Central nervous system LCH Involvement of the central nervous system (CNS) may manifest as

mass lesions in the white or grey matter, endocrine abnormalities (eg, diabetes insipidus) or as
neurodegenerative syndrome. Patients with CNS involvement require therapy directed at their CNS
lesions. Treatment options include chemotherapy that crosses the blood brain barrier, resection, radiation
therapy, or a combination of these. These options have not been compared directly in a prospective trial.
Cladribine is effective for CNS mass lesions [32]. Clofarabine is active in both LCH and Erdheim-Chester
disease and is a reasonable empiric therapy if these two entities cannot be distinguished (eg, isolated
pituitary lesions) [33]. Cytarabine can reverse the symptoms and signs of the neurodegenerative
syndrome [34].

Case reports have demonstrated remissions following the resection of solitary mass lesions with or
without subsequent low dose radiation therapy [35].

Mass lesions of the white or grey matter, hypothalamus, and pituitary have been effectively treated with
cladribine [32,36].

Three treatments for the neurodegenerative syndrome of LCH have been published.

Retinoic acid was used for 10 patients with MRI findings remaining stable, but clinical efficacy was
difficult to judge as only one-year follow-up was reported [37].

Another study, which used intravenous immunoglobulin and oral prednisone with or without
vinblastine methotrexate in five patients, reported most remained stable over a mean follow-up period
of 30 months [38].

A study using cytarabine with or without vincristine reported improvement in the clinical (6/8) and MRI
findings (5/8) [34]. Seven of these patients have been followed for more than 10 years after stopping
therapy and all but two have stable neurologic and radiographic findings.

Treatment of the pituitary damaged by LCH has not been shown to reverse the DI [11], although some
patients have reported a need for lower doses of antidiuretic hormone (desmopressin, DDAVP) after
receiving chemotherapy.