Autoimmune Hepatitis in Pediatric Patients

Autoimmune Hepatitis Clinical Trial

Official title:

Multicentric Study on the Long-term Follow-up of Autoimmune Hepatitis in the Pediatric Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04247490
• Other study ID #: 637/2019/Oss/AOUFe
• Status: Completed
• Start date: December 17, 2019
• Est. completion date: December 31, 2019

Study information

• Verified date: January 2020
• Source: University Hospital of Ferrara
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

Autoimmune hepatitis (AIH) is an immune-mediated and inflammatory liver disorder. It is currently divided into types 1 and 2, differentiated and defined by the presence of specific autoantibodies. The objectives are to describe the prevalence and incidence of type 1 and 2 autoimmune hepatitis and to analyze the clinics, biochemical and histopathological profiles at diagnosis and follow-up, initial therapy, response to therapy and long-term follow-up in three Italian centers of patients with type 1 and type 2 AIH.

Description:

Background: Autoimmune hepatitis (AIH) is an immune-mediated and inflammatory liver disorder with typical onset in pediatric age. It is currently divided into types 1 and 2, differentiated and defined by the presence of specific autoantibodies. AIH type 1 (AIH-1) is characterised by smooth muscle and/or antinuclear autoantibodies (SMA/ANA) while anti-liver kidney microsomal (anti-LKM) or liver cytosol type 1 (anti-LC1) identified AIH type 2 (AIH-2). The conventional treatment is based on corticosteroids and azathioprine as first line therapy. The optimal duration is not clear definied, however it should be extended for at least five years due to the frequent relapses.

Aims of the study: The primary objective is to describe the prevalence and incidence of type 1 and 2 autoimmune hepatitis on a selected italian pediatric population. Secondary objectives are to analyze the clinical, biochemical and histopathological profiles at diagnosis and follow-up, type of treatment received, response to therapy and long-term follow-up.

Methods: The investigators conducted a retrospective and prospective cohort study on patients aged ≤ 18 years who the diagnosis of AIH 1 and 2 between 01/01/1989 and 31/12/2019 and were followed in three Italian pediatric centers.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 109
• Est. completion date: December 31, 2019
• Est. primary completion date: December 31, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 18 Years

Eligibility

Inclusion Criteria:

• Age = 18 years at the time of diagnosis

• Diagnosis of autoimmune hepatitis type 1 or 2 according to the Mieli-Vergani score: a pre-treatment score> 15 or post-treatment> 17 indicates a definite diagnosis of Autoimmune Hepatitis; a pre-treatment score between 10 and 15 or post-treatment between 12 and 17 indicates a probable diagnosis.

Exclusion Criteria:

• Age> 18 years at the time of diagnosis

• Presence of other causes of liver disease

• Presence of overlap syndrome (hepatito-cholangitis)

Related Conditions & MeSH terms

• Autoimmune Hepatitis
• Hepatitis
• Hepatitis A
• Hepatitis, Autoimmune

Intervention

Diagnostic Test:
• pediatric AIH
pediatric patients with clinical, laboratory (transaminase and IgG increase), instrumental (ultrasound, liver biopsy) characteristics

Locations

Country	Name	City	State
Italy	University Hospital of Ferrara	Cona	Ferrara

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital of Ferrara

Country where clinical trial is conducted

Italy, 

References & Publications (6)

Alvarez F, Berg PA, Bianchi FB, Bianchi L, Burroughs AK, Cancado EL, Chapman RW, Cooksley WG, Czaja AJ, Desmet VJ, Donaldson PT, Eddleston AL, Fainboim L, Heathcote J, Homberg JC, Hoofnagle JH, Kakumu S, Krawitt EL, Mackay IR, MacSween RN, Maddrey WC, Man — View Citation

Jiménez-Rivera C, Ling SC, Ahmed N, Yap J, Aglipay M, Barrowman N, Graitson S, Critch J, Rashid M, Ng VL, Roberts EA, Brill H, Dowhaniuk JK, Bruce G, Bax K, Deneau M, Guttman OR, Schreiber RA, Martin S, Alvarez F. Incidence and Characteristics of Autoimmune Hepatitis. Pediatrics. 2015 Nov;136(5):e1237-48. doi: 10.1542/peds.2015-0578. Epub 2015 Oct 19. — View Citation

Maggiore G, Porta G, Bernard O, Hadchouel M, Alvarez F, Homberg JC, Alagille D. Autoimmune hepatitis with initial presentation as acute hepatic failure in young children. J Pediatr. 1990 Feb;116(2):280-2. — View Citation

Mieli-Vergani G, Vergani D, Baumann U, Czubkowski P, Debray D, Dezsofi A, Fischler B, Gupte G, Hierro L, Indolfi G, Jahnel J, Smets F, Verkade HJ, Hadžic N. Diagnosis and Management of Pediatric Autoimmune Liver Disease: ESPGHAN Hepatology Committee Posit — View Citation

Nastasio S, Sciveres M, Matarazzo L, Maggiore G. Old and New Treatments for Pediatric Autoimmune Hepatitis. Curr Pediatr Rev. 2018;14(3):187-195. doi: 10.2174/1573396314666180516130314. Review. — View Citation

Verma S, Torbenson M, Thuluvath PJ. The impact of ethnicity on the natural history of autoimmune hepatitis. Hepatology. 2007 Dec;46(6):1828-35. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Prevalence and incidence	The primary objective was to calculate the proportion of AIH type 1 and 2 patients present in the Italian pediatric population at a given time (prevalence) and the proportion of new AIH type 1 and 2 patients in the Italian pediatric population (0-18 years) in a given period of time (incidence).	from 01/01/1989 to 31/12/2019
Secondary	Characteristics of AIH in pediatric patients	Describe the baseline characteristics (age of onset, clinical-laboratory, radiological and histological characteristics), the response to therapy (medical and / or the possible use of liver transplantation) and the follow-up of patients with autoimmune hepatitis type 1 and 2.	from 01/01/1989 to 31/12/2019