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 language | English |
|---|---|
| Pages (from-to) | 331-336 |
| Number of pages | 6 |
| Journal | Journal of Pediatric Gastroenterology and Nutrition |
| Volume | 39 |
| Issue number | 4 |
| Publication status | Published - Oct 2004 |
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ASJC Scopus subject areas
- Gastroenterology
- Histology
- Medicine (miscellaneous)
- Food Science
- Pediatrics, Perinatology, and Child Health
Cite this
Serum transaminases in children with Wilson's disease. / Iorio, Raffaele; D'Ambrosi, Mariangela; Marcellini, Matilde; Barbera, Cristiana; Maggiore, Giuseppe; Zancan, Lucia; Giacchino, Raffaella; Vajro, Pietro; Marazzi, Maria Grazia; Francavilla, Ruggiero; Michielutti, Fabio; Resti, Massimo; Frediani, Tullio; Pastore, Maria; Mazzarella, Giuseppina; Fusco, Giuseppina; Cirillo, Francesco; Vegnente, Angela.
In: Journal of Pediatric Gastroenterology and Nutrition, Vol. 39, No. 4, 10.2004, p. 331-336.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Serum transaminases in children with Wilson's disease.
AU - Iorio, Raffaele
AU - D'Ambrosi, Mariangela
AU - Marcellini, Matilde
AU - Barbera, Cristiana
AU - Maggiore, Giuseppe
AU - Zancan, Lucia
AU - Giacchino, Raffaella
AU - Vajro, Pietro
AU - Marazzi, Maria Grazia
AU - Francavilla, Ruggiero
AU - Michielutti, Fabio
AU - Resti, Massimo
AU - Frediani, Tullio
AU - Pastore, Maria
AU - Mazzarella, Giuseppina
AU - Fusco, Giuseppina
AU - Cirillo, Francesco
AU - Vegnente, Angela
PY - 2004/10
Y1 - 2004/10
N2 - 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.
AB - 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.
UR - http://www.scopus.com/inward/record.url?scp=30744436753&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=30744436753&partnerID=8YFLogxK
M3 - Article
C2 - 15448420
AN - SCOPUS:30744436753
VL - 39
SP - 331
EP - 336
JO - Journal of Pediatric Gastroenterology and Nutrition
JF - Journal of Pediatric Gastroenterology and Nutrition
SN - 0277-2116
IS - 4
ER -