Outcome of patients with sarcoma and other mesenchymal tumours participating in phase I trials: A subset analysis of a European phase I database

Philippe A. Cassier, V. Polivka, I. Judson, J. C. Soria, N. Penel, S. Marsoni, J. Verweij, J. H. Schellens, R. Morales-Barrera, P. Schöffski, E. E. Voest, C. Gomez-Roca, T. R J Evans, R. Plummer, E. Gallerani, S. B. Kaye, D. Olmos

Research output: Contribution to journalArticlepeer-review


Background: Although sarcomas account for only 1% of all solid tumours, patients with sarcomas comprise a larger proportion of patients entering phase I trials, due to the limited number of registered or active drugs for these diseases. To help in patient selection, we evaluated the utility of the predictive Royal Marsden Score which had been derived in carcinoma patients. In addition, we analysed efficacy and toxicity regarding the sarcoma population enrolled in phase I trials. Patients and methods: We used data from a European Database comprising 2182 patients treated in phase I trials in 14 European institutions between 2005 and 2007. Results: One hundred and seventy-eight patients diagnosed with advanced sarcoma or other mesenchymal tumours were identified and accounted for 217 phase I trial participations during the study period. Histological type, class of drug, number of metastatic sites, high serum lactate dehydrogenase activity (LDH), low albumin and high white blood cell count were independent prognostic factors. Poor performance status (PS), liver metastases and high leucocyte count were associated with increased risk of early death. The class of drug used was the strongest predictor of progression-free survival (PFS) duration, inhibitors of angiogenesis and histone deacetylase giving the best results. Poor PS, high serum LDH and low lymphocyte count correlated with shorter PFS. In this heterogeneous population, PFS with investigational agents appeared comparable with that previously published for patients receiving standard treatments beyond first line. Conclusion: Prognostic factors in sarcoma patients do not differ from a broader phase I population. Efficacy measures suggest that some patients with sarcoma derive benefit from therapy in this setting which could therefore be considered for patients with no remaining standard therapeutic option.

Original languageEnglish
Pages (from-to)1222-1228
Number of pages7
JournalAnnals of Oncology
Issue number6
Publication statusPublished - 2014


  • Clinical trial
  • Phase I
  • Sarcoma
  • Targeted therapies

ASJC Scopus subject areas

  • Oncology
  • Hematology


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