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造血干细胞移植治疗难治性慢性炎症性脱髓鞘性多发性神经病的疗效:系统评价和荟萃分析。

Efficacy of hematopoietic stem cell transplantation treatment in refractory chronic inflammatory demyelinating polyradiculoneuropathy: a systematic review and meta-analysis.

机构信息

Department of Neurology, Huashan Hospital Fudan University, Shanghai, China.

National Center for Neurological Disorders (NCND), Shanghai, China.

出版信息

Eur J Neurol. 2023 Aug;30(8):2570-2582. doi: 10.1111/ene.15857. Epub 2023 May 29.

Abstract

BACKGROUND AND PURPOSE

Treatment options for chronic inflammatory demyelinating polyneuropathy (CIDP) are intravenous immunoglobulin, plasmapheresis, corticosteroids and immunosuppressive drugs. However, a substantial proportion of patients with CIDP remain refractory to treatment and develop severe functional disability. A systematic review and a meta-analysis of the efficacy of hematopoietic stem cell transplantation (HSCT) treatment in refractory CIDP patients was performed.

METHODS

The study is based on queries in the PubMed, Cochrane Central Register of Controlled Trials, Embase, Web of Science and clinicaltrials.gov databases on 4 December 2022. Articles that met our eligibility criteria were included after screening. Patients' characteristics, treatment regime and outcome measures were extracted.

RESULTS

Eighty-nine patients in 11 studies were included. The pooled estimate of responsiveness amongst the four included studies was 87.04% (95% confidence interval 66.7%-99.5%) and the pooled estimate of freedom of all immune modulating or suppressive drugs was 80.75% (95% confidence interval 71.2%-90.2%).

CONCLUSION

This meta-analysis and systematic review suggested that HSCT can be effective in the treatment of refractory CIDP. Whilst there are risks involved, HSCT may be a beneficial and viable therapy for refractory CIDP when carefully evaluated.

摘要

背景与目的

治疗慢性炎症性脱髓鞘性多发性神经病(CIDP)的选择包括静脉注射免疫球蛋白、血浆置换、皮质类固醇和免疫抑制剂。然而,相当一部分 CIDP 患者对治疗仍然没有反应,导致严重的功能障碍。我们对造血干细胞移植(HSCT)治疗难治性 CIDP 患者的疗效进行了系统评价和荟萃分析。

方法

本研究基于 2022 年 12 月 4 日在 PubMed、Cochrane 对照试验中心注册库、Embase、Web of Science 和 clinicaltrials.gov 数据库中的查询。经过筛选,符合我们纳入标准的文章被纳入。提取了患者的特征、治疗方案和结局指标。

结果

11 项研究共纳入 89 名患者。四项纳入研究的综合应答率为 87.04%(95%置信区间 66.7%-99.5%),所有免疫调节或抑制药物的综合无效率为 80.75%(95%置信区间 71.2%-90.2%)。

结论

这项荟萃分析和系统评价表明,HSCT 可有效治疗难治性 CIDP。虽然存在风险,但 HSCT 可能是一种有益和可行的治疗难治性 CIDP 的方法,需要仔细评估。

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