Abiraterone in Classic Congenital Adrenal Hyperplasia: Results of Medical Therapy Before Adrenalectomy

Author:

Stuckey Bronwyn G A123ORCID,Dedic Deila4,Zhang Rui5ORCID,Rabbah Amira6,Turcu Adina F6ORCID,Auchus Richard J67ORCID

Affiliation:

1. Keogh Institute for Medical Research , Nedlands, Western Australia 6009 , Australia

2. Department of Endocrinology and Diabetes, Sir Charles Gairdner Hospital , Nedlands, Western Australia 6009 , Australia

3. Medical School, University of Western Australia , Nedlands, Western Australia 6009 , Australia

4. Murdoch Endocrinology , Murdoch, Western Australia 6150 , Australia

5. Department of Biochemistry, PathWest Laboratory Medicine , Nedlands, Western Australia 6009 , Australia

6. Department of Internal Medicine/Division of Metabolism, Endocrinology, and Metabolism, University of Michigan , Ann Arbor, MI 48109 , USA

7. Department of Pharmacology, University of Michigan , Ann Arbor, MI 48109 , USA

Abstract

Abstract We present the case of a 20-year-old woman with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency, with uncontrolled hyperandrogenemia despite supraphysiological glucocorticoid therapy. We used abiraterone acetate, an inhibitor of the 17-hydroxylase/17,20-lyase enzyme, to suppress adrenal androgen synthesis and allow physiological glucocorticoid and mineralocorticoid therapy, as a proof-of-concept, before proceeding to bilateral adrenalectomy. We report the patient's clinical course, the changes in adrenal steroids, and the immunohistochemistry of the adrenals.

Funder

Janssen-Cilag Pty Ltd

Publisher

The Endocrine Society

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