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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