Baseline demographic, clinical, and biological characteristics of 68 patients with mastocytosis treated by 2-CdA
| Characteristics . | No. of patients (%) . | Median (range) . |
|---|---|---|
| Total no. of patients | 68 | |
| WHO subtypes | ||
| Indolent mastocytosis | 36 (53) | |
| CM/MIS | 6 (9) | |
| ISM | 28 (41) | |
| SSM | 2 (3) | |
| Advanced mastocytosis | 32 (47) | |
| ASM | 14 (21) | |
| SM-AHNMD | 17 (25) | |
| MCL | 1 (1.4) | |
| Female | 35 (51) | |
| Age at treatment’s initiation, y | 54 (17-83) | |
| Time from diagnosis to treatment, mo | 47 (1-473) | |
| Time from appearance of symptoms to 2-CdA’s initiation, mo | 160 (2-473) | |
| Clinical findings | ||
| Mediators release symptoms | ||
| Fatigue | 54 (79) | |
| Flush | 36 (53) | |
| Pruritus | 36 (53) | |
| Diarrhea | 36 (53) | |
| Abdominal pain | 33 (48.5) | |
| Neuropsychiatric symptoms | 28 (41) | |
| Headache/chronic pain | 24 (35) | |
| Nausea, vomiting | 21 (30) | |
| Dyspnea | 14 (20.5) | |
| Mast cells infiltration-related symptoms | ||
| Urticaria pigmentosa | 51 (75) | |
| Bone involvement (osteopenia, osteoporosis, compression fracture, osteosclerosis) | 42 (62) | |
| Hepatomegaly | 35 (51) | |
| Splenomegaly | 33 (48.5) | |
| Weight loss, fever, chills, night sweats (B-symptoms) | 25 (36.7) | |
| Lymphadenopathy | 18 (26) | |
| Ascites | 10 (15) | |
| Biological findings | ||
| Hemoglobin, g/dL | 64 (94) | 12.1 (6.5-15.8) |
| Hb <10 | 18 (28) | 8.9 (6.5-9.7) |
| White blood cell count, ×109/L | 64 (94) | 6.7 (2.1-108) |
| WBC >10 | 10 (16) | 13.1 (10.6-108) |
| Absolute neutrophil count | 63 (93) | 3.8 (0.49-14) |
| ANC <1 | 2 (3) | 0.7 (0.4-0.9) |
| Absolute eosinophil count, ×109/L | 58 (85) | 0.2 (0-99.9) |
| AEC >0.5 | 14 (24) | 1.88 (0.6-99.9) |
| Platelet count, ×109/L | 65 (96) | 215 (16-966) |
| Platelet <100 | 14 (22) | 52 (16-87) |
| Liver parameters (total bilirubin, SAP, AST/ALT > UNL) | 25 (37) | — |
| Albumin, >35-55 g/L | 39 (57) | 43 (35-53) |
| Albumin <35 | 8 (20) | 30 (22.7-32.9) |
| Serum tryptase, ng/mL | 54 (80) | 78 (2.7-1240) |
| Serum tryptase >20 | 47 (87) | 95 (20.2-1240) |
| C-Findings | 26 (38) | |
| KIT D816V mutation detected | 58 (81) | — |
| From bone marrow aspiration in 25 patients | 19 (76) | — |
| From skin biopsy in 49 patients | 39 (80) | — |
| Previous mastocytosis therapies | 34 (50) | |
| Interferon-α | 13 (19) | |
| TKIs (imatinib, masitinib, dasatinib, imatinib then masitinib) | 6/6/1/3 | |
| Thalidomide | 5 (7) | |
| Hydroxyurea | 3 (4) |
| Characteristics . | No. of patients (%) . | Median (range) . |
|---|---|---|
| Total no. of patients | 68 | |
| WHO subtypes | ||
| Indolent mastocytosis | 36 (53) | |
| CM/MIS | 6 (9) | |
| ISM | 28 (41) | |
| SSM | 2 (3) | |
| Advanced mastocytosis | 32 (47) | |
| ASM | 14 (21) | |
| SM-AHNMD | 17 (25) | |
| MCL | 1 (1.4) | |
| Female | 35 (51) | |
| Age at treatment’s initiation, y | 54 (17-83) | |
| Time from diagnosis to treatment, mo | 47 (1-473) | |
| Time from appearance of symptoms to 2-CdA’s initiation, mo | 160 (2-473) | |
| Clinical findings | ||
| Mediators release symptoms | ||
| Fatigue | 54 (79) | |
| Flush | 36 (53) | |
| Pruritus | 36 (53) | |
| Diarrhea | 36 (53) | |
| Abdominal pain | 33 (48.5) | |
| Neuropsychiatric symptoms | 28 (41) | |
| Headache/chronic pain | 24 (35) | |
| Nausea, vomiting | 21 (30) | |
| Dyspnea | 14 (20.5) | |
| Mast cells infiltration-related symptoms | ||
| Urticaria pigmentosa | 51 (75) | |
| Bone involvement (osteopenia, osteoporosis, compression fracture, osteosclerosis) | 42 (62) | |
| Hepatomegaly | 35 (51) | |
| Splenomegaly | 33 (48.5) | |
| Weight loss, fever, chills, night sweats (B-symptoms) | 25 (36.7) | |
| Lymphadenopathy | 18 (26) | |
| Ascites | 10 (15) | |
| Biological findings | ||
| Hemoglobin, g/dL | 64 (94) | 12.1 (6.5-15.8) |
| Hb <10 | 18 (28) | 8.9 (6.5-9.7) |
| White blood cell count, ×109/L | 64 (94) | 6.7 (2.1-108) |
| WBC >10 | 10 (16) | 13.1 (10.6-108) |
| Absolute neutrophil count | 63 (93) | 3.8 (0.49-14) |
| ANC <1 | 2 (3) | 0.7 (0.4-0.9) |
| Absolute eosinophil count, ×109/L | 58 (85) | 0.2 (0-99.9) |
| AEC >0.5 | 14 (24) | 1.88 (0.6-99.9) |
| Platelet count, ×109/L | 65 (96) | 215 (16-966) |
| Platelet <100 | 14 (22) | 52 (16-87) |
| Liver parameters (total bilirubin, SAP, AST/ALT > UNL) | 25 (37) | — |
| Albumin, >35-55 g/L | 39 (57) | 43 (35-53) |
| Albumin <35 | 8 (20) | 30 (22.7-32.9) |
| Serum tryptase, ng/mL | 54 (80) | 78 (2.7-1240) |
| Serum tryptase >20 | 47 (87) | 95 (20.2-1240) |
| C-Findings | 26 (38) | |
| KIT D816V mutation detected | 58 (81) | — |
| From bone marrow aspiration in 25 patients | 19 (76) | — |
| From skin biopsy in 49 patients | 39 (80) | — |
| Previous mastocytosis therapies | 34 (50) | |
| Interferon-α | 13 (19) | |
| TKIs (imatinib, masitinib, dasatinib, imatinib then masitinib) | 6/6/1/3 | |
| Thalidomide | 5 (7) | |
| Hydroxyurea | 3 (4) |
ALT, alanine aminotransferase; ASM, aggressive systemic mastocytosis; AST, aspartate aminotransferase; CM, cutaneous mastocytosis; ISM, indolent systemic mastocytosis; MCL, mast cell leukemia; MIS, mastocytosis in the skin; SAP, serum alkaline phosphatase; SM-AHNMD, systemic mastocytosis with an associated clonal hematologic non–mast cell lineage disease; SSM, smoldering systemic mastocytosis; TKI, tyrosine kinase inhibitors; UNL, upper normal limit.