Long-term Outcomes of Oral Vinorelbine in Advanced, Progressive Desmoid Fibromatosis and Influence of

Administration, Oral Adolescent Adult Aged Antineoplastic Agents, Phytogenic / administration & dosage Biomarkers, Tumor / genetics Female Fibromatosis, Aggressive / drug therapy Follow-Up Studies Humans Male Middle Aged Mutation Prognosis Response Evaluation Criteria in Solid Tumors Retrospective Studies Survival Rate Vinorelbine / administration & dosage Young Adult beta Catenin / genetics

Journal

Clinical cancer research : an official journal of the American Association for Cancer Research

ISSN: 1557-3265

Titre abrégé: Clin Cancer Res

Pays: United States

ID NLM: 9502500

Informations de publication

Date de publication:
01 12 2020

Historique:

received: 13 05 2020

revised: 15 07 2020

accepted: 28 08 2020

pubmed: 3 9 2020

medline: 15 12 2021

entrez: 3 9 2020

Statut: ppublish

Résumé

Desmoid-type fibromatosis (DF) are locally aggressive neoplasms, with a need for effective systemic treatment in case of progression to avoid the short- and long-term complications of local treatments. We retrospectively analyzed the outcomes of adult patients with DF treated with oral vinorelbine (90 mg once weekly) at Gustave Roussy Cancer Institute (Villejuif, Paris, France). Only patients with documented progressive disease according to RECIST v1.1 for more than 3 months (±2 weeks) before treatment initiation were included. From 2009 to 2019, 90 out of 438 patients with DF were eligible for this analysis. Vinorelbine was given alone in 56 patients (62%), or concomitantly with endocrine therapy in 34 patients, for a median duration of 6.7 months. A partial response was observed in 29% and stable disease in another 57%. With a median follow-up of 52.4 months, the median time to treatment failure (TTF) was not reached. Progression-free rates at 6 and 12 months were 88.7% and 77.5%, respectively. Concomitant endocrine therapy was associated with longer TTF in women [HR, 2.16; 95% confidence interval (CI), 1.06-4.37; Oral vinorelbine is an effective, affordable, and well-tolerated regimen in patients with advanced, progressive DF. Prolonged activity was observed in patients with tumors harboring

Identifiants

DOI: 10.1158/1078-0432.CCR-20-1847 PMID: 32873570

pubmed: 32873570

pii: 1078-0432.CCR-20-1847

doi: 10.1158/1078-0432.CCR-20-1847

doi:

Substances chimiques

Antineoplastic Agents, Phytogenic 0

Biomarkers, Tumor 0

CTNNB1 protein, human 0

beta Catenin 0

Vinorelbine Q6C979R91Y

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

Pagination

6277-6283

Commentaires et corrections

Type : CommentIn

Informations de copyright

Références

The management of desmoid tumours: a joint global consensus-based guideline approach for adult and paediatric patients. Eur J Cancer. 2020;127:96–107.

Tejpar S, Nollet F, Li C, Wunder JS, Michils G, dal Cin P, et al. Predominance of beta-catenin mutations and beta-catenin dysregulation in sporadic aggressive fibromatosis (desmoid tumor). Oncogene. 1999;18:6615–20.

Colombo C, Miceli R, Lazar AJ, Perrone F, Pollock RE, Le Cesne A, et al. CTNNB1 45F mutation is a molecular prognosticator of increased postoperative primary desmoid tumor recurrence: an independent, multicenter validation study. Cancer. 2013;119:3696–702.

Salas S, Dufresne A, Bui B, Blay J-Y, Terrier P, Ranchere-Vince D, et al. Prognostic factors influencing progression-free survival determined from a series of sporadic desmoid tumors: a wait-and-see policy according to tumor presentation. J Clin Oncol. 2011;29:3553–8.

Fiore M, Rimareix F, Mariani L, Domont J, Collini P, Le Péchoux C, et al. Desmoid-type fibromatosis: a front-line conservative approach to select patients for surgical treatment. Ann Surg Oncol. 2009;16:2587–93.

Garbay D, Le Cesne A, Penel N, Chevreau C, Marec-Berard P, Blay J-Y, et al. Chemotherapy in patients with desmoid tumors: a study from the French Sarcoma Group (FSG). Ann Oncol. 2012;23:182–6.

Gounder MM, Mahoney MR, Van Tine BA, Ravi V, Attia S, Deshpande HA, et al. Sorafenib for advanced and refractory desmoid tumors. N Engl J Med. 2018;379:2417–28.

Toulmonde M, Pulido M, Ray-Coquard I, Andre T, Isambert N, Chevreau C, et al. Pazopanib or methotrexate-vinblastine combination chemotherapy in adult patients with progressive desmoid tumours (DESMOPAZ): a non-comparative, randomised, open-label, multicentre, phase 2 study. Lancet Oncol. 2019;20:1263–72.

Napolitano A, Provenzano S, Colombo C, Vitellaro M, Brunello A, Badalamenti G, et al. Familial adenomatosis polyposis-related desmoid tumours treated with low-dose chemotherapy: results from an international, multi-institutional, retrospective analysis. ESMO Open. 2020;5:pii:e000604.

Palassini E, Frezza AM, Mariani L, Lalli L, Colombo C, Fiore M, et al. Long-term efficacy of methotrexate plus vinblastine/vinorelbine in a large series of patients affected by desmoid-type fibromatosis. Cancer J. 2017;23:86–91.

Rowinsky EK, Noe DA, Trump DL, Winer EP, Lucas VS, Wargin WA, et al. Pharmacokinetic, bioavailability, and feasibility study of oral vinorelbine in patients with solid tumors. J Clin Oncol. 1994;12:1754–63.

Aapro MS, Conte P, Esteban González E, Trillet-Lenoir V. Oral vinorelbine: role in the management of metastatic breast cancer. Drugs. 2007;67:657–67.

Gralla RJ, Gatzemeier U, Gebbia V, Huber R, O'Brien M, Puozzo C. Oral vinorelbine in the treatment of non-small cell lung cancer: rationale and implications for patient management. Drugs. 2007;67:1403–10.

Azzarelli A, Gronchi A, Bertulli R, Tesoro Tess JD, Baratti D, Pennacchioli E, et al. Low-dose chemotherapy with methotrexate and vinblastine for patients with advanced aggressive fibromatosis. Cancer. 2001;92:1259–64.

Weiss AJ, Horowitz S, Lackman RD. Therapy of desmoid tumors and fibromatosis using vinorelbine. Am J Clin Oncol. 1999;22:193–5.

Thomas de Montpréville V, Lacroix L, Rouleau E, Mamodaly M, Leclerc J, Tutuianu L, et al. Non-small cell carcinoma with CTNNB1 (beta-catenin) mutations: a clinicopathological study of 26 cases. Ann Diagn Pathol. 2020;46:151522.

Eisenhauer EA, Therasse P, Bogaerts J, Schwartz LH, Sargent D, Ford R, et al. New response evaluation criteria in solid tumours: revised RECIST guideline (version 1.1). Eur J Cancer. 2009;45:228–47.

Marty M, Fumoleau P, Adenis A, Rousseau Y, Merrouche Y, Robinet G, et al. Oral vinorelbine pharmacokinetics and absolute bioavailability study in patients with solid tumors. Ann Oncol. 2001;12:1643–9.

Levêque D, Jehl F. Clinical pharmacokinetics of vinorelbine. Clin Pharmacokinet. 1996;31:184–97.

Rowinsky EK, Lucas VS, Hsieh A-LY, Wargin WA, Hohneker JA, Lubejko B, et al. The effects of food and divided dosing on the bioavailability of oral vinorelbine. Cancer Chemother Pharmacol. 1996;39:9–16.

Colombo C, Belfiore A, Paielli N, De Cecco L, Canevari S, Laurini E, et al. β-catenin in desmoid-type fibromatosis: deep insights into the role of T41A and S45F mutations on protein structure and gene expression. Mol Oncol. 2017;11:1495–507.

Patel SR, Evans HL, Benjamin RS. Combination chemotherapy in adult desmoid tumors. Cancer. 1993;72:3244–7.

Hansmann A, Adolph C, Vogel T, Unger A, Moeslein G. High-dose tamoxifen and sulindac as first-line treatment for desmoid tumors. Cancer. 2004;100:612–20.

Kasper B, Gruenwald V, Reichardt P, Bauer S, Rauch G, Limprecht R, et al. Imatinib induces sustained progression arrest in RECIST progressive desmoid tumours: final results of a phase II study of the German Interdisciplinary Sarcoma Group (GISG). Eur J Cancer. 2017;76:60–7.

Penel N, Le Cesne A, Bui BN, Perol D, Brain EG, Ray-Coquard I, et al. Imatinib for progressive and recurrent aggressive fibromatosis (desmoid tumors): an FNCLCC/French Sarcoma Group phase II trial with a long-term follow-up. Ann Oncol. 2011;22:452–7.

Heinrich MC, McArthur GA, Demetri GD, Joensuu H, Bono P, Herrmann R, et al. Clinical and molecular studies of the effect of imatinib on advanced aggressive fibromatosis (desmoid tumor). J Clin Oncol. 2006;24:1195–203.

Kasper B, Gruenwald V, Reichardt P, Bauer S, Hohenberger P, Haller F. Correlation of CTNNB1 mutation status with progression arrest rate in RECIST progressive desmoid-type fibromatosis treated with imatinib: translational research results from a phase 2 study of the German Interdisciplinary Sarcoma Group (GISG-01). Ann Surg Oncol. 2016;23:1924–7.

Hamada S, Futamura N, Ikuta K, Urakawa H, Kozawa E, Ishiguro N, et al. CTNNB1 S45F mutation predicts poor efficacy of meloxicam treatment for desmoid tumors: a pilot study. PLoS One. 2014;9:e96391.

Timbergen MJM, Colombo C, Renckens M, Kim HS, Rosmalen JV, Salas S, 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 Dec 2 [Epub ahead of print].

Wu G, He X. Threonine 41 in beta-catenin serves as a key phosphorylation relay residue in beta-catenin degradation. Biochemistry. 2006;45:5319–23.

Liu C, Li Y, Semenov M, Han C, Baeg G-H, Tan Yi, et al. Control of beta-catenin phosphorylation/degradation by a dual-kinase mechanism. Cell. 2002;108:837–47.

Desurmont T, Lefèvre JH, Shields C, Colas C, Tiret E, Parc Y. Desmoid tumour in familial adenomatous polyposis patients: responses to treatments. Fam Cancer. 2015;14:31–9.

Greer JA, Amoyal N, Nisotel L, Fishbein JN, MacDonald J, Stagl J, et al. A Systematic review of adherence to oral antineoplastic therapies. Oncologist. 2016;21:354–76.