First-line imatinib is effective and safe among pediatric patients with chronic myeloid leukemia (CML), according to a study being presented at the 2017 American Society of Hematology Annual Meeting (ASH) in Atlanta, Georgia.1

For the prospective CML-PAED-II study, researchers enrolled 156 patients with newly diagnosed CML. Patients received imatinib 300 mg/m2, 400 mg/m2, and 500 mg/m2 for chronic phase (CML-CP), accelerated phase (CML-AP), and blast phase (CML-BP) disease, respectively. Of the enrolled patients, 148 had evaluable data.

The median follow-up was 25 months. Patients with CML-CP had an 18-month event-free survival rate of 97% (95% CI, 94.2%-99.9%).

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Complete hematologic response at month 3 was observed in 98% of patients, complete cytogenic response (CCyR) at month 12 was observed in 63% of patients, and molecular response (MR3.0) at month 18 was observed in 59% of patients. After 3 years of receiving continuous first-line imatinib or 2nd-generation tyrosine kinase inhibitor (TKI) therapy, 86% and 74% of patients achieved CCyR and MR3.0, respectively.

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Approximately 66% of patients reported at least 1 adverse event (AE); the most frequently reported AE of any grade was imatinib-related anemia and the most frequently reported grade 3 to 4 hematologic AE was neutropenia. Gastrointestinal and musculoskeletal AEs were also common.

Eighteen percent of patients had to suspend therapy and 6% of patients discontinued treatment because of intolerable AEs; 27% of patients failed therapy because of unsatisfactory response or intolerance. Nineteen percent of patients underwent stem cell transplantation (SCT), of which 3 patients died of relapse and 2 died of SCT-related complications.

The authors concluded that “[l]ong term outcome and effects of potentially life-long TKI treatment have to be registered in cooperation with adult hematologists in extended surveillance follow-up studies.”

Read more of Cancer Therapy Advisor‘s coverage of the American Society of Hematology (ASH) 2017 meeting by visiting the conference page.


  1. Suttorp M, Schulze P, Glauche I, et al. Response to front-line imatinib treatment in children and adolescents with CML – data from a large pediatric cohort. Poster presentation at: American Society of Hematology 59th Annual Meeting & Exposition; December 9-12, 2017; Atlanta, GA.

This article originally appeared on ONA