Table 2 Primary endpoint and secondary efficacy endpoints within 8 weeks

Primary endpoint

 n (%)

99 (58.9)

110 (64.3)

5 (5.9)

 OR (95% CI; p)^*,†

25.97 (9.83–68.63; < 0.0001)

32.81 (12.39–86.87; < 0.0001)

–

Secondary efficacy end points within 8 weeks

Proportion of patients who responded at least once

 n (%)

142 (84.5)

148 (86.5)

19 (22.4)

 OR (95% CI; p)^*,†

24.11 (11.76–49.40; < 0.0001)

27.83 (13.49–57.40; < 0.0001)

–

Proportion of patients who responded at ≥ 75% of assessments

 n (%)

55 (32.7)

98 (57.3)

2 (2.4)

 OR (95% CI; p)^*,†

24.63 (5.71–106.21; < 0.0001)

79.65 (18.36–345.53; < 0.0001)

–

Proportion of patients achieving platelet counts ≥ 30 × 10⁹/L at least once that were at least twice their baseline platelet counts

 n (%)

147 (87.5)

155 (90.6)

24 (28.2)

 OR (95% CI; p)^*,†

18.01 (9.31–34.85; < 0.0001)

24.93 (12.37–50.24, < 0.0001)

–

Maximum continuous duration of response

n^#

125

138

6

 Median (range), days

22.0 (6.0–53.0)

22.0 (6.0–54.0)

8.5 (7.0–42.0)

Total duration of response

n^#

125

138

6

 Median (range), days

23.0 (6.0–53.0)

33.0 (6.0–54.0)

8.5 (7.0–42.0)

Proportion of patients required rescue therapy

 n (%; 95% CI)^&

22 (13.1; 8.4–19.2)

17 (9.9; 5.9–15.4)

32 (37.6; 27.4–48.8)

 p value^¶

 < 0.0001

 < 0.0001

–

Bleeding (WHO bleeding scale), n (%)^§

 Yes

107 (64.1)

97 (56.7)

67 (78.8)

 Grade 1

96 (57.5)

87 (50.9)

50 (58.8)

 Grade 2

8 (4.8)

10 (5.8)

16 (18.8)

 Grade 3

3 (1.8)

0

1 (1.2)

 Grade 4

0

0

0

 No

60 (35.9)

74 (43.3)

18 (21.2)

 p value for present of bleed symptoms^¶

0.0208

0.0005

–