Chronic hypoparathyroidism (HypoPT) is a rare disease associated with a variety of symptoms and diminished health-related quality of life (HRQoL). This study aimed to characterise and quantify the symptom burden, HRQoL, and overall disease impact in patients receiving recombinant human parathyroid hormone (rhPTH1-84, Natpar®) for inadequately controlled HypoPT and in symptomatic patients receiving standard therapy (ST; calcium and/or vitamin D supplements). A web-based patient survey was developed with input from members of the US Hypoparathyroidism Association and from physician experts. Eligibility criteria included receiving rhPTH1-84 or having symptoms of HypoPT while on ST. All respondents were ≥18 years old, US residents, diagnosed with HypoPT, and currently taking prescription and/or over-the-counter therapies to manage their condition. The surveys were completed at one point in time and focused on patient characteristics, recall of symptom burden from prior to and while taking their current treatment, impact of HypoPT on life and work, and HRQoL evaluated by SF-36 v2. 90 patients (mean age, 54 years; women, 83%) with HypoPT (mean duration, 8.8 years) currently on rhPTH1-84 (mean duration of therapy, 19 months) and 47 patients (mean age, 50 years; women, 93%) with HypoPT (mean duration, 16.3 years) currently symptomatic on ST completed the survey. Patients on rhPTH1-84 experienced an average of 9.1 symptoms (range, 034) and patients on ST experienced an average of 20.2 symptoms (range, 1-39) over a recall period of up to 12 months. Patients currently on rhPTH1-84 recalled experiencing an average of 17.0 symptoms (range, 340) prior to rhPTH1-84 therapy. A numerically greater proportion of patients on ST reported significant disease-associated interference with their lives (49%) and impact on work responsibilities (31%) versus patients on rhPTH1-84 (27% and 15%, respectively). rhPTH1-84 patients scored numerically higher on HRQoL (SF-36v2 domain scores range, 44.8-49.8) compared with ST patients (range, 33.940.9). HypoPT is associated with significant symptom burden. Patients on rhPTH1-84 recalled a reduction in symptoms after starting therapy, while most patients on ST reported minimal improvement of their HypoPT-related symptoms. Patients on rhPTH1-84 reported numerically higher physical and mental domain scores as measured by SF-36v2 (higher score indicating better HRQoL) compared with ST patients. This cross-sectional real-world non-interventional study does not control for unobserved treatment selection bias and underlying clinical differences that may impact treatment effectiveness between treatment groups. Retrospective reporting of baseline symptoms and other questions, which depend on patient recall represents an additional limitation.
19 - 22 May 2018
European Society of Endocrinology