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December 19, 2025International Journal of CancerOpen Access

Efficacy, safety, and relapse outcomes of MAPK inhibitors in pediatric Langerhans cell histiocytosis: A real‐world study

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Authors

XTXue TangJSJingjing SunZWZhi Wan

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Overview

Real-world study shows MAPK inhibitors improve disease outcomes in pediatric LCH, suggesting benefit of combination therapy.

Key Points

  • This study aims to evaluate the efficacy and safety of MAPK inhibitors in managing pediatric Langerhans cell histiocytosis.
  • Real-world study design involving 36 children with LCH
  • Treatment with MAPK inhibitors (vemurafenib, dabrafenib, trametinib) either alone or with chemotherapy
  • Assessment of Disease Activity Score, interleukin-2 receptor, and tumor necrosis factor-α levels
  • Median Disease Activity Score improved significantly from 5 to 1 after 3 months (p < .0001)
  • 94.4% objective response rate observed at 3 months
  • Relapse more frequent in monotherapy group (75%) compared to combination group (20%) (p = .047)
  • Grade ≥3 adverse events occurred in 11.1% of patients but resolved with dose adjustment

Cite This Study

Tang et al. (2025) studied this question.

synapsesocial.com/papers/69449a892f0218eca9508544https://doi.org/10.1002/ijc.70300
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