Vaso-occlusive painful events in sickle cell disease: Impact on child well-being

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.