Table 1.
Baseline characteristics of patients
| Characteristics | Overall population (N = 10) |
|---|---|
| Median age (range), y | 72.5 (40-84) |
| Female sex, n (%) | 6 (60) |
| ECOG performance status, n (%) | |
| 0-1 | 7 (70) |
| 2-3 | 3 (30) |
| Median number of prior therapies (range), n | 0.5 (0-2) |
| Number of prior therapies per patient, n (%) | |
| 0 | 5 (50) |
| 1 | 4 (40) |
| 2 | 1 (10) |
| Type of prior therapy, n (%) | |
| Splenectomy | 3 (30) |
| Cladribine | 3 (30) |
| Midostaurin | 1 (10) |
| Imatinib | 1 (10) |
| Decitabine* | 1 (10) |
| Clinicopathologic diagnosis, n (%) | |
| ASM | 3 (30) |
| SM-AHN | 5 (50) |
| CMML-1 | 3 (30) |
| MDS/MPN-U | 1 (10) |
| CEL, NOS | 1 (10) |
| MCL | 2 (20) |
| CMML-1 | 1 (10) |
| Mutation status, n (%) | |
| KIT D816V mutation | 10 (100) |
| Median number of IWG-MRT-ECNM organ damage findings† (range), n | 1 (1-3) |
| Number of IWG-MRT-ECNM organ damage findings per patient, n (%) | |
| 1 | 6 (60) |
| 2 | 2 (20) |
| 3 | 2 (20) |
| Types of IWG-MRT-ECNM organ damage findings, n (%) | |
| Elevated alkaline phosphatase | 5 (50) |
| Symptomatic splenomegaly | 3 (30) |
| Anemia | 4 (40) |
| Transfusion-dependent | 2 (20) |
| Transfusion-independent | 2 (20) |
| Thrombocytopenia | 4 (40) |
| Transfusion-dependent | 2 (20) |
| Transfusion-independent | 2 (20) |
| Neutropenia | 1 (10) |
| Ascites | 1 (10) |
| Median serum tryptase (range), ng/mL | 295 (81-903) |
| Median bone marrow core biopsy mast cell burden‡ (range), % involvement | 55 (15-60) |
| Median bone marrow mast cell CD30+ expression¶ (range), % | 70 (29-96) |
*
AHN-directed therapy for MDS/MPN-U.
†
For the full listing and definitions of eligible organ damage criteria, please see the IWG-MRT-ECNM consensus guidelines.29
‡
Measured via tryptase, CD117, and CD25 immunohistochemistry staining on the core biopsy specimen.
¶
Measured via multiparametric FCM methods using anti-BerH83 antibody on aspirate specimen.
CEL, chronic eosinophilic leukemia, not otherwise specified; CMML-1, chronic myelomonocytic leukemia, <10% blasts; ECOG, Eastern Cooperative Oncology Group; KIT, CD117, c-Kit receptor; MCL, mast cell leukemia; MDS/MPN-U, myelodysplastic/myeloproliferative neoplasm, unclassifiable; MPN-SAF, Myeloproliferative Neoplasm Symptom Assessment Form.