Clinical trials in desmoid-type fibromatosis in children and adults: A systematic review
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Read the article on pubmed.ncbi.nlm.nih.gov ↗Article · Sep 2022 · 1 min read
In brief
In brief
Systematic review of 14 clinical trials examining desmoid-type fibromatosis treatment in pediatric and adult populations reveals wide variation in tumor response rates and highlights treatment disparities between age groups, primarily due to limited tyrosine kinase inhibitor access in pediatric oncology. The review emphasizes the need for more randomized trials and patient-reported outcome studies in this chronic, quality-of-life-impacting condition.
Written by the GCMD Library team from the article.
Desmoid-type fibromatosis (DTF) is a rare locally aggressive soft tissue neoplasm, which occurs in children and adults, with a peak incidence in young adults. For the majority of the patients, DTF is a chronic and symptomatic disease, which affects health-related quality of life. Systemic treatment regimens tend to differ for patients treated by pediatric oncologists compared to medical oncologists. This systematic review identified 14 clinical trials in children and adults with DTF. Tumor response and progression-free survival rates varied widely between studies and study populations. Treatment choices for patients with DTF are based on a paucity of (randomized) trials. Treatment principles of DTF are similar in pediatric and adult oncology, but the treatment itself is different. This seems mostly driven by a lack of tyrosine kinase inhibitor (TKI) accessibility in pediatric oncology. An insufficient number of studies examined patient-reported outcomes, which are extremely important for patients with a chronic disease like DTF.
DOI: 10.1002/pbc.29831