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Table 1 Efficacy outcomes in final analysis data set of OPTiM

From: Final analyses of OPTiM: a randomized phase III trial of talimogene laherparepvec versus granulocyte-macrophage colony-stimulating factor in unresectable stage III–IV melanoma

  Talimogene laherparepvec (n = 295) GM-CSF (n = 141) Descriptive P-valuea Difference
% 95% CIb
Response per investigator assessment in the intent-to-treat population (Stage IIIB–IVM1c disease)
 DRR, n (%) 57 (19.3) 2 (1.4) < 0.0001 17.9 12.0–23.1
 CR, n (%) 50 (16.9) 1 (0.7)
 PR, n (%) 43 (14.6) 8 (5.7)
 ORR, % (95% CI)b 31.5 (26.3–37.2) 6.4 (3.0–11.8) < 0.0001 25.1 17.4–31.7
 SD, n (%) 132 (44.7) 71 (50.4)
 DCR, n (%) 225 (76.3) 80 (56.7) 19.5 9.7–29.3
 Progressive disease, n (%) 62 (21.0) 42 (29.8)
 Not assessed, n (%) 8 (2.7) 19 (13.5)
Estimated OS probability in the intent-to-treat population (Stage IIIB–IVM1c disease), % (95% CI)
 At 12 months 73.7 (68.3–78.4) 69.1 (60.6–76.2) 4.6 −4.7–13.8
 At 24 months 49.8 (44.0–55.4) 40.3 (32.0–48.4) 9.5 −0.5–19.6
 At 36 months 38.9 (33.3–44.4) 30.4 (22.9–38.3) 8.4 −1.2–18.0
 At 48 months 34.5 (28.9–40.1) 23.9 (16.8–31.7) 10.6 1.2–20.0
 At 60 months 33.4 (27.7–39.2) NE NE NE
DRR, ORR, CR and DCR per investigator assessment according to disease stage
 DRR, n/N (%)
  IIIB/C 29/88 (33.0) 0/43 (0) < 0.0001 33.0 19.1–43.9
  IVM1a 18/75 (24.0) 0/43 (0) 0.0003 24.0 10.5–35.5
  IIIB–IVM1a 47/163 (28.8) 0/86 (0) < 0.0001 28.8 20.3–36.5
  IVM1b 4/64 (6.3) 1/26 (3.8) 1.0000 2.4 −15.8–12.8
  IVM1c 6/67 (9.0) 1/29 (3.4) 0.6710 5.5 −11.5–16.2
 ORR, n/N (%)
  IIIB/C 46/88 (52.3) 2/43 (4.7) < 0.0001 47.6 31.1–59.0
  IVM1a 29/75 (38.7) 2/43 (4.7) < 0.0001 34.0 17.6–46.6
  IIIB–IVM1a 75/163 (46.0) 4/86 (4.7) < 0.0001 41.4 30.6–49.9
  IVM1b 9/64 (14.1) 2/26 (7.7) 0.5002 6.4 −13.8–19.5
  IVM1c 9/67 (13.4) 3/29 (10.3) 1.0000 3.1 −16.3–16.5
 CR, n/N (%)
  IIIB/C 31/88 (35.2) 0/43 (0)
  IVM1a 15/75 (20.0) 1/43 (2.3)
  IIIB–IVM1a 46/163 (28.2) 1/86 (1.2)
  IVM1b 2/64 (3.1) 0/26 (0)
  IVM1c 2/67 (3.0) 0/29 (0)
 PR, n/N (%)
  IIIB/C 15/88 (17.0) 2/43 (4.7)
  IVM1a 14/75 (18.9) 1/43 (2.3)
  IIIB–IVM1a 29/163 (17.8) 3/86 (3.5)
  IVM1b 7/64 (10.9) 2/26 (7.7)
  IVM1c 7/67 (10.4) 3/29 (10.3)
 DCR, n/N (%)
  IIIB/C 75/88 (85.2) 23/43 (53.5) 31.7 13.9–48.6
  IVM1a 54/75 (72.0) 24/43 (55.8) 16.2 −2.7–34.6
  IIIB–IVM1a 129/163 (79.1) 47/86 (54.7) 24.5 11.5–37.0
  IVM1b 50/64 (78.1) 16/26 (61.5) 16.6 −4.9–39.3
  IVM1c 46/67 (68.7) 17/29 (58.6) 10.0 −11.4–32.2
  1. aP-values calculated using Fisher’s Exact Test
  2. bThe Clopper-Pearson method was used to calculate exact CIs for binary endpoints. Wilson’s score method with continuity correction was used to calculate an approximate CI for between-group differences in binary rates
  3. CI confidence interval, CR complete response, DCR disease control rate, DRR durable response rate, GM-CSF granulocyte-macrophage colony-stimulating factor, NE not estimable, ORR overall response rate, OS overall survival, PR partial response