Case study finds ustekinumab effective in treating pityriasis rubra pilaris related to CARD14 gene variant, suggesting potential off-label use.
Background. Few cases of pityriasis rubra pilaris (PRP) associated with CARD14 gene variants have been reported among pediatric patients in the literature. The inefficacy of standard treatment methods necessitates management with genetically engineered biologic drugs for off-label indications. Case description . 5-year-old child with PRP and pathogenic variant in the CARD14 gene was administered with ustekinumab, genetically engineered biologic drug, for off-label indication due to inefficacy of previous therapy with topical glucocorticoids (methotrexate and acitretin). High efficacy of this therapy was noted. No side effects have been reported. Conclusion. Genetically engineered biologic therapy with ustekinumab can be suggested in the recommended regimen in patients with PRP and pathogenic variant in the CARD14 gene in case of inefficacy of topical glucocorticoids, methotrexate, oral retinoids, and tumor necrosis factor alpha inhibitors.
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Murashkin et al. (2025) studied this question.
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