Should expert opinion be included in PCOS guidelines when evidence is limited?
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Clinicians and patients deserve expert recommendations based on what we know right now.
The most recent international polycystic ovary syndrome guideline, published in 2018, was a monumental effort that addressed metabolic, reproductive and psychological well-being issues related to PCOS, based on a synthesis of the available evidence. Was the evidence base perfect? Far from it. However, the guideline authors did the best they could, based on available evidence, to provide guidance to clinicians, health care professionals and patients, raising awareness of PCOS encouraging shared decision-making and communication about diagnosis, assessment and management. The authors graded the evidence, identified gaps in knowledge and advocated for additional research to fill the gaps. Regarding diagnostic criteria, the authors clarified the factors to consider, highlighted the phenotypic diversity and emphasized that abnormal findings on ultrasound were not essential. Guidance regarding management was also provided.
PCOS is a prevalent condition affecting approximately 10% of women globally, and is understudied and poorly understood. However, the 2018 PCOS guideline raised the profile of this condition among clinicians, many of whom will benefit from guidance on how to diagnose, manage and treat it. Although working with imperfect and incomplete data, the guideline authors did their best during the course over a lengthy period to synthesize and interpret the available evidence.
Clinicians, patients and other stakeholders deserve expert recommendations based on the best available evidence. The knowledge may be limited, but it still has value. Millions of women with PCOS are dealing with these issues right now, in the present, and their needs can’t be deferred until a perfect evidence base is in place. Guidelines will change as more evidence becomes available. Some recommendations might even be overturned — that is common in medicine. Some recommendations will stand the test of time. We need to do the best we can with the tools and resources available to us.
JoAnn E. Manson, MD, DrPH, FACE, is chief of the division of preventive medicine at Brigham and Women’s Hospital and professor of medicine and the Michael and Lee Bell Professor of Women’s Health at Harvard Medical School.
Expert-based PCOS recommendations can muddy the waters.
You will often hear a health care provider say, “Well, it’s in the guideline.” Many times, people do not differentiate between the levels of evidence. Then something becomes the standard of care without any evidence. That said, the practice of medicine is always a mixture of the art and the evidence. The fact the we have 35 evidence-based recommendations for PCOS is, to me, remarkable. Thirty years ago, there were zero.
More evidence is needed. The largest study that needs to be done should characterize the cardiovascular event risk for women with PCOS. Is their risk less than for other women without PCOS, which some experts surmise? If so, let’s find out why. If their risk is greater, then we need to intervene. That’s the No. 1 issue and addresses what we should primarily be treating in PCOS. For the woman with PCOS who is not seeking fertility — which is most women for most of their lives — the big question is, what is the best chronic therapy that controls the symptoms of PCOS and at the same time prevents the most dreaded sequelae? From a public health standpoint, those sequelae are endometrial cancer and type 2 diabetes.
Why don’t we have these answers? The sequalae of PCOS develop over time and among older women. Federally funded research typically has 5-year windows, and it is difficult to see those hard endpoints develop in a 5-year window among young women. We tend to look at surrogate outcomes that may or may not correlate with events down the road. Although very common, PCOS is an orphan disorder. The Eunice Kennedy Shriver National Institute of Child Health and Human Development and National Institute of Diabetes and Digestive and Kidney Diseases largely view PCOS as an infertility disorder. The National Cancer Institute views endometrial cancer as very treatable and easily diagnosed. No one wants to claim “ownership” of PCOS, other than reproductive or medical endocrinologists.
Most people view expert opinion as better than having no opinion. In the absence of a recommendation, people are more likely to unintentionally do something that holds potential for harm.
When it comes to guidelines, a lot of it is about provider education. Societies that put out these guidelines often grade their recommendations. People must pay attention to that and acknowledge it.
Richard S. Legro, MD, FACOG, is a physician and professor of obstetrics and gynecology and public health services at Penn State College of Medicine, Milton S. Hershey Medical Center in Hershey, Pennsylvania.
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