TY - JOUR
T1 - Long-term follow-up of nilotinib in patients with advanced tenosynovial giant cell tumours
T2 - Long-term follow-up of nilotinib in TGCT
AU - Spierenburg, Geert
AU - Grimison, Peter
AU - Chevreau, Christine
AU - Stacchiotti, Silvia
AU - Piperno-Neumann, Sophie
AU - Le Cesne, Axel
AU - Ferraresi, Virginia
AU - Italiano, Antoine
AU - Duffaud, Florence
AU - Penel, Nicolas
AU - Metzger, Severine
AU - Chabaud, Sylvie
AU - van der Heijden, Lizz
AU - Pérol, David
AU - van de Sande, Michiel A.J.
AU - Blay, Jean Yves
AU - Gelderblom, Hans
N1 - Publisher Copyright:
© 2022 The Author(s)
PY - 2022/9/1
Y1 - 2022/9/1
N2 - Background: Diffuse-type tenosynovial giant cell tumour (D-TGCT) is a non-malignant but locally aggressive tumour driven by overexpression of colony-stimulating factor-1 (CSF1). CSF1R inhibitors are potential therapeutic strategies for patients not amenable to surgery. We report here the long-term outcome of nilotinib in patients with advanced D-TGCT treated within a phase II prospective international study (ClinicalTrials.gov: NCT01261429). Methods: Patients were enrolled between December 2010–September 2012 at 11 cancer centres. Eligible patients had histologically confirmed D-TGCT, not amenable to surgery. Patients received nilotinib until evidence of progression, toxicity or a maximum of one year. Long-term data were retrospectively collected after the completion of the phase II trial. Patients with nilotinib treatment ≥12 weeks and follow-up ≥12 months were included for long-term analysis. Results: Forty-eight of 56 enrolled patients were included. Median treatment duration was 11 months; 31 (65%) patients completed the treatment protocol. After 102 months of follow-up (median; range 12–129), 25 patients (52%) had progression. The median progression-free survival (PFS) was 77 months. The five-year PFS rate was 53%. Fifteen patients (n = 15/46; 33%) experienced clinical worsening after 11 months (median). Twenty-seven patients (58%) received additional treatment, after which eleven patients (n = 11/27; 41%) had a second relapse. Nine patients required a subsequent treatment, primarily other CSF1R inhibitors (n = 6/9; 67%). No unfavourable long-term effects were observed. Conclusion: This long-term analysis of nilotinib for advanced D-TGCT showed that about half of the patients had progression and underwent additional treatment after 8.5 years follow-up. Contrarily, several patients had ongoing disease control after limited treatment duration, demonstrating the mixed effect of nilotinib.
AB - Background: Diffuse-type tenosynovial giant cell tumour (D-TGCT) is a non-malignant but locally aggressive tumour driven by overexpression of colony-stimulating factor-1 (CSF1). CSF1R inhibitors are potential therapeutic strategies for patients not amenable to surgery. We report here the long-term outcome of nilotinib in patients with advanced D-TGCT treated within a phase II prospective international study (ClinicalTrials.gov: NCT01261429). Methods: Patients were enrolled between December 2010–September 2012 at 11 cancer centres. Eligible patients had histologically confirmed D-TGCT, not amenable to surgery. Patients received nilotinib until evidence of progression, toxicity or a maximum of one year. Long-term data were retrospectively collected after the completion of the phase II trial. Patients with nilotinib treatment ≥12 weeks and follow-up ≥12 months were included for long-term analysis. Results: Forty-eight of 56 enrolled patients were included. Median treatment duration was 11 months; 31 (65%) patients completed the treatment protocol. After 102 months of follow-up (median; range 12–129), 25 patients (52%) had progression. The median progression-free survival (PFS) was 77 months. The five-year PFS rate was 53%. Fifteen patients (n = 15/46; 33%) experienced clinical worsening after 11 months (median). Twenty-seven patients (58%) received additional treatment, after which eleven patients (n = 11/27; 41%) had a second relapse. Nine patients required a subsequent treatment, primarily other CSF1R inhibitors (n = 6/9; 67%). No unfavourable long-term effects were observed. Conclusion: This long-term analysis of nilotinib for advanced D-TGCT showed that about half of the patients had progression and underwent additional treatment after 8.5 years follow-up. Contrarily, several patients had ongoing disease control after limited treatment duration, demonstrating the mixed effect of nilotinib.
KW - CSF1
KW - CSF1R inhibitor
KW - Nilotinib
KW - PVNS
KW - TGCT
KW - Tyrosine kinase inhibitor
UR - http://www.scopus.com/inward/record.url?scp=85135398450&partnerID=8YFLogxK
U2 - 10.1016/j.ejca.2022.06.028
DO - 10.1016/j.ejca.2022.06.028
M3 - Article
C2 - 35932628
AN - SCOPUS:85135398450
SN - 0959-8049
VL - 173
SP - 219
EP - 228
JO - European Journal of Cancer
JF - European Journal of Cancer
ER -