Wilson disease in children has a varied presentation. Wilson disease with acute liver failure is associated with very high mortality and morbidity. The standard therapy i.e chelation (with either D- penicillamine or trientene can be used as a temporizing agent to treat the enormous release of copper into the blood stream; however, substantial removal is not achieved for at least 1 to 3 months. Plasma exchange provides a means of rapid means of removal of copper. As per American Society for Apheresis, TPE in wilson disease with acute liver failure can rapidly remove an average of 20 mg of copper per TPE treatment. Decreased serum copper may decrease hemolysis, prevent progression of kidney failure and provide clinical stabilization. TPE can also remove large molecular weight toxins (aromatic amino acids, ammonia, endotoxins) and other factors, which may be responsible for hepatic coma. The frequency of said TPE is not defined as most evidence is based on case reports and case series.
Copper is highly protein bound and the volume of distribution for copper is large. Under normal conditions, 90-95% of serum copper is ceruloplasmin-bound with the remaining 5-10% being nonceruloplasmin-bound. TPE efficiently removes both ceruloplasmin- and albumin-bound copper. FFP used for exchange can be helpful in treating the associated coagulopathy. TPE has been used as a bridge to liver transplantation as well as seen to improve survival with native liver, the optimum protocol for same remains uncertain.
Wilson disease with New Wilson Index of ≥ 11 and INR ≥ 2.5
Children aged 3 years to 18 years
Grade 3 or grade 4 hepatic encephalopathy
Septic shock
Disseminated intravascular coagulation
Marked hemodynamic instability requiring a high dose of vasopressors (norepinephrine >0.5 mcg/kg/min)
Parallel
Plasma Exchange
Biological/Vaccine• Plasma exchange (1.5 times plasma exchange) * Blood volume: 80ml/kg * Plasma volume = Blood volume x (1 - Hematocrit/100) * TPE volume = 1.5 x plasma volume * Duration: 4 hours
Standard Medical Treatment
Other interventionStandard Medical Treatment
To compare the reduction in NWI (New Wilson Index) between both groups at the end of three sessions of plasma exchange
The New Wilson Index is a composite score containing of bilirubin, albumin, INR, AST, total leucocyte count ranging from minimum score of 0 till maximum of 20 with a higher score correlating with a worse outcome. A score of NWI \>=11 is associated with increased mortality and considered an indication for consideration for liver transplantation in patients with Wilson disease.
Comparison of change in serum and urine copper levels on day 7 after initiation of plasmapheresis as compared to baseline in alternate versus daily plasma exchange group.
Comparison of overall and native liver survival at day 90 between the two groups
Comparison of change in dialysate copper levels at the end of 3rd session between both groups.
Comparison of total number of sessions of plasma exchange between both groups as on day 28.
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