What is the treatment for PCOS (Polycystic Ovary Syndrome)?

Treatment of Polycystic Ovary Syndrome (PCOS)

The treatment of PCOS should focus on lifestyle modifications as the cornerstone therapy, with pharmacological interventions tailored to specific symptoms and reproductive goals, including metformin for metabolic manifestations, combined oral contraceptives for menstrual irregularities and hirsutism in non-pregnancy-seeking women, and clomiphene citrate as first-line for ovulation induction in those seeking pregnancy. 1, 2, 3

First-Line Treatment: Lifestyle Modifications

• Weight loss of as little as 5% of initial body weight significantly improves both metabolic and reproductive abnormalities in PCOS, including ovulation rates and pregnancy outcomes 2, 1
• A recommended energy deficit of 30% or 500-750 kcal/day combined with regular physical activity should be implemented for optimal results 1, 4
• Lifestyle modifications should be used alongside medical treatments for best outcomes, particularly for insulin resistance and hyperandrogenism 5, 4

Pharmacological Management Based on Reproductive Goals

For Women Not Seeking Pregnancy:

• Combined oral contraceptives (COCPs) are the first-line pharmacological treatment for menstrual irregularities and hyperandrogenism 1, 3
• COCPs suppress ovarian androgen secretion and increase sex hormone binding globulin, though they may increase triglyceride and HDL cholesterol levels 2, 1
• Metformin is indicated for most women with PCOS due to its positive effects on insulin resistance, menstrual irregularities, and hirsutism 5, 2
• For hirsutism treatment, a combination approach is often needed:
  • Antiandrogens (like spironolactone) with ovarian suppression agents 6
  • Topical eflornithine can be used as an adjunct therapy 5
  • Laser hair removal may be effective but requires multiple treatments and doesn't address the underlying hormonal cause 6

For Women Seeking Pregnancy:

• Clomiphene citrate is the first-line medication for ovulation induction, with approximately 80% of patients ovulating and half conceiving 2, 3
• If clomiphene treatment fails, low-dose gonadotropin therapy is recommended due to lower risk of ovarian hyperstimulation 2, 1
• Metformin may be used alone or in combination with clomiphene for ovulation induction 5, 3
• While metformin appears safe in pregnancy, its effects on early pregnancy outcomes are not fully established 2, 7

Management of Metabolic Aspects

• Regular screening for metabolic abnormalities is essential, including fasting glucose and lipid profile 1, 3
• Metformin is the first-line medication for metabolic manifestations such as hyperglycemia and insulin resistance 3, 8
• GLP-1 receptor agonists (like liraglutide and semaglutide) show promise for weight management in PCOS, though more high-quality studies are needed 2
• The 3-mg dose of liraglutide has been shown to optimize weight loss, though many studies used suboptimal doses 2
• Orlistat may benefit weight reduction and improve HOMA-IR, insulin, and testosterone levels, but has high adverse effects 2

Long-term Monitoring and Complications

• Regular screening for type 2 diabetes mellitus and cardiovascular disease is necessary due to increased risk in PCOS patients 1, 8
• Mental health assessment should be included in monitoring, as women with PCOS have higher rates of depression, anxiety, and eating disorders 1
• Avoid exceeding recommended clomiphene dosage and duration to prevent complications 1
• Don't neglect metabolic screening even in normal-weight PCOS patients 1, 3

Emerging Treatments

• Semaglutide is the most potent long-acting GLP-1 receptor agonist but has only been studied in one small pilot study in PCOS patients 2
• The FDA has approved tirzepatide, a dual-acting GLP-1 receptor agonist with gastric inhibitory polypeptide receptor activator, which may offer future treatment options 2
• More high-quality, multicenter studies of newer agents in PCOS are urgently needed, incorporating reproductive, metabolic, and psychological outcomes 2