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Quantifying Benefit-Risk Trade-Offs Toward Prophylactic Treatment Among Adult Patients With Hemophilia A in China: Discrete Choice Experiment Study

BACKGROUND: Hemophilia A is a chronic condition that requires meticulous treatment and management. Patient preferences for prophylactic treatment can substantially influence adherence, outcomes, and quality of life, yet these preferences remain underexplored, particularly in China. OBJECTIVE: This s...

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Autores principales: Wang, Limin, Liu, Shimeng, Jiang, Shan, Li, Chaofan, Lu, Liyong, Fang, Yunhai, Li, Shunping
Formato: Online Artículo Texto
Lenguaje:English
Publicado: JMIR Publications 2023
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10413247/
https://www.ncbi.nlm.nih.gov/pubmed/37494098
http://dx.doi.org/10.2196/45747
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author Wang, Limin
Liu, Shimeng
Jiang, Shan
Li, Chaofan
Lu, Liyong
Fang, Yunhai
Li, Shunping
author_facet Wang, Limin
Liu, Shimeng
Jiang, Shan
Li, Chaofan
Lu, Liyong
Fang, Yunhai
Li, Shunping
author_sort Wang, Limin
collection PubMed
description BACKGROUND: Hemophilia A is a chronic condition that requires meticulous treatment and management. Patient preferences for prophylactic treatment can substantially influence adherence, outcomes, and quality of life, yet these preferences remain underexplored, particularly in China. OBJECTIVE: This study aimed to investigate the preferences for prophylactic treatment among Chinese adult patients with hemophilia A without inhibitors, considering clinical effectiveness, side effects, dosing mode, and dosing frequency. METHODS: A discrete choice experiment was used to elicit patient preferences for prophylactic treatment of hemophilia. The study was conducted across 7 provinces in China with socioeconomic and geographical diversity. Subgroup analysis was performed according to education level, geographic location, and treatment type, alongside the exploration of benefit-risk trade-offs. RESULTS: A total of 113 patients completed the discrete choice experiment questionnaire, and we included 102 responses for analysis based on predetermined exclusion criteria. The study found that patients prioritized reducing annual bleeding times and avoiding the risk of developing inhibitors over treatment process attributes. Subgroup analysis revealed that lower-educated patients and those from rural areas attached more importance to the dosing mode, likely due to barriers to self-administration. Patients demonstrated a clear understanding of benefit-risk trade-offs, exhibiting a willingness to accept an increased risk of developing inhibitors for improved clinical outcomes. CONCLUSIONS: This study provides valuable insights into the preferences of patients with hemophilia A for prophylactic treatment in China. Understanding these preferences can enhance shared decision-making between patients and clinicians, fostering personalized prophylactic treatment plans that may optimize adherence and improve clinical outcomes.
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spelling pubmed-104132472023-08-11 Quantifying Benefit-Risk Trade-Offs Toward Prophylactic Treatment Among Adult Patients With Hemophilia A in China: Discrete Choice Experiment Study Wang, Limin Liu, Shimeng Jiang, Shan Li, Chaofan Lu, Liyong Fang, Yunhai Li, Shunping JMIR Public Health Surveill Original Paper BACKGROUND: Hemophilia A is a chronic condition that requires meticulous treatment and management. Patient preferences for prophylactic treatment can substantially influence adherence, outcomes, and quality of life, yet these preferences remain underexplored, particularly in China. OBJECTIVE: This study aimed to investigate the preferences for prophylactic treatment among Chinese adult patients with hemophilia A without inhibitors, considering clinical effectiveness, side effects, dosing mode, and dosing frequency. METHODS: A discrete choice experiment was used to elicit patient preferences for prophylactic treatment of hemophilia. The study was conducted across 7 provinces in China with socioeconomic and geographical diversity. Subgroup analysis was performed according to education level, geographic location, and treatment type, alongside the exploration of benefit-risk trade-offs. RESULTS: A total of 113 patients completed the discrete choice experiment questionnaire, and we included 102 responses for analysis based on predetermined exclusion criteria. The study found that patients prioritized reducing annual bleeding times and avoiding the risk of developing inhibitors over treatment process attributes. Subgroup analysis revealed that lower-educated patients and those from rural areas attached more importance to the dosing mode, likely due to barriers to self-administration. Patients demonstrated a clear understanding of benefit-risk trade-offs, exhibiting a willingness to accept an increased risk of developing inhibitors for improved clinical outcomes. CONCLUSIONS: This study provides valuable insights into the preferences of patients with hemophilia A for prophylactic treatment in China. Understanding these preferences can enhance shared decision-making between patients and clinicians, fostering personalized prophylactic treatment plans that may optimize adherence and improve clinical outcomes. JMIR Publications 2023-07-26 /pmc/articles/PMC10413247/ /pubmed/37494098 http://dx.doi.org/10.2196/45747 Text en ©Limin Wang, Shimeng Liu, Shan Jiang, Chaofan Li, Liyong Lu, Yunhai Fang, Shunping Li. Originally published in JMIR Public Health and Surveillance (https://publichealth.jmir.org), 26.07.2023. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work, first published in JMIR Public Health and Surveillance, is properly cited. The complete bibliographic information, a link to the original publication on https://publichealth.jmir.org, as well as this copyright and license information must be included.
spellingShingle Original Paper
Wang, Limin
Liu, Shimeng
Jiang, Shan
Li, Chaofan
Lu, Liyong
Fang, Yunhai
Li, Shunping
Quantifying Benefit-Risk Trade-Offs Toward Prophylactic Treatment Among Adult Patients With Hemophilia A in China: Discrete Choice Experiment Study
title Quantifying Benefit-Risk Trade-Offs Toward Prophylactic Treatment Among Adult Patients With Hemophilia A in China: Discrete Choice Experiment Study
title_full Quantifying Benefit-Risk Trade-Offs Toward Prophylactic Treatment Among Adult Patients With Hemophilia A in China: Discrete Choice Experiment Study
title_fullStr Quantifying Benefit-Risk Trade-Offs Toward Prophylactic Treatment Among Adult Patients With Hemophilia A in China: Discrete Choice Experiment Study
title_full_unstemmed Quantifying Benefit-Risk Trade-Offs Toward Prophylactic Treatment Among Adult Patients With Hemophilia A in China: Discrete Choice Experiment Study
title_short Quantifying Benefit-Risk Trade-Offs Toward Prophylactic Treatment Among Adult Patients With Hemophilia A in China: Discrete Choice Experiment Study
title_sort quantifying benefit-risk trade-offs toward prophylactic treatment among adult patients with hemophilia a in china: discrete choice experiment study
topic Original Paper
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10413247/
https://www.ncbi.nlm.nih.gov/pubmed/37494098
http://dx.doi.org/10.2196/45747
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