Despite treatment, all patients with hypoparathyroidism experience physical and psychological symptoms that negatively impact their quality of life, a study finds.
The findings were presented in a poster, “Understanding the Patient-centered Impact of Hypoparathyroidism on Functioning and Well-being,” by Ascendis Pharma during the Endocrine Society’s annual meeting, ENDO 2019, which took place March 23–26 in New Orleans.
Limited data is available about the short-term symptoms and burden of illness that is experienced by people with hypoparathyroidism.
“I have lived with HP (hypoparathyroidism) for over 25 years and have personally experienced how this debilitating disease affects all areas of life — physical, emotional and social,” Deb Murphy, president of the HypoPARAthyroidism Association, said in a press release.
To understand what it’s like to live with hypoparathyroidism, researchers at Ascendis Pharma conducted a study to evaluate the impact of hypoparathyroidism on patients’ quality of life.
“This study highlights that currently available therapies are still not enough. It is critical to not only increase awareness of HP, but to develop new treatment options that address all aspects of the disease and improve patients’ quality of life,” Murphy said.
The researchers interviewed 42 adult patients with post-surgical or idiopathic (unknown cause) hypoparathyroidism. To be included in this study, patients had to be stable on a standard-of-care treatment (either oral calcium, vitamin D supplements, and/or parathyroid hormone therapy) for at least three months.
Most patients (83.3%) were female and had hypoparathyroidism as a result of surgery to remove the parathyroid glands. Despite treatment, 71% of patients reported that managing their condition was “somewhat,” “a lot,” or “extremely difficult.” In addition, 64% of patients reported that it was “difficult to find primary care physicians, specialists, and emergency department physicians” with sufficient knowledge about their condition.
All patients reported that they experienced physical problems that were attributed to hypoparathyroidism, such as paresthesia (tingling and numbness), muscle cramps, and fatigue. Additionally, 86% of patients suffered from “brain fog,” which refers to cognitive issues such as difficulties with memory and forgetfulness, talking, ability to concentrate, fuzzy thinking, and comprehending information.
These patients also reported experiencing significant psychological issues: 81% said they had anxiety, 64% experienced frustration, and 62% had depression.
The patients’ physical functioning was also affected. Most patients (95%) experienced problems with physical functioning and consequently had fewer social interactions. In particular, they experienced problems exercising, being mobile, and participating in social interactions.
All patients said that the disease interfered with their daily life, namely with the ability to do things around the home (79%), and their overall productivity (67%).
“These results clearly show the need for a treatment option that can address all aspects of HP to help alleviate the significant quality-of-life issues these patients face every day,” said Jonathan Leff, MD, Ascendis Pharma’s chief medical officer.
Ascendis Pharma is the developer of TransCon PTH, a long-acting prodrug that, when inside the body, releases the unmodified active parathyroid hormone in a controlled manner, increasing hormone levels to within normal range for a 24-hour period. This is expected to normalize blood and urinary calcium levels, serum phosphate levels, and bone turnover.
The company will soon begin a Phase 2 trial (PaTH) in up to 40 sites worldwide this year. The study will examine the safety, tolerability, and early efficacy of TransCon PTH in approximately 40 adult patients with hypoparathyroidism.