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Clinical benefit of early phase clinical trial participation for advanced sarcoma patients

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Abstract

Purpose

Standard systemic treatment options for patients with advanced sarcoma are limited. Depending on the histological subtype, patients receive differing lines of therapy usually consisting of doxorubicin, ifosfamide and/or trabectedin. After progression on conventional therapies, some patients are offered more experimental options including Phase I clinical trials. The aim of this study was to evaluate the clinical benefit for sarcoma patients treated within the Phase I Unit of a single referral centre.

Methods

The response, toxicity and outcome of sarcoma patients treated within Phase I clinical trials at the Royal Marsden between August 1998 and December 2010 were analysed.

Results

One hundred and thirty-three patients were treated. The median number of prior systemic therapies was 3 (range 0–6). The median age of these patients was 48.0 years (range 12.5–81.9), with a male/female ratio of 71/62. One patient (0.8%) achieved a complete response and 2 (1.6%) partial responses. The non-progression rate at 3 and 6 months was 31.5% (95% CI, 23.4–39.6%) and 11.0% (95% CI 5.6–16.5%), respectively. The median progression-free survival was 2.1 months (95% CI, 1.7–2.5), and median overall survival was 7.6 months (95% CI, 4.8–10.4). Twenty-four (18.0%) patients experienced grade 3 or 4 toxicity, and 16 (12.0%) stopped trial treatment due to toxicity.

Conclusion

Phase I clinical trials could be considered a therapeutic option in sarcoma patients with no remaining standard treatment due to the low risk of toxicity and the potential for clinical benefit.

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Acknowledgments

Dr Robin Jones is supported by the Bob and Eileen Gilman Family Sarcoma Research Program. David Olmos is supported by a research fellowship from the Spanish Society of Medical Oncology (SEOM).

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Authors and Affiliations

  1. Sarcoma Unit, Royal Marsden Hospital, Fulham Road, London, SW3 6JJ, UK

    Robin L. Jones, David Olmos, Khin Thway, Cyril Fisher, Sophie Postel-Vinay, Michelle Scurr & Ian R. Judson

  2. Drug Development Unit, Royal Marsden Hospital, Downs Road, Sutton, Surrey, SM2 5PT, UK

    Robin L. Jones, David Olmos, Sophie Postel-Vinay, Michelle Scurr, Johann de Bono, Stan B. Kaye & Ian R. Judson

  3. Radiology Department, Royal Marsden Hospital, Downs Road, Sutton, Surrey, SM2 5PT, UK

    Nina Tunariu

  4. University of Washington/Fred Hutchinson Cancer Research Center, 825 Eastlake Avenue E, G3630, Seattle, WA, 98109-1023, USA

    Robin L. Jones

Authors
  1. Robin L. Jones
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  2. David Olmos
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  3. Khin Thway
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  4. Cyril Fisher
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  5. Nina Tunariu
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  6. Sophie Postel-Vinay
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  7. Michelle Scurr
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  8. Johann de Bono
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  9. Stan B. Kaye
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  10. Ian R. Judson
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Corresponding author

Correspondence to Robin L. Jones.

Additional information

R. L. Jones and D. Olmos have equally contributed to this work.

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Jones, R.L., Olmos, D., Thway, K. et al. Clinical benefit of early phase clinical trial participation for advanced sarcoma patients. Cancer Chemother Pharmacol 68, 423–429 (2011). https://doi.org/10.1007/s00280-010-1484-9

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  • DOI: https://doi.org/10.1007/s00280-010-1484-9

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