在日本患者中高葡萄糖皮质激素依赖和有限的治疗反应VEXAS综合征:一个多中心的回顾性研究
Ayaka Maeda1, Yohei Kirino1, Naomi Tsuchida1,2,3
1Department of Stem Cell and Immune Regulation, Yokohama City University Graduate School of Medicine, 3-9 Fukuura, Kanazawa-Ku, Yokohama 236-0004.
Modern rheumatology
|October 9, 2025
概括
在日本,治疗VEXAS综合征具有挑战性,治疗选择有限,难以逐渐减少皮质糖类药物. 迫切需要新的疗法来改善患者的治疗结果,并减少类固醇依赖.
科学领域:
- 类风湿病学 类风湿病学
- 遗传学 遗传学 是一个
- 免疫学 免疫学 免疫学
背景情况:
- 维克萨斯综合征是一种严重的自身炎症性疾病,由体质UBA1基因变异引起.
- 治疗选择有限和治疗策略的不确定性使VEXAS综合征的管理变得复杂.
研究的目的:
- 描述日本VEXAS综合征患者的临床治疗和结果.
- 评估当前治疗的疗效,并确定治疗改进的领域.
主要方法:
- 来自46名男性VEXAS综合征患者的临床数据的回顾性分析.
- 使用法国VEXAS组标准对12名患者进行缓解评估.
主要成果:
- 所有患者都接受了全身性皮质糖类药物,通常需要持续高剂量,难以逐渐减少.
- 只有42%的评估患者实现了完全缓解;托西利祖马布是最常见的免疫抑制剂.
- 与国际实践相比,简氏激酶抑制剂和阿扎丁的使用有限.
结论:
- 在日本,维克萨斯综合征的治疗面临着重大挑战.
- 迫切需要新的,有效的疗法来克服葡萄糖皮质激素的依赖,并提高患者的治疗结果.
相关概念视频
Drugs for Treatment of Crohn's Disease in IBD Using Glucocorticoids
434
Glucocorticoids, a class of anti-inflammatory drugs, are pivotal in treating moderate to severe Crohn's disease by inducing remission. They exhibit their anti-inflammatory action by inhibiting the production of inflammatory cytokines such as tumor necrosis factor (TNF)-α, interleukin (IL)-1, and chemokines like IL-8. In addition, they reduce the expression of inflammatory cell adhesion molecules and inhibit gene transcription of nitric oxide synthase, phospholipase A2, cyclooxygenase-2...
434
Antiasthma Drugs: Inhaled Corticosteroids and Glucocorticoids
1.5K
Inhaled corticosteroids (ICS) are anti-inflammatory drugs used primarily in treating persistent asthma and providing long-term maintenance. They target the bronchial mucosa, the lining of the airways, to control inflammation, a critical factor in asthma progression and exacerbation.
ICS work through a multifaceted mechanism of action. They suppress the inflammatory response caused by the proliferation of TH cells. They also reduce the transcription of the IL-2 gene, which is involved in the...
ICS work through a multifaceted mechanism of action. They suppress the inflammatory response caused by the proliferation of TH cells. They also reduce the transcription of the IL-2 gene, which is involved in the...
1.5K
Drugs for Treatment of Crohn's Disease in IBD Using Immunomodulatory Agents
488
Crohn's disease is an inflammatory bowel disorder marked by chronic inflammation of the GI tract. Various treatment strategies for Crohn's disease are employed, such as immunomodulatory agents, glucocorticoids, and biologics or anti-TNF therapy. Azathioprine (Imuran), a commonly used immunomodulatory drug for Crohn's disease, is converted in the body to mercaptopurine, which inhibits purine biosynthesis and cell proliferation. Both are utilized in severe cases of Inflammatory Bowel...
488
Therapeutic Drug Monitoring: Affecting Factors
200
Therapeutic Drug Monitoring (TDM) is the clinical practice of measuring specific drug levels in a patient's blood or body tissues to manage and optimize therapy. TDM is crucial for drugs with narrow therapeutic windows, like warfarin and phenytoin, where incorrect doses can lead to treatment failure or severe side effects. This monitoring ensures the dosage administered is within a safe and effective range. The factors affecting therapeutic drug monitoring include:Patient-Specific Factors:a.
200
COPD: Management Using Bronchodilators and Corticosteroids
726
Chronic obstructive pulmonary isease (COPD) involves a group of progressive lung disorders characterized by persistent airflow limitation and chronic respiratory symptoms. Asthma-COPD Overlap Syndrome (ACOS), encompassing features of both asthma and Chronic obstructive pulmonary disease (COPD), is a group of progressive lung disorders that includes chronic bronchitis, emphysema, and refractory (non-reversible) asthma. ACOS leads to complex clinical presentations that combine the inflammatory...
726
Nephrotic Syndrome II : Assessment and Medical Management
200
IntroductionNephrotic syndrome is a kidney disorder marked by excessive protein loss in the urine, leading to various systemic complications. This condition often results from damage to the glomeruli—the kidney's filtering units—causing proteinuria, low blood protein levels, and fluid retention. Understanding the assessment, diagnosis, and management of nephrotic syndrome is essential for effective treatment and prevention of further kidney damage.AssessmentPatient History: Document...
200


