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Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?


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.

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.

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Additional indexing

Item Type:Journal Article, refereed, original work
Communities & Collections:04 Faculty of Medicine > University Children's Hospital Zurich > Medical Clinic
Dewey Decimal Classification:610 Medicine & health
Language:English
Date:2012
Deposited On:19 Feb 2013 08:25
Last Modified:07 Dec 2017 19:32
Publisher:American Society of Hematology
ISSN:0006-4971
Additional Information:This research was originally published in Blood. 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; Cavazzana-Calvo, Marina (2012). Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood? Blood, 119(12):2949-2955. Copyright by the American Society of Hematology.
Publisher DOI:https://doi.org/10.1182/blood-2011-06-363572
PubMed ID:22308292

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