Intravenous immunoglobulins improve survival in monoclonal gammopathy-associated systemic capillary-leak syndrome

摘要: Abstract Background Monoclonal gammopathy-associated systemic capillary-leak syndrome, also known as Clarkson disease, is a rare condition characterized by recurrent life-threatening episodes of capillary hyperpermeability in the context monoclonal gammopathy. This study was conducted to better describe clinical characteristics, natural history, and long-term outcome syndrome. Methods We cohort analysis all patients included European disease (EureClark) registry between January 1997 March 2016. From diagnosis last follow-up, studied outcomes (eg, frequency severity attacks, death, evolution toward multiple myeloma) type preventive treatments administered were monitored every 6 months. Results Sixty-nine (M/F sex ratio 1:1; mean ± SD age at onset 52 12 years) study. All had gammopathy immunoglobulin G type, with kappa light chains 47 (68%). Median (interquartile range) follow-up duration 5.1 (2.5-9.7) years. Twenty-four (35%) died after 3.3 (0.9-8) Fifty-seven (86%) received least one treatment, including intravenous immunoglobulins (IVIg) n = 48 (73.8%), theophylline 22 (33.8%), terbutaline thalidomide 5 (7.7%). In 65 5- 10-year survival rates 78% (n = 35) 69% 17), respectively. Multivariate found treatment IVIg (hazard 0.27; 95% confidence interval, 0.10-0.70; P  = .007) 0.35; 0.13-0.96; .041) be independent predictors mortality. Conclusions largest date well-defined Preventive strongest factor associated survival, suggesting use first line prevention therapy.