Updated findings from the pivotal phase 2 Ponatinib Ph+ ALL and CML Evaluation (PACE) trial found robust activity and sustained benefit for the investigational tyrosine kinase inhibitor ponatinib in heavily pretreated patients with accelerated or blast-phase chronic myeloid leukemia (CML) or Philadelphia chromosome–positive (Ph+) acute lymphoblastic leukemia (ALL). Twelve-month data were presented for 179 patients who were resistant to or intolerant of dasatinib or nilotinib, or had the T315I mutation.
Major hematologic response, the primary end point, was as follows: for accelerated-phase patients, 58% for the resistant group and 50% for the mutation group; for blast-phase and Ph+ ALL patients, 35% for the resistant group and 33% for the mutated group. Cytologic responses were obtained in 52%, 67%, 40%, and 43%, respectively. Progression-free survival at 12 months was 55% for patients with accelerated-phase CML and 15% for those with blast-phase/Ph+ ALL, while overall survival was 84% and 33%, respectively.
Reference
Kantarjian HM, Kim D-W, Pinilla-Ibarz J; the PACE Study Group. Efficacy and safety of ponatinib in patients with accelerated phase or blast phase chronic myeloid leukemia (AP-CML or BP-CML) or Philadelphia chromosome–positive acute lymphoblastic leukemia (PH+ ALL): 12-month follow-up of the PACE trial. Presented at: 54th American Society of Hematology Annual Meeting; December 8-11, 2012; Atlanta, GA. Abstract 915.
