Most common medications for people with PCOS

Polycystic ovarian syndrome (PCOS) affects up to 2 out of 10 people with ovaries. The “syndrome” in PCOS means that it is a group of symptoms (1). These symptoms can vary widely from person to person. Some people have no symptoms at all while others might see hirsutism (male-pattern hair growth, usually on the chin and face), hair loss, acne, irregular or missed periods, infertility, and metabolic changes like unwanted weight gain and insulin resistance (2).

While there’s no cure for PCOS, there are actually quite a few options for management. Lifestyle changes like diet and exercise are considered the first step to managing PCOS symptoms but sometimes, these changes aren’t enough (3). In those cases, your healthcare provider may want to discuss additional options with you. Below, you’ll find a list of the most common and effective medications used for symptom management.

If you want to dig deeper into the diagnosis, detailed symptoms, and types of PCOS, check out our PCOS Crash Course. 

Medications for menstrual cycle-regulation

One of the hallmarks of managing PCOS is regulating ovulation so that periods return to normal. If you’re trying to get pregnant, it can be nearly impossible to do so if your ovary is not releasing eggs. If you’re not trying to get pregnant, it’s still important to shed your endometrium (uterine lining)  regularly to lower the risk of uterine and other estrogen-related cancers.  

Hormonal birth control is often used to help regulate periods and improve quality of life for people with PCOS. For people with symptoms of acne, oily skin, unwanted hair growth or loss, and irregular periods, birth control pills, patches, and rings that contain estrogen and progestin can decrease androgen production which improves all these symptoms (3). These and other progestin-only birth controls, like hormonal IUDs, also help protect the endometrium against abnormal cell growth. Given the 3x higher risk of estrogen-related cancers like endometrial cancer for people with PCOS, this protective benefit can’t be overlooked (3). 

Lastly, hormonal birth control can oftentimes help improve anxiety and depression symptoms. Mental health counseling and self-care practices are still important to include in the management of these symptoms. 

There are many different hormonal birth controls that exist and each will work differently for one person versus another. This is why it’s so important to find a method that works for you and your symptoms. Common side effects of birth control are usually mild and go away with continued use or switching to another option. These side effects  include unscheduled bleeding (especially in the first 3 months), nausea, bloating and abdominal cramps, headaches and breast tenderness (4). In particular, birth control methods that are either progestin-only or lower in estrogen have been shown to increase androgen levels as a side effect (5). This will likely worsen acne and hair-related symptoms such as male pattern baldness and hirsutism. 

The most serious side effect of hormonal birth control is the potential for a blood clot or stroke.The risk is small but can become an issue for people with significant risk factors like smoking or cardiovascular problems. For most people, these risks are very low. 

One final caveat to keep in mind is that hormonal birth control is not going to “fix” or cure PCOS, it is only masking symptoms. It’s still crucial to focus on healthy lifestyle practices and other interventions. For more on hormonal birth control, check out our post on whether birth control is helpful or harmful for those with PCOS.

Medications for systemic symptoms

Because many symptoms of PCOS overlap in terms of their causes and effects, be aware that the medications below can have effects in multiple areas. For example, medications like metformin can help with metabolic health and ovulatory health because those pathways are intertwined. This is why it is so important to discuss all your options with a healthcare provider  so you can work together to find the best solution for you and your body.

Medications for metabolic health

The metabolic effects of PCOS like insulin resistance are often the biggest problem people with PCOS deal with. Insulin resistance means the body doesn’t respond to insulin as well as it should. This leads to high glucose (sugar) levels in the blood. Over time, the body requires more and more insulin, which can eventually lead to diabetes. 

The high levels of insulin that result from insulin resistance can also disrupt the hormones that your ovaries and pituitary gland (an important endocrine gland in our brain) release, leading to more ovulation dysfunction. Alternatives to hormonal birth control for people trying to manage these metabolic concerns include an over the counter supplement inositol, and the prescription medication, metformin (6, 7). 

Metformin used to be considered a first-line treatment for PCOS because it increases ovulation rates, which helps regulate periods and makes it easier to get pregnant. It does this by improving the metabolic and hormonal changes that lead to insulin resistance in people with PCOS. Metformin can sometimes also help with early weight loss when combined with other lifestyle interventions. 

Side effects of metformin include nausea/vomiting, diarrhea, stomach cramps, and decreased appetite (8). Continued metformin use can lead to vitamin B12 deficiency.  Although very rare, metformin's most serious side effect is lactic acidosis (9). This is caused by dehydration which leads to a buildup of lactic acid and a pH imbalance in the body.

Inositol is a natural sugar that our bodies make. It exists in several forms known as isomers.  Myo-inositol (MI) and D-chiro-inositol (DCI) are the two main types that are used in fertility supplements.This is because MI functions to improve ovulation while DCI reduces insulin levels in the body (10).

Research shows that people with PCOS make less mature eggs than people without it (10). This has been specifically linked to having too little MI and too much DCI in the follicular fluid, which surrounds the eggs as they grow inside the ovaries (11). The MI to DCI ratio in general body tissues of healthy menstruators is 40 to 1 and 100 to 1 in their follicular fluid. In people with PCOS, the average ratio of MI to DCI in this same fluid is 0.2 to 1 (12). The exact dosage of inositol is not yet agreed upon but a 40 to 1 MI to DCI ratio is optimal for restoring ovulation in people with PCOS (13). 

Inositol is generally considered very safe in adults. Side effects, if any, tend to be mild and can include nausea, stomach pain, fatigue, headache, and dizziness (6).

Medications for hair and skin

• Minoxidil: Commonly known as Rogaine, minoxidil is an FDA-approved topical drug used to treat female pattern baldness and hair loss. It’s applied directly on the scalp every day to help to regrow hair. The most common side effects of topical minoxidil are scalp irritation and changes to the color and texture of the hair (14).
• Anti-androgenic medications: These medicines work by blocking the effects of androgens. Some also suppress production of androgens by the ovaries. 

• Spironolactone is a diuretic approved by the FDA to treat retained fluid (15). It is also used to treat PCOS hair loss and acne because it blocks androgens. Studies show that 44–74% of people who took spironolactone saw improvement in their hair loss (14). It may take several months to see significant results (14). Common side effects of spironolactone can include dizziness, nausea, muscle cramps, fatigue, and breast tenderness. 
• Flutamide is another drug that has shown promise for hair loss (16). This drug is used much less often than spironolactone because in rare cases, it may cause severe liver damage (14).
• Finasteride and Dutasteride are two FDA-approved oral medications for hair growth to treat male-pattern baldness and hair loss (16). Studies show that both drugs are effective in improving acne and hair growth in people with hyperandrogenism. However, they are contraindicated in people who are or may become pregnant due to risks to the fetus and in people with a family history of breast cancer (16). 

• Eflornithine:  Eflornithine is a topical cream that can be used to slow down the growth of unwanted facial hair (17). Noticeable improvement usually takes about 4 to 8 weeks (17). It does not remove hair or cure unwanted facial hair, but rather slows growth and makes hair less visible and coarse (18). As soon as it’s stopped, hair will begin to regrow and will return to pre-treatment levels after about 8 weeks. The most common side effect is scalp irritation and folliculitis (18). 
• Hormonal contraceptives: As mentioned earlier, birth control can also be effective in people who have acne. It’s important to be consistent and patient since many of these treatments can take 2 to 3 months before they start to work. Some hormonal birth controls can make acne and hair concerns worse so always discuss with your provider if you want your birth control to do more than prevent pregnancy. 

Medications for fertility

Knowing that there are options for when the time comes to try to get pregnant can go a long way in relieving anxiety for people with PCOS.

Ovulation induction

If lifestyle changes and medications like inositol and/or metformin have already been tried, the next step is usually ovulation induction (OI). These are safe medications that can be prescribed to encourage ovulation. There are two OI agents that are widely used: clomiphene citrate (Clomid) and letrozole (Femara).

Clomid works by blocking the body’s response to estrogen while letrozole suppresses estrogen production (19). The lack of response to estrogen with Clomid and the low levels of estrogen from letrozole prompt the pituitary gland to produce more follicle stimulating hormone (FSH), which does exactly what its name says: stimulates the growth of follicles containing eggs in the ovary. Both medications are generally not used for more than six cycles.

Of the two, clomid used to be first-line, but studies have shown that letrozole has better ovulation, pregnancy, and birth rates so many providers now choose to go straight to Letrozole (20). 

Unfortunately, OI doesn’t always work. In these cases, providers will typically move on to injectable fertility drugs, known as gonadotropins. These are made of the follicle-stimulating hormone (FSH), luteinizing hormone (LH), or some combination of the two (19). 

Gonadotropins can be given alone but are often given as part of a regimen that includes one of the above ovulation induction agents (19). These regimens are typically started with the lowest effective dose to avoid one of the potential risks of gonadotropin use: ovarian hyperstimulation syndrome (OHSS). This can be a very serious complication in which the ovaries overreact to the fertility drugs and become dangerously filled with fluid. This fluid can leak into the belly and chest leading to other problems. People with PCOS are at a higher risk of developing OHSS. Symptoms like rapid weight gain, severe abdominal pain and nausea, shortness of breath, or trouble urinating could be a sign that you’re getting OHSS and should prompt you to call your doctor immediately (21).

Intrauterine insemination

OI is often combined with intrauterine insemination (IUI). IUI is an assisted reproductive technique in which specially washed semen is placed directly into the uterus using a small catheter. This is timed for after you have been given low dose ovulation induction agents to try to produce a couple more eggs per cycle, rather than just the usual one (19). The idea is to increase your chances of pregnancy by placing as much sperm as possible where it needs to be. The semen could be from a sperm donor or your partner. This method is generally done for about three cycles before moving on to IVF.

In vitro fertilization

In vitro fertilization (IVF) is generally well-known nowadays. It involves using injectable fertility drugs to stimulate the ovaries so that they will produce as many mature eggs as (safely) possible. The eggs are retrieved from the ovaries during an in-office procedure known as an egg retrieval (during which you are put to sleep). Those eggs are then combined with sperm in the lab with the hopes that the sperm will fertilize some of the eggs. Fertilization of half those eggs is considered a success. After about 3-5 days of growth, the embryos are either frozen for later use or one of them is transferred into the uterus. This is called an embryo transfer. Two weeks later, a pregnancy test is done to see if the cycle worked (22).

There is obviously a bit more nuance to choosing one or more of these options, especially because every person is different, as is their PCOS and their body. It’s important to remember this when talking to your doctor about which of these methods will be best for you and your specific situation. Try to be open-minded and don’t ever be afraid to ask questions!

‍

References:

1. Ding, T., Hardiman, P. J., Petersen, I., Wang, F. F., Qu, F., & Baio, G. (2017). The prevalence of polycystic ovary syndrome in reproductive-aged women of different ethnicity: a systematic review and meta-analysis. Oncotarget, 8(56), 96351–96358. https://doi.org/10.18632/oncotarget.19180
2. U.S. Department of Health & Human Services. (2021, August 10). Insulin resistance and diabetes. Centers for Disease Control and Prevention. Retrieved August 16, 2022, from https://www.cdc.gov/diabetes/basics/insulin-resistance.html 
3. Teede, H. J., Misso, M. L., Costello, M. F., Dokras, A., Laven, J., Moran, L., Piltonen, T., Norman, R. J., & International PCOS Network (2018). Recommendations from the international evidence-based guideline for the assessment and management of polycystic ovary syndrome. Human reproduction (Oxford, England), 33(9), 1602–1618. https://doi.org/10.1093/humrep/dey256
4. Cooper DB, Patel P, Mahdy H. Oral Contraceptive Pills. [Updated 2022 May 24]. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2022 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK430882/
5. Villines, Z. (2017, November 17). Best birth control pills for PCOS: Options and how they work. Medical News Today. Retrieved August 16, 2022, from https://www.medicalnewstoday.com/articles/320055#how-birth-control-can-affect-pcos 
6. Kalra, B., Kalra, S., & Sharma, J. B. (2016). The inositols and polycystic ovary syndrome. Indian journal of endocrinology and metabolism, 20(5), 720–724. https://doi.org/10.4103/2230-8210.189231
7. Practice Committee of the American Society for Reproductive Medicine. Electronic address: ASRM@asrm.org, & Practice Committee of the American Society for Reproductive Medicine (2017). Role of metformin for ovulation induction in infertile patients with polycystic ovary syndrome (PCOS): a guideline. Fertility and sterility, 108(3), 426–441. https://doi.org/10.1016/j.fertnstert.2017.06.026
8. Crown copyright. (2022, March 24). Side effects of metformin. NHS. Retrieved August 16, 2022, from https://www.nhs.uk/medicines/metformin/side-effects-of-metformin/ 
9. Fitzgerald, E., Mathieu, S., & Ball, A. (2009). Metformin associated lactic acidosis. BMJ, 339–360. https://www.bmj.com/content/339/bmj.b3660 
10. Unfer, V., Facchinetti, F., Orrù, B., Giordani, B., & Nestler, J. (2017). Myo-inositol effects in women with PCOS: a meta-analysis of randomized controlled trials. Endocrine connections, 6(8), 647–658. https://doi.org/10.1530/EC-17-0243
11. Pundir, J., Psaroudakis, D., Savnur, P., Bhide, P., Sabatini, L., Teede, H., Coomarasamy, A., & Thangaratinam, S. (2018). Inositol treatment of anovulation in women with polycystic ovary syndrome: a meta-analysis of randomised trials. BJOG : an international journal of obstetrics and gynaecology, 125(3), 299–308. https://doi.org/10.1111/1471-0528.14754
12. Unfer, V., Carlomagno, G., Papaleo, E. et al. Hyperinsulinemia Alters Myoinositol to D-chiroinositol Ratio in the Follicular Fluid of Patients With PCOS. Reprod. Sci. 21, 854–858 (2014). https://doi.org/10.1177/1933719113518985
13. Nordlo, M., Basciani, S., & Camajani, E. (2019). The 40:1 myo-inositol/D-chiro-inositol plasma ratio is able to restore ovulation in PCOS patients: comparison with other ratios. European Review for Medical and Pharmacological Sciences, 23(12), 5512–5521. https://doi.org/10.26355/eurrev_201906_18223 
14. Carmina, E., Azziz, R., Bergfeld, W., Escobar-Morreale, H. F., Futterweit, W., Huddleston, H., Lobo, R., & Olsen, E. (2019). Female Pattern Hair Loss and Androgen Excess: A Report From the Multidisciplinary Androgen Excess and PCOS Committee. The Journal of clinical endocrinology and metabolism, 104(7), 2875–2891. https://doi.org/10.1210/jc.2018-02548
15. Rathnayake, D., & Sinclair, R. (2010). Innovative use of spironolactone as an antiandrogen in the treatment of female pattern hair loss. Dermatologic clinics, 28(3), 611–618. https://doi.org/10.1016/j.det.2010.03.011
16. Goodman, N. F., Cobin, R. H., Futterweit, W., Glueck, J. S., Legro, R. S., Carmina, E., American Association of Clinical Endocrinologists (AACE), American College of Endocrinology (ACE), & Androgen Excess and PCOS Society (AES) (2015). AMERICAN ASSOCIATION OF CLINICAL ENDOCRINOLOGISTS, AMERICAN COLLEGE OF ENDOCRINOLOGY, AND ANDROGEN EXCESS AND PCOS SOCIETY DISEASE STATE CLINICAL REVIEW: GUIDE TO THE BEST PRACTICES IN THE EVALUATION AND TREATMENT OF POLYCYSTIC OVARY SYNDROME--PART 1. Endocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists, 21(11), 1291–1300. https://doi.org/10.4158/EP15748.DSC
17. Valejo Coelho, M. M., & Carvalho, R. (2019). Use of Eflornithine Hydrochloride Cream to Reduce Facial Hair Growth in Flapped Skin. Dermatologic surgery : official publication for American Society for Dermatologic Surgery [et al.], 45(12), 1717–1718. https://doi.org/10.1097/DSS.0000000000001769
18. Agrawal N. K. (2013). Management of hirsutism. Indian journal of endocrinology and metabolism, 17(Suppl 1), S77–S82. https://doi.org/10.4103/2230-8210.119511
19. Tanbo, T., Mellembakken, J., Bjercke, S., Ring, E., Åbyholm, T., & Fedorcsak, P. (2018). Ovulation induction in polycystic ovary syndrome. Acta obstetricia et gynecologica Scandinavica, 97(10), 1162–1167. https://doi.org/10.1111/aogs.13395
20. Legro, R. S., Brzyski, R. G., Diamond, M. P., Coutifaris, C., Schlaff, W. D., Casson, P., Christman, G. M., Huang, H., Yan, Q., Alvero, R., Haisenleder, D. J., Barnhart, K. T., Bates, G. W., Usadi, R., Lucidi, S., Baker, V., Trussell, J. C., Krawetz, S. A., Snyder, P., Ohl, D., … NICHD Reproductive Medicine Network (2014). Letrozole versus clomiphene for infertility in the polycystic ovary syndrome. The New England journal of medicine, 371(2), 119–129. https://doi.org/10.1056/NEJMoa1313517
21. Kumar, P., Sait, S. F., Sharma, A., & Kumar, M. (2011). Ovarian hyperstimulation syndrome. Journal of human reproductive sciences, 4(2), 70–75. https://doi.org/10.4103/0974-1208.86080
22. Ho, J. (2021, July 8). Patient education: In vitro fertilization (IVF) (Beyond the Basics). UpToDate. Retrieved August 16, 2022, from https://www.uptodate.com/contents/in-vitro-fertilization-ivf-beyond-the-basics 

‍