Ponatinib-based therapy in adults with relapsed or refractory Philadelphia chromosome-positive acute lymphoblastic leukemia: results of the real-life OPAL study

Abstract

The OPAL study is a French multicenter observational retrospective analysis of adults with relapsed/refractory Philadelphia chromosome-positive acute lymphoblastic leukemia treated in a real-life setting by ponatinib. Twenty-nine patients were included since 2012. Median age was 55  years. The initial dose of ponatinib, combined to chemotherapy in half of the patients, was 45 mg/day in most instances. The remission rate was 90% and seven patients received allogeneic stem cell transplantation. Median disease-free and overall survival were only 3.5 and 9.9 months respectively. The outcome of patients with BCR-ABL mutation was similar to that of unmutated patients. With a median duration of exposure to ponatinib of 4 months, only 3 cardio-vascular events were recorded despite a high incidence of risk factors, and overall safety was acceptable. These results underline the place of ponatinib to induce early response in advanced disease and the need of new combinations to improve long-term outcome.

Keywords:

• Acute lymphoblastic leukemia
• Philadelphia chromosome
• ponatinib

Acknowledgements

The authors thank Ms Elodie Gadolet, CRA, for the collection of the data nationwide.

Author contributions

ST and FH designed the study in accordance with the GRAALL Scientific Board chaired by HD, analyzed the data and wrote the paper. MU, DB, XT, ER, TL, MPGH, AB, SL, YH, and PC enrolled patients and provided data. EB and SB provided methodological support and performed statistical analysis. VL coordinated data collection. All authors have proof read the manuscript and agree on its content.

Disclosure statement

No potential conflict of interest was reported by the author(s).