Biomed Pap Med Fac Univ Palacky Olomouc Czech Repub. 2024, 168(1):50-54 | DOI: 10.5507/bp.2023.023

Immunoablative therapy followed by autologous hematopoietic stem cell transplantation as the first-line disease-modifying therapy in patients with multiple sclerosis

Martin Lachnit1, Kamila Zondra Revendova2, 3, Pavel Hradilek2, 3, Radovan Bunganic2, 3, Zdenek Koristek1, 4, Tomas Jelinek1, 4, Monika Skutova2, Radim Piza2, Ondrej Volny2, 3, Roman Hajek1, 4, Michal Bar2, 3
1 Department of Hematooncology, University Hospital Ostrava, Czech Republic
2 Department of Neurology, University Hospital Ostrava, Czech Republic
3 Department of Clinical Neurosciences, Faculty of Medicine, University of Ostrava, Czech Republic
4 Department of Hematooncology, Faculty of Medicine, University of Ostrava, Czech Republic

Introduction: Immunoablative therapy followed by autologous hematopoietic stem cell transplantation (AHSCT) is one of the possible disease-modifying therapies (DMTs) for patients with multiple sclerosis (MS). In this case series, we would like to present six patients with MS, who underwent AHSCT as the first-line DMT.

Case reports: Six MS patients with a rapid progression of disability with or without relapses underwent AHSCT as the first-line DMT at the University Hospital Ostrava between 2018 and 2021. The conditioning regimens for AHSCT used were a medium-intensity regime BEAM (Carmustine, Etoposid, Cytarabin, Melphalan) and low-intensity regime based on Cyclophosphamide. Four out of six patients showed some disability progression after AHSCT, so the rapid progression of MS was just slowed down by AHSCT. One patient developed activity on magnetic resonance imaging three months after AHSCT, and two experienced mild relapses during the follow-up period. None of our patients developed grade 4 non-hematological toxicity; all infections were mild. In one patient, an allergic reaction probably to dimethyl sulfoxide was observed.

Conclusion: Our case series of 6 patients shows that AHSCT is a promising therapeutic approach to slow down the rapid progression of clinical disability in MS patients with a good safety profile.

Keywords: multiple sclerosis, immunoablative therapy, autologous hematopoietic stem cell transplantation

Received: February 12, 2023; Revised: May 25, 2023; Accepted: May 26, 2023; Prepublished online: June 16, 2023; Published: March 12, 2024  Show citation

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Lachnit, M., Revendova, K.Z., Hradilek, P., Bunganic, R., Koristek, Z., Jelinek, T., ... Bar, M. (2024). Immunoablative therapy followed by autologous hematopoietic stem cell transplantation as the first-line disease-modifying therapy in patients with multiple sclerosis. Biomedical papers, 168(1), 50-54. doi: 10.5507/bp.2023.023
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    References

    1. Montalban X, Gold R, Thompson AJ, Otero-Romero S, Amato MP, Chandraratna D, Clanet M, Comi G, Derfuss T, Fazekas F, Hartung HP, Havrdova E, Hemmer B, Kappos L, Liblau R, Lubetzki C, Marcus E, Miller DH, Olsson T, Pilling S, Selmaj K, Siva A, Sorensen PS, Sormani MP, Thalheim C, Wiendl H, Zipp F. ECTRIMS/EAN Guideline on the pharmacological treatment of people with multiple sclerosis. Mult Scler 2018;24(2):96-120. Go to original source...
    2. Piehl F. Current and emerging disease-modulatory therapies and treatment targets for multiple sclerosis. Intern Med 2021;289(6):771-91. Go to original source...
    3. Havrdova E, Pitha J, Nytrova P, Liba Z. Česká neurologická společnost ČLS JEP. KKNEU0031 - Klinický doporučený postup pro diagnostiku a léčbu roztroušené sklerózy a neuromyelitis optica a onemocnění jejího širšího spektra, verze 2.0. 2020 Mar. Available from: https://www.czech-neuro.cz/content/uploads/2020/04/rs_odborna-2.0_final_pub_ web-2.pdf. (In Czech)
    4. Arrambide G, Iacobaeus E, Amato MP, Derfuss T, Vukusic S, Hemmer B, Brundin L, Tintore M; 2018 ECTRIMS Focused Workshop Group. Aggressive multiple sclerosis (2): Treatment. Mult Scler 2020;26(9):1045-63. Go to original source...
    5. Hauser SL, Bar-Or A, Comi G, Giovannoni G, Hartung HP, Hemmer B, Lublin F, Montalban X, Rammohan KW, Selmaj K, Traboulsee A, Wolinsky JS, Arnold DL, Klingelschmitt G, Masterman D, Fontoura P, Belachew S, Chin P, Mairon N, Garren H, Kappos L; OPERA I and OPERA II Clinical Investigators. Ocrelizumab versus Interferon Beta-1a in Relapsing Multiple Sclerosis. N Engl J Med 2017;376(3):221-34. Go to original source...
    6. Gärtner J, Hauser SL, Bar-Or A, Montalban X, Cohen JA, Cross AH, Deiva K, Ganjgahi H, Häring DA, Li B, Pingili R, Ramanathan K, Su W, Willi R, Kieseier B, Kappos L. Efficacy and safety of ofatumumab in recently diagnosed, treatment-naive patients with multiple sclerosis: Results from ASCLEPIOS I and II. Mult Scler 2022;28(10):1562-75. Go to original source...
    7. Das J, Snowden JA, Burman J, Freedman MS, Atkins H, Bowman M, Burt RK, Saccardi R, Innocenti C, Mistry S, Laud PJ, Jessop H, Sharrack B. Autologous haematopoietic stem cell transplantation as a first-line disease-modifying therapy in patients with 'aggressive' multiple sclerosis. Mult Scler 2021;27(8):1198-204. Go to original source...
    8. Sormani MP, Muraro PA, Schiavetti I, Signori A, Laroni A, Saccardi R, Mancardi GL. Autologous hematopoietic stem cell transplantation in multiple sclerosis: A meta-analysis. Neurology 2017; 88(22):2115-22. Go to original source...
    9. Burt RK, Balabanov R, Han X, Sharrack B, Morgan A, Quigley K, Yaung K, Helenowski IB, Jovanovic B, Spahovic D, Arnautovic I, Lee DC, Benefield BC, Futterer S, Oliveira MC, Burman J. Association of nonmyeloablative hematopoietic stem cell transplantation with neurological disability in patients with relapsing-remitting multiple sclerosis. JAMA 2015;313(3):275-84. Go to original source...
    10. Carreras E, Dufour C, Mohty M, Kröger N, editors. The EBMT Handbook: Hematopoietic Stem Cell Transplantation and Cellular Therapies [Internet]. 7th ed. Cham (CH): Springer; 2019. Go to original source...
    11. Häußler V, Ufer F, Pöttgen J, Wolschke C, Friese MA, Kröger N, Heesen C, Stellmann JP. aHSCT is superior to alemtuzumab in maintaining NEDA and improving cognition in multiple sclerosis. Ann Clin Transl Neurol 2021;8(6):1269-78. Go to original source...
    12. Zhukovsky C, Sandgren S, Silfverberg T, Einarsdottir S, Tolf A, Landtblom AM, Novakova L, Axelsson M, Malmestrom C, Cherif H, Carlson K, Lycke J, Burman J. Autologous haematopoietic stem cell transplantation compared with alemtuzumab for relapsing-remitting multiple sclerosis: an observational study. J Neurol Neurosurg Psychiatry 2021;92(2):189-94. Go to original source...
    13. Boffa G, Lapucci C, Sbragia E, Varaldo R, Raiola AM, Currò D, Roccatagliata L, Capello E, Laroni A, Mikulska M, Gualandi F, Uccelli A, Angelucci E, Mancardi GL, Inglese M. Aggressive multiple sclerosis: a single-centre, real-world treatment experience with autologous haematopoietic stem cell transplantation and alemtuzumab. Eur J Neurol 2020;27(10):2047-55. Go to original source...
    14. Windrum P, Morris TC, Drake MB, Niederwieser D, Ruutu T; EBMT Chronic Leukaemia Working Party Complications Subcommittee. Variation in dimethyl sulfoxide use in stem cell transplantation: A survey of EBMT centres. Bone Marrow Transplant 2005;36(7):601-3. Go to original source...

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