From the Guidelines
The most effective treatment for Polycystic Ovary Syndrome (PCOS) is a multi-faceted approach that includes lifestyle modifications, such as weight loss and regular exercise, as well as targeted medications tailored to specific symptoms, as supported by the latest evidence from 2024 1.
Key Components of Treatment
- Weight loss of 5-10% through caloric restriction and regular exercise (at least 150 minutes weekly of moderate activity) to improve insulin sensitivity and restore ovulation
- Combined hormonal contraceptives containing ethinyl estradiol with progestins like drospirenone or cyproterone acetate to regulate menstrual cycles and reduce androgen effects
- Metformin (starting at 500mg daily, increasing to 1500-2000mg daily in divided doses) for metabolic symptoms, particularly in women with insulin resistance or diabetes risk
- Letrozole (2.5-7.5mg daily for 5 days) as the preferred ovulation induction agent for those seeking pregnancy, due to higher live birth rates compared to clomiphene
- Spironolactone (50-200mg daily) or finasteride (2.5-5mg daily) for hirsutism, with contraception required due to teratogenicity
- Inositol supplements (myo-inositol 2-4g daily) showing promise for improving ovulation and metabolic parameters
Consideration of Bariatric Surgery
- Bariatric surgery is emerging as a potential adjunct therapy for PCOS in patients with excess weight, with evidence from 2024 suggesting improvements in anthropometric, reproductive, hormonal, and metabolic outcomes 1
- However, the efficacy and safety of bariatric surgery compared to existing strategies for weight reduction in PCOS require further study
Prioritization of Lifestyle Management
- Lifestyle management is the first-line management in the intervention hierarchy in PCOS, with a focus on weight and healthy lifestyle behaviors, as emphasized in the international evidence-based guidelines for the assessment and management of PCOS 2018 1
- Multicomponent lifestyle intervention, including diet, exercise, and behavioral strategies, is central to PCOS management, with evidence supporting its benefits for reproductive, metabolic, and anthropometric outcomes 1
From the FDA Drug Label
Clomiphene citrate is indicated for the treatment of ovulatory dysfunction in women desiring pregnancy. Impediments to achieving pregnancy must be excluded or adequately treated before beginning clomiphene citrate therapy Those patients most likely to achieve success with clomiphene therapy include patients with polycystic ovary syndrome (see WARNINGS: Ovarian Hyperstimulation Syndrome)
The latest most evidence-based peer-reviewed treatment for PCOS (Polycystic Ovary Syndrome) includes the use of clomiphene citrate for ovulatory dysfunction.
- Key points to consider when using clomiphene citrate for PCOS:
- Patients should have adequate levels of endogenous estrogen.
- Clomiphene citrate therapy cannot be expected to substitute for specific treatment of other causes of ovulatory failure.
- The lowest dose consistent with expected clinical results should be used to minimize the hazard associated with occasional abnormal ovarian enlargement.
- Patients with PCOS should be started on the lowest recommended dose and shortest treatment duration for the first course of therapy 2.
- Important considerations:
- Ovarian hyperstimulation syndrome (OHSS) has been reported to occur in patients receiving clomiphene citrate therapy for ovulation induction.
- Early warning signs of OHSS include abdominal pain and distention, nausea, vomiting, diarrhea, and weight gain 2.
From the Research
Treatment Options for PCOS
- The current treatment options for Polycystic Ovary Syndrome (PCOS) include metformin, lifestyle interventions, and oral contraceptive pills (OCPs) 3, 4, 5.
- Metformin has been shown to improve insulin resistance, reduce androgen production, and improve ovulatory rates in women with PCOS 3.
- Lifestyle interventions, such as dietary changes and physical activity, have been found to improve reproductive, anthropometric, metabolic, and psychological outcomes in women with PCOS 4.
Comparison of Treatment Options
- A systematic review found that metformin may be less effective in improving hirsutism compared to OCPs in women with PCOS, particularly in those with a body mass index (BMI) between 25 and 30 kg/m2 6.
- The review also found that metformin may increase the incidence of severe gastrointestinal adverse events compared to OCPs, but may decrease the incidence of severe other adverse events 6.
- Another review found that metformin may improve live birth rates compared to placebo, but may also increase the risk of gastrointestinal side effects 7.
Recommendations
- The Endocrine Society Guidelines recommend the use of metformin during adolescence, particularly in the presence of excess body weight 3.
- Lifestyle modification is considered the first-line treatment for PCOS, regardless of fertility status, without the addition of metformin 5.
- OCPs should be used as a first-line treatment for long-term management in patients with no reproductive requirements 5.
- For patients with fertility requirements, ovulation therapy is an effective treatment, and metformin may be used in combination with clomiphene citrate or letrozole to improve reproductive outcomes 5, 7.