Endocrine Abstracts

IntroductionA high calcium-phosphate product is thought to increase the risk of soft tissue calcification in hypoparathyroidism, including cataracts. Based on small case series, cataracts in hypoparathyroidism typically develop in the posterior subcapsular region of the lens. However, the mechanism of cataract formation is still not well understood.Materials and methodsIn a large cohort of 1014 patients with ...

Chronic hypoparathyroidism is a rare mineral homeostasis disorder managed by conventional therapy (oral calcium and active vitamin D), alone or with adjunctive rhPTH(1-84). Chronic hypoparathyroidism is associated with considerable symptom burden, which can interfere with daily living. We report results from a web-based, cross-sectional survey conducted among adults with chronic hypoparathyroidism. The objectives were to quantify the impact on overall life interference of (1) ...

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)...

Hypoparathyroidism, a rare disorder characterized by absent or low levels of parathyroid hormone (PTH), often results from thyroid surgery. However, nonsurgical etiologies are present in >10% of patients. Data about this group of patients are limited.In this post hoc REPLACE (NCT00732615, EudraCT2008-005063-34) analysis, baseline characteristics and response to 50–100 μg/day rhPTH(1-84) in patients with postsurgical or nonsurgical ...

Hypoparathyroidism is a rare endocrine deficiency due to inadequate amounts of parathyroid hormone (PTH) and is characterised by hypocalcaemia and hyperphosphataemia. Current management regimens with large amounts of oral calcium and active vitamin D do not adequately control mineral homeostasis and can lead to complications in many patients. The phase III REPLACE and RELAY clinical trials established the efficacy and safety of once-daily s.c. injection of r...

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: Patients with hypoparathyroidism experience significant physical and cognitive symptoms and reduced health-related quality of life (HRQoL). Conventional therapy for hypoparathyroidism does not fully alleviate diminished HRQoL. The Hypoparathyroidism Patient Experience Scales (HPES) were developed to assess disease-specific physical and cognitive symptoms as well as the impact of hypoparathyroidsm on HRQol. TransCon PTH, an investigational long-acting prodrug of par...

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...