Which way to go?
Confident treatment advice versus weak evidence
There are “serious problems” with a treatment guideline used to justify medicalised gender change for minors, according to a pioneer of the evidence-based medicine movement.
Professor Gordon Guyatt told the journal BMJ that systematic reviews of the evidence carried out for the Endocrine Society’s 2017 treatment guideline had failed to take into account the effect of transgender hormonal interventions on gender dysphoria, arguably “the most important outcome.”
“He also noted that the Endocrine Society had at times paired strong recommendations — phrased as ‘we recommend’ — with weak evidence,” BMJ investigations reporter Jennifer Block said in a new feature article published today.
Dr Guyatt is distinguished professor in the Department of Health Research Methods, Evidence and Impact at Canada’s McMaster University, where he was one of the founders of the evidence-based medicine movement in the 1990s, with an emphasis on developing the criteria necessary for a “trustworthy guideline”.
Video: A new documentary critiques the affirmative model
No more cherry picking of data
A recent paper, The Myth of ‘Reliable Research’ in Pediatric Gender Medicine, explained the rationale for systematic reviews of the literature:
“Rather than arbitrarily selecting studies and simply restating their results and conclusions, systematic reviews of evidence analyse all of the available evidence meeting pre-specified criteria and scrutinise the studies for methodological bias and errors, issuing an overarching conclusion about what’s known about the effects of an intervention based on the totality of the evidence.”
The paper says that when Dutch clinicians began to pioneer medical transition with adolescents in the late 1980s to early 1990s, “it was not uncommon for medical professionals to practise medicine based on ‘empirical evidence,’ relying on expert opinion and often backed by only minimal research.”
“[But] the former era of eminence-based, expert-opinion-led medicine, under which the innovative clinical practice of paediatric gender transition proliferated, has been replaced by a new standard, evidence-based medicine, which demands rigour in the research that underpins population-level treatment recommendations.”
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Weak and uncertain
Since 2020, amid increasing international debate about invasive treatments for gender dysphoria, systematic reviews undertaken in Finland, Sweden, the United Kingdom and the American state of Florida have found the evidence for paediatric gender change to be weak and uncertain.
However, especially in the United States, confident treatment guidelines and policy statements from health professional societies — eminence-based medicine — have been invoked to claim a medical consensus in favour of these “gender-affirming” hormonal and surgical interventions with minors.
“These documents are often cited to suggest that medical treatment is both uncontroversial and backed by rigorous science,” Block said in the BMJ.
She noted the assertion by a 2022 article in Scientific American that, “All of those medical societies find [gender-affirming] care to be evidence-based and medically necessary.”
U.S. assistant secretary for health Dr Rachel Levine, a paediatrician and trans woman, claimed last year:
“There is no argument among medical professionals — paediatricians, paediatric endocrinologists, adolescent medicine physicians, adolescent psychiatrists, psychologists, etc. — about the value and the importance of gender-affirming care.”
Guidelines and policy statements endorsing the gender-affirming treatment approach are coming in for closer scrutiny, including by the U.K. independent review of youth dysphoria care under the leadership of paediatrician Dr Hilary Cass.
The BMJ investigation also targets the updated treatment guideline issued last year by the World Professional Association for Transgender Health (WPATH).
This guideline failed to give a clear account of the systematic reviews of evidence it had commissioned, according to Mark Helfand, professor of medical informatics and clinical epidemiology at Oregon Health and Science University.
Professor Helfand also highlighted instances where the strength of evidence presented by WPATH to justify a treatment recommendation was “at odds with what their own systematic reviewers found.”
“[Weak evidence] doesn’t just mean something esoteric about study design, it means there’s uncertainty about whether the long-term benefits outweigh the harms,” Helfand told the BMJ.
Professor Guyatt challenged WPATH’s claim that limited evidence made it impossible to carry out a systematic review of outcome data for adolescents.
He said “systematic reviews are always possible” — even if few or no studies met the eligibility criteria — and treatment recommendations made without a systematic review fell short of standards for “trustworthy guidelines.”
WPATH acknowledged that its guideline partly relied on “consensus-based expert opinion,” which ranks low in the quality hierarchy of evidence.
Block reported Professor Helfand’s view that, “In the absence of high-quality evidence and the presence of a patient population in need — who are willing to take on more personal risk — consensus-based guidelines are not unwarranted … ‘But don’t call them evidence-based’.”
The academy has blocked attempts by concerned paediatricians to mobilise the membership in favour of a systematic review as the foundation for a new, more rigorous policy.
The 2018 statement was subjected to a highly critical “fact check” by clinical psychologist and researcher Dr James M. Cantor, who concluded that the academy had misrepresented the state of the evidence.
The Cantor critique remains unanswered but today’s BMJ article quotes the lead author of the academy’s statement, Dr Jason Rafferty, assistant professor of paediatrics and psychiatry at Brown University, as saying that the process leading up to the 2018 policy “doesn’t quite fit the definition of systematic review, but it is very comprehensive.”
“What our policy statement is not meant to be is a protocol or guidelines in and of themselves,” he said.
The BMJ also noted that a prominent U.S. gender clinician and researcher, Dr Robert Garofalo, chief of adolescent medicine at the Lurie Children’s Hospital in Chicago, had acknowledged last year that the evidence base represented “a challenge … it is a discipline where the evidence base is now being assembled [and] it’s truly lagging behind [clinical practice], I think, in some ways.”
Dr Garofalo was a “contributor” to the American Academy of Pediatrics’ 2018 policy statement.
U.S. paediatrician Dr Sarah Palmer, one of the academy’s members seeking a careful review of the 2018 policy, said the term “gender-affirming care” was being broadly defined by clinicians “to mean go ahead and do anything that affirms.”
“One of the main things I’ve seen it used for is masculinising chest surgery, also known as mastectomy, in teenage patients,” she told the BMJ.
“I’ve seen a quick evolution, from kids with a very rare case of gender dysphoria who were treated with a long course of counselling and exploration before hormones were started, [to treatment progressing] very quickly—even at the first visit to gender clinic— and there’s no psychologist involved anymore.”