Abstract 4512: Impact of clinical response and treatment tolerability on HRQoL in patients treated with ponatinib or imatinib for newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL)

Abstract In the PhALLCON trial (NCT03589326), ponatinib demonstrated superior minimal residual disease (MRD)-negative complete remission (CR) rate, patient-reported treatment tolerability, and health-related quality of life (HRQoL) over imatinib in combination with reduced intensity chemotherapy in adults with newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL). To further substantiate the effect of treatment benefits on HRQoL, we assessed the effect of clinical response and treatment tolerability on changes in HRQoL over time in PhALLCON trial patients. HRQoL was assessed using prespecified primary domains from the Functional Assessment of Cancer Therapy-Leukemia (FACT-Leu) questionnaire and the EQ-5D-5L. Treatment tolerability was assessed using the FACT-GP5 item “bothered by treatment side effects”. Linear mixed-effects regression models were used to examine changes in HRQoL over time, with CR status and treatment tolerability as time-varying predictors, while controlling for significant covariates. The analysis included 238 patients (ponatinib 159, imatinib 79). Achieving CR was linked to significant HRQoL improvement in all FACT-Leu domains and the EQ-5D visual analogue scale (EQ-VAS), with clinically meaningful improvements in the FACT-Leu Trial Outcome Index, FACT-G Total Score, and FACT-Leu Total Score domains (Table). Treatment-related side effects led to significant and clinically meaningful deterioration in all scales. Higher levels of “bother” were associated with greater worsening in HRQoL. Given the 2-fold higher MRD-negative CR rate of ponatinib compared to imatinib, these analyses further support the HRQoL benefit of ponatinib over imatinib in patients with Ph+ ALL. Citation Format: 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, Yanyu Wu, Meliessa Hennessy, Alexander Vorog, Shien Guo. Impact of clinical response and treatment tolerability on HRQoL in patients treated with ponatinib or imatinib for newly diagnosed Philadelphia chromosome-positive (Ph+) acute lymphoblastic leukemia (ALL) [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2025; Part 1 (Regular Abstracts); 2025 Apr 25-30; Chicago, IL. Philadelphia (PA): AACR; Cancer Res 2025;85(8_Suppl_1):Abstract nr 4512.