Treatment Options for Polycystic Ovary Syndrome (PCOS)
Lifestyle modification with multicomponent intervention (diet, exercise, and behavioral strategies) is the first-line treatment for all women with PCOS, regardless of weight or reproductive goals, followed by targeted pharmacotherapy based on whether pregnancy is desired. 1, 2
First-Line Treatment: Lifestyle Modification
All women with PCOS should begin with comprehensive lifestyle intervention before or alongside pharmacological treatment. 1
Weight Management
- Target a 30% energy deficit or 500-750 kcal/day reduction for weight loss 2, 3
- Even modest weight loss of just 5% of initial body weight significantly improves metabolic and reproductive abnormalities, including ovulation and pregnancy rates 3, 4
- Weight loss improves insulin resistance, hyperandrogenism (measured by free androgen index), and BMI 5
- Exercise shows positive effects even without weight loss, so physical activity should be emphasized regardless of weight changes 3
Exercise Recommendations
- Both aerobic and resistance exercise enhance insulin sensitivity, promote weight loss, and improve metabolic and reproductive outcomes 4
- Exercise improves the free androgen index independent of weight changes 5
Dietary Approaches
- No specific diet is superior; focus on caloric restriction and healthy eating patterns 1
- Evidence supports: low glycemic index foods, high-fiber diets, omega-3 fatty acid-rich diets, ketogenic diets, Mediterranean diets, and anti-inflammatory diets 4
- All dietary approaches work primarily through improving insulin sensitivity and hormonal balance 4
Behavioral Strategies
- Include education modules, adherence support, and mental health management 1
- Address depression, anxiety, and eating disorders, which occur at higher rates in PCOS patients 2
Treatment Based on Reproductive Goals
For Women NOT Attempting Pregnancy
Combined oral contraceptives (COCs) are first-line pharmacological treatment for women with PCOS not seeking pregnancy 2
- COCs suppress ovarian androgen secretion and increase sex hormone binding globulin 2
- Address menstrual irregularity, hirsutism, and acne
- Continue lifestyle modifications alongside hormonal therapy 1
Metformin as adjunctive therapy:
- Improves insulin sensitivity and reduces risk factors for diabetes and cardiovascular disease 2
- Reduces insulin levels, which decreases ovarian androgen production 3
- Maintains or improves glucose tolerance over time 3
For Women Attempting Pregnancy
Step 1: Optimize lifestyle first - Weight loss and exercise must be implemented before pharmacological ovulation induction 3
Step 2: Clomiphene citrate is first-line pharmacological treatment for ovulation induction 2, 3, 6
- Approximately 80% of patients ovulate and half of those who ovulate conceive 2, 3
- FDA-approved specifically for ovulatory dysfunction in women with PCOS 6
- Start on or about the 5th day of the cycle 6
- Limit to about six cycles total (including three ovulatory cycles) 6
- Use the lowest effective dose to minimize risk of ovarian hyperstimulation syndrome, particularly in PCOS patients who are unusually sensitive to gonadotropin stimulation 6
Step 3: If clomiphene fails, use low-dose gonadotropin therapy due to lower risk of ovarian hyperstimulation compared to higher doses 2
Metformin role in fertility:
- Improves ovulation frequency and appears safe during pregnancy 3
- Effects on early pregnancy outcomes not fully established 3
- Can be used as adjunct to clomiphene 3
Preconception and Pregnancy Management
Before attempting pregnancy:
- Screen for metabolic abnormalities: fasting glucose, 2-hour glucose tolerance test, and lipid profile 3
- Provide preconception counseling about increased pregnancy risks 3
During pregnancy:
- Closer monitoring throughout pregnancy with regular assessment of blood pressure, kidney function, and proteinuria 3
- Prescribe low-dose aspirin from week 12 to week 36 to reduce preeclampsia risk 3
Long-Term Metabolic Screening and Monitoring
Regular screening for metabolic complications is essential regardless of weight or symptom severity 2
- Screen for type 2 diabetes mellitus and cardiovascular disease risk factors 2
- Calculate BMI and waist-hip ratio to assess metabolic risk 2
- Monitor fasting glucose and lipid profiles 2
- Assess mental health regularly for depression, anxiety, and eating disorders 2
Critical Pitfalls to Avoid
- Do not neglect metabolic screening in normal-weight PCOS patients - insulin resistance occurs regardless of BMI 1, 2
- Do not exceed recommended clomiphene dosage and duration - risk of ovarian hyperstimulation syndrome increases, especially in PCOS patients 2, 6
- Do not skip pelvic examination before each clomiphene course - must rule out ovarian cysts before treatment 6
- Do not use clomiphene in patients with ovarian enlargement except those with polycystic ovary syndrome 6
- Do not delay lifestyle intervention - it must be first-line, not an afterthought 1, 3
- Watch for visual symptoms with clomiphene - discontinue immediately and perform complete ophthalmological evaluation if visual changes occur 6