Contributions
Abstract: EP1175
Type: E-Poster Presentation
Session title: Quality of life, palliative care, ethics and health economics
Background
Currently available second and third generation tyrosine kinase inhibitors (TKIs), offer a variety of treatment options for chronic-phase chronic myeloid leukemia (CP-CML) patients and prescribing physicians. Indirect comparisons suggest the efficacy of these TKIs are similar, however each have distinct dosing requirements and safety profiles. Patient preferences for TKI dosing and safety have yet to be quantified, but are valuable for optimizing healthcare decisions and outcomes.
Aims
The aim of this study was to quantify CP-CML patient preferences and perspectives regarding treatment attributes for TKI therapies.
Methods
US-based patients (N=200) with self-reported CP-CML completed an online discrete choice experiment. Choice tasks were based on key treatment attributes: 1) dosing requirements (number of tablets and consumption with/without food), and incidence of 2) nausea, 3) fatigue, 4) pain in muscles and/or joints, 5) diarrhea, 6) blockage to a major blood vessel and 7) pleural effusion. Attributes were identified from the published literature, approved TKI labels, qualitative interviews with CML patients (N=12) and consultation with clinical experts. Hierarchical Bayesian estimation was used to calculate part-worth utilities to estimate relative importance (RI) and category-level preference of each attribute. Latent class analysis investigated preference patterns among patients.
Results
The sample included 149 females and 51 males (median age 54 years). Patients had been diagnosed for <1 year (n=31), 1-5 years (n=74), >5-10 years (n=47) and >10 years (n=48). Current treatments included dasatinib (n=62), imatinib (n=61), nilotinib (n=43), bosutinib (n=16) and ponatinib (n=1). Blockage to a major blood vessel emerged with the highest RI: 34%. RI of remaining attributes were comparable: pleural effusion (12%); fatigue (12%); pain in muscles and/or joints (11%); dosing requirements (11%); diarrhea (10%); nausea (9%). Similar findings were observed in demographic (gender, age) and clinical characteristic (time since diagnosis, current TKI) subgroups. Evaluation of mean part-worth utilities for individual attribute levels indicated preference for once daily dosing compared with twice daily dosing on an empty stomach. Lower incidences of diarrhea (20% vs 85%) were preferred, but duration of diarrhea (1-5 days vs 10+ days) had limited impact on preferences. Latent class analysis identified a number of distinct groups of participants which varied with respect to those attributes driving preferences in each group. Blockage to a major blood vessel was important in all groups, but was not always the most important attribute.
Conclusion
The likelihood of experiencing serious adverse events (blockage to a major blood vessel) was a key driver of CP-CML treatment preferences. However, heterogeneity in treatment preference patterns highlight the importance of discussions regarding dosing and treatment toxicities for optimizing individual treatment decisions.
Keyword(s): Chronic myeloid leukemia, Tyrosine kinase inhibitor
Abstract: EP1175
Type: E-Poster Presentation
Session title: Quality of life, palliative care, ethics and health economics
Background
Currently available second and third generation tyrosine kinase inhibitors (TKIs), offer a variety of treatment options for chronic-phase chronic myeloid leukemia (CP-CML) patients and prescribing physicians. Indirect comparisons suggest the efficacy of these TKIs are similar, however each have distinct dosing requirements and safety profiles. Patient preferences for TKI dosing and safety have yet to be quantified, but are valuable for optimizing healthcare decisions and outcomes.
Aims
The aim of this study was to quantify CP-CML patient preferences and perspectives regarding treatment attributes for TKI therapies.
Methods
US-based patients (N=200) with self-reported CP-CML completed an online discrete choice experiment. Choice tasks were based on key treatment attributes: 1) dosing requirements (number of tablets and consumption with/without food), and incidence of 2) nausea, 3) fatigue, 4) pain in muscles and/or joints, 5) diarrhea, 6) blockage to a major blood vessel and 7) pleural effusion. Attributes were identified from the published literature, approved TKI labels, qualitative interviews with CML patients (N=12) and consultation with clinical experts. Hierarchical Bayesian estimation was used to calculate part-worth utilities to estimate relative importance (RI) and category-level preference of each attribute. Latent class analysis investigated preference patterns among patients.
Results
The sample included 149 females and 51 males (median age 54 years). Patients had been diagnosed for <1 year (n=31), 1-5 years (n=74), >5-10 years (n=47) and >10 years (n=48). Current treatments included dasatinib (n=62), imatinib (n=61), nilotinib (n=43), bosutinib (n=16) and ponatinib (n=1). Blockage to a major blood vessel emerged with the highest RI: 34%. RI of remaining attributes were comparable: pleural effusion (12%); fatigue (12%); pain in muscles and/or joints (11%); dosing requirements (11%); diarrhea (10%); nausea (9%). Similar findings were observed in demographic (gender, age) and clinical characteristic (time since diagnosis, current TKI) subgroups. Evaluation of mean part-worth utilities for individual attribute levels indicated preference for once daily dosing compared with twice daily dosing on an empty stomach. Lower incidences of diarrhea (20% vs 85%) were preferred, but duration of diarrhea (1-5 days vs 10+ days) had limited impact on preferences. Latent class analysis identified a number of distinct groups of participants which varied with respect to those attributes driving preferences in each group. Blockage to a major blood vessel was important in all groups, but was not always the most important attribute.
Conclusion
The likelihood of experiencing serious adverse events (blockage to a major blood vessel) was a key driver of CP-CML treatment preferences. However, heterogeneity in treatment preference patterns highlight the importance of discussions regarding dosing and treatment toxicities for optimizing individual treatment decisions.
Keyword(s): Chronic myeloid leukemia, Tyrosine kinase inhibitor