Since Summer 2021, the feasibility study stage of a major UK research programme – called TOAST (Treating Oesophageal Atresia to Prevent Stricture) – has been going on. This trial is the first one in which TOFS has formally taken part, as Patient and Public Involvement partner, and we have been working with the trials team since late 2019.
The main point of this feasibility study was to see whether the proposed full trial would be acceptable to a sample of parents and also to a set of clinicians. Encouragingly, the outcomes were positive, and formal go-ahead for the next stages of the trial has now been given by the funder, the National Institute for Health Research, as recommended by. the Trial’s Steering Committee.
There is a lot of background preparation work now to be done, mostly by the Clinical trials team at Oxford University’s NPEU, but the aim is now to start the main trial in the Spring / early Summer 2022.
The main trial will be a “gold standard” randomised controlled trial to establish whether or not the routine use of antacid drugs (such as Omeprazole) with OA/TOF babies is helpful to them. The multi-year programme is expected to involve some 12 to 15 of the 26 UK surgical centres where OA/TOF babies are initially treated.
Some 18 TOFS members, all parents of young OA children, helped greatly by talking in detail to the researchers – thank you! Moreover, the design of the trial has been tweaked in several detailed respects as a result of the inputs especially from parents. One example of this is that the Parent Information Leaflet will now include an everyday-language version of the “symptomatic reflux treatment pathway”, which aims to ensure that any infant on the trial who seems to develop symptoms of reflux has those symptoms properly attended to. The feasibility study also asked parents about the range of outcomes which are to be observed in the trial, and useful feedback emerged from this.
The trial was seen to be acceptable to all 18 parents, with 15 saying they would definitely have given consent for their new OA baby to take part – and the remaining three also saying yes, with some provisos.
Some 51 UK clinicians – all of whom are currently involved in care for OA babies – responded to a survey. The responses will help to adjust some aspects of the intended trial. But overall, three quarters of the clinicians viewed the trial as acceptable, with only 2% viewing it as unacceptable.
As you may imagine, the trial team is talking in some more detail to address the concerns of the handful of clinicians.
This TOAST research programme will be the biggest trial of its type in the World and will be a major UK contribution to knowledge about how best to treat OA/TOF babies. Its results should make it clear either that OA/TOF babies should only be given antacids in cases where there is a diagnosis of reflux or that all OA/TOF babies should have antacids regardless. We anticipate that the trial’s outcomes will make it possible to strengthen or alter the ESPGHAN / NASPGHAN (European Society for Paediatric Gastroenterology, Hepatology and Nutrition / North American Society For Pediatric Gastroenterology, Hepatology & Nutrition) guidelines which were published in 2016.
A bit of background:
It is thought that around half of the UK’s OA/TOF babies are routinely given antacids, i.e whether or not there is any sign of a given infant suffering from acidic reflux.
Indeed, the ESPGHAN / NASPGHAN guidelines suggest this. However they do so only on the basis of expert opinion, and say that this is backed by a low level of evidence: this is a signal that more research would be welcome. Our international federation of OA/TOF support groups (EAT) reviewed these guidelines before publication, and TOFS is generally supportive of them. We have drawn members’ attention to these guidelines a number of times in Chew. Moreover we are asking the British body BSPGHAN what we need to do to have such guidelines formally adopted in the UK – or suitably adapted for UK use.
Research done on this subject around the world to date has been limited, with no major controlled trials and it has not so far been clearly established that routine use of antacids with OA/TOF babies is beneficial. Some research suggests that this practice might be harmful.
In a typical randomised controlled trial, a randomly-chosen half of the participants are given the medication being trialled, and the other half an identical – seeming placebo. If the medication is effective, the outcomes for the two groups of participants are notably different.
For more information, please visit our TOAST trial page.