Objectives To determine clinical and health-related quality of life outcomes, and to derive an “ideal” composite profile of children alive 10 years after pediatric liver transplantation (LT) performed in the US and Canada. Study design This was a multicenter cross-sectional analysis characterizing patients enrolled in the Studies of Pediatric Liver Transplantation database registry who have survived >10 years from LT. Results A total of 167 10-year survivors were identified, all of whom received daily immunosuppression therapy. Comorbidities associated with the post-LT course included post-transplantation lymphoproliferative disease (in 5% of patients), renal dysfunction (9%), and impaired linear growth (23%). Health-related quality of life, as assessed by the PedsQL 4.0 Generic Core Scales, revealed lower patient self-reported total scale scores for 10-year survivors compared with matched healthy children (77.2 ± 12.9 vs 84.9 ± 11.7; P < .001). At 10 years post-LT, only 32% of patients achieved an ideal profile of a first allograft stable on immunosuppression monotherapy, normal growth, and absence of common immunosuppression-induced sequelae. Conclusion Success after pediatric LT has moved beyond patient survival. Availability of an ideal composite profile at follow-up provides opportunities for patients, families, and healthcare providers to identify broader sets of outcomes at earlier stages, ultimately contributing to improved outcomes after pediatric LT.

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The measurement properties of the newly developed Pediatric Quality of Life Inventory TM (PedsQL TM ) 3.0 Transplant Module in pediatric solid organ transplant recipients were evaluated. Participants included pediatric recipients of liver, kidney, heart and small bowel transplantation who were cared for at seven medical centers across the United States and their parents. Three hundred and thirty-eight parents of children ages 2-18 and 274 children ages 5-18 completed both the PedsQL TM 4.0 Generic Core Scales and the Transplant Module. Findings suggest that child self-report and parent proxy-report scales on the Transplant Module demonstrated excellent reliability (total scale score for child self-report a = 0.93; total scale score for parent proxy-report a = 0.94). Transplant-specific symptoms or problems were significantly correlated with lower generic HRQOL, supporting construct validity. Children with solid organ transplants and their parents reported statistically significant lower generic HRQOL than healthy children. Parent and child reports showed moderate to good agreement across the scales. In conclusion, the PedsQL TM Transplant Module demonstrated excellent initial feasibility, reliability and construct validity in pediatric patients with solid organ transplants.

Non-adherence with recommended immunosuppressant medications is common post-pediatric liver transplant and is the most important reason for organ rejection in long-term survivors. However, there is currently no validated, standard method to measure adherence, with a well-defined threshold, making it extremely difficult to evaluate interventions to improve adherence. Previous studies have suggested that the degree of fluctuation of medication blood levels over time can provide an idea about how regularly the medication is being taken. The present study, conducted at UCLA medical center, sought to identify a specific threshold value of the s.d. of individual tacrolimus blood levels in pediatric liver transplant recipients which would be associated with rejection episodes in these patients. A threshold of 3.0 has been identified in other studies, and was supported by the analysis of retrospective data from 96 subjects. However, further analysis found that a s.d. of 2.5 appeared to have a better fit with the data. These findings suggest the utility of monitoring the s.d. of routine tacrolimus blood levels in pediatric liver transplant recipients for detecting non-adherence to immunosuppressant medication prior to clinical rejection, allowing earlier interventions.