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Effect of chelation therapy in pediatric Wilson's disease: Liver and endoscopic outcome
Mridul Chandra Das1, Moinak Sen Sarma1, Anshu Srivastava1
1Department of Pediatric Gastroenterology, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow, India.
Insights
Chelation therapy effectively manages pediatric hepatic Wilson's disease (WD), improving liver function and portal hypertension. Prognosis is accurately predicted by the Pediatric End-Stage Liver Disease (PELD) and Nazar scores.
Area of Science:
- Hepatology
- Pediatric Gastroenterology
- Genetic Liver Diseases
Background:
- Limited literature exists on pediatric hepatic Wilson's disease (WD).
- This study addresses the efficacy of chelation therapy in managing hepatocellular function and portal hypertension in pediatric WD patients.
Purpose of the Study:
- To evaluate the effectiveness of chelation therapy in pediatric hepatic Wilson's disease.
- To assess the impact of treatment on liver function and portal hypertension.
- To identify predictors of outcome in pediatric WD.
Main Methods:
- 111 pediatric Wilson's disease patients with at least 9 months of follow-up were analyzed.
- Response to chelation therapy was categorized into complete remission, partial remission, disease progression, or drug toxicity.
- Pediatric End-Stage Liver Disease (PELD) and Nazar scores were compared, alongside endoscopic evaluations for esophageal varices.
Main Results:
- 71% of patients achieved favorable outcomes (remission), while 29% showed disease progression, and 10.8% experienced drug toxicity.
- Esophageal varices, present in two-thirds of patients, did not progress, and large varices were effectively managed.
- Baseline PELD and Nazar scores were independent predictors of outcome, showing a strong positive correlation (r=0.864, P<.001).
Conclusions:
- D-penicillamine monotherapy is effective for managing most pediatric hepatic Wilson's disease cases, even with severe liver disease.
- The Pediatric End-Stage Liver Disease (PELD) score and Nazar score are reliable tools for predicting patient outcomes.
- Chelation therapy demonstrates efficacy in improving liver function and managing complications like portal hypertension in pediatric WD.
Background:
As there is paucity of exclusive literature on pediatric hepatic Wilson's disease (WD), this study was undertaken to evaluate the efficacy of chelation on hepatocellular function and portal hypertension in WD.
Methods:
Wilson's disease patients with ≥9 months of follow-up were evaluated for response to chelation therapy in the following categories: (a) complete remission, (b) partial remission (c) progression of disease; (d) drug toxicity. Pediatric end-stage liver disease (PELD), Nazar and New Wilson Index scores were compared. Hemodynamically stable patients underwent esophagogastroduodenoscopy (baseline and surveillance) and received prophylaxis (primary or secondary). Endoscopic outcome was assessed at follow-up.
Results:
Of the 111 WD children (aged 9 [3-15] years; PELD score 16 [-11 to 60]), 65 with follow-up of 3.6 (0.8-12) years on chelation (83% D-penicillamine monotherapy, 17% D-penicillamine and zinc) were analyzed. 81% had severe disease at presentation. Favorable outcome (complete and or partial remission), progression of disease and drug toxicity were seen in 71%, 29% and 10.8%, respectively. Two-thirds had esophageal varices which did not show progression. Large esophageal varices (16%) were effectively downgraded in 3 (2-6) therapeutic endoscopic sessions. Nazar score and PELD score at baseline were independent predictors of outcome with favorable correlation with each other (r = .864, P < .001). PELD cutoff 9.45 (AUC: 71%, sensitivity: 87%, specificity: 50%; P = .009) and Nazar score cut off 3.5 (AUC: 68%, sensitivity: 83%, specificity: 50%; P = .02) were associated with poor prognosis.
Conclusions:
Despite severe liver disease, the majority of hepatic WD can be managed on D-penicillamine monotherapy. PELD score and Nazar score effectively determine the outcome.
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