[0:16]Desmoid tumors or aggressive fibromatosis are rare soft tissue tumors. They are locally aggressive but they do not spread so it's not a metastatic disease. It's very rare and with this low incidence come a few problems because it requires expertise to make right decisions on how to treat patients and also it requires expertise to make the right diagnosis. For example, there is a biomarker that helps pathologists to make the diagnosis, and that is mutations in the beta catenin gene.
[1:02]Desmoid fibromatosis have for a long time been treated very aggressively. That means they have been perceived as sarcomas, have been treated with aggressive surgery, wide margins, frequently also radiotherapy, but we have learned in recent years that these tumors very frequently spontaneously stop growing and even start to shrink. So, whenever you make the diagnosis of a desmoid fibromatosis, the first thing is refer these patients to centers who have experience, sarcoma centers, centers of excellence to make a decision is there an indication for an actually active treatment. The most important trigger for a treatment is if patients have symptomatic disease. And when you think about a treatment, you have several options of how you can treat a tumor.
[2:09]When we have a patient with symptomatic disease, we have several options at our disposal. The first one is local treatments, that includes surgery, that includes radiotherapy, but it also includes locally ablative treatments such as cryotherapy. And on top of that, we have found out in recent years that there are quite a few number of drugs that can have beneficial effects on patients and lead to successful tumor shrinkage and with tumor shrinkage of course symptom relief. And among those drugs are VFR inhibitors such as sorafenib, but more recently also drugs that are called gamma secretase inhibitors. They are not approved yet, but they may be on their way to be approved. So far, the the most powerful trials in that disease have shown that these treatments are superior to placebo. So we see way more patients having tumor reductions when you treat them compared to patients with placebo. We don't know enough about those treatments if um the duration of treatment, let's say over one or two years will cure the patients from these desmoid tumors to grow ever again. And that is going to be part of future research to see how long do we need to treat, can we um safely retreat, does it make sense? Do we need to combine treatments to finally cure patients of their disease? And I think there is a chance that this is going to happen. What I would like everyone who crosses a desmoid tumor in in their practice should keep in mind these tumors are rare, you're not gaining anything by treating these patients on your own. And and it is absolutely in the patient's interest to be consulted at the time of diagnosis so that a multidisciplinary team of experts can steer the disease and very frequently that can be done in collaboration with the local private practition or with the local caretakers, but that will make sure that patients are not overtreated or undertreated and may not receive drugs that are really innovative and will help them to to successfully treat their disease.
