Polycystic ovary syndrome medical therapy
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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Aditya Ganti M.B.B.S. [2]
Overview
The first step in the management of PCOS is weight loss if the patient is obese, and treatment of type 2 diabetes with metformin. In significantly overweight patients, weight loss alone usually effects a cure and should always be vigorously attempted. Diet and exercise are recommended in all women with PCOS. The next step is initiation of treatment to break the self-perpetuating anovulatory cycling, either by stimulating ovulation or suppressing androgenic and ovarian activity. The selection of treatment depends on whether pregnancy is desired. All anti-androgen treatments will take at least 3 months to affect hirsutism. The drug regimen for PCOS depends upon the desire for fertility of the patient.
Pharmacotherapy
The first step in the management of PCOS is weight loss if the patient is obese, and treatment of type 2 diabetes with metformin. In significantly overweight patients, weight loss alone usually effects a cure and should always be vigorously attempted. Diet and exercise are recommended in all women with PCOS. The next step is initiation of treatment to break the self-perpetuating anovulatory cycling, either by stimulating ovulation or suppressing androgenic and ovarian activity. The selection of treatment depends on whether pregnancy is desired. All anti-androgen treatments will take at least 3 months to affect hirsutism. The goals of treatment are:
- Exclude androgen-secreting tumors, endometrial tumors, and endometrial hyperplasia
- Reduce ovarian androgen secretion and/or antagonist activity at target tissues
- Interrupt the self-sustaining abnormal hormonal cycle
- Normalize the endometrium
- Restore fertility by correcting anovulation, if desired
- Reduce insulin resistance
If fertility is not desired
- Preferred regimen (1): Combined [[|OCP|oral contraceptive pills]] one tablet of formulations containing 30 to 35 μg estrogen orally daily for 21 days, then nothing for 7 days
- Preferred regimen (2): Progesterone-only contraceptive pills (eg, norethindrone, norgestrel ) are the treatment of choice if combined oral contraceptive pills are contraindicated
- Alternative regimen(1): Medroxyprogesterone may be used, although it is not approved by the U.S. Food and Drug Administration (FDA) for this indication
- Alternative regimen(2): Glucocorticoids (eg, hydrocortisone, cortisone, dexamethasone ) may be used to suppress adrenal androgen production, although they are not approved by the FDA for this indication
- Alternative regimen(3): Spironolactone and flutamide are androgen receptor antagonists that may be added to the oral contraceptive pill, but they are not approved by the FDA for this indication; flutamide is not usually recommended because of its unproven efficacy and associated risk of hepatic impairment
If fertility is desired
- Preferred regimen (1): Clomiphene, alone or in combination with glucocorticoids, is the first-choice treatment
- Preferred regimen (2): Follicle-stimulating hormone may be administered in conjunction with timed human chorionic gonadotropin for ovulation induction
- Preferred regimen (3): Metformin
Symptomatic Medical therapy
Treatment of Hirsutism and Acne
- Cyproterone acetate is an anti-androgen, which blocks the action of male hormones that are believed to contribute to acne and the growth of unwanted facial and body hair.
- Cyproterone acetate is also contained in the contraceptive pill Dianette®.
- Spironolactone also has some benefits, again through anti-androgen activity, and metformin can also help.
- Eflornithine is a drug which is applied to the skin in cream form (Vaniqa®), and acts directly on the hair follicles to inhibit hair growth.
- The average reduction in hair growth is generally in the region of 25%, which may not be enough to eliminate the social embarrassment of hirsutism, or the inconvenience of plucking/shaving.
- Individuals may vary in their response to different therapies, and it is usually worth trying other drug treatments if one does not work, but drug treatments do not work well for all individuals.
- Alternatives include electrolysis and various forms of laser therapy.
Treatment of Menstrual Irregularity and Prevention of Endometrial Hyperplasia/Cancer
- Menstruation can be regulated with a contraceptive pill.
- Most brands of contraceptive pill result in a withdrawal bleed every 28 days.
- Dianette® (a contraceptive pill containing cyproterone acetate) is also beneficial for hirsutism and is therefore often prescribed in PCOS.
- If a regular menstrual cycle is not desired, then a standard contraceptive pill is not appropriate.
- Women who are having irregular menses do not necessarily require any therapy; most experts consider that if a menstrual bleed occurs at least every three months, then the endometrium (womb lining) is being shed sufficiently often to prevent an increased risk of endometrial abnormalities or cancer.
- If menstruation occurs less often or not at all, some form of progestogen replacement is recommended. Some women prefer a uterine progestogen implant such as the Mirena® coil, which provides simultaneous contraception and endometrial protection for years, though often with unpredictable minor bleeding.
- An alternative is oral progestogen taken at intervals (e.g. every three months) to induce a predictable menstrual bleed.