TY - JOUR
T1 - Multicenter survey on the outcome of transplantation of hematopoietic cells in patients with the complete form of DiGeorge anomaly
AU - Janda, Ales
AU - Sedlacek, Petr
AU - Hönig, Manfred
AU - Friedrich, Wilhelm
AU - Champagne, Martin
AU - Matsumoto, Tadashi
AU - Fischer, Alain
AU - Neven, Benedicte
AU - Contet, Audrey
AU - Bensoussan, Danielle
AU - Bordigoni, Pierre
AU - Loeb, David
AU - Savage, William
AU - Jabado, Nada
AU - Bonilla, Francisco A.
AU - Slatter, Mary A.
AU - Davies, E. Graham
AU - Gennery, Andrew R.
PY - 2010/9/30
Y1 - 2010/9/30
N2 - 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.
AB - 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.
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U2 - 10.1182/blood-2010-03-275966
DO - 10.1182/blood-2010-03-275966
M3 - Article
C2 - 20530285
AN - SCOPUS:77957714384
SN - 0006-4971
VL - 116
SP - 2229
EP - 2236
JO - Blood
JF - Blood
IS - 13
ER -