Treatment of Polycystic Ovary Syndrome (PCOS)
All women with PCOS, regardless of body weight, should receive multicomponent lifestyle intervention as first-line treatment, combining dietary modification (500-750 kcal/day deficit), structured physical activity (150 minutes/week moderate-intensity or 75 minutes/week vigorous-intensity), and behavioral strategies, because insulin resistance affects all PCOS patients independent of BMI. 1, 2
Initial Assessment and Risk Stratification
Before initiating treatment, exclude other causes of androgen excess including Cushing's syndrome, androgen-secreting tumors, nonclassic congenital adrenal hyperplasia, thyroid disease, hyperprolactinemia, and acromegaly 2. Perform essential laboratory tests: TSH, prolactin, total or free testosterone, fasting glucose, and fasting lipid panel 2. Calculate BMI and measure waist circumference using ethnic-specific cutoffs—Asian, Hispanic, and South Asian populations require lower thresholds due to higher cardiometabolic risk 1, 2.
First-Line Treatment: Multicomponent Lifestyle Intervention
Dietary Management
Prescribe a balanced diet with an energy deficit of 500-750 kcal/day (targeting 1,200-1,500 kcal/day total), tailored to individual energy requirements, body weight, and physical activity levels 1, 3. No specific diet type has proven superior—focus on patient preferences and cultural needs while maintaining nutritional balance 1, 3. Evidence supports low glycemic index foods, high-fiber diets, omega-3 fatty acid-rich diets, ketogenic diets, Mediterranean diets, and anti-inflammatory diets for improving insulin sensitivity and hormonal balance 1, 4. Avoid unduly restrictive or nutritionally unbalanced diets 1.
Physical Activity Prescription
Prescribe at least 150 minutes/week of moderate-intensity exercise (brisk walking, cycling 8-15 km/h, low-impact aerobics, yoga) or 75 minutes/week of vigorous-intensity activity (jogging/running, high-impact aerobics, competitive sports), performed in at least 10-minute bouts 1, 3. Include muscle-strengthening activities involving major muscle groups on 2 non-consecutive days per week 1, 3. For patients with excess weight requiring greater weight loss, increase to at least 250 minutes/week of moderate-intensity or 150 minutes/week of vigorous-intensity activity 1, 3. Target 10,000 steps daily, including 30 minutes of structured physical activity 1. Minimize sedentary, screen, and sitting time throughout the day 1.
Both aerobic and resistance exercise improve insulin sensitivity and metabolic outcomes in PCOS, with benefits occurring independently of significant weight loss 1, 3. Start with realistic 10-minute activity bouts and progressively increase physical activity by 5% weekly 1.
Behavioral Strategies
Implement SMART (specific, measurable, achievable, realistic, timely) goal setting with self-monitoring using fitness tracking devices for step count and exercise intensity 1, 3. Include behavioral change techniques: goal-setting, stimulus control, problem-solving, assertiveness training, slower eating, reinforcing changes, and relapse prevention 1, 3. Consider comprehensive behavioral or cognitive behavioral interventions to increase support and adherence 1.
Monitoring and Realistic Goals
Target 5-10% weight loss in patients with excess weight—this achievable goal yields significant clinical improvements within 6 months 1, 3. Monitor weight and waist circumference regularly during weight loss and maintenance 1, 3. Implement fortnightly review for the first 3 months with structured dietary and physical activity plans, then regular review for the first 12 months to ensure adherence and adjust interventions 1.
Medical Management for Women NOT Attempting to Conceive
Hormonal Therapy
Prescribe combined oral contraceptive pills as first-line hormonal therapy for women not attempting to conceive 1, 2. COCs suppress ovarian androgen secretion, increase sex hormone-binding globulin, regulate menstrual cycles, prevent endometrial hyperplasia, and reduce hirsutism and acne 1, 2. A typical regimen is drospirenone 3 mg/ethinyl estradiol 20 μg in a 24-active/4-inert pill regimen, taken daily 1. COCs reduce the risk of endometrial cancer but are associated with increases in circulating triglyceride and HDL cholesterol levels 1.
Insulin-Sensitizing Agents
Prescribe metformin 500-2000 mg daily for patients with insulin resistance or glucose intolerance, starting at 500 mg daily and titrating up to 1000-2000 mg daily in divided doses 1. Metformin improves glucose tolerance over time and may have a positive impact on risk factors for diabetes and cardiovascular disease 1, 2. Consider GLP-1 receptor agonists (liraglutide, semaglutide, exenatide) in combination with lifestyle interventions for weight loss and metabolic control 1.
Antiandrogen Therapy
For hirsutism, combined medical interventions (antiandrogen plus ovarian suppression agent) may be most effective 1, 2. Medroxyprogesterone acetate suppresses circulating androgen and pituitary gonadotropin levels, though optimal progestin, duration, and frequency to prevent endometrial cancer in PCOS is not known 1.
Medical Management for Women ATTEMPTING to Conceive
Ovulation Induction
Prescribe clomiphene citrate as first-line pharmacological treatment for ovulation induction 1, 2, 5. Approximately 80% of patients ovulate and 50% conceive with clomiphene 1, 2. Clomiphene is indicated for ovulatory dysfunction in women desiring pregnancy, with properly timed coitus in relationship to ovulation 5. Each course should be started on or about the 5th day of the cycle, with long-term cyclic therapy not recommended beyond a total of about six cycles (including three ovulatory cycles) 5.
Weight control and regular exercise programs should be started before medication 1. If clomiphene treatment fails, low-dose gonadotropin therapy should be used, which induces a high rate of monofollicular development with a lower risk of ovarian hyperstimulation 1.
Preconception Metformin
Consider metformin 1 g twice daily for 12 weeks or until pregnancy in the pregestational stage 1.
Critical Pitfalls to Avoid
Do not dismiss lifestyle intervention in lean PCOS patients simply because they have normal BMI—insulin resistance requires management regardless of weight. 1 Insulin resistance is present irrespective of BMI and affects both lean and overweight women with PCOS, contributing to hyperandrogenism through effects on the pituitary, liver, and ovaries 1.
Ensure health professional interactions are respectful and patient-centered, avoiding weight-related stigma which negatively impacts treatment engagement 1, 3. Perform pelvic examination prior to the first and each subsequent course of clomiphene citrate treatment 5. Patients with polycystic ovary syndrome who are unusually sensitive to gonadotropin may have an exaggerated response to usual doses of clomiphene citrate and should be started on the lowest recommended dose 5.
Monitor for ovarian hyperstimulation syndrome (OHSS), which may progress rapidly (within 24 hours to several days) and become a serious medical disorder 5. Early warning signs include abdominal pain and distention, nausea, vomiting, diarrhea, and weight gain 5. If enlargement of the ovary occurs, additional clomiphene citrate therapy should not be given until the ovaries have returned to pretreatment size 5.
Discontinue clomiphene treatment if visual symptoms occur (blurring, scotomata, phosphenes) and perform complete ophthalmological evaluation promptly 5.
Psychological Management
Screen patients with PCOS for eating disorders (binge eating disorder, night eating syndrome), which are highly prevalent and create a vicious cycle worsening both obesity and hormonal disturbances 1. Refer patients with moderately severe depression to psychiatry or behavioral health for evaluation and potential pharmacotherapy, as depression dramatically reduces adherence to lifestyle interventions 1. Address body image concerns and psychological distress related to hair loss and PCOS symptoms, which significantly impact treatment adherence 1.
Special Population Considerations
Adolescents
Prevention of weight gain and monitoring should begin from adolescence, as weight gain escalates from this period 1. Adolescents with PCOS should aim for at least 60 minutes of moderate to vigorous intensity physical activity daily, including activities that strengthen muscle and bone at least 3 times weekly 1. Hormonal contraceptives and metformin are the treatment options in adolescents with PCOS 1.
High-Risk Ethnic Groups
Asian, Hispanic, and South Asian populations require lower BMI and waist circumference thresholds for intervention and greater consideration for lifestyle intervention due to high cardiometabolic risk 1, 2. Use ethnic-specific BMI and waist circumference categories when optimizing lifestyle and weight 1, 3.