Rituximab therapy for patients with Langerhans cell histiocytosis-associated neurologic dysfunction.

ABSTRACT

OBJECTIVE: Since patients with langerhans cell histiocytosis and neurologic dysfunction (LCH-ND) often have incomplete treatment responses we sought a new treatment regimen. Because of clinical benefit from rituximab in multiple sclerosis patients with neurodegeneration, we evaluated its use in patients with LCH-ND. PARTICIPANTS: Eight LCH-ND patients who had failed prior therapies. METHODS: Charts of the 8 patients treated with rituximab were reviewed. Signs/symptoms and MRI responses were assessed. RESULTS: Seven of eight patients experienced some clinical improvement gait abnormalities and tremors in four children, proprioceptive deficits in 2, and dysarthria/dysphagia in 2. Five of eight patients demonstrated improvement in intellectual/behavioral/psychological symptoms. CONCLUSION: These findings suggest that prospective studies are warranted to define safety and efficacy of rituximab for patients with LCH-ND.