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, I2 = 0 %). Also, the PFS and RFS were 69 % and 53 % respectively (PFS: 69 %, 95 %CI 42 %, 96 %; Q = 8.63, P = 0.01, I2 = 73.07 %; RFS: 53 %, 95 %CI 27 %, 79 %; Q = 12.33, P = 0.01, I2 = 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.