{"title":"Dupilumab successfully controlled eczema in Wiskott-Aldrich Syndrome over 52 weeks.","authors":"Changhua Zhu, Yaxuan Zheng, Lianming Liao, Jincong Chen, Lihang Lin, Huichun Su","doi":"10.1038/s41390-025-03941-1","DOIUrl":null,"url":null,"abstract":"<p><strong>Impact statement: </strong>Wiskott-Aldrich syndrome (WAS) is a rare genetic disorder characterized by immunodeficiency, thrombocytopenia, and eczema. The Management of eczema remains still a major clinical challenge. We present a rare case of eczema associated with WAS in an 8-year-old boy whose lesions significantly improve after a 4-week treatment of dupilumab, a interleukin(IL)-4α receptor monoclonal antibody. Although eczema relapsed after stopping dupilumab, and alleviated after dupilumab was resumed. Finally, the Eczema Area and Severity Index (EASI) score decreased to less than 7 after the patient receive dupilumab 300 mg every 4 weeks over 52 weeks. This case suggests that dupilumab is a promising therapy alternative for patient with eczema in WAS.</p>","PeriodicalId":19829,"journal":{"name":"Pediatric Research","volume":" ","pages":""},"PeriodicalIF":3.1000,"publicationDate":"2025-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Pediatric Research","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1038/s41390-025-03941-1","RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"PEDIATRICS","Score":null,"Total":0}
引用次数: 0
Abstract
Impact statement: Wiskott-Aldrich syndrome (WAS) is a rare genetic disorder characterized by immunodeficiency, thrombocytopenia, and eczema. The Management of eczema remains still a major clinical challenge. We present a rare case of eczema associated with WAS in an 8-year-old boy whose lesions significantly improve after a 4-week treatment of dupilumab, a interleukin(IL)-4α receptor monoclonal antibody. Although eczema relapsed after stopping dupilumab, and alleviated after dupilumab was resumed. Finally, the Eczema Area and Severity Index (EASI) score decreased to less than 7 after the patient receive dupilumab 300 mg every 4 weeks over 52 weeks. This case suggests that dupilumab is a promising therapy alternative for patient with eczema in WAS.
期刊介绍:
Pediatric Research publishes original papers, invited reviews, and commentaries on the etiologies of children''s diseases and
disorders of development, extending from molecular biology to epidemiology. Use of model organisms and in vitro techniques
relevant to developmental biology and medicine are acceptable, as are translational human studies