title: Pazopanib in advanced desmoplastic small round cell tumours: a multi-institutional experience.
abstract: BACKGROUND
  We retrospectively reviewed data from nine pre-treated metastatic desmoplastic small round cell tumour (DSRCT) patients who received pazopanib.
  
  
  METHODS
  Three patients received pazopanib within the EORTC phase II 62043, three in the EORTC phase III 62072, and three in the context of UK named patient program.
  
  
  RESULTS
  Nine patients were retrieved from the databases, the median age was 30 years (range: 21-47), they were all males. All had received prior chemotherapy. At the time of treatment start, 4 patients (44%) had ECOG PS 0, 4 (44%) PS 1, 1 (11%) PS 2. Best response was partial response (PR) in 2/9 (22%) patients, stable disease (SD) in 5/9 (56%) and progressive disease (PD) in 2/9 (22%) with a clinical benefit rate (PR + SD > 12 weeks) of 78%. Median PFS and OS were 9.2 (95%CI: 0-23.2) and 15.4 (95%CI: 1.5-29.3) months respectively. With a median follow-up of 20 months, 2/9 (22%) patients are still alive, all progressed. The most common toxicities included neutropenia (G1-2 45%; G3-4 11%), anaemia (G1-2 45%), fatigue (G1-2 67%), diarrhoea (G1-2 45%; G3-4 11%), nausea (G1-2 45%), hypertension (G1-2 45%) and increase in liver enzymes (G1-2 34%; G3-4 11%). Three patients (34%) required a dose reduction. One of the patients discontinued treatment because of persistent increase in total bilirubin level, one due to patient's choice.
  
  
  CONCLUSIONS
  In this series, pazopanib showed interesting activity in DSRCT patients who progressed after prior chemotherapy without major toxicity.
authors.affiliation: Medical Oncology, University Campus Bio-Medico, Via Alvaro del Portillo 200, Rome 00128, Italy.
authors.forename: Anna Maria
authors.initials: AM
authors.lastname: Frezza
authors.affiliation: Sarcoma Unit, Royal Marsden Hospital, London, UK.
authors.forename: Charlotte
authors.initials: C
authors.lastname: Benson
authors.affiliation: Sarcoma Unit, Royal Marsden Hospital, London, UK.
authors.forename: Ian R
authors.initials: IR
authors.lastname: Judson
authors.affiliation: EORTC HQ, Brussels, Belgium.
authors.forename: Saskia
authors.initials: S
authors.lastname: Litiere
authors.affiliation: EORTC HQ, Brussels, Belgium.
authors.forename: Sandrine
authors.initials: S
authors.lastname: Marreaud
authors.affiliation: Medical Oncology, Erasmus MC Cancer Institute, Rotterdam, The Netherlands.
authors.forename: Stefan
authors.initials: S
authors.lastname: Sleijfer
authors.affiliation: Medical Oncology, Centre Leon Berard, Lyon, France.
authors.forename: Jean-Yves
authors.initials: JY
authors.lastname: Blay
authors.affiliation: GlaxoSmithKline, Oncology, Uxbridge, UK.
authors.forename: Raz
authors.initials: R
authors.lastname: Dewji
authors.affiliation: Sarcoma Unit, Royal Marsden Hospital, London, UK.
authors.forename: Cyril
authors.initials: C
authors.lastname: Fisher
authors.affiliation: Department of Medical Oncology, Radboud University Medical Center, Nijmegen, The Netherlands.
authors.forename: Winette
authors.initials: W
authors.lastname: van der Graaf
authors.affiliation: Medical Oncology, NHS Lothians, Edinburgh, UK.
authors.forename: Larry
authors.initials: L
authors.lastname: Hayward
keywords: Desmoplastic small round cell tumour; Pazopanib; Tyrosine kinase inhibitor
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