Autologous hematopoietic stem cell transplantation with low-intensity conditioning regimens in relapsing remitting multiple sclerosis: Clinical outcomes and quality of life

Abstract

The effect of autologous hematopoietic stem cell transplantation (AHSCT) with low-intensity conditioning regimens, in terms of clinical and patient-reported out-comes, was studied in patients with relapsing-remitting multiple sclerosis (RRMS). In total, 258 RRMS patients were enrolled in a single-center study. The median fol-low-up duration was 30 months. Low-intensity conditioning regimens (two based on reduced BEAM and one on cyclophosphamide) were applied. Outcomes of AHSCT were evaluated from both the physicians’ and patients’ perspectives. Reversal of the disability progres-sion, relapse-free survival (RFS), progression-free survival (PFS), as well as changes in quality of life (QoL), and severity of symptoms were analyzed. Transplantation procedure was well tolerated by the patients, and there were no cases of transplantation-related mortality. In addition, no deaths were registered throughout the follow-up period. The vast majority of patients exhibited clinical improve-ment, or were in stable condition during the entire fol-low-up period. The estimated proportions of RFS and PFS were 83% and 86%, respectively, at 7 years after AHSCT. No differences in RFS were found between the patients who received reduced BEAM±ATG and high-dose cyclophosphamide+rituximab conditioning regi-mens. AHSCT resulted in significant and sustained QoL improvement, as well as decrease of symptom burden. The results of our study support feasibility of autolo-gous HSCT with low-intensity conditioning regimens in RRMS. Multicentre cooperative studies should be done to optimize the treatment protocol of mini-AHSCT.