Multicenter survey on the outcome of transplantation of hematopoietic cells in patients with the complete form of DiGeorge anomaly.

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.

• 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)

Chemical References

• HLA Antigens

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