What are the treatment options for a woman with Polycystic Ovary Syndrome (PCOS)?

Treatment of Polycystic Ovary Syndrome (PCOS)

All women with PCOS must begin with lifestyle modification targeting 5-10% weight loss through diet and exercise as the mandatory foundation of treatment, regardless of body weight, reproductive goals, or symptom severity. 1, 2

Foundational Lifestyle Intervention (Required for All Patients)

• Target a 500-750 kcal/day energy deficit with total intake of 1,200-1,500 kcal/day, adjusted for individual energy requirements. 1, 2
• Any balanced dietary approach creating an energy deficit is acceptable—no specific diet type (low-carb, Mediterranean, etc.) has proven superior in PCOS compared to general population recommendations. 1, 2
• Prescribe at least 150 minutes/week of moderate-intensity exercise or 75 minutes/week of vigorous activity, plus muscle-strengthening activities on 2 non-consecutive days/week. 1, 2
• Exercise provides metabolic benefits even without weight loss, so continue exercise recommendations regardless of weight loss success. 3, 1
• Weight loss of as little as 5% of initial body weight improves metabolic, reproductive, and hormonal abnormalities in PCOS. 3, 2

Treatment Algorithm Based on Reproductive Goals

For Women NOT Attempting to Conceive:

Combined oral contraceptive pills (OCPs) are the first-line pharmacological treatment. 1, 2, 4

• OCPs suppress ovarian androgen secretion, increase sex hormone-binding globulin, regulate menstrual cycles, prevent endometrial hyperplasia, and reduce hirsutism and acne. 1, 2, 4
• Add metformin 500-2000 mg daily when insulin resistance or glucose intolerance is documented, lifestyle modifications alone are insufficient for metabolic control, or the patient has obesity or elevated cardiovascular risk factors. 1, 2
• Metformin improves glucose tolerance and insulin sensitivity, though its role in preventing cardiovascular disease remains unknown. 3, 2

For Women WITH Hirsutism:

Combine OCP with spironolactone 50-200 mg daily, as this combination provides superior results compared to monotherapy. 1

• The combination of an antiandrogen (spironolactone) and ovarian suppression (OCP) appears most effective, though the optimal specific agents are not definitively established. 3
• Topical eflornithine hydrochloride cream is the only FDA-labeled treatment specifically for hirsutism, though additional benefits or risks in PCOS are unknown. 3
• Mechanical hair removal (plucking, shaving, waxing), electrolysis, and laser vaporization are adjunctive options, but concomitant medical management to reduce androgen levels is usually necessary. 3

For Women Attempting to Conceive:

Clomiphene citrate is the first-line pharmacological treatment for ovulation induction. 3, 1, 2, 5

• Approximately 80% of PCOS patients ovulate with clomiphene citrate, and half of those who ovulate conceive. 3, 2
• Start clomiphene citrate on or about the 5th day of the cycle, and limit to six total cycles (including three ovulatory cycles). 1, 5
• If clomiphene treatment fails, use low-dose gonadotropin therapy rather than high-dose, as low-dose treatment induces monofollicular development with lower risk of ovarian hyperstimulation. 3, 2
• Metformin appears safe during pregnancy and may reduce miscarriage risk, though effects on early pregnancy are not fully known. 3, 4
• Thiazolidinediones have poor documentation regarding effects on early pregnancy and should not be used in pregnancy. 3, 4

Mandatory Metabolic Screening (All Patients Regardless of Weight)

Screen all PCOS patients for metabolic abnormalities at diagnosis and at least annually. 1, 2

• Obtain fasting glucose followed by 2-hour glucose tolerance test to screen for diabetes. 1, 2
• Obtain fasting lipid profile. 1, 2
• Calculate BMI and waist-hip ratio. 1, 2
• Use ethnic-specific BMI and waist circumference categories for Asian, Hispanic, and South Asian populations who are at higher cardiometabolic risk. 1, 2

Emerging Therapies

GLP-1 receptor agonists (semaglutide, liraglutide, exenatide) show promise for weight reduction and metabolic improvement when combined with lifestyle interventions, and may be considered for patients with obesity or elevated cardiovascular risk factors when lifestyle and metformin are insufficient. 1, 2

• Bariatric surgery is emerging as a potential adjunct therapy for PCOS patients with clinically severe obesity, with reported improvements in hirsutism, menstrual regularity, and fertility. 1

Critical Pitfalls to Avoid

• Do not delay lifestyle intervention—it must be the foundation of treatment, not an afterthought. 1, 4
• Do not assume normal weight excludes metabolic dysfunction; screen all PCOS patients regardless of BMI. 1, 4
• Do not use spironolactone as monotherapy in patients attempting to conceive; use clomiphene citrate instead. 1, 4
• Do not exceed recommended clomiphene dosage and duration; limit to six total cycles. 1, 4, 5
• Do not use thiazolidinediones in pregnancy due to poorly documented effects on early pregnancy. 4
• The benefit and role of laparoscopic ovarian drilling by laser or diathermy are undetermined in PCOS. 3