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Determining the minimally clinically important difference for the pediatric liver transplant quality of life
Simone Kortbeek1, Fatema T Johara2,3, Karina Kwan2,3
1Section of Pediatric Gastroenterology, Hepatology, and Nutrition, Alberta Children's Hospital, University of Calgary, Calgary, Alberta, Canada.
Insights
A change of 6.5 points or more on the Pediatric Liver Transplant Quality of Life (PeLTQL) questionnaire indicates a meaningful improvement for pediatric liver transplant recipients. This provides valuable data for assessing patient health status.
Area of Science:
- Pediatric Gastroenterology and Hepatology
- Clinical Outcomes Research
- Quality of Life Assessment
Background:
- The Pediatric Liver Transplant Quality of Life (PeLTQL) questionnaire measures outcomes in pediatric liver transplant (LT) recipients.
- Establishing minimally clinically important difference (MCID) values is crucial for interpreting score changes.
- Previous studies lacked defined MCID thresholds for the PeLTQL.
Purpose of the Study:
- To determine the first minimally clinically important difference (MCID) values for the Pediatric Liver Transplant Quality of Life (PeLTQL) questionnaire.
- To provide a benchmark for meaningful change in health status for pediatric LT recipients.
Main Methods:
- Retrospective cohort study utilizing anchor and distribution-based methods.
- Included sequential PeLTQL data from 65 pediatric liver transplant recipients (2013-2022).
- Employed an internal anchor question and triangulation of methods for MCID estimation.
Main Results:
- MCID for self-reported PeLTQL scores ranged from 4.53 to 8.46.
- MCID for proxy-reported PeLTQL scores ranged from 4.47 to 8.85.
- Triangulated MCID estimates were 6.45 for self-reports and 6.78 for proxy reports.
Conclusions:
- A change of 6.5 or more points in the PeLTQL score signifies a meaningful improvement in health status.
- This MCID value empowers clinical teams to incorporate patient perspectives into health status reassessment and management.
- The established MCID enhances the clinical utility of the PeLTQL in pediatric liver transplant care.
Purpose:
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.
Conclusion:
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.
