RT Journal Article
T1 Familial adenomatosis polyposis-related desmoid tumours treated with low-dose chemotherapy: results from an international, multi-institutional, retrospective analysis.
A1 Napolitano, Andrea
A1 Provenzano, Salvatore
A1 Colombo, Chiara
A1 Vitellaro, Marco
A1 Brunello, Antonella
A1 Badalamenti, Giuseppe
A1 Nannini, Margherita
A1 Ibrahim, Toni
A1 Hohenberger, Peter
A1 Gasperoni, Silvia
A1 Gennatas, Spyridon
A1 Jones, Robin L
A1 Hindi, Nadia
A1 Martin-Broto, Javier
A1 Spalato Ceruso, Mariella
A1 Silletta, Marianna
A1 Dei Tos, Angelo Paolo
A1 Gronchi, Alessandro
A1 Stacchiotti, Silvia
A1 Santini, Daniele
A1 Tonini, Giuseppe
A1 Palassini, Elena
A1 Vincenzi, Bruno
K1 chemotherapy
K1 desmoid
K1 familial adenomatosis polyposis
K1 methotrexate
K1 vinca alkaloids
AB Desmoid tumour (DT) is a locally aggressive fibroblastic proliferative disease representing the most common extraintestinal manifestation of familial adenomatosis polyposis (FAP). As data on the activity of chemotherapy in these patients are limited, we examined the outcomes of patients treated with low-dose methotrexate (MTX)+vinca alkaloids (vinorelbine or vinblastine). We retrospectively reviewed clinical and outcome data from all patients with confirmed FAP-associated DTs treated with weekly MTX+vinca alkaloids in seven European sarcoma reference centres between January 2000 and December 2018. Radiological responses were assessed using RECIST V.1.0 and V.1.1. The Kaplan-Meier method associated to the log-rank test was used to estimate and compare survival curves. We identified 37 patients (median age 29 years, range 7-44). According to RECIST, 20/37 (54.1%) patients achieved partial response (PR), 15/37 (40.5%) patients had stable disease and 2/37 (5.4%) had progressive disease as best response. Overall, the median progression-free survival (PFS) was 6.5 years (range, 0.3-12.1 years). In the subset of patients achieving PR as best response, the median PFS was not reached. In a subset of 11 patients with progressive disease offered MTX+vinca alkaloids rechallenge (after chemotherapy withdrawal following prolonged disease control), the disease control rate was 100%, resulting in a median PFS after rechallenge of 5.8 years. This is the largest series on the activity of low-dose chemotherapy in patients with FAP-related DT. In this population, MTX+vinca alkaloids is an active combination, as already reported in patients with sporadic DT.
YR 2020
FD 2020
LK http://hdl.handle.net/10668/14969
UL http://hdl.handle.net/10668/14969
LA en
DS RISalud
RD Apr 8, 2025