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December 8, 2025BloodOpen Access

Efficacy of frontline fixed duration chemotherapy in MAPK-pathway altered adult langerhans cell histiocytosis: A multi-institutional international retrospective study

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Authors

MPMia PoleksicGSGrant M. SpearsBSBichitra Nath Shukla

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Overview

Retrospective study shows chemotherapy improves progression-free survival in MAPK-altered LCH patients, indicating substantial benefits despite treatment limitations.

Key Points

  • To evaluate the efficacy of chemotherapy in adult patients with MAPK pathway-altered Langerhans Cell Histiocytosis.
  • Multi-institutional international retrospective cohort study
  • Analyzed adult patients with BRAF or MAP2K alterations treated with chemotherapy
  • Used Kaplan-Meier method for time-to-event analysis
  • Median progression-free survival was 2.61 years
  • Overall response rate was 89%, with 26% achieving complete responses
  • Patients with single-system disease had higher 3-year PFS at 80.2% compared to 42.2% in multisystem disease.

Cite This Study

Poleksic et al. (2025) studied this question.

synapsesocial.com/papers/69362f694fa91c937236def9https://doi.org/10.1182/blood-2025-2999
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Also Consider

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  1. 1Clinical, radiological and molecular responses to combination chemotherapy with <scp>MAPK</scp> pathway inhibition in relapsed and refractory Langerhans cell histiocytosis2024 · 17 citations
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  3. 3From mutation to management: Advancing Langerhans cell histiocytosis treatment through combination therapies2024 · 1 citations
  4. 4Long-term MAPK inhibition of childhood refractory-Langerhans cell histiocytosis: an observational study on 288 patients2026 · 6 citations
  5. 5Current Approaches Among Medical Providers Treating Pediatric Langerhans Cell Histiocytosis2026