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

AbstractObjective: 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.