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Long term survival in Indian childhood cirrhosis treated with D-penicillamine
A R Bavdekar1, S A Bhave, A M Pradhan
1Department of Paediatrics, King Edward Memorial Hospital, Pune, India.
Insights
Penicillamine treatment can improve outcomes for children with Indian childhood cirrhosis (ICC). Long-term follow-up shows significant recovery, suggesting ICC is not an inborn copper metabolism error.
Area of Science:
- Hepatology
- Pediatric Gastroenterology
- Clinical Toxicology
Background:
- Indian childhood cirrhosis (ICC) is a severe, often fatal pediatric liver disease.
- Early intervention with penicillamine may alter the disease's prognosis.
- The long-term effects and underlying etiology of ICC require further investigation.
Purpose of the Study:
- To assess the long-term outcomes of children treated with penicillamine for Indian childhood cirrhosis.
- To evaluate the clinical, biochemical, histological, and sonographic recovery from ICC.
- To investigate the potential role of copper metabolism in the pathogenesis of ICC.
Main Methods:
- A retrospective review of 29 children with ICC treated with penicillamine in Pune, India (1980-1987).
- Assessment included clinical examination, liver function tests, liver biopsy, and duplex Doppler ultrasound.
- Follow-up duration was at least five years from the initiation of penicillamine treatment.
Main Results:
- All patients showed significant reduction in hepatosplenomegaly and normalized liver function tests.
- No significant toxic effects of penicillamine were observed, apart from asymptomatic proteinuria.
- Histological review indicated recovery from ICC, with most cases progressing to inactive micronodular cirrhosis or near-normal histology.
Conclusions:
- Penicillamine treatment can lead to significant clinical and biochemical recovery in children with ICC.
- The observed recovery patterns challenge the hypothesis that ICC results from an inborn error of copper metabolism.
- Long-term penicillamine therapy may be necessary for children with persistent micronodular cirrhosis.
Abstract:
Indian childhood cirrhosis (ICC) is an almost uniformly fatal disease whose outcome may be modified with penicillamine if given at a sufficiently early stage. Twenty nine children with ICC seen in Pune, India, in 1980-7, who had survived at least five years from onset of penicillamine treatment, were reviewed aged 6.3 to 13 years. They were assessed clinically, biochemically, histologically, and by duplex Doppler ultrasound examination. None had symptoms suggestive of liver disease. There were no toxic effects of penicillamine other than asymptomatic proteinuria. Hepatosplenomegaly reduced significantly and liver function tests returned to normal in all. In four children, significant hepatosplenomegaly was associated with an abnormal duplex Doppler hepatic vein flow pattern and micronodular cirrhosis on biopsy. Clinical findings, growth and development, and ultrasound examination were normal in the remainder. Review of serial liver biopsy specimens showed a sequence of recovery from ICC through inactive micronodular cirrhosis to virtually normal histological appearances. The four children who still have micronodular cirrhosis beyond four years from onset remain on penicillamine treatment. In the others penicillamine was stopped after 1-7 (mean 3.5) years without relapse, strong evidence that ICC is not due to an inborn error of copper metabolism.