Treatment of Dyskeratosis Congenita-Associated Pulmonary Fibrosis With Danazol

被引:10
作者
Zlateska, Bozana [1 ,2 ]
Ciccolini, Amanda [1 ,2 ]
Dror, Yigal [1 ,2 ]
机构
[1] Univ Toronto, Res Inst, Genet & Genome Biol Program, Toronto, ON, Canada
[2] Hosp Sick Children, Dept Paediat, Div Haematol Oncol, Toronto, ON M5G 1X8, Canada
关键词
Dyskeratosis congenita; pulmonary fibrosis; Danazol;
D O I
10.1002/ppul.23235
中图分类号
R72 [儿科学];
学科分类号
100202 ;
摘要
Individuals with Dyskeratosis Congenita (DC) are at increased risk for complications in variety of systems including pulmonary fibrosis. Idiopathic and DC-associated pulmonary fibrosis are progressive and fatal disorders without known treatment. Here we describe, for the first time, marked improvement in the clinical and laboratory parameters of the pulmonary disease of a child who suffered from TINF2-associated DC and severe pulmonary fibrosis after initiation of therapy with Danazol. We recommend that the clinical efficacy of Danazol in slowing down the progression of pulmonary fibrosis in patients with telomere-related disorders is evaluated in prospective studies. (C) 2015 Wiley Periodicals, Inc.
引用
收藏
页码:E48 / E51
页数:4
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