Clinical lymphoma, myeloma & leukemia · 2025
Molecular Milestones and Survival Outcomes of Ponatinib Treatment in Patients With Chronic Myeloid Leukemia and Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia: A Real-World Analysis.
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Tseng Tai-Chung, Chang Yu-Sung, Tsai Xavier Cheng-Hong, Lin Ming-En, Tien Feng-Ming, Huang Huai-Hsuan, Lin Yun-Chu, Liu Jia-Hau, Liu Ming-Chih, Lin Chien-Chin, Cheng Chieh-Lung, Hsu Szu-Chun, Yao Ming, Tseng Mei-Hsuan, Peng Yen-Ling, Ko Bor-Sheng, Yang Yung-Li, Jou Shiann-Tarng, Hou Hsin-An, Chou Wen-Chien
Аннотация
BACKGROUND: Ponatinib, a third-generation tyrosine kinase inhibitor, has demonstrated its activity against chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). However, real-world data, particularly in Asian populations, remain limited.
METHODS: Patients with CML or Ph+ ALL treated with ponatinib in Taiwan between March 2013 and November 2024 were retrospectively collected. Molecular and cytogenetic data, efficacy outcomes (complete hematologic response [CHR], molecular response [MR], progression-free survival [PFS], overall survival [OS]), and adverse events were analyzed.
RESULTS: Among 52 patients (27 CML and 25 Ph+ ALL; median follow-up: 50.7 months), 94% received ponatinib due to relapsed or refractory disease. Among patients without prior CHR, 89% reached CHR at 12 months. Among patients without prior MR, 57% had MR2.0, 53% MR3.0, and 26% MR5.0 at 12 months. MR2.0 at 6 months and MR3.0 at 12 months correlated with improved outcome in patients with CML. Additional chromosomal abnormalities (ACAs) were identified in 36% of patients and were associated with inferior survival, whereas kinase domain mutations in 78% of studied patients, including T315I (57%), did not affect the outcome. MR3.0 at any time predicted superior OS and PFS in patients with CML, and MR5.0 with superior PFS in patients with Ph+ ALL. One patient (1.9%) had an arterial occlusive event.
CONCLUSION: Ponatinib demonstrated substantial real-world efficacy in pretreated patients with CML and Ph+ ALL, with MR3.0 in CML and MR5.0 in Ph+ ALL emerging as favorable prognostic markers. In contrast, the presence of ACAs was associated with shorter survival.
METHODS: Patients with CML or Ph+ ALL treated with ponatinib in Taiwan between March 2013 and November 2024 were retrospectively collected. Molecular and cytogenetic data, efficacy outcomes (complete hematologic response [CHR], molecular response [MR], progression-free survival [PFS], overall survival [OS]), and adverse events were analyzed.
RESULTS: Among 52 patients (27 CML and 25 Ph+ ALL; median follow-up: 50.7 months), 94% received ponatinib due to relapsed or refractory disease. Among patients without prior CHR, 89% reached CHR at 12 months. Among patients without prior MR, 57% had MR2.0, 53% MR3.0, and 26% MR5.0 at 12 months. MR2.0 at 6 months and MR3.0 at 12 months correlated with improved outcome in patients with CML. Additional chromosomal abnormalities (ACAs) were identified in 36% of patients and were associated with inferior survival, whereas kinase domain mutations in 78% of studied patients, including T315I (57%), did not affect the outcome. MR3.0 at any time predicted superior OS and PFS in patients with CML, and MR5.0 with superior PFS in patients with Ph+ ALL. One patient (1.9%) had an arterial occlusive event.
CONCLUSION: Ponatinib demonstrated substantial real-world efficacy in pretreated patients with CML and Ph+ ALL, with MR3.0 in CML and MR5.0 in Ph+ ALL emerging as favorable prognostic markers. In contrast, the presence of ACAs was associated with shorter survival.