A study of prospective registry data supported routine ponatinib use in patients with chronic myeloid leukemia (CML) or Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) who are resistant or intolerant to treatment with tyrosine kinase inhibitors (TKIs). These findings were published in the Annals of Hematology.

Ponatinib was authorized for use in Europe in 2013; however, few studies have assessed its routine use. To better understand the real-world clinical outcomes, patients with CML or Ph+ ALL treated with ponatinib at 20 sites in Belgium between 2016 and 2019 were assessed for outcomes.

This study involved 50 patients, median age 58 years (range, 19 to 83), 38% were women, and 46% received 3 or more prior lines of TKIs. Most patients had CML (33).

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Ponatinib was administered due to intolerance, relapse or refractoriness, disease progression, and mutational status in 42%, 27%, 18%, and 12%, respectively, of patients with CML; and 35%, 30%, 12%, and 24%, respectively, of patients with Ph+ ALL.

After ponitinib treatment for a median 380 days in the CML cohort and a median 123 days in the Ph+ ALL cohorts, major molecular response was achieved by 58% and 41% of patients with CML and Ph+ ALL, respectively. A single patient in each cohort achieved BCR-ABL1 mRNA £1%.

Estimated overall survival was 85.3% in the CML cohort and 85.6% in the Ph+ ALL cohort over 3 years, and estimated progression-free survival was 81.6% and 48.9%, respectively.

More than half (68%) of patients reported adverse reactions, most commonly rash and dry skin. A total of 5 deaths occurred, none of which were attributed to ponatinib treatment.

This study was limited by its small sample size.

The study authors concluded that observations made during routine clinical practice were similar to previously reported data and supported the use of ponatinib in patients with Ph+ ALL or CML who experienced resistance or intolerance with TKIs.

Disclosure: Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.


Devos T, Havelange V, Theunissen K, et al. Clinical outcomes in patients with Philadelphia chromosome-positive leukemia treated with ponatinib in routine clinical practice—data from a Belgian registry. Ann Hematol. 2021;100(7):1723-1732. doi:10.1007/s00277-021-04507-x

This article originally appeared on Oncology Nurse Advisor