Langerhans cell histiocytosis
ICD-10 C96.5 · ICD-11 2B31.2

Treatment of Langerhans Cell Histiocytosis in LCH-Associated Sclerosing Cholangitis with BRAF-V600E Mutation

This protocol addresses adult patients with Langerhans cell histiocytosis (LCH) presenting with LCH-associated sclerosing cholangitis — a form of critical liver involvement — and a confirmed BRAF-V600E mutation.

Clinical Scenario

LCH-associated sclerosing cholangitis represents a severe hepatic complication of Langerhans cell histiocytosis. The optimum therapy in this setting is unknown. BRAF-V600E mutational testing is essential for all LCH patients to guide diagnosis and treatment decisions.

Treatment Approach

Due to poor outcomes associated with conventional chemotherapy in this scenario, targeted therapy — including kinase inhibition — is among the approaches considered, selected on the basis of patient-specific factors. The complete evidence-based regimen and full selection criteria are available in the structured protocol.

Instant Access to Structured Evidence-Based Regimens

References

  1. For LCH-associated sclerosing cholangitis, the optimum therapy is unknown.
  2. Due to poor outcomes with chemotherapy, targeted agents are preferable.
  3. All patients with LCH should undergo BRAF-V600E mutational testing to aid in diagnosis and treatment.
  4. For disease that is relapsed or refractory following chemotherapy, it is reasonable to consider an alternate chemotherapy agent or a kinase inhibitor based on patient-specific factors and drug availability.
DOI: 10.1182/blood.2021014343
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