Clinical trials of penicillamine in Indian childhood cirrhosis.

The outcome in 15 children with advanced Indian childhood cirrhosis (ICC) treated with penicillamine 20 mg/kg/day was not significantly different from that in untreated children. Among children admitted to a further double blind trial who had ICC but who had not yet developed jaundice or ascites 10 treated with penicillamine and 10 treated with penicillamine plus prednisolone had a significantly improved survival. Fourteen of 29 treated cases made a clinical recovery and were alive 489 to 1460 days from the start of treatment. Biopsy specimens in survivors showed a return to normal liver histology in three, residual fibrosis in six, and inactive micronodular cirrhosis in five. Thus penicillamine, while not shown to be beneficial in advanced ICC, lowered mortality from 93% to 52% in preicteric cases of ICC.