Efficacy of the Janus kinase 1/2 inhibitor ruxolitinib in the treatment of vasculopathy associated with TMEM173-activating mutations in 3 children - 18/04/17
Doi : 10.1016/j.jaci.2016.07.015
Marie-Louise Frémond, MD a, b, c, ∗, Mathieu Paul Rodero, PhD b, c, ∗, Nadia Jeremiah, PhD b, d, Alexandre Belot, MD, PhD e, f, Eric Jeziorski, MD g, Darragh Duffy, PhD h, i, Didier Bessis, MD j, Guilhem Cros, MD a, Gillian I. Rice, PhD k, Bruno Charbit, MSc h, Anne Hulin, PharmD, PhD l, Nihel Khoudour, MD l, Consuelo Modesto Caballero, MD m, Christine Bodemer, MD, PhD b, n, Monique Fabre, MD o, Laureline Berteloot, MD p, Muriel Le Bourgeois, MD q, Philippe Reix, MD r, Thierry Walzer, PhD f, Despina Moshous, MD, PhD a, b, s, Stéphane Blanche, MD, PhD a, b, Alain Fischer, MD, PhD a, b, t, u, Brigitte Bader-Meunier, MD a, b, d, Fréderic Rieux-Laucat, PhD b, d, ∗, Yanick Joseph Crow, MD, PhD b, c, k, ∗, Bénédicte Neven, MD, PhD a, b, d 
a Pediatric Hematology-Immunology and Rheumatology Department, Hôpital Necker-Enfants Malades, Paris, France
b Paris Descartes University, Sorbonne-Paris-Cité, Institut Imagine, Paris, France
c INSERM UMR 1163, Laboratory of Neurogenetics and Neuroinflammation, Paris, France
d INSERM UMR 1163, Laboratory of Immunogenetics of Pediatric Autoimmunity, Paris, France
e Pediatric Rheumatology, Nephrology and Dermatology Department, Hospices Civils de Lyon, Lyon, France
f CIRI, Centre International de Recherche en Infectiologie, INSERM, U1111, CNRS UMR5308, Ecole Normale Supérieure de Lyon, Université Lyon 1, Lyon, France
g Pediatrics Department, Centre Hospitalier Universitaire de Montpellier, Montpellier, France
h INSERM U1223, Laboratory of Dendritic Cell Immunobiology, Institut Pasteur, Paris, France
i Center for Translational Research, Institut Pasteur, Paris, France
j Dermatology Department, Centre Hospitalier Universitaire de Montpellier, Montpellier, France
k Manchester Centre for Genomic Medicine, Institute of Human Development, Faculty of Medical and Human Sciences, Manchester Academic Health Sciences Centre, University of Manchester, Manchester, United Kingdom
l Pharmacology and Toxicology Laboratory, Hôpital Universitaire Henri Mondor, Créteil, France
m Pediatric Rheumatology Department, Hospital Universitari Vall d'Hebron, Barcelona, Spain
n Department of Dermatology, Hôpital Necker-Enfants Malades, Paris, France
o Department of Pathology, Hôpital Necker-Enfants Malades, Paris, France
p Department of Pediatric Radiology, Hôpital Necker-Enfants Malades, Paris, France
q Department of Pediatric Pneumology, Hôpital Necker-Enfants Malades, Paris, France
r Pneumology Department, Centre de Référence de la Mucoviscidose, Hospices Civils de Lyon and Claude-Bernard Lyon 1 University, Lyon, France
s INSERM UMR 1163, Laboratory of Genome Dynamics in the Immune System, Paris, France
t INSERM UMR 1163, Paris, France
u Collège de France, Paris, France
| M.-L.F. is supported by the Institut National de la Santé et de la Recherche Médicale (grant no. 000427993). Y.J.C. is supported by the European Research Council (GA 309449: Fellowship) and a state subsidy managed by the National Research Agency (France) under the “Investments for the Future” program bearing the reference ANR-10-IAHU-01. F.R.-L. is supported by a state subsidy managed by the National Research Agency (France) under the “Lumugène” project bearing the reference ANR-14-CE14-0026. |
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| Disclosure of potential conflict of interest: D. Duffy receives grant support from the ANR. A. Hulin receives travel support from Atellas and Bayer and serves as a consultant for Region Centre Val de Loire. The rest of the authors declare that they have no relevant conflicts of interest. |
© 2016
American Academy of Allergy, Asthma & Immunology. Publié par Elsevier Masson SAS. Tous droits réservés.
Vol 138 - N° 6
P. 1752-1755 - décembre 2016 Retour au numéro
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