Uso off-label de medicamentos em pediatria: uma revisão integrativa em ambientes hospitalares
DOI:
10.24276/rrecien2025.15.43.233Palavras-chave:
Pediatria, Criança, Uso Off-Label, MedicamentoResumo
Descrever a prevalência, indicações e usos mais frequentes de medicamentos off-label em pacientes pediátricos. Realizou-se uma revisão integrativa da literatura com busca em bases de dados, PubMed, Scopus e BVS, utilizando os descritores “pediatria”, “criança” e “uso off-label”. Foram selecionados 30 artigos após triagem de 372 estudos identificados. A prevalência de prescrição OL variou de 18,7% a 96%. Os principais motivos de prescrição OL foram a inexistência de alternativa terapêutica para a faixa etária (3,0% a 73,5%) e indisponibilidade de dose para pediatria (34,8%). As classes terapêuticas mais comumente prescritas OL foram os anti-infecciosos para uso sistêmico, seguidos por medicamentos do sistema nervoso e os serviços de saúde em que a prescrição OL é mais frequente são os hospitalares. O uso de medicamentos off-label é frequente em pediatria, especialmente em ambientes hospitalares. A prática está associada à falta de ensaios clínicos específicos para crianças e a necessidade de adaptação de doses. Os resultados destacam a necessidade de estudos clínicos específicos para pediatria e protocolos de prescrição padronizados.
Descritores: Pediatria, Criança, Uso Off-Label, Medicamento.
Off-label use of medications in pediatrics: an integrative review in hospital settings
Abstract: To assess the prevalence of off-label drug use in pediatrics. An integrative literature review was conducted by searching databases such as PubMed, Scopus, and BVS, using the keywords "pediatrics," "child," and "off-label". A total of 30 articles were selected after screening 372 identified studies. The prevalence of OL prescription ranged from 18.7% to 96%, being more frequent in hospital settings and neonatal intensive care units (NICUs). The primary reasons for OL prescription were age (3.0% to 73.5%) and inappropriate dosage (34.8%). The most prescribed OL therapeutic classes were systemic anti-infectives, followed by nervous system drugs. The use of off-label drugs is frequent in pediatrics, especially in hospital settings. This practice is associated with factors such as the lack of specific clinical trials for children and the need for dose adjustment. The results highlight the need for pediatric-specific clinical studies and standardized prescription protocols.
Descriptors: Pediatrics, Child, Off-label Use, Drugs.
Uso fuera de etiqueta de medicamentos en pediatría: una revisión integradora en entornos hospitalarios
Resumen: Describir la prevalencia, indicaciones y usos más frecuentes de medicamentos fuera de indicación en pacientes pediátricos. Se realizó una revisión integrativa de la literatura con búsqueda en bases de datos como PubMed, Scopus y BVS, utilizando los descriptores “pediatría,” “niño,” y “Uso Fuera de lo Indicado”. Se seleccionaron 30 artículos tras la revisión de 372 estudios identificados. La prevalencia de prescripción fuera de indicación varió de 18,7% a 96%. Las principales razones de prescripción fuera de indicación fueron la edad (3,0% a 73,5%) y la dosificación inadecuada (34,8%). Las clases terapéuticas más comúnmente prescritas fuera de indicación fueron los antiinfecciosos para uso sistémico, seguidos de medicamentos para el sistema nervioso. El uso de medicamentos fuera de indicación es frecuente en pediatría, especialmente en entornos hospitalarios. Esta práctica está asociada con la falta de ensayos clínicos específicos para niños y la necesidad de ajustar dosis. Los resultados destacan la necesidad de estudios clínicos específicos para pediatría y protocolos estandarizados de prescripción.
Descriptores: Pediatría, Niño, Uso Fuera de lo Indicado, Medicamento.
Downloads
Referências
Joseph PD, Craig JC, Caldwell PH. Clinical trials in children. Br J Clin Pharmacol. 2015; 79(3):357-69.
Turner S, Gill A, Nunn AJ, Hewitt B, Choonara I. Use of “off-label” and unlicensed drugs in paediatric intensive care unit. Lancet. 1998; 351(9100):343-5.
Diel JDA, Facchini LA, Dall’Agnol MM. Off-label drug use in children and adolescents: a population-based study. Rev Saúde Pública. 2020; 54:119.
Langerová P, Vrtal J, Urbánek K. Off-label and unlicensed use of medicinal products in children hospitalized in the Czech Republic: identification of the most common interventions and associated risks. BMC Pediatr. 2014; 14:225.
Meng L, Gorrell R, Kuczynski S, Vaillancourt R, Tseng A. Off-label drug use and its evaluation in pediatric guidelines: a literature review. Frontiers in Pharmacology. 2022; 13:846619.
Allen HC, Garbe MC, Lees J, Aziz N, Chaaban H, Miller JL, et al. Off-label medication use in children, more common than we think: a systematic review of the literature. J Okla State Med Assoc. 2018; 111(8):776-783.
Lee JH, Kim SC, Park YH, Kim HJ. Off-label and unlicensed drug use in pediatric inpatients in a Korean teaching hospital. Korean J Pediatr. 2018; 61(7):228-33.
Aamir M, Khan JA, Shakeel F, Raja AA, Hussain Z, Khan TM. Off-label and unlicensed drug use: perceptions and practices of pediatricians and pharmacists in Pakistan. PLoS One. 2018; 13(12).
Garcia-Lopez I, Cuervas-Mons Vendrell M, Martín Romero I, de Noriega I, Benedí González J, Martino-Alba R. Off-label and unlicensed drugs in pediatric palliative care: a prospective observational study. J Pain Symptom Manage. 2020; 60(5):923-932.
Giurin MS, Trojniak MP, Arbo A, Carrozzi M, Abbracciavento G, Monasta L, et al. Safety of off-label pharmacological treatment in pediatric neuropsychiatric disorders: a global perspective from an observational study at an Italian Third Level Children’s Hospital. Front Pharmacol. 2022; 13:837692.
Pesiou S, Barcelo R, Fradera M, Torres F, Pontes C. Utilisation of drugs for the treatment of psychiatric diseases in the pediatric population: focus on off-label use. Front Pharmacol. 2023; 14:1157135.
Lee JH, Kim SC, Kwon SH, Shin SH, Park YH, Kim HJ. Off-label and unlicensed drug use in a pediatric intensive care unit in Korea: a retrospective study. Pediatr Crit Care Med. 2023; 24(2).
Arroyo M, Gil-Navarro MV, Gavín E, Domínguez-Gil H, González-Haba E. Off-label drug use in a pediatric intensive care unit: a prospective observational study. Front Pediatr. 2023; 11:1004551.
Trinidade AF, Melo MS, Queiroz PS, Braga JU. Off-label drug use in hospitalized children: a prospective observational study in a Brazilian teaching hospital. J Pediatr (Rio J). 2021; 97(5):544-551.
Jaberi E, Boussaha I, Dode X, Grenet G, Kassai B, Nguyen KA. Unlicensed/off-label drug prescriptions at hospital discharge in children: an observational study using routinely collected health data. Healthcare (Basel). 2024; 12(2):208.
Costa HT, Coelho HL, Ribeiro LC. Off-label and unlicensed drug utilization in neonatal units: a prospective cohort study in Brazil. Eur J Clin Pharmacol. 2018; 74(4):479-85.
Couselo-Rodriguez C, Batalla A, Martínez-Fernández S, Dávila-Pousa C, Soto-García D, Vilanova-Trillo L, Flórez Á. Off-label prescription in paediatric dermatology: a retrospective observational study in a tertiary hospital. Acta Derm Venereol. 2023; 103.
Meng M, Ge B, Lei W, Wu Y, Tian M, Lu Y, et al. Paediatric off-label use of drugs in Gansu, China: a multicentre cross-sectional study. BMJ Open. 2024; 14.
Aamir M, et al. Unlicensed and off-label use of drugs in pediatric surgical units at tertiary care hospitals of Pakistan. International Journal of Clinical Pharmacy. 2017; 39(4):860-866.
Casãn VA, et al. Utilización de medicamentos fuera de ficha técnica y sin licencia en una unidad de cuidados. Farmacia Hosp. 2017; 3:371-381.
García-Lópes I, et al. Utilización de medicamentos en condiciones off-label y unlicensed: resultados de un estudio piloto realizado en una unidad de cuidados intensivos pediátricos. Anales de Pediatría. 2017; 86(1):28-36.
Tefera YG, et al. Off-label drug use in hospitalized children: a prospective observational study at gondar university referral hospital, northwestern ethiopia. Pharmacology Research & Perspectives, v. 5, n. 2, mar. 2017.
Dornelles AD, Silveira LM, Silva MG. Prevalence of off-label drug use in a general pediatric ward of a university hospital in Southern Brazil. Rev Paul Pediatr. 2019; 37(4):441-6.
Alonso AS, Silva MT, Almeida F, Ribeiro H. Off-label medication use in the neonatal intensive care unit. An Pediatr (Barc). 2019; 91(4):237-43.
Landwehr C, Richardson KL, Russell NJ, Hewson K, Conroy C, Downey G. Cross-sectional survey of off-label and unlicensed prescribing in an Australian paediatric hospital. Int J Clin Pharm. 2019; 41(4):919-27.
Kouti L, Hashemi M, Nematollahi M. Off-label and unlicensed drug use in neonatal intensive care units in Iran: a cross-sectional study. BMC Pediatr. 2019; 19(1):121.
Rajan S, Verma N, Bhatia S, Dahiya N, Arora A, Arora P. Off-label drug use in pediatric outpatients: an observational study from India. J Pharm Bioallied Sci. 2019; 11(4): 361-4.
Tukayo Y, Hasan F, Imansyah D, Ismail S. Off-label use of medicines in Indonesian pediatric inpatients. Paediatr Int Child Health. 2020; 40(1):18-24.
Shatif, R. et al. Pharmacoepidemiological assessment of off-label drug use in pediatric ambulatory departments at four tertiary care hospital in Pakistan. Tropical Journal Of Pharmaceutical Research. 2020; 19(10):2219-2225.
Vieira HT, Carvalho PR, Rocha AS, Oselame GB, Gonçalves TCB. Off-label and unlicensed drug use in neonatal intensive care units: FDA versus Brazilian guidelines. Rev Paul Pediatr. 2021.
Biggar WD, Duchesne M, McNeely E, Ray P, Michel RN. Long-term benefits of deflazacort treatment for boys with Duchenne muscular dystrophy in their second decade of life. J Neuromuscul Dis. 2022; 9(1):41-53.
Di Salvo M, Laurini GS, Motola D, Valpiani G, Sapigni E, Pompilio A, et al. PAttern of drug use in PEdiatrics: An observational study in Italian hospitals (the PAPEOS study). Br J Clin Pharmacol. 2024; 90:1050-1057.
Venckute G, Zekaite-Vaisniene E, Oniunaite U, Jankauskaite L. Younger children with respiratory tract infections are more exposed to off-label treatments: an exploratory retrospective study in a pediatric emergency setting. Children (Basel). 2024; 11(6):735.
Lim M, Shulman DS, Roberts H, Li A, Clymer J, Bona K, et al. Off-label prescribing of targeted anticancer therapy at a large pediatric cancer center. Cancer Med. 2020; 9(18):6658-6666.
Cuzzolin L, Atzei A, Fanos V. Off-label and unlicensed drug use in neonatal intensive care units: a nationwide survey. Ital J Pediatr. 2006; 32(1):52-6.
Andrade P, Santos A, Figueiredo-Braga M. Pediatric off-label use of medicines: the urgent need to reformulate the drug market. Acta Pediatr Port. 2017; 48(1):19-25.
Publicado
- Visualizações 0
- pdf downloads: 0
Como Citar
Edição
Seção
Este trabalho está licenciado sob uma licença Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International License.
