BACKGROUND: Spinal muscular atrophy is an autosomal-recessive, progressive neuromuscular disease associated with extensive morbidity. Children with spinal muscular atrophy have potentially increased life spans due to improved nutrition, respiratory…
Despite vast improvements in disease-based treatments, many children live with life-threatening disorders that cause distressing symptoms. These symptoms can be difficult to comprehensively assess and manage. Yet, frequent and accurate symptom…
CONTEXT: Specialized pediatric palliative care (SPPC) is increasingly involved in the care of seriously ill children, yet the evidence on its impact has not been comprehensively reviewed. OBJECTIVE(S): To assess the effects of providing SPPC to…
Context Specialized pediatric palliative care (SPPC) is increasingly involved in the care of seriously ill children, yet the evidence on its impact has not been comprehensively reviewed. Objective The objective of this study was to assess the effects…
BACKGROUND: Clinicians are the standard source for adverse event (AE) reporting in oncology trials, despite the subjective nature of symptomatic AEs. The authors designed a pediatric patient-reported outcome (PRO) instrument for symptomatic AEs to…
Objectives To identify pediatric patient-reported outcomes (PROs) that are associated with chronic conditions and to evaluate the effects of chronic disease activity on PROs. Study design Participants 8-24 years-old and their parents were enrolled…
Age is often used to determine when children can begin completing patient-reported outcome (PRO) instruments or transition to adult instruments. This study’s purpose was to determine relationships between literacy, age, and race and their influence…
Objectives To evaluate whether Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric patient-reported outcome (PRO) measures can serve as valid endpoints in a clinical trial of a chronic pediatric illness. Study design We…
BACKGROUND: Children with advanced cancer experience symptoms despite access to quality care. Symptom research has previously relied upon retrospective designs and parent proxy rather than prospective measurement with self-report. OBJECTIVE: This…
