甲状旁腺功能减退症成人用泵持续皮下注射 rhPTH(1-84) 和 rhPTH(1-34)

IF 3 Q2 ENDOCRINOLOGY & METABOLISM
Journal of the Endocrine Society Pub Date : 2024-03-29 eCollection Date: 2024-03-12 DOI:10.1210/jendso/bvae053
Nipith Charoenngam, Erin Bove-Fenderson, Daniel Wong, Natalie E Cusano, Michael Mannstadt
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引用次数: 0

摘要

背景:重组甲状旁腺激素(rhPTH)通过泵持续皮下注射,已被提议作为慢性甲状旁腺功能减退症患者的一种治疗替代方案,这些患者在接受常规治疗(钙剂和活性维生素D)或每日注射rhPTH(1-84)或rhPTH(1-34)后仍有症状或高钙尿症。然而,有关这种新型疗法疗效的实际证据却很有限:我们报告了来自美国 3 个不同临床机构的 12 名甲状旁腺功能减退症成人患者(11 名女性,30-70 岁;1 名男性,30 岁)的临床和生化结果。这些患者从传统疗法过渡到每日注射 rhPTH(1-84) 或 rhPTH(1-34),然后又转为通过泵疗法持续给药 rhPTH(1-84)/rhPTH(1-34) 。与传统疗法(11 例患者)和每日单次/多次注射 rhPTH(8 例患者)相比,大多数患者在接受 PTH 泵疗法期间的平均血清钙浓度均有所升高。尽管如此,与之前的疗法相比,10 名患者在接受 PTH 泵疗法期间的 24 小时尿钙浓度中位数较低(平均值± SD 差异:-130 ± 222 毫克/24 小时)。所有患者都表示,在接受泵治疗期间,低血钙症状明显减轻。三名患者至少有一次出现荚膜衰竭,一名患者出现输液部位反应:在这组 12 例慢性甲状旁腺功能减退症患者使用 rhPTH(1-84)/rhPTH(1-34) 泵给药治疗的病例中,大多数患者的临床和生化指标都有所改善。我们的观察结果表明,rhPTH泵给药的益处值得进一步研究。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Continuous Subcutaneous Delivery of rhPTH(1-84) and rhPTH(1-34) by Pump in Adults With Hypoparathyroidism.

Context: Continuous subcutaneous infusion of recombinant parathyroid hormone (rhPTH) through a pump has been proposed as a therapeutic alternative for patients with chronic hypoparathyroidism who remain symptomatic or hypercalciuric on conventional treatment (calcium and active vitamin D) or daily injections of rhPTH(1-84) or rhPTH(1-34). However, the real-world evidence of the outcome of this novel therapy is limited.

Case descriptions: We report the clinical and biochemical outcomes of 12 adults with hypoparathyroidism (11 women, age 30-70 years, and 1 man, age 30 years) from 3 different clinical sites in the United States who were transitioned from conventional therapy to daily injections of rhPTH(1-84) or rhPTH(1-34) and then switched to continuous administration of rhPTH(1-84)/rhPTH(1-34) via pump therapy. In most patients, mean serum calcium concentrations increased while on PTH pump therapy compared with both conventional therapy (in 11 patients) and single/multiple daily rhPTH injections (in 8 patients). Despite this, 10 patients had lower median 24-hour urinary calcium levels while on PTH pump therapy compared with prior therapy (mean ± SD difference: -130 ± 222 mg/24 hours). All patients reported a qualitative decrease in hypocalcemic symptoms while receiving pump therapy. Three patients had pod failure at least once, and 1 patient developed an infusion site reaction.

Conclusion: In this case series of 12 patients with chronic hypoparathyroidism treated with rhPTH(1-84)/rhPTH(1-34) administered via a pump, improvement in clinical and biochemical parameters were observed in the majority of the patients. Our observations indicate benefits of pump administration of rhPTH that warrant further investigation.

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来源期刊
Journal of the Endocrine Society
Journal of the Endocrine Society Medicine-Endocrinology, Diabetes and Metabolism
CiteScore
5.50
自引率
0.00%
发文量
2039
审稿时长
9 weeks
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