<p>In the Phase 3 PhALLCON trial (NCT03589326), ponatinib demonstrated superior efficacy and comparable safety profile versus imatinib in adults with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL). Here we report patient-reported outcomes (PRO) from PhALLCON assessed as exploratory endpoints using the Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu) and EQ-5D-5L. Primary PRO domains included FACT-G physical well-being, FACT-Leu subscale (FACT-LeuS), Trial Outcome Index (TOI), FACT-Leu total score, FACT-G total score, and EQ-5D visual analogue scale. Differences in least-squares mean score changes from baseline to the end of induction (EOI)/consolidation (EOC) and time to confirmed improvement/deterioration were analyzed. Overall treatment tolerability was assessed using the FACT-GP5. Analyses included 238 patients (ponatinib 159, imatinib 79) with ≥1 PRO assessment. Least-squares mean changes from baseline favored ponatinib, with significant and meaningful differences in FACT-LeuS, TOI, and FACT-Leu total score at EOI and across the primary domains except for FACT-LeuS at EOC. Median time to confirmed improvement was shorter with ponatinib versus imatinib for key measures. Ponatinib-treated patients tended to report being less bothered by treatment side effects as assessed by FACT-GP5. These findings highlight ponatinib’s potentially favorable impact on health-related quality of life, supporting its use as frontline treatment for Ph+ ALL.</p>

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Patient-reported outcomes in Philadelphia chromosome-positive acute lymphoblastic leukemia patients treated with ponatinib or imatinib: results from the PhALLCON trial

  • Ajibade Ashaye,
  • Ling Shi,
  • Ibrahim Aldoss,
  • Pau Montesinos,
  • Pankit Vachhani,
  • Vanderson Rocha,
  • Cristina Papayannidis,
  • Jessica T. Leonard,
  • Maria R. Baer,
  • Jose-Maria Ribera,
  • James McCloskey,
  • Jianxiang Wang,
  • Sujun Gao,
  • Deepali Rane,
  • Shien Guo

摘要

In the Phase 3 PhALLCON trial (NCT03589326), ponatinib demonstrated superior efficacy and comparable safety profile versus imatinib in adults with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL). Here we report patient-reported outcomes (PRO) from PhALLCON assessed as exploratory endpoints using the Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu) and EQ-5D-5L. Primary PRO domains included FACT-G physical well-being, FACT-Leu subscale (FACT-LeuS), Trial Outcome Index (TOI), FACT-Leu total score, FACT-G total score, and EQ-5D visual analogue scale. Differences in least-squares mean score changes from baseline to the end of induction (EOI)/consolidation (EOC) and time to confirmed improvement/deterioration were analyzed. Overall treatment tolerability was assessed using the FACT-GP5. Analyses included 238 patients (ponatinib 159, imatinib 79) with ≥1 PRO assessment. Least-squares mean changes from baseline favored ponatinib, with significant and meaningful differences in FACT-LeuS, TOI, and FACT-Leu total score at EOI and across the primary domains except for FACT-LeuS at EOC. Median time to confirmed improvement was shorter with ponatinib versus imatinib for key measures. Ponatinib-treated patients tended to report being less bothered by treatment side effects as assessed by FACT-GP5. These findings highlight ponatinib’s potentially favorable impact on health-related quality of life, supporting its use as frontline treatment for Ph+ ALL.