Neurodegenerative Langerhans cell histiocytosis: long-term follow-up of 63 patients from the Italian Registry

Key Points

Overall, 60% of patients with neurodegenerative Langerhans cell histiocytosis were asymptomatic upon diagnosis, showing potential for stable outcomes.
At long-term follow-up, 57% of treated patients experienced stability or improvement, primarily among symptomatic individuals.
Analysis of patient data from the Italian LCH registry included long-term observations on neurological symptoms and MRI findings.
Univariable analysis indicated an increased risk for clinical symptoms with LCH reactivations and severe brain MRI abnormalities.

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Analysis of treatment effects on stability in patients with neurodegenerative Langerhans cell histiocytosis reveals high asymptomatic rates.

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Trambusti et al. (2025) studied this question.

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