Table 1.
Main Features, Doses, and Response Rates of MDS or MDS/MPN Patients Treated With Danazol
| Age and sex | |
| Age at diagnosis (y, range) | 69 (27–87) |
| Female (n, %) | 23/57 (40) |
| Male (n, %) | 34/57 (60) |
| WHO-2016 diagnosis | |
| MDS-MLD | 28/57 (49) |
| MDS-SLD | 7/57 (12) |
| MDS-EB1 | 11/57 (19) |
| MDS-EB2 | 2/57 (4) |
| MDS-RS-MLD | 1/57 (2) |
| CMML | 5/57 (9) |
| MDS/MPN (not CMML) | 1/57 (2) |
| MDS-U | 2/57 (4) |
| Bone marrow features at diagnosis (n, %) | |
| Available core biopsy at diagnosis | 43/57 (75) |
| Hypocellular (cellularity <25%) | 9/43 (21) |
| Fibrosis MF 0–1 | 30/43 (70) |
| Fibrosis MF 2 | 11/43 (26) |
| MF-not evaluated | 2/43 (5) |
| IPSS at diagnosis (n, %), only for MDS (n = 51) | |
| Low | 17/51 (33) |
| Int-1 | 34/51 (67) |
| IPSS-R at diagnosis (n, %), only for MDS (n = 51) | |
| Very low | 2/51 (4) |
| Low | 21/51 (41) |
| Intermediate | 15/51 (29) |
| High | 9/51 (18) |
| Very high | 4/51 (8) |
| Blood counts before treatment | |
| Hb (g/dL) | 11.1 (6.9–15.7) |
| PLT (103/μL) | 20 (2–49) |
| ANC (103/μL) | 2.00 (0.26–20.4) |
| Danazol dosing and timing | |
| Mean daily dose (mg, range) | 500 (88–667) |
| Dose reductions due to any cause (n, %) | 19/57 (33) |
| Median treatment duration (mo, range) | 12.0 (0.6–149.5) |
| Concomitant treatments at danazol start (n, %) | |
| Erythropoiesis stimulating agents | 5/57 (9) |
| Corticosteroids | 12/57 (21) |
| Cyclosporin A | 1/57 (2) |
| No concomitant treatments | 41/57 (72) |
| Response data | |
| Median follow-up (mo, range) | 16.2 (0.7–171) |
| PLT response (n, %) | 29/57 (51) |
| Median time to PLT response (mo, range) | 3.3 (1.3–24.6) |
| Erythroid hematological improvement (n, %) | 10/24 (42) |
| Neutrophil hematological improvement (n, %) | 4/9 (44) |
ANC = absolute neutrophil count; CMML = chronic myelomonocytic leukemia; Hb = hemoglobin; IPSS = International Prognostic Scoring System; IPSS-R = revised International Prognostic Scoring System; MDS/MPN = myelodysplastic syndrome/myeloproliferative neoplasms; MDS EB1 = myelodysplastic syndrome with excess blast 1; MDS EB2 = myelodysplastic syndrome with excess blast 2; PLT = platelet.