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


CONCLUSIONS
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.
Royal Marsden Hospital, London, SW3 6JJ, UK. rjones@seattlecca.org
Robin L
RL
Jones
David
D
Olmos
Khin
K
Thway
Cyril
C
Fisher
Nina
N
Tunariu
Sophie
S
Postel-Vinay
Michelle
M
Scurr
Johann
J
de Bono
Stan B
SB
Kaye
Ian R
IR
Judson
D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000009:adverse effects / Q000627:therapeutic use*; D054316:Biomarkers, Pharmacological / Q000097:blood; D002648:Child; D017321:Clinical Trials, Phase I as Topic; D005260:Female; D005500:Follow-Up Studies; D006779:Hospitals, Public; D006801:Humans; D008131:London; D008297:Male; D008875:Middle Aged; D009367:Neoplasm Staging; D010352:Patient Dropouts / Q000706:statistics & numerical data; D011379:Prognosis; D012189:Retrospective Studies; D012509:Sarcoma / Q000097:blood / Q000175:diagnosis / Q000188:drug therapy* / Q000473:pathology; D016019:Survival Analysis; D055815:Young Adult
D000970:Antineoplastic Agents; D054316:Biomarkers, Pharmacological