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VEXAS 综合征:临床表现、诊断与治疗。

VEXAS syndrome: Clinical manifestations, diagnosis, and treatment.

机构信息

Departamento de Inmunología y Reumatología, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Tlalpan, Mexico City, Mexico.

Departamento de Patología, Instituto Nacional de Ciencias Médicas y Nutrición Salvador Zubirán, Tlalpan, Mexico City, Mexico.

出版信息

Reumatol Clin (Engl Ed). 2024 Jan;20(1):47-56. doi: 10.1016/j.reumae.2023.12.004. Epub 2023 Dec 29.

Abstract

VEXAS (Vacuoles, E1 enzyme, X-linked, Autoinflammatory, Somatic) syndrome is an adult-onset autoinflammatory syndrome characterized by somatic mutations in the UBA1 gene and is considered the prototype of hematoinflammatory diseases. Patients with VEXAS syndrome exhibit inflammatory and hematological manifestations that can lead to clinical diagnoses such as relapsing polychondritis, polyarteritis nodosa, Sweet syndrome, and myelodysplastic syndrome. Diagnosis requires bone marrow evaluation to identify cytoplasmic vacuoles in myeloid and erythroid precursors. However, genetic confirmation of mutations in UBA1 is necessary. Treatment is challenging and often involves glucocorticoids and immunosuppressants with variable responses. Hypomethylating agents and allogenic haemopoietic stem cell transplant are considered promising therapies. Prognosis is influenced by genetic and clinical factors. The aim of this review is to provide an overview of the pathogenesis, clinical presentation, treatment, and prognosis of VEXAS syndrome for the Latin American medical community.

摘要

VEXAS(空泡、E1 酶、X 连锁、自身炎症、体细胞)综合征是一种成人发病的自身炎症综合征,其特征是 UBA1 基因的体细胞突变,被认为是血液炎症性疾病的原型。VEXAS 综合征患者表现出炎症和血液学表现,可导致复发性多软骨炎、结节性多动脉炎、Sweet 综合征和骨髓增生异常综合征等临床诊断。诊断需要骨髓评估以识别髓系和红系前体细胞中的细胞质空泡。然而,需要对 UBA1 中的突变进行基因确认。治疗具有挑战性,通常涉及糖皮质激素和免疫抑制剂,但反应各不相同。低甲基化剂和同种异体造血干细胞移植被认为是有前途的治疗方法。预后受遗传和临床因素的影响。本文旨在为拉丁美洲医学界提供 VEXAS 综合征的发病机制、临床表现、治疗和预后的概述。

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