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

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

Research output: Contribution to journalArticle

38 Scopus citations

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 graftversus-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/ mm3, respectively, CD4 +/CD45RA+ cells remained very low, whereas mitogen responses were normalized.

Original languageEnglish (US)
Pages (from-to)2229-2236
Number of pages8
JournalBlood
Volume116
Issue number13
DOIs
StatePublished - Sep 30 2010

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

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    Janda, A., Sedlacek, P., Hönig, M., Friedrich, W., Champagne, M., Matsumoto, T., Fischer, A., Neven, B., Contet, A., Bensoussan, D., Bordigoni, P., Loeb, D., Savage, W., Jabado, N., Bonilla, F. A., Slatter, M. A., Davies, E. G., & Gennery, A. R. (2010). Multicenter survey on the outcome of transplantation of hematopoietic cells in patients with the complete form of DiGeorge anomaly. Blood, 116(13), 2229-2236. https://doi.org/10.1182/blood-2010-03-275966