Long-term treatment of Barth syndrome with pantothenic acid: a retrospective study

被引：11

作者：

Rugolotto, S

Prioli, MD

Toniolo, D

Pellegrino, P

Catuogno, S

Burlina, AB ^{[1
]}

机构：

[1] Univ Padua, Azienda Osped, Dept Pediat, Metab Unit, Padua, Italy

[2] Univ Verona, Mother & Child Dept, Pediat Sect, I-37100 Verona, Italy

[3] Univ Verona, Dept Cardiol, I-37100 Verona, Italy

[4] CNR, Inst Genet Biochem & Evolut, Padua, Italy

来源：

MOLECULAR GENETICS AND METABOLISM | 2003年 / 80卷 / 04期

关键词：

Barth syndrome; 3-methylglutaconic aciduria type II; neutropenia; dilated cardiomyopathy; pantothenic acid;

D O I：

10.1016/j.ymgme.2003.07.002

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

We report on three patients (two are brothers) with confirmed Barth syndrome treated with pantothenic acid. This treatment is still controversial and only one study has reported positive results to date. In our patients, long-term treatment has failed to reduce the number of infectious episodes and prevent dilated cardiomyopathy. Our cases show that this treatment is not as effective in Barth syndrome as was previously claimed. (C) 2003 Published by Elsevier Inc.

引用

页码：408 / 411

页数：4

共 13 条

[1] HEART-TRANSPLANTATION FOR DILATED CARDIOMYOPATHY [J].