Table 1.
Patient and Disease Characteristics
| Characteristic | Pre-transplant therapy | |
|---|---|---|
| AZA | IC | |
| No. of patients | 35 | 33 |
| Age (y), range (median) | 4–74 (60) | 2–64 (47) |
| Gender, M/F (No. of patients) | 22/13 | 17/16 |
| Etiology (No. of patients [%]) | ||
| De Novo | 29 (83%) | 28 (85%) |
| Secondary | 6 (17%) | 5 (15%) |
| Disease duration in months (range [median]) | 4–112 (9) | 0–62 (7) |
| WHO Classification (No. of patients [%]) | ||
| RARS | 2 (6%) | 2 (6%) |
| RCMD | 9 (26%) | 3 (9%) |
| RAEB-1 | 3 (9%) | 7 (21%) |
| RAEB-2 | 12 (34%) | 10 (30%) |
| tAML | 2 (6%) | 11 (33%) |
| CMML | 3 (9%) | 0 (0%) |
| JMML | 1 (3%) | 0 (0%) |
| MDS-U | 3 (9%) | 0 (0%) |
| IPSS risk group (No. of patients [%]) | ||
| Low | 6 (17%) | 0 (0%) |
| Intermediate-1 | 11 (31%) | 10 (30%) |
| Intermediate-2 | 9 (26%) | 8 (24%) |
| High | 9 (26%) | 15 (45%) |
| Cytomegalovirus serology (No. of patients [%]) | ||
| Positive | 27 (77%) | 20 (61%) |
| Negative | 8 (23%) | 13 (39%) |
| Myeloblast at transplant (No. of patients [%]) | ||
| <5% | 18 (51%) | 19 (58%) |
| 5–9% | 8 (23%) | 5 (15%) |
| 10–19% | 7 (20%) | 5 (15%) |
| ≥20% | 2 (6%) | 4 (12%) |
| Graft-versus-host-disease prophylaxis (No. of patients [%]) | ||
| MTX, cyclosporine | 1 (3%) | 27 (82%) |
| MTX, tacrolimus | 15 (43%) | 0 (0%) |
| MTX, cyclosporine, tacrolimus | 0 (0%) | 1 (3%) |
| MTX, tacrolimus, rapamycin | 1 (3%) | 0 (0%) |
| Cyclosporine, MMF | 15 (43%) | 4 (12%) |
| Tacrolimus and MMF | 2 (6%) | 0 (0%) |
| Tacrolimus, MMF, Cy | 1 (3%) | 0 (0%) |
| T-cell depletion | 0 (0%) | 1 (3%) |
| Donor (No. of patients [%]) | ||
| HLA-identical sibling | 13 (37%) | 16 (48%) |
| HLA-identical unrelated | 13 (37%) | 17 (52%) |
| Alternative related donor | 1 (3%) | 0 (0%) |
| HLA-mismatched unrelated | 5 (14%) | 0 (0%) |
| Umbilical Cord Blood | 3 (9%) | 0 (0%) |
| Conditioning regimen (No. of patients [%]) | ||
| tBuCy | 12 (34%) | 21 (64%) |
| BuTBI | 0 (0%) | 12 (36%) |
| CyTBI | 1 (3%) | 0 (0%) |
| FluBu | 1 (3%) | 0 (0%) |
| FluTBI(200) | 11 (31%) | 0 (0%) |
| TreoFluTBI(200) | 6 (17%) | 0 (0%) |
| FluTBI(200) + I-131 | 2 (6%) | 0 (0%) |
| FluCyTBI(200) + I-131 | 1 (3%) | 0 (0%) |
| FluCyTBI(200) + ATG | 1 (3%) | 0 (0%) |
| Conditioning intensity (No. of patients [%]) | ||
| High intensity | 14 (40%) | 33 (100%) |
| Reduced intensity | 21 (60%) | 0 (0%) |
ATG = antithymocyte globulin; AZA = Azacitidine; Bu = busulfan; CMML = chronic myelomonocytic leukemia; Cy = cyclophosphamide; I-131 = radiolabeled anti-CD45 antibody; IC= induction chemotherapy; IPSS = International Prognostic Scoring System; JMML = juvenile myelomonocytic leukemia; MDS-U = myelodysplastic syndrome unclassified; MMF = mycophenolate mofetil; MTX = methotrexate; RAEB = refractory anemia with excess blasts; RARS = refractory anemia with ring sideroblasts; RCMD = refractory cytopenias with multilineage dysplasia; tAML = acute myeloid leukemia transformed from myelodysplastic syndrome; TBI = total body irradiation; Treo = treosulfan.