Thihan Padukkavidana, PhD, from Takeda Oncology, discusses what physicians can learn about chronic myeloid leukemia (CML) treatment based on recent data from the OPTIC trial, an ongoing open label phase 2 study testing different dosages of ponatinib for safety and efficacy. Post-hoc analysis data from this trial was recently presented at ASH 2021. 

CML is a rare blood cancer characterized by the unregulated growth of myeloid cells in the bone marrow and their accumulation in the blood. Approximately 15% of new leukemia cases are CML. CML is considered very difficult to treat. Each year, there are about 8,450 new cases of CML in the United States and 1,130 will die of CML.

The OPTIC trial is an ongoing open label phase 2 study testing different dosages of ponatinib for safety and efficacy. At ASH 2021, post-hoc analysis data demonstrated clinical benefit across all 3 dosing regimens of ponatinib (45mg, 30mg and 15mg) regardless of T315I mutation status at baseline, which is in line with previous analyses and supports the benefit of using ponatinib post-2nd generation TKI failure for patients with resistant disease.

As Dr. Padukkavidana explains, the post-hoc analysis data demonstrate the safety and efficacy of the response-based dosing regimen used in the OPTIC trial. Previously, there have been concerns about the safety of ponatinib, particularly with regard to treatment-emergent arterial occlusive events. What the recent data presentation was able to demonstrate was that using a response-based dosing regimen, physicians can efficiently and effectively treat patients who are resistant to care which maximizes benefits while mitigating these safety concerns.

For more information about CML, Ph+ ALL, and other rare cancers, visit checkrare.com/diseases/cancers/