Treatment response summary
| . | All patients (N = 27) . |
|---|---|
| Response at week 32* | |
| Composite response† | 1 (8) |
| Hematocrit control‡ | 9 (56) |
| Spleen volume reduction > 35% at any time point§ | 7 (33) |
| Hematocrit control | |
| At C3D28¶ | 19 (73) |
| At C5D28ǁ | 15 (68) |
| CHR at week 32‡ | 8 (50) |
| CHR at any time point | 14 (52) |
| ELN response at week 32‡ | |
| CR | 3 (19) |
| PR | 8 (50) |
| PD | 0 |
| No response | 5 (31) |
| ELN response (CR or PR) at any time point | 21 (78) |
| BM histologic remission at week 32# | 2 (13) |
| Median TSS reduction from baseline at week 32 (IQR)** | −25.4 (−62.5 to −5.1) |
| TSS reduction ≥ 50% from baseline at week 32** | 6 (43) |
| TSS reduction ≥ 50% from baseline at any time point†† | 12 (48) |
| . | All patients (N = 27) . |
|---|---|
| Response at week 32* | |
| Composite response† | 1 (8) |
| Hematocrit control‡ | 9 (56) |
| Spleen volume reduction > 35% at any time point§ | 7 (33) |
| Hematocrit control | |
| At C3D28¶ | 19 (73) |
| At C5D28ǁ | 15 (68) |
| CHR at week 32‡ | 8 (50) |
| CHR at any time point | 14 (52) |
| ELN response at week 32‡ | |
| CR | 3 (19) |
| PR | 8 (50) |
| PD | 0 |
| No response | 5 (31) |
| ELN response (CR or PR) at any time point | 21 (78) |
| BM histologic remission at week 32# | 2 (13) |
| Median TSS reduction from baseline at week 32 (IQR)** | −25.4 (−62.5 to −5.1) |
| TSS reduction ≥ 50% from baseline at week 32** | 6 (43) |
| TSS reduction ≥ 50% from baseline at any time point†† | 12 (48) |
Unless otherwise noted, data are n (%).
CR, complete remission; PD, progressive disease; PR, partial remission; TSS, Total Symptom Score.
The response-evaluable population included 16 patients who had undergone response assessment at week 32 or had withdrawn prior to week 32 because of a lack of response or PD. Composite response was the primary end point at week 32 in patients with splenomegaly. For all other patients, the primary end point was hematocrit control at week 32. Hematocrit control was defined as ≤1 phlebotomy between the start of the study and week 8 and no phlebotomies after week 8. Protocol-defined indications for phlebotomy were hematocrit > 45%, which was 3% higher than the screening value, or hematocrit > 48%, regardless of screening value.
Evaluable patients, n = 13.
Evaluable patients, n = 16.
Evaluable patients, n = 21.
Evaluable patients, n = 26.
Evaluable patients, n = 22.
Evaluable patients, n = 15.
Evaluable patients, n = 14.
Evaluable patients, n = 25.