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CNS lesions
Source: Langerhans Cell Histiocytosis Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: January 6, 2025 · Captured 2026-09-09.
Selected source text with whitespace normalised. This Triangle page is not an NCI PDQ summary.
CNS LCH lesions include the following:
Mass lesions or tumors in the cerebrum, cerebellum, or choroid plexus.
Mass lesions of the hypothalamic-pituitary axis that are always associated with diabetes insipidus and are often associated with other endocrinopathies.
Drugs that cross the blood-brain barrier, such as cladribine, or other nucleoside analogs, such as cytarabine, are used for active CNS LCH lesions.
Treatment options for patients with CNS LCH lesions include the following:
Chemotherapy (cladribine). Treatment of mass lesions with cladribine has been effective in 13 reported cases.[114,115]; [116][Level of evidence C2] Mass lesions included enlargement of the hypothalamic-pituitary axis, parenchymal mass lesions, and leptomeningeal involvement. Doses of cladribine ranged from 5 mg/m2 to 13 mg/m2, given at varying frequencies.[116][Level of evidence C2]
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Chemotherapy (other regimens). Patients with LCH and mass lesions in the hypothalamic-pituitary region, the choroid plexus, the grey matter, or the white matter may also respond to standard LCH chemotherapy.[117,118][Level of evidence C3] Treatment with vinblastine with or without corticosteroids for patients with CNS mass lesions (20 patients; mainly pituitary) demonstrated objective responses in 15 patients. Of 20 patients, 5 achieved complete responses and 10 achieved partial responses.
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Preserved source evidence · Not medical advice