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Targeted Therapy in Pediatric Langerhans Cell Histiocytosis: Describing a Novel Strategy to Minimize Long‐Term Exposure While Maintaining Efficacy

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Retrospective analysis demonstrates trametinib's effectiveness in pediatric LCH, suggesting a safer dosing strategy.

Trametinib showed favorable responses in pediatric patients with LCH, highlighting its potential as an effective treatment.
Among 15 pediatric patients treated with trametinib, 11 had detected MAPK pathway mutations, reinforcing targeted therapy importance.
Patients experienced mild adverse effects, mainly rash and diarrhea, which were manageable through dosing adjustments.
A self-weaning dosing strategy successfully minimized long-term medication exposure while maintaining disease control.

Wojciechowska et al. (2025) studied this question.

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