Charoenngam Nipith, Bove-Fenderson Erin, Wong Daniel, Cusano Natalie E, Mannstadt Michael
Endocrine Unit, Massachusetts General Hospital, Harvard Medical School, Boston, MA 02114, USA.
Sutter Health, Sacramento, CA 95816, USA.
J Endocr Soc. 2024 Mar 29;8(5):bvae053. doi: 10.1210/jendso/bvae053. eCollection 2024 Mar 12.
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.
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.
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.
对于接受常规治疗(钙和活性维生素D)或每日注射重组人甲状旁腺激素(rhPTH)(1-84)或rhPTH(1-34)后仍有症状或高钙尿症的慢性甲状旁腺功能减退患者,有人提出通过泵持续皮下输注rhPTH作为一种治疗选择。然而,这种新疗法疗效的真实世界证据有限。
我们报告了来自美国3个不同临床地点的12例甲状旁腺功能减退成人患者(11名女性,年龄30 - 70岁,1名男性,年龄30岁)的临床和生化结果。这些患者从常规治疗过渡到每日注射rhPTH(1-84)或rhPTH(1-34),然后改为通过泵疗法持续输注rhPTH(1-84)/rhPTH(1-34)。在大多数患者中,与常规治疗(11例患者)和单次/多次每日rhPTH注射(8例患者)相比,接受PTH泵治疗时平均血清钙浓度升高。尽管如此,与先前治疗相比,10例患者在接受PTH泵治疗时24小时尿钙中位数水平较低(平均±标准差差异:-130±222mg/24小时)。所有患者在接受泵治疗时均报告低钙血症症状有定性改善。3例患者至少有一次泵故障,1例患者出现输注部位反应。
在这个通过泵给予rhPTH(1-84)/rhPTH(1-34)治疗的12例慢性甲状旁腺功能减退患者的病例系列中,大多数患者的临床和生化参数有改善。我们的观察结果表明泵给予rhPTH有好处,值得进一步研究。