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Updated: Nov 20, 2025
Author Spotlight: Exploring the Lifespan Dynamics of Healthy Human Hematopoiesis
Published on: December 8, 2023
Long-term Clinical Outcomes of Hematopoietic Stem Cell Transplantation in Multiple Sclerosis
Giacomo Boffa1, Luca Massacesi1, Matilde Inglese2
1From the Department of Neurology, Rehabilitation, Ophthalmology, Genetics, Maternal and Child Health (G.B., M.I., A.U., G.M.) and Biostatistics Unit (A.S., M.P.S.), University of Genoa; San Martino Hospital (G.B.), Genoa; Department of Neurosciences Drugs (L. Massacesi, A.M., A.M.R.), Child Health and Department of Neurology 2 (L. Massacesi, A.M., A.M.R.), and Cell Therapy and Transfusion Medicine Unit (C.I., R. Saccardi), Careggi University Hospital, Florence; Ospedale Policlinico San Martino (M.I., A.U., G.M.), IRCCS, Genoa; Department of Neurology (M.C., A.B.), San Luigi Gonzaga Hospital, Orbassano; Department of Neurology (L. Moiola, G.C.) and Department of Haematology and Bone Marrow Transplant (R.G., F.C.), Vita-Salute San Raffaele University, San Raffaele Scientific Institute, Milan; Department NEUROFARBA (M.P.A.), Section Neurological Sciences, University of Florence IRCCS Fondazione Don Carlo Gnocchi; Department of Neurology (S.C.) and Department of Haematology (R. Scimè), Villa Sofia Hospital, Palermo; Department of Haematology and Bone Marrow Transplant Unit (F.G., E.A.), Policlinico San Martino IRCCS, Genoa; Department of Clinical and Biological Sciences (M.D.G.), Haematopoietic Stem Cell Transplant Unit, University of Turin, San Luigi Gonzaga Hospital, Orbassano; Multiple Sclerosis Center (J.F.), Department of Medical Sciences and Public Health University of Cagliari; Binaghi Hospital (J.F.), Cagliari; Department of Neurology (G.B.Z.), Ospedale Generale Regionale "F. Miulli," Acquaviva delle Fonti, BA; and IRCCS Scientific Clinical Institutes Maugeri (G.M.), Pavia-Genoa Nervi, Italy.
Objective:
To determine whether autologous hematopoietic stem cell transplantation (aHSCT) is able to induce durable disease remission in people with multiple sclerosis (MS), we analyzed the long-term outcomes after transplantation in a large cohort of patients with MS.
Methods:
To be included, a minimum dataset (consisting of age, MS phenotype, Expanded Disability Status Scale [EDSS] score at baseline, information on transplantation technology, and at least 1 follow-up visit after transplantation) was required.
Results:
Two hundred ten patients were included (relapsing-remitting [RR] MS 122 [58%]). Median baseline EDSS score was 6 (1-9); mean follow-up was 6.2 (±5.0) years. Among patients with RRMS, disability worsening-free survival (95% confidence interval [CI]) was 85.5% (76.9%-94.1%) at 5 years and 71.3% (57.8%-84.8%) at 10 years. In patients with progressive MS, disability worsening-free survival was 71.0% (59.4%-82.6%) and 57.2% (41.8%-72.7%) at 5 and 10 years, respectively. In patients with RRMS, EDSS significantly reduced after aHSCT (p = 0.001; mean EDSS change per year -0.09 [95% CI -0.15% to -0.04%]). In patients with RRMS, the use of the BCNU+Etoposide+Ara-C+Melphalan (BEAM) + anti-thymocyte globulin (ATG) conditioning protocol was independently associated with a reduced risk of no evidence of disease activity 3 failure (hazard ratio 0.27 [95% CI 0.14-0.50], p < 0.001). Three patients died within 100 days from aHSCT (1.4%); no deaths occurred in patients transplanted after 2007.
Conclusions:
aHSCT prevents disability worsening in the majority of patients and induces durable improvement in disability in patients with RRMS. The BEAM + ATG conditioning protocol is associated with a more pronounced suppression of clinical relapses and MRI inflammatory activity.
Classification Of Evidence:
This study provides Class IV evidence that for people with MS, aHSCT induces durable disease remission in most patients.
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