Autologous Hematopoietic Stem Cell Transplantation in Multiple Sclerosis Patients: Monocentric Case Series and Systematic Review of the Literature

Abstract

Multiple sclerosis (MS) is a chronic, inflammatory and immune-mediated disease of the central nervous system (CNS), commonly affecting young adults and potentially associated with life-long disability. About 14 disease-modifying treatments (DMTs) are currently approved for the treatment of MS. However, despite the use of highly effective therapies, some patients exhibit a highly active disease with an aggressive course from onset and a higher risk of long-term disability accrual. In the last few years, several retrospective studies, clinical trials, meta-analyses and systematic reviews have investigated autologous hematopoietic stem cell transplantation (AHSCT) as a possible therapeutic option in order to address this unmet clinical need. These studies demonstrated that AHSCT is a highly efficacious and relatively safe therapeutic option for the treatment of highly active MS. Particularly, over recent years, the amount of evidence has grown, with significant improvements in the development of patient selection criteria, choice of the most suitable transplant technique and clinical experience. In this paper, we present six patients who received AHSCT in our MS center and we systematically reviewed recent evidence about the long-term efficacy and safety of AHSCT and the placement of AHSCT in the rapidly evolving therapeutic armamentarium for MS.

Autologous haematopoietic stem cell transplantation for multiple sclerosis: a position paper and registry outline

“… 1 , 2 While only two randomised controlled trials (RCT) have been completed so far, which had included 21 and 110 patients, respectively, 3 , 4 several retrospective data analyses, meta-analyses and systematic reviews have gathered data from more than 4000 AHSCTs documented in publications worldwide. 5 – 8 However, many questions about using AHSCT in MS are still unsolved. The three ongoing RCT studies, STAR-MS in the United Kingdom, BEAT-MS in the United States and RAM-MS in Norway, 9 together with the upcoming NET-MS study in Italy, aim to confirm effects in highly active relapsing-remitting MS in comparison with high-efficacy treatments such as B cell depleting and other monoclonal antibodies.…”