BACKGROUND: Advanced biliary tract carcinoma has a very poor prognosis, with chemotherapy being the mainstay of treatment. Sorafenib, a multikinase inhibitor of VEGFR-2/-3, PDGFR-beta, B-Raf, and C-Raf, has shown to be active in preclinical models of cholangiocarcinoma. METHODS: We conducted a phase II trial of single-agent sorafenib in patients with advanced biliary tract carcinoma. Sorafenib was administered at a dose of 400 mg twice a day. The primary end point was the disease control rate at 12 weeks. RESULTS: A total of 46 patients were treated. In all, 26 (56%) had received chemotherapy earlier, and 36 patients completed at least 45 days of treatment. In intention-to-treat analysis, the objective response was 2% and the disease control rate at 12 weeks was 32.6%. Progression-free survival (PFS) was 2.3 months (range: 0-12 months), and the median overall survival was 4.4 months (range: 0-22 months). Performance status was significantly related to PFS: median PFS values for ECOG 0 and 1 were 5.7 and 2.1 months, respectively (P=0.0002). The most common toxicities were skin rash (35%) and fatigue (33%), requiring a dose reduction in 22% of patients. CONCLUSIONS: Sorafenib as a single agent has a low activity in cholangiocarcinoma. Patients having a good performance status have a better PFS. The toxicity profile is manageable.

Sorafenib in patients with advanced biliary tract carcinoma: a phase II trial / Bengala, C; Bertolini, Federica; Malavasi, Norma; Boni, C; Aitini, E; Dealis, Cristina; Zironi, S; Depenni, R; Fontana, Annalisa; DEL GIOVANE, Cinzia; Luppi, G; Conte, Pierfranco. - In: BRITISH JOURNAL OF CANCER. - ISSN 0007-0920. - STAMPA. - 102:1(2010), pp. 68-72. [10.1038/sj.bjc.6605458]

Sorafenib in patients with advanced biliary tract carcinoma: a phase II trial

BERTOLINI, Federica;MALAVASI, Norma;DEALIS, Cristina;FONTANA, Annalisa;DEL GIOVANE, Cinzia;CONTE, Pierfranco
2010

Abstract

BACKGROUND: Advanced biliary tract carcinoma has a very poor prognosis, with chemotherapy being the mainstay of treatment. Sorafenib, a multikinase inhibitor of VEGFR-2/-3, PDGFR-beta, B-Raf, and C-Raf, has shown to be active in preclinical models of cholangiocarcinoma. METHODS: We conducted a phase II trial of single-agent sorafenib in patients with advanced biliary tract carcinoma. Sorafenib was administered at a dose of 400 mg twice a day. The primary end point was the disease control rate at 12 weeks. RESULTS: A total of 46 patients were treated. In all, 26 (56%) had received chemotherapy earlier, and 36 patients completed at least 45 days of treatment. In intention-to-treat analysis, the objective response was 2% and the disease control rate at 12 weeks was 32.6%. Progression-free survival (PFS) was 2.3 months (range: 0-12 months), and the median overall survival was 4.4 months (range: 0-22 months). Performance status was significantly related to PFS: median PFS values for ECOG 0 and 1 were 5.7 and 2.1 months, respectively (P=0.0002). The most common toxicities were skin rash (35%) and fatigue (33%), requiring a dose reduction in 22% of patients. CONCLUSIONS: Sorafenib as a single agent has a low activity in cholangiocarcinoma. Patients having a good performance status have a better PFS. The toxicity profile is manageable.
2010
102
1
68
72
Sorafenib in patients with advanced biliary tract carcinoma: a phase II trial / Bengala, C; Bertolini, Federica; Malavasi, Norma; Boni, C; Aitini, E; Dealis, Cristina; Zironi, S; Depenni, R; Fontana, Annalisa; DEL GIOVANE, Cinzia; Luppi, G; Conte, Pierfranco. - In: BRITISH JOURNAL OF CANCER. - ISSN 0007-0920. - STAMPA. - 102:1(2010), pp. 68-72. [10.1038/sj.bjc.6605458]
Bengala, C; Bertolini, Federica; Malavasi, Norma; Boni, C; Aitini, E; Dealis, Cristina; Zironi, S; Depenni, R; Fontana, Annalisa; DEL GIOVANE, Cinzia; Luppi, G; Conte, Pierfranco
File in questo prodotto:
File Dimensione Formato  
6605458.pdf

Open access

Tipologia: Versione pubblicata dall'editore
Dimensione 223.28 kB
Formato Adobe PDF
223.28 kB Adobe PDF Visualizza/Apri
Pubblicazioni consigliate

Licenza Creative Commons
I metadati presenti in IRIS UNIMORE sono rilasciati con licenza Creative Commons CC0 1.0 Universal, mentre i file delle pubblicazioni sono rilasciati con licenza Attribuzione 4.0 Internazionale (CC BY 4.0), salvo diversa indicazione.
In caso di violazione di copyright, contattare Supporto Iris

Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11380/640274
Citazioni
  • ???jsp.display-item.citation.pmc??? 82
  • Scopus 204
  • ???jsp.display-item.citation.isi??? 178
social impact