For patients with severe neuromyelitis optica spectrum disorder (NMOSD), autologous hematopoietic stem cell transplantation (AHSCT) may be used as an alternative to traditional immunotherapies, according to a systematic review and meta-analysis.

These data indicated that the Extended Disability Status Scale score prior to and following AHSCT had a pooled standardized mean difference of -0.81 (95% CI, -1.07 – -0.15), reported Fardin Nabizadeh, of the Iran University of Medical Sciences in Tehran, and colleagues.

Notably, they also identified 3 secondary autoimmune disease cases, which were thyroiditis, myasthenia gravis, and hyperthyroidism.

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The findings were published in the Journal of Clinical Neuroscience.

Read more about NMOSD complications

These findings contribute to the rather scarce data in the literature regarding AHSCT as a treatment for NMOSD that currently exists. Although the results of this technique can be considered overall successful, it may not be used as a first-line therapeutic option due to potentially severe complications, the authors noted.

“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, the characteristics of patients likely to enter long-term remission, and major factors involved in the effectiveness of the therapy. In addition, the physicians should pay attention to serious side effects of AHSCT,” the study authors wrote.

The researchers gathered 9 papers on AHSCT in patients with NMOSD from the Google scholar, Scopus, PubMed, and Web of Science databases. Of the 9 papers included, there was 1 clinical trial, 2 cohort studies, and 6 case reports. The investigations were conducted in Australia (n=1), Italy (n=1), Canada (n=1), Singapore (n=1), China (n=1), and the United States (n=4).

Across those studies, the investigators identified 39 patients who had severe NMOSD for an average of 108 months. Females comprised more than half of the cohort. There were 29 patients who were positive for AQP4-IgG, 5 who were negative, and 5 who had an unknown status.

Cyclophosphamide was administered to 22 patients prior to and following AHSCT therapy. During AHSCT, Patients also received medications like dexamethasone (n=1), melphalan (n=1), thiotepa (n=1), anti-thymocyte globulin (n=19),  filgrastim (n=13). granulocyte colony-stimulating factor (n=1), fludarabine (n=1), BEAM (n=10), etoposide (n=1), and rituximab (n=17).  

Other findings showed that the cohort had a 69% progression-free survival during treatment (95% CI, 42%-96%) and a 53% relapse-free survival during treatment (95% CI, 27%-79 %).

The researchers also found a number of complications. Electrolyte imbalances presented as hypophosphatemia, hypocalcemia, and hypokalemia occurred in 9, 3, and 2 cases, respectively. The rest of the adverse reactions correspond to nausea and vomiting, diarrhea, bone pain, and torso pain.

Looking ahead, investigations are needed to compare and address the mechanisms in charge of autologous and allogeneic transplantation. Other research efforts should be made to address the need for investigations in Asian countries and to explore the impact of ethnicity in responding to treatment, the researchers noted.


Nabizadeh F, Masrouri S, Sharifkazemi H, Azami M, Nikfarjam M, Moghadasi A. Autologous hematopoietic stem cell transplantation in neuromyelitis optica spectrum disorder: a systematic review and meta-analysis. J of Clin Neurosci. Published online September 6, 2022. doi:10.1016/j.jocn.2022.08.020