Successful Use of Metyrapone Suppositories in an Infant with Neonatal Cushing and McCune Albright Syndrome: A Case Report.

ABSTRACT:

Introduction

Perinatal hypercorticism, regardless of its cause, has a high mortality or leads to life-long lasting complications. Some publications reported on the use of metyrapone in children with McCune Albright syndrome (MAS) and hypercorticism but also mentioned its poor tolerability.

Case presentation

We present the case of a toddler in whom we diagnosed MAS based on pseudo-precocious puberty and hypercorticism at the age of 10 months. In light of hepatopathy, we decided to start the off-label treatment with metyrapone. Being aware of the challenges of this treatment with the only available product (gelatine capsule containing liquid metyrapone) and reports on local irritation and significant side effects after the oral and intrarectal administration of liquid metyrapo

SUBMITTER: Ertl DA 

PROVIDER: S-EPMC11854969 | biostudies-literature | 2025

REPOSITORIES: biostudies-literature