Introduction: Completion of the Pediatric Liver Transplant Quality of Life (PeLTQL) questionnaire, a patient-reported outcome measure for pediatric liver transplant (LT) recipients, generates a total and three subdomain scores (Social Emotional, Coping & Adjustment and Future Health). Published methodologies exist to derive the minimally clinically important difference (MCID), referring to the change in score that reflects a meaningful change for the patient. We used two established approaches to derive the MCID values for the PeLTQL.

Method: All self- and proxy- reported PeLTQL surveys completed during routine surveillance visits at SickKids LT clinic were reviewed. Surveys with data from two sequential visits were included in analysis. Anchor and distribution-based (0.5 standard deviation, standard error of measurement) methods were used to ascertain MCID values for PeLTQL. Given the retrospective study design, an internal anchor question was used. 

Results: PeLTQL data from 65 LT recipients (26 [40%] male, 17 [42%] biliary atresia, median age at LT 3.08 years [IQR 0.99 - 7.30]), and their caregivers were included for analysis.  Median patient age at time of baseline PeLTQL completion was 9.05 (IQR 5.70 - 11.43) years. The MCID for self-PeLTQL total scores ranged from 4.53 to 8.10 and from 4.49 to 8.75 for caregiver responses (Table 1).

Conclusion: The results of this study may guide clinicians in the interpretation of PeLTQL results with attention to respondents with a change in score of >4.5 points. Prospective multi-center studies utilizing an external anchor question will add to knowledge about MCID values for the PeLTQL.