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

doi:10.1002/pbc.29831

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 journal › Review article › Academic › peer-review

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Abstract

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 language	English
Article number	e29831
Journal	Pediatric Blood and Cancer
Volume	69
Issue number	9
DOIs	https://doi.org/10.1002/pbc.29831
Publication status	Published - Sep 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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Clinical trials in desmoid-type fibromatosis in children and adultsFinal published version, 404 KBLicence: CC BY-NC-ND

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title = "Clinical trials in desmoid-type fibromatosis in children and adults: A systematic review",

abstract = "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.",

author = "{van Maren}, {Simone A.} and {van Noesel}, {Max M.} and Olga Husson and {van der Graaf}, {Winette T.A.}",

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: {\textcopyright} 2022 The Authors. Pediatric Blood & Cancer published by Wiley Periodicals LLC.",

year = "2022",

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AU - Husson, Olga

AU - van der Graaf, Winette T.A.

N1 - 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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AB - 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.

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

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