Autologous hematopoietic stem cell transplantation for relapsing-remitting multiple sclerosis: A single-center institutional care protocol

Authors

DOI:

https://doi.org/10.46765/2675-374X.2025v7n1e322

Keywords:

Multiple sclerosis, Stem cell transplantation, Clinical protocol

Abstract

Relapsing-remitting multiple sclerosis (RRMS) may remain clinically and radiologically active despite sequential exposure to high-efficacy disease-modifying therapies. In carefully selected patients with highly active inflammatory RRMS, autologous hematopoietic stem cell transplantation (AHSCT) has demonstrated superior inflammatory control and durable rates of no evidence of disease activity, with low treatment-related mortality rates in experienced centers. AHSCT promotes immune renewal through high-dose immunoablation followed by autologous stem cell rescue, aiming to restore immune tolerance. Here, we describe a structured institutional standard-of-care protocol for AHSCT in RRMS, developed to support safe referrals and harmonize neurology-hematology collaboration within a Brazilian tertiary center. By consolidating a transparent and safety-focused care pathway aligned with national and international guidance, this manuscript aims to facilitate standardized patient selection, optimize risk mitigation, and strengthen multidisciplinary communication between referring neurologists and transplant teams.

References

1. Walton C, King R, Rechtman L, Kaye W, Leray E, Marrie RA, et al. Rising prevalence of multiple sclerosis worldwide: insights from the Atlas of MS, third edition. Mult Scler. 2020;26(14):1816-21. https://doi.org/10.1177/1352458520970841 DOI: https://doi.org/10.1177/1352458520970841

2. Kvistad CE, Lehmann AK, Kvistad SAS, Holmøy T, Lorentzen ÅR, Trovik LH, et al. Autologous hematopoietic stem cell transplantation for multiple sclerosis: long-term follow-up data from Norway. Mult Scler. 2024; 30(6): 751-4. https://doi.org/10.1177/13524585241231665 DOI: https://doi.org/10.1177/13524585241231665

3. Muraro PA, Mariottini A, Greco R, Burman J, Iacobaeus E, Inglese M, et al. Autologous haematopoietic stem cell transplantation for treatment of multiple sclerosis and neuromyelitis optica spectrum disorder: recommendations from ECTRIMS and the EBMT. Nat Rev Neurol. 2025;21(3):140-58. https://doi.org/10.1038/s41582-024-01050-x

4. Muraro PA, Pasquini M, Atkins HL, Bowen JD, Farge D, Fassas A, et al. Autologous haematopoietic stem cell transplantation for treatment of multiple sclerosis. Nat Rev Neurol. 2017;13(7):391-405. https://doi.org/10.1038/nrneurol.2017.81 DOI: https://doi.org/10.1038/nrneurol.2017.81

5. Abrahamsson SV, Angelini DF, Dubinsky AN, Morel E, Oh U, Jones JL, et al. Non-myeloablative autologous haematopoietic stem cell transplantation expands regulatory T cells and resets the T-cell repertoire in multiple sclerosis. J Clin Invest. 2013;123(7):3084-91. https://doi.org/10.1093/brain/awt182 DOI: https://doi.org/10.1093/brain/awt182

6. Burt RK, Balabanov R, Burman J, Sharrack B, Snowden JA, Oliveira MC, et al. Effect of nonmyeloablative hematopoietic stem cell transplantation vs continued disease-modifying therapy on disease progression in relapsing-remitting multiple sclerosis: a randomized clinical trial. JAMA. 2019;321(2):165-74. https://doi.org/10.1001/jama.2018.18743 DOI: https://doi.org/10.1001/jama.2018.18743

7. Muraro PA, Pasquini M, Atkins HL, Bowen JD, Farge D, Fassas A, et al. Long-term outcomes after autologous hematopoietic stem cell transplantation for multiple sclerosis. JAMA Neurol. 2017;74(4):459-69. https://doi.org/10.1001/jamaneurol.2016.5867 DOI: https://doi.org/10.1001/jamaneurol.2016.5867

8. Häußler V, Ufer F, Pöttgen J, Wolschke C, Friese MA, Kröger N, et al. aHSCT is superior to alemtuzumab in maintaining NEDA and improving cognition in multiple sclerosis. Ann Clin Transl Neurol. 2021; 8(6): 1269-78. https://doi.org/10.1002/acn3.51366 DOI: https://doi.org/10.1002/acn3.51366

9. Muraro PA, Mariottini A, Greco R, Burman J, Iacobaeus E, Inglese M, et al. Autologous haematopoietic stem cell transplantation in multiple sclerosis and neuromyelitis optica spectrum disorder: updated consensus recommendations. Nat Rev Neurol. 2024;21. https://doi.org/10.1038/s41582-024-01050-x DOI: https://doi.org/10.1038/s41582-024-01050-x

10. Burman J, Iacobaeus E, Svenningsson A, Lycke J, Fagius J, Andersen O, et al. Autologous haematopoietic stem cell transplantation for multiple sclerosis: a registry-based study. J Neurol Neurosurg Psychiatry. 2014;85:1046-53. https://doi.org/10.1136/jnnp-2013-307207 DOI: https://doi.org/10.1136/jnnp-2013-307207

11. Snowden JA, Saccardi R, Allez M, Ardizzone S, Arnold R, Cervera R, et al. Haematopoietic stem cell transplantation for autoimmune diseases. In: Carreras E, Dufour C, Mohty M, Kröger N, editors. The EBMT Handbook: Hematopoietic Stem Cell Transplantation and Cellular Therapies. Cham: Springer; 2019. p. 537-58. https://doi.org/10.1007/978-3-030-02278-5_56 DOI: https://doi.org/10.1007/978-3-030-02278-5_56

12. Mariottini A, Nozzoli C, Carli I, Landi F, Gigli V, Repice AM, et al. Cost and effectiveness of autologous haematopoietic stem cell transplantation and high-efficacy disease-modifying therapies in relapsingremitting multiple sclerosis. Neurol Sci. 2024;45(7):3379-87. https://doi.org/10.1007/s10072-024-07308-y DOI: https://doi.org/10.1007/s10072-024-07308-y

13. Voltarelli JC, Moraes DA, Ribeiro AAF, Oliveira MC, Rodrigues M, Brum DG, et al. Consenso brasileiro para transplante de células-tronco hematopoéticas para tratamento de doenças autoimunes. Rev Bras Hematol Hemoter. 2010;32(Suppl 1):125-35. https://doi.org/10.1590/S1516-84842010005000023 DOI: https://doi.org/10.1590/S1516-84842010005000023

14. Rodrigues MC, Hamerschlak N, de Moraes DA, Simões BP, Rodrigues M, Ribeiro AA, et al. Guidelines of the Brazilian Society of Bone Marrow Transplantation on hematopoietic stem cell transplantation as a treatment for the autoimmune diseases systemic sclerosis and multiple sclerosis. Rev Bras Hematol Hemoter. 2013;35(2):134-43. DOI: https://doi.org/10.5581/1516-8484.20130035

15. Conselho Federal de Medicina. Parecer CFM nº 9/2021: uso de transplante de medula óssea na esclerose múltipla. Brasília (DF): CFM; 2021. Available from: https://sistemas.cfm.org.br/normas/arquivos/pareceres/BR/2021/9_2021.pdf. Accessed 2025 Jan 10.

16. Sharrack B, Saccardi R, Alexander T, Badoglio M, Burman J, Farge D, et al. Autologous haematopoietic stem cell transplantation and other cellular therapy in multiple sclerosis and immune-mediated neurological diseases: updated guidelines and consensus statement. J Neurol Neurosurg Psychiatry. 2020; 91(3): 241-51. https://doi.org/10.1038/s41409-019-0684-0 DOI: https://doi.org/10.1038/s41409-019-0684-0

17. Roxby AC, Gottlieb GS, Limaye AP. Strongyloidiasis in transplant patients. Clin Infect Dis. 2009; 49(9):1411-23. https://doi.org/10.1086/630201 DOI: https://doi.org/10.1086/630201

18. Peixoto D, Prestes DP. Parasitic infections of the stem cell transplant recipient and the hematologic malignancy patient, including toxoplasmosis and strongyloidiasis. Infect Dis Clin North Am. 2019; 33(2): 567-91. https://doi.org/10.1016/j.idc.2019.02.009 DOI: https://doi.org/10.1016/j.idc.2019.02.009

19. Ljungman P, de la Camara R, Cordonnier C, Einsele H, Engelhard D, Reusser P, et al. Management of CMV, HHV-6, HHV-7 and Kaposi-sarcoma herpesvirus (HHV-8) infections in patients with hematological malignancies and after SCT. Bone Marrow Transplant. 2008;42(4):227-40. https://doi.org/10.1038/bmt.2008.162 DOI: https://doi.org/10.1038/bmt.2008.162

20. Nabizadeh F, Pirahesh K, Rafiei N, Afrashteh F, Ahmadabad MA, Zabeti A, et al. Autologous hematopoietic stem-cell transplantation in multiple sclerosis: a systematic review and meta-analysis. Neurol Ther. 2022;11(4):1553-69. https://doi.org/10.1007/s40120-022-00389-x DOI: https://doi.org/10.1007/s40120-022-00389-x

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Published

04/22/2026

How to Cite

Silva, P. D. M., Abreu, S. J. de, Vargas, A., & Vecino, M. C. A. de. (2026). Autologous hematopoietic stem cell transplantation for relapsing-remitting multiple sclerosis: A single-center institutional care protocol. JOURNAL OF BONE MARROW TRANSPLANTATION AND CELLULAR THERAPY, 7(1). https://doi.org/10.46765/2675-374X.2025v7n1e322