Endocrine Abstracts

Chronic hypoparathyroidism affects ~2.5/10 000 individuals in the European Union and is classified as a rare disorder. There is limited knowledge of the natural history and epidemiology of this disease. Publications frequently represent a single center’s experience and information across the population of patients with hypoparathyroidism is lacking and needed. To fill the gap, a voluntary global hypoparathyroidism patient registry (PARADIGHM) was initiated by NPS Pharmaceuticals, Inc. The goal is to characterize the epidemiology, natural history, and clinical features of hypoparathyroidism globally and long term.

PARADIGHM is a global, prospective, observational registry based on clinical practices. Patients of any age who have hypoparathyroidism are eligible for inclusion, regardless of etiology or treatment received. Recruitment is planned for 7 years with minimum follow-up of 10 years. Target sample size is 900. Patient enrollment is voluntary through physicians qualifying as investigator sites. Data collected include i) hypoparathyroidism-relevant clinical features and treatment history extracted from medical records, ii) patient-reported outcomes and healthcare use, and iii) mortality.

Registry enrollment began in the USA in July 2013 and is targeted to begin in the European Union in 2014. In addition to baseline demographics, family and medical history, and hypoparathyroidism characteristics and treatment, we expect to report changing features of the disease during follow-up. This includes patients’ signs, symptoms, and medical test results; management practices and disease control; risks of clinical events (eg, hypercalcemia or organ impairment); patient quality of life; disease impact on work; hospitalization and emergency room use; and mortality.

PARADIGHM™ will provide previously unavailable long-term, prospective data from a large sample of patients with hypoparathyroidism. Findings will assist providers in clinical decision making through enhanced understanding of the variability and progression of hypoparathyroidism.