Puberty blockers study: much ado about nothing?
The BMJ has a great tradition in exposing improper activities and unearthing scandals. So we read the four page article on the puberty blockers study avidly, yet were surprised when no substantive defects in the study or its conduct were revealed. We have no connection to the trial.
The criticisms raised in the article are minor but dressed up as major: this small cohort study correctly followed its protocol, and by definition there was no control group. A randomised trial is all but impossible in these young people who are highly reluctant not to take puberty blockers. The article implies that the study put participants at risk since subsequently most go on to further interventions, but this is confusing the decision by the clinical team to give treatment versus studying how participants fared. Likewise the authors imply that the study didn't warn of possible bone side-effects, yet the young person in the "patient’s perspective" says " the only certainties are….your bones go a bit weird, hence why you may have a few bone density tests…"
The article states the drug was "off label", but in fact 50% of drugs used in paediatrics are "off label", because few are formally tested on children. Many protocols for small cohort studies don't define serious adverse events precisely, and the article concedes that "similar concerns are common to many studies of puberty blockers". Failing to submit an annual progress report to a Research Ethics Committee (REC) is common. Submission is not a regulatory requirement and this does not indicate the study was unethical.
The authors seem to prejudge the study’s results. They imply that the researcher who said the interim results were positive was misleading, yet report that all the young people wished to continue puberty blockers. Some young people reported Self Harm (SH) ideation and acts, but contrary to what is suggested, we simply do not know whether this was related to the treatment - rates of SH increase dramatically between 12 and 15 years,1 and gender dysphoric young people have particularly high rates of SH. This was not pointed out by the authors. And while 4 young people showed an increased propensity to SH over time, 3 decreased – this is in no way an example of “bad science, where we’ve given treatments that have increased suicidality in teenagers” .
This is field that arouses strong passions, with patients who are often, understandably, very dissatisfied with their lot. It is therefore important for major scholarly journals to throw light and take a measured approach. This biased article does a disservice to progress in helping young people since it is not balanced - no editorial or counter view is offered. Such diatribes can make researchers leave contentious fields, as has already happened in chronic fatigue syndrome.
1. Stallard et al. BMC Psychiatry 2013, 13:328
Competing interests: No competing interests