Ponatinib Currently Not Indicated As First Line CML Chronic Phase Therapy

MedicalResearch.com Interview with:

Prof Jeffrey H Lipton, PhD, MD, FRCPC  Princess Margaret Cancer Centre Toronto, ON Canada

Prof. Jeffrey Lipton

Prof Jeffrey H Lipton, PhD, MD, FRCPC
Princess Margaret Cancer Centre
Toronto, ON Canada

MedicalResearch.com: What is the background and purpose for this study?

Dr. Lipton: Ponatinib is a third generation tyrosine kinase inhibitor that has been shown to be extremely effective in treating patients with chronic myeloid leukemia resistant to other drugs.  Because of this, it was decided to look at it in newly diagnosed patients in a randomized study against imatinib.  The study was terminated prematurely because of evidence of vascular toxicity that became evident in the phase 1 and 2 studies of ponatinib in previously treated patients with resistant disease.  

MedicalResearch.com: What are the main findings?

Dr. Lipton: The current study demonstrated superior responses to ponatinib at all levels of depth and speed, but nothing could be determined about durability or survival because of the early termination.  A higher incidence of vascular toxicities was also seen when the results obtained were analyzed.  As a result, ponatinib is currently not indicated for first line therapy in CML chronic phase.

MedicalResearch.com: What should clinicians and patients take away from your report?

Dr. Lipton: It is hoped that current studies looking at lower doses or planned dose reductions in resistant patients will demonstrate ongoing efficacy and reduced toxicity.  This may open the door for renewed interest in a study in newly diagnosed patients, but using lower doses of ponatinib.


Ponatinib versus imatinib for newly diagnosed chronic myeloid leukaemia: an international, randomised, open-label, phase 3 trial

Lipton, Jeffrey H et al.

The Lancet Oncology , Volume 0 , Issue 0
DOI: http://dx.doi.org/10.1016/S1470-2045(16)00080-2
Published Online: 12 April 2016

[wysija_form id=”5″]

Last Updated on April 13, 2016 by Marie Benz MD FAAD