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The Desmoid Tumor Research Foundation

June 2022

Clinical trials in Desmoid-type Fibromatosis in children and (young) adults: A systematic review

Author Information

van Maren SA (1), van Noesel MM (2,3), Husson O (4,5,6,7), van der Graaf (WTA (4,8).

(1) Radboud University Medical Center, Nijmegen, The Netherlands.(2) Princess Máxima Center for pediatric oncology, and Division Cancer & Imaging. (3) University Medical Center Utrecht, Utrecht, The Netherlands. (4) Department of Medical Oncology, Netherlands Cancer Institute, Amsterdam, The Netherlands. (5) Division of Clinical Studies, Institute of Cancer Research, London, UK.(6) Department of Surgical Oncology, Erasmus MC Cancer Institute, Rotterdam, The Netherlands.(7) Division of Psychosocial Research and Epidemiology, Netherlands Cancer Institute, Amsterdam, The Netherlands.(8) Department of medical oncology, Erasmus MC Cancer Institute, Erasmus University Medical Center, Rotterdam, The Netherlands.

Publication

Pediatr Blood Cancer

Summary

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 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.

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