RESUMO
In recent years, off-label and unlicensed drug use has extensively developed in the paediatric population. For a long time, clinical trials in the paediatric population were considered complicated to perform because of ethical problems, causing frequent off-label use. Off-label drug use remains an important public health issue, especially for children with rare conditions or with diseases not responsive to conventional treatments. The present paper is a narrative review of the literature of off-label drug use in children. The aim of our study is to summarize the main works dealing with the off-label use of biological drugs in paediatrics. Further studies analyzing their efficacy, safety, and cost-benefit ratios are needed to extend the use of biological therapies to the paediatric population.
Assuntos
Uso Off-Label , Pediatria , Anticorpos Monoclonais , Criança , Rotulagem de Medicamentos , Humanos , Saúde PúblicaRESUMO
OBJECTIVES: Practice-to-recommendations gaps exist in croup management and have not been critically investigated. This study examined the therapeutic management of croup among a national sample of Italian pediatric providers. METHODS: A survey was administered online to a sample of primary care and hospital-based pediatricians. Demographic data, perception regarding disease severity, treatment and knowledge of croup, choices of croup treatment medications, and knowledge of and adherence to treatment recommendations were compared between hospital and primary care pediatricians. Oral corticosteroids alone, oral corticosteroids with or without nebulized epinephrine and nebulized epinephrine plus oral or inhaled corticosteroids were considered the correct management in mild, moderate and severe croup, respectively. The determinants for correct management were examined using multivariate logistic regression analysis. RESULTS: Six hundred forty-nine pediatricians answered at least 50% of the survey questions and were included in the analysis. Providers reported extensive use of inhaled corticosteroids for mild and moderate croup. Recommended treatment for mild, moderate and severe croup was administered in 46/647 (7.1%), 181/645 (28.0%) and 263/643 (40.9%) participants, respectively. Provider's age and knowledge of Westley Croup Score were significant predictors for correct management of mild croup. Being a hospital pediatrician and perception of croup as a clinically relevant condition were significant for moderate croup. CONCLUSIONS: Significant differences exist between recommended guidelines and clinical practice in croup management. This study suggests wide variability in both the treatment of croup and clinical decision making strategies among hospital and primary care pediatricians. Addressing this issue could lead to noteworthy clinical and economic benefits.
Assuntos
Corticosteroides , Crupe , Pediatras , Humanos , Crupe/tratamento farmacológico , Itália , Pediatras/estatística & dados numéricos , Masculino , Feminino , Corticosteroides/administração & dosagem , Corticosteroides/uso terapêutico , Inquéritos e Questionários , Administração por Inalação , Epinefrina/administração & dosagem , Epinefrina/uso terapêutico , Padrões de Prática Médica/estatística & dados numéricos , Índice de Gravidade de Doença , Adulto , Administração Oral , Pessoa de Meia-Idade , Nebulizadores e Vaporizadores , Fidelidade a Diretrizes/estatística & dados numéricos , Conhecimentos, Atitudes e Prática em Saúde , Criança , Atenção Primária à Saúde/estatística & dados numéricosRESUMO
Cow's milk allergy (CMA) is one of the most common food allergies in infants, and its prevalence has increased over recent years. In the present paper, we focus on CMA as a model of food allergies in children. Understanding the diagnostic features of CMA is essential in order to manage patients with this disorder, guide the use of an elimination diet, and find the best moment to start an oral food challenge (OFC) and liberalize the diet. To date, no shared tolerance markers for the diagnosis of food allergy have been identified, and OFC remains the gold standard. Recently, oral immunotherapy (OIT) has emerged as a new therapeutic strategy and has changed the natural history of CMA. Before this, patients had to strictly avoid the food allergen, resulting in a decline in quality of life and subsequent nutritional, social, and psychological impairments. Thanks to the introduction of OIT, the passive approach involving rigid exclusion has changed to a proactive one. Both the heterogeneity in the diagnostic process among the studies and the variability of OIT data limit the comprehension of the real epidemiology of CMA, and, consequentially, its natural history. Therefore, well-planned randomized controlled trials are needed to standardize CMA diagnosis, prevention, and treatment strategies.