Table 1.
Clinical, laboratory, outcome and treatment characteristics of patients with indolent systemic mastocytosis (ISM) and advanced SM (AdvSM).
| Variables | ISM | AdvSM | p-Value |
|---|---|---|---|
| Number of patients (n) | 40 | 121 | - |
| Age in years, median (range) | 54 (29–83) | 76 (30–90) | <0.0001 |
| Male, n (%) | 17 (43) | 81 (67) | 0.006 |
| Hemoglobin, g/dL; median (range) | 13.9 (11.7–16.8) | 10.8 (5.8–15.8) | <0.0001 |
| Platelets, ×109/L; median (range) | 283 (87–461) | 114 (12–958) | 0.0002 |
| MC-infiltration in BM histology, % | not applicable | 30 (0–100) | - |
| Serum tryptase, µg/L; median (range) | 46 (8–166) | 180 (11–1382) | <0.0001 |
| Alkaline phosphatase, U/L; median (range) | 76 (15–166) | 200 (33–1279) | <0.0001 |
| Diagnosis | |||
| ASM, n (%) | - | 18 (15) | - |
| MCL, n (%) | - | 2 (2) | - |
| SM/MCL-AHN, n (%) | - | 101 (83) | - |
| Progression to | |||
| Secondary AML, n (%) | - | 11 (61) | - |
| Secondary MCL, n (%) | - | 7 (39) | - |
| Outcome | |||
| Follow-up, years, median (range) | 5 (0–21) | 3 (0–25) | n.s. |
| Death, n (%) | 0 (100) | 60 (50) | <0.0001 |
| Overall survival, median, years | not reached | 4.8 | <0.0001 |
| Treatment | |||
| Midostaurin, n (%) | 1 (3) | 57 (47) | <0.0001 |
AHN, associated hematological neoplasm; AML, acute myeloid leukemia; ASM, aggressive systemic mastocytosis; BM, bone marrow; MCL, mast cell leukemia; n, number.