Objective: To report outcomes of Chronic Myeloid Leukemia (CML) treated with upfront imatinib.
Methods: Outcomes of children (≤18 y) with chronic phase CML (CML-CP) treated with imatinib over a 5 y (2003-2008) period were retrospectively analyzed to quantify responses, progression free survival (PFS) and overall survival (OS).
Results: Thirty-one patients (age range: 6-18 y) received therapy with imatinib 260-300 mg/m(2). Thirty (97 %) achieved complete hematological response at a median of 2 mo from start of treatment. Major and complete cytogenetic response rates at 2 y were 82 % and 70 % respectively. After a median follow up of 49.2 mo the 5 year PFS and OS were 68 % and 76 % respectively. Out of the 16 patients with documented Complete Cytogenetic Response (CCR) at 2 y, none progressed during subsequent follow up. There were no serious toxicities. Most patients who progressed, died of the disease.
Conclusions: Imatinib is a reasonable first line therapy in pediatric CML-CP, which is effective and well tolerated. Outcomes are comparable to those reported from the West. Availability of second line agents and increased access to stem cell transplantation could further improve outcomes.