Off-label treatment is often seen in pediatrics, especially in pediatric intensive care and neonatal departments. It is linked to a broad range of factors, including restriction of pharmacological studies for ethical reasons, different pharmacokinetic and pharmacodynamic properties of medicines due to age and physiological differences in children, and the difficulty of manufacturing and dosage determination. These gaps increase the likelihood of adverse reactions. Various measures are being taken to avoid unnecessary risks to children without depriving them of potentially effective pharmacotherapy. This study aimed to analyse the studies reported in literature on the relationship between off-label prescribing, the latest recommendations, and the impact of this treatment on the pediatric population. The literature shows that off-label are often prescribed due to children’s younger age, respiratory or rare diseases. Relevant groups of commonly misused medicines are as follows anti-infectives, respiratory medicines or treatment for neurological conditions. It has been observed that the most common off-label prescriptions are related to inappropriate dosage, patient age or indications for use. To deal with the problem, researchers are encouraging the research community to find new, scientifically supported and effective treatments by increasing the sharing of high-quality research and data. This is to achieve trustworthy, evidence-based medical care and to protect children from ineffective or even harmful treatments. For off-label treatment, the European Academy of Paediatrics and the European Society of Perinatal and Paediatric Pharmacology have issued new guidelines to ensure the safest possible care for children.
Astma vaikų amžiuje yra viena dažniausių lėtinių uždegiminių kvėpavimo takų ligų. Astmos paūmėjimas – tai staigus astmos simptomų paūmėjimas, kuris neretai sukelia respiracinį distresą. Dažnai įprastas gydymasastmos paūmėjimo metu gali būti neveiksmingas, todėl tokiu atveju vaikai kreipiasi į vaikų skubiosios pagalbos skyrių. Pagrindiniai astmos paūmėjimo veiksniai yra įvairūs. Virusinės kvėpavimo takų infekcijos – vienos dažniausių provokacinių veiksnių. Kiti veiksniai: alergenai, oro užterštumas, rūkymas, bloga astmos kontrolė. Astmos paūmėjimo metu pagalbos eiga – greitas ir objektyvus simptomų įvertinimas bei tiksli diferencinė diagnostika, tinkamai skirtas medikamentinis gydymas ir gyvybinių funkcijų užtikrinimas, siekiant pagerinti oksigenaciją, sumažinti respiracinio distreso simptomus bei ligos atkryčio riziką, būklės vertinimas dinamikoje, pagalbos teikimas pagal stebimą būklę, tolesnio gydymo plano sudarymas. Pagrindiniai astmos paūmėjimo gydymo principai: hipoksemijos korekcija, bronchospazmo sumažinimas, kvėpavimo takų uždegimo valdymas, būklės vertinimas dinamikoje, tolesnio gydymo stacionare indikacijų vertinimas, kitas gydymas (pvz., antipiretikai, pakankamo skysčių kiekio užtikrinimas), tolesnių paciento veiksmų planas. Po astmos paūmėjimo pacientą išleidžiant į namus, būtina suformuluoti aiškias ir tikslias tolesnes rekomendacijas, ką ir kaip vartoti namuose, kada kreiptis į šeimos gydytoją arba gydytoją specialistą.
Off-label drug use is prevalent in the pediatric population and represents a patient safety concern. We aimed to identify factors for off-label drug use in our pediatric emergency department (PED). Methods. We performed a retrospective data analysis. All patients aged 0–18 referred to PED from 1 September to 1 October 2022, were included. Further analysis was performed when respiratory tract infections were diagnosed. Data collected: gender, age, triage group, chronic diseases, vital signs, and PED-prescribed treatment (medications, dosages, methods of administration). Statistical analysis used SPSS 28.0, with significance at p < 0.05. Results. Data from 473 patients were analyzed, median age 3.5 years. Chronic diseases were present in 17.1% of children. 387 medications were prescribed, 47.5% being off-label. Off-label treatment was common for external otitis, acute laryngitis, and acute bronchitis (p < 0.001). There was incorrect administration of tobramycin with dexamethasone for otitis (n = 16, 100%) and inappropriate use of salbutamol inhalations by age (34.8%, n = 16). Some medications were given orally instead of injections (ondansetron n = 5, 62.5%; dexamethasone n = 82, 98.7%) or intranasally instead of intravenously (IV) (midazolam n = 7, 87.5%). IV adrenalin was prescribed for inhalations (n = 46). Younger children were more likely to receive off-label treatment (p < 0.001). Conclusion. Our study highlights the widespread issue of off-label and unlicensed drug prescribing in pediatric emergency care. Further research is necessary, because this reliance on off-label prescribing raises concerns about patient safety and compliance, especially given the limited clinical trials and therapeutic options available.
