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Canadian Study on Treatment Choices and Outcomes for Children With CML

By: Kelly M. Hennessey, PhD
Posted: Monday, August 17, 2020

Upon their introduction in the early 2000s, tyrosine kinase inhibitors (TKIs) revolutionized the treatment of chronic myeloid leukemia (CML). Previously, the treatment of choice for CML was hematopoietic stem cell transplant (HSCT). Results from a retrospective analysis support the use of TKI monotherapy for children with CML. Sarah Alexander, MD, of The Hospital for Sick Children, Ontario, Canada, and colleagues published their results in Pediatric Blood & Cancer.

The study was conducted across five pediatric oncology centers in Ontario, Canada, and included 52 patients up to 18 years of age who were diagnosed with CML between 1985 and 2018. There were 22 patients in the pre-TKI era, 18 of whom were treated with HSCT. No donors were available for four patients. At a median follow-up of 6 years, the overall survival of the patients in the pre-TKI cohort was 63.6%.

From 2002 to 2018, of the 30 patients who received TKI treatment, 23 received imatinib as first-line therapy, 4 received dasatinib, and 3 elected to receive HSCT without TKI therapy. Nine patients underwent HSCT after TKI therapy; three died due to HSCT complications; and no deaths were attributed to the primary disease process. At a median follow-up of 3 years, the overall survival of patients who received TKI treatment was 90%; those treated with TKI therapy alone had significantly better outcomes. Survival outcomes were similar between patients who received HSCT before and after 2002.

“Our data support the current recommendations to switch to an alternative second-generation TKI with HSCT delayed where possible,” explained the researchers. “TKI therapy is a safe, effective treatment for childhood CML that has decreased mortality in children with this disease.”

Disclosure: For full disclosures of the study authors, visit onlinelibrary.wiley.com.



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