What question did this study set out to answer?

To investigate clinical features, treatments, and outcomes in Erdheim-Chester disease patients.

December 8, 2025Blood

Clinical presentation and prognosis in erdheim-chester disease: Analysis of an international cohort of 1044 patients within the ecdga network

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Authors

FPFrancesco PegoraroEuropean Society for Sexual Medicine ZAZahir AmouraRoche (Switzerland)VRVanderson RochaUniversidade de São Paulo

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Implication

International cohort study reports clinical features and survival outcomes in Erdheim-Chester disease, suggesting prognosis factors for better patient management.

Key Points

To investigate clinical features, treatments, and outcomes in Erdheim-Chester disease patients.
Included 1044 patients diagnosed with ECD across 13 referral centers
Analyzed clinical presentation, treatment, and follow-up data
Identified predictors of survival using Cox regression
Median age at diagnosis was 57 years, with a male-to-female ratio of 2.2
20% experienced bone pain, while 18% had kidney and urinary manifestations
60% of tested patients harbored the BRAFV600E mutation, with significant mortality factors found in the analysis

Cite This Study

Pegoraro et al. (2025) studied this question.

synapsesocial.com/papers/69362f604fa91c937236dd39 https://doi.org/10.1182/blood-2025-192

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Implication

International cohort study reports clinical features and survival outcomes in Erdheim-Chester disease, suggesting prognosis factors for better patient management.

Clinical presentation and prognosis in erdheim-chester disease: Analysis of an international cohort of 1044 patients within the ecdga network

Discussion

Member takes

Implication

Key Points

Cite This Study

Clinical presentation and prognosis in erdheim-chester disease: Analysis of an international cohort of 1044 patients within the ecdga network

Discussion

Member takes

Implication

Key Points

Cite This Study

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