Real-world outcomes of ponatinib in heavily pretreated patients with chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia
Year: 2026
Type: article
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).
Source: Annals of Hematology
Authors: Seok Lee, Hyeoung-Joon Kim, June-Won Cheong, Ho-Jin Shin, Jee Hyun Kong, Sang Kyun Sohn, Sung-Soo Yoon, Dae Young Zang, Chul Won Jung, Jeong-A Kim, Sung-Eun Lee, Won Sik Lee, Yunsuk Choi, Inho Kim, Jae Joon Han, Min Kyoung Kim, So Young Chong, Da Jung Kim, Da Jung Kim, Deog-Yeon Jo, Hawk Kim, Jae-Yong Kwak, Je-Hwan Lee, Jieun Uhm, Sewon Lee, Min-Yi Lee, Dong-Wook Kim, Dong-Wook Kim
Institutions: The Catholic University of Korea Seoul St. Mary's Hospital, Ewha Womans University Mokdong Hospital, Catholic University of Korea, Gachon University Gil Medical Center, Chonnam National University Hwasun Hospital, Gachon University Medical Center, Yonsei University, Severance Hospital, Yonsei University Health System, Pusan National University Hospital, Pusan National University, Wonju Severance Christian Hospital, Kyungpook National University Hospital, Seoul National University, Seoul National University Hospital, Hallym University Sacred Heart Hospital, Samsung Medical Center, Sungkyunkwan University, The Catholic University of Korea St. Vincent's Hospital, Inje University, Inje University Busan Paik Hospital, Inje University Paik Hospital, Asan Medical Center, University of Ulsan, Kyung Hee University, Yeungnam University Medical Center, Yeungnam University College, Yeungnam University, CHA University Bundang Medical Center, CHA University, Eulji University, Kosin University Gospel Hospital, Eulji University Uijeongbu Eulji Medical Center, Kosin University, Chungnam National University, Chungnam National University Hospital, Jeonbuk National University Hospital, Jeonbuk National University, Hanyang University Seoul Hospital, Hanyang University, Otsuka (South Korea)
Language: English
Subfield: Hematology
Field: Medicine
Domain: Health Sciences
Open Access status: gold
Estimated APC paid: $4,590
OpenAlex ID: W7162135663