Endocrine Abstracts

Significant knowledge gaps exist regarding the humanistic effects of hypoparathyroidism, a rare, debilitating disorder. We report results from a global survey to characterise the burden of hypoparathyroidism from the patient perspective. An anonymous survey was conducted in patients with uncontrolled chronic hypoparathyroidism (as determined by patients’ report of persistent symptoms and/or poorly controlled calcium levels as told by their physician) despite receiving con...

PARADIGHM™ is a global registry (NCT01922440) of patients diagnosed with hypoparathyroidism (HPT) ≥6 months regardless of aetiology and management. Routine medical care data were entered using electronic case report forms; the 36-item Short Form Health Survey was completed by patients. Baseline-recorded data are reported for 492 patients enrolled as of 1 December 2016 from 41 centres. At baseline, 78% were women, mean (S.D.) age was 49 (17)...

Although chronic hypoparathyroidism is known to negatively affect health-related quality of life (HRQoL), burden of illness is less well defined in patients whose disease is not adequately controlled on conventional therapy. We report burden of illness for the European subset of a patient survey in 13 countries globally. 398 adults with chronic hypoparathyroidism not adequately controlled on conventional therapy (recruited by treating physician or patient association) complete...

Significant knowledge gaps exist regarding the humanistic effects of chronic hypoparathyroidism (HPT), a rare, debilitating condition. We provide interim results from a global survey conducted to characterise the burden associated with chronic HPT from the patient perspective. An anonymous, self-reported survey (online or paper) was conducted in patients with chronic HPT, not adequately controlled with standard therapy by self-perception, from 12 countries through physicians o...

Background: RACE is an open-label study that assessed the long-term safety and efficacy of recombinant human parathyroid hormone 1–84 (rhPTH[1–84]) for the treatment of hypoparathyroidism in adults (ClinicalTrials.gov identifier NCT01297309). Here, we present 6-year safety and efficacy data.Methods: Patients initially received 25 or 50 μg of rhPTH(1–84) subcutaneously, once daily, with stepwise dose adjustments of 25 μg (up or do...

PARADIGHM™, a global, prospective, observational registry of patients with chronic hypoparathyroidism (HPT), began enrolment in 2013 to collect data on the natural history of chronic HPT (ClinicalTrials.gov NCT01922440). Since initiation, recombinant human PTH, rhPTH(1-84), has been approved in the United States and Europe as an adjunctive treatment for adult patients with HPT. The protocol for the registry (now a European Medicines Agency–designated postm...

PARADIGHM is an actively recruiting, prospective, observational registry (NCT01922440/EUPAS16927). The primary objective is to evaluate the safety and effectiveness of recombinant human parathyroid hormone, rhPTH(1-84), treatment in patients with chronic hypoparathyroidism under routine clinical care. The secondary objective is tocharacterize the clinical course of chronic hypoparathyroidism under conditions of routine clinical practice. At enrollment, registry inclusion crite...