January 13, 2025 - Moti Gorin; Jilles Smids; John Lantos
The US Supreme Court soon will decide the constitutionality of Tennessee’s ban on medicalized gender transition for youth. That nearly half US states have banned hormonal and surgical interventions for the treatment of gender dysphoria in patients younger than 18 years while these interventions are endorsed by most US medical societies indicates how much turmoil exists regarding pediatric gender medicine. Many European countries also are questioning the wisdom of these interventions and some have shifted toward a less medicalized approach. These changes reflect the views of leaders in medicine and health policy rather than those of legislators or judges.
The contrast between US and European physicians is clear in their policy statements. The lead author of the 2018 policy statement of the American Academy of Pediatrics (AAP),1 Jason Rafferty, has explained that children themselves should judge whether treatment should be given. “[T]he child’s sense of reality and feeling of who they are is the navigational beacon to sort of orient treatment around,” he said.2 By contrast, Sweden’s National Board of Health and Welfare concluded that “the risks of puberty blockers and gender-affirming treatment are likely to outweigh the expected benefits of these treatments.”3 The board recommends that children with gender dysphoria be carefully evaluated and compassionately treated but that hormonal interventions be limited to “the context of research” or “exceptional cases” only.3
Both groups claim to be following current evidence. In the US, physicians interpret that evidence as showing that gender-affirming care is safe and effective. Health authorities in Sweden, Finland, and the UK conducted systematic reviews of the scientific evidence underpinning pediatric gender medicine.4 All have concluded the evidence base is weak. Because of this, they now recommend that first-line treatment for gender dysphoria in youth should be holistic psychosocial support.4 In the UK, puberty blockers are no longer prescribed to youth for gender dysphoria outside clinical trials. Meanwhile, parents and other caregivers, including many pediatricians, are left disoriented by heated public debate and competing claims among experts.
The focus in Europe is on the fact that many studies of hormonal or surgical interventions were poorly designed. There are no prospective randomized trials. Often, eligibility criteria for participation in the studies are not clearly stated. Measured outcomes often are not comparable. Then, as a National Health Service–commissioned systematic review of clinical guidelines for transgender health revealed, such studies are cited, uncritically, by one professional society after another. For example, the latest guidelines from the World Professional Association for Transgender Health (WPATH) cited Endocrine Society guidelines, which in turn cited earlier WPATH guidelines, which were themselves based on poorly designed studies. “The circularity of this approach,” wrote Hilary Cass, OBE, author of the Cass Review, “may explain why there has been an apparent consensus on key areas of practice despite the evidence being poor.” Reflecting the conclusion of guideline reviewers, the Cass review noted that WPATH and Endocrine Society guidelines, on which US clinicians heavily rely, score poorly on developmental rigor (p 130). Only 2 guidelines—those of Sweden and Finland—were recommended by the majority of guideline appraisers for clinical use. The final Cass review has become the basis for health policy in the UK, though there have been some critiques. References can be found in the most widely noted critique, a white paper5 that has itself been criticized in a recent peer-reviewed article.6
The different perspectives of European and US health authorities suggest there is deep disagreement about risks and benefits. Differences between health care systems and culture also may lead to discrepancies in how policymakers balance ethical considerations such as the values of autonomy and patient welfare. All authorities cite clinical research but different studies are given different weight. The most controversial disagreement is about whether medical transition is “lifesaving.” Some studies conclude that it reduces thoughts of self-harm or suicide7 while others find no effect on suicide mortality.8 Similar debates surround claims about persistence, desistance, and detransition.
In one sense, this kind of debate is not unique. Physicians often disagree about how best to treat health problems. Usually, the debates are resolved through further research and intraprofessional discussion. In gender medicine, however, there is another crucial element. Pediatric medical transition is caught up in the intense polarization that characterizes US politics. Rather than civil discussion, we get vigorous and sometimes toxic advocacy for either the status quo or legal bans. This threatens the usual sorts of consensus building. Consequently, children and their families find themselves caught up in an intense culture war. This is dangerous for distressed young people who need knowledgeable physicians to provide evidence-based recommendations. US health experts must tune out the political noise and renew their focus on scientific, clinical, and ethical considerations.
Disagreements about the interpretation of research lead to different assessments of the risks and benefits of current treatments. Differing assessments then lead to conflicting conclusions about physicians’ ethical obligations. A favorable risk-benefit ratio is the ethical basis for any medical intervention. There is general agreement that hormonal and surgical treatments have risks. Medical transition may result in infertility and sexual dysfunction. Anesthesia and surgery have known risks of complications and death. Puberty blockers may have detrimental effects on cognitive function.9 The anticipated benefits from treatment are psychological—reductions in gender dysphoria, depression, anxiety, and suicidality. These risks and benefits are difficult to compare but efforts to do so should at least follow the methodology generally used for outcome studies. Moreover, there are no rigorous, well-designed studies showing how many US patients present to gender clinics, how these patients are evaluated, or how decisions about treatment are made. Disturbingly, some of the best data come from insurance companies rather than clinician-scientists. Insurance data suggest most US youth who receive a diagnosis of gender dysphoria are not treated with blockers, hormones, or surgery.10 We have no idea how they are faring because no studies have looked. The natural history of gender dysphoria in youth is poorly understood, as are the comparative success rates associated with different approaches.
The lack of good data, especially in the US, reflects deep professional inertia. For example, in 2023, the AAP announced it would conduct a systematic review of the evidence on which its 2018 policy statement was based. That review should have been done before the statement was issued, not after. Now, it is too little, too late. Vulnerable patients, their families, and their physicians deserve better.
Given this state of knowledge, it is ethically problematic to view the routine use of hormonal or surgical interventions in youth with gender dysphoria as evidence-based. We need high-quality studies to better understand the risks and benefits of various interventions. In the meantime, pediatricians should inform parents of disagreements within the field. Parents need to know how current practices in the US diverge from those in Europe. They need to know that systematic reviews of the clinical research find many flaws. Clinicians should also be aware that the Endocrine Society and WPATH guidelines as well as the AAP’s policy statement differ substantially from the approach adopted in the UK and a growing number of European countries.
There are widely used models for collaborative evaluation of innovative therapies for children. In pediatric oncology and other fields, clinicians work together to maintain national collaborative efforts in which data collection and analyses are standardized. A similar approach should be attempted for pediatric gender medicine, one that will allow the development of research protocols to address fundamental questions about diagnosis, safety, efficacy, and long-term outcomes. This may be more complicated than in other fields, especially in the current political climate. But, that is no reason not to try. After all, the problems will not go away or get any easier. As US physicians and medical societies have learned over the past several years, if the medical profession does not have a closer look at this field of medicine, lawmakers and judges will continue to determine policy on their own. The time for health authorities to get serious is now.
References
1.
Rafferty J, Yogman M, Baum R, et al; Committee on Psychosocial Aspects of Child and Family Health; Committee on Adolescence; Section on Lesbian, Gay, Bisexual, and Transgender Health and Wellness. Ensuring comprehensive care and support for transgender and gender-diverse children and adolescents. Pediatrics. 2018;142(4):e20182162. doi:10.1542/peds.2018-2162PubMedGoogle ScholarCrossref
2.
Block J. Youth gender medicine has become a hall of mirrors. The Boston Globe. November 7, 2023. Accessed December 14, 2024. https://www.bostonglobe.com/2023/11/07/opinion/gender-affirming-care-trans-kids/
3.
Swedish National Board of Health and Welfare. Care of children and adolescents with gender dysphoria: summary of national guidelines. Published December 2022. Accessed December 14, 2024. https://www.socialstyrelsen.se/globalassets/sharepoint-dokument/artikelkatalog/kunskapsstod/2023-1-8330.pdf
4.
Kozlowska K, Ambler GR, Dechêne G, et al. Evolving national guidelines for the treatment of children and adolescents with gender dysphoria: international perspectives. Human Systems. Published online November 2, 2024. doi:10.1177/26344041241269298Google ScholarCrossref
5.
McNamara M, Baker K, Connelly K. An evidence-based critique of the Cass Review on gender-affirming care for adolescent gender dysphoria. Yale Law School. Accessed January 2, 2024. https://law.yale.edu/sites/default/files/documents/integrity-project_cass-response.pdf
6.
Cheung CR, Abbruzzese E, Lockhart E, Maconochie IK, Kingdon CC. Gender medicine and the Cass Review: why medicine and the law make poor bedfellows. Arch Dis Child. Published October 15, 2024. doi:10.1136/archdischild-2024-327994Google ScholarCrossref
7.
Tordoff DM, Wanta JW, Collin A, Stepney C, Inwards-Breland DJ, Ahrens K. Mental health outcomes in transgender and nonbinary youths receiving gender-affirming care. JAMA Netw Open. 2022;5(2):e220978. doi:10.1001/jamanetworkopen.2022.0978
ArticlePubMedGoogle ScholarCrossref
8.
Ruuska SM, Tuisku K, Holttinen T, Kaltiala R. All-cause and suicide mortalities among adolescents and young adults who contacted specialised gender identity services in Finland in 1996-2019: a register study. BMJ Ment Health. 2024;27(1):e300940. doi:10.1136/bmjment-2023-300940PubMedGoogle ScholarCrossref
9.
Baxendale S. The impact of suppressing puberty on neuropsychological function: a review. Acta Paediatr. 2024;113(6):1156-1167. doi:10.1111/apa.17150PubMedGoogle ScholarCrossref
10.
Respaut R, Terhune C. Putting numbers on the rise in children seeking gender care. Reuters Investigates. Published October 6, 2022. Accessed December 14, 2024. https://www.reuters.com/investigates/special-report/usa-transyouth-data/
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