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Efficacy and safety of D-penicillamine, trientine, and zinc in pediatric Wilson disease patients.
Lee, Eun Joo; Woo, Min Hyung; Moon, Jin Soo; Ko, Jae Sung.
Afiliação
  • Lee EJ; Department of Pediatrics, Severance Hospital, Yonsei University College of Medicine, Seoul, Korea.
  • Woo MH; Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine, Seoul, Korea.
  • Moon JS; Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine, Seoul, Korea.
  • Ko JS; Department of Pediatrics, Seoul National University Children's Hospital, Seoul National University College of Medicine, Seoul, Korea. kojs@snu.ac.kr.
Orphanet J Rare Dis ; 19(1): 261, 2024 Jul 09.
Article em En | MEDLINE | ID: mdl-38982450
ABSTRACT

OBJECTIVES:

Wilson disease (WD) is a rare genetic disease affecting copper metabolism and the biliary tract's copper excretion. Lifelong medication is necessary to prevent liver failure, neurological complications, and death. Although D-penicillamine (DPA), trientine, and zinc are used to treat WD, there is limited research on the long-term outcomes of these drugs, especially in children. This study aimed to evaluate the efficacy and safety of DPA, trientine, and zinc in patients diagnosed with WD during childhood.

METHODS:

Ninety out of 92 patients were included in the analysis, excluding two patients who underwent liver transplantation without drug treatment due to an acute liver failure diagnosis. Treatment outcomes and reasons for discontinuation of therapy in 148 treatment blocks (37 DPA, 50 trientine, and 61 zinc) were analyzed using Kaplan-Meier analysis.

RESULTS:

The median age at diagnosis was 8.3 years. There was a statistically significant difference in drug changes due to treatment ineffectiveness among the three drugs trientine (22/50, 44%), zinc (15/61, 25%), and DPA (2/37, 5%) (all p < 0.05). Regarding drug changes due to adverse effects, the rate was the highest for DPA, followed by zinc and trientine. There were significant differences between DPA and zinc, zinc and trientine (all p < 0.05), but no significant difference was observed between DPA and zinc (p = 0.22).

CONCLUSIONS:

In pediatric WD, DPA, zinc, and trientine have therapeutic effects in that order. However, DPA and zinc are associated with more adverse effects compared to trientine.
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Penicilamina / Trientina / Zinco / Degeneração Hepatolenticular Limite: Adolescent / Child / Child, preschool / Female / Humans / Male Idioma: En Ano de publicação: 2024 Tipo de documento: Article

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Penicilamina / Trientina / Zinco / Degeneração Hepatolenticular Limite: Adolescent / Child / Child, preschool / Female / Humans / Male Idioma: En Ano de publicação: 2024 Tipo de documento: Article