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生长激素缺乏症成年女性应用索马鲁肽引起的可逆性脂肪萎缩。

Somapacitan-induced reversible lipoatrophy in an adult woman with hypopituitarism.

机构信息

Faculty of Medicine, University Medical Centre Ljubljana, University of Ljubljana, Zaloška cesta 7, Ljubljana, SI-1000, Slovenia.

出版信息

Pituitary. 2024 Oct;27(5):737-739. doi: 10.1007/s11102-024-01440-w. Epub 2024 Aug 9.

DOI:10.1007/s11102-024-01440-w
PMID:39120809
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11513695/
Abstract

BACKGROUND

Lipoatrophy is rare adverse event (AE) in daily recombinant human growth hormone (rhGH). Data on lipoatrophy in newly developed long-acting GH (LAGH) are scarce. We report the first case of lipoatrophy in adult patient treated with LAGH somapacitan.

CASE PRESENTATION

A 38-year-old woman with congenital panhypopituitarism was transitioned from daily rhGH 0.4 mg QD to somapacitan dose 4 mg QW due to non-adherence to daily rhGH. Despite adequate education and regular changing of injection sites, the patient reported reduced subcutaneous tissue at all four injection sites, after the 4th application of somapacitan. Somapacitan was discontinued at patient preference and lipoatrophy completely reversed after 3 months.

CONCLUSIONS

Lipoatrophy caused by somapacitan was completely reversible. We speculate that high initial dose and volume of somapacitan caused delayed diffusion and a direct local lipolytic effect in our patient. Although, titration of somapacitan was initiated as previously reported in REAL2 study protocol, recent clinical guidelines advise more gradual increase of somapacitan dose also in women on oral estogens that are switched from daily rhGH. Importantly, our case and the two previously described cases in children in the REAL 3 study showed that lipoatrophy caused by somapacitan was transient and completely reversible, and that discontinuation of the drug is not always mandatory.

摘要

背景

脂肪萎缩是重组人生长激素(rhGH)治疗中罕见的不良反应(AE)。关于新开发的长效 GH(LAGH)中脂肪萎缩的数据很少。我们报告了首例使用长效 somapacitan 治疗的成年患者脂肪萎缩病例。

病例介绍

一名 38 岁女性患有先天性全垂体功能减退症,由于不依从每日 rhGH 治疗,从每日 rhGH 0.4 mg QD 转换为 somapacitan 剂量 4 mg QW。尽管进行了充分的教育和定期更换注射部位,但在接受第 4 次 somapacitan 治疗后,患者报告所有四个注射部位的皮下组织减少。由于患者的偏好,somapacitan 已停用,3 个月后脂肪萎缩完全逆转。

结论

somapacitan 引起的脂肪萎缩是完全可逆的。我们推测,somapacitan 的初始高剂量和大体积导致其在我们患者中扩散延迟,并产生直接的局部脂肪分解作用。尽管 REAL2 研究方案中已开始进行 somapacitan 的滴定,但最近的临床指南建议在从每日 rhGH 转换为口服雌激素的女性中,更缓慢地增加 somapacitan 剂量。重要的是,我们的病例和 REAL 3 研究中之前描述的两名儿童病例表明,somapacitan 引起的脂肪萎缩是短暂的和完全可逆的,并且不一定需要停用药物。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/319d/11513695/f12596fe07b1/11102_2024_1440_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/319d/11513695/f12596fe07b1/11102_2024_1440_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/319d/11513695/f12596fe07b1/11102_2024_1440_Fig1_HTML.jpg

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本文引用的文献

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Weekly Somapacitan in GH Deficiency: 4-Year Efficacy, Safety, and Treatment/Disease Burden Results From REAL 3.每周索马帕坦治疗 GH 缺乏症:REAL 3 研究的 4 年疗效、安全性和治疗/疾病负担结果。
J Clin Endocrinol Metab. 2023 Sep 18;108(10):2569-2578. doi: 10.1210/clinem/dgad183.
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Guidance for the treatment of adult growth hormone deficiency with somapacitan, a long-acting growth hormone preparation.生长激素缺乏症成人治疗用长效生长激素制剂somapacitan 的治疗指南。
Front Endocrinol (Lausanne). 2022 Dec 23;13:1040046. doi: 10.3389/fendo.2022.1040046. eCollection 2022.
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Eur J Endocrinol. 2018 May;178(5):491-499. doi: 10.1530/EJE-17-1073. Epub 2018 Mar 2.