Some professionals disagree on the treatment for gender dysphoria in young people

More children and adolescents are identifying as transgender and offered medical treatment, especially in the US. But some providers and European authorities are urging caution because of a lack of strong evidence.

In a new report from The BMJ Investigations Unit, Jennifer Block, investigations reporter, looks into the evidence base behind this surge in treatment.

More adolescents with no history of gender dysphoria are presenting at gender clinics. For example, a recent analysis of insurance claims found that nearly 18,000 US minors began taking puberty blockers or hormones from 2017 to 2021, the number rising each year.

Meanwhile, the number of US private clinics focused on providing hormones and surgeries have grown from just a few a decade ago to more than 100 today.

American medical professional groups are aligned in support of "gender affirming care" for gender dysphoria, which may include hormone treatment to suppress puberty and promote secondary sex characteristics, and surgical removal or augmentation of breasts, genitals, and other physical features.

Three organizations in particular have had a major role in shaping the US approach to gender dysphoria care: The World Professional Association for Transgender Health (WPATH), the American Academy of Pediatrics, and the Endocrine Society, all of which have guidelines or policies that support early medical treatment for gender dysphoria in young people.

These endorsements are often cited to suggest that medical treatment is both uncontroversial and backed by rigorous science, but governing bodies around the world have come to different conclusions regarding the safety and efficacy of certain treatments, notes Block.

For example, Sweden's National Board of Health and Welfare, which sets guidelines for care, determined earlier this year that the risks of puberty blockers and treatment with hormones "currently outweigh the possible benefits" for minors.

And NHS England, which is in the midst of an independent review of gender identity services, recently stated that there is "scarce and inconclusive evidence to support clinical decision-making" for minors with gender dysphoria, and that for most who present before puberty it will be a "transient phase," requiring clinicians to focus on psychological support and to be "mindful" of the risks of even social transition

Experts are also questioning the evidence underpinning these guidelines.

Professor Mark Helfand at Oregon Health and Science University identified several deficiencies in WPATH's recommendations, such as lack of a grading system to indicate the quality of the evidence, while Professor Gordon Guyatt at McMaster University found "serious problems" with the Endocrine Society guidelines, including pairing strong recommendations with weak evidence.

Helfand explains that calling a recommendation 'evidence-based' should mean a treatment has not just been systematically studied, but that there was also a finding of high quality evidence supporting its use.

Despite these concerns, WPATH recommends that youth have access to treatments following comprehensive assessment, stating "the emerging evidence base indicates a general improvement in the lives of transgender adolescents."

Eli Coleman, lead author of WPATH's Standards of Care and former director of the Institute for Sexual and Gender Health at the University of Minnesota, told The BMJ that WPATH's new guidelines emphasise "careful assessment prior to any of these interventions" by clinicians who have appropriate training and competency to assure that minors have "the emotional and cognitive maturity to understand the risks and benefits."

But without an objective diagnostic test, others remain concerned, pointing to examples of teenagers being "fast-tracked to medical intervention" with little or no mental health involvement.

And in her interim report of a national review into services for young people with gender identity issues, Hilary Cass noted that some NHS staff reported feeling "under pressure to adopt an unquestioning affirmative approach and that this is at odds with the standard process of clinical assessment and diagnosis that they have been trained to undertake in all other clinical encounters."

For Guyatt, claims of certainty represent both the success and failure of the evidence-based medicine movement. "When there's been a rigorous systematic review of the evidence and the bottom line is 'we don't know,'" he says, then "anybody who then claims they do know is not being evidence based."