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Autoimmune Disorders

Autologous hematopoietic stem cell transplantation for pediatric multiple sclerosis: a registry-based study of the Autoimmune Diseases Working Party (ADWP) and Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT)

Bone Marrow Transplantation volume 52, pages 1133–1137 (2017)Cite this article

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Abstract

Autologous hematopoietic stem cell transplantation (aHSCT) is a promising therapy for multiple sclerosis (MS), which has mainly been used in adults. The purpose of this study was to investigate efficacy and adverse events of aHSCT in the treatment of children with MS using data from the European Society for Blood and Marrow Transplantation registry. Twenty-one patients with a median follow-up time of 2.8 years could be identified. PFS at 3 years was 100%, 16 patients improved in expanded disability status scale score and only 2 patients experienced a clinical relapse. The procedure was generally well tolerated and only two instances of severe transplant-related toxicity were recorded. There was no treatment-related mortality, although one patient needed intensive care. aHSCT may be a therapeutic option for children with disease that does not respond to standard care.

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Acknowledgements

We thank Myriam Labopin for valuable advice on statistical analyses and Tobias Alexander, ADWP secretary.

Author information

Author notes

  1. J Burman and K Kirgizov: These authors contributed equally to this work.

Authors and Affiliations

  1. Department of Neuroscience, Uppsala University Hospital, Uppsala University, Uppsala, Sweden

    J Burman

  2. BMT Department, Russian Children’s Research Hospital, Moscow, Russia

    K Kirgizov

  3. Department of Medical Sciences, Uppsala University, Uppsala, Sweden

    K Carlson

  4. EBMT Paris Study Office, Hôpital Saint Antoine, Paris, France

    M Badoglio

  5. Department of Neurosciences, University of Genoa, Genoa, Italy

    G L Mancardi

  6. Neurology Clinic, Multiple Sclerosis Center, Università G. D'Annunzio Chieti-Pescara, Chieti, Italy

    G De Luca

  7. Neuroimmunology Unit, Hospital Universitari i Politècninc La Fe, Valencia, Spain

    B Casanova

  8. Department of Hematology, Affiliated Drum Tower Hospital of Nanjing University Medical School, Nanjing, China

    J Ouyang

  9. Department of Neurology, Neurosurgery and Genetics of Pediatric Faculty, Pirogov Russian National Research Medical University, Moscow, Russia

    R Bembeeva

  10. Jüdisches Krankenhaus, Berlin, Germany

    J Haas

  11. Division for Stem Cell Transplantation and Immunology, Department for Children and Adolescents, G.W. Goethe University Hospital, Frankfurt am Main, Germany

    P Bader

  12. Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust and University of Sheffield, Sheffield, UK

    J Snowden

  13. Paris 7 University, INSERM U1160, Paris, France

    D Farge

  14. Saint Louis Hospital, Unité de Médecine interne: Maladies auto-immunes et Pathologie Vasculaire, UF 04, Assistance Publique des Hôpitaux de Paris, Paris7 Denis Diderot University, Paris, France

    D Farge

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  1. J Burman
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  4. M Badoglio
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  5. G L Mancardi
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  7. B Casanova
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  8. J Ouyang
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  10. J Haas
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Corresponding author

Correspondence to J Burman.

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Burman, J., Kirgizov, K., Carlson, K. et al. Autologous hematopoietic stem cell transplantation for pediatric multiple sclerosis: a registry-based study of the Autoimmune Diseases Working Party (ADWP) and Pediatric Diseases Working Party (PDWP) of the European Society for Blood and Marrow Transplantation (EBMT). Bone Marrow Transplant 52, 1133–1137 (2017). https://doi.org/10.1038/bmt.2017.40

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  • DOI: https://doi.org/10.1038/bmt.2017.40

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