Pazopanib for treatment of typical solitary fibrous tumours: a multicentre, single-arm, phase 2 trial

Javier Martin-Broto, Josefina Cruz, Nicolas Penel, Axel Le Cesne, Nadia Hindi, Pablo Luna, David S Moura, Daniel Bernabeu, Enrique de Alava, Jose Antonio Lopez-Guerrero, Joaquin Dopazo, Maria Peña-Chilet, Antonio Gutierrez, Paola Collini, Marie Karanian, Andres Redondo, Antonio Lopez-Pousa, Giovanni Grignani, Juan Diaz-Martin, David MarcillaAntonio Fernandez-Serra, Cristina Gonzalez-Aguilera, Paolo G Casali, Jean-Yves Blay, Silvia Stacchiotti

Research output: Contribution to journalArticlepeer-review


BACKGROUND: Solitary fibrous tumour is an ultra-rare sarcoma, which encompasses different clinicopathological subgroups. The dedifferentiated subgroup shows an aggressive course with resistance to pazopanib, whereas in the malignant subgroup, pazopanib shows higher activity than in previous studies with chemotherapy. We designed a trial to test pazopanib activity in two different cohorts of solitary fibrous tumour: the malignant-dedifferentiated cohort, which was previously published, and the typical cohort, which is presented here.

METHODS: In this single-arm, phase 2 trial, adult patients (aged ≥18 years) diagnosed with confirmed metastatic or unresectable typical solitary fibrous tumour of any location, who had progressed in the previous 6 months (by Choi criteria or Response Evaluation Criteria in Solid Tumors [RECIST]) and an Eastern Cooperative Oncology Group (ECOG) performance status of 0-2 were enrolled at 11 tertiary hospitals in Italy, France, and Spain. Patients received pazopanib 800 mg once daily, taken orally, until progression, unacceptable toxicity, withdrawal of consent, non-compliance, or a delay in pazopanib administration of longer than 3 weeks. The primary endpoint was proportion of patients achieving an overall response measured by Choi criteria in patients who received at least 1 month of treatment with at least one radiological assessment. All patients who received at least one dose of the study drug were included in the safety analyses. This study is registered in, NCT02066285, and with the European Clinical Trials Database, EudraCT 2013-005456-15.

FINDINGS: From June 26, 2014, to Dec 13, 2018, of 40 patients who were assessed, 34 patients were enrolled and 31 patients were included in the response analysis. Median follow-up was 18 months (IQR 14-34), and 18 (58%) of 31 patients had a partial response, 12 (39%) had stable disease, and one (3%) showed progressive disease according to Choi criteria and central review. The proportion of overall response based on Choi criteria was 58% (95% CI 34-69). There were no deaths caused by toxicity, and the most frequent adverse events were diarrhoea (18 [53%] of 34 patients), fatigue (17 [50%]), and hypertension (17 [50%]).

INTERPRETATION: To our knowledge, this is the first prospective trial of pazopanib for advanced typical solitary fibrous tumour. The manageable toxicity and activity shown by pazopanib in this cohort suggest that this drug could be considered as first-line treatment for advanced typical solitary fibrous tumour.

FUNDING: Spanish Group for Research on Sarcomas (GEIS), Italian Sarcoma Group (ISG), French Sarcoma Group (FSG), GlaxoSmithKline, and Novartis.

Original languageEnglish
Pages (from-to)456-466
Number of pages11
JournalThe Lancet. Oncology
Issue number3
Publication statusPublished - Mar 2020


  • Aged
  • Female
  • Follow-Up Studies
  • Humans
  • Male
  • Middle Aged
  • Neoplasm Metastasis
  • Prognosis
  • Prospective Studies
  • Protein Kinase Inhibitors/therapeutic use
  • Pyrimidines/therapeutic use
  • Response Evaluation Criteria in Solid Tumors
  • Solitary Fibrous Tumors/drug therapy
  • Sulfonamides/therapeutic use
  • Survival Rate


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