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)

被引：20

作者：

Burman, J. ^{[1
]}

Kirgizov, K. ^{[2
]}

Carlson, K. ^{[3
]}

Badoglio, M. ^{[4
]}

Mancardi, G. L. ^{[5
]}

De Luca, G. ^{[6
]}

Casanova, B. ^{[7
]}

Ouyang, J. ^{[8
]}

Bembeeva, R. ^{[9
]}

Haas, J. ^{[10
]}

Bader, P. ^{[11
]}

Snowden, J. ^{[12
,13
]}

Farge, D. ^{[14
,15
]}

机构：

[1] Uppsala Univ, Uppsala Univ Hosp, Dept Neurosci, Uppsala, Sweden

[2] Russian Childrens Res Hosp, BMT Dept, Moscow, Russia

[3] Uppsala Univ, Dept Med Sci, Uppsala, Sweden

[4] Hop St Antoine, EBMT Paris Study Off, Paris, France

[5] Univ Genoa, Dept Neurosci, Genoa, Italy

[6] Univ G dAnnunzio, Multiple Sclerosis Ctr, Neurol Clin, Chieti, Italy

[7] Hosp Univ & Politecninc La Fe, Neuroimmunol Unit, Valencia, Spain

[8] Nanjing Univ, Dept Hematol, Affiliated Drum Tower Hosp, Med Sch, Nanjing, Jiangsu, Peoples R China

[9] Pirogov Russian Natl Res Med Univ, Neurosurg & Genet Pediat Fac, Dept Neurol, Moscow, Russia

[10] Jud Krankenhaus, Berlin, Germany

[11] GW Goethe Univ Hosp, Dept Children & Adolescents, Div Stem Cell Transplantat & Immunol, Frankfurt, Germany

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

[13] Univ Sheffield, Sheffield, S Yorkshire, England

[14] Paris 7 Univ, INSERM U1160, Paris, France

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

来源：

BONE MARROW TRANSPLANTATION | 2017年 / 52卷 / 08期

关键词：

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

D O I：

10.1038/bmt.2017.40

中图分类号：

Q6 [生物物理学];

学科分类号：

071011 ;

摘要：

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.

引用

页码：1133 / 1137

页数：5

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