INFORM is a registry study which was started in 2015 which is actually a large molecular platform providing comprehensive molecular diagnostics but at the same time also provides clinical follow-up so that we also can look back at what certain alterations and treatments brought for benefit for our paediatric patients.
What were your methods?
As I said before, it’s a registry study meaning that it’s not an interventional study so there was no clinical intervention in that sense. Patients received their diagnostics and treating physicians could use those reports of molecular alterations to base their clinical decision-making on. So they were completely free to decide what to do with this information.
What did you find?
After the molecular alterations were identified we had a weekly molecular tumour board and in this molecular tumour board, an interdisciplinary group of experts, weighed the alterations from very high level priority to very low level priority. We found in this study that most, especially the very high level priority level targets, really benefitted paediatric patients when they were treated accordingly with targeted drugs.
What did you conclude from these findings?
The most important conclusion is that precision oncology for paediatric patients is feasible, also in an international multicentre setting which we did, we had eight countries in there and at the moment it’s already a lot more. But also that patients really benefitted from these diagnostics but, a critical point, this was only a small group, there is still a large group of patients who didn’t benefit from treatment because they didn’t receive the according treatment or because they didn’t have a good target for potential treatments. So in the future we still need more, additional layers of molecular or other diagnostics for the other group of patients which at the moment don’t benefit. But also we need more drugs and especially clinical trials in children.
What will be the clinical implications of this study?
With INFORM we have shown that paediatric precision oncology is possible and since we are also giving benefit to patients we should offer these diagnostics to all high risk relapse patients and maybe also in a somewhat earlier stage. And we have shown that it is possible to provide benefits for patients so we need to work on more access to drugs in clinical trials or off label as we did now most of the time. Because you need to know that in paediatric oncology the accessibility to new innovative drugs is so far not as good as in adult oncology where we have lots and lots of new drugs, new treatments, new trials. But paediatric oncology is lagging behind there but we have shown with INFORM that the targets are there but the treatments are lagging behind.
In the last five years we were able to provide a benefit for a small but significant group of patients, now that we will be able to provide new diagnostics or new drugs in the next five years. If we would make such a step every couple of years that will be really great and a good way forward.
