Title: Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?
Authors: Fernandes, Juliana F ×
Rocha, Vanderson
Labopin, Myriam
Neven, Benedicte
Moshous, Despina
Gennery, Andrew R
Friedrich, Wilhelm
Porta, Fulvio
Diaz de Heredia, Cristina
Wall, Donna
Bertrand, Yves
Veys, Paul
Slatter, Mary
Schulz, Ansgar
Chan, Ka Wah
Grimley, Michael
Ayas, Mouhab
Gungor, Tayfun
Ebell, Wolfram
Bonfim, Carmem
Kalwak, Krzysztof
Taupin, Pierre
Blanche, Stéphane
Gaspar, H Bobby
Landais, Paul
Fischer, Alain
Gluckman, Eliane
Eurocord and Inborn Errors Working Party of European Group for Blood and Marrow Transplantation #
Contributors: Meyts, Isabelle
Issue Date: Mar-2012
Publisher: W.B. Saunders
Series Title: Blood vol:119 issue:12 pages:2949-2955
Abstract: Pediatric patients with SCID constitute medical emergencies. In the absence of an HLA-identical hematopoietic stem cell (HSC) donor, mismatched related-donor transplantation (MMRDT) or unrelated-donor umbilical cord blood transplantation (UCBT) are valuable treatment options. To help transplantation centers choose the best treatment option, we retrospectively compared outcomes after 175 MMRDTs and 74 UCBTs in patients with SCID or Omenn syndrome. Median follow-up time was 83 months and 58 months for UCBT and MMRDT, respectively. Most UCB recipients received a myeloablative conditioning regimen; most MMRDT recipients did not. UCB recipients presented a higher frequency of complete donor chimerism (P = .04) and faster total lymphocyte count recovery (P = .04) without any statistically significance with the preparative regimen they received. The MMRDT and UCBT groups did not differ in terms of T-cell engraftment, CD4(+) and CD3(+) cell recoveries, while Ig replacement therapy was discontinued sooner after UCBT (adjusted P = .02). There was a trend toward a greater incidence of grades II-IV acute GVHD (P = .06) and more chronic GVHD (P = .03) after UCBT. The estimated 5-year overall survival rates were 62% ± 4% after MMRDT and 57% ± 6% after UCBT. For children with SCID and no HLA-identical sibling donor, both UCBT and MMRDT represent available HSC sources for transplantation with quite similar outcomes.
ISSN: 0006-4971
Publication status: published
KU Leuven publication type: IT
Appears in Collections:Laboratory of Pediatric Immunology
× corresponding author
# (joint) last author

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