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Treatment of CD40 l...
Treatment of CD40 ligand deficiency by hematopoietic stem cell transplantation: a survey of the European experience, 1993-2002
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Gennery, A. R. (author)
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Khawaja, K. (author)
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Veys, P. (author)
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Bredius, R. G. (author)
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Notarangelo, L. D. (author)
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Mazzolari, E. (author)
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Fischer, A. (author)
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Landais, P. (author)
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Cavazzana-Calvo, M. (author)
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Friedrich, W. (author)
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- Fasth, Anders, 1945 (author)
- Gothenburg University,Göteborgs universitet,Institutionen för kvinnors och barns hälsa, Avdelningen för pediatrik,Institute for the Health of Women and Children, Dept of Paediatrics
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Wulffraat, N. M. (author)
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Matthes-Martin, S. (author)
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Bensoussan, D. (author)
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Bordigoni, P. (author)
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Lange, A. (author)
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Pagliuca, A. (author)
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Andolina, M. (author)
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Cant, A. J. (author)
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Davies, E. G. (author)
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(creator_code:org_t)
- 2003-10-02
- 2004
- English.
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In: Blood. - : American Society of Hematology. - 0006-4971 .- 1528-0020. ; 103:3, s. 1152-7
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https://gup.ub.gu.se...
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https://doi.org/10.1...
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Abstract
Subject headings
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- CD40 ligand (CD40L) deficiency causes recurrent sinopulmonary infection, Pneumocystis carinii pneumonia, and Cryptosporidium parvum infection. Approximately 40% to 50% of patients survive to the third decade: long-term survival is unclear. Hematopoietic stem cell transplantation (HSCT) is curative. We present a retrospective analysis of 38 European patients undergoing HSCT for CD40L deficiency in 8 European countries between 1993 and 2002. Donor stem cell source included 14 HLA-identical siblings, 22 unrelated donors, and 2 phenotypically matched parental stem cells (12 T-cell depleted). Of the patients, 34 engrafted and 26 (68%) survived; 3 had autologous reconstitution, 22 (58%) were cured, and 1 engrafted but has poor T-cell immune reconstitution. There were 18 evaluated patients who responded to vaccination. Of the patients, 12 (32%) died from infection-related complications, with severe cryptosporidiosis in 6. Grades 2 to 4 graft-versus-host disease (GvHD) associated with infection occurred in 6 of 12 fatal cases. HSCT cured 58% of patients, 72% of those without hepatic disease. Early T-cell function following whole marrow HSCT may limit cryptosporidial disease, but survival was similar after T-cell-depleted HSCT. Preexisting lung damage was the most important adverse risk factor. Further studies will determine optimal timing and type of HSCT.
Keyword
- Adolescent
- Adult
- CD40 Ligand/*genetics/*metabolism
- Child
- Child
- Preschool
- Data Collection
- Europe
- Genetic Diseases
- X-Linked/genetics/immunology/*therapy
- Graft Survival
- Graft vs Host Disease/etiology
- *Hematopoietic Stem Cell Transplantation/adverse effects
- Humans
- Hypergammaglobulinemia/genetics/immunology/*therapy
- *Immunoglobulin M
- Infant
- Opportunistic Infections/etiology
- Retrospective Studies
Publication and Content Type
- ref (subject category)
- art (subject category)
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Blood
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To the university's database
- By the author/editor
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Gennery, A. R.
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Khawaja, K.
-
Veys, P.
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Bredius, R. G.
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Notarangelo, L. ...
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Mazzolari, E.
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show more...
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Fischer, A.
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Landais, P.
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Cavazzana-Calvo, ...
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Friedrich, W.
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Fasth, Anders, 1 ...
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Wulffraat, N. M.
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Matthes-Martin, ...
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Bensoussan, D.
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Bordigoni, P.
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Lange, A.
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Pagliuca, A.
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Andolina, M.
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Cant, A. J.
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Davies, E. G.
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show less...
- Articles in the publication
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Blood
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University of Gothenburg