Serum transaminases in children with Wilson's disease.

Raffaele Iorio, Mariangela D'Ambrosi, Matilde Marcellini, Cristiana Barbera, Giuseppe Maggiore, Lucia Zancan, Raffaella Giacchino, Pietro Vajro, Maria Grazia Marazzi, Ruggiero Francavilla, Fabio Michielutti, Massimo Resti, Tullio Frediani, Maria Pastore, Giuseppina Mazzarella, Giuseppina Fusco, Francesco Cirillo, Angela Vegnente

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Abstract

OBJECTIVES: The response of serum transaminase levels to penicillamine and zinc treatment in Wilson's disease is poorly understood. The aim of this multicenter retrospective study was to evaluate transaminase levels after penicillamine and zinc treatment in children with Wilson's disease. PATIENTS AND METHODS: One hundred and nine patients with Wilson's disease (median age at diagnosis, 7.2 years; range, 1 to 18 years), treated for at least 12 months and observed in the last 20 years at 11 Paediatric Departments were studied. Clinical, laboratory and histologic features at diagnosis and initial treatment were recorded. Efficacy parameters were normalization of serum transaminase level and improved clinical and/or laboratory signs. One hundred and two patients had clinical or laboratory signs of liver disease. RESULTS: Fifty-six of 87 patients (64%) given penicillamine normalized serum alanine aminotransferase (ALT) levels within a median of 17 months (range, 2 to 96 months). Of the 29 patients with persistent hyper-ALT, 17 (59%) switched to zinc; only four of these normalized ALT on zinc within a median period of 38 months (range, 7 to 48 months). Eleven (50%) of the 22 patients given zinc alone normalized ALT within a median period of 6 months (range, 1 to 36 months). Of the 11 patients with persistent hyper-ALT, five switched to penicillamine. Three of the five normalized ALT within a median period of 6 months (range, 6 to 9 months). Overall, in penicillamine-treated and zinc-treated patients with persistent hypertransaminasemia, ALT decreased from a basal median of 236 IU/L (range, 54 to 640 IU/L) to a median of 78 (range, 46 to 960 IU/L) at the end of follow-up (P = 0.0245). Poor compliance was suspected in only 10% of cases. No predictive factor of persistent hypertransaminasemia was identified. Liver disease did not worsen in any patient during the study. CONCLUSIONS: Although the efficacy of penicillamine and zinc is well documented, it is notable that a subset of children with Wilson's disease-related liver disease (36%) had hypertransaminasemia despite appropriate treatment with penicillamine or zinc.

Original languageEnglish
Pages (from-to)331-336
Number of pages6
JournalJournal of Pediatric Gastroenterology and Nutrition
Volume39
Issue number4
Publication statusPublished - Oct 2004

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ASJC Scopus subject areas

  • Gastroenterology
  • Histology
  • Medicine (miscellaneous)
  • Food Science
  • Pediatrics, Perinatology, and Child Health

Cite this

Iorio, R., D'Ambrosi, M., Marcellini, M., Barbera, C., Maggiore, G., Zancan, L., Giacchino, R., Vajro, P., Marazzi, M. G., Francavilla, R., Michielutti, F., Resti, M., Frediani, T., Pastore, M., Mazzarella, G., Fusco, G., Cirillo, F., & Vegnente, A. (2004). Serum transaminases in children with Wilson's disease. Journal of Pediatric Gastroenterology and Nutrition, 39(4), 331-336.