Treatment Options for Polycystic Ovary Syndrome (PCOS)
Multicomponent lifestyle intervention including diet, exercise, and behavioral strategies is the first-line treatment for all women with PCOS, regardless of weight status, with pharmacological therapy reserved for specific symptoms or fertility needs. 1, 2, 3
First-Line Treatment: Lifestyle Modification
Dietary Interventions
All women with PCOS should follow a balanced dietary approach with no specific diet type proven superior to others. 1, 2, 3 The evidence supports multiple dietary patterns including low glycemic index foods, Mediterranean diet, high-fiber diets, and omega-3 fatty acid-rich foods, all of which improve insulin sensitivity and hormonal balance. 4
For women with excess weight requiring weight loss:
- Target an energy deficit of 30% or 500-750 kcal/day (total intake 1,200-1,500 kcal/day), adjusted for individual energy requirements, body weight, and physical activity levels. 2, 3
- Aim for 5-10% weight loss within 6 months, which yields significant clinical improvements in metabolic and reproductive outcomes. 2, 5
- Avoid unduly restrictive or nutritionally unbalanced diets. 3
Physical Activity Requirements
For prevention of weight gain and health maintenance, prescribe a minimum of 150 minutes/week of moderate-intensity physical activity or 75 minutes/week of vigorous-intensity activity (or equivalent combination). 2, 3
For weight loss and greater metabolic benefits, prescribe a minimum of 250 minutes/week of moderate-intensity activities or 150 minutes/week of vigorous-intensity activity (or equivalent combination). 2, 3
- Include muscle-strengthening activities on 2 non-consecutive days per week. 3
- Activity should be performed in at least 10-minute bouts, aiming for at least 30 minutes daily on most days. 3
- Both aerobic exercise (brisk walking, cycling 8-15 km/h, low-impact aerobics, yoga) and resistance training improve insulin sensitivity and metabolic outcomes. 2, 4
- Minimize sedentary, screen, and sitting time. 3
Behavioral Strategies
Implement SMART (specific, measurable, achievable, realistic, timely) goal setting and self-monitoring as core behavioral interventions. 2, 3
Additional behavioral strategies include:
- Goal-setting and self-monitoring 2, 3
- Stimulus control and problem-solving 2, 3
- Assertiveness training and slower eating 2, 3
- Reinforcing changes and relapse prevention 2, 3
- Comprehensive behavioral or cognitive behavioral interventions to increase engagement and adherence 3
- Address psychological factors including anxiety, depression, body image concerns, and disordered eating 3, 5
Pharmacological Treatment for Specific Indications
For Ovulatory Dysfunction and Infertility
Clomiphene citrate is the first-line pharmacological therapy for ovulation induction in women with PCOS desiring pregnancy. 6, 7
Key prescribing considerations for clomiphene:
- Indicated specifically for ovulatory dysfunction in women with PCOS desiring pregnancy 6
- Start each course on or about the 5th day of the cycle 6
- Long-term cyclic therapy should not exceed approximately six cycles (including three ovulatory cycles) 6
- Patients must be evaluated to ensure they are not pregnant, have no ovarian cysts (except polycystic ovaries), have no abnormal vaginal bleeding, and have normal liver function 6
- Women with PCOS may have exaggerated response to usual doses; start with the lowest recommended dose and shortest treatment duration for the first course. 6
Critical warning: Ovarian hyperstimulation syndrome (OHSS) can occur with clomiphene therapy, progressing rapidly within 24 hours to several days into a serious medical disorder with gross ovarian enlargement, ascites, dyspnea, oliguria, and pleural effusion. 6 Early warning signs include abdominal pain and distention, nausea, vomiting, diarrhea, and weight gain. 6
For Metabolic Abnormalities
Metformin is beneficial for metabolic and glycemic abnormalities and for improving menstrual irregularities, but has limited or no benefit in treating hirsutism, acne, or infertility. 7
- Metformin is particularly useful in women with insulin resistance and metabolic dysfunction 8, 7
- It can be used as complementary pharmacological support to lifestyle intervention 9
For Menstrual Abnormalities and Hyperandrogenism
Hormonal contraceptives are first-line pharmacological management for menstrual abnormalities, hirsutism, and acne in PCOS. 7
- Hormonal contraceptives are also appropriate treatment options in adolescents with PCOS 7
Monitoring and Follow-Up
Regular monitoring of weight and waist circumference is essential during weight loss and maintenance phases. 2
- Use ethnic-specific BMI and waist circumference categories for ethnic groups at high cardiometabolic risk 3, 5
- Comprehensive metabolic evaluation is crucial for women with obesity 5
- Screen for risk factors including endometrial cancer, mood disorders, obstructive sleep apnea, diabetes, and cardiovascular disease 7
Special Populations and Considerations
Ethnic groups with PCOS who are at high cardiometabolic risk require greater consideration for lifestyle intervention with ethnic-specific BMI and waist circumference thresholds. 2, 3, 5
In adolescents with PCOS, hyperandrogenism is central to the diagnosis, and treatment options include hormonal contraceptives and metformin. 7
Healthy lifestyle interventions may contribute to health and quality of life benefits even in the absence of weight loss. 2, 3
Common Pitfalls and Caveats
- Weight gain over time is significantly greater in women with PCOS than in unaffected women, with progressive increase in waist-to-hip ratio between ages 20-45 years, making early intervention crucial. 2
- Avoid weight-related stigma in health professional interactions, as this negatively impacts engagement with treatment. 2
- Thiazolidinediones have an unfavorable risk-benefit ratio overall and are not recommended. 7
- The role of weight loss in improving PCOS status per se remains uncertain, but lifestyle intervention provides clear benefits for overall health in overweight/obese patients. 7
- High-fat diets exacerbate obesity, impair hypothalamic-pituitary-ovarian axis function, and worsen metabolic and reproductive consequences of PCOS. 10