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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 \u22651 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\u2019s potentially favorable impact on health-related quality of life, supporting its use as frontline treatment for Ph+ ALL.<\/jats:p>","DOI":"10.1038\/s41375-025-02608-4","type":"journal-article","created":{"date-parts":[[2025,4,16]],"date-time":"2025-04-16T07:59:07Z","timestamp":1744790347000},"page":"1342-1350","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Patient-reported outcomes in Philadelphia chromosome-positive acute lymphoblastic leukemia patients treated with ponatinib or imatinib: results from the PhALLCON trial"],"prefix":"10.1038","volume":"39","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-3731-0769","authenticated-orcid":false,"given":"Ajibade","family":"Ashaye","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ling","family":"Shi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9564-4498","authenticated-orcid":false,"given":"Ibrahim","family":"Aldoss","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3275-5593","authenticated-orcid":false,"given":"Pau","family":"Montesinos","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pankit","family":"Vachhani","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vanderson","family":"Rocha","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cristina","family":"Papayannidis","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jessica T.","family":"Leonard","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maria R.","family":"Baer","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1042-6024","authenticated-orcid":false,"given":"Jose-Maria","family":"Ribera","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"James","family":"McCloskey","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9437-9151","authenticated-orcid":false,"given":"Jianxiang","family":"Wang","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Sujun","family":"Gao","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Deepali","family":"Rane","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shien","family":"Guo","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2025,4,16]]},"reference":[{"key":"2608_CR1","doi-asserted-by":"publisher","first-page":"12","DOI":"10.3322\/caac.21820","volume":"74","author":"RL Siegel","year":"2024","unstructured":"Siegel RL, Giaquinto AN, Jemal A. 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LS and ShG reports employment with Evidera, which received payment for the statistical analysis during the conduct of the study. IA reports honoraria from Takeda, Amgen, Pfizer, Jazz, Sobi, Kite, Syndax, and Adaptive; consulting\/advisory roles at Takeda, Amgen, Pfizer, Jazz, Sobi, Kite, Adaptive, and Syndax; participation in speakers bureau at Pfizer; and research funding from AbbVie and MacroGenics. PM reports consulting\/advisory roles at Takeda, Daiichi Sankyo, and Bristol Myers Squibb; participation in speakers bureau at Servier; and research funding from AbbVie, Takeda, Daiichi Sankyo, Novartis, and Servier. PV reports consulting\/advisory roles at Abbvie, Amgen, Blueprint Medicines, Cogent Biosciences, Incyte, CTI BioPharma, Corp (now Sobi), Genentech, Geron, GlaxoSmith Kline, Karyopharm, Merck, MorphoSys, Novartis, Pfizer, Stemline, and Takeda. VR reports consulting\/advisory roles at Pfizer, Takeda, AbbVie, Astellas, Kite, and Amgen. CP reports consulting\/advisory roles at AbbVie, Astellas, Novartis, Bristol Myers Squibb, Menarini Stemline, Blueprint Medicines, Incyte, GlaxoSmithKline, Amgen, Pfizer, and Janssen; and personal fees from Astellas, Novartis, Pfizer, and Amgen. JTL reports consulting\/advisory roles at Adaptive Biotechnologies, Kite\/Gilead, Pfizer, and Takeda; and support for travel, accommodations, and expenses from Adaptive Biotechnologies. MRB reports institutional research funding from AbbVie, Ascentage Pharma, Astellas, FORMA Therapeutics, Kite\/Gilead, Kura Oncology, and Takeda. J-MR reports consulting\/advisory roles at Incyte, Pfizer, Bristol Myers Squibb, Novartis, and Takeda; and research funding from Amgen. JM reports personal fees from Bristol Myers Squibb, Takeda, Blueprint Medicine, PharmaEssentia, CTI, GlaxoSmithKline, Incyte, Amgen, and Jazz Pharmaceuticals. JW reports an advisory role at Abbvie. SuG has no conflict of interest to disclose.","order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}},{"value":"All methods used in this study were performed in accordance with the relevant guidelines and regulations. The PhALLCON trial was conducted in compliance with United States (US) Title 21 Code of Federal Regulations (CFR) Parts 50, 54, 56, and 312; the International Council for Harmonisation (ICH) of Technical Requirements for Pharmaceuticals for Human Use Good Clinical Practice (GCP) Guideline E6; the Declaration of Helsinki; and applicable local regulations. The clinical study protocol and its amendments, as well as the informed consent form (ICF) and Investigator\u2019s Brochure, were reviewed and approved by the Institutional Review Board\/Independent Ethics Committee (IRB\/IEC) of the participating study centers. This study was conducted in compliance with the informed consent regulations outlined in GCP. All patients completed the initial consent process before enrolling to the study. During this process, the person obtaining consent explained the study objectives and potential risks and benefits to patients using the ICF approved by the IRB\/IEC. Patients were given adequate time to ask questions and make a voluntary decision to participate. Each patient signed and dated the ICF before any protocol-specific procedures were performed.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}}]}}