Patterns and organ treatment response of Erdheim-Chester disease with cardiac involvement
Authors' conclusion
We pioneered the evaluation of heart response of ECD considering both pericardial effusion and cardiac metabolic response within our cohort, revealing a correlation between these two indicators.
Study context
- Population
- Patients with Erdheim-Chester disease (ECD) with cardiac involvement, diagnosed January 2010–August 202321 evaluable
- Participants
- 40 patients
- Outcome
- Heart response (integrating pericardial effusion and metabolic responses)
Methodological detailssingle-centre
- Single-centre
Main finding
As reported in the abstract
Heart response
Heart response (21 evaluable)
18
86%
Complete response
5
24%
Partial response
13
62%
Observational data: associations, not effects.
Further findings
Pericardial effusion response
- Complete response (CR) rate
- 33%
- Partial response (PR) rate
- 56%
Cardiac mass response
- PR
- 33%
Cardiac metabolic response
- Complete metabolic response
- 32%
- Partial metabolic response
- 53%
Correlation of pericardial effusion response and cardiac metabolic response
- R (95% CI 0.12 to 0.83), p<0.001
- 0.73
Survival · median follow-up 50.2 months (range: 1.0-102.8 months)
- Estimated 5-year overall survival
- 78.9%
- Median progression-free survival
- 59.4 months (95% CI 26.2 to 92.7 months)
Heart response with BRAF inhibitors
- Better heart response with BRAF inhibitors regardless of treatment lines
- P = 0.037
Original publication
Patterns and organ treatment response of Erdheim-Chester disease with cardiac involvement. Heart. Published 15 May 2024. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →