ArticleOrphanet journal of rare diseases2026
Targeted therapy for Langerhans cell histiocytosis with maxillofacial involvement in 20 children.
Article in Orphanet journal of rare diseases, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Not yet cited in PubMed.
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Abstract
backgroundTo evaluate targeted therapy for Langerhans cell histiocytosis (LCH) with maxillofacial manifestations, we conducted a retrospective study of 20 children admitted from January 2016 to June 2021.
methodsThe maxillofacial LCH exhibited diverse clinical features and laboratory indices. Seventeen patients were refractory to standard treatments, and three patients directly underwent targeted therapy. Sixteen patients with BRAF
resultsThe cohort included nine males and eleven females, with a median 0.92 years at diagnosis and 1.70 years at initiation of targeted therapy. Three patients had single-system LCH and 17 patients had multi-system LCH. Lesions affected the mandibular bone in 17 patients, the temporal bone in 12 patients, and the maxilla in 10 patients. Ki-67 expression before targeted therapy correlates with maxillary involvement (coefficient 0.61; P < 0.05). The cfBRAF
conclusionOverall, targeted therapy demonstrated good efficacy with mild tolerable adverse events.
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