Vladimir Y Melnichenko¹, Tatiana I Ionova², Tatiana P Nikitina², Ilya S Nikolaev¹, Anastasia K Panchenko¹, Natalia M Porfirieva³, Anatoliy А Rukavitcyn¹ and Denis A Fedorenko¹*

¹Department of Hematology, Chemotherapy and Bone Marrow Transplantation, Pirogov National Medical and Surgical Center, Moscow, Russia
²Clinic of High Medical Technologies named after N.I. Pirogov, Saint-Petersburg State University Hospital, Saint-Petersburg, Russia
³Multinational Center for Quality of Life Research, Saint-Petersburg, Russia

*Corresponding Author:Denis A Fedorenko; Department of Hematology, Chemotherapy and Bone Marrow Transplantation, Pirogov National Medical and Surgical Center, Moscow, Russia.

Received: October 09, 2025; Published: October 27, 2025

Abstract

AHSCT is highly effective and promising treatment option for MS. We evaluated clinical outcomes and associated factors in patients with MS undergoing non-myeloablative AHSCT. Patients with various types of MS who underwent AHSCT from 2006 to 2023 were enrolled in a single-center study. Three non-myeloablative conditioning regimens were used - BEAM-based with ATG or Rituximab (25%), Cyclophosphamide with Rituximab (62%), and Fludarabine + Cyclophosphamide with Rituximab (13%). For assessment of clinical outcomes EDSS evaluation and MRI were performed, for QoL assessment SF-36 was used at baseline and at different time points after AHSCT. Totally, 898 patients with relapsing-remitting (RRMS, n = 477, 53%), primary progressive (PPMS, n = 141, 16%), and secondary progressive MS (SPMS, n = 280, 31%) were enrolled. Median age - 40 yrs, 40% were males. Mean (SD) disease duration - 6.8 (5.9) yrs. Median baseline EDSS - 4 (range 1.5-8.5). There were 6 cases of TRM: RRMS - 3, SPMS - 2, PPMS - 1; Cyclophosphamide was used in all cases. Toxicity profile was the best for Fludarabine-based conditioning. Median follow-up after AHSCT was 30 months (Q1; Q3 - 12; 54). EDSS improvement for the entire group at all-time intervals after transplantation as compared with baseline was observed (p < 0.001). At 12 months after AHSCT the decrease of EDSS in RRMS was from median 3.0 to 1.5, in SPMS - from 6.0 tо 5.0, and in PPMS - from 5.5 tо 4.5. The estimated 5-years EFS probability for the entire group was 83.6%, EFS for RRMS - 87.3%, EFS for SPMS - 80.4%, and for PPMS - 77.6%. No differences in EFS depending on conditioning regimen were found. Factors associated with AHSCT failure were - age ≥ 40, progressive MS, baseline EDSS ≥ 4, previous standard treatment, baseline physical health component score < 40 by SF-36. Three risk groups were identified: in high risk group EFS was worse than in low and in intermediate risk groups (low risk group - 89.7%, intermediate risk group - 79.3%, and high-risk group - 58.2%). AHSCT was accompanied by significant improvement in patient’s QoL. QoL dramatically improved by all SF-36 scales at 24 months after AHSCT as compared to baseline and at long term follow-up. The results demonstrate that non-myeloablative AHSCT is effective in patients with different types of MS in terms of clinical response and QoL improvement. The safest non-myeloablative conditioning is Fludarabine-based. The best candidates for AHSCT are patients aged < 40 years, with relapsing MS, treatment-naive, with baseline EDSS < 4, and with baseline physical health summary score ≥40. EFS is better in MS patients of low and intermediate risk than in patients of high risk.

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

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Citation

Citation: Denis A Fedorenko., et al. “Treatment Outcomes and Factors Associated with the Success of Autologous Hematopoietic Stem Cell Transplantation (AHSCT) in Multiple Sclerosis".Acta Scientific Neurology 8.11 (2025): 44-57.

Copyright

Copyright: © 2025 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.