Prediction of Portal Hypertension in Patients With CVID (CVID-pHT)
Study Details
Study Description
Brief Summary
Patients with CVID will be offered to participate in this observational trial during the routine annual visit in the outpatient clinic at the Center of chronic Immunodeficiency (CCI) of the University Medical Center Freiburg, Germany.
Clinical and laboratory data at the time of presentation will be assessed. Additionally, parameters of abdominal ultrasound, duplex sonography of the liver and spleen, and liver and spleen stiffness at the time of presentation will be evaluated. If applicable, clinical and/or interventional parameters indicating clinically significant portal hypertension (i.e. presence of varices or portal-hypertensive gastropathy in esophago-gastroduodenoscopy, presence of ascites) within 12 months prior and after the index visit will be assessed. During the visit, serum/plasma samples and peripheral blood mononuclear cells (PBMC) are collected and stored in an associated biobank.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
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Detailed Description
Patients with CVID can be included in the study. The study cohort will consist of three subgroups: 1) Patients with CVID and autoimmunity, but no liver involvement. 2) Patients with CVID and liver involvement, defined by elevated liver enzymes, abnormal ultrasound and/or abnormal liver biopsy, but no signs of clinically significant portal hypertension and 3) Patients with CVID and clinically significant non-cirrhotic portal hypertension. Further, patients with CVID and diagnosed non-cirrhotic portal hypertension between 01/01/2004 and the start of the prospective part of this study (01/01/2023) will be included retrospectively in this study.
Patients will be recruited during the routine annual visit in the outpatient clinic at the Center of chronic Immunodeficiency (CCI) of the University Medical Center Freiburg, Germany. During the visit a standard ultrasound protocol, including size and morphology of liver and spleen, duplex-sonography of the liver, and measurement of liver and spleen stiffness via transient elastography will be performed as a part of the routine clinical diagnostic. Results from endoscopy or other interventions (esophago-gastroduodenoscopy, invasive measurement of hepatic venous-portal pressure gradient, or TIPS implantation, if applicable) within 12 months prior and after the index visit will be assessed. All non-invasive and invasive interventions will be conducted solely based on clinical decisions made independently from the study inclusion as the study is designed as an observational study.
Detailed patient characteristics, epidemiologic, clinical, imaging and laboratory parameters will be assessed and included in an electronic database.
Apart from these data, patients will be asked to participate in biobank sampling including serum/plasma and peripheral blood mononuclear cells (PBMC) samples from the peripheral veins.
All patients recruited in this study will be followed-up for at least 12 months.
Outcome parameters include parameters from abdominal ultrasound, duplex-sonography and stiffness of liver and spleen, presence or development of clinically significant portal hypertension and non-invasive fibrosis scores based on laboratory and ultrasound parameters.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Patients with CVID and autoimmunity and no liver involvement. Patients with CVID and autoimmune disease, but no liver involvement (normal liver enzymes and normal abdominal ultrasound) |
Diagnostic Test: Ultrasound including color doppler ultrasound
Patients with CVID receive color doppler ultrasound of the liver including assessment of portal vein diameter, portal vein flow velocity, hepatic vein diameter and assessment of arterial perfusion. Further liver and spleen stiffness measurement by transient elastography is performed.
Other Names:
|
| Patients with CVID and liver involvement and no clinical significant portal hypertension Patients with CVID and liver involvement (elevated liver enzymes, abnormal ultrasound and/or abnormal liver biopsy), but no clinically significant portal hypertension |
Diagnostic Test: Ultrasound including color doppler ultrasound
Patients with CVID receive color doppler ultrasound of the liver including assessment of portal vein diameter, portal vein flow velocity, hepatic vein diameter and assessment of arterial perfusion. Further liver and spleen stiffness measurement by transient elastography is performed.
Other Names:
|
| Patients with CVID and clinically significant non-cirrhotic portal hypertension Patients with CVID and clinically significant non-cirrhotic portal hypertension (indicated by the presence of ascites or esophageal varices / portal-hypertensive gastropathy). |
Diagnostic Test: Ultrasound including color doppler ultrasound
Patients with CVID receive color doppler ultrasound of the liver including assessment of portal vein diameter, portal vein flow velocity, hepatic vein diameter and assessment of arterial perfusion. Further liver and spleen stiffness measurement by transient elastography is performed.
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Differences in liver stiffness between CVID patients with and without liver involvement and with clinically significant portal hypertension. [Study inclusion, after 12 months]
Liver stiffness will be assessed by transient elastography.
- Differences in spleen stiffness between CVID patients with and without liver involvement and with clinically significant portal hypertension. [Study inclusion, after 12 months]
Spleen stiffness will be assessed by transient elastography.
Secondary Outcome Measures
- Differences in portal vein diameter between CVID patients with and without liver involvement and with clinically significant portal hypertension. [Study inclusion, after 12 months]
Portal vein diameter will be assessed by color doppler ultrasound
- Differences in portal vein flow velocity between CVID patients with and without liver involvement and with clinically significant portal hypertension. [Study inclusion, after 12 months]
Portal vein flow velocity will be assessed by color doppler ultrasound
- Differences in alanine aminotransfersases (ALT) between CVID patients with and without liver involvement and with clinically significant portal hypertension. [Study inclusion, after 12 months]
ALT is assessed during routine clinical practice
- Differences in aspartat aminotransfersases (AST) between CVID patients with and without liver involvement and with clinically significant portal hypertension. [Study inclusion, after 12 months]
AST is assessed during routine clinical practice
- Differences in liver function tests (bilirubin, albumin, INR) between CVID patients with and without liver involvement and with clinically significant portal hypertension. [Study inclusion, after 12 months]
Liver function liver function tests (bilirubin, albumin, INR) are assessed during routine clinical practice
- Differences in platelets between CVID patients with and without liver involvement and with clinically significant portal hypertension. [Study inclusion, after 12 months]
Platelets are assessed during routine clinical practice
Eligibility Criteria
Criteria
Inclusion Criteria:
- Patients with CVID
Exclusion Criteria:
-
no written informed consent
-
concomitant chronic liver disease (viral hepatitis, alcoholic liver disease, steatitic liver disease, hemochromatosis, primary biliary cholangitis, primary sclerosis cholangitis, M. Wilson, alpha-1-antitrypsin deficiency)
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University Medical Center Freiburg, Department of Medicine II | Freiburg | Germany | 79106 |
Sponsors and Collaborators
- University Hospital Freiburg
Investigators
- Principal Investigator: Dominik Bettinger, MD, University Hospital Freiburg
- Principal Investigator: Klaus Warnatz, MD, University Hospital Freiburg
- Principal Investigator: Marlene Reincke, MD, University Hospital Freiburg
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- CVID-pHT