The DOI matches its Crossref record; 20 of the 20 references it cites were found in a registry or an index.
Risks and Benefits of Phase 1 Oncology Trials, 1991 through 2002
Open accessAbstract
BACKGROUND: Previous reviews of phase 1 oncology trials reported a rate of response to treatment of 4 to 6 percent and a toxicity-related death rate of 0.5 percent. These results may not reflect the rates in current phase 1 oncology trials. METHODS: We reviewed all nonpediatric phase 1 oncology trials sponsored by the Cancer Therapy Evaluation Program at the National Cancer Institute between 1991 and 2002. We report the rates of response to treatment, of stable disease, of grade 4 toxic events, and of treatment-related deaths. RESULTS: We analyzed 460 trials involving 11,935 participants, all of whom were assessed for toxicity and 10,402 of whom were assessed for a response to therapy. The overall response rate (i.e., for both complete and partial responses) was 10.6 percent, with considerable variation among trials. "Classic" phase 1 trials of single investigational chemotherapeutic agents represented only 20 percent of the trials and had a response rate of 4.4 percent. Studies that included at least one anticancer agent approved by the Food and Drug Administration constituted 46.3 percent of the trials and had a response rate of 17.8. An additional 34.1 percent of participants had stable disease or a less-than-partial response. The overall rate of death due to toxic events was 0.49 percent. Of 3465 participants for whom data on patient-specific grade 4 toxic events were available, 14.3 percent had had at least one episode of grade 4 toxic events. CONCLUSIONS: Overall response rates among phase 1 oncology trials are higher than previously reported, although they have not changed for classic phase 1 trials, and toxicity-related death rates have remained stable. Rates of response and toxicity vary, however, among the various types of phase 1 oncology trials.
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Cited by
Le Tourneau, C., Delord, J.-P., Gonçalves, A., Gavoille, C., Dubot, C., Isambert, N., Campone, M., Trédan, O., Massiani, M.-A., Mauborgne, C., et al. (2015) · The Lancet Oncology · doi:10.1016/s1470-2045(15)00188-6 · Cited by 1,137
Dose Escalation Methods in Phase I Cancer Clinical Trials
Le Tourneau, C., Lee, J. J., & Siu, L. L. (2009) · JNCI: Journal of the National Cancer Institute · doi:10.1093/jnci/djp079 · Cited by 867
Clinical trial results with oncolytic virotherapy: a century of promise, a decade of progress
Liu, T.-C., Galanis, E., & Kirn, D. (2007) · Nature Clinical Practice Oncology · doi:10.1038/ncponc0736 · Cited by 450
Janku, F., Wheler, J. J., Westin, S. N., Moulder, S. L., Naing, A., Tsimberidou, A. M., Fu, S., Falchook, G. S., Hong, D. S., Garrido-Laguna, I., et al. (2012) · Journal of Clinical Oncology · doi:10.1200/jco.2011.36.1196 · Cited by 447
Hidalgo, M., Bruckheimer, E., Rajeshkumar, N. V., Garrido-Laguna, I., De Oliveira, E., Rubio-Viqueira, B., Strawn, S., Wick, M. J., Martell, J., & Sidransky, D. (2011) · Molecular Cancer Therapeutics · doi:10.1158/1535-7163.mct-11-0233 · Cited by 406
Metadata
- Authors
- Horstmann, E., McCabe, M. S., Grochow, L., Yamamoto, S., Rubinstein, L., Budd, T., Shoemaker, D., Emanuel, E. J., & Grady, C.
- Venue
- New England Journal of Medicine
- Publisher
- Massachusetts Medical Society
- Published
- 2005-03-03
- Volume
- 352
- Issue
- 9
- Pages
- 895-904
- ISSN
- 0028-4793 (ISSN-L)
- 1533-4406
- OpenAlex
- W2149626936
- Cited by
- 441
- References
- 20
- FWCI
- 24.34
- Type
- journal article
- Language
- en
- Open access
- bronze
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