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Treatment options for adult LCH
Source: Langerhans Cell Histiocytosis Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: January 6, 2025 · Captured 2026-09-09.
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Context: Adult LCH / Treatment of Adult LCH
The lack of clinical trials limits the ability to make evidence-based recommendations for adult patients with LCH.
Many investigators have previously recommended treatment according to the guidelines for childhood LCH. It is unclear, however, whether adult LCH responds as well as the childhood form of the disease. In addition, the drugs used in the treatment of children are not as well tolerated when used in adults. Excessive neurological toxicity from vinblastine, for example, prompted closure of the LCH-A1 trial. BRAF and MEK inhibitors are increasingly used as initial treatment for many adults.[1] For more information, see the Targeted therapies for the treatment of single-system and multisystem disease section.
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An international expert consensus panel has proposed a treatment algorithm for adult patients and is summarized below.[1]
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Bone-only LCH: Treatment includes curettage, bisphosphonates, oral methotrexate, or hydroxyurea. Radiation therapy may be used for patients with progressive disease and fewer than three lesions. Patients with more than three lesions may receive chemotherapy (cladribine, cytarabine, or others) or MAPK inhibitors.
Skin-only LCH: Treatment includes hydroxyurea, oral methotrexate, thalidomide or lenalidomide, or topical therapy.
Single-system pulmonary LCH: Treatment is smoking cessation. Patients with progressive disease receive chemotherapy with similar drugs used for bone LCH.
Multisystem LCH: Treatment includes chemotherapy with similar drugs used for bone LCH.
Critical organ involvement (bone marrow, spleen, liver, brain): Patients with LCH and BRAF V600E variants receive a BRAF inhibitor. For patients without BRAF V600E variants, a MEK inhibitor may be used if tests are positive for MAPK variants. If no variants are found, patients receive chemotherapy based on the regimen used for bone LCH.
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Preserved source evidence · Not medical advice