RESUMO
OBJECTIVES: To evaluate the longitudinal evolution of work productivity loss and activity impairment in caregivers of children with inflammatory bowel disease (IBD). We also evaluated the associations between these impairments, IBD-related factors, and caregivers' health-related quality of life (HRQOL) and estimated the indirect costs related to work absenteeism. STUDY DESIGN: Since January 2017, children with newly diagnosed IBD were enrolled prospectively in the Pediatric Inflammatory Bowel Disease Network for Safety, Efficacy, Treatment and Quality improvement of care study. The impact of pediatric-onset IBD on caregivers' socioeconomic functioning (work and daily activities) and HRQOL was assessed using the Work Productivity and Activity Impairment for caregivers questionnaire and the European Quality of Life Five Dimension Five Level questionnaire, at diagnosis and 3 and 12 months of age. Generalized estimating equation models were applied to evaluate outcomes longitudinally, adjusted for IBD type, disease activity, and child's age at diagnosis. RESULTS: Up to July 2021, 491 children with IBD were eligible for analysis of caregivers' Work Productivity and Activity Impairment questionnaire. At diagnosis, the mean caregivers' employment rate was 78.4%; the adjusted mean work productivity loss was 44.6% (95% CI, 40.2%-49.0%), and the adjusted mean activity impairment was 34.3% (95% CI, 30.8%-37.7%). Work productivity loss and activity impairment significantly decreased over time and were associated with disease activity, but not with IBD type or child's age. Caregivers' HRQOL was associated with both impairments. Costs related to work absenteeism were at least 6272 ($7276) per patient during the first year after diagnosis. CONCLUSIONS: Caregivers of children with IBD experience significant impairments in work and daily activities, especially at diagnosis. The impact decreases thereafter and is associated with disease activity and caregivers' HRQOL. Work absenteeism results in high indirect costs.
Assuntos
Cuidadores , Doenças Inflamatórias Intestinais , Criança , Doença Crônica , Eficiência , Humanos , Doenças Inflamatórias Intestinais/terapia , Estudos Prospectivos , Qualidade de Vida , Inquéritos e QuestionáriosRESUMO
OBJECTIVE: To describe the ambulatory proton pump inhibitor (PPI) prescription in French children, its trends, and the impact of French (2014) and international (2018) clinical guidelines. STUDY DESIGN: We described PPI prescription rates based on national dispensation data in French children (IQVIA's Xponent database, 2009-2019). Using a segmented linear regression, we assessed the impact of clinical guidelines on PPI prescription rates. Analyses were performed for the overall pediatric population and by age subgroups (infants <2 years old, children 2-11 years old, adolescents 12-17 years old). RESULTS: During the study period, 8 060 288 pediatric PPI prescriptions were filled, with a mean PPI prescription rate of 52.5 per 1000 inhabitants per year. Between 2009 and 2019, the PPI prescription rate increased by 41% in the overall pediatric population (+110% in infants). The PPI prescription rate showed seasonal patterns with peaks in winter. After the release of French guidelines, significant decreases in trends of prescription rates occurred overall (change in trend -0.28, 95% CI -0.34;-0.23) and across all age groups. In infants, this change in trend was not sufficient to reverse the PPI prescription rate that was still increasing over time. In children, the PPI prescription rate slightly decreased and in adolescents, it was stable. After the release of international guidelines, a significant decrease in trend occurred in adolescents only (change in trend -0.26, 95% CI -0.47; -0.04). CONCLUSIONS: The pediatric PPI prescription rate in France was high, displayed a major increase over the last decade, mainly among infants, and was modestly affected by clinical guidelines.
Assuntos
Prescrições , Inibidores da Bomba de Prótons , Adolescente , Criança , Pré-Escolar , Bases de Dados Factuais , Prescrições de Medicamentos , França/epidemiologia , Humanos , Lactente , Padrões de Prática Médica , Inibidores da Bomba de Prótons/uso terapêutico , Projetos de PesquisaRESUMO
IPEX (immune-dysregulation, polyendocrinopathy, enteropathy, X-linked) syndrome is an autoimmune disorder with an often lethal outcome in spite of immunosuppressive therapy. We report the successful use of sirolimus in 3 patients with IPEX. The efficacy of sirolimus is probably due to its different mode of action compared to calcineurin-dependent agents.