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难治性重症肌无力患者孕期使用依库珠单抗:一例报告

Eculizumab during Pregnancy in a Patient with Treatment-Refractory Myasthenia Gravis: A Case Report.

作者信息

Vu Tuan, Harvey Brittany, Suresh Niraja, Farias Jerrica, Gooch Clifton

机构信息

Department of Neurology, University of South Florida, Tampa, Florida, USA.

出版信息

Case Rep Neurol. 2021 Feb 1;13(1):65-72. doi: 10.1159/000511957. eCollection 2021 Jan-Apr.

Abstract

The recombinant humanized monoclonal antibody eculizumab has been shown to be effective and well tolerated in patients with anti-acetylcholine receptor antibody-positive, treatment-refractory generalized myasthenia gravis (gMG). Myasthenia gravis (MG) often affects women of child-bearing potential. However, management can be challenging during pregnancy, and current treatment options are limited due to potential teratogenicity. Data are currently lacking on the use of eculizumab in pregnant women with gMG. This case report describes a successful pregnancy in a young woman with treatment-refractory gMG treated with eculizumab before, during, and after pregnancy. Eculizumab appeared to have a favorable benefit-risk profile in this setting, with no treatment-related adverse effects noted in either the patient or the neonate. The patient remains neurologically stable on eculizumab, which she has now been receiving for 5 years. This first report of the use of eculizumab during pregnancy in a patient with treatment-refractory gMG suggests a potential role for eculizumab in this setting, although further clinical experience is necessary to support its use during pregnancy in women with MG.

摘要

重组人源化单克隆抗体依库珠单抗已被证明在抗乙酰胆碱受体抗体阳性、治疗难治性全身型重症肌无力(gMG)患者中有效且耐受性良好。重症肌无力(MG)常影响有生育潜力的女性。然而,孕期管理具有挑战性,且由于潜在致畸性,目前的治疗选择有限。目前缺乏依库珠单抗在gMG孕妇中应用的数据。本病例报告描述了一名年轻女性在妊娠前、妊娠期间和妊娠后接受依库珠单抗治疗难治性gMG后成功妊娠的情况。在此情况下,依库珠单抗似乎具有良好的效益风险比,患者和新生儿均未出现与治疗相关的不良反应。该患者在接受依库珠单抗治疗后神经功能保持稳定,目前已接受治疗5年。这是关于依库珠单抗在治疗难治性gMG患者孕期应用的首例报告,提示依库珠单抗在此情况下可能具有潜在作用,尽管需要更多临床经验来支持其在MG女性孕期的应用。

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