Long-term follow-up of nilotinib in patients with advanced tenosynovial giant cell tumours: Long-term follow-up of nilotinib in TGCT

Geert Spierenburg, Peter Grimison, Christine Chevreau, Silvia Stacchiotti, Sophie Piperno-Neumann, Axel Le Cesne, Virginia Ferraresi, Antoine Italiano, Florence Duffaud, Nicolas Penel, Severine Metzger, Sylvie Chabaud, Lizz van der Heijden, David Pérol, Michiel A.J. van de Sande, Jean Yves Blay, Hans Gelderblom

Résultats de recherche: Contribution à un journalArticleRevue par des pairs

10 Citations (Scopus)

Résumé

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.

langue originaleAnglais
Pages (de - à)219-228
Nombre de pages10
journalEuropean Journal of Cancer
Volume173
Les DOIs
étatPublié - 1 sept. 2022
Modification externeOui

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