Allogeneic hematopoietic cell transplantation outcomes for children and young adults with treatment-related myelodysplastic syndrome or acute myeloid leukaemia

Abstract

Objective: Therapy-related myeloid malignancies (tMDS or tAML) are well-known complications of cancer therapy for which outcomes are poor. Whether clinical characteristics can predict disease outcome in children and young adults, as they do in older adults, is not well known.

Method: Twenty-five children with tMDS/tAML underwent allogeneic hematopoietic stem cell transplantation (allo-HCT) at the University of Minnesota between 1990 and 2010. Twenty-one patients received myeloablative conditioning. Graft sources included umbilical cord blood in fourteen patients [single (n=9), double (n=5)] and bone marrow in 11. Prior to transplant twenty-one patients received AML-directed chemotherapy and 19 were in complete remission at the time of allo-HCT. Twelve patients had high-risk cytogenetic abnormalities involving 11q23 (n=6), chromosome 5 (n=4) and/or chromosome 7 (n=5).

Result: The 5-year OS and DFS was 23% (CI 95%: 9-41%) and 17% (CI 95%: 5-35%) respectively. Transplant related mortality (TRM) and relapse at 1-year were 39% (CI 95%: 19-59%) and 22% (CI 95%: 5-38%). We found no significant differences in survival based on remission status, pre-transplant cytogenetics or conditioning regimen.

Conclusion: Outcomes of children and young adults with tMDS/tAML continue to be poor with TRM and relapse remaining the major obstacles to cure.