Clinical, laboratory, and treatment characteristics and outcome of 35 patients with advSM, stratified by absence (S/A/Rneg) or presence (S/A/Rpos) of mutations in the SRSF2/ASXL1/RUNX1 (S/A/R) gene panel
| Variables . | All patients . | S/A/Rneg . | S/A/Rpos . | P* . |
|---|---|---|---|---|
| No. of patients | 35 | 12 | 23 | |
| Age in y, median (range) | 67 (48-76) | 64 (48-76) | 68 (55-75) | NS |
| Males, n (%) | 24 (69) | 5 (42) | 19 (83) | .02 |
| Diagnosis | ||||
| ASM, n (%) | 4 (6) | 4 (33) | 0 | NS |
| MCL, n (%) | 4 (11) | 3 (25) | 1 (4) | NS |
| SM-AHN | 27 (83) | 5 (42) | 22 (96) | .01 |
| SM, n (%) | 20 (57) | 4 (80) | 16 (73) | NS |
| MCL, n (%) | 7 (26) | 1 (20) | 6 (27) | NS |
| Disease-related parameters | ||||
| Hemoglobin (G/dL) median (range) | 10 (6-15) | 11 (7-15) | 9 (6-14) | .04 |
| <10 g/dL, n (%) | 21 (60) | 4 (33) | 17 (74) | .03 |
| Platelets (×109/L) median (range) | 97 (7-426) | 113 (30-344) | 94 (7-426) | NS |
| <100 × 109/L, n (%) | 20 (57) | 6 (50) | 14 (61) | NS |
| Hemoglobin <10 g/dL and/or platelets <100 × 109/L, n (%) | 30 (86) | 8 (67) | 22 (96) | .04 |
| MC infiltration in BM (%) median (range) | 50 (15-95) | 40 (15-90) | 50 (20-95) | NS |
| Serum tryptase (µg/D) median (range) | 246 (33-1690) | 214 (65-1690) | 296 (33-1200) | NS |
| >200 µg/L, n (%) | 21 (60) | 7 (58) | 14 (61) | NS |
| KIT D816V+ EAB in PB (%) median (range) | 40 (4-64) | 35 (4-63) | 40 (7-64) | NS |
| >30%, n (%) | 24 (69) | 8 (67) | 16 (70) | NS |
| Albumin (g/L) median (range) | 34 (23-45) | 34 (23-43) | 34 (26-45) | NS |
| <35 g/L, n (%) | 21 (60) | 7 (58) | 14 (61) | NS |
| AP (U/L) median (range) | 287 (61-1067) | 179 (61-479) | 355 (134-1067) | .004 |
| >150 U/L, n (%) | 29 (83) | 8 (67) | 22 (96) | .04 |
| Spleen volume (mL) median (range), n = 28 | 918 (333-2058) | 800 (333-1796) | 919 (338-2058) | NS |
| Marked splenomegaly (≥1200 mL) | 10 (36) | 2 (22) | 8 (42) | NS |
| Treatment-related parameters | ||||
| Time on midostaurin therapy (mo) median (range) | 13 (1-88) | 13 (5-88) | 11 (1-57) | NS |
| ≥6 mo on midostaurin, n (%) | 29 (83) | 11 (92) | 18 (78) | NS |
| Number of patients on midostaurin at time of reporting | 11 (31) | 7 (58) | 4 (17) | .02 |
| Outcome | ||||
| Median OS, mo (95% CI) | 30 (6-54) | NR | 27 (16-38) | .01† |
| Death, n (%) | 20 (57) | 3 (25) | 17 (74) |
| Variables . | All patients . | S/A/Rneg . | S/A/Rpos . | P* . |
|---|---|---|---|---|
| No. of patients | 35 | 12 | 23 | |
| Age in y, median (range) | 67 (48-76) | 64 (48-76) | 68 (55-75) | NS |
| Males, n (%) | 24 (69) | 5 (42) | 19 (83) | .02 |
| Diagnosis | ||||
| ASM, n (%) | 4 (6) | 4 (33) | 0 | NS |
| MCL, n (%) | 4 (11) | 3 (25) | 1 (4) | NS |
| SM-AHN | 27 (83) | 5 (42) | 22 (96) | .01 |
| SM, n (%) | 20 (57) | 4 (80) | 16 (73) | NS |
| MCL, n (%) | 7 (26) | 1 (20) | 6 (27) | NS |
| Disease-related parameters | ||||
| Hemoglobin (G/dL) median (range) | 10 (6-15) | 11 (7-15) | 9 (6-14) | .04 |
| <10 g/dL, n (%) | 21 (60) | 4 (33) | 17 (74) | .03 |
| Platelets (×109/L) median (range) | 97 (7-426) | 113 (30-344) | 94 (7-426) | NS |
| <100 × 109/L, n (%) | 20 (57) | 6 (50) | 14 (61) | NS |
| Hemoglobin <10 g/dL and/or platelets <100 × 109/L, n (%) | 30 (86) | 8 (67) | 22 (96) | .04 |
| MC infiltration in BM (%) median (range) | 50 (15-95) | 40 (15-90) | 50 (20-95) | NS |
| Serum tryptase (µg/D) median (range) | 246 (33-1690) | 214 (65-1690) | 296 (33-1200) | NS |
| >200 µg/L, n (%) | 21 (60) | 7 (58) | 14 (61) | NS |
| KIT D816V+ EAB in PB (%) median (range) | 40 (4-64) | 35 (4-63) | 40 (7-64) | NS |
| >30%, n (%) | 24 (69) | 8 (67) | 16 (70) | NS |
| Albumin (g/L) median (range) | 34 (23-45) | 34 (23-43) | 34 (26-45) | NS |
| <35 g/L, n (%) | 21 (60) | 7 (58) | 14 (61) | NS |
| AP (U/L) median (range) | 287 (61-1067) | 179 (61-479) | 355 (134-1067) | .004 |
| >150 U/L, n (%) | 29 (83) | 8 (67) | 22 (96) | .04 |
| Spleen volume (mL) median (range), n = 28 | 918 (333-2058) | 800 (333-1796) | 919 (338-2058) | NS |
| Marked splenomegaly (≥1200 mL) | 10 (36) | 2 (22) | 8 (42) | NS |
| Treatment-related parameters | ||||
| Time on midostaurin therapy (mo) median (range) | 13 (1-88) | 13 (5-88) | 11 (1-57) | NS |
| ≥6 mo on midostaurin, n (%) | 29 (83) | 11 (92) | 18 (78) | NS |
| Number of patients on midostaurin at time of reporting | 11 (31) | 7 (58) | 4 (17) | .02 |
| Outcome | ||||
| Median OS, mo (95% CI) | 30 (6-54) | NR | 27 (16-38) | .01† |
| Death, n (%) | 20 (57) | 3 (25) | 17 (74) |