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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 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)

Fecha de publicación: 01/08/2017 Fecha Ahead of Print: 20/03/2017

Autores de IIS La Fe

Bonaventura Casanova Estruch

Autor

Participantes ajenos a IIS La Fe

Burman, J
Kirgizov, K
Carlson, K
Badoglio, M
Mancardi, GL
De Luca, G
Ouyang, J
Bembeeva, R
Haas, J
Bader, P
Snowden, J
Farge, D

Grupos

Investigación en Neuroinmunología y Esclerosis Múltiple

Leader

Bonaventura Casanova Estruch

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.

Datos de la publicación

ISSN/ISSNe:: 0268-3369, 1476-5365
Tipo:: Article
Páginas:: 1133-1137
DOI:: 10.1038/bmt.2017.40
Factor de Impacto:: 1,935 SCImago ℠
Cuartil:: Q1 SCImago ℠

Documentos

No hay documentos

Métricas

Filiaciones

Burman, J:: Department of Neuroscience, Uppsala University Hospital, Uppsala University, Uppsala, Sweden

Uppsala Univ, Uppsala Univ Hosp, Dept Neurosci, Uppsala, Sweden
Kirgizov, K:: BMT Department, Russian Children's Research Hospital, Moscow, Russia

Russian Childrens Res Hosp, BMT Dept, Moscow, Russia
Carlson, K:: Department of Medical Sciences, Uppsala University, Uppsala, Sweden

Uppsala Univ, Dept Med Sci, Uppsala, Sweden
Badoglio, M:: EBMT Paris Study Office, Hôpital Saint Antoine, Paris, France

Hop St Antoine, EBMT Paris Study Off, Paris, France
Mancardi, GL:: Department of Neurosciences, University of Genoa, Genoa, Italy

Univ Genoa, Dept Neurosci, Genoa, Italy
De Luca, G:: Neurology Clinic, Multiple Sclerosis Center, Università G. D'Annunzio Chieti-Pescara, Chieti, Italy

Univ G dAnnunzio, Multiple Sclerosis Ctr, Neurol Clin, Chieti, Italy
Casanova, B:: Neuroimmunology Unit, Hospital Universitari i Politècninc La Fe, Valencia, Spain

Hosp Univ & Politecninc La Fe, Neuroimmunol Unit, Valencia, Spain
Ouyang, J:: Department of Hematology, Affiliated Drum Tower Hospital of Nanjing University Medical School, Nanjing, China

Nanjing Univ, Dept Hematol, Affiliated Drum Tower Hosp, Med Sch, Nanjing, Jiangsu, Peoples R China
Bembeeva, R:: Neurosurgery and Genetics of Pediatric Faculty, Department of Neurology, Pirogov Russian National Research Medical University, Moscow, Russia

Pirogov Russian Natl Res Med Univ, Neurosurg & Genet Pediat Fac, Dept Neurol, Moscow, Russia
Haas, J:: Jüdisches Krankenhaus, Berlin, Germany

Jud Krankenhaus, Berlin, Germany
Bader, P:: Division for Stem Cell Transplantation and Immunology, Department for Children and Adolescents, G.W. Goethe University Hospital, Frankfurt am Main, Germany

GW Goethe Univ Hosp, Dept Children & Adolescents, Div Stem Cell Transplantat & Immunol, Frankfurt, Germany
Snowden, J:: Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust and University of Sheffield, Sheffield, UK

Sheffield Teaching Hosp NHS Fdn Trust, Dept Haematol, Sheffield, S Yorkshire, England

Univ Sheffield, Sheffield, S Yorkshire, England
Farge, D:: 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

Paris 7 Univ, INSERM U1160, Paris, France

Paris7 Denis Diderot Univ, St Louis Hosp, AP HP,UF 04, Unite Med Interne Malad Autoimmunes & Pathol Vasc, Paris, France

Keywords

INTENSITY CONDITIONING REGIMEN; CHILDHOOD; CHILDREN; ONSET; IMPAIRMENT; DISABILITY; TRIAL; MS

Proyectos asociados

PROYECTO CAIBER

CAI08/01/0061 . INSTITUTO DE SALUD CARLOS III; FUNDACIÓN PARA LA INVESTIGACIÓN DEL HOSPITAL UNIVERSITARIO LA FE DE LA COMUNIDAD VALENCIANA . 2009

RED DE BIOBANCOS (BIOBANCOS)

RD09/0076/00021 . INSTITUTO DE SALUD CARLOS III; FUNDACIÓN PARA LA INVESTIGACIÓN DEL HOSPITAL UNIVERSITARIO LA FE DE LA COMUNIDAD VALENCIANA . 2010

PATRONES DE RM CEREBRAL Y MEDULAR EN RELACION CON LA PRESENCIA DE AUTOANTIGENOS NEURONALES RECONOCIDOS POR INMUNOGLOBULINAS M PRESENTES EN EL LIQUIDO CEFALORRAQUIDEO DE PACIENTES DE ESCLEROSIS MULTIPLE.

Investigador Principal: ISABEL BOSCÁ BLASCO

MERCK SERONO DR. CASANOVA . ALMIRALL S.A; FUNDACIÓN BIOGEN IDEC; MERCK S.L.U; FUNDACIÓN PARA LA INVESTIGACIÓN DEL HOSPITAL UNIVERSITARIO LA FE DE LA COMUNIDAD VALENCIANA . 2010