Background: The Pediatric Cardiac Quality of Life Inventory (PCQLI) is a disease-specific pediatric cardiac quality of life (QOL) instrument that is reliable, valid and generalizable internationally. Responsiveness represents the change in QOL over time that would be expected to occur when clinical status changes. Validating responsiveness is critical to use of the PCQLI to assess for changes in QOL longitudinally and after interventions. The purpose of this study was to demonstrate PCQLI responsiveness in the pediatric cardiac population. Methods: Pediatric patients from 10 centers who underwent arrhythmia ablation, heart transplantation, or valve surgery (pulmonary valve replacement, RV-to-PA conduit replacement, aortic or mitral valve surgery) who were 8-18 years were enrolled. Patient and parent-proxy PCQLI Total, Disease Impact (DI) and Psychosocial Impact (PI) subscale scores were assessed pre-procedure and 3-12 months post-procedure. Clinical status of the patient was assessed by a clinician at the follow-up visit and rated markedly improved, improved, no change, worse, or much worse. Clinical status was dichotomized into markedly improved/improved and no change/worse/much worse for analysis. Paired t-tests were used to examine change over time. Holm method of correction for multiple comparisons was utilized to assess for statistical significance. Results: There were 195 patient/parent-proxies who participated in the study. Patients were: 12.6 + 3.0 years; white (82%); male (56%); time to follow-up was 7.0 + 2.5 months; procedural groups: 79 (41%) ablation, 28 (14%) heart transplant, 88 (45%) valve surgery. Eighty-four patients (43%) had markedly improved clinical status, 80 (41%) were improved, 27 (14%) were unchanged, and 4 (2%) were worse at follow-up. PCQLI patient and parent-proxies scores increased significantly (p≤0.013) in almost all subscales (Table 1). All PCQLI scores were significantly higher (p<0.001) in the markedly improved/improved clinical status group at follow-up, while there were no differences in any of the PCQLI scores in the no difference/worse/much worse group at follow-up (Table 2). Patients experiencing marked improvement/improved clinical status at follow-up and their parent-proxies reported an 8.7 (CI: 6.4 – 10.9) and 9.2 (CI: 7.0 – 11.4) point increase in their PCQLI Total scores, respectively. Patients who did not have improved clinical status at follow-up and their parent-proxies reported a 4.0 (CI: 0.0-7.9) and 1.7 (CI: -2.9 to 6.3) point increase in PCQLI Total scores, respectively. Conclusion: The PCQLI shows responsiveness in QOL in the pediatric cardiac arrhythmia, transplant, and valve intervention populations. Patients who had improved clinical status post-procedure and their parent-proxies reported a significant increase in QOL compared to patients who did not improve clinically post-procedure and their parent-proxies. Given these data the PCQLI may be used as a patient reported outcome measure for future longitudinal follow-up and interventional trials as a means to assess meaningful clinical impact from the patient and parent-proxy perspectives.

Table 2.

Change in PCQLI Scores from baseline to 3 to 12 months post-procedure by clinical status

Change in PCQLI Scores from baseline to 3 to 12 months post-procedure by clinical status
Change in PCQLI Scores from baseline to 3 to 12 months post-procedure by clinical status
Table 1.

Change in PCQLI Scores from baseline to 3 to 12 months post-procedure by procedural group

Change in PCQLI Scores from baseline to 3 to 12 months post-procedure by procedural group
Change in PCQLI Scores from baseline to 3 to 12 months post-procedure by procedural group