1. Intravenous Immunoglobulins Tapering and Withdrawal in Systemic Capillary Leak Syndrome (Clarkson Disease)
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Quentin Moyon, Marc Pineton de Chambrun, Marie Gousseff, Alexis Mathian, Miguel Hie, Geoffrey Urbanski, Franco Verlicchi, Stanislas Faguer, Antoine Dossier, Jean-Christophe Lega, Sophie Riviere, David Saadoun, Julie Graveleau, Marie-Josée Lucchini-Lecomte, Christine Christides, Sylvie Le Moal, Béatrice Bibes, Giuseppe Malizia, Marc Ruivard, Gilles Blaison, Laurent Alric, Christian Agard, Martin Soubrier, Jean-François Viallard, Hervé Levesque, Georges-Etienne Rivard, Nathalie Tieulie, Arnaud Hot, Pierre-Yves Lovey, Thomas Hanslik, François Lhote, Vincent Eble, Jorge Álvarez Troncoso, Avinash Aujayeb, Paul Quentric, Dov Taieb, Fleur Cohen-Aubart, Marc Lambert, Zahir Amoura, CHU Pitié-Salpêtrière [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU), Unité de Recherche sur les Maladies Cardiovasculaires, du Métabolisme et de la Nutrition = Research Unit on Cardiovascular and Metabolic Diseases (ICAN), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Institut de Cardiométabolisme et Nutrition = Institute of Cardiometabolism and Nutrition [CHU Pitié Salpêtrière] (IHU ICAN), Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-CHU Pitié-Salpêtrière [AP-HP], Centre d'Immunologie et des Maladies Infectieuses (CIMI), Institut National de la Santé et de la Recherche Médicale (INSERM)-Sorbonne Université (SU)-Centre National de la Recherche Scientifique (CNRS), Centre hospitalier Bretagne Atlantique (Morbihan) (CHBA), Centre Hospitalier Universitaire d'Angers (CHU Angers), PRES Université Nantes Angers Le Mans (UNAM), Institut des Maladies Métaboliques et Casdiovasculaires (UPS/Inserm U1297 - I2MC), Université Toulouse III - Paul Sabatier (UT3), Université de Toulouse (UT)-Université de Toulouse (UT)-Institut National de la Santé et de la Recherche Médicale (INSERM), CIC - CHU Bichat, Institut National de la Santé et de la Recherche Médicale (INSERM), Laboratoire de Biométrie et Biologie Evolutive - UMR 5558 (LBBE), Université Claude Bernard Lyon 1 (UCBL), Université de Lyon-Université de Lyon-Institut National de Recherche en Informatique et en Automatique (Inria)-VetAgro Sup - Institut national d'enseignement supérieur et de recherche en alimentation, santé animale, sciences agronomiques et de l'environnement (VAS)-Centre National de la Recherche Scientifique (CNRS), Hôpital Saint Eloi (CHRU Montpellier), Centre Hospitalier Régional Universitaire [Montpellier] (CHRU Montpellier), Immunologie - Immunopathologie - Immunothérapie [CHU Pitié Salpêtrière] (I3), CHU Charles Foix [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Institut National de la Santé et de la Recherche Médicale (INSERM)-CHU Pitié-Salpêtrière [AP-HP], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Sorbonne Université (SU)-Sorbonne Université (SU), Centre hospitalier de Saint-Nazaire, Centre Hospitalier Henri Duffaut (Avignon), CHU Estaing [Clermont-Ferrand], CHU Clermont-Ferrand, Hôpital pasteur [Colmar], Université de Toulouse (UT), Centre hospitalier universitaire de Nantes (CHU Nantes), Hôpital Haut-Lévêque [CHU Bordeaux], CHU Bordeaux [Bordeaux], Université de Rouen Normandie (UNIROUEN), Normandie Université (NU), Centre de recherche du CHU Sainte-Justine / Research Center of the Sainte-Justine University Hospital [Montreal, Canada], Université de Montréal (UdeM)-CHU Sainte Justine [Montréal], Hôpital Pasteur [Nice] (CHU), Hôpital Edouard Herriot [CHU - HCL], Hospices Civils de Lyon (HCL), Université de Versailles Saint-Quentin-en-Yvelines - UFR Sciences de la santé Simone Veil (UVSQ Santé), Université de Versailles Saint-Quentin-en-Yvelines (UVSQ), Service de médecine interne [CHU Ambroise Paré], Assistance publique - Hôpitaux de Paris (AP-HP) (AP-HP)-Hôpital Ambroise Paré [AP-HP], Hôpital Delafontaine, Centre Hospitalier de Saint-Denis [Ile-de-France], Centre Hospitalier Eure-Seine - Hôpital d'Evreux - Vernon (Evreux), CHU Lille, SNFMI-2021, This clinical research was supported by a grant from Octapharma to the Association pour la Recherche, l'Enseignement et la Thérapeutique en Médecine Interne (ARTHEMI) but all treatment decisions, including intravenous immunoglobulin (IVIG) type, were left at physician's discretion. Conflicts of interest: M. Pineton de Chambrun was supported for this study by a grant from La Société Française Nationale de Médecine Interne (SNFMI-2021). The rest of the authors declare that they have no relevant conflicts of interest., Conflicts of interest: M. Pineton de Chambrun was supported for this study by a grant from La Société Française Nationale de Médecine Interne (SNFMI-2021). The rest of the authors declare that they have no relevant conflicts of interest., and This clinical research was supported by a grant from Octapharma to the Association pour la Recherche, l’Enseignement et la Thérapeutique en Médecine Interne (ARTHEMI) but all treatment decisions, including intravenous immunoglobulin (IVIG) type, were left at physician’s discretion.
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Adult ,Incidence ,[SDV]Life Sciences [q-bio] ,Paraproteinemias ,Immunoglobulins, Intravenous ,Tapering ,Intravenous immunoglobulins ,Middle Aged ,Withdrawal ,Systemic capillary leak syndrome ,Humans ,Immunology and Allergy ,Capillary Leak Syndrome ,Clarkson disease ,Retrospective Studies - Abstract
International audience; Background: The systemic capillary leak syndrome (SCLS), also known as Clarkson disease, is a very rare condition characterized by recurrent life-threatening episodes of vascular hyperpermeability in the presence of a monoclonal gammopathy. Extended intravenous immunoglobulin (IVIG) treatment is associated with fewer recurrences and improved survival, but the optimal treatment dosage and duration remain unknown. Objective: We aim to evaluate the safety of IVIG tapering and withdrawal in patients with SCLS. Methods: We conducted a retrospective multicenter study including all adult patients with monoclonal gammopathy–associated SCLS from the EurêClark registry who received at least 1 course of IVIG. The primary end point was overall survival according to IVIG withdrawal. Results: Fifty-nine patients of mean ± SD age 51 ± 13 years were included. Overall cumulative probabilities of 2-, 5-, 10- and 15-year survival were 100%, 85%, 72%, 44%, respectively. The IVIG was withdrawn at least once in 18 patients (31%; W+ group) and never in 41 patients (69%; W– group). Cumulative probabilities of 10-year survival in W+ versus W– groups were 50% and 83% (log rank test, P = .02), respectively. Relapse rate and the median number of relapses in the W+ versus the W– groups were 72% versus 58% (P = 0.3) and 2.5 (0.3–4) versus 1 (0–2) (P = .03), respectively. The IVIG tapering was not statistically associated with increased person-year incidence of attacks using a mixed linear model. Conclusions: The IVIG withdrawal was associated with increased mortality and higher rate of recurrence in SCLS patients. The IVIG tapering might be cautiously considered in stable SCLS patients.
- Published
- 2022