Endocrine Abstracts

The Hypoparathyroidism Symptom Diary (HypoPT-SD) was developed for the daily assessment of key symptoms and impact of HypoPT as reported by patients with chronic HypoPT and includes 13-items: a 7-item Symptom subscale, 4-item Impact subscale, and single-item anxiety and depression scores. The current analysis was undertaken to confirm test-retest reliability and construct validity of HypoPT SD Symptom subscale scores using data collected during two open-label, single-arm studi...

In Europe, rhPTH(1-84) is an approved adjunctive treatment for adults with chronic hypoparathyroidism that cannot be adequately controlled with conventional therapy. Here we present data from the longest rhPTH(1-84)-treated study cohort of patients with hypoparathyroidism. In a single-centre, single-arm, phase 4 study (NCT02910466), long-term rhPTH(1-84) treatment (25, 50, 75, or 100 μg/day subcutaneously) was evaluated in adults with chronic hypoparathyroidism who mainta...

Hypoparathyroidism is a rare disorder characterised by hypocalcaemia and insufficient or undetectable parathyroid hormone (PTH). Recombinant human PTH, rhPTH(1-84), has been approved in the United States and Europe as an adjunctive treatment for adult patients with hypoparathyroidism. When hypoparathyroidism is established, long-term administration of rhPTH(1-84) is a treatment option. Thus, long-term safety and efficacy data for rhPTH(1-84) are needed. To this end, we evaluat...

Standard therapy (ST) for chronic hypoparathyroidism (HPT) includes calcium and active vitamin D supplementation, which can be associated with an increased risk of renal complications. This study compared renal function change, assessed by estimated glomerular filtration rate (eGFR) over 5 years between HPT patients receiving rhPTH1-84 as an adjunct to ST and a historical control cohort without rhPTH1-84. rhPTH1-84-treated HPT patients were selected from two single-arm, long-t...

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: Hypoparathyroidism (HP) is characterized by low serum calcium (sCa) and high serum phosphate (sP). Standard of care (SoC), active vitamin D and calcium, raises sCa and sP and increases burden of illness on HP patients by worsening hypercalciuria and the CaxP product. Parathyroid hormone (PTH)(1–84)(t1/2 ~2–3 hrs) is approved and can raise sCa and allow partial withdrawal of SoC, but does not sufficiently control urine calcium or symptoma...

BackgroundHypoparathyroidism is characterized by insufficient levels of parathyroid hormone (PTH), resulting in hypocalcemia, hyperphosphatemia, hypercalciuria, and a reduced quality of life (QoL). PTH replacement therapy should restore physiologic levels of PTH and restore downstream physiologic levels of calcitriol, promoting independence from Ca and active vitamin D supplements and normalization of QoL. TransCon PTH is an investigational long-acting p...

Background: Individuals with hypoparathyroidism often experience a range of symptoms associated with reduced health-related quality of life (HRQoL) and work productivity. Conventional therapy aims to alleviate hypocalcemia and acute symptoms but fails to restore normal parathyroid hormone (PTH) physiology or improve HRQoL. In the PaTHway trial, 79% of participants treated with TransCon PTH vs. 5% placebo (P<0.0001) met the primary efficacy endpoint (normal serum c...