The American Society for Blood and Bone Marrow Transplantation (ASBMT) recently recommended that autologous hematopoietic cell transplantation (AHCT) be indicated for patients with treatment-refractory relapsing multiple sclerosis (MS) with a high risk of future disability. The ASBMT made this decision based on their expert panel’s evaluation of existing research; their report was recently published in Biology of Blood and Marrow Transplantation.

The panel examined 8 clinical trials, 8 retrospective studies, and 3 meta-analyses and reviews and concluded that AHCT is a safe and efficacious treatment for active relapsing MS. For this reason, the ASBMT designated this new AHCT indication as “standard of care, clinical evidence available”, which is a change from the ASBMT’s 2015 designation of AHCT for MS as a “developmental” indication. This new designation means that although results have not yet been obtained from large studies, AHCT has demonstrated efficacy.

The ASBMT advised that qualified neurologists and transplant physicians be involved in deciding the suitability of AHCT for patients with relapsing MS and determining the appropriate treatment protocols.

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Moreover, they recommended that treatment outcomes, along with baseline data, should be reported by transplant facilities within the United States and Canada to the Center for International Blood and Marrow Transplant Research. 

The authors of the ASBMT position statement noted that although the evaluated clinical and retrospective studies took a variety of forms, a control cohort was included in only 2 studies. In addition, most studies with AHCT included patients with more severe manifestations of MS. However, the trend that was observed consistently across the studies was a rapid and durable improvement in signs and symptoms. The authors considered AHCT to outperform other traditional disease-modifying therapies for this condition.

The authors indicated that AHCT is more likely to benefit patients who are younger and have short disease duration. AHCT is also more likely to be effective for more active disease compared with, for example, progressive MS that is lacking recent inflammatory activity.

The ASBMT position statement reflected the society’s endorsement of AHCT for treatment-refractory relapsing MS. The statement did not identify best treatment practices for performing AHCT and was focused on the suitability of this therapy for patients.

Disclosure: Some authors have declared affiliations with the pharmaceutical industry. Please refer to the original study for a full list of disclosures.

Reference

  1. Cohen JA, Baldassari LE, Atkins HL, et al. Autologous hematopoietic cell transplantation for treatment-refractory relapsing multiple sclerosis: position statement from the American Society for Blood and Marrow Transplantation [published online February 14, 2019]. Biol Blood Marrow Transplant. doi: 10.1016/j.bbmt.2019.02.014