Oncologists’ preferences for frontline TKI treatment of Ph+ acute lymphoblastic leukemia: a discrete choice experiment

ABSTRACT Aims To evaluate hematologist-oncologists’ preferences for frontline treatment of Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) using tyrosine kinase inhibitors (TKIs) + chemotherapy. Participants & methods An online discrete choice experiment was conducted among US-based hematologist-oncologists. Participants viewed profiles of hypothetical TKI + chemotherapy treatments with varied levels of benefit and risks (minimal residual disease-negative complete remission [MRD-negative CR], arterial occlusive events, grade 3–4 hepatotoxicity, grade 3–4 hematotoxicity) and chose their recommended treatment for five patient profiles: “less-complex” baseline; age ≥ 65; ECOG score 3; diabetes; hypertension. Data were analyzed using mixed multinomial logit models. Results 121 hematologist-oncologists participated. Increasing MRD−negative CR was most important to hematologist-oncologists, driving 65%–87% of decision-making across patient profiles. Relative importance of benefits/risks varied by patient profile. Pooled across patient profiles, hepatotoxicity was the most concerning risk, driving 14% of decision-making. Based on PhALLCON data and elicited preferences, hematologist-oncologists were predicted to select the profile of ponatinib + chemotherapy over imatinib + chemotherapy for all included patient profiles. Predicted probabilities of choosing ponatinib over imatinib ranged from 87%–98% across patient profiles. Conclusions Hematologist-oncologists prioritized achieving MRD-negative CR when recommending frontline treatments for Ph+ ALL, accepting some risks if offset by meaningful improvement in MRD-negative CR.