Chronic Myeloid Leukemia Coverage from Every Angle

Real-World Ponatinib Outcomes in Patients With CML From Nationwide Belgian Registry

By: Celeste L. Dixon
Posted: Tuesday, January 14, 2020

Real-world data appear to support the long-term efficacy and safety of ponatinib that have been reported in clinical trials, according to a Belgian team of investigators. Timothy Devos, MD, PhD, of Katholieke Universiteit Leuven, Belgium, and colleagues presented these results at the 2019 American Society of Hematology (ASH) Annual Meeting & Exposition (Abstract 4161).

The authors utilized a national registry to follow 33 patients with chronic myeloid leukemia (CML) and 17 with Philadelphia chromosome–positive acute lymphoblastic leukemia who began ponatinib in March 2016 or later. A total of 40% of these patients were intolerant to previous tyrosine kinase inhibitors, 28% were refractory to previous tyrosine kinase inhibitors, 16% had disease progression on previous therapy, and 16% had the T315I mutation. Most patients with CML and many patients with ALL obtained deep molecular responses, the investigators reported.

“Major molecular response was achieved as best response in 19 (58%) CML patients and 7 (41%) ALL patients, while 2 (6%) of CML and 3 (18%) ALL patients achieved complete cytogenetic response as best response,” wrote Dr. Devos and coauthors. The median follow-up for all patients, including those enrolled recently, was 15.0 and 4.5 months for patients with CML and ALL, respectively.

A total of 91% of patients with CML and 94% of those with ALL had received at least two prior tyrosine kinase inhibitors, and 54% of patients with CML and 29% of patients with ALL had received at least three prior tyrosine kinase inhibitors. Although they plan to continue follow-up to further assess the long-term clinical effectiveness of ponatinib in this real-life population, Dr. Devos and colleagues declared their support for its use in patients with these hematologic malignancies who are resistant or intolerant to previous tyrosine kinase inhibitors or have the T315I mutation, especially given that no new safety signals emerged for this agent.

Disclosure: For full disclosures of the study authors, visit

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