High prevalence of primary adrenal insufficiency in Zellweger spectrum disorders

被引:0
作者
Kevin Berendse
Marc Engelen
Gabor E Linthorst
AS Paul van Trotsenburg
Bwee Tien Poll-The
机构
[1] Emma Children's Hospital/Academic Medical Centre,Department of Paediatric Neurology
[2] Emma Children's Hospital/Academic Medical Centre,Laboratory for Genetic Metabolic Diseases
[3] Emma Children's Hospital/Academic Medical Centre,Department of Endocrinology and Metabolism
[4] Emma Children's Hospital/Academic Medical Centre,Department of Paediatric Endocrinology
来源
Orphanet Journal of Rare Diseases | / 9卷
关键词
Adrenal insufficiency; peroxisomal disorders; Very long-chain fatty acids; Zellweger spectrum disorders; X-linked adrenoleukodystrophy;
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摘要
Zellweger spectrum disorders are a group of autosomal recessive disorders characterized by impaired peroxisome functions. The clinical spectrum is broad, ranging from the classical most severe Zellweger syndrome to patients with a relatively mild phenotype. Treatment options are limited to symptomatic and supportive therapy. During routine follow-up we discovered patients with asymptomatic primary adrenal insufficiency. It is important to detect impaired adrenal function because it has treatment implications. Primary adrenal insufficiency was found in 7/24 patients examined, with 4/7 being asymptomatic. Systematic evaluation of adrenal function, through a Synacthen test, should be included in the clinical management of these patients.
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