Chen Nan, Zhang Zhi-Yong, Liu Da-Wei, Liu Wei, Tang Xue-Mei, Zhao Xiao-Dong
Division of Immunology, Children's Hospital of Chongqing Medical University, Ministry of Education Key Laboratory of Child Development and Disorders, Key Laboratory of Pediatrics in Chongqing, Chongqing International Science and Technology Cooperation Center for Child, Development and Disorders, Chongqing, 400014, China.
Eur J Pediatr. 2015 Oct;174(10):1311-8. doi: 10.1007/s00431-015-2527-3. Epub 2015 Apr 16.
Autoimmune disease (AD) is common in patients with Wiskott-Aldrich syndrome (WAS) and patients with WAS who has an AD usually constitute a high-risk group with poor outcome. However, knowledge of AD in WAS is limited in China. In this study, medical records of 53 patients with WAS at Children´s Hospital of Chongqing Medical University from April 2004 to January 2014 were evaluated retrospectively and 14 patients (26%) had at least one AD. Autoimmune hemolytic anemia (AIHA) was the most common and detected in 12 patients (23%), other complications included immune thrombocytopenia (n = 1), immune neutropenia (n = 1), autoimmune arthritis (n = 1), and renal injury (n = 1). No significant differences were found in the level of serum immunoglobulins and lymphocyte subsets between the AD group and non-AD group. Although eight patients with AD received hematopoietic stem cell transplantation (HSCT), three patients died of pulmonary infection after HSCT.
AD is frequent in Chinese patients with WAS and AIHA was the most common. AD is a poor prognosis factor for WAS and should be treated as early as possible by HSCT.
• Autoimmune disease is common in patients with WAS. • Manifestations, follow-up finding, and treatment approaches of autoimmune disease in Chinese patients with WAS have received less attention in the literature. What is New: • This study is firstly intended for evaluation of the clinical and immune characteristics of autoimmune disease in a large series Chinese patients with WAS. • AD is frequent in Chinese patients with WAS and AIHA is the most common.
自身免疫性疾病（AD）在威斯科特-奥尔德里奇综合征（WAS）患者中很常见，患有AD的WAS患者通常构成预后不良的高危群体。然而，在中国，关于WAS患者中AD的了解有限。在本研究中，回顾性评估了重庆医科大学附属儿童医院2004年4月至2014年1月期间53例WAS患者的病历，14例患者（26%）至少患有一种AD。自身免疫性溶血性贫血（AIHA）最为常见，12例患者（23%）检测到该疾病，其他并发症包括免疫性血小板减少症（n = 1）、免疫性中性粒细胞减少症（n = 1）、自身免疫性关节炎（n = 1）和肾损伤（n = 1）。AD组和非AD组之间血清免疫球蛋白水平和淋巴细胞亚群无显著差异。尽管8例AD患者接受了造血干细胞移植（HSCT），但3例患者在HSCT后死于肺部感染。
AD在中国WAS患者中很常见，AIHA最为常见。AD是WAS患者预后不良的因素，应尽早通过HSCT进行治疗。
• 自身免疫性疾病在WAS患者中很常见。• 中国WAS患者自身免疫性疾病的表现、随访结果和治疗方法在文献中较少受到关注。新发现：• 本研究首次旨在评估大量中国WAS患者自身免疫性疾病的临床和免疫特征。• AD在中国WAS患者中很常见，AIHA最为常见。
