title: Worsening of Wilson disease following penicillamine therapy.
abstract: BACKGROUND
  Penicillamine is a standard therapy for Wilson disease (WD) but some patients have paradoxical worsening. Predictors of such deterioration have not been evaluated. This study documents frequency and predictors of deterioration following treatment in WD.
  
  
  METHODS
  59 consecutive patients with neurologic WD and 4 asymptomatic siblings were prospectively evaluated. Their clinical, laboratory, ultrasound abdomen and cranial MRI findings with and without worsening were compared. Patients were treated with oral penicillamine and/or zinc and followed up at 1, 3 and 6 months or earlier if needed. Deterioration was defined by >10% worsening in baseline Burke-Fahn-Marsden score or appearance of new neurological sign.
  
  
  RESULTS
  Patients' median age was 13 years and 13 were females. 19 patients (30.2%) worsened following treatment; 10 within 1 month, 7 in 1-3 months, and 2 after 3 months of treatment. Deterioration was associated with drooling, leukopenia, thrombocytopenia, splenomegaly and evidence of chronic liver disease. None of the asymptomatic patients following zinc therapy deteriorated.
  
  
  CONCLUSIONS
  In the deteriorating group, withdrawal of penicillamine resulted in improvement/stabilization in 11 patients, 2 improved by trientine therapy and 4 continued to deteriorate till 3 months. 30.2% patients with WD deteriorated following penicillamine, especially those with chronic liver disease, leukopenia and thrombocytopenia.
authors.affiliation: Department of Neurology, Sanjay Gandhi Post Graduate Institute of Medical Sciences, Lucknow, India.
authors.forename: Jayantee
authors.initials: J
authors.lastname: Kalita
authors.forename: Vijay
authors.initials: V
authors.lastname: Kumar
authors.forename: Satish
authors.initials: S
authors.lastname: Chandra
authors.forename: Bishwanath
authors.initials: B
authors.lastname: Kumar
authors.forename: Usha Kant
authors.initials: UK
authors.lastname: Misra
mesh_terms: D000293:Adolescent; D002614:Chelating Agents / Q000009:adverse effects*; D002648:Child; D005260:Female; D006527:Hepatolenticular Degeneration / Q000188:drug therapy*; D006801:Humans; D008297:Male; D010396:Penicillamine / Q000009:adverse effects*; D016896:Treatment Outcome
chemical_list: D002614:Chelating Agents; D010396:Penicillamine