Non-invasive Assessment of Liver Fibrosis in a French Cohort of Pediatric Patients With Type III Glycogen Storage Disease: Current State and Perspectives

University Hospital, Strasbourg, France30 enrolled

Overview

Patients with type III glycogen storage disease (GSDIII) can develop liver fibrosis, which can be complicated by liver failure or even hepatocellular carcinoma. Since the beginning of the 21st century, non-invasive techniques for assessing fibrosis, such as liver elastography, have been developed. These techniques often make it possible to avoid liver biopsies during patient follow-up and have already been validated in the management of several diseases in adults. These techniques are also beginning to be recommended for monitoring certain chronic liver diseases in children.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Liver Fibrosis

Eligibility

Sex: ALLMin age: 1 MonthMax age: 21 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Minors: from birth to 17 years * Adults: 18 to 21 years * Subjects being monitored for type III glycogen storage disease and having had at least one liver elastography measurement during their follow-up. Exclusion Criteria: \- Patients monitored for type III glycogen storage disease but who had never undergone liver elastography during their follow-up.

Locations (1)

Service de pédiatrie 1 - CHU de Strasbourg - France

Strasbourg, France

Outcomes

Primary Outcomes

Liver elastography values

Liver fibrosis is classified into four stages, from 0 to 4: * F0 corresponds to the absence of fibrosis * F4 corresponds to established cirrhosis.

Time frame: 1 hour after consultation