Abstract
Interim encouraging results of a D-penicillamine trial in ICC (presented to ESPGAN 1984) are now confirmed. 30 children with biopsy-proven ICC who had not yet developed jaundice or ascites were treated double blind with: D-penicillamine (Group P, n=10); penicillamine + prednisolone 2 mg/kg/d for 4 weeks then 5 mg/d (Group PP, n=10); or placebo (Group Plac). Entry parameters were found to be comparable between groups. 9/10 placebo treated children died, median survival 58 days; 1 survives to date (2.7y). 5 in Group P, and 5 in Group PP survive after 1.3-4.0y. Life table analysis (Breslow) showed a significant therapeutic benefit (P vs Plac p=0.01; PP vs Plac p=0.03) but no difference between active treatment groups (p=0.71). Inclusion of a further 13 children treated non-blind did not introduce entry differences between groups but increased significance of survival differences: P'(n=15) vs Plac'(n=14) p=0.002; PP'(n=14) vs Plac' p=0.002. 14/29 P' or PP' treated children survive, are well and have follow-up liver biopsies showing inactive micronodular cirrhosis in 5, portal fibrosis in 6, and minimal abnormalities in 3.
Conclusions: D-penicillamine increased survival in pre-icteric ICC from 1/14 to 14/29, with reversal of cirrhosis in 9, and prednisolone conferred no additional benefit.
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Tanner, M., Bhave, S., John, I. et al. 4. D-PENICILLAMINE INCREASES SURVIVAL IN PRE-ICTERIC INDIAN CHILDHOOD CIRRHOSIS (ICC). Pediatr Res 22, 96 (1987). https://doi.org/10.1203/00006450-198707000-00025
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DOI: https://doi.org/10.1203/00006450-198707000-00025