Chronic Myeloid Leukemia Coverage from Every Angle

EHA25 Virtual: Low-Dose Ponatinib for Patients With CML

By: Lauren Harrison, MS
Posted: Monday, June 15, 2020

For patients with chronic myeloid leukemia (CML) who were intolerant to previous tyrosine kinase inhibitors, low-dose ponatinib appears to be a viable treatment option, with a decreased incidence of specific adverse events and maintained efficacy compared with higher doses. The recommended starting dose of 45 mg was previously associated with adverse cardiovascular events. This analysis was presented by Alessandra Iurlo, MD, PhD, of Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy, and colleagues during the virtual edition of the 25th European Hematology Association Annual Congress (EHA25 virtual; Abstract EP752).

From May 2012 through December 2019, 46 patients with CML who were intolerant to previous tyrosine kinase inhibitors were treated with ponatinib at 13 different centers. Patients were given a median dose of 22.5 mg of ponatinib. At the start of therapy, this cohort had a male-to-female ratio of 2:1, with a median age of 64.5 years. Of note, 41.3% of patients had hypertension.

After a median follow-up of 22.2 months, 76.1% of patients had an increase in the depth of their molecular response, including 8 patients with a major molecular response and 15 patients with a durable molecular response. Among those who achieved a deep molecular response, one patient was able to attain treatment-free remission. The remaining 23.9% of patients included in the study maintained the same level of molecular response achieved prior to the study.

Adverse events were reported in 36.9% of patients, which included pancreatitis in 10.9%, hypertension in 8.7%, and cardiovascular events in 8.7%. One patient experienced an acute myocardial infarction, and one had an ischemic stroke, but both had preexisting cardiovascular risk factors. These two patients were started on a 30-mg dose of ponatinib. The patient who experienced the myocardial infarction had received nilotinib as a previous therapy.

Disclosure: For full disclosures of study authors, visit

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