Minimal Clinically Important Difference for the PeLTQL in Pediatric Liver Transplantation: A Multicenter Analysis
Vicky L Ng1, Karina Kwan1, Sherrie Logan2
1Division of Gastroenterology, Hepatology, and Nutrition, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada.
Insights
Establishing minimal clinically important difference (MCID) values for the Pediatric Liver Transplant Quality of Life (PeLTQL) questionnaire is crucial for pediatric liver transplant recipients. Changes greater than 6 points in PeLTQL total scores may indicate a need for closer clinical attention.
Area of Science:
- Pediatric transplantation outcomes
- Patient-reported outcome measures
- Quality of life assessment
Background:
- Patient-reported outcomes (PROs) are vital in chronic disease management, including pediatric liver transplantation.
- Clinicians need clear thresholds to interpret changes in PRO scores for meaningful clinical impact.
- The Starzl Network Patient Reported Outcomes (SPaRO) study aimed to establish minimal clinically important difference (MCID) values for the Pediatric Liver Transplant Quality of Life (PeLTQL) questionnaire.
Purpose of the Study:
- To derive MCID estimates for the PeLTQL questionnaire in pediatric liver transplant recipients.
- To provide clinicians with thresholds for interpreting changes in quality of life scores.
- To enhance the clinical utility of longitudinal PRO data in this population.
Main Methods:
- The SPaRO study recruited pediatric liver transplant recipients (aged 8-18) at least 1 year post-transplant from 7 SNEPT sites.
- Participants and caregivers completed the PeLTQL questionnaire twice using a mobile or web-based platform.
- MCIDs were estimated using anchor-based, distribution-based, and predictive modeling methods.
Main Results:
- 98 patients and 86 caregivers completed assessments.
- Triangulated MCID values for the PeLTQL total score were 6.4 (self-report) and 5.6 (proxy-report).
- Predictive modeling estimated MCID values at 7.7 (self-report) and 6.8 (proxy-report).
Conclusions:
- MCID thresholds for the PeLTQL total score are approximately 6-8 points (self-report) and 6-7 points (proxy-report).
- A change greater than 6 points in the PeLTQL total score may signify a clinically meaningful improvement or decline.
- Prospective validation of these MCID values is ongoing.
Background:
Patient-reported outcomes are increasingly recognized as essential complements to biological indices in chronic disease including pediatric liver transplantation. However, clinicians lack clear thresholds to determine when changes in patient-reported outcome measure (PROM) scores represent clinically meaningful change. Establishing minimal clinically important difference (MCID) values is critical to interpret longitudinal PROM data and guide action. The Starzl Network Patient Reported Outcomes (SPaRO) study provided a unique opportunity to derive MCID estimates of the Pediatric Liver Transplant Quality of Life (PeLTQL) questionnaire.
Methods:
In SPaRO, English- or Spanish-speaking pediatric LT recipients aged 8-18 years and at least 1 year post-LT were recruited from 7 Starzl Network for Excellence in Pediatric Transplantation (SNEPT) sites. Participants and caregivers completed the PeLTQL (self- and proxy-report) questionnaire twice via a mobile application or web-based platform. MCIDs were estimated using anchor-based and distribution-based methods including predictive modeling.
Results:
A total of 98 patients and 86 caregivers completed two PeLTQL assessments between March 2022 and October 2023. Triangulated MCID values for the total score (TS) were 6.4 (self-report) and 5.6 (proxy-report). Predictive modeling yielded estimates of 7.7 (self) and 6.8 (proxy).
Conclusions:
In this multicenter cohort, MCID thresholds for the PeLTQL TS were approximately 6-8 points (self-report) and 6-7 points (proxy-report), consistent with prior single-center estimates. Changes in the PeLTQL TS of greater than 6 points may help clinicians identify patients who warrant closer attention. Prospective validation using an independent anchor is underway.
Trial Registration:
ClinicalTrials.gov Identifier: NCT05241847.
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