Overall survival with ponatinib versus allogeneic stem cell transplantation in Philadelphia chromosome-positive leukemias with the T315I mutation
Managing Principals Mei Sheng Duh, M.P.H., Sc.D., and Maral DerSarkissian, Ph.D., collaborated with Franck E. Nicolini, M.D., Ph.D., and a larger team of world-renowned hematologists/oncologists to study the effectiveness of ponatinib, a drug used to treat Philadelphia chromosome-positive (Ph+) leukemias (i.e., chronic myeloid leukemia [CML] and acute lymphoblastic leukemia [ALL]) with the threonine-to-isoleucine mutation at codon 315 (T315I). The findings of the study, which was a collaborative effort with ARIAD Pharmaceuticals, Inc. (now part of Takeda Pharmaceutical Company Limited), were originally published in Cancer and were subsequently reported by ASH Online, a prestigious online journal covering hematology oncology.
To date, few effective treatment options have been developed for patients with Ph+ CML or Ph+ ALL with the T315I mutation or relapsed/refractory disease. The research team found that treatment with ponatinib significantly improved overall survival for patients with T315I-positive chronic-phase CML, compared with hematopoietic cell transplantation (HCT). They also found that patients with advanced acute disease who underwent HCT had better survival than those who received ponatinib. The analysis, based on data from the phase II PACE trial and the European Bone Marrow Transplant (EBMT) registry, has implications for prolonging survival among those suffering from chronic-phase CML.