Abstract |
Seventeen patients transplanted with hematopoietic cells to correct severe T lymphocyte immunodeficiency resulting from complete DiGeorge anomaly were identified worldwide, and retrospective data were obtained using a questionnaire-based survey. Patients were treated at a median age of 5 months (range, 2-53 months) between 1995 and 2006. Bone marrow was used in 11 procedures in 9 cases: 6 from matched unrelated donors, 4 from human leukocyte antigen (HLA)-identical siblings, and one haploidentical parent with T-cell depletion. Unmobilized peripheral blood was used in 8 cases: 5 from HLA-identical siblings, one from a matched unrelated donor, one from an HLA-identical parent, and one unrelated matched cord blood. Conditioning was used in 5 patients and graft-versus-host disease prophylaxis in 11 patients. Significant graft-versus-host disease occurred in 9 patients, becoming chronic in 3. Median length of follow-up was 13 months, with transplantation from HLA-matched sibling showing the best results. Median survival among deceased patients (10 patients) was 7 months after transplantation (range, 2-18 months). The overall survival rate was 41%, with a median follow-up of 5.8 years (range, 4-11.5 years). Among survivors, median CD3 and CD4 counts were 806 (range, 644-1224) and 348 (range, 225-782) cells/mm(3), respectively, CD4(+)/CD45RA(+) cells remained very low, whereas mitogen responses were normalized.
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Authors | Ales Janda, Petr Sedlacek, Manfred Hönig, Wilhelm Friedrich, Martin Champagne, Tadashi Matsumoto, Alain Fischer, Benedicte Neven, Audrey Contet, Danielle Bensoussan, Pierre Bordigoni, David Loeb, William Savage, Nada Jabado, Francisco A Bonilla, Mary A Slatter, E Graham Davies, Andrew R Gennery |
Journal | Blood
(Blood)
Vol. 116
Issue 13
Pg. 2229-36
(Sep 30 2010)
ISSN: 1528-0020 [Electronic] United States |
PMID | 20530285
(Publication Type: Journal Article, Multicenter Study, Research Support, Non-U.S. Gov't)
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Chemical References |
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Topics |
- Bone Marrow Transplantation
- Child, Preschool
- Cord Blood Stem Cell Transplantation
- DiGeorge Syndrome
(blood, immunology, therapy)
- Female
- Graft vs Host Disease
(etiology)
- HLA Antigens
- Hematopoietic Stem Cell Transplantation
(adverse effects, methods)
- Humans
- Infant
- Lymphocyte Count
- Lymphopoiesis
- Male
- Retrospective Studies
- Surveys and Questionnaires
- Transplantation Conditioning
- Treatment Outcome
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