Doctors see Cushing’s symptoms as considerable, favor better therapies

Endocrinologists in US list life quality gains as key in choosing treatment

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by Andrea Lobo |

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A survey of U.S. doctors treating people with Cushing’s syndrome noted a considerable burden of symptoms, ranging from fatigue to muscle weakness, that weigh on patients’ life quality and personal finances, and a need for better treatments.

These endocrinologists  — physicians who specialize in disorders affecting hormone-producing glands — also reported that improving a patient’s health-related quality of life was their key factor in choosing a specific treatment.

“New [Cushing’s syndrome] treatments are needed that have long-term efficacy, fewer side effects, and effective reimbursement,” the researchers wrote.

Survey findings were detailed in the study, “A medical chart audit to assess endocrinologist perceptions of the burden of endogenous Cushing’s syndrome,” published in the journal Pituitary. The work was supported by Strongbridge Biopharma, a subsidiary of Xeris Pharmaceuticals, which markets Recorlev (levoketoconazole), an approved treatment for Cushing’s disease.

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69 endocrinologists, all treating Cushing’s patients, responded to survey

Cushing’s syndrome refers to a group of conditions characterized by high cortisol levels. In Cushing’s disease, the most common form of endogenous Cushing’s, a tumor in the brain’s pituitary gland leads to the increased production of adrenocorticotropic hormone (ACTH), which prompts the adrenal glands to produce excessive amounts of cortisol.

Of note, endogenous Cushing’s refers to disease forms in which excess cortisol levels are brought on by problems within the body, generally a tumor.

Due to the variety of Cushing’s symptoms, patients often visit several specialists before receiving a definitive disease diagnosis. These include endocrinologists, doctors who specialize in disorders of the endocrine system, the network of hormone-producing glands in the body.

Although the disease is known to affect patients’ overall quality of life, “studies reporting both the patient burden (via medical charts) and physician perceptions of burden are lacking,” the scientists wrote.

Researchers in the U.S. sought to characterized Cushing’s overall burden — its symptoms, treatments, and use of healthcare resources — based on patients’ medical charts and endocrinologists’ perceptions of available treatments and factors influencing therapy decisions.

The study surveyed 69 endocrinologists, who provided information covering 273 Cushing’s patients. Physicians had been in practice for a mean of 17.3 years and, in the six months before the survey, had seen a mean of 30 patients with endogenous Cushing’s.

The majority of these patients were women (60%), with a mean age at diagnosis of 40.2.

Patients’ medical chart data indicated a high symptom burden. At the time of diagnosis, 34% of patients experienced one to three symptoms, 33% had four to six different symptoms, 20% had seven to nine, and 13% had 10 or more. The most common included fatigue, weight gain, acne, muscle weakness, decreased sex drive, headache, swelling, emotional instability, and excessive hair growth on the face, chest, or back.

Among Cushing’s treatments, 28.9% underwent surgery alone, 31.9% were solely on medications, and 31.1% were treated with surgery combined with medications. A small percentage of patients were treated with radiotherapy, either alone or in combination with surgery and/or medications.

According to the researchers, “although symptoms decreased post-treatment, a large proportion of subjects still exhibited these symptoms.”

Patients had a mean of one hospitalization each year, lasting a mean of 4.3 days. They also had a mean of 0.6 emergency room visits and 4.3 outpatient visits per year.

How a treatment affected quality of life, specific symptoms seen as key

While life quality was most important to endocrinologists in evaluating and selecting a Cushing’s treatment, other factors they also considered as key were approaches to improve cardiovascular health and those addressing specific symptoms, especially for mental health, like depression and anxiety, or symptoms affecting the skeletal and muscular or neurological systems.

Physicians reported the highest satisfaction with surgery, favoring its initial efficacy followed by its durability, safety and tolerability, and side effects.They also favor it when considering their patients’ overall experience. Based on the same criteria, these doctors next reported being more satisfied with pharmaceutical treatments than with radiation therapy or other interventions.

Still, the study noted that many patients continue to experience symptoms despite surgery, as reflected in the “importance providers place on the availability of post-surgery treatment options.”

Endocrinologists strongly agreed that Cushing’s patients have significant and unmet clinical needs, and that out-of-pocket costs for medications are a significant burden. They also felt strongly that better support services are needed for medications, as they typically lead to better treatment adherence.

“Patients with [Cushing’s syndrome] continue to experience symptoms such as fatigue, weight gain, muscle weakness, and emotional instability even after seeking and receiving treatment,” the researchers wrote.

“Future research is needed to develop a treatment paradigm that alleviates disease burden in patients with [Cushing’s syndrome] and that results in long-term disease control with a favorable side effect profile,” they added.