Multicenter, Nationwide, Observational, Retrospective and Prospective Study Based on the Development of a Autoimmune Hepatitis Patient's Database Linked to a Biological Sample Storage
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 10,000
- 试验地点
- 1
- 主要终点
- Number of participants with Normalization of AST and ALT <1 x upper limit of normal (ULN)) and normal IgG levels within 6 months
研究概览
简要总结
Autoimmune Hepatitis (AIH) is chronic fibroinflammatory disease of the liver characterized by chronic, relapsing liver inflammation, and a risk for progression to liver failure and need for liver transplantation. No AIH-specific registry does exist in Italy, so that the actual epidemiology of the disease in the country is unknown.
This is an observational, retrospective and prospective, multicenter study evaluating incidence, prevalence and disease course of AIH in subjects > 1 years old in Italy.
详细描述
Autoimmune Hepatitis (AIH) is a chronic fibroinflammatory disease of the liver characterized by chronic, relapsing inflammation, and a risk for progression to liver failure and development of hepatocellular cancer. Both children and adults are affected. A significant fraction of patients with AIH has a diminished life expectancy, despite treatment. Around 80% of patients respond to current therapies, but their quality of life and health is hugely impaired by side effects. The remaining proportion of patients (the difficult-to-treat category) do not respond to treatment and progress to liver cirrhosis and its complications; thus, timely identification of these individuals is a key aspect of epidemiological research in AIH.Taken together and combined with patients' debilitating quality of life issues, these data highlight the considerable disease burden and clinical impact of AIH on patients' outcomes.
Most epidemiological studies on AIH are retrospective case series based on tertiary referral series with relevant selection biases. Population-based studies, that include all cases in a defined geographical area, provide more accurate estimates of incidence, survival and mortality rate for the individual with AIH. Typically, multiple case-finding approaches have been used, including surveys, laboratory reports, liver histology databases, transplant registries, and death certificates. Only few population-based studies have been performed and limit the research population to a few dozen patients, reporting incidence rates ranging from 1.07 to 1.9 per 100,000 inhabitants. Reported point prevalence is 16.9 per 100,000.
To the investigators knowledge, there have been no epidemiologic studies in AIH carried out in Italy.
The investigators aim to develop a national registry on this rare disease to describe incidence and prevalence of AIH in Italy; identify and define distinct phenotypes and sub-phenotypes of AIH patients; identify factors influencing the progression of AIH and affecting mortality; assess safety and long-term efficacy of novel therapies.
This is a multicentre, nationwide, observational and prospective study based on the development of a patient's database linked to a biological sample storage. The enrollment period will cover 120 months to include the required number of patients. Based on litteraure, the estimated prevalence of AIH in Italy ranges from 6,000 to 15,000. Based on this, the investigators estimate to enroll approximately 10,000 patients. The minimum follow-up time will be 1 year.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •All AIH patients living in Italy and aged at least 1 year can be included in the database.
- •Willing and able to give informed consent prior to any study specific procedure being
- •Diagnosis of AIH according to the most recent published guidelines (EASL)
排除标准
- •Subject unwilling to participate at the study
结局指标
主要结局
Number of participants with Normalization of AST and ALT <1 x upper limit of normal (ULN)) and normal IgG levels within 6 months
时间窗: Overall duration of the study (11 years)
Number of participants with Normalization of AST and ALT \<1 x upper limit of normal (ULN)) and normal IgG levels within 6 monthswithin 6 months
Number of participants with Hepatitis Activity Index <4/18
时间窗: Overall duration of the study (11 years)
Number of participants with Hepatitis Activity Index \<4/18
Deep biochemical remissionof disease progression
时间窗: Overall duration of the study (11 years)
ALT less than 50% ULN and IgG\<12g/l
Number of participants with variceal bleeding requiring hospital admission
时间窗: Overall duration of the study (11 years)
Number of participants with variceal bleeding requiring hospital admission
Number of participants with platelet count < 150.000/mm3 on more than one consecutive sample
时间窗: Overall duration of the study (11 years)
Number of participants with platelet count \< 150.000/mm3 on more than one consecutive sample
Number of participants with albumin levels < 3.5 mg/dL on more than one consecutive sample
时间窗: Overall duration of the study (11 years)
Number of participants with albumin levels \< 3.5 mg/dL on more than one consecutive sample
Number of participants with lack of biochemical response
时间窗: Overall duration of the study (11 years)
Number of participants with lack of biochemical response
Intolerance to treatment
时间窗: Overall duration of the study (11 years)
Any adverse event possibly related to treatment as assessed by the treating physician leading to potential discontinuation of the drug
Number of participants with An increase in serum ALT levels above ULN on at least two occasions with an interval of 4 weeks
时间窗: Overall duration of the study (11 years)
Number of participants with An increase in serum ALT levels above ULN on at least two occasions with an interval of 4 weeks, with or without concomitant clinical symptoms and reinstitution of drug therapy after exclusion of other plausible causes for the elevated serum ALT
Number of participants with <50% decrease of AST and ALT within 4 weeks after initiation of treatment
时间窗: Overall duration of the study (11 years)
Number of participants with \<50% decrease of AST and ALT within 4 weeks after initiation of treatment
Relapse
时间窗: Overall duration of the study (11 years)
An increase in serum ALT levels above three times the ULN and/or an increase in serum IgG levels to more than 20 g/l
Number of participants with first evidence of ascites requiring hospital admission
时间窗: Overall duration of the study (11 years)
Number of participants with first evidence of ascites requiring hospital admission
Number of participants with hepatic encephalopathy requiring hospital admission
时间窗: Overall duration of the study (11 years)
Number of participants with hepatic encephalopathy requiring hospital admission
Number of participants with serum bilirubin levels > 1.0 mg/dl on more than one consecutive sample
时间窗: Overall duration of the study (11 years)
Number of participants with serum bilirubin levels \> 1.0 mg/dl on more than one consecutive sample
次要结局
- Number of participants achieving HAI score < 4 at follow-up biopsies(Overall duration of the study (11 years))
- Liver stiffness(Overall duration of the study (11 years))
- The time from the diagnosis of AIH to an event(Overall duration of the study (11 years))