Conley Mary Ellen, Saragoussi Delphine, Notarangelo Luigi, Etzioni Amos, Casanova Jean Laurent
Department of Pediatrics, University of Tennessee College of Medicine, Memphis, TN 38101, USA.
Clin Immunol. 2003 Dec;109(3):272-7. doi: 10.1016/j.clim.2003.08.005.
Wiskott-Aldrich syndrome is a rare immunodeficiency characterized by thrombocytopenia, eczema, recurrent infections, autoimmunity, and an increased incidence of malignancy. Clinical severity is highly variable. As a first step toward the development of therapeutic guidelines, an international study to evaluate current treatment strategies was undertaken. A total of 73 centers from 24 countries participated. These centers provide care for 507 patients with Wiskott-Aldrich syndrome. Treatment strategies were strikingly variable in both small and large centers. Most options were used in at least some patients in the majority of centers, indicating that treatment is individualized; however, there were some clear trends. Most centers use intravenous gammaglobulin and prophylactic antibiotics in the majority of patients. Splenectomy was used more sparingly. Stem cell transplant was more likely to be used in centers providing care for 10 or more patients. Studies that better define prognostic markers and optimal therapy are needed.
威斯科特-奥尔德里奇综合征是一种罕见的免疫缺陷病，其特征为血小板减少、湿疹、反复感染、自身免疫以及恶性肿瘤发病率增加。临床严重程度差异很大。作为制定治疗指南的第一步，开展了一项评估当前治疗策略的国际研究。来自24个国家的73个中心参与其中。这些中心为507例威斯科特-奥尔德里奇综合征患者提供治疗。无论在小型还是大型中心，治疗策略都存在显著差异。大多数治疗方案在大多数中心至少有部分患者使用，这表明治疗是个体化的；然而，也有一些明显的趋势。大多数中心在大多数患者中使用静脉注射免疫球蛋白和预防性抗生素。脾切除术的使用则较为谨慎。干细胞移植更有可能在为10名或更多患者提供治疗的中心使用。需要开展能更好地确定预后标志物和最佳治疗方法的研究。
