Understanding the burden of illness associated with hypoparathyroidism reported among patients in the PARADOX study.

OBJECTIVE: Hypoparathyroidism is a rare disease caused by lack of parathyroid hormone (PTH) leading to hypocalcemia, hyperphosphatemia, and a variety of symptoms. This study aimed to quantify the clinical and social burden of illness from the perspective of affected patients. METHODS: A web-based instrument was developed with input from patients, clinical experts, and the Hypoparathyroidism Association. Qualifying participants were ≥18 years old, diagnosed with hypoparathyroidism for ≥6 months, and U.S. residents. Questions focused on demographics, diagnosis perceptions, current attitudes, medical management, current symptoms, acute episodes, comorbidities, personal life, and employment. RESULTS: A total of 374 adults (mean age, 49 ± 12 years; female, 85%) with hypoparathyroidism (mean duration, 13 ± 12 years; severe condition, 30.5%) completed the survey. Patients reported visiting a mean of 6 ± 8 physicians before and after their diagnosis. The majority strongly agreed with feeling unprepared to manage the condition at diagnosis (56%), that controlling their hypoparathyroidism is harder than expected (60%), and that they were concerned about long-term complications of their current medications (75%). More than 10 symptoms were experienced by 72% of patients in the preceding 12 months, despite current management regimens. Symptoms were experienced for a mean of 13 ± 9 hours/day. Hospital stays or emergency department visits were required by 79% of patients. 45% reported significant interference with their lives, 85% reported an inability to perform household activities, and 20% experienced a disease-associated change in employment status. CONCLUSION: Patients with hypoparathyroidism have a high burden of illness and experience a broad spectrum of symptoms, with a multidimensional impact on their lives.