Autologous Stem Cell Transplant vs Disease-Modifying Therapy for Multiple Sclerosis

The availability of an increasingly wide array of disease-modifying therapies (DMTs) for multiple sclerosis (MS), including the currently more than 20 US Food and Drug Administration –approved therapies, illustrates remarkable progress in treatment of the disease. These available treatments achieve their efficacy via a variety of different mechanisms of action but also exhibit varying degrees of overall effectiveness, safety, and tolerability. Despite treatment with these ther apies, there are some people with highly active MS who experience continued relapses, new magnetic resonance imaging (MRI) lesions, or disability worsening. Immunoablation/myeloablation followed by autologous hematopoietic stem cell transplant (AHSCT) has been proposed as an option for such patients . AHSCT is a multistep procedure comprising (1) mobilization of hematopoietic stem cells from the bone marrow to the blood, typically by the administration of cyclophosphamide followed by granulocyte colony-stimulating factor; (2) harvest (collection), typically by leukapheresis and sometimes with s election of CD34+ cells, followed by cryopreservation; (3) administration of an ablative conditioning regimen to eliminate existing immune cells; and (4) reinfusion of the peripheral blood stem cell graft to shorten the aplastic phase. Each of these steps has the potential for adverse effects, somet imes severe, particularly the conditioning and transplant steps. Late complications are mostly related to viral r...
Source: JAMA Neurology - Category: Neurology Source Type: research