Chronic Myeloid Leukemia Coverage from Every Angle

Study Finds Asciminib Active in Heavily Pretreated Patients With CML

By: Kayci Reyer
Posted: Monday, January 6, 2020

According to research published in The New England Journal of Medicine, the allosteric BCR-ABL1 inhibitor asciminib showed clinical activity in patients with chronic myeloid leukemia (CML) who had undergone significant prior treatment. The study sought to determine the recommended and/or maximum tolerated dose of asciminib in this patient population.

“Asciminib was active in heavily pretreated patients with [CML] who had resistance to or unacceptable side effects from [tyrosine kinase inhibitors], including patients in whom ponatinib had failed and those with a T315I mutation,” concluded Timothy P. Hughes, MD, of the University of Adelaide, Australia, and colleagues.

The phase I study included 150 patients with either chronic phase (n = 141) or accelerated phase (n = 9) CML. A total of 70% of patients (n = 105) had previously received at least three tyrosine kinase inhibitors. Patients received oral asciminib once or twice daily at doses ranging from 10 mg to 200 mg.

At a median follow-up of 14 months, the maximum tolerated dose was not reached. The majority of patients (92%) with chronic phase disease who had previously had a hematologic relapse experienced a complete hematologic response, whereas more than half (54%) of those who did not have a baseline complete cytogenetic response had achieved one by the time of follow-up.

Among evaluable patients, major molecular response was achieved or maintained at a follow-up of 12 months in 48%, including 57% (8/14) of those for whom ponatinib was not effective and 28% of those with a baseline T315I mutation. A total of 40 of 44 patients achieving a major molecular response maintained it.

Toxic effects of asciminib treatment reported in this study included lipase elevations and pancreatitis; fatigue, headache, and arthralgia were among common adverse events.

Disclosure: For full disclosures of the study authors, visit

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