Therapeutic efficacy of Ponatinib

The PhALLCON study is a phase Ⅲ, randomized, international multicenter, open-label clinical trial designed to evaluate the efficacy and safety of ponatinib versus imatinib combined with reduced-intensity chemotherapy as a first-line treatment regimen for adult patients with newly diagnosed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL).

A total of 245 patients were enrolled in the study and randomized at a ratio of 2:1 to receive either ponatinib plus reduced-intensity chemotherapy or imatinib plus reduced-intensity chemotherapy. The median ages of patients in the ponatinib group and imatinib group were 54 years and 52 years, respectively. A total of 164 patients received ponatinib at an initial dose of 30 mg/day, while 81 patients received imatinib at an initial dose of 600 mg/day. All patients continuously received ponatinib or imatinib in combination with reduced-intensity chemotherapy during the induction, consolidation, and maintenance treatment phases. After the completion of combination therapy, patients continued to receive monotherapy with ponatinib or imatinib until relapse after complete remission, disease progression, hematopoietic stem cell transplantation, initiation of alternative therapy, or occurrence of intolerable toxic reactions.

The primary endpoint of the study was the rate of minimal residual disease (MRD)-negative complete remission at the end of induction therapy (after 3 treatment cycles). The key secondary endpoint of event-free survival has not yet reached statistical maturity.

FDA,2022.02