., A.G., W.L., O.B., L.L., S.R., K.P., F.R.-C., F.L., L. Groussin, X.B., B.R., J.B.), Faculté de Médecine Paris Descartes, Université Paris Descartes, Sorbonne Paris Cité (G.A., S.E., A.G., O.B., L.L., M.S., K.P., F.R.-C., L. Groussin, X.B., J.B.), Department of Endocrinology, Referral Center for Rare Adrenal Diseases (G.A., R.L., O.B., L. Guignat, L. Groussin, X.B., J.B.), and Department of Pathology (M.S.), Assistance Publique–Hôpitaux de Paris, Hôpital Cochin, and Unit of Endocrinology, Department of Obstetrics and Gynecology, Hôpital Tenon (N.C.-B.) — all in Paris; Unit of Endocrinology, Centre Hospitalier du Centre Bretagne, Site de Kério, Noyal-Pontivy (B.T.), Unit of Endocrinology, Hôtel Dieu du Creusot, Le Creusot (A.P.), and Department of Endocrinology Lyon-Est, Groupement Hospitalier Est, Bron (F.B.-C.) — all in France; and the Section on Endocrinology and Genetics, Program on Developmental Endocrinology and Genetics and the Pediatric Endocrinology Inter-Institute Training Program, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD (C.A.S.).
Address reprint requests to Dr. Bertherat at Service des Maladies Endocriniennes et Métaboliques, Centre de Référence des Maladies Rares de la Surrénale, Hôpital Cochin, 27 rue du Faubourg St. Jacques, 75014 Paris, France, or at jerome.bertherat@cch.aphp.fr.
Access this article: Subscribe to NEJM | Purchase this article

Friday, May 10, 2013

Important! Please Ask your Member of Congress to join the Rare Disease Congressional Caucus



Help us strengthen the rare disease community's voice on Capitol Hill!  Please take 3 minutes to ask your Member of Congress to join the Rare Disease Caucus at http://bit.ly/RareAlert.

It's easy - the Action Center has a draft letter that will automatically be sent to your Member of Congress - just put in your name and address & click send.  We also encourage you to personalize the letter to share information about your specific disease.  If your Congress Member is already on the Caucus, the letter will automatically populate as a thank you letter instead - these are just as important to send!

It can take up to 10 letters from constituents for a Member to respond so please share this Action Alert with your friends, family & colleagues.  Join our Facebook event & invite your friends:   http://on.fb.me/17Mlpjg 

The Rare Disease Congressional Caucus will help bring public and Congressional awareness to the unique needs of the rare disease community – patients, physicians, scientists, and industry, and create opportunities to address roadblocks in access to and development of crucial treatments.  The Caucus will give a permanent voice to the rare disease community on Capitol Hill.  Working together, we can find solutions that turn hope into treatments.

Tuesday, March 19, 2013

For Cushing's Awareness Challenge Bloggers


Cushie bloggers - who would like to share your post on April 8 (Cushing's Awareness Day) with our friends at Adrenal Insufficiency United, on their blog?

Also, they are interested in sharing others of our posts from April.  Please let me know if I have your permission to share your blog post(s) with them.  I won't without your permission.

Thanks!

Thursday, March 14, 2013

Cushing's Awareness Blogging Challenge 2013

Do you blog? Want to get started?

Since April 8 is Cushing's Awareness Day, several people got their heads together to create the Second Annual Cushing's Awareness Blogging Challenge.

All you have to do is blog about something Cushing's related for the 30 days of April and add one of the logos below to your blog.

Robin designed this year's version of our "official logo" to put on your blog.


Cushing's Awareness Challenge 2013
challenge-2013nb 


 If your blog wants you to upload an image from your desktop, right-click on the image above and choose "save-as". Remember where you saved it to! 

 To link to the image with the yellow border, use this URL: http://www.cushings-help.com/images/challenge-2013b.jpg 

 To link to the image without a border, use this URL: http://www.cushings-help.com/images/challenge-2013nb.jpg 

 In all cases, the URL for the site is http://www.cushings-help.com 

Please let me know the URL to your blog in the comments area of this post or and I will list it on CushieBloggers ( http://cushie-blogger.blogspot.com/

The more people who participate, the more the word will get out about Cushing's. 

  Suggested topics - or add your own!

In what ways have Cushing's made you a better person?
What have you learned about the medical community since you have become sick?
If you had one chance to speak to an endocrinologist association meeting, what would you tell them about Cushing's patients?
What would you tell the friends and family of another Cushing's patient in order to garner more emotional support for your friend?
Challenges with Cushing's? How have you overcome challenges? Stuff like that.
I have Cushing's Disease....(personal synopsis)
How I found out I have Cushing's
What is Cushing's Disease/Syndrome? (Personal variation, i.e. adrenal or pituitary or ectopic, etc.)
My challenges with Cushing's
Overcoming challenges with Cushing's (could include any challenges)
If I could speak to an endocrinologist organization, I would tell them...
. What would I tell others trying to be diagnosed? What would I tell families of those who are sick with Cushing's?
Treatments I've gone through to try to be cured/treatments I may have to go through to be cured.
What will happen if I'm not cured?
I write about my health because...
10 Things I Couldn’t Live Without.
My Dream Day.
What I learned the hard way
Miracle Cure. (Write a news-style article on a miracle cure. What’s the cure? How do you get the cure? Be sure to include a disclaimer)
Health Madlib Poem. Go to http://languageisavirus.com/cgi-bin/madlibs.pl and fill in the parts of speech and the site will generate a poem for you.
Give yourself, your condition, or your health focus a mascot. Is it a real person? Fictional? Mythical being? Describe them. Bonus points if you provide a visual!
5 Challenges and 5 Small Victories.
The First Time I...
Make a word cloud or tree with a list of words that come to mind when you think about your blog, health, or interests. Use a thesaurus to make it branch more.
How much money have you spent on Cushing's, or, How did Cushing's impact your life financially?
Why do you think Cushing's may not be as rare as doctors believe?
What is your theory about what causes Cushing's?
How has Cushing's altered the trajectory of your life? What would you have done? Who would you have been?
What three things has Cushing's stolen from you? What do you miss the most? What can you do in your Cushing's life to still achieve any of those goals? What new goals did Cushing's bring to you?
How do you cope?
What do you do to improve your quality of life as you fight Cushing's?
Your thoughts...?

Monday, December 17, 2012

US FDA approves Novartis' Signifor for first medication to treat Cushing's disease

Basel

Monday, December 17, 2012, 16:00 Hrs  [IST]

 

The US Food and Drug Administration (FDA) has approved Novartis' Signifor (pasireotide) injection for the treatment of adult patients with Cushing's disease for whom pituitary surgery is not an option or has not been curative. 

 

Signifor is the first medicine to be approved in the US that addresses the underlying mechanism of Cushing's disease, a serious, debilitating endocrine disorder caused by the presence of a non-cancerous pituitary tumour which ultimately leads to excess cortisol in the body.

 

This approval follows a unanimous recommendation from the FDA Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) in support of the use of Signifor.

 

"The FDA approval of Signifor for Cushing's disease brings a novel pituitary-directed therapy to patients with limited treatment options," said Hervé Hoppenot, president, Novartis Oncology. "Today's milestone reinforces Novartis' commitment to addressing unmet needs and advancing treatments for rare pituitary-related disorders."

 

Cushing's disease most commonly affects adults as young as 20 to 50 years and affects women three times more often than men. It may present with weight gain, central obesity, a round, red full face, severe fatigue and weakness, striae (purple stretch marks), high blood pressure, depression and anxiety. Cushing's disease can cause severe illness and death with mortality up to four times higher than in the healthy population.

 

The approval is based on data from PASPORT-CUSHINGS (PASireotide clinical trial PORTfolio - CUSHING'S disease), the largest randomized Phase III study to evaluate a medical therapy in patients with Cushing's disease. Results from the PASPORT-CUSHINGS study found that a decrease in mean urinary-free cortisol (UFC), the key measure of biochemical control of the disease, was sustained during the treatment period in most patients with a subset of patients reaching normal levels. The study also showed that certain clinical manifestations of Cushing's disease tended to improve.

 

"Patients with Cushing's disease may suffer from debilitating manifestations, and there are many serious health complications associated with the disease," said Mary Andrews, CEO and Co-Founder of the US non-profit, The MAGIC Foundation. "The FDA approval of Signifor offers the option of a medical therapy that may help certain patients with Cushing's disease."

 

In April 2012, the European Commission approved Signiforfor the treatment of adult patients with Cushing's disease for whom surgery is not an option or for whom surgery has failed. Other worldwide regulatory filings for pasireotide for this use are also underway.

Signifor (pasireotide) is approved in the US for the treatment of adult patients with Cushing's disease for whom pituitary surgery is not an option or has not been curative, and in the European Union for the treatment of adult patients with Cushing's disease for whom surgery is not an option or for whom surgery has failed.

 

For the treatment of Cushing's disease, Signifor has been studied as a twice-daily subcutaneous (sc) injection and is currently being evaluated as a long-acting release (LAR), once-monthly intramuscular (IM) injection as part of a global Phase III program in Cushing's disease and acromegaly. Signifor is a multireceptor targeting somatostatin analog that binds with high affinity to four of the five somatostatin receptor subtypes (sst 1, 2, 3 and 5).

 

From http://pharmabiz.com/NewsDetails.aspx?aid=72752&sid=2

Thursday, December 6, 2012

RARE Video Project

Global Genes | RARE Project would like your voice to be heard!

Share your home videos!

We are currently looking for your home videos that illustrate what life is like for rare disease patients and caregivers on this complex and often emotional journey.  This could be an assortment of moments that you’ve captured on your phone, a camera, or a digital recording device.  We want the key moments, the most beautiful, personal moments that represent not only the diseases, but you and your child(ren) as well.

A few examples of what we are looking for in these clips:

Births

In Pain

Overcoming

Birthdays

Sadness

Happiness

Hospital Visits

Loss

Laughter

Medicines

Struggle

Important Events

At Play

Tears

Family

Be creative, think outside the box.   Look for clips that are shot well, with nice light.  Give us variety!  Old footage, new footage, maybe even something your child has shot.  We want personal, private moments.  That is what will send the strongest message.

What are we going to do with this?

We are working with an award-winning filmmaking team to select submissions that will be compiled together to create a visual storyline of the years of your ongoing journey.  This is your chance to share the moments that you see and experience with a global audience.

This is your moment to be heard.

More information at http://globalgenes.org/rare-video-submission-form/

Cushing Syndrome Overview

Cushing’s syndrome (pronounced KOOSH-ingz SIN-drohm) is a condition that occurs when a person’s body tissues are exposed over time to too much of the hormone cortisol (pronouncedKAWR-tuh-sawl). The syndrome can be caused by taking certain medicines or, less commonly, it can be caused by noncancerous or cancerous tumors. Cushing’s syndrome includes a range of symptoms, but they can be treated and, in most cases, the syndrome can be cured. The NICHD is one of the many federal agencies that support and conduct research on the causes of Cushing’s syndrome, detection of its symptoms as soon as possible, and development of improved treatments.

For more information about this topic, select the Condition Information, Research Information, Clinical Trials, or Resources and Publications link in the menu on the left.

Fast Facts

Common Name

  • Cushing's syndrome

Scientific Name

  • Hyperadrenocorticism (pronounced HAHY-per-uh-dree-noh-KAWR-ti-siz-uhm)
  • Hypercortisolism (pronounced HAHY-per-KAWR-ti-sol-iz-uhm)

Causes

The most common cause of Cushing’s syndrome is taking medication that contains the hormone cortisol. This leaves the body with more cortisol than it would normally contain from the natural production of cortisol.1 Less commonly, a cancerous or noncancerous tumor in the body can cause too much cortisol production.2

Number of People Affected

Among 1 million people, two or three will develop endogenous (non-medicine-related) Cushing’s syndrome each year in the United States.3 Women are three times more likely than men to have the condition.4

Common Symptoms

The symptoms of Cushing’s syndrome vary, especially in mild cases, but patients may have some or most of the following1:

  • Upper-body obesity, with thin arms and legs
  • A round, red face
  • Skin problems, such as acne, reddish-blue streaks, or easy bruising
  • Muscle and bone weakness, including backache
  • Fat that collects between the shoulders
  • Poor growth in children5

Common Treatments

In most cases Cushing’s syndrome can be cured. The treatment depends on what is causing the excess cortisol in the body.6,7

Cushing’s syndrome can be treated by the following:

  • Medication. If medication is to blame, a health care provider can reduce the dose or change the type of drug.
  • Overproduction. If the body is making too much cortisol because of a tumor, treatments may include oral medication, surgery, radiation, or a combination of these approaches.

 


  1. Stewart P. M., & Krone, N. P. (2011). The adrenal cortex. In Kronenberg, H. M., Shlomo, M., Polonsky, K. S., & Larsen, P. R. (Eds.). Williams textbook of endocrinology (12th ed.) (chap. 15). Philadelphia, PA: Saunders Elsevier. [top]
  2. Nieman, L. K., & Ilias, I. (2005) Evaluation and treatment of Cushing’s syndrome. Journal of American Medicine, 118(12), 1340-1346. PMID 16378774. [top]
  3. Lindholm, J., Juul, S., Jørgensen, J. O. L, Astrup, J., Bjerre, P., Feldt-Rasmussen, U., et al. (2001). Incidence and late prognosis of Cushing’s syndrome: A population-based study. Journal of Clinical Endocrinology and Metabolism, 86(1), 117-123. PMID 11231987[top]
  4. Steffensen, C., Bak, A. M., Rubeck, K. Z., & Jørgensen, J. O. (2010). Epidemiology of Cushing’s syndrome. Neuroendocrinology, 92(Suppl 1), 1-5. PMID 20829610[top]
  5. Batista, D. L., Riar, J., Keil, M., & Stratakis, C.A. (2007). Diagnostic tests for children who are referred for the investigation of Cushing syndrome. Pediatrics120(3), e575-e586. [top]
  6. Nieman, L. K., Biller, B. M. K., Findling, J. W., Newell-Price, J., Savage, M. O., et al. (2008). The diagnosis of Cushing’s syndrome: An Endocrine Society clinical practice guideline. Retrieved April 8, 2012, fromhttp://www.endo-society.org/guidelines/final/upload/Cushings_Guideline.pdf (PDF - 510 KB). [top]
  7. Boscaro, M., & Arnaldi, G. (2009). Approach to the patient with possible Cushing’s syndrome. Journal of Clinical Endocrinology and Metabolism, 94(9), 3121. [top]

 

Last Updated Date: 11/30/2012
Last Reviewed Date: 11/30/2012

Tuesday, December 4, 2012

Mifepristone: is there a place in the treatment of Cushing's disease?

Carmichael JD, Fleseriu M.

Source

Cedars-Sinai Medical Center, 8700 Beverly Blvd, Los Angeles, CA, 90048, USA, john.carmichael@cshs.org.

Abstract

The purpose was to review the use of mifepristone in the treatment of Cushing's syndrome (CS) in the context of other recently published studies. We review the use of mifepristone, as published in the recent Study of the Efficacy and Safety of Mifepristone in the Treatment of Endogenous Cushing's Syndrome (SEISMIC). We also review the multiple case reports and case series of mifepristone use in CS. A review of other medications used in the treatment of Cushing's disease (CD), including pasireotide and cabergoline also provides context for the discussion of the role of mifepristone in the treatment of CD. The results show that the treatment of CD has been primarily surgical with medical therapy reserved for adjuvant therapy when primary treatment fails or other therapies require time for optimal efficacy. Two recent large prospective studies, using pasireotide and mifepristone provide new clinical insights to the medical treatment of CD in particular. Mifepristone has been used to treat excessive cortisol production by blocking the action of cortisol at the level of the glucocorticoid receptor. Until recently, the majority of clinical experience with mifepristone on the treatment of excess cortisol was derived from case reports and small case series. Based on the SEISMIC study, mifepristone was FDA approved for hyperglycemia associated with CS. In conclusion the role of mifepristone in the treatment of CD remains one of adjuvant therapy. Its place among other choices for medical therapy has yet to be firmly established and an evidenced-based approach toward the use of novel medications in the treatment of CD has not been made. Selection of medication depends on drug approval and availability in individual countries and requires cautious assessment of potential adverse effects, consideration of patient comorbidities, and efficacy.

 

PMID: 23192246 [PubMed - as supplied by publisher]