LONG-TERM SURVIVAL IN INDIAN CHILDHOOD CIRRHOSIS TREATED WITH D-PENICILLAMINE

Indian childhood cirrhosis (ICC) is an almost uniformly fatal disease whose outcome may be modified with penicillamine if given at a suffici ently early stage. Twenty nine children with ICC seen in Pune, India, in 1980-7, who had survived at least five years fi om onset of penicil lamine treatment, were reviewed aged 6.3 to 13 years. They were assess ed clinically, biochemically, histologically, and by duplex Doppler ul trasound examination. None had symptoms suggestive of liver disease. T here were no toxic effects of penicillamine other than asymptomatic pr oteinuria. Hepatosplenomegaly reduced significantly and Liver function tests returned to normal in all. In four children, significant hepato splenomegaly was associated with an abnormal duplex Doppler hepatic ve in flow pattern and micronodular cirrhosis on biopsy. Clinical finding s, growth and development, and ultrasound examination were normal in t he remainder. Review of serial liver biopsy specimens showed a sequenc e of recovery from ICC through inactive micronodular cirrhosis to virt ually normal histological appearances. The four children who still hav e micronodular cirrhosis beyond four years from onset remain on penici llamine treatment. In the others penicillamine was stopped after 1-7 ( mean 3.5) years without relapse, strong evidence that ICC is not due t o an inborn error of copper metabolism.