Paediatric Unit, "San Giuseppe" Hospital, Empoli, Italy.
Department of Clinical and Experimental Medicine, Section of Pediatrics, University of Pisa, Pisa, Italy.
Curr Pediatr Rev. 2024;20(3):253-264. doi: 10.2174/1573396320666230912103124.
The Hyper IgE Syndromes are rare primary immunodeficiencies characterized by eczema, recurrent skin and respiratory infections and elevated serum IgE levels. Nowadays a geneticmolecular characterization is possible and allows the distinction in various monogenic pathologies, which share some clinical characteristics but also important differences. In addition to long-known STAT3 and DOCK8 gene mutations, in fact, also ZNF341, CARD11, ERBB2IP, IL6R and IL6ST genes mutations can cause the disease. The main clinical manifestations are represented by newborn rash, eczema similar to atopic dermatitis, bacterial and viral skin infections, cold abscesses, respiratory infections with possible pulmonary complications, allergies, gastrointestinal manifestations, malignancies and connective tissue abnormalities. Diagnosis is still a challenge because, especially in the early stages of life, it is difficult to distinguish from other pathologies characterized by eczema and high IgE, such as atopic dermatitis. Several scores and diagnostic pathways have been developed, but it is essential to seek a genetic diagnosis. Treatment is based on prevention and early treatment of infections, meticulous skincare, intravenous immunoglobulins and HSCT, which, in some HIES subtypes, can modify the prognosis. Prognosis is related to the affected gene, but also to early diagnosis, timely treatment of infections and early HSCT.
高免疫球蛋白 E 综合征是一种罕见的原发性免疫缺陷病,其特征为湿疹、反复的皮肤和呼吸道感染以及血清 IgE 水平升高。如今,可以进行基因分子特征分析,从而将其区分成各种单基因疾病,这些疾病具有一些共同的临床特征,但也存在重要的差异。除了众所周知的 STAT3 和 DOCK8 基因突变外,实际上,ZNF341、CARD11、ERBB2IP、IL6R 和 IL6ST 基因突变也可导致该病。主要的临床表现包括新生儿皮疹、类似于特应性皮炎的湿疹、细菌和病毒皮肤感染、寒性脓肿、可能伴有肺部并发症的呼吸道感染、过敏、胃肠道表现、恶性肿瘤和结缔组织异常。由于尤其是在生命的早期阶段,很难将其与其他以湿疹和高 IgE 为特征的疾病区分开来,因此该病的诊断仍然具有挑战性。已经开发了几种评分和诊断途径,但寻求基因诊断至关重要。治疗基于预防和早期治疗感染、细致的皮肤护理、静脉注射免疫球蛋白和 HSCT,在某些 HIES 亚型中,这些方法可以改善预后。预后与受影响的基因有关,但也与早期诊断、及时治疗感染和早期 HSCT 有关。