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

Liver Failure Clinical Trial

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT03589820
• Other study ID #: XWX1
• Status: Enrolling by invitation
• Phase: N/A
• Start date: June 1, 2018
• Est. completion date: July 31, 2020

Study information

• Verified date: July 2018
• Source: Third Affiliated Hospital, Sun Yat-Sen University
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 60
• Est. completion date: July 31, 2020
• Est. primary completion date: June 28, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 65 Years

Eligibility

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.

Related Conditions & MeSH terms

• Liver Failure
• Wilson Disease

Intervention

Other:
• artificial liver support system
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.

Locations

Country	Name	City	State
China	The Third Affiliated Hospital of Sun Yat-sen University	Guangzhou	Guangdong

Sponsors (1)

Lead Sponsor	Collaborator
Third Affiliated Hospital, Sun Yat-Sen University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	survival rate	Whether patients will survive is observed in the follow-up.	48 weeks
Secondary	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