Chronic Myeloid Leukemia Coverage from Every Angle

Evaluating Outcomes for Children With Advanced Phase of CML at Time of Diagnosis

By: Joseph Fanelli
Posted: Monday, July 22, 2019

According to findings from the International Chronic Myeloid Leukemia Pediatric Study presented in the European Journal of Cancer, children diagnosed with advanced phase chronic myeloid Leukemia (CML) at the time of diagnosis appear to have better survival rates than those who experienced advanced phases of disease while receiving  tyrosine kinase inhibitors (TKIs). According to Frédéric Millot, MD, of the University Hospital, Poitiers, France, and colleagues, these findings indicate that treatment approaches for advanced phases of CML at the time of diagnosis in children might differ from those when disease progresses with upfront TKI treatment.

“Upfront treatment recommendations for newly diagnosed CML [accelerated phase] and CML-[blast phase] in pediatric patients are based on TKI, but the optimal treatment remains to be determined,” the authors observed.

This observational, prospective study ( identifier NCT01281735) reportedly included the largest database analysis of the characteristics and outcomes of childhood CML. Of the 479 children (aged 17 and younger) enrolled in this study, 36 (7.5%) displayed advanced phase CML at the time of diagnosis, according to the European LeukemiaNet criteria.

Of the 36 patients, 19 (4%) were diagnosed with CML in the accelerated phase, and 17 (3.5%) were diagnosed with CML in the blast phase. For both groups of patients, their initial treatment included TKIs with or without chemotherapy, with 33 (92%) reaching complete hematologic response. Subsequently, 17 patients had hematopoietic stem cell transplantation.

As of the last follow-up, 18 of the 19 patients with advanced phase CML detected at the time of diagnosis were alive, with 16 achieving major molecular response and 1 each experiencing disease progression and molecular relapse. Of the 17 patients diagnosed with blast phase CML at the time of diagnosis, 13 were alive, with at least a major molecular response. For the two groups, 5-year overall survival rates were 94% and 74% for patients with advanced phase and blast phase CML, respectively.

Disclosure: The study authors’ disclosure information can be found at

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