Details

Autor(en) / Beteiligte

• de Chambrun, Marc Pineton, MD
• Gousseff, Marie, MD
• Mauhin, Wladimir, MD
• Lega, Jean-Christophe, MD, PhD
• Lambert, Marc, MD, PhD
• Rivière, Sophie, MD
• Dossier, Antoine, MD
• Ruivard, Marc, MD, PhD
• Lhote, François, MD
• Blaison, Gilles, MD
• Alric, Laurent, MD, PhD
• Agard, Christian, MD, PhD
• Saadoun, David, MD, PhD
• Graveleau, Julie, MD
• Soubrier, Martin, MD, PhD
• Lucchini-Lecomte, Marie-Josée, MD
• Christides, Christine, MD
• Bosseray, Annick, MD
• Levesque, Hervé, MD, PhD
• Viallard, Jean-François, MD, PhD
• Tieulie, Nathalie, MD
• Lovey, Pierre-Yves, MD
• Le Moal, Sylvie, MD
• Bibes, Béatrice, MD
• Malizia, Giuseppe, MD
• Abgueguen, Pierre, MD
• Lifermann, François, MD
• Ninet, Jacques, MD, PhD
• Hatron, Pierre-Yves, MD
• Amoura, Zahir, MD, MSc

Titel

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

Ist Teil von

• The American journal of medicine, 2017-10, Vol.130 (10), p.1219.e19-1219.e27

Ort / Verlag

United States: Elsevier Inc

Erscheinungsjahr

2017

Link zum Volltext

Quelle

MEDLINE

Beschreibungen/Notizen

• 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 hyper-permeability in the context of a monoclonal gammopathy. This study was conducted to better describe the clinical characteristics, natural history, and long-term outcome of monoclonal gammopathy-associated systemic capillary-leak syndrome. Methods We conducted a cohort analysis of all patients included in the European Clarkson disease (EurêClark) registry between January 1997 and March 2016. From diagnosis to last follow-up, studied outcomes (e.g., the frequency and severity of attacks, death, and evolution toward multiple myeloma) and the type of preventive treatments administered were monitored every 6 months. Results Sixty-nine patients (M/F sex ratio 1:1; mean ± SD age at disease onset 52 ± 12 years) were included in the study. All patients had monoclonal gammopathy of immunoglobulin G type, with kappa light chains in 47 (68%). Median (interquartile range, IQR) follow-up duration was 5.1 (2.5-9.7) years. Twenty-four patients (35%) died after 3.3 (0.9-8) years. Fifty-seven (86%) patients received at least one preventive treatment, including intravenous immunoglobulins (IVIg) n = 48 (73.8%), theophylline n = 22 (33.8%), terbutaline n = 22 (33.8%), and thalidomide n = 5 (7.7%). In the 65 patients with follow-up, 5- and 10-year survival rates were 78% (n = 35) and 69% (n = 17) respectively. Multivariate analysis found preventive treatment with IVIg [hazard ratio (HR) 0.27 (0.10-0.70), p=0.007] and terbutaline [HR 0.35 (0.13-0.96), p=0.041] to be independent predictors of mortality. Conclusions We describe the largest cohort to date of patients with well-defined monoclonal gammopathy-associated systemic capillary-leak syndrome. Preventive treatment with IVIg was the strongest factor associated with survival, suggesting the use of IVIg as the first line in prevention therapy.