BEAM or cyclophosphamide in autologous haematopoietic stem cell transplantation for relapsing-remitting multiple sclerosis

The most widely used conditioning regimens in autologous haematopoietic stem cell transplantation (ASCT) for multiple sclerosis (MS) are BEAM with anti-thymocyte globulin (ATG) and high-dose cyclophosphamide with ATG (Cy/ATG). In this retrospective study, we compare efficacy and safety of these regi...

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Bibliographic Details
Published in:Bone marrow transplantation (Basingstoke) 2024-11, Vol.59 (11), p.1601-1610
Main Authors: Silfverberg, Thomas, Zjukovskaja, Christina, Noui, Yassine, Carlson, Kristina, Burman, Joachim
Format: Article
Language:English
Subjects:
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Adult
Adverse events
Antilymphocyte Serum - therapeutic use
Autografts
Autoimmune diseases
Carmustine - administration & dosage
Carmustine - therapeutic use
Cell Biology
Clinical trials
Cyclophosphamide
Cyclophosphamide - therapeutic use
Cytarabine - administration & dosage
Cytarabine - therapeutic use
Effectiveness
Electronic health records
Electronic medical records
Female
Globulins
Hematology
Hematopoietic Stem Cell Transplantation - methods
Hematopoietic stem cells
Humans
Internal Medicine
Male
Medicine
Medicine & Public Health
Melphalan - administration & dosage
Melphalan - therapeutic use
Middle Aged
Multiple sclerosis
Multiple Sclerosis, Relapsing-Remitting - therapy
Pediatrics
Pediatrik
Public Health
Retrospective Studies
Safety
Stem cell transplantation
Stem Cells
Thymocytes
Transplantation
Transplantation Conditioning - methods
Transplantation, Autologous - methods
Vidarabine - administration & dosage
Vidarabine - analogs & derivatives
Vidarabine - therapeutic use
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Summary:The most widely used conditioning regimens in autologous haematopoietic stem cell transplantation (ASCT) for multiple sclerosis (MS) are BEAM with anti-thymocyte globulin (ATG) and high-dose cyclophosphamide with ATG (Cy/ATG). In this retrospective study, we compare efficacy and safety of these regimens when used for relapsing-remitting MS. We assessed 231 patients treated in Sweden before January 1, 2020. The final cohort comprised 33 patients treated with BEAM/ATG and 141 with Cy/ATG. Prospectively collected data from the Swedish MS registry were used for efficacy, and electronic health records for procedure-related safety. The Kaplan–Meier estimate of ‘no evidence of disease activity’ (NEDA) at 5 years was 81% (CI 68–96%) with BEAM/ATG and 71% (CI 63–80%) with Cy/ATG, p  = 0.29. Severe adverse events were more common with BEAM/ATG, mean 3.1 vs 1.4 per patient, p  = 
ISSN:0268-3369
1476-5365
1476-5365
DOI:10.1038/s41409-024-02397-x