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

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 bette...

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Veröffentlicht in:	The American journal of medicine 2017-10, Vol.130 (10), p.1219.e19-1219.e27
Hauptverfasser:	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
Format:	Artikel
Sprache:	eng
Schlagworte:	Capillary Leak Syndrome - drug therapy Capillary Leak Syndrome - etiology Capillary Leak Syndrome - mortality Capillary Leak Syndrome - pathology Clarkson disease Female Hematology Human health and pathology Humans Immunoglobulins, Intravenous - therapeutic use Internal Medicine Intravenous immunoglobulins Life Sciences Male Middle Aged Monoclonal gammopathy-associated systemic capillary-leak syndrome Paraproteinemias - complications Paraproteinemias - diagnostic imaging Paraproteinemias - mortality Paraproteinemias - pathology Survival Analysis Systemic capillary-leak syndrome Terbutaline - therapeutic use Theophylline - therapeutic use
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Zusammenfassung:	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.
ISSN:	0002-9343 1555-7162
DOI:	10.1016/j.amjmed.2017.05.023