Onkológia 6/2020

Real life data on ponatinib treatment of chronic myeloid leukemia patients in Slovakia

Ponatinib is a very effective oral tyrosine kinase inhibitor (TKI) of unmutated and mutated BCR-ABL, including variant T315I. Ponatinib is indicated in patiens with chronic myeloid leukemia (CML) who are resistant or do not tolerate previous treatment with second generation TKI and when imatinib can not be used. Ponatinib is very effective TKI even in very heavily pretreated patients. However an increased incidence of cardiovascular diseases such as arterial hypertension and vascular occlusive disease have been observed. We present the results of a retrospective analysis of patients with CML treated with ponatinib in routine clinical practice outside of clinical trials. We performed the analyses in cooperation with the Institute of Biostatistics and Analyses in Brno, Czech Republic. Primary objective: To collect and analyse data on treatment with ponatinib in patients with CML /Ph+ ALL in Slovakia. Study design and study population: This study was a retrospective, non-interventional study of patients treated with ponatinib. The treatment efficacy was evaluated according to ELN guidelines with the evaluation of hematologic, cytogenetic and molecular response. Analysed population consists of 22 CML CF patients The age median at the start of treatment was 61,6 years. The study population of patients was heavily pretreated with several previous lines of TKI. 4 patients had T315I mutation. 54,4% of patients had a history of another comorbidities. Patients most often started with daily dose 45 mg. Results: Almost half of CP CML patients achieved MMR (45,5%). Ponatinib showed its efficacy with 27,3% probability of achieving MMR after 1 year of therapy in CP-CML patients. Disease progression was the major reason for treatment termination. Arterial hypertension developed in 4 and coronary artery disease in 2 patients. Conclusion: Ponatinib has an important position in management of patients with CML that are resistant to other TKI. Doctors should consider risk versus benefit of the treatment for patients with a cardiovascular anamnesis.

Keywords: ponatinib, chronic myeloid leukemia, tyrosine kinase inhibitor, resistant, mutations