Analysis of risk factors influencing outcome in children with myelodysplastic syndrome after unrelated cord blood transplantation

摘要

We describe 70 children with myelodysplastic syndrome [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 myelodysplastic syndrome. Median age at transplantation was 7 years and the median follow-up, 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 × 10⁷/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 to 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 to other karyotypes (30%), p=0.017. These data suggest transplantation of mismatched UCB graft is an acceptable alternative for children without a matched sibling or suitably matched unrelated adult donor.