Liver Failure, Wilson Disease
Conditions
Keywords
Wilson Disease, Liver Failure, plasma exchange, continuous hemodiafiltration
Brief summary
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
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.
Interventions
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.
Sponsors
Third Affiliated Hospital, Sun Yat-Sen University
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
1. Clinical diagnosis of Wilson Disease: serum ceruloplasmin \< 0.2g/L, 24 hours urinary copper \> 40μg, Kayser-Fleischer ring present; 2. Do not receive drugs to promote the excretion of copper ever before; 3. Serum total bilirubin \> 10 times upper limit of normal, prothrombin time activity \< 40% or prothrombin time international ratio \> 1.5.
Exclusion criteria
1. Other active liver diseases; 2. Hepatocellular carcinoma or other malignancy; 3. Pregnancy or lactation; 4. Human immunodeficiency virus infection or congenital immune deficiency diseases; 5. Severe diabetes, autoimmune diseases; 6. Other important organ dysfunctions.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| survival rate | 48 weeks | Whether patients will survive is observed in the follow-up. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| liver function improvement | 48 weeks | 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. |
Countries
China
Outcome results
None listed