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).
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.
Department of Neuroscience, Uppsala University Hospital, Uppsala University, Uppsala, Sweden.
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http://orcid.org/0000-0002-7045-1806
J
Burman
BMT Department, Russian Children's Research Hospital, Moscow, Russia.
K
K
Kirgizov
Department of Medical Sciences, Uppsala University, Uppsala, Sweden.
K
K
Carlson
EBMT Paris Study Office, Hôpital Saint Antoine, Paris, France.
M
M
Badoglio
Department of Neurosciences, University of Genoa, Genoa, Italy.
G L
GL
Mancardi
Neurology Clinic, Multiple Sclerosis Center, Università G. D'Annunzio Chieti-Pescara, Chieti, Italy.
G
G
De Luca
Neuroimmunology Unit, Hospital Universitari i Politècninc La Fe, Valencia, Spain.
B
B
Casanova
Department of Hematology, Affiliated Drum Tower Hospital of Nanjing University Medical School, Nanjing, China.
J
J
Ouyang
Neurosurgery and Genetics of Pediatric Faculty, Department of Neurology, Pirogov Russian National Research Medical University, Moscow, Russia.
R
R
Bembeeva
Jüdisches Krankenhaus, Berlin, Germany.
J
J
Haas
Division for Stem Cell Transplantation and Immunology, Department for Children and Adolescents, G.W. Goethe University Hospital, Frankfurt am Main, Germany.
P
P
Bader
Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust and University of Sheffield, Sheffield, UK.
J
J
Snowden
Paris 7 University, INSERM U1160, Paris, France.|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.
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Farge
D000293:Adolescent; D002648:Child; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods*; D006801:Humans; D008297:Male; D009103:Multiple Sclerosis / Q000628:therapy*; D012042:Registries; D014182:Transplantation, Autologous; D016896:Treatment Outcome