Autoimmune liver diseases (AILD), which include Primary Sclerosing Cholangitis (PSC) and Autoimmune Hepatitis (AIH) are a common etiological factor for chronic liver disease among adolescents. This is a longitudinal study to identify surrogate endpoints with an accurate predictive value for the progression of hepatobiliary damage in subjects with pediatric onset AILD. This study will involve collection of MRI-based data at the time of enrollment and at year 1 and 2 of follow up, and collection of clinical data for 10 years following enrollment. There is a strong possibility that MRI quantitative techniques may be more sensitive to disease progression than standard clinical and laboratory tests. To investigate predictivity of MRI based biomarkers, summary measures of MRCP/MREL from baseline, Year 1 and Year 2, e.g. change rate, maximum, and average will be calculated as predictors for Year 10 clinical outcomes. The same predictors will also be used to model native liver survival in a proportional hazard regression. Findings from this study may be used to assess disease progression and to predict complications and survival of liver disease patients.
Study Type
OBSERVATIONAL
Enrollment
150
Cincinnati Children's Hospital and Medical Center
Cincinnati, Ohio, United States
RECRUITINGChange of intrahepatic bile duct irregularities between V0 (baseline visit) and V1 (visit after12 months) or V2 (visit after 24 months).
Change of intrahepatic bile duct irregularities between V0 and V1 or V2 by MRCP (scored by Majoie classification on 4 point scale of 0-3).
Time frame: 24 months
Change of extra-hepatic duct irregularities between V0 (baseline visit) and V1 (visit after 12 months) or V2 (visit after 24 months).
Change of extra-hepatic duct irregularities between V0 and V1 or V2 by MRCP (scored by Majoie classification on 5 point scale 0-4).
Time frame: 24 months
Mean shear stiffness of the liver
Change in mean shear stiffness (kPa) of the liver by MREL between V0 (baseline visit) and V1 ( visit after 12 months) or V2 (visit after 24 months).
Time frame: 24 months
long-term clinical outcomes: survival with the native liver
Annual assessment of survival with the native liver (Yes=1, No=0) will be done within 10 years of follow-up.
Time frame: 120 months
long-term clinical outcomes: hospital admissions for cholangitis
Annual assessment of long-term clinical outcomes will be done within 10 years of follow-up. Any hospital admissions for cholangitis (Yes=1, No=0) since last visit will be recorded at the time of follow-up.
Time frame: 120 months
long-term clinical outcomes:endoscopic interventions for biliary strictures
Annual assessment of long-term clinical outcomes will be done within 10 years of follow-up. Endoscopic interventions for biliary strictures (Yes=1, No=0) since last visit will be recorded at the time of follow-up.
Time frame: 120 months
long-term clinical outcomes:diagnosis of cholangiocarcinoma
Annual assessment of long-term clinical outcomes will be done within 10 years of follow-up. If there is diagnosis of cholangiocarcinoma (Yes=1, No=0) since last visit will be recorded.
Time frame: 120 months
long-term clinical outcomes: variceal bleeding
Annual assessment of long-term clinical outcomes will be done within 10 years of follow-up. Presence or absence of variceal bleeding (Yes=1, No=0) since last visit will be recorded.
Time frame: 120 months
long-term clinical outcomes: ascites
Annual assessment of long-term clinical outcomes will be done within 10 years of follow-up. Presence or absence of ascites (Yes=1, No=0) since last visit will be recorded.
Time frame: 120 months
Changes in liver/spleen volumes
Changes in liver/spleen volumes (mL) between V0 (baseline visit) and V1 (visit after 12 months) or V2 (visit after 24 months).
Time frame: 24 months
Changes in T1rho, T1 and T2 mapping
Readouts of T1rho, T1 and T2 mapping between baseline MRI at V0 (baseline visit) and repeat ones at V1 (after12 months) or V2 (after 24 months) in msec.
Time frame: 24 months
Clinical endpoints of AILD: Pruritus
Annual assessment for Pruritus (on visual analogue scale of 0-10) will be done within 10 years of follow-up.
Time frame: 120 months
Clinical endpoints of AILD
Annual assessment for Clinical diagnosis of hepatopulmonary syndrome (Yes=1, No=0) and/or hepatic encephalopathy (Yes=1, No=0) will be done within 10 years of follow-up.
Time frame: 120 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.