Table 2. Hematologic Response in Patients Treated with Immunosuppression and Eltrombopag*.
| Cohort and Response | Rate at 3 Mo | Rate at 6 Mo | P Value |
|---|---|---|---|
| Cohort 1 | |||
| No. of patients | 30 | 30 | |
| Response — no. (% [95% CI]) | |||
| Overall response | 23 (77 [61–93]) | 24 (80 [65–95]) | |
| Partial response | 18 (60 [41–79]) | 14 (47 [28–66]) | |
| Complete response | 5 (17 [3–31]) | 10 (33 [15–31]) | 0.01 |
| Cohort 2 | |||
| No. of patients | 31 | 31 | |
| Response — no. (% [95% CI]) | |||
| Overall response | 24 (77 [62–93]) | 27 (87 [75–100]) | |
| Partial response | 16 (52 [33–70]) | 19 (61 [43–79]) | |
| Complete response | 8 (26 [9–42]) | 8 (26 [9–42]) | 0.06 |
| Cohort 3 | |||
| No. of patients | 31 | 31 | |
| Response — no. (% [95% CI]) | |||
| Overall response | 27 (87 [75–100]) | 29 (94 [84–103]) | |
| Partial response | 12 (39 [21–57]) | 11 (35 [18–53]) | |
| Complete response | 15 (48 [30–67]) | 18 (58 [40–76]) | <0.001 |
| All cohorts | |||
| No. of patients | 92 | 92 | |
| Response — no. (% [95% CI]) | |||
| Overall response | 74 (80 [72–89]) | 80 (87 [80–94]) | <0.001† |
| Partial response | 46 (50 [40–60]) | 44 (48 [37–58]) | |
| Complete response | 28 (30 [21–40]) | 36 (39 [29–49]) | <0.001 |
Complete response was defined as an absolute neutrophil count of at least 1000 per cubic millimeter, a hemoglobin level of at least 10 g per deciliter, and a platelet count of at least 100,000 per cubic millimeter. Partial response was defined as blood counts no longer meeting the criteria for severe aplastic anemia but not meeting the criteria for complete response. The results for overall response correspond to the proportions of patients with a partial or complete response. Three patients in cohort 1 withdrew from the study before 6 months, as did two patients in cohort 2 and one in cohort 3 (Fig. S1A, S1B, and S1C in the Supplementary Appendix). The P value is for testing the null hypothesis that the rate of complete response at 6 months would be 30% or greater. The 95% confidence intervals were computed on the basis of normal approximation.
The overall response rate in all cohorts was an exploratory end point. The P value is for testing the comparison of the overall response rate to the overall response rate in a historical cohort (67 of 102 patients [66%]).