Abstract
BACKGROUND: This study describes how painful events affect the health-related quality of life (HRQL) of children with sickle cell disease (SCD) and determines the responsiveness of a generic HRQL measure in SCD. Our hypotheses were twofold: (1) HRQL is significantly impaired at presentation to the emergency department for a painful event and (2) PedsQL 4.0 Acute Version Generic Core Scales is responsive to change in the evolution of a painful event. PROCEDURE: This prospective cohort study included 57 children with SCD. HRQL was measured with the Acute Version of the PedsQL 4.0 Generic Core Scales, completed by child (self-report) and caregiver (proxy report) at presentation and 7 days post-discharge. Independent comparisons of HRQL scores were made between children in the study cohort and a published reference sample of children with SCD in baseline health (historical SCD controls). RESULTS: Median PedsQL scores at presentation were significantly lower than historical SCD controls in all domains for child self-report and all domains except social and school functioning in parent-proxy. Clinically and statistically significant changes in HRQL between presentation and post-discharge resulted in similar HRQL scores at 7 days post-discharge to historical SCD controls. CONCLUSIONS: The PedsQL is responsive to change; thus a useful tool to measure the impact of interventions in future SCD clinical trials. Painful events significantly diminish all domains of HRQL and this improves 7 days post-discharge.