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    Langerhans Cell Histiocytosis Treatment (PDQ®): Treatment - Health Professional Information [NCI] - Treatment of Childhood LCH


    Treatment of CNS disease

    CNS lesions

    There are three types of LCH CNS lesions:

    • 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.
    • Neurodegenerative syndrome. T2 fluid attenuated inversion recovery (FLAIR) hyperintense signals are present, most often in the cerebellar white matter, pons, basal ganglia, and sometimes, in the cerebrum.

    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 of mass lesions with cladribine has been effective in 13 reported cases.[30,31]; [32][Level of evidence: 3iiiDiii] 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.[32][Level of evidence: 3iiiDiii]
    • 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.[33,34] Treatment with vinblastine with or without corticosteroids for patients with CNS mass lesions (20 patients; mainly pituitary) demonstrated an objective response in 15 patients, with 5 of 20 patients demonstrating a complete response and 10 of 20 patients demonstrating a partial response.

    CNS neurodegenerative syndrome

    Drugs used in active LCH, such as dexamethasone and cladribine, along with other agents, such as retinoic acid, intravenous immunoglobulin (IVIg), infliximab, and cytarabine with or without vincristine have been used in small numbers of patients with mixed results. Many of these agents may result in the complete or partial resolution of radiographic findings, but definitive clinical response rates have not been rigorously defined.[35,36,37,38,39]; [32][Level of evidence: 3iiiDiii]

    • Retinoic acid was given at a dose of 45 mg/m2 daily for 6 weeks, then 2 weeks per month for 1 year.[35] Patients were reported to have stable clinical status.
    • IVIg (400 mg/m2) was given monthly with chemotherapy consisting of oral prednisolone with or without oral or intravenous methotrexate and oral 6-mercaptopurine for at least 1 year.[36] Magnetic resonance imaging (MRI) findings were stable but clinical efficacy was difficult to judge because patients were reported to have no progression in their neurologic symptoms.
    • A study using cytarabine with or without vincristine for up to 24 months reported improvement in the clinical and MRI findings in some patients and stabilization of disease in the others.[38][Level of evidence: 3iiiC] Seven of eight patients have been followed for more than 8 years after stopping therapy and have had stable neurologic and radiographic findings.
    • In the Japan LCH Study Group-96 Protocol, cytarabine failed to prevent the onset of neurodegenerative syndrome. Patients received cytarabine 100 mg/m2 daily on days 1 to 5 during induction and 150 mg/m2 on day 1 of each maintenance cycle (every 2 weeks for 6 months). Three of 91 patients developed neurodegenerative disease, which is similar to the rate experienced on the Histiocyte Society studies.[28]
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