Uso emergente fuera de ficha técnica de inhibidores de Janus cinasa (JAK) y fosfodiesterasa 4 (PDE4) en dermatología pediátrica

• Carmen Cánovas Seva ^[2] ; Paula Carrasco Nogales ^[1] ; María Isabel Rodríguez Blanco ^[2] ; Ana Batalla ^[2]
  1. [1] Universidade de Santiago de Compostela

     Universidade de Santiago de Compostela

     Santiago de Compostela, España

     
  2. [2] Complejo Hospitalario Universitario Santiago de Compostela, Santiago de Compostela, España
• Localización: Piel: Formación continua en dermatología, ISSN 0213-9251, Vol. 41, Nº. 7 (Agosto-Septiembre), 2026, págs. 616-626
• Idioma: español
• DOI: 10.1016/j.piel.2026.01.013
• Títulos paralelos:
  • Emerging off-label use of Janus kinase (JAK) and phosphodiesterase 4(PDE4) inhibitors in pediatric dermatology
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• Resumen
  • español

    Introducción los inhibidores de la Janus cinasa (JAK) y de la fosfodiesterasa 4 (PDE4) se prescriben de forma creciente fuera de ficha técnica (FFT) en pacientes pediátricos. El objetivo de este trabajo fue revisar el uso FFT de inhibidores JAK y PDE4 en práctica clínica real, en niños y adolescentes.

    Material y métodos se realizó una revisión narrativa de la literatura en PubMed y Web of Science, utilizando la guía PRISMA 2020 para estructurar la búsqueda y selección de estudios. Se incluyeron casos clínicos, series de casos y estudios observacionales que describieran pacientes menores de 18 años tratados con inhibidores JAK o PDE4 para indicaciones dermatológicas no aprobadas.

    Resultados se incluyeron 34 estudios que recogían 72 pacientes pediátricos, de los cuales 57 disponían de datos clínicos individualizados. El fármaco más frecuentemente utilizado fue baricitinib (35/57; 61,4%), seguido de ruxolitinib tópico y upadacitinib (8/57; 14,0% cada uno). Las indicaciones más habituales fueron la dermatomiositis juvenil (25/57; 43,9%) y la alopecia areata (12/57; 21,0%). La respuesta clínica cutánea pudo evaluarse en 56 pacientes, observándose remisión en 14/56 (25,0%) y mejoría clínica en 37/56 (66,1%). Se notificaron eventos adversos en 5/32 pacientes (15,6%).

    Conclusiones baricitinib fue la pequeña molécula más frecuentemente utilizada FFT. Las indicaciones FFT más prevalentes fueron la dermatomiositis juvenil y la alopecia areata. Existió un aceptable perfil de efectividad y seguridad. Los resultados obtenidos proceden de casos clínicos y estudios observacionales, por lo que son necesarios nuevos estudios de mayor grado de evidencia científica para confirmar estos resultados.

  • English

    Introduction Janus kinase (JAK) and phosphodiesterase-4 (PDE4) inhibitors are increasingly prescribed off-label in pediatric patients. The objective of this study was to review the off-label use of JAK and PDE4 inhibitors in real-world clinical practice in children and adolescents.

    Materials and methods A narrative review of the literature was conducted in PubMed and Web of Science, using the PRISMA 2020 guidelines to structure the search and study selection process. Case reports, case series, and observational studies describing patients younger than 18 years treated with JAK or PDE4 inhibitors for non-approved dermatologic indications were included.

    Results Thirty-four studies were included, comprising 72 pediatric patients, of whom 57 had individual clinical data available. The most frequently used drug was baricitinib (35/57; 61.4%), followed by topical ruxolitinib and upadacitinib (8/57; 14.0% each). The most common indications were juvenile dermatomyositis (25/57; 43.9%) and alopecia areata (12/57; 21.0%). Cutaneous clinical response could be evaluated in 56 patients, with remission observed in 14/56 (25.0%) and clinical improvement in 37/56 (66.1%). Adverse events were reported in 5/32 patients (15.6%).

    Conclusions Baricitinib was the most frequently used off-label agent. The most common indications were juvenile dermatomyositis and alopecia areata. An acceptable effectiveness and safety profile was observed. As the results derive from case reports and observational studies, further studies with higher levels of scientific evidence are required in order to confirm these results.

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