Should autologous hematopoietic stem cell transplantation be offered as a first-line disease modifying therapy to patients with multiple sclerosis?

Abstract

In multiple sclerosis (MS), progression independent of new focal inflammation may commence shortly after disease onset, and it is increasingly revealed that the risk of disability accrual is reduced by early use of high-efficacy disease-modifying therapies (HE-DMTs). People with aggressive MS may therefore benefit from early treatment with autologous haematopoietic stem cell transplantation (AHSCT), a procedure inducing maximal immunosuppression followed by immune reconstitution, demonstrated to be superior to DMTs in one randomized clinical trial. However, in current practice prior failure to HE-DMTs is typically required to establish the indication for AHSCT. In the present article, the available evidence on the potential role of AHSCT as first-line treatment in aggressive MS and the rationale for its early use will be summarized. Proposed definitions of aggressive MS that could help identifying MS patients eligible for early treatment with AHSCT will also be discussed.

Keywords: Aggressive multiple sclerosis; Disease-modifying therapy; Hematopoietic stem cell transplantation; Multiple sclerosis; Treatment-naïve.