Minimal Clinically Important Difference for the PeLTQL in Pediatric Liver Transplantation: A Multicenter Analysis

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.

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.

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).

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.

ClinicalTrials.gov Identifier: NCT05241847.