Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson's Disease-related Liver Failure

Study Overview
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Brief Summary
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This study is to investigate the clinical efficacy of artificial liver support system using combination of plasma exchange and continuous hemodiafiltration in treatment of Wilson's Disease - related liver failure. 30 patients will receive treatment of plasma exchange and continuous hemodiafiltration and internal medicine. 30 patients will receive treatment of internal medicine.

Detailed Description
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Wilson's disease (WD) is an inherited disorder in which defective biliary excretion of copper leads to its accumulation, particularly in liver and brain. Presenting symptoms of liver disease can be highly variable, ranging from asymptomatic, with only biochemical abnormalities, to overt cirrhosis with all its complications. Acute liver failure (ALF) presentation is the most dramatic and may arise with catastrophic suddenness. It is considered rare, constituting only 3% of ALF cases in the pediatric ALF series in USA and associated with a high mortality reaching 95%. Patients with acute liver failure due to WD require liver transplantation, which is lifesaving. Kido J et al reported clinical outcomes of patients with liver failure were improved by artificial liver support system using combination of plasma exchange and continuous hemodiafiltration. So the investigators design this protocol: 30 patients will receive treatment of plasma exchange and continuous hemodiafiltration and internal medicine, 30 patients will receive treatment of internal medicine.

Official Title
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Clinical Efficacy of Artificial Liver Support System Using Combination of Plasma Exchange and Continuous Hemodiafiltration in Treatment of Wilson's Disease - Related Liver Failure

Conditions
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Wilson Disease, Liver Failure

Intervention / Treatment
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* Other: artificial liver support system


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Clinical diagnosis of Wilson Disease: serum ceruloplasmin < 0.2g/L, 24 hours urinary copper > 40μg, Kayser-Fleischer ring present; Do not receive drugs to promote the excretion of copper ever before; Serum total bilirubin > 10 times upper limit of normal, prothrombin time activity < 40% or prothrombin time international ratio > 1.5. Exclusion Criteria: Other active liver diseases; Hepatocellular carcinoma or other malignancy; Pregnancy or lactation; Human immunodeficiency virus infection or congenital immune deficiency diseases; Severe diabetes, autoimmune diseases; Other important organ dysfunctions.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Artificial liver support system group<br>30 patients will receive treatment of artificial liver support system using combination of plasma exchange and continuous hemodiafiltration and internal medicine. | Other: artificial liver support system<br>* Patients will receive the treatment of artificial liver support system using combination of plasma exchange and continuous hemodiafiltration and internal medicine. The volume of fresh frozen plasma used in plasma exchange is 2000 millilitre. The time for continuous hemodiafiltration is about 8 hours.<br>|
| No Intervention: Control group<br>30 patients will receive treatment of internal medicine. | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| survival rate | Whether patients will survive is observed in the follow-up. | 48 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| liver function improvement | Symptoms (i.e. fatigue, appetite, nausea, vomiting, jaundice, consciousness) and laboratory tests (i.e. blood cells, alanine transaminase, total bilirubin, prothrombin time, creatinine, ceruloplasmin, serum copper) are observed in the follow-up. | 48 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Wilson Disease, Liver Failure, plasma exchange, continuous hemodiafiltration