Hagop Kantarjian, MD, from The University of Texas MD Anderson Cancer Center, discusses data on the efficacy and safety of ponatinib in chronic-phase chronic myeloid leukemia (CML) patients. This data was presented at ASH 2020.

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 PACE trial, which was completed in January 2019, demonstrated a significant and durable antileukemic response in chronic-phase CML patients after treatment with ponatinib. The two year survival rate for chronic-phase CLL patients was 85%. However, post hoc analysis suggested a relationship between high levels of toxicity in higher doses of ponatinib. 

The OPTIC trial, initiated in June 2015, is an ongoing, open label phase II study testing different dosages of ponatinib for safety and efficacy. The two year survival rate for the OPTIC trial is currently at 93%.

Interim analysis of data from both the PACE and OPTIC trials suggests very low toxicity in the OPTIC trial compared with the PACE trial, but ponatinib remains favorable overall compared to other tyrosine kinase inhibitors (TKIs) for the treatment of patients with chronic phase CML who failed prior 2nd generation TKIs, regardless of a patient’s mutation status.

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