BackgroundDetecting change in health status over time and ascertaining meaningful changes are critical elements when using health-related quality of life (HRQL) instruments to measure patient-centered outcomes. The PedsQL™ Sickle Cell Disease module, a disease specific HRQL instrument, has previously been shown to be valid and reliable. Our objectives were to determine the longitudinal validity of the PedsQL™ Sickle Cell Disease module and the change in HRQL that is meaningful to patients.MethodsAn ancillary study was conducted utilizing a multi-center prospective trial design. Children ages 4–21 years with sickle cell disease admitted to the hospital for an acute painful vaso-oclusive crisis were eligible. Children completed HRQL assessments at three time points (in the Emergency Department, one week post-discharge, and at return to baseline (One to three months post-discharge). The primary outcome was change in HRQL score. Both distribution (effect size, standard error of measurement (SEM)) and anchor (global change assessment) based methods were used to determine the longitudinal validity and meaningful change in HRQL. Changes in HRQL meaningful to patients were identified by anchoring the change scores to the patient’s perception of global improvement in pain.ResultsModerate effect sizes (0.20–0.80) were determined for all domains except the Communication I and Cognitive Fatigue domains. The value of 1 SEM varied from 3.8–14.6 across all domains. Over 50% of patients improved by at least 1 SEM in Total HRQL score. A HRQL change score of 7–10 in the pain domains represented minimal perceived improvement in HRQL and a HRQL change score of 18 or greater represented moderate to large improvement.ConclusionsThe PedsQL™ Sickle Cell Disease Module is responsive to changes in HRQL in patients experiencing acute painful vaso-occlusive crises. The study data establish longitudinal validity and meaningful change parameters for the PedsQL™ Sickle Cell Disease Module.Trial RegistrationClinicalTrials.gov (study identifier: NCT01197417). Date of registration: 08/30/2010Electronic supplementary materialThe online version of this article (doi:10.1186/s12955-017-0700-2) contains supplementary material, which is available to authorized users.
Objectives: Emergency department (ED) identification and radiographic evaluation of children with intra-abdominal injuries who need acute intervention can be challenging. To date, it is unclear if a clinical prediction rule is superior to unstructured clinician judgment in identifying these children. The objective of this study was to compare the test characteristics of clinician suspicion with a derived clinical prediction rule to identify children at risk of intra-abdominal injuries undergoing acute intervention following blunt torso trauma.Methods: This was a planned subanalysis of a prospective, multicenter observational study of children (<18 years old) with blunt torso trauma conducted in 20 EDs in the Pediatric Emergency Care Applied Research Network (PECARN). Clinicians documented their suspicion for the presence of intra-abdominal injuries needing acute intervention as <1, 1 to 5, 6 to 10, 11 to 50, or >50% prior to knowledge of abdominal computed tomography (CT) scanning (if performed). Intra-abdominal injuries undergoing acute intervention were defined by a therapeutic laparotomy, angiographic embolization, blood transfusion for abdominal hemorrhage, or intravenous fluid administration for 2 or more days in those with pancreatic or gastrointestinal injuries. Patients were considered to be positive for clinician suspicion if suspicion was documented as ≥1%. Suspicion ≥ 1% was compared to the presence of any variable in the prediction rule for identifying children with intra-abdominal injuries undergoing acute intervention.
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