Severe fatigue, decreased physical activity in patients with Addison’s disease

Posted on March 18, 2016 by MaryO

van der Valk ES, et al. Clin Endocrinol. 2016;doi:10.1111/cen.13059.

Dutch adults with primary adrenal insufficiency reported abnormal or severe fatigue, reduced physical activity and significantly reduced quality of life vs. healthy controls, according to recent survey results.

In a cross-sectional study, Eline S. van der Valk, MD, of Amphia Hospital in Breda, the Netherlands, and colleagues also found that patients with Addison’s disease reported physical activity levels that were significantly lower than those reported by other Dutch chronically ill patients.

“The clinical relevance of the impaired [quality of life] and increased fatigue found in our study is supported by the size of the differences in scores and the restriction in physical activity in patients with [Addison’s disease], an important activity in daily life,” the researchers wrote. “Physical inactivity could be very detrimental in [Addison’s disease] because the prevalence of other cardiovascular risk factors is already increased, and it has been demonstrated that patients with [Addison’s disease] have an up to twofold increased mortality rate from [CVDs].”

Researchers analyzed survey data from 328 Dutch adults with Addison’s disease on stable glucocorticoid replacement therapy with hydrocortisone or cortisone acetate (mean age, 53 years; 223 women; mean duration of disease after diagnosis, 15.6 years). Participants attended outpatient clinics at University Medical Center Utrecht and Radboud University Nijmegen Medical Centre, or were members of the Dutch Association of Addison and Cushing Patients. They completed general and health-related quality of life (Short Form 36; Checklist Individual Strength) and physical activity questionnaires. Scores were compared with a random sample of 1,718 adults who completed a Dutch National Health Survey (controls).

Within the cohort, 53% of participants had isolated Addison’s disease; 74.1% received hydrocortisone therapy; 25.9% received cortisone acetate therapy; 87.2% received fludrocortisone therapy; and 23.2% received dehydroepiandrosterone replacement therapy.

Researchers found that 45.7% of participants with Addison’s disease met the standard of physical activity (Dutch standard of healthy physical exercise, defined as moderately intensive physical exercise for 30 minutes daily 5 days per week; “Fitnorm,” defined as 20 minutes of intensive physical exercise at least 3 days per week) vs. 67.8% of controls (P < .01). Researchers found 61% of participants with Addison’s disease reported abnormal fatigue, and 43% reported severe fatigue. Mean fatigue scores were significantly higher vs. controls (mean difference, 32.6; 95% CI, 24-41).

In both men and women with Addison’s disease, researchers found that quality of life scores in all component summaries were significantly decreased compared with controls, particularly in participants aged 65 years and younger. – by Regina Schaffer

Disclosure: The researchers report no relevant financial disclosures.

From http://www.healio.com/endocrinology/adrenal/news/online/%7Ba8914384-d40e-41ab-aa1c-134d856d2edd%7D/severe-fatigue-decreased-physical-activity-in-patients-with-addisons-disease

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Rare Disease Day, 2016!

Posted on February 29, 2016 by MaryO

rare-disease-day-robin

There are events all over the world today.  What are *You* doing to raise awareness for Cushing’s, Addison’s or other rare disease you have?

Many thanks to Robin for the great graphic!

 

The USA joined Rare Disease Day in 2009, making the campaign a truly international affair. Diverse events and campaigns have been organised since then, including educational programmes in schools and a collection of photographs entitled “Handprints across America” with the Rare Disease logo across the USA. In 2013 President Barack Obama sent a letter proclaiming his support of the day. In 2015, the day was a nation-wide affair, with events everywhere from California to New York to Texas. More than 35 states participated, holding conferences, artistic events, fundraising walks, and benefit dinners. In Chicago, a “Rock Rare Diseases” event created a playlist that was featured at many hospitals on the special day.

NORD, the National Organization for Rare Disorders, is committed to the identification, treatment, and cure of rare diseases through programmes of education, advocacy, research and patient services. They can be contacted directly to help you find a patient organisation locally which may have more information about a specific rare disease or disorder. Find their contact information on the bottom of this page.

You can also get involved! Do you know of any events not listed here? Email us at rarediseaseday@eurordis.org.

On Monday, February 29th, Rare Disease Week on Capitol Hill kicks off! Hundreds of advocates from around the country will be in Washington, D.C. for a full week of events. Space remains for the Caucus Briefing on Thursday March 3rd and the Rare Artist Reception. Can’t make it to Washington D.C.? NORD is helping coordinate State House Events across the U.S.

On Wednesday, March 2nd, the EveryLife Foundation for Rare Diseases is holding a Virtual Lobby Day for advocates who cannot attend the events in D.C. The event will ask advocates to contact Congress and ask that they co-sponsor the OPEN ACT, legislation to double the number of rare disease treatments. Please share widely on social media.

On Thursday, March 3rd, the first bicameral Congressional Rare Disease Caucus briefing will be held in the Auditorium of the U.S. Capitol Visitor Center at noon. Attendees will hear from the co-chairs of the Caucus and a panel discussion featuring key thought-leaders from the patient, regulatory, and industry communities who will discuss the Rare Disease Ecosystem.

On Wednesday, March 9th, the Senate Health, Education, Labor, and Pensions Committee (HELP) will hold its second hearing as part of its biomedical innovation initiative. To date, the Advancing Hope Act (Priority Review Voucher program at FDA) is scheduled for consideration. The final hearing is slated for April 6th, although no bills have been announced for consideration.

On Wednesday, March 16th, the EveryLife Foundation for Rare Diseases will hold a public webinar on newborn screening. The Foundation has just launched newborn screening legislation in California to expand and streamline screening for rare diseases.

On Wednesday, March 23rd, RDLA will hold its next monthly webinar. The agenda is OPEN! Please send suggestions for action items or policy issues to Vignesh Ganapathy at vganapathy@everylifefoundation.org.

Filed under: adrenal, adrenal crisis, Cancer, Cushing's, pituitary, Rare Diseases | Tagged: , , , , | Leave a comment »

Addison’s disease may cause psychosis, say researchers

Posted on December 10, 2015 by MaryO

adrenal-glands

 

Research suggests that chronic adrenal insufficiency, more commonly known as Addison’s disease, may be responsible for psychiatric symptoms in those who suffer with it. Unfortunately, these symptoms are poorly understood and inadequately studied. In one case, a 41-year-old construction worker was admitted to a psychiatric clinic complaining of depression. He had trouble sleeping and concentrating and had lost 6 pounds due to a loss of appetite. He was placed on 20mg of fluoxetine but returned 2 weeks later complaining that the therapy did not work, and even reported hallucinating his ex-wife, who had recently died in a car accident. He returned again later 4 months later and was found to have a weak pulse, major hypotension, and hyponatremia and hyperkalemia. It was at this point that he was diagnosed with Addison’s disease.

The disease was first described by Thomas Addison in the mddle of the 19th century. It involves inadequate secretion from the adrenal glands, leading to lower secretion of glucocorticoids. Its usual symptom pigmentation involves fatigue, weight loss, nausea, vomiting, weakness and abdominal pain. Among its psychiatric symptoms are psychosis and delirium.

Filed under: adrenal, adrenal crisis, Rare Diseases | Tagged: , , , , | 1 Comment »

COR-003 Clinical Trial for Cushing’s Syndrome

Posted on November 19, 2015 by MaryO

CureClick_Trial_Card_CushingsBLU2

 

This trial is testing the safety and effectiveness of an investigational drug for the treatment of Cushing’s Syndrome. Under the supervision of qualified physicians, cortisol levels and symptoms of Cushing’s Syndrome will be closely followed along with any signs of side effects.

More about the study:

The study drug (COR-003) is administered by tablets.

  • There will be 90 participants in this trial
  • There is no placebo used in the trial

If you are interested, please find the full study details and eligibility criteria listed here.

Eligibility Criteria:

Participants must:

  • be at least 18 years old
  • have been diagnosed with endogenous Cushing’s Syndrome by a medical professional (not caused by the use of steroid medications)

Participants must not:

  • have been treated with radiation for Cushing’s Syndrome in the past 4 years
  • be currently using weight loss medication
  • have been diagnosed with uncontrolled hypertension, some forms of cancer, adrenal carcinoma, Hepatitis B / C, or HIV

Please complete the online questionnaire to check if you’re eligible for the trial.

If you’re not familiar with clinical trials, here are some FAQs:

What are clinical trials?

Clinical trials are research studies to determine whether investigational drugs or treatments are safe and effective for humans. All new investigational medications and devices must undergo several clinical trials, often involving thousands of people.

Why participate in a clinical trial?

You will have access to investigational treatments that would be available to the general public only upon approval. You will also receive study-related medical care and attention from clinical trial staff at research facilities. Clinical trials offer hope for many people and an opportunity to help researchers find better treatments for others in the future.

Learn why I’m posting about this Clinical Trial

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Global Cushing’s Syndrome Market Size 2015

Posted on October 19, 2015 by MaryO

Cushing’s as money makers for drug companies 😦

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Steroidogenesis inhibitors were responsible for approximately 28% of total drug sales in the 6MM in 2013, equating to around $50m. As a consequence of this trend, GlobalData expects overall revenues generated by this drug class to increase by approximately 390% to reach around $247m, encompassing 49% of total drug sales in the 6MM in 2018.

The expansion in this segment of the CS market is fuelled by the introduction of premium-priced pharmacological agents such as Novartis’ LCI699 and Cortendo AB’s NormoCort (COR-003) in the US, as well as the arrival of HRA Pharma’s Ketoconazole HRA (ketoconazole) to the European CS stage. One of the greatest unmet needs in this indication is a lack of effective drugs directed against the underlying cause of Cushing’s disease (the pituitary tumor).

Despite this demand, pharmaceutical companies are continuing to adopt a strategy that simply targets the adrenal glands. As a result, there is a vast amount of room for new or existing players to penetrate the market and capture considerable patient share.

Highlights

Key Questions Answered

Although the current standard of care (ketoconazole) is cheap and reasonably effective in most CS patients, it possesses worrying safety profiles, inconvenient dosing schedules, is difficult to obtain and can display waning efficacy over time. Newer medical treatments, for example, Novartis’ Signifor (pasireotide) and Corcept Therapeutics’ Korlym (mifepristone) address only some of these issues; yet, present their own limitations. The CS market is still marked by the existence of a multitude of unmet needs. What are the main unmet needs in this market? Will the drugs under development fulfil the unmet needs of the CS market?

The late-stage CS pipeline is sparsely populated; however, those drugs in development will be a strong driver of CS market growth. Which of these drugs will attain high sales revenues during 2013-2018? Which of these drugs will have the highest peak sales at the highest CAGR, and why?

Key Findings

One of the main drivers influencing growth in the Cushing’s syndrome market will be the introduction of second-generation steroidogenesis inhibitors, LCI699 and NormoCort (COR-003), in the US, which will rival existing standard of care medical treatments.

Another strong driver will be the arrival of Corcept Therapeutics’ Korlym (mifepristone) and HRA Pharma’s Ketoconazole HRA (ketoconazole) to the European CS market. Both drugs will stimulate significant growth here.

The launch of Novartis’ Signifor LAR (pasireotide) in the 6MM will equip physicians with a less frequently administered formulation of Signifor.

Reasons for inadequate CS treatment include poor physician awareness of the condition, delayed diagnosis, a lack of efficacious drugs for individuals suffering from severe hypersecretion, and a shortage of effective medicines targeting the source of Cushing’s disease.

Scope

Overview of Cushing’s syndrome, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.

Annualized Cushing’s syndrome therapeutics market revenues, annual cost of therapies and treatment usage pattern data from 2013 and forecast for five years to 2018.

Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the Cushing’s syndrome therapeutics market.

Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.

Analysis of the current and future market competition in the global Cushing’s syndrome therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Reasons to buy

Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline. Additionally a list of acquisition targets included in the pipeline product company list.

Develop business strategies by understanding the trends shaping and driving the Cushing’s syndrome therapeutics market.

Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the Cushing’s syndrome therapeutics market in the future.

Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.

Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.

Track drug sales in the 6MM Cushing’s syndrome therapeutics market from 2013-2018.

Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

From http://www.medgadget.com/2015/10/global-cushings-syndrome-market-size-2015-share-trend-analysis-price-research-report-forecast.html

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