Combination molecular therapies for type 1 spinal muscular atrophy
Combination molecular therapies for type 1 spinal muscular atrophy
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DOI：
10.1002/mus.27034
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发表时间：
2020-08-10
期刊：
影响因子：
3.4
通讯作者：
Veerapandiyan, Aravindhan
中科院分区：
文献类型：
--
作者：
Harada, Yohei;Rao, Vamshi K.;Veerapandiyan, Aravindhan
背景资料联合分子疗法，增加运动神经元存活蛋白脊髓性肌萎缩症1型（SMA 1）缺乏。方法回顾性分析我中心采用联合治疗SMA 1患者的经验。结果5例患儿接受了诺西那生和奥那生阿贝伐他汀（奥那生）治疗。4例患者在Onasemnogene治疗前接受nusinersen治疗。Nusinersen持续3次。肝酶明显升高导致两名患者延长皮质类固醇治疗，其中一名患者住院并进行肝活检;另外两名患者的肝酶升高较轻。1例患者先接受Onasemnogene，然后接受nusinersen。未观察到不良反应。所有患者均有所改善。结论SMA 1患者可以耐受联合分子治疗。需要进一步的研究来确定是否存在联合治疗比单药治疗更有效的情况。在Onasemnogene治疗中，可能需要延长皮质类固醇的使用和肝毒性监测。
Background Data on combining molecular therapies that increase survival motor neuron protein for spinal muscular atrophy type 1 (SMA1) is lacking. Methods This was a retrospective study describing our centers' experiences in treating SMA1 patients with combination therapy. Results Five children received nusinersen and onasemnogene abeparvovec-xioi (onasemnogene). Four were receiving nusinersen prior to onasemnogene. Nusinersen was continued in three. Marked liver enzyme elevations resulted in prolonged corticosteroid treatment in two patients with hospitalization and liver biopsy in one; milder liver enzyme elevations were noted in the other two. One patient received onasemnogene first, and then nusinersen. No adverse effects were noted. All patients improved. Conclusions Combination molecular therapy is tolerated in SMA1 patients. Further studies are needed to determine whether there are circumstances in which combination therapy would be more efficacious than either monotherapy. Prolonged corticosteroid use and liver toxicity monitoring may be necessary with onasemnogene therapy.