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<p>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&#xa0;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%, <i>p</i> = 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)</p>

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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年5月22日

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  • 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,
  • Deog-Yeon Jo,
  • Hawk Kim,
  • Jae-Yong Kwak,
  • Je-Hwan Lee,
  • Jieun Uhm,
  • Sewon Lee,
  • Min-Yi Lee,
  • Dong-Wook Kim

摘要

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)