Clinical trial NCT04247490: Autoimmune Hepatitis in Pediatric Patients
Official title: Multicentric Study on the Long-term Follow-up of Autoimmune Hepatitis in the Pediatric Patients
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Autoimmune Hepatitis
Keywords: Autoimmune hepatitis
incidence
prevalence
immunosuppressive treatmen
liver transplantation
Lead sponsor: University Hospital of Ferrara
Sponsor class: OTHER
First posted: 2020-01-30
Enrollment: 109
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population is represented by all the patients who presented the onset of Autoimmune hepatitis type 1 and type 2 from 01/01/1989 to 31/12/2019 and were followed at the Pediatric Hepatology Centers of Ferrara (Hospital Saint Anna of Cona), Naples (Federico II University Hospital) and Palermo (Mediterranean Institute for Transplants and Highly Specialized Therapies).

Brief 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.

Detailed 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.

Eligibility criteria: 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)

Primary outcomes:
measure: Prevalence and incidence
timeFrame: from 01/01/1989 to 31/12/2019
Secondary outcomes:
measure: Characteristics of AIH in pediatric patients
timeFrame: from 01/01/1989 to 31/12/2019
Other outcomes:
Locations:
city: Cona
country: Italy
facility: University Hospital of Ferrara
state: Ferrara
Study officials:
References:
citation: Jimenez-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.
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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, Hadzic N. Diagnosis and Management of Pediatric Autoimmune Liver Disease: ESPGHAN Hepatology Committee Position Statement. J Pediatr Gastroenterol Nutr. 2018 Feb;66(2):345-360. doi: 10.1097/MPG.0000000000001801.