Abstract

The effect of HDIT + AHSCT with low-intensity conditioning regimens in patients with various types of multiple sclerosis (MS) in terms of clinical and patient-reported outcomes was studied. In total, 418 patients with relapsing-remitting (RRMS) and secondary progressive MS (SPMS) were enrolled in a single-center study from October 2006 to October 2018. Median follow-up was 29.8 months. Outcomes of AHSCT were evaluated both from physician’s and patient’s perspective at 3, 6, 12 months after AHSCT and at long-term follow-up. EDSS changes, proportion of patients who achieved NEDA-3, event-free survival (EFS), safety, and quality of life (QoL) changes were evaluated separately in patients with RRMS and SPMS. Paired t-test, Wilcoxon test and Generalized Estimating Equations and were used for comparisons. Kaplan-Meyer method was used to evaluate EFS in terms of relapse-free survival (RFS) and progression-free survival (PFS) after AHSCT. Good tolerability of transplantation procedure was demonstrated in both patient groups. There were no cases of transplantation-related mortality. Response to treatment was achieved in the vast majority of patients. Significant improvement in disability for the entire group at all time-points after transplantation as compared with baseline was observed. The EDSS score improved in 32% and 17% of RRMS patients and in 32% and 36% SPMS patients, at 2 years and 4 years, respectively. At follow-up of 12 months postransplant, 94.6% RRMS patients and 85.6% SPMS patients achieved NEDA-3. At 7-year follow-up after AHSCT the estimated RFS in RRMS were 83%; PFS in SPMS was 77%. No differences in EFS were found according to conditioning regimens in both RRMS and SPMS. EFS in RRMS and SPMS was similar in the subgroups of patients depending on age and disease duration. RFS was dramatically better in patients with EDSS < 4 as compared to patients with EDSS ≥ 4 in RRMS patients; no differences were shown for PFS in SPMS patients depending on EDSS. In terms of patient’s perspective AHSCT resulted in significant and sustained improvement of patient’s QoL both in RRMS and SPMS. The results obtained point to feasibility of AHSCT with low-intensity conditioning regimens in RRMS and SPMS patients. Multicenter cooperative studies are worthy to optimize the protocol of AHSCT with low-intensity conditioning regimens in patients with MS.

Keywords: Autologous Hematopoietic Stem Cell Transplantation; Low-Intensity Conditioning Regimens; Relapsing-Remitting Multiple Sclerosis; Secondary Progressive Multiple Sclerosis; Quality of Life

References

Citation

Citation: Denis A Fedorenko., et al. “Сlinical and Patient-Reported Outcomes of Autologous Hematopoietic Stem Cell Transplantation (AHSCT) In Patients with Multiple Sclerosis: Single Center Experience". Acta Scientific Neurology 5.5 (2022): 09-19.

Copyright

Copyright: © 2022 Denis A Fedorenko., et al. This is an open-access article distributed under the terms of the Creative Commons Attribution License, which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.