Real-world outcomes of ponatinib in heavily pretreated patients with chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia

To evaluate the real-world efficacy and safety of ponatinib, we conducted a multicenter, prospective post-marketing surveillance study in patients with chronic phase (CP), accelerated phase (AP), or blast phase (BP) chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL), who were resistant or intolerant to prior tyrosine kinase inhibitors (TKIs) or harbored the T315I mutation. A total of 148 patients were enrolled across 24 sites: 115 with CML (92 CP-CML, 12 AP-CML, 11 BP-CML) and 33 with Ph + ALL. Ponatinib was administered as third-line therapy in 70 patients (47.3%), and 127 (85.8%) initiated treatment at 45 mg once daily. Sixty-three patients (42.6%) began ponatinib due to resistance or intolerance to prior TKIs. In CP-CML, the overall major molecular response (MMR) rate was 43.2% (35/81), with a cumulative MMR incidence of 23.9% at week 24 among patients without prior MMR. In Ph + ALL, the overall MMR and complete cytogenetic response (CCyR) rates were 82.4% (14/17) and 75.0% (3/4), respectively. CP-CML patients receiving ponatinib as earlier-line therapy achieved higher MR4.5 rates than those treated later (47.4% vs. 16.1%, p = 0.011). The most common adverse drug reactions were rash (21.6%, 32/148), hypertension (12.2%, 18/148), and pyrexia (10.1%, 15/148); two vascular occlusive events occurred. Ponatinib showed favorable real-world efficacy and manageable safety in Korean patients. Early, high-dose therapy suggests a trend toward deep molecular responses, supporting its clinical benefit in real-world practice. (ClinicalTrials.gov Identifier: NCT03709017; registered October 15, 2018).

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Journal
Annals of Hematology
Published
2026-05-22
DOI
https://doi.org/10.1007/s00277-026-07053-6
Primary Topic
Chronic Myeloid Leukemia Treatments
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article
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article

Real-world outcomes of ponatinib in heavily pretreated patients with chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia

Chul Won Jung, Jieun Uhm, Jee Hyun Kong, Deog‐Yeon Jo et al.
Annals of Hematology
Chronic Myeloid Leukemia Treatments
article

Real-world outcomes of ponatinib in heavily pretreated patients with chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia

Chul Won Jung, Jieun Uhm, Jee Hyun Kong, Deog‐Yeon Jo, June‐Won Cheong, So Young Chong, Jeong‐A Kim, Dae Young Zang, Sewon Lee, Inho Kim, Won Sik Lee, Jae-Yong Kwak, Je-Hwan Lee, Seok Lee, Hawk Kim, Min Kyoung Kim, Sung-Eun Lee, Sang Kyun Sohn, Sung-Soo Yoon, Jae Joon Han, Hyeoung-Joon Kim, Da Jung Kim, Dong-Wook Kim, Yunsuk Choi, Min-Yi Lee, Ho-Jin Shin
article en

Abstract

To evaluate the real-world efficacy and safety of ponatinib, we conducted a multicenter, prospective post-marketing surveillance study in patients with chronic phase (CP), accelerated phase (AP), or blast phase (BP) chronic myeloid leukemia (CML) or Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph + ALL), who were resistant or intolerant to prior tyrosine kinase inhibitors (TKIs) or harbored the T315I mutation. A total of 148 patients were enrolled across 24 sites: 115 with CML (92 CP-CML, 12 AP-CML, 11 BP-CML) and 33 with Ph + ALL. Ponatinib was administered as third-line therapy in 70 patients (47.3%), and 127 (85.8%) initiated treatment at 45 mg once daily. Sixty-three patients (42.6%) began ponatinib due to resistance or intolerance to prior TKIs. In CP-CML, the overall major molecular response (MMR) rate was 43.2% (35/81), with a cumulative MMR incidence of 23.9% at week 24 among patients without prior MMR. In Ph + ALL, the overall MMR and complete cytogenetic response (CCyR) rates were 82.4% (14/17) and 75.0% (3/4), respectively. CP-CML patients receiving ponatinib as earlier-line therapy achieved higher MR4.5 rates than those treated later (47.4% vs. 16.1%, p = 0.011). The most common adverse drug reactions were rash (21.6%, 32/148), hypertension (12.2%, 18/148), and pyrexia (10.1%, 15/148); two vascular occlusive events occurred. Ponatinib showed favorable real-world efficacy and manageable safety in Korean patients. Early, high-dose therapy suggests a trend toward deep molecular responses, supporting its clinical benefit in real-world practice. (ClinicalTrials.gov Identifier: NCT03709017; registered October 15, 2018).

Annals of Hematology
Otsuka (Japan) (JP), Ulsan College (KR), Ewha Womans University (KR), Yonsei University (KR), Eulji University (KR), Severance Hospital (KR), Kyungpook National University Hospital (KR), Kosin University Gospel Hospital (KR), Asan Medical Center (KR), Samsung Medical Center (KR), Seoul National University Hospital (KR), Kyung Hee University (KR), University of Ulsan (KR), Gachon University Gil Medical Center (KR), The Catholic University of Korea St. Vincent's Hospital (KR), Korea Pharma (South Korea) (KR), Wonju Severance Christian Hospital (KR), Inje University Busan Paik Hospital (KR), Kyung Hee University Medical Center (KR), Pusan National University Hospital (KR), Otsuka (South Korea) (KR), CHA University Bundang Medical Center (KR), Hanyang University Seoul Hospital (KR), Chungnam National University Hospital (KR), Chonnam National University Hwasun Hospital (KR), CHA University (KR), Hallym University Sacred Heart Hospital (KR), The Catholic University of Korea Seoul St. Mary's Hospital (KR), Yeungnam University (KR), Jeonbuk National University (KR), Sungkyunkwan University (KR), Catholic University of Korea (KR)
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Chronic Myeloid Leukemia Treatments
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