Treatment of Langerhans Cell Histiocytosis with Associated Sclerosing Cholangitis, BRAF-V600E Negative

This protocol addresses an adult with Langerhans cell histiocytosis-associated sclerosing cholangitis involving critical liver involvement, in whom BRAF-V600E mutation testing is negative.

Clinical situation

LCH-associated sclerosing cholangitis represents a pattern of critical liver involvement that poses particular management challenges. The optimal therapy for this presentation remains an area of active investigation. BRAF-V600E mutational status is an essential step in guiding treatment selection for all patients with LCH and should be established at diagnosis.

Treatment approach (partial overview)

Management in this BRAF-V600E-negative setting involves consideration of a kinase inhibitor or a conventional chemotherapy agent, selected according to patient-specific factors and availability — the full regimen details, sequencing, and decision criteria are in the structured protocol.Full regimen, dosing, and algorithm available via the link below.

Instant Access to Structured Evidence-Based Regimens

References

DOI: 10.1182/blood.2021014343

For LCH-associated sclerosing cholangitis, the optimum therapy is unknown.

Due to poor outcomes with chemotherapy, targeted agents are preferable.

All patients with LCH should undergo BRAF-V600E mutational testing to aid in diagnosis and treatment.

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.

View source ↗