ABSTRACT
BACKGROUND: Crohn's disease (CD) is a chronic inflammatory condition of the gastrointestinal tract that affects people across the age spectrum but often starts in childhood or early adulthood. Despite this, almost all published research examining the symptomatic and health-related quality of life (HRQL) experiences of CD has been conducted in an adult population. Studies providing a comprehensive overview of the lived experience of pediatric and adolescent CD are virtually non-existent. The experiences of younger children aged 2-7 years are especially unknown. RESULTS: A total of 49 participants (31 children and 18 parents) were interviewed. This included 11 dyads (i.e., parents and children from the same family). Analyses were conducted based on reporter-type (patient self-report vs parent observer-report) and age subgroups (ages 2-4 vs 5-7 vs 8-11 vs 12-17). Key symptoms were identified across the age subgroups and reporter types. Abdominal/stomach pain, passing gas/feeling gassy, diarrhea/liquid stools, fatigue/tiredness, bowel urgency, blood in stools, stomach cramping, constipation, and incomplete evacuation were discussed most frequently. The most common HRQL impacts included impact on physical activity, school, social life, and mood (i.e., feeling sad/low), and were mostly consistent between reporter type and across age spectrum. Concept agreement between parents and children in the dyad analysis was > 60% for most symptoms and impacts. CONCLUSIONS: Qualitative interviews revealed the substantial symptom and HRQL burden of pediatric CD from the child and parent perspectives and that disease experiences were largely consistent across the age range and based on both reporter perspectives. This is an important first step towards implementing a robust measurement strategy for the assessment of symptoms and HRQL impacts in pediatric CD.
ABSTRACT
BACKGROUND: This study was conducted to evaluate content validity of the IntraVenous and SubCutaneous Treatment Administration Satisfaction Questionnaires (TASQ-IV and TASQ-SC), for use in a clinical trial population of participants with paroxysmal nocturnal hemoglobinuria (PNH) undergoing eculizumab treatment. METHODS: Participants underwent semi-structured combined brief introduction to disease history and full cognitive debriefing interviews to establish symptoms and key impacts of PNH and to explore the clarity and relevance of both sets of instructions (TASQ-IV and TASQ-SC). The clarity, relevance, response options, and recall period of the TASQ-IV items were also explored. RESULTS: Ten participants with PNH were recruited. Fatigue was the most commonly reported symptom (n = 7); the most commonly reported impact of PNH was on physical activity (n = 4). Nine participants indicated understanding and relevance of the TASQ-IV instructions; three participants suggested changes. Of the 20 TASQ-IV items, ≥ 15 were considered understandable, relevant and to have suitable response options (n ≥ 8). The TASQ-SC instructions were understood by all participants; seven participants indicated relevance. While a few participants suggested minor changes for the items, these reflected the one-off completion of the measure in an interview setting and were thus not considered sufficient to justify modification of the measure for clinical trial completion. CONCLUSIONS: Most participants understood the TASQ-IV and TASQ-SC instructions (n = 9 and 10, respectively) and the TASQ-IV items were considered clear, relevant and to have suitable response options, demonstrating face and content validity of the instruments for the clinical trial setting.
