Hematologist-Oncologist Preferences for Treating Newly Diagnosed Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ ALL) using Tyrosine Kinase Inhibitors in Combination with Chemotherapy: A Discrete Choice Experiment
Author(s)
Ajibade Ashaye, MBA, MPH, MSc, MD1, Natasha Ramachandran, MSc2, Matthew Quaife, PhD2, Yanyu Wu, PhD3, Alvaro Alberto Gutierrez Vargas, PhD2, Angelica Jiongco, MSc2, Vamsi Kota, MBBS4, Bipin Savani, MD3, Caitlin Thomas, BSc, MSc2;
1Takeda Pharmaceuticals Inc., Director, Cambridge, MA, USA, 2Evidera, London, United Kingdom, 3Takeda Development Center Americas, Inc., Cambridge, MA, USA, 4Augusta University, Augusta, GA, USA
1Takeda Pharmaceuticals Inc., Director, Cambridge, MA, USA, 2Evidera, London, United Kingdom, 3Takeda Development Center Americas, Inc., Cambridge, MA, USA, 4Augusta University, Augusta, GA, USA
Presentation Documents
OBJECTIVES: The treatment landscape for newly-diagnosed Ph+ ALL is evolving, with FDA approval of the first TKI (ponatinib) in combination with chemotherapy in 2024. This study aimed to understand trade-offs that physicians are willing to make between benefits and risks when making frontline treatment decisions.
METHODS: A twelve-task, online discrete choice experiment was conducted among hematologist-oncologist physicians in the US, who chose between hypothetical profiles with varied levels of minimal residual disease-negative complete remission (MRD-negative CR; 15%-35%), arterial occlusive events (AOE; 1%- 3%), grade 3-4 hepatoxicity (10%-25%) and grade 3-4 hematotoxicity (65%-85%). Respondents were first asked to make treatment choices for a 45-year-old patient, with an ECOG performance status score of 0, without comorbidities; then in each task they were also asked to make choices for patient profiles who were either aged 65+, have an ECOG score of 3, diabetes, or hypertension. Mixed multinomial logit models were used to analyze choice data. Clinically-relevant relative attribute importance (cRAI) scores and minimum acceptable increases in MRD-negative CR required to tolerate increased risks were calculated.
RESULTS: Participants comprised 121 physicians predominantly working in academic (44%) or community (38%) hospitals, in urban (54%) or suburban (42%) areas. Across all patient profiles, improving MRD-negative CR was most important to physicians (cRAI=74.2%), followed by avoiding hepatoxicity (cRAI=14.8%), AOEs (cRAI=9.0%) and hematotoxicity (cRAI=2.0%). To tolerate a 1% increase in the risk of an AOE, hematotoxicity, or hepatoxicity, physicians required an increase in MRD-negative CR of 2.1%, 0.3% or 0.4%, respectively. Considering the different patient profiles, physicians placed more importance on hepatoxicity for patients with an ECOG status score of 3 (cRAI=20.6%) and on AOEs for patients with diabetes (cRAI=19.8%).
CONCLUSIONS: Despite varied relative importance for treatment benefits and risks across the different patient profiles, increasing MRD-negative CR remained the primary consideration for physicians, irrespective of the patient profile.
METHODS: A twelve-task, online discrete choice experiment was conducted among hematologist-oncologist physicians in the US, who chose between hypothetical profiles with varied levels of minimal residual disease-negative complete remission (MRD-negative CR; 15%-35%), arterial occlusive events (AOE; 1%- 3%), grade 3-4 hepatoxicity (10%-25%) and grade 3-4 hematotoxicity (65%-85%). Respondents were first asked to make treatment choices for a 45-year-old patient, with an ECOG performance status score of 0, without comorbidities; then in each task they were also asked to make choices for patient profiles who were either aged 65+, have an ECOG score of 3, diabetes, or hypertension. Mixed multinomial logit models were used to analyze choice data. Clinically-relevant relative attribute importance (cRAI) scores and minimum acceptable increases in MRD-negative CR required to tolerate increased risks were calculated.
RESULTS: Participants comprised 121 physicians predominantly working in academic (44%) or community (38%) hospitals, in urban (54%) or suburban (42%) areas. Across all patient profiles, improving MRD-negative CR was most important to physicians (cRAI=74.2%), followed by avoiding hepatoxicity (cRAI=14.8%), AOEs (cRAI=9.0%) and hematotoxicity (cRAI=2.0%). To tolerate a 1% increase in the risk of an AOE, hematotoxicity, or hepatoxicity, physicians required an increase in MRD-negative CR of 2.1%, 0.3% or 0.4%, respectively. Considering the different patient profiles, physicians placed more importance on hepatoxicity for patients with an ECOG status score of 3 (cRAI=20.6%) and on AOEs for patients with diabetes (cRAI=19.8%).
CONCLUSIONS: Despite varied relative importance for treatment benefits and risks across the different patient profiles, increasing MRD-negative CR remained the primary consideration for physicians, irrespective of the patient profile.
Conference/Value in Health Info
2025-05, ISPOR 2025, Montréal, Quebec, CA
Value in Health, Volume 28, Issue S1
Code
PCR208
Topic
Patient-Centered Research
Disease
SDC: Oncology, SDC: Rare & Orphan Diseases