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March 13, 2026Pediatric Blood & Cancer

Trametinib Monotherapy in the Treatment of Pediatric Refractory/Relapse Langerhans Cell Histiocytosis

Wenqian Wang Zishi Fang Jian Ge Yunze Zhao Dong Wang Lei Cui Chenxin Zhou Zhigang Li Tianyou Wang R X Zhang

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Key Points

Evaluate the effectiveness and safety of trametinib in pediatric patients with refractory/relapse LCH.
Monotherapy treatment with trametinib administered to pediatric patients with refractory LCH.
Assessment of effectiveness and safety throughout the treatment period.
Trametinib demonstrated effectiveness in treating refractory/relapse LCH in pediatric patients.
The treatment was found to be safe with manageable side effects.

Implication

Results show trametinib provides effective treatment for pediatric patients with refractory LCH, suggesting a new therapeutic option.

Abstract

Overall, TRA was effective and safe in the treatment of patients with refractory/relapse LCH, offering a convenient therapeutic alternative.

Discussion

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Cite This Study

Wang et al. (2026) studied this question.

synapsesocial.com/papers/69b5ff5c83145bc643d1bcc7 https://doi.org/10.1002/1545-5017.70162

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Keep reading

Synapse has enriched 5 closely related papers on similar clinical questions. Consider them for comparative context:

1Targeted Therapy in Pediatric Langerhans Cell Histiocytosis: Describing a Novel Strategy to Minimize Long‐Term Exposure While Maintaining Efficacy2025 · 3 citations
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3Dabrafenib or trametinib in pediatric Langerhans cell histiocytosis with bone lesions: a retrospective efficacy and safety studyCohort Study · 2026 · 3 citations
4Luvometinib in recurrent/refractory pediatric Langerhans cell histiocytosis: a single-arm, multi-center, phase 2 trialHuman Clinical Trial · 2026
5Langerhans Cell Histiocytosis with BRAF p.N486_P490del or MAP2K1 p.K57_G61del Treated by the MEK Inhibitor TrametinibOriginal Research · 2020 · 2 citations

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Efficacy, safety, and relapse outcomes of <scp>MAPK</scp> inhibitors in pediatric Langerhans cell histiocytosis: A real‐world study2025
Efficacy of the Allosteric MEK Inhibitor Trametinib in Relapsed and Refractory Juvenile Myelomonocytic Leukemia: a Report from the Children’s Oncology Group2024
Targeted therapy for Langerhans cell histiocytosis with maxillofacial involvement in 20 children2026

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