The Burden of Atypical Hemolytic Uremic Syndrome and The Clinical Characteristics of Patients in Egyptian Hospitals A Multicenter, Observational, Retrospective Cohort Study in Egypt
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- AstraZeneca
- 入组人数
- 200
- 试验地点
- 14
- 主要终点
- Demographic and clinical characteristics of patients with aHUS.
研究概览
简要总结
Atypical hemolytic uremic syndrome (aHUS) is a rare, progressive, and life-threatening disease that occurs at any age, with incidence rate of 0.75 to 2.0 cases per million population per year. aHUS is a thrombotic microangiopathy (TMA) commonly caused by dysregulation of the complement system, affecting several organs, especially the kidneys. aHUS can be familial or sporadic, and approximately 50% to 60% of patients have specific identifiable genetic complement mutations and antibodies.
Although aHUS is a rare disease, it has a significant impact on the quality of life because of its poor prognosis: a 25% global mortality rate; more than 50% of untreated patients advance to endstage renal disease (ESRD); and more than 75% of adults with renal failure require prompt dialysis. The risk of relapse is also high in many patients, either in the native or transplanted kidneys, so long-term management and close monitoring are essential.
Advancements in aHUS therapies, especially the availability of anti-complement therapy, have enhanced the natural course of aHUS through hematologic remission induction, kidney function stabilization or improvement, and graft failure prevention. Since complement inhibitors are still unavailable in Egypt, it is important to understand the aHUS manifestations of pediatrics and adults in Egyptian hospitals, aiming for early diagnosis and proper management.
In this study, we primarily aim to describe the aHUS burden on the patients by gathering their demographic and clinical characteristics, documented disease course, and long-term complications. Our secondary objectives include an estimate of the prevalence of patients diagnosed with aHUS out of all patients with TMA and gathering information about the clinical outcomes of available therapies in real-world settings, as there is no data from the country on aHUS management.
研究设计
- 研究类型
- Observational
- 观察模型
- Case Control
- 时间视角
- Retrospective
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patients aged one month or older who have been diagnosed with TMA between 01-Jan-2010 and 31-Dec-2023.
排除标准
- •None. All records of patients with TMA will be screened for aHUS diagnosis.
结局指标
主要结局
Demographic and clinical characteristics of patients with aHUS.
时间窗: 10 Years
Duration between the first aHUS treatment initiation and aHUS diagnosis
次要结局
- The prevalence of patients diagnosed with aHUS(10 Years)
- clinical outcomes of current treatment(10 Years)
- aHUS Treatment Patterns(10 Years)
- aHUS Treatment Outcomes(10 Years)