Nabizadeh Fardin, Masrouri Soroush, Sharifkazemi Hossein, Azami Mobin, Nikfarjam Mahsa, Moghadasi Abdorreza Naser
Neuroscience Research Group (NRG), Universal Scientific Education and Research Network (USERN), Tehran, Iran; School of Medicine, Iran University of Medical Sciences, Tehran, Iran.
School of Medicine, Shahid Beheshti University of Medical Science, Tehran, Iran.
J Clin Neurosci. 2022 Nov;105:37-44. doi: 10.1016/j.jocn.2022.08.020. Epub 2022 Sep 6.
Treatment options for neuromyelitis optica spectrum disorder (NMOSD) are corticosteroids, immunosuppressive drugs, emerging monoclonal antibodies, rituximab, eculizumab, satralizumab, and inebilizumab. Due to disabling and deadly nature of NMOSD, there is a great motivation among physicians for finding new treatment options. Recently, several studies have been conducted on the therapeutic effects of autologous hematopoietic stem cell transplantation (AHSCT) on NMOSD patients.
Several databases including PubMed, Scopus, Web of Science, and Google scholar were searched for studies on AHSCT in NMOSD patients.
After screening titles and abstracts, and reviewing full texts, nine studies with 39 severe cases of NMOSD met the criteria of our study. The pooled standardized mean difference (SMD) for EDSS score before and after treatment was -0.81 (95 %CI:-1.07, -0.15; Q = 1.99, P = 0.58, I = 0 %). Also, the PFS and RFS were 69 % and 53 % respectively (PFS: 69 %, 95 %CI 42 %, 96 %; Q = 8.63, P = 0.01, I = 73.07 %; RFS: 53 %, 95 %CI 27 %, 79 %; Q = 12.33, P = 0.01, I = 71.87 %). Also, there were three cases with secondary autoimmune diseases including myasthenia gravis, hyperthyroidism, and thyroiditis.
According to the present study, AHSCT could be an alternative therapy for NMOSD in severe cases instead of conventional immunotherapies. However, physicians should pay attention to its serious complications. The diversity of results from the published trials on the efficacy and safety of AHSCT calls for further investigations on determining the ideal AHSCT conditioning and the characteristics of patients.
视神经脊髓炎谱系障碍（NMOSD）的治疗选择包括皮质类固醇、免疫抑制药物、新兴的单克隆抗体、利妥昔单抗、依库珠单抗、萨特利珠单抗和依奈利珠单抗。由于NMOSD具有致残性和致命性，医生们有很大的动力去寻找新的治疗方案。最近，已经针对自体造血干细胞移植（AHSCT）对NMOSD患者的治疗效果开展了多项研究。
检索了包括PubMed、Scopus、科学网和谷歌学术在内的多个数据库，以查找关于AHSCT治疗NMOSD患者的研究。
在筛选标题和摘要并审阅全文后，9项涉及39例重度NMOSD病例的研究符合我们的研究标准。治疗前后扩展残疾状态量表（EDSS）评分的合并标准化均数差（SMD）为-0.81（95%置信区间：-1.07，-0.15；Q = 1.99，P = 0.58，I² = 0%）。此外，无进展生存期（PFS）和复发自由生存期（RFS）分别为69%和53%（PFS：69%，95%置信区间42%，96%；Q = 8.63，P = 0.01，I² = 73.07%；RFS：53%，95%置信区间27%，79%；Q = 12.33，P = 0.01，I² = 71.87%）。另外，有3例出现继发性自身免疫性疾病，包括重症肌无力、甲状腺功能亢进和甲状腺炎。
根据本研究，对于重度NMOSD病例，AHSCT可作为传统免疫疗法之外的一种替代治疗方法。然而，医生应注意其严重并发症。已发表的关于AHSCT疗效和安全性试验结果的多样性，要求进一步开展研究以确定理想的AHSCT预处理方案和患者特征。
