Janda A, Sedláček 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 FA, Slatter MA, Davies EG, Gennery AR. Blood. 2010 Jun 7. IF: 10.890
Abstract:
Seventeen patients transplanted with hematopoietic cells to correct severe T lymphocyte immunodeficiency due to 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 (2–53) months between 1995 to 2006. Bone marrow was used in 11 procedures in 9 cases: six from matched unrelated donors, four from HLA-identical siblings and one haploidentical parent with T cell-depletion. Unmobilized peripheral blood was used in eight cases: five 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 five and graft versus host disease (GvHD) prophylaxis in eleven patients. Significant GvHD occurred in nine patients, becoming chronic in three. Median length of follow-up was 13 months, transplantation from HLA-matched sibling showing the best results. Median survival amongst deceased patients (10 patients) was 7 months after transplantation (2–18 months). The overall survival rate was 41%, with a median follow-up of 5.8 (4–11.5) years. Amongst survivors, median (range) CD3 and CD4 counts was 806 (644–1224) and 348 (225–782) cells/mm3 respectively, CD4+/CD45RA+ cells remained very low, while mitogen responses were normalized.
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