Table 2.
Primary endpoint and secondary efficacy endpoints within 8 weeks
| Platelet response | HETROM-2.5, n = 168 | HETROM-5, n = 171 | Placebo, n = 85 |
|---|---|---|---|
| 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 | – |
Response to treatment was defined as a platelet count of ≥ 50 × 109/L. HETROM-2.5, dosage was titrated from an initial dose of once-daily 2.5 mg hetrombopag; HETROM-5, dosage was titrated from an initial dose of once-daily 5 mg hetrombopag; OR, odd ratio; CI, confidence interval
*Hetrombopag versus Placebo
†Logistic regression analysis adjusted for baseline platelet count
&95% CI was calculated using the Clopper–Pearson method
¶p value from Fisher's exact test for comparison between Hetrombopag and Placebo
§HETROM-2.5 group, n = 167; one patient had no bleeding assessment
#Number of patients achieving response to treatment at consecutive scheduled visits