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


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 > or =24 months and evidence of disease control were statistically significant predictors of improved OS.


CONCLUSIONS
Current phase I oncology trials are safe and are associated with clinical benefit in a substantial proportion of patients.
Department of Medicine, Institut Gustave Roussy, 39 rue Camille Desmoulins, 94805 Villejuif Cedex, France.
A
A
Italiano
C
C
Massard
R
R
Bahleda
A-L
AL
Vataire
E
E
Deutsch
N
N
Magné
J-P
JP
Pignon
G
G
Vassal
J-P
JP
Armand
J-C
JC
Soria
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000009:adverse effects / Q000493:pharmacokinetics / Q000494:pharmacology* / Q000627:therapeutic use*; D018931:Antineoplastic Agents, Hormonal / Q000627:therapeutic use; D017321:Clinical Trials, Phase I as Topic; D018572:Disease-Free Survival; D005260:Female; D005602:France / Q000453:epidemiology; D006801:Humans; D007167:Immunotherapy; D053208:Kaplan-Meier Estimate; D008297:Male; D008875:Middle Aged; D015999:Multivariate Analysis; D009369:Neoplasms / Q000188:drug therapy / Q000276:immunology / Q000401:mortality* / Q000532:radiotherapy / Q000628:therapy*; D016896:Treatment Outcome
D000970:Antineoplastic Agents; D018931:Antineoplastic Agents, Hormonal