Efficacy of the Janus kinase 1/2 inhibitor ruxolitinib in the treatment of vasculopathy associated with TMEM173-activating mutations in 3 children

被引：179

作者：

Fremond, Marie-Louise ^{[1
,2
,3
]}

Rodero, Mathieu Paul ^{[2
,3
]}

Jeremiah, Nadia ^{[2
,4
]}

Belot, Alexandre ^{[5
,6
]}

Jeziorski, Eric ^{[7
]}

Duffy, Darragh ^{[8
,9
]}

Bessis, Didier ^{[10
]}

Cros, Guilhem ^{[1
]}

Rice, Gillian I. ^{[11
]}

Charbit, Bruno ^{[8
]}

Hulin, Anne ^{[12
]}

Khoudour, Nihel ^{[12
]}

Caballero, Consuelo Modesto ^{[13
]}

Bodemer, Christine ^{[2
,14
]}

Fabre, Monique ^{[15
]}

Berteloot, Laureline ^{[16
]}

Le Bourgeois, Muriel ^{[17
]}

Reix, Philippe ^{[18
,19
]}

Walzer, Thierry ^{[6
]}

Moshous, Despina ^{[1
,2
,20
]}

Blanche, St Ephane ^{[1
,2
]}

Fischer, Alain ^{[1
,2
,21
,22
]}

Bader-Meunier, Brigitte ^{[1
,2
,4
]}

Rieux-Laucat, Frederic ^{[2
,4
]}

Crow, Yanick Joseph ^{[2
,3
,11
]}

Neven, Benedicte ^{[1
,2
,4
]}

机构：

[1] Hop Necker Enfants Malad, Pediat Hematol Immunol & Rheumatol Dept, Paris, France

[2] Paris Descartes Univ, Sorbonne Paris Cite, Inst Imagine, Paris, France

[3] INSERM, UMR 1163, Lab Neurogenet & Neuroinflammat, Paris, France

[4] INSERM, UMR 1163, Lab Immunogenet Pediat Autoimmun, Paris, France

[5] Hosp Civils Lyon, Nephrol & Dermatol Dept, Pediat Rheumatol, Lyon, France

[6] Univ Lyon 1, Ecole Normale Super Lyon, INSERM, CIRI,U1111,CNRS,UMR5308, Lyon, France

[7] Ctr Hosp Univ Montpellier, Dept Pediat, Montpellier, France

[8] INSERM, U1223, Lab Dendrit Cell Immunobiol, Paris, France

[9] Inst Pasteur, Ctr Translat Res, Paris, France

[10] Ctr Hosp Univ Montpellier, Dept Dermatol, Montpellier, France

[11] Univ Manchester, Manchester Acad Hlth Sci Ctr, Fac Med & Human Sci, Manchester Ctr Genom Med,Inst Human Dev, Manchester, Lancs, England

[12] Hop Univ Henri Mondor, Pharmacol & Toxicol Lab, Creteil, France

[13] Hosp Univ Vall dHebron, Dept Pediat Rheumatol, Barcelona, Spain

[14] Hop Necker Enfants Malad, Dept Dermatol, Paris, France

[15] Hop Necker Enfants Malad, Dept Pathol, Paris, France

[16] Hop Necker Enfants Malad, Dept Pediat Radiol, Paris, France

[17] Hop Necker Enfants Malad, Dept Pediat Pneumol, Paris, France

[18] Hosp Civils Lyon, Ctr Reference Mucoviscidose, Dept Pneumol, Lyon, France

[19] Claude Bernard Lyon 1 Univ, Lyon, France

[20] INSERM, UMR 1163, Lab Genome Dynam Immune Syst, Paris, France

[21] INSERM, UMR 1163, Paris, France

[22] Coll France, Paris, France

来源：

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY | 2016年 / 138卷 / 06期

基金：

欧洲研究理事会;

关键词：

AICARDI-GOUTIERES SYNDROME;

D O I：

10.1016/j.jaci.2016.07.015

中图分类号：

R392 [医学免疫学];

学科分类号：

100108 [医学免疫学];

摘要：

[No abstract available]

引用

页码：1752 / 1755

页数：12

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