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

108 Downloads (Pure)

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 - 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.

Access to Document

10.1002/pbc.29831Licence: CC BY-NC-ND

Clinical trials in desmoid-type fibromatosis in children and adultsFinal published version, 404 KBLicence: CC BY-NC-ND

Cite this

@article{0135fb5c703d4bbfba975bcef07828da,

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",

month = sep,

doi = "10.1002/pbc.29831",

language = "English",

volume = "69",

journal = "Pediatric Blood and Cancer",

issn = "1545-5009",

publisher = "Wiley-Liss Inc.",

number = "9",

}

TY - JOUR

T1 - Clinical trials in desmoid-type fibromatosis in children and adults

T2 - A systematic review

AU - van Maren, Simone A.

AU - van Noesel, Max M.

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.

PY - 2022/9

Y1 - 2022/9

N2 - 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.

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.

UR - http://www.scopus.com/inward/record.url?scp=85133634316&partnerID=8YFLogxK

U2 - 10.1002/pbc.29831

DO - 10.1002/pbc.29831

M3 - Review article

C2 - 35714333

AN - SCOPUS:85133634316

SN - 1545-5009

VL - 69

JO - Pediatric Blood and Cancer

JF - Pediatric Blood and Cancer

IS - 9

M1 - e29831

ER -

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

Abstract

Bibliographical note

Access to Document

Other files and links

Fingerprint

Cite this