Clinical trials in desmoid-type fibromatosis in children and adults: A systematic review

Simone A. van Maren, Max M. van Noesel, Olga Husson, Winette T.A. van der Graaf*

*Corresponding author for this work

Research output: Contribution to journalReview articleAcademicpeer-review

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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.

Original languageEnglish
Article numbere29831
JournalPediatric Blood and Cancer
Issue number9
Publication statusPublished - Sept 2022

Bibliographical note

Funding Information:
The authors are grateful to Milou Reuvers (MR) for providing her help with the screening of the retrieved citations during the selection procedure.

Publisher Copyright:
© 2022 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC.


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