Table 1 Baseline characteristics
Safety population (n = 62) | Interim analysis efficacy population (n = 32) | |
|---|---|---|
Median age, years (range) | 69 (31–88) | 68 (37–85) |
Male/female, n (%) | 34 (55)/28 (45) | 18 (56)/14 (44) |
ECOG performance status, n (%) | ||
0–1 | 43 (69) | 21 (66) |
2–3 | 19 (31) | 11 (34) |
AdvSM subtype as per central assessment, n (%) | ||
ASM | 9 (15) | 2 (6) |
SM-AHN | 43 (69) | 26 (81) |
MCL | 10 (16) | 4 (13) |
KIT D816V mutation status in peripheral blood by central ddPCR, n (%) | ||
Positive | 59 (95) | 30 (94) |
Negative | 3 (5) | 2 (6) |
KIT D816V variant allele fraction in blood, median percentage (range) | 18 (0–47) | 15 (0–45) |
SRSF2/ASXL1/RUNX1 mutation as per central assay, n (%) | ||
Positive | 26 (42) | 17 (53) |
Negative | 36 (58) | 15 (47) |
Previous antineoplastic therapy, n (%) | ||
Any | 42 (68) | 23 (72) |
Midostaurin | 34 (55) | 17 (53) |
Cladribine | 8 (13) | 4 (13) |
Imatinib | 5 (8) | 4 (13) |
Interferon | 6 (10) | 2 (6) |
Bone marrow biopsy mast cell burden median percentage (range) | 45 (1–95) | 50 (10–95) |
Serum tryptase level, median ng ml–1 (range) | 283 (24–1,600) | 293 (24–1,600) |
Spleen volume, median ml (range) | 748 (44–2,601) | 939 (150–2,270) |
