Chronic myeloid leukemia (CML) in children and young adults is uncommon. Young patients have long life expectancies and low morbidity with hematopoietic cell transplantation (HCT). Prolonged tyrosine kinase inhibitor (TKI) use may cause significant morbidity. In addition indication for HCT in patients in first chronic phase is not established.
We hence retrospectively evaluated outcomes in 449 CML patients with early disease receiving myeloablative HCT reported to the CIBMTR. We analyzed various factors affecting outcome specifically the effect of age and pre-HCT TKI in pediatric patients (<18 yrs, n= 177) and young adults (18–29 years, n=272) with the goal of identifying prognostic factors.
Post-HCT probability of 5 y overall survival (OS) and leukemia free survival (LFS) were 75% and 59% respectively. OS and LFS were 76%, 57% in <18 yr and 74%, 60% in 18–29 yr group respectively by univariate analysis (p= 0.1 and 0.6). Five-year OS for human leukocyte antigen (HLA) matched sibling donor (MSD) and bone marrow (BM) stem cell source were 83% and 80% respectively. In multivariate analysis, there was no effect of age (<18 vs 18–29) or pre-HCT TKI therapy on OS, LFS, transplant related mortality (TRM) or relapse. Favorable factors for OS were MSD (p<0.001) and recent HCT (2003–2010) (p=0.04). LFS was superior with MSD (P<0.001), BM as graft source (P=0.001) and performance score >90 (P=0.03) compared to unrelated or mismatched, peripheral blood stem cells donors and recipients with lower performance scores. Older age was associated with increased incidence of chronic graft vs. host disease (cGVHD) (p=0.0002).
In the current era, HCT outcomes are similar in young patients and children with early CML, and best outcomes are achieved with BM grafts and MSD.