The UK is preparing to launch a clinical trial to investigate the use of puberty blockers in young people experiencing gender dysphoria, a move aimed at resolving the ongoing debate about the drugs' efficacy and safety. With funding expected to be confirmed soon by the National Institute for Health and Care Research, the trial seeks to provide much-needed evidence in a field marked by controversy and uncertainty. The central question is whether the benefits of puberty blockers outweigh the potential harms for adolescents questioning their gender identity.
Background and Context
The debate around puberty blockers intensified after NHS England restricted their routine prescription to under-18s, citing insufficient evidence of safety and clinical effectiveness. This decision followed a significant increase in referrals to gender identity services, particularly among young people registered female at birth. Dr. Hilary Cass's review of gender identity services further highlighted the lack of a cautious and careful approach in the field, criticizing the reliance on limited studies with inadequate long-term follow-up.
Ethical Considerations
One of the primary challenges in designing the trial is addressing ethical dilemmas. Some argue that withholding puberty blockers from young people experiencing gender distress would be unethical, given the belief that these drugs can improve mental health. The World Professional Association of Transgender Health (WPATH) has voiced concerns about making trial participation the only avenue to access care they consider medically necessary and potentially lifesaving.
Conversely, other clinicians question the evidence supporting the mental health benefits of puberty blockers and point to potential negative impacts on brain development and bone density. Dr. Louise Irvine, co-chair of the Clinical Advisory Network on Sex and Gender, emphasizes the need for tangible and significant benefits to justify disrupting a crucial phase of human development.
Trial Design and Potential Approaches
Several trial designs are under consideration to rigorously assess the effects of puberty blockers. One approach involves randomly assigning participants to either receive puberty blockers or a placebo. However, concerns have been raised about the feasibility of a placebo group, as participants may realize they are not receiving the drug and drop out of the trial.
An alternative design proposes comparing young people receiving puberty blockers to those undergoing social transition, such as changing their name, pronouns, and dress. Another option involves delaying the administration of puberty blockers in one group while providing psychological and emotional support, allowing researchers to assess whether gender-related distress subsides with support alone.
Researchers also consider exploring the optimal timing for administering puberty blockers by giving drugs at different stages of puberty to different groups. This would help determine if starting the drugs earlier improves wellbeing by reducing gender-specific body changes, or if it has a greater negative impact on bone density and brain development.
Measuring Outcomes
The trial aims to measure various outcomes to determine whether the intervention improves the participants' quality of life. This includes assessing mental health, self-esteem, self-confidence, time spent in education, and time spent with family and friends. Potential harms, such as reduced bone density and impacts on learning, will also be studied.
Brain scans may be used to understand the impact of puberty suppression on normal brain development, which is influenced by both puberty and chronological age. Participants will continue to receive treatment for other conditions, such as anxiety, depression, eating disorders, and autism, to ensure that any differences in results are attributable to the drug.
The Path Forward
Recruitment for the trial is expected to begin in 2025. The results of this trial are eagerly awaited by scientists, healthcare professionals, parents, and young people around the world, as they could significantly influence the future of gender-affirming care for adolescents.
