Patients' Perspectives on the Definition of Cure in Chronic Myeloid Leukemia: A US Based Survey

Abstract Introduction: The development of tyrosine kinase inhibitors (TKIs) has markedly improved the prognosis of patients (pts) with chronic myeloid leukemia (CML), with the perception by healthcare professionals that this is now a chronic disease to be managed. However, the need for continuous TKI therapy may result in ongoing toxicities, limits on fertility, and financial hardship. The H. Jean Khoury Cure CML consortium (HJKC3) is a collaborative effort of physicians and researchers at 17 academic centers. The HJKC3-001 2017 Patient Survey sought to define pts' expectations for treatment in CML to serve as a guidepost for future research in this area. Methods: Pts with CML were recruited by HJKC3 physicians, CML advocacy groups, and social media. An online survey platform (Qualtrics®) was used to obtain informed consent and administer the questionnaire. The anonymous survey was designed to gauge priorities for research in CML, understand patient definitions of cure, and elicit patient interest in future directions for CML therapy. Patient demographic and health characteristics were also collected. The data were analyzed using descriptive statistics. Results: Of the 458 pts who completed the survey, the median age of respondents was 54 years (range 18-81); 88% of pts identified as non-Hispanic white, 2% as non-Hispanic black, 2% as non-Hispanic Asian, 4% as Hispanic, and 4% other. Patients rated their overall health as poor (4%), fair (18%), good (40%), very good (28%) and excellent (9%). All but one respondent said that more research was needed for CML, with pts indicating their preferences for where they considered the need was greatest (Table 1). Overwhelmingly, 94% of respondents considered cure in CML as not taking any more pills. All but three respondents had received treatment with a TKI, with 26% (n=119) of pts having previously stopped their TKI medication for at least one month. When presented with the possibility of stopping all future treatment for CML with additional treatment, 97% of pts were willing to add another oral medication to their TKI while 89% of pts would accept intravenous treatment in addition to a TKIs. Half of the pts had discussed treatment discontinuation with their physician, with 45% considering this option in an attempt at treatment-free-remission. Of the pts that stopped taking their TKIs for at least one month, 65% did so because of side effects and another 10% because of cost. Conclusion: This survey demonstrates that pts do not consider disease control with life-long oral medication as cure; rather, cure requires the absence of treatment. Overwhelmingly, pts indicated the importance of continuing CML research with an ultimate goal of treatment-free cure. The advent of oral TKIs has been a tremendous success for pts with this disease. Nevertheless, it remains a source of disruption in pts' lives, particularly through side effects and costs. The HJKC3 was initiated with the goal of curing CML. Disclosures Atallah: Novartis: Consultancy; Jazz: Consultancy; Pfizer: Consultancy; BMS: Consultancy; Abbvie: Consultancy. Mauro:Bristol-Myers Squibb: Consultancy; Pfizer: Consultancy; Takeda: Consultancy; Novartis: Consultancy, Research Funding. Goldberg:COTA Inc.: Employment, Equity Ownership. Cortes:Daiichi Sankyo: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; Pfizer: Consultancy, Research Funding; Astellas Pharma: Consultancy, Research Funding; Arog: Research Funding. Deininger:Pfizer: Consultancy, Membership on an entity's Board of Directors or advisory committees; Blueprint: Consultancy. Druker:ARIAD: Research Funding; Third Coast Therapeutics: Membership on an entity's Board of Directors or advisory committees; Patient True Talk: Consultancy; Amgen: Membership on an entity's Board of Directors or advisory committees; MolecularMD: Consultancy, Equity Ownership, Membership on an entity's Board of Directors or advisory committees; Novartis Pharmaceuticals: Research Funding; Henry Stewart Talks: Patents & Royalties; Leukemia & Lymphoma Society: Membership on an entity's Board of Directors or advisory committees, Research Funding; McGraw Hill: Patents & Royalties; Aptose Therapeutics: Consultancy, Equity Ownership, Membership on an entity's Board of Directors or advisory committees; Cepheid: Consultancy, Membership on an entity's Board of Directors or advisory committees; GRAIL: Consultancy, Membership on an entity's Board of Directors or advisory committees; Bristol-Meyers Squibb: Research Funding; Oregon Health & Science University: Patents & Royalties; Gilead Sciences: Consultancy, Membership on an entity's Board of Directors or advisory committees; Monojul: Consultancy; Vivid Biosciences: Membership on an entity's Board of Directors or advisory committees; Blueprint Medicines: Consultancy, Equity Ownership, Membership on an entity's Board of Directors or advisory committees; Millipore: Patents & Royalties; Fred Hutchinson Cancer Research Center: Research Funding; Beta Cat: Membership on an entity's Board of Directors or advisory committees; ALLCRON: Consultancy, Membership on an entity's Board of Directors or advisory committees; Aileron Therapeutics: Consultancy; Celgene: Consultancy. Larson:Novartis: Consultancy, Research Funding; Ariad/Takeda: Consultancy, Research Funding; Pfizer: Consultancy, Research Funding; BristolMyers Squibb: Consultancy, Research Funding. Lipton:Bristol-Myers Squibb: Consultancy, Research Funding; ARIAD: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; Pfizer: Consultancy, Research Funding. Ritchie:Incyte: Consultancy, Speakers Bureau; NS Pharma: Research Funding; Bristol-Myers Squibb: Research Funding; Astellas Pharma: Research Funding; ARIAD Pharmaceuticals: Speakers Bureau; Novartis: Consultancy, Other: Travel, Accommodations, Expenses, Research Funding, Speakers Bureau; Pfizer: Consultancy, Research Funding; Celgene: Consultancy, Other: Travel, Accommodations, Expenses, Speakers Bureau. Shah:Bristol-Myers Squibb: Research Funding; ARIAD: Research Funding. Sweet:Celgene: Honoraria, Speakers Bureau; Jazz: Speakers Bureau; Celgene: Honoraria, Speakers Bureau; Agios: Consultancy; Phizer: Consultancy; Astellas: Consultancy; Astellas: Consultancy; Jazz: Speakers Bureau; Phizer: Consultancy; BMS: Honoraria; Novartis: Consultancy, Honoraria, Speakers Bureau; Agios: Consultancy; Novartis: Consultancy, Honoraria, Speakers Bureau; BMS: Honoraria.