Table 4. Clinical responses.
| Patient group | Splenic reduction ⩾50%, n/N (%)a | Median (range) cycles to splenic reduction ⩾50%a | Splenic reduction of 100%, n/N (%)a | Splenic clinical improvement, n/N (%)a,b | Hb/Plt clinical improvement, n/N (%)b,c |
|---|---|---|---|---|---|
| NS-018 dose cohort | |||||
| 75 mg QD | 0/2 (0) | — | — | — | 0/3 (0) |
| 125 mg QD | 0/4 (0) | — | — | — | Plt: 1/4 (25) |
| 200 mg QD | 3/5 (60) | 3 | 1/5 (20) | 1/5 (20) | 0/6 (0) |
| 300 mg QD | 5/9 (56) | 2 (1–8) | 5/9 (56) | 4/9 (44) | Hb: 2/9 (22) |
| 400 mg QD | 3/3 (100) | 1 | 0/3 (0) | 1/3 (33) | 0/3 (0) |
| 100 mg BID | 2/3 (66) | 7.5 (3–12) | 0/3 (0) | 1/3 (33) | Hb: 1/3 (33) |
| 200 mg BID | 1/3 (33) | 1 | 1/3 (33) | 1/3 (33) | 0/4 (0) |
| 250 mg BID | 1/4 (25) | 3 | 1/4 (25) | 0/4 (0) | 0/4 (0) |
| 300 mg BID | 3/6 (50) | 1 | 3/6 (50) | 2/6 (33) | Hb: 1/7 (14) |
| 400 mg BID | 4/6 (67) | 1.5 (1–2) | 1/6 (17) | 4/6 (67) | 0/6 (0) |
| Prior JAK2 inhibitor treatment | 9/19 (47) | 2 (1–12) | 5/19 (26) | 6/19 (32) | Hb: 3/18 (17) Plt: 1/18 (6) |
| All dose cohorts | 20/36 (56) | 1 (1–12) | 11/36 (31) | 13/36 (36) | Hb: 4/40 (10) Plt: 1/40 (3) |
Abbreviations: BID, twice-daily dosing; Hb, hemoglobin; JAK, Janus-activated kinase; N, number of evaluable patients; Plt, platelet; QD, once-daily dosing.
a
Splenic response was assessed in patients who had received ⩾1 cycle of treatment and with baseline splenomegaly ⩾5 cm.
b
All measures of clinical improvement had to be confirmed for ⩾8 weeks.22
c
Hb/Plt response was assessed in patients who had received ⩾1 cycle of treatment.