Off label drug use in the treatment of polycystic ovary syndrome

This review explores the off-label use of medications used to manage aspects of polycystic ovary syndrome, including androgen excess, irregular menses, and metabolic changes.

Wendy Vitek, M.D., Snigdha Alur, M.D., Kathleen M. Hoeger, M.D., M.P.H.

Volume 103, Issue 3, Pages 605-611


Polycystic ovary syndrome (PCOS) is a complex lifelong disorder with an etiology and pathophysiology that is not yet entirely understood. Women with PCOS have clinical presentations that may vary from adolescence to menopause, including menstrual irregularity/anovulation and symptoms of hyperandrogenism, such as acne and hirsutism. Over a lifetime, treatment needs and requirements can change. Unfortunately, there are no Food and Drug Administration–approved medications that are approved solely for the purpose of PCOS, but the symptoms and presentation of PCOS are often amenable to several approved agents, such as oral contraceptives for the indication of acne and clomiphene citrate for the indication of induction of ovulation. However, to meet the needs of women with PCOS, off-label use of medications has flourished. This review explores the data for those agents that do not carry an indication for PCOS but have been used for treating the signs and symptoms of PCOS.

  • Kathleen Hoeger

    Thank you for the excellent question.

    I agree with your suggestion that we should refrain from using metformin in all
    women diagnosed with PCOS. In accord with the Endocrine Society clinical
    practice guideline on the diagnosis and management of PCOS, metformin should be
    considered in women with T2DM or IGT who fail lifestyle modification and in
    women with irregular cycles who have contraindications to or cannot tolerate
    combination oral contraceptive pills or progestins. The data does not
    support using metformin as a sole agent for the treatment of hirsutism,
    prevention of miscarriage or for the treatment of obesity, although very modest
    weight reduction is seen often with use.

  • In this excellent comprehensive review, some of the controversies in the treatment of PCOS are highlighted. Probably the clinically most important uncertainty surrounds Metformin treatment. A lot of patients I see have already been started on Metformin in the community, most of the time without a clear diagnosis or clear indication. Which PCOS patients should be treated with Metformin? All? Or only those with impaired glucose tolerance? And for what indication(s)? Since Metformin often has significant side effects, clarification of this issue through further research and guidelines would be useful.

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