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  • Open Access

Final planned overall survival (OS) from OPTiM, a randomized Phase III trial of talimogene laherparepvec (T-VEC) versus GM-CSF for the treatment of unresected stage IIIB/C/IV melanoma (NCT00769704)

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Journal for ImmunoTherapy of Cancer20142 (Suppl 3) :P263

  • Published:


  • Melanoma
  • Overall Survival
  • Herpes Simplex
  • Antitumor Immune Response
  • ECOG Performance Status


T-VEC is an oncolytic immunotherapy derived from herpes simplex virus type-1 designed to selectively replicate within tumors and to produce GM-CSF to enhance systemic antitumor immune responses. OPTiM, a randomized Phase III trial of T-VEC vs GM-CSF in patients with unresected melanoma with regional or distant metastases met the primary objective of an improvement in durable response rate (response lasting continuously for ≥6 months) with T-VEC versus GM-CSF (16% vs 2%, respectively; P<0.001). Most common adverse events with T-VEC were fatigue, chills, and pyrexia. No ≥grade 3 adverse events occurred in ≥3% of patients in either arm (Andtbacka et al., J Clin Oncol 2013,32[suppl]:LBA9008). At the primary analysis (PA) of secondary OS endpoint, with median follow-up of 44 (range, 32-59) months and 189 events in the T-VEC arm and 101 events in the GM-CSF arm, median (95%CI) OS was 23.3 (19.5-29.6) months for T-VEC and 18.9 (16.0-23.7) months for GM-CSF (hazard ratio [HR]=0.79; 95%CI = 0.62-1.00; P = 0.051) (Kaufman et al., J Clin Oncol 2014,32[suppl]:9008a). A planned analysis of OS at 3 years from the last randomization is presented here.


Eligible patients were ≥18 years old; had ECOG performance status (PS) ≤1; unresectable melanoma stage IIIB/C/IV; injectable cutaneous, subcutaneous (SC) or nodal lesions; LDH≤1.5X upper limit of normal; ≤3 visceral lesions (excluding lung), none>3 cm. Patients were randomized 2:1 to intralesional T-VEC (initially ≤4 mL x106 pfu/mL, then after 3 wks, ≤4 mL x108 pfu/mL q2w) or SC GM-CSF (125 µg/m2qd × 14 ds q4w).


Of 436 patients in the intent-to-treat analysis, 295 (68%) patients received T-VEC and 141 (32%) patients received GM-CSF; 57% were men; median age 63 yrs. At time of the final OS analysis with median follow-up of 49 months [range, 37-63], only 1 additional event occurred (T-VEC arm). Median (95%CI) OS was 23.3 months (95%CI = 19.5-29.6) for T-VEC and 18.9 months (16.0-23.8) for GM-CSF; HR = 0.80 (95%CI = 0.62-1.01), P = 0.06 (descriptive). Five-year survival for the T-VEC arm was 33.4% (95%CI = 27.7-39.2). T-VEC effect on OS was most pronounced in patients with stage IIIB/C/IVM1a melanoma (HR = 0.57; 95%CI = 0.41-0.81, P = 0.001 [descriptive]) and in patients with treatment-naive disease (HR = 0.52; 95%CI = 0.36-0.75, P < 0.001 [descriptive]).


With >4 years of median follow-up for survival, a persistent relevant OS effect was demonstrated with further follow-up. Long-term follow-up continues in the registry trial (NCT02173171). T-VEC represents a novel potential therapy for patients with regionally and distantly metastatic melanoma.

Authors’ Affiliations

Huntsman Cancer Institute, Salt Lake City, UT, USA
The University of Carolina at Chapel Hill, Chapel Hill, NC, USA
Minnesota Oncology, MN, USA
Mary Crowley Cancer Research Center, TX, USA
University of Louisville, Louisville, KY, USA
Emory University, Atlanta, GA, USA
Northern California Melanoma Center, San Francisco, CA, USA
Vanderbilt University Medical Center, Nashville, TN, USA
St. Luke's University Hospital & Health Network, Bethlehem, PA, USA
University of Iowa, Iowa City, IA, USA
The Institute of Cancer Research/The Royal Marsden Hospital, London, UK
National Institute for Health Research Biomedical Research Centre, London, UK
Amgen Inc, Thousand Oaks, CA, USA
Amgen, Woburn, MA, USA
Rutgers Cancer Institute of New Jersey, New Brunswick, NJ, USA


© I. Andtbacka et al.; licensee BioMed Central Ltd. 2014

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