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.
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 diseasepatients 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 WDchildren (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.