TY - JOUR
T1 - Treatment preferences of patients with relapsed or refractory multiple myeloma in the United States, United Kingdom, Italy, Germany, France, and Spain
T2 - results from a discrete choice experiment
AU - Thomas, Caitlin
AU - Ailawadhi, Sikander
AU - Popat, Rakesh
AU - Kleinman, David
AU - Ross, Melissa M.
AU - Gorsh, Boris
AU - Mulnick, Sarah
AU - O’Neill, Alicia
AU - Paka, Prani
AU - Hanna, Maya
AU - Krucien, Nicolas
AU - Molinari, Alexa
AU - Gelhorn, Heather L.
AU - Perera, Sue
N1 - Publisher Copyright:
Copyright © 2023 Thomas, Ailawadhi, Popat, Kleinman, Ross, Gorsh, Mulnick, O’Neill, Paka, Hanna, Krucien, Molinari, Gelhorn and Perera.
PY - 2023
Y1 - 2023
N2 - Introduction: Newer treatment options for relapsed/refractory multiple myeloma (RRMM) with efficacy and safety profiles that differ from traditional therapies have facilitated personalized management strategies to optimize patient outcomes. In the context of such personalized management, understanding how treatment characteristics influence patients’ preferences is essential. This study assessed patients’ preferences for RRMM treatment attributes and determined trade-offs between potential benefits, administration procedures, and adverse effects. Methods: Patients’ preferences were evaluated using a discrete choice experiment (DCE). Patients with RRMM who reported failing two lines of anti-myeloma treatment (immunomodulatory agent and a proteasome inhibitor [PI]) or ≥ 3 lines (including ≥1 PI, immunomodulatory agent, or anti-CD38 monoclonal antibody), were recruited across the US, UK, Italy, Germany, France, and Spain. DCE attributes and levels were identified using a targeted literature review, a review of clinical data for relevant RRMM treatments, qualitative patient interviews, and input from clinical and myeloma patient experts. The DCE was administered within an online survey from February–June 2022. Preference data were analyzed using an error-component logit model and willingness to make trade-offs for potential benefits, and relative attribute importance scores were calculated. Results: Overall, 296 patients from the US (n = 100), UK (n = 49), Italy (n = 45), Germany (n = 43), France (n = 39), and Spain (n = 20) participated in the DCE. Mean (standard deviation) age was 63.8 (8.0) years, 84% had a caregiver, and patients had a median of 3 (range: 2–8) prior lines of therapy. Efficacy attributes most influenced patients’ preferences, with increasing overall response rate (25–85%) and overall survival (6 months to 2 years) contributing to ~50% of treatment decision-making. Administration procedures were also considered important to patients. Avoiding individual side effects was considered relatively less important, with patients willing to tolerate increases in side effects for gains in efficacy. Patient characteristics such as rate of disease progression, sociodemographics, or clinical characteristics also influenced treatment preferences. Conclusion: Patients with RRMM were willing to tolerate increased risk of side effects for higher efficacy. Preferences and risk tolerance varied between patients, with preference patterns differing by certain patient characteristics. This highlights the importance of shared decision-making for optimal treatment selection and patient outcomes.
AB - Introduction: Newer treatment options for relapsed/refractory multiple myeloma (RRMM) with efficacy and safety profiles that differ from traditional therapies have facilitated personalized management strategies to optimize patient outcomes. In the context of such personalized management, understanding how treatment characteristics influence patients’ preferences is essential. This study assessed patients’ preferences for RRMM treatment attributes and determined trade-offs between potential benefits, administration procedures, and adverse effects. Methods: Patients’ preferences were evaluated using a discrete choice experiment (DCE). Patients with RRMM who reported failing two lines of anti-myeloma treatment (immunomodulatory agent and a proteasome inhibitor [PI]) or ≥ 3 lines (including ≥1 PI, immunomodulatory agent, or anti-CD38 monoclonal antibody), were recruited across the US, UK, Italy, Germany, France, and Spain. DCE attributes and levels were identified using a targeted literature review, a review of clinical data for relevant RRMM treatments, qualitative patient interviews, and input from clinical and myeloma patient experts. The DCE was administered within an online survey from February–June 2022. Preference data were analyzed using an error-component logit model and willingness to make trade-offs for potential benefits, and relative attribute importance scores were calculated. Results: Overall, 296 patients from the US (n = 100), UK (n = 49), Italy (n = 45), Germany (n = 43), France (n = 39), and Spain (n = 20) participated in the DCE. Mean (standard deviation) age was 63.8 (8.0) years, 84% had a caregiver, and patients had a median of 3 (range: 2–8) prior lines of therapy. Efficacy attributes most influenced patients’ preferences, with increasing overall response rate (25–85%) and overall survival (6 months to 2 years) contributing to ~50% of treatment decision-making. Administration procedures were also considered important to patients. Avoiding individual side effects was considered relatively less important, with patients willing to tolerate increases in side effects for gains in efficacy. Patient characteristics such as rate of disease progression, sociodemographics, or clinical characteristics also influenced treatment preferences. Conclusion: Patients with RRMM were willing to tolerate increased risk of side effects for higher efficacy. Preferences and risk tolerance varied between patients, with preference patterns differing by certain patient characteristics. This highlights the importance of shared decision-making for optimal treatment selection and patient outcomes.
KW - benefit–risk
KW - discrete choice experiment
KW - multiple myeloma
KW - patient preferences
KW - trade-offs
KW - treatment attributes
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U2 - 10.3389/fmed.2023.1271657
DO - 10.3389/fmed.2023.1271657
M3 - Article
AN - SCOPUS:85178940435
SN - 2296-858X
VL - 10
JO - Frontiers in Medicine
JF - Frontiers in Medicine
M1 - 1271657
ER -