Patients with advanced head and neck cancers have similar progression-free survival on phase I trials and their last food and drug administration-approved treatment.
OBJECTIVE
To compare clinical outcomes of metastatic head and neck cancer patients treated in phase I clinical trials with clinical outcomes of those patients who had their last Food and Drug Administration (FDA)-approved therapy in the setting of metastatic disease.


METHODS
We retrospectively reviewed the outcomes of 61 consecutive patients with head and neck tumors treated in 36 phase I trials at The University of Texas M.D. Anderson Cancer Center between July 2004 and September 2009.


RESULTS
The most common histology was head and neck squamous cell carcinoma (62%). Median age was 55 years (range, 26-80). Eastern Cooperative Oncology Group performance status was 0 to 1 for 95% of patients. Fifty-nine patients had received FDA-approved drugs as the backbone of their last systemic therapy before inclusion in phase I trials (median, 2 systemic therapies). Progression-free survival (PFS) on phase I trials was not inferior to PFS on their last FDA-approved therapies (12 versus 10.7 weeks, log-rank P = 0.87). Fifty-three patients were evaluable for response by Response Evaluation Criteria in Solid Tumors criteria. Four (7%) had partial responses and 16 (26%) had stable disease for > or =4 months. In univariate analysis, number of metastatic sites, lactate dehydrogenase (LDH) levels at baseline, and Royal Marsden Hospital prognosis scores were significant predictors of survival. Only LDH was significant in multivariate analysis (hazard ratio, 6.35; P < or = 0.0001).


CONCLUSIONS
For patients with heavily pretreated advanced head and neck tumors, PFS on phase I trials is not inferior to PFS with their last FDA-approved therapy. The only significant predictor of survival in the multivariate analysis was baseline LDH.
Garrido-Laguna
Ignacio
I
Department of Investigational Cancer Therapeutics (Phase I Clinical Trials Program), University of Texas M.D. Anderson Cancer Center, 1515Holcombe Boulevard, Houston, TX 77030, USA. igarrido@mdanderson.org
Janku
Filip
F
Falchook
Gerald S
GS
Fu
Siqing
S
Hong
David S
DS
Naing
Aung
A
Aaron
Joanne
J
Wang
Xuemei
X
Kies
Merrill
M
Kurzrock
Razelle
R
D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000627:therapeutic use*; D017321:Clinical Trials, Phase I as Topic; D018572:Disease-Free Survival; D006258:Head and Neck Neoplasms / Q000188:drug therapy* / Q000401:mortality* / Q000473:pathology; D006801:Humans; D008875:Middle Aged; D012189:Retrospective Studies
D000970:Antineoplastic Agents