[Systemic treatment for management of aggressive fibromatosis in both adult and children: Level of evidence and unsolved questions].

[Systemic treatment for management of aggressive fibromatosis in both adult and children: Level of evidence and unsolved questions]. Bull Cancer. 2019 Dec 04;: Authors: Penel N, Ryckewaert T, Orbach D Abstract We summarize here available data about systemic treatments of desmoids tumors, as well in adult patients as pediatric patients. Until 2018, the level of evidence associated with these treatments (NSAI, hormonal therapies, tyrosine kinase inhibitors, chemotherapy) was very low, based on retrospective studies or non-randomized phase 2 trials with limited number of cases. In 2018-2019, 2 large randomized trials have been published, including one large superiority phase 3 trial comparing sorafenib to placebo. This trial clearly demonstrates the clinical benefit of sorafenib over placebo (level of evidence IA). To conclude, wait-and-see policy must be the first-line approach, systemic treatment is indicated in case of disease progression. Randomized trials are feasible in this exceptional disease. PMID: 31812284 [PubMed - as supplied by publisher]
Source: Bulletin du Cancer - Category: Cancer & Oncology Authors: Tags: Bull Cancer Source Type: research