What question did this study set out to answer?

The study aims to assess the effectiveness and safety of targeted therapies, dabrafenib and trametinib, in pediatric patients with LCH and skeletal involvement.

April 1, 2026BMC CancerOpen Access

Dabrafenib or trametinib in pediatric Langerhans cell histiocytosis with bone lesions: a retrospective efficacy and safety study

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Authors

HOHaiping OuyangChildren's Hospital of Chongqing Medical University HYHailun YaoChildren's Hospital of Chongqing Medical University CHChengyuan HeChildren's Hospital of Chongqing Medical University

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Implication

Retrospective analysis evaluates dabrafenib or trametinib in pediatric LCH, suggesting promising outcomes and safety.

Key Points

The study aims to assess the effectiveness and safety of targeted therapies, dabrafenib and trametinib, in pediatric patients with LCH and skeletal involvement.
Conducted a single-center retrospective analysis
Included 30 children with pathologically confirmed, bone-involved LCH
Evaluated imaging-based objective response rate (ORR) and disease control rate (DCR)
Performed subgroup analysis based on BRAF mutation status
23.3% (7/30) achieved active disease-better status
60.0% (18/30) had active disease-stable status, resulting in a DCR of 83.3%
All patients survived
Drug-related adverse events occurred in 20.0% of patients, with mild toxicities noted

Cite This Study

Ouyang et al. (2026) studied this question.

synapsesocial.com/papers/69ccb7b016edfba7beb89bd2 https://doi.org/10.1186/s12885-026-15940-4

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Dabrafenib or trametinib in pediatric Langerhans cell histiocytosis with bone lesions: a retrospective efficacy and safety study

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Cite This Study

Dabrafenib or trametinib in pediatric Langerhans cell histiocytosis with bone lesions: a retrospective efficacy and safety study

Discussion

Member takes

Implication

Key Points

Cite This Study

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