Early hematopoietic stem cell transplant is associated with favorable outcomes in children with MDS

Pediatr Blood Cancer. 2013 Apr;60(4):705-10. doi: 10.1002/pbc.24390. Epub 2012 Nov 14.

Abstract

Background: Although hematopoietic stem cell transplantation (HSCT) is the treatment of choice for childhood myelodysplastic syndrome (MDS), there is no consensus regarding patient or disease characteristics that predict outcomes.

Procedure: We reviewed 37 consecutive pediatric MDS patients who received myeloablative HSCT between 1990 and 2010 at a single center.

Results: Twenty had primary MDS and 17 had secondary MDS. Diagnostic cytogenetics included monosomy 7 (n = 21), trisomy 8 (n = 7) or normal/other (n = 8). According to the modified WHO MDS classification, thirty had refractory cytopenia and seven had refractory anemia with excess blasts. IPSS scores were: low risk (n = 1), intermediate-1 (n = 15), and intermediate-2 (n = 21). OS and DFS at 10 years in the entire cohort was 53% and 45%. Relapse at 10 years was 26% and 1 year TRM was 25%. In multivariate analysis, factors associated with improved 3 years DFS were not receiving pre-HSCT chemotherapy (RR = 0.30, 95% CI 0.10-0.88; P = 0.03) and a shorter interval (<140 days) from time of diagnosis to transplant (RR = 0.27, 95% CI 0.09-0.80; P = 0.02). Three years DFS in patients who did not receive pre-HSCT chemotherapy and those who had a shorter interval to transplant (n = 16) was 80%.

Conclusion: These results suggest that children with MDS should be referred for allogeneic HSCT soon after diagnosis and that pre-HSCT chemotherapy does not appear to improve outcomes.

MeSH terms

  • Adolescent
  • Antineoplastic Agents / therapeutic use
  • Child
  • Child, Preschool
  • Disease-Free Survival
  • Female
  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Infant
  • Kaplan-Meier Estimate
  • Male
  • Myelodysplastic Syndromes / drug therapy
  • Myelodysplastic Syndromes / mortality*
  • Myelodysplastic Syndromes / surgery*
  • Neoplasm Recurrence, Local / epidemiology
  • Retrospective Studies
  • Treatment Outcome
  • Young Adult

Substances

  • Antineoplastic Agents