Treatment outcome and survival in participants of phase I oncology trials carried out from 2003 to 2006 at Institut Gustave Roussy

• Published in: Annals of oncology Vol. 19; no. 4; pp. 787 - 792
• Main Authors: Italiano, A., Massard, C., Bahleda, R., Vataire, A.-L., Deutsch, E., Magné, N., Pignon, J.-P., Vassal, G., Armand, J.-P., Soria, J.-C.
• Format: Journal Article
• Language: English
• Published: Oxford Oxford University Press 01.04.2008; Oxford Publishing Limited (England)
• Subjects: Adult; Aged; Aged, 80 and over; anticancer drugs; Antineoplastic agents; Antineoplastic Agents - adverse effects; Antineoplastic Agents - pharmacokinetics; Antineoplastic Agents - pharmacology; Antineoplastic Agents - therapeutic use; Antineoplastic Agents, Hormonal - therapeutic use; Biological and medical sciences; Clinical Trials, Phase I as Topic; Disease-Free Survival; early clinical trials; Female; France - epidemiology; Humans; Immunotherapy; Kaplan-Meier Estimate; Male; Medical sciences; Middle Aged; Multivariate Analysis; Neoplasms - drug therapy; Neoplasms - immunology; Neoplasms - mortality; Neoplasms - radiotherapy; Neoplasms - therapy; oncology; Pharmacology. Drug treatments; phase I trials; targeted therapies; Treatment Outcome; France; phase I trials; targeted therapies; anticancer drugs; early clinical trials; oncology; Antineoplastic agent; Human; Drug; Prognosis; Targeted therapy; Malignant tumor; Survival; Cancerology; Treatment; Phase I trial; Clinical trial; Early; Cancer
• ISSN: 0923-7534; 1569-8041
• DOI: 10.1093/annonc/mdm548

Background: The oncology community usually perceives phase I oncology trials as associated with poor or limited benefits and substantial risks. There is scarce data concerning outcome and survival of patients enrolled in current phase I oncology trials. Patients and methods: We reviewed all phase I oncology trials conducted by investigators from the Adult Phase I Unit at Institut Gustave Roussy from 2003 to 2006. We report data concerning patient demographics, treatment outcome, toxicity, survival and type of care after trial exit. Results: We analyzed 10 trials involving 180 participants. The overall response rate was 7.2%. Disease control (objective response plus stable disease) was achieved in 48.2% of patients. The rate of toxic death was 0.5%. In all, 38% of patients had at least one episode of grade 3 or 4 toxic events. The median progression-free survival and the median overall survival (OS) were 2.3 and 8.7 months, respectively. On multivariate analysis, a time between diagnosis of disease and inclusion in the phase I trial ≥24 months and evidence of disease control were statistically significant predictors of improved OS. Conclusion: Current phase I oncology trials are safe and are associated with clinical benefit in a substantial proportion of patients.