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

Bronwyn G A Stuckey; Deila Dedic; Rui Zhang; Amira Rabbah; Adina F Turcu; Richard J Auchus

doi:10.1210/jcemcr/luae077

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

JCEM Case Rep. 2024 May 24;2(6):luae077. doi: 10.1210/jcemcr/luae077. eCollection 2024 Jun.

Authors

Bronwyn G A Stuckey^{1

2

3}, Deila Dedic⁴, Rui Zhang⁵, Amira Rabbah⁶, Adina F Turcu⁶, Richard J Auchus^{6

7}

Affiliations

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

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.

Keywords: 21-hydroxylase deficiency; abiraterone acetate; adrenalectomy; classic congenital adrenal hyperplasia.

Publication types

Case Reports