Table 2.
Efficacy findings.
| PV Cohort |
ET Cohort |
|||||
|---|---|---|---|---|---|---|
| 100 mg (N = 14) | 200 mg (N = 14) | Total (N = 28) | 100 mg (N = 5) | 200 mg (N = 6) | Total (N = 11) | |
| Overall response | ||||||
| n | 14 | 14 | 28 | 5 | 6 | 11 |
| Yes, n (%) | 0 | 2 (14.3) | 2 (7.1) | 0 | 0 | 0 |
| 90% CI | 0–19.3 | 2.6–38.5 | 1.3–20.8 | 0–45.1 | 0–39.3 | 0–23.8 |
| Hematocrit response, ≥ 45% at baseline | ||||||
| n | 7 | 10 | 17 | N/A | N/A | N/A |
| Yes, n (%) | 0 | 1 (10.0) | 1 (5.9) | N/A | N/A | N/A |
| 90% CI | 0–34.8 | 0.5–39.4 | 0.3–25.0 | N/A | N/A | N/A |
| Platelet response, ≥ 400 x 109/L at baseline | ||||||
| n | 7 | 5 | 12 | 5 | 6 | 11 |
| Yes, n (%) | 0 | 1 (20.0) | 1 (8.3) | 0 | 0 | 0 |
| 90% CI | 0–34.8 | 1–65.7 | 0.4–33.9 | 0–45.1 | 0–39.3 | 0–23.8 |
| WBC count response, ≥ 10 x 109/L at baseline | ||||||
| n | 10 | 6 | 16 | 1 | 0 | 1 |
| Yes, n (%) | 0 | 1 (16.7) | 1 (6.3) | 1 (100.0) | 0 | 1 (100.0) |
| 90% CI | 0–25.9 | 0.9–58.2 | 0.3–26.4 | 5–100 | N/A | 5–100 |
| Spleen responsea | ||||||
| n | 4 | 3 | 7 | 0 | 0 | 0 |
| Yes, n (%) | 1 (25.0) | 1 (33.3) | 2 (28.6) | 0 | 0 | 0 |
| 90% CI | 1.3–75.1 | 1.7–86.5 | 5.3–65.9 | N/A | N/A | N/A |
| Response in modified MPN-SAF TSS, n (%) | ||||||
| Yes | 0 | 1 (7.1) | 1 (3.6) | 0 | 1 (16.7) | 1 (9.1) |
| No | 14 (100.0) | 13 (92.9) | 27 (96.4) | 5 (100.0) | 5 (83.3) | 10 (90.9) |
| JAK2V617F allele burden response in patients with JAK2V617F mutation at baseline | ||||||
| Median baseline allele burden | ||||||
| n | 22 | 5 | ||||
| % (range) | 21.8 (1.4, 73.2) | 5.3 (0.3, 10.3) | ||||
| Median change in allele burden at week 24, | ||||||
| n | 13 | 3 | ||||
| % (range) | 99 (49, 172)b | 155 (62, 179)b | ||||
CI, confidence interval; ET, essential thrombocythemia; JAK, Janus kinase; MPN-SAF TSS, Modified Myeloproliferative Neoplasm Symptom Assessment Form Total Symptom Score; N/A, not available; PV, polycythemia vera; WBC, white blood cell.
Overall Response: Patient must meet hematocrit, WBC, platelet, and spleen response criteria in PV cohort; must meet WBC, platelet, and spleen response in ET cohort at some point during the treatment period. These criteria do not have to be met for the same 4 weeks.
Hematocrit response: Hematocrit < 45% in the absence of phlebotomy that lasts ≥ 4 weeks (applicable to PV cohort only).
Platelet response: Platelet count ≤ 400 × 109/L that lasts ≥ 4 weeks.
WBC response: WBC < 10 × 109/L that lasts ≥ 4 weeks.
Spleen response: Resolution or 50% reduction of palpable splenomegaly postbaseline.
MPN-SAF TSS response: Proportion of patients with ≥ 10-point decrease from baseline in modified MPN-SAF TSS that lasted ≥ 12 weeks.
Patients with baseline spleen size ≥ 5 cm are evaluated for spleen response and serve as denominator for spleen response rate calculation.
Not significant by Wilcoxon test.