Desmoid-type fibromatosis: toward a holistic management.
Journal
Current opinion in oncology
ISSN: 1531-703X
Titre abrégé: Curr Opin Oncol
Pays: United States
ID NLM: 9007265
Informations de publication
Date de publication:
01 07 2021
01 07 2021
Historique:
pubmed:
12
5
2021
medline:
28
8
2021
entrez:
11
5
2021
Statut:
ppublish
Résumé
Desmoid-type fibromatosis, a rare locally aggressive fibroblastic proliferation, is a treatment challenge. This review aimed to explore recent data about the management of desmoid-type fibromatosis. New data underline the role of kinases and ɣ-secretase in stimulating cell proliferation and invasiveness in desmoid-type fibromatosis. This explains the proven activity of multikinase inhibitors (sorafenib or pazopanib) in the management of desmoid-type fibromatosis or the emerging role of a ɣ-secretase inhibitor. An international guideline for management was recently published, and this guideline take into account patient point of view. Lastly, recent studies highlight the multidimensional burden of desmoid-type fibromatosis, particularly health-related quality of life (HRQoL). Active surveillance with planned MRI is the first-line management in desmoid-type fibromatosis. A site-specific and stepwise approach should be considered for progressive desmoid-type fibromatosis. Further, a risk-benefit analysis that considers the side effects and long-term sequelae should be conducted before deciding to start any treatment. A less aggressive approach should be considered. Multikinase inhibitors are effective, but their tolerability and side effects should be discussed with the patients. The symptoms and HRQoL should be integrated in decision-making. Desmoid-type fibromatosis patients should be offered support to address their needs supportive care.
Identifiants
pubmed: 33973549
doi: 10.1097/CCO.0000000000000743
pii: 00001622-202107000-00010
doi:
Substances chimiques
Enzyme Inhibitors
0
Protein Kinases
EC 2.7.-
Amyloid Precursor Protein Secretases
EC 3.4.-
Types de publication
Journal Article
Review
Langues
eng
Sous-ensembles de citation
IM
Pagination
309-314Informations de copyright
Copyright © 2021 Wolters Kluwer Health, Inc. All rights reserved.
Références
Penel N, Coindre JM, Bonvalot S, et al. Management of desmoid tumours: a nationwide survey of labelled reference centre networks in France. Eur J Cancer 2016; 58:90–96.
Desmoid Tumor Working Group. The management of desmoid tumours: a joint global consensus-based guideline approach for adult and paediatric patients. Eur J Cancer 2020; 127:96–107.
Timbergen MJM, Colombo C, Renckens M, et al. The prognostic role of β-catenin mutations in desmoid-type fibromatosis undergoing resection only: a meta-analysis of individual patient data. Ann Surg 2019.
Crago AM, Chmielecki J, Rosenberg M, et al. Near universal detection of alterations in CTNNB1 and Wnt pathway regulators in desmoid-type fibromatosis by whole-exome sequencing and genomic analysis. Genes Chromosomes Cancer 2015; 54:606–615.
Shang H, Braggio D, Lee YJ, et al. Targeting the notch pathway: a potential therapeutic approach for desmoid tumors. Cancer 2015; 121:4088–4096.
Hughes DP, Kummar S, Lazar AJ. New, tolerable γ-secretase inhibitor takes desmoid down a notch. Clin Cancer Res 2015; 21:7–9.
Trautmann M, Rehkämper J, Gevensleben H, et al. Novel pathogenic alterations in pediatric and adult desmoid-type fibromatosis – a systematic analysis of 204 cases. Sci Rep 2020; 10:3368.
Siozopoulou V, Marcq E, Jacobs J, et al. Desmoid tumors display a strong immune infiltration at the tumor margins and no PD-L1-driven immune suppression. Cancer Immunol Immunother 2019; 68:1573–1583.
Timbergen MJM, Schut AW, Grünhagen DJ, et al. Active surveillance in desmoid-type fibromatosis: a systematic literature review. Eur J Cancer 2020; 137:18–29.
Bishop AJ, Landry JP, Roland CL, et al. Certain risk factors for patients with desmoid tumors warrant reconsideration of local therapy strategies. Cancer 2020; 126:3265–3273.
Penel N, Chibon F, Salas S. Adult desmoid tumors: biology, management and ongoing trials. Curr Opin Oncol 2017; 29:268–274.
Murahashi Y, Emori M, Shimizu J, et al. The value of the black fiber sign on T1-weighted images for predicting stability of desmoid fibromatosis managed conservatively. Eur Radiol 2020; 30:5768–5776.
Cassidy MR, Lefkowitz RA, Long N, et al. Association of MRI T2 signal intensity with desmoid tumor progression during active observation: a retrospective cohort study. Ann Surg 2020; 271:748–755.
Ingley KM, Burtenshaw SM, Theobalds NC, et al. Clinical benefit of methotrexate plus vinorelbine chemotherapy for desmoid fibromatosis (DF) and correlation of treatment response with MRI. Cancer Med 2019; 8:5047–5057.
Zhu HC, Li XT, Ji WY, et al. Desmoid-type fibromatosis: tumour response assessment using magnetic resonance imaging signal and size criteria. Clin Imaging 2020; 68:111–120.
Azzarelli A, Gronchi A, Bertulli R, et al. Low-dose chemotherapy with methotrexate and vinblastine for patients with advanced aggressive fibromatosis. Cancer 2001; 92:1259–1264.
Nishida Y, Hamada S, Urakawa H, et al. Desmoid with biweekly methotrexate and vinblastine shows similar effects to weekly administration: a phase II clinical trial. Cancer Sci 2020; 111:4187–4194.
Mir O, Honoré C, Chamseddine AN, et al. Long-term outcomes of oral vinorelbine in advanced, progressive desmoid fibromatosis and influence of CTNNB1 mutational status. Clin Cancer Res 2020; 26:6277–6283.
Gennatas S, Chamberlain F, Smrke A, et al. A timely oral option: single-agent vinorelbine in desmoid tumors. Oncologist 2020; 25:e2013–e2016.
Ferrari A, Orbach D, Affinita MC, et al. Evidence of hydroxyurea activity in children with pretreated desmoid-type fibromatosis: a new option in the armamentarium of systemic therapies. Pediatr Blood Cancer 2019; 66:e27472.
Toulmonde M, Pulido M, Ray-Coquard I, et al. Pazopanib versus combination chemotherapy using vinblastine and methotrexate in adult with progressive desmoid tumors: a randomized phase II study. Lancet Oncol 2019; 20:1263–1272.
Gounder MM, Mahoney MR, Van Tine BA, et al. Sorafenib for advanced and refractory desmoids tumors. N Engl J Med 2018; 379:2417–2428.
Nishida Y, Sakai T, Koike H, Ito K. Pazopanib for progressive desmoid tumours: children, persistent effects, and cost. Lancet Oncol 2019; 20:e555.
Kummar S, O'Sullivan Coyne G, Do KT, et al. Clinical activity of the γ-secretase inhibitor PF-03084014 in adults with desmoid tumors (aggressive fibromatosis). J Clin Oncol 2017; 35:1561–1569.
Timbergen MJM, van de Poll-Franse LV, Grünhagen DJ, et al. Identification and assessment of health-related quality of life issues in patients with sporadic desmoid-type fibromatosis: a literature review and focus group study. Qual Life Res 2018; 27:3097–3111.
Husson O, Younger E, Dunlop A, et al. Desmoid fibromatosis through the patients’ eyes: time to change the focus and organisation of care? Support Care Cancer 2019; 27:965–980.
Ingley KM, Klein R, Theobalds N, et al. High prevalence of persistent emotional distress in desmoid tumor. Psychooncology 2020; 29:311–320.
Gounder MM, Maddux L, Paty J, Atkinson TM. Prospective development of a patient-reported outcomes instrument for desmoid tumors or aggressive fibromatosis. Cancer 2020; 126:531–539.