Transplantation in patients with SCID: mismatched related stem cells or unrelated cord blood?
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.
Eurocord, Hôpital Saint Louis, Paris, France.
Juliana F
JF
Fernandes
Vanderson
V
Rocha
Myriam
M
Labopin
Benedicte
B
Neven
Despina
D
Moshous
Andrew R
AR
Gennery
Wilhelm
W
Friedrich
Fulvio
F
Porta
Cristina
C
Diaz de Heredia
Donna
D
Wall
Yves
Y
Bertrand
Paul
P
Veys
Mary
M
Slatter
Ansgar
A
Schulz
Ka Wah
KW
Chan
Michael
M
Grimley
Mouhab
M
Ayas
Tayfun
T
Gungor
Wolfram
W
Ebell
Carmem
C
Bonfim
Krzysztof
K
Kalwak
Pierre
P
Taupin
Stéphane
S
Blanche
H Bobby
HB
Gaspar
Paul
P
Landais
Alain
A
Fischer
Eliane
E
Gluckman
Marina
M
Cavazzana-Calvo
D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000009:adverse effects / Q000401:mortality; D005260:Female; D006086:Graft vs Host Disease / Q000453:epidemiology* / Q000401:mortality; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000401:mortality; D006648:Histocompatibility; D006801:Humans; D015994:Incidence; D007223:Infant; D007231:Infant, Newborn; D053208:Kaplan-Meier Estimate; D008297:Male; D016016:Proportional Hazards Models; D012189:Retrospective Studies; D016511:Severe Combined Immunodeficiency / Q000276:immunology* / Q000401:mortality / Q000601:surgery*; D019172:Transplantation Conditioning / Q000379:methods; D016896:Treatment Outcome