Efficacy and complications of autologous hematopoietic stem cell transplantation in multiple sclerosis

Abstract

Introduction. Autologous hematopoietic stem cell transplantation (AHSCT) is a therapeutic strategy with curative potential for relapsing-remitting multiple sclerosis (RRMS) [1]. AHSCT is a modern alternative to disease-modifying therapies, since a low therapeutic response has been observed in RRMS patients [6]. The action mechanism is based on the immunosuppression of autoreactive lymphocytes to subsequently remodel the immune response through self-hematopoietic stem cells [1, 3], restoring immune tolerance and suppressing the inflammatory response [2]. Materials and methods. For this study, was performed a search of the specialized scientific literature from 2019-2025, the articles were identified through the search engine PubMed, Google Scholar. Results. A prevalence of 68%-88% of patients undergoing AHSCT maintained no evidence of disease activity: NEDA-3 (no relapses, no magnetic resonance imaging activity and no MS progression) in the following 3-5 years after therapy [4, 8]. The efficacy rate is 86.9-91.3% due to the absence of clinical relapses at 5 years after transplantation [1, 2]. The success rate is higher in patients under 45-50, with an expanded disability status scale (EDSS) <5.5-6 [4], and disease duration of less than 5-10 years, thus having a higher chance of remission [1]. The mortality is 2.1% [2]. To confirm the biological efficacy, cerebrospinal fluid is extracted, and it has been observed that CXCL13 decreases in the first year and sCD27 normalizes over the next two years [6]. Complications vary depending on individual tolerance and 17% of patients suffer from Uhthoff's phenomenon in the first 60 days [4], myelosuppression in the first 100 days [7], neutropenia (58%-70%) [8], bacterial sepsis, pneumonia, urinary tract infections, hemorrhagic cystitis, dyspeptic disorders and venous thrombosis [4]. Autoimmune thyroiditis or immune thrombocytopenia occur in 4-6%, Epstein-Barr virus and cytomegalovirus are reactivated in 80% of cases, and there have been recorded oncological risks such as breast cancer, glioblastoma, prostate cancer post-AHSCT [4]. Arrhythmias, renal failure and infertility are found in a minor percentage [5, 7]. Conclusions. With over 80% efficacy, AHSCT is the most effective treatment for RRMS, and despite adverse reactions, this therapy offers optimal therapeutic prospects for patients with active forms of MS.