A Mexican research team has performed a literature review on the use of autologous hematopoietic stem cell transplantation (auto-HSCT) to treat patients with several immune-mediated neurological diseases, including neuromyelitis optica spectrum disorder (NMOSD). They reported that of the 42 NMOSD patients included, 24 were relapse-free at last follow-up and 13 had negative anti-AQ4 antibodies after HSCT.

“In this review, we included a total of 173 patients with uncommon neuroimmune diseases who underwent HSCT. From the total sample, 76.8% of the patients experienced an overall response (133/173),” the researchers explained. “This rate is important, considering that most included patients were refractory to more than three immunotherapies, including intravenous immunoglobulin and plasma exchange.”

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The review is published in Cytotherapy. The authors identified 11 published studies specifically reporting on HSCT in 42 patients with NMOSD. Thirty-eight patients underwent auto-HCST and eight received allo-HSCT, and the conditioning regimens varied substantially between studies.

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However, almost all patients underwent a reduced-intensity conditioning regimen including cyclophosphamide (Cy) and anti-thymocyte globulin (ATG). The use of ATG in NMOSD is important because it inhibits B and T cell interactions, which is a key component of the pathogenesis of NMOSD. Some 100 patients also received rituximab, a B-cell depleting compound that has demonstrated improved expanded disability scale scores and lower rates of relapse among patients with NMOSD.

In addition to the general improvements observed in the included patients, no serious complications from HSCT were noted. Although the patient numbers were smaller than those for studies on HSCT in multiple sclerosis—in which good results have also been obtained—the results here highlight the efficacy of HSCT in the treatment of several severe, refractory immune neurological diseases, also including chronic inflammatory demyelinating polyneuropathy, stiff-person syndrome, and severe refractory myasthenia gravis.

The authors recommend inter-center collaboration to determine the optimal patient profile for achieving a long-term response to this approach.


Jaime-Pérez JC, Jesús, Meléndez-Flores D, Ramos-Dávila EM, et al. Hematopoietic stem cell transplantation for uncommon immune-mediated neurological disorders: a literature review. Cytotherapy. Published online February 19, 2022. doi: doi.org/10.1016/j.jcyt.2021.12.006