Abstract：
OBJECTIVE: The Pediatric Liver Transplant Quality of Life (PeLTQL) questionnaire is a disease-specific patient reported outcome measure for pediatric liver transplant (LT) recipients. To-date, threshold values above which a change in PeLTQL score is considered meaningful to patients are unavailable. This study proposes the first values for the minimally clinically important difference (MCID) for the PeLTQL.
METHODS: In this retrospective cohort study, anchor and distribution-based methods were used to estimate the MCID for the PeLTQL. Questionnaires completed between March 2013, and July 2022 were included if data from two sequential visits were available. An internal anchor question was used for anchor-based determination of the MCID. A final MCID estimate was ascertained from triangulation of all methods.
RESULTS: PeLTQL data from 65 LT recipients (26 [40%] male, 17 [42%] biliary atresia, median age at LT 3.08 years [interquartile range 0.99-7.30]), and their caregivers were included for analysis. Median patient age at time of baseline PeLTQL completion was 13.84 (10.90-15.86) years. The MCID for self-PeLTQL total scores ranged from 4.53 to 8.46, and from 4.47 to 8.85 for proxy responses. By triangulation, the MCID of the PeLTQL total score was 6.45 and 6.78 for self and proxy responses respectively.
CONCLUSIONS: A change in PeLTQL score of 6.5 or more points suggests a change in health status that is meaningful to the patient, providing the clinical team an opportunity to engage the patient\'s voice in reassessing current health status and management strategies.
METHODS: In this retrospective cohort study, anchor and distribution-based methods were used to estimate the MCID for the PeLTQL. Questionnaires completed between March 2013, and July 2022 were included if data from two sequential visits were available. An internal anchor question was used for anchor-based determination of the MCID. A final MCID estimate was ascertained from triangulation of all methods.
RESULTS: PeLTQL data from 65 LT recipients (26 [40%] male, 17 [42%] biliary atresia, median age at LT 3.08 years [interquartile range 0.99-7.30]), and their caregivers were included for analysis. Median patient age at time of baseline PeLTQL completion was 13.84 (10.90-15.86) years. The MCID for self-PeLTQL total scores ranged from 4.53 to 8.46, and from 4.47 to 8.85 for proxy responses. By triangulation, the MCID of the PeLTQL total score was 6.45 and 6.78 for self and proxy responses respectively.
CONCLUSIONS: A change in PeLTQL score of 6.5 or more points suggests a change in health status that is meaningful to the patient, providing the clinical team an opportunity to engage the patient\'s voice in reassessing current health status and management strategies.
摘要：
目的：小儿肝移植生活质量（PeLTQL）问卷是儿童肝移植（LT）受者的疾病特异性患者报告结果指标。到目前为止，如果超过该阈值,PeLTQL评分的变化被认为对患者有意义,则无法获得该阈值.这项研究提出了PeLTQL的最小临床重要差异（MCID）的第一个值。
方法：在这项回顾性队列研究中，使用基于锚定和分布的方法来估计PeLTQL的MCID。如果可以获得两次连续访问的数据，则包括在2013年3月至2022年7月之间完成的问卷调查。内部锚问题用于MCID的基于锚的确定。从所有方法的三角测量确定最终的MCID估计值。
结果：来自65名LT接受者（26[40％]男性，17[42%]胆道闭锁,LT3.08岁的中位年龄[IQR0.99-7.30])，和他们的照顾者被纳入分析。基线时患者年龄中位数为13.84（10.90-15.86）岁。自我PeLTQL总分的MCID范围为4.53至8.46，而代理响应的MCID范围为4.47至8.85。通过三角测量,自我应答和代理应答的PeLTQL总分的MCID分别为6.45和6.78.
结论：PeLTQL评分6.5分或以上的变化表明健康状况的变化对患者有意义，为临床团队提供一个机会，让患者的声音重新评估当前的健康状况和管理策略。
方法：在这项回顾性队列研究中，使用基于锚定和分布的方法来估计PeLTQL的MCID。如果可以获得两次连续访问的数据，则包括在2013年3月至2022年7月之间完成的问卷调查。内部锚问题用于MCID的基于锚的确定。从所有方法的三角测量确定最终的MCID估计值。
结果：来自65名LT接受者（26[40％]男性，17[42%]胆道闭锁,LT3.08岁的中位年龄[IQR0.99-7.30])，和他们的照顾者被纳入分析。基线时患者年龄中位数为13.84（10.90-15.86）岁。自我PeLTQL总分的MCID范围为4.53至8.46，而代理响应的MCID范围为4.47至8.85。通过三角测量,自我应答和代理应答的PeLTQL总分的MCID分别为6.45和6.78.
结论：PeLTQL评分6.5分或以上的变化表明健康状况的变化对患者有意义，为临床团队提供一个机会，让患者的声音重新评估当前的健康状况和管理策略。
