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Description
  • 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.
  • 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. (en)
Title
  • Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?
  • Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood? (en)
skos:prefLabel
  • Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?
  • Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood? (en)
skos:notation
  • RIV/00064203:_____/12:8572!RIV13-MZ0-00064203
http://linked.open...avai/predkladatel
http://linked.open...avai/riv/aktivita
http://linked.open...avai/riv/aktivity
  • I
http://linked.open...iv/cisloPeriodika
  • 12
http://linked.open...vai/riv/dodaniDat
http://linked.open...aciTvurceVysledku
http://linked.open.../riv/druhVysledku
http://linked.open...iv/duvernostUdaju
http://linked.open...titaPredkladatele
http://linked.open...dnocenehoVysledku
  • 174976
http://linked.open...ai/riv/idVysledku
  • RIV/00064203:_____/12:8572
http://linked.open...riv/jazykVysledku
http://linked.open.../riv/klicovaSlova
  • severe combined immunodeficiency; bone-marrow-transplantation; term immune reconstitution; host disease; experience; children; phenotype; anemia; graft (en)
http://linked.open.../riv/klicoveSlovo
http://linked.open...odStatuVydavatele
  • US - Spojené státy americké
http://linked.open...ontrolniKodProRIV
  • [B0D761798816]
http://linked.open...i/riv/nazevZdroje
  • Blood
http://linked.open...in/vavai/riv/obor
http://linked.open...ichTvurcuVysledku
http://linked.open...cetTvurcuVysledku
http://linked.open...UplatneniVysledku
http://linked.open...v/svazekPeriodika
  • 119
http://linked.open...iv/tvurceVysledku
  • Blanche, S.
  • Fernandes, J.
  • Fischer, A.
  • Moshous, D.
  • Starý, Jan
  • Cavazzana-Calvo, M.
  • Kalwak, K.
  • Schulz, A.
  • Veys, P.
  • Neven, B.
  • Gennery, A.
  • Labopin, M.
  • Ayas, M.
  • Bertrand, Y.
  • Bonfim, C.
  • Camci, Y.
  • Chan, K.
  • Courteille, V.
  • Czogala, W.
  • Ebell, W.
  • Friedrich, W.
  • Gaspar, H.
  • Gluckman, E.
  • Grimley, M.
  • Gungor, T.
  • Heredia, C.
  • Landais, P.
  • Nademi, Z.
  • Pashano, V.
  • Porta, F.
  • Qasim, W.
  • Rocha, V.
  • Slatter, M.
  • Taupin, P.
  • Wall, D.
http://linked.open...ain/vavai/riv/wos
  • 000302121700033
issn
  • 0006-4971
number of pages
is http://linked.open...avai/riv/vysledek of
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