Analysis of risk factors influencing outcome in children with myelodysplastic syndrome after unrelated cord blood transplantation.
We describe 70 children with myelodysplastic syndrome (MDS) (refractory cytopenia (n=31) and refractory anemia with excess blasts (n=30) or blasts in transformation (n=9)) who received umbilical cord blood (UCB) transplantation with a single UCB unit and a myeloablative conditioning regimen. Approximately 20% of children had secondary MDS. Median age at transplantation was 7 years and the median follow-up was 3 years. The day-60 probability of neutrophil recovery was 76%; recovery was faster after transplantation of matched or 1-locus mismatched UCB, irradiation-containing conditioning regimen, cell dose >6 × 10(7)/kg and monosomy 7. Risks of treatment failure (recurrent disease or death) were lower in patients with monosomy 7 and transplantations after 2001. The 3-year disease-free survival (DFS) was 50% for transplantations after 2001 compared with 27% for the earlier period (P=0.018). Transplantations after 2001 occurred within 6 months after diagnosis and used UCB units with higher cell dose. DFS was highest in patients with monosomy 7 (61%) compared with other karyotypes (30%), P=0.017. These data suggest that transplantation of mismatched UCB graft is an acceptable alternative for children without a matched sibling or suitably matched unrelated adult donor.
Eurocord Office, Hôpital Saint Louis, Assistance Publique des Hôpitaux de Paris, Paris, France.
A B M
AB
Madureira
M
M
Eapen
F
F
Locatelli
P
P
Teira
M-J
MJ
Zhang
S M
SM
Davies
A
A
Picardi
A
A
Woolfrey
K-W
KW
Chan
G
G
Socié
A
A
Vora
Y
Y
Bertrand
C M
CM
Sales-Bonfim
E
E
Gluckman
C
C
Niemeyer
V
V
Rocha
D000293:Adolescent; D002648:Child; D002675:Child, Preschool; D036101:Cord Blood Stem Cell Transplantation / Q000009:adverse effects; D018572:Disease-Free Survival; D006086:Graft vs Host Disease / Q000209:etiology; D006801:Humans; D007223:Infant; D007231:Infant, Newborn; D009190:Myelodysplastic Syndromes / Q000097:blood / Q000401:mortality / Q000601:surgery*; D012008:Recurrence; D012307:Risk Factors; D016896:Treatment Outcome