Chronic Hypoparathyroidism - Current and Emerging Therapies.

IF 3.7 3区医学 Q2 ENDOCRINOLOGY & METABOLISM

Endocrine Practice Pub Date : 2025-07-16 DOI:10.1016/j.eprac.2025.07.011

Sarah Khan, Aliya A Khan

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Abstract

Hypoparathyroidism (HypoPT) is a rare endocrine disorder characterized by low serum calcium due to insufficient parathyroid hormone (PTH) and results in significant complications. Conventional treatment for hypoparathyroidism with calcium salts and activated vitamin D has limitations such as inadequate control of symptomatic hypocalcemia and large pill burden. PTH replacement therapy offers a more physiologic alternative to conventional therapy. PTH (1-34) has a short half-life of 1 hour and is used off-label for the treatment of hypoparathyroidism. rhPTH (1-84) has a half life of 3 hours and improves symptom control but its production is being halted due to manufacturing issues. Palopegteriparatide has a 60-hour half life and results in an infusion like PTH release profile. This molecule is effective in lowering urinary calcium, maintaining serum eucalcemia, lowering serum phosphate and reducing pill burden and is now approved by the FDA and EMA. Emerging therapies include Eneboparatide which is currently being evaluated in phase 3 clinical trials. Encalaret is a calcilytic molecule which is in phase 3 clinical trials and is a promising molecule for autosomal dominant hypocalcemia type 1. New molecules include an oral PTH1receptor agonist which is in phase 1 clinical trials. A weekly PTH molecule is also being evaluated in phase 2 clinical trials.

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慢性甲状旁腺功能减退-当前和新兴的治疗方法。

甲状旁腺功能减退症（HypoPT）是一种罕见的内分泌疾病，其特征是由于甲状旁腺激素（PTH）不足而导致血清钙降低，并导致严重的并发症。常规治疗甲状旁腺功能减退用钙盐和活性维生素D有局限性，如对症状性低钙控制不足和药片负担大。甲状旁腺激素替代疗法提供了一种比传统疗法更具生理性的替代疗法。甲状旁腺素（1-34）的半衰期很短，只有1小时，在说明书外用于治疗甲状旁腺功能减退症。rhPTH（1-84）的半衰期为3小时，可以改善症状控制，但由于制造问题，其生产正在停止。Palopegteriparatide有60个小时的半衰期，结果是像PTH释放谱一样的输注。该分子在降低尿钙、维持血清钙、降低血清磷酸盐和减轻药片负担方面有效，现已获得FDA和EMA的批准。新兴疗法包括eneoparatide，目前正在进行3期临床试验。Encalaret是一种正处于3期临床试验的溶钙分子，是一种治疗常染色体显性低钙血症1型的有前景的分子。新分子包括一种口服pth1受体激动剂，目前正处于1期临床试验。每周一次的甲状旁腺激素分子也正在二期临床试验中进行评估。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Endocrine Practice ENDOCRINOLOGY & METABOLISM-

CiteScore

7.60

自引率

2.40%

发文量

546

审稿时长

41 days

期刊介绍： Endocrine Practice (ISSN: 1530-891X), a peer-reviewed journal published twelve times a year, is the official journal of the American Association of Clinical Endocrinologists (AACE). The primary mission of Endocrine Practice is to enhance the health care of patients with endocrine diseases through continuing education of practicing endocrinologists.