Table 1.
Baseline Characteristics of Patients Who Underwent Randomization and Received the Study Drug, Intention-to-Treat Population
| Characteristic | Brincidofovir (N = 303) | Placebo (N = 149) |
|---|---|---|
| Age, yr, median (range) | 56 (18-77) | 54 (20-75) |
| Male sex, n (%) | 163 (53.8) | 98 (65.8) |
| Race, n (%) | ||
| White | 255 (84.2) | 123 (82.6) |
| African American | 24 (7.9) | 14 (9.4) |
| Asian | 17 (5.6) | 10 (6.7) |
| Other | 7 (2.3) | 2 (1.3) |
| Hispanic or Latino ethnicity, n (%) | 27 (8.9) | 13 (8.7) |
| Weight, kg, median (range) | 78.7 (42.2-122.0) | 75.3 (44.0-138.3) |
| Body mass index, kg/m2, median (range) | 26.2 (16.7-44.6) | 26.1 (17.9-43.0) |
| Indication for allogeneic HCT, n (%) | ||
| Acute myelogenous leukemia | 129 (42.6) | 64 (43.0) |
| Myelodysplastic syndrome | 52 (17.2) | 24 (16.1) |
| Non-Hodgkin lymphoma | 28 (9.2) | 18 (12.1) |
| Acute lymphocytic leukemia | 29 (9.6) | 13 (8.7) |
| Chronic myelogenous leukemia | 10 (3.3) | 6 (4.0) |
| Chronic lymphocytic leukemia | 10 (3.3) | 6 (4.0) |
| Aplastic anemia | 9 (3.0) | 7 (4.7) |
| Other diseases | 36 (11.9) | 11 (7.4) |
| Graft source, n (%) | ||
| Peripheral blood | 241 (79.5) | 113 (75.8) |
| Bone marrow | 41 (13.5) | 24 (16.1) |
| Cord blood | 19 (6.3) | 11 (7.4) |
| Other* | 2 (.7) | 7 (0.7) |
| Donor type, n(%)† | ||
| Matched unrelated | 148 (48.8) | 62 (41.8) |
| Matched related | 97 (32.0) | 52 (34.9) |
| Mismatched | 23 (7.6) | 15 (10.1) |
| Haploidentical | 14 (4.6) | 8 (5.4) |
| Donor CMV seropositive, n (%) | 154 (50.8) | 84 (56.4) |
| Myeloablative conditioning regimen, n (%) | 162 (53.5) | 86 (57.7) |
| Ex vivo T cell depletion,‡ n (%) | 36 (11.9) | 20 (13.4) |
| Alemtuzumab use, n (%) | 26 (8.6) | 12 (8.1) |
| ATG globulin use, n (%) | 85 (28.1) | 47 (31.5) |
| Acute GVHD at baseline, n (%) | 10 (3.3) | 6 (4.0) |
| Glucocorticoids at ≥1 mg/kg prednisone equivalent, n (%) | 2 (.7) | 3 (2.0) |
| Immunosuppressant use at baseline, n (%) | ||
| Tacrolimus | 251 (82.8) | 121 (81.2) |
| Mycophenolate | 87 (28.7) | 45 (30.2) |
| Cyclosporine | 43 (14.2) | 24 (16.1) |
| Sirolimus | 28 (9.2) | 10 (6.7) |
| Time to randomization after HCT, d, median (range) | 15 (3-33) | 14 (2-29) |
| Absolute neutrophil count <500 cells/μL at baseline, n (%) | 116 (38.3) | 58 (38.9) |
| CMV DNA PCR at randomization, n (%) | ||
| Not detected | 288 (95.0) | 137 (91.9) |
| Detected, <151 copies/mL§ | 15 (5.0) | 12 (8.1) |
| Risk of CMV disease progression,** n (%) | ||
| Higher risk | 223 (73.6) | 109 (73.2) |
| Lower risk | 80 (26.4) | 40 (26.8) |
Three patients from 1 site underwent allogeneic HCT using a combination of adult haploidentical peripheral blood and cord blood grafts.
Cord-blood and “other” grafts not included.
T cell depletion, CD34+ selection, and/or other.
No patient had a quantifiable CMV virus load at randomization.
Stratification criterion at randomization. Patients were considered at higher risk of CMV disease progression if they received cord blood or ex vivo T cell-depleted grafts or grafts from unrelated, mismatched, or haploidentical donors; received antithymocyte globulin or alemtuzumab; or were being treated with ≥1 mg/kg/day of prednisone (or equivalent) for acute GVHD or other conditions. Patients who received grafts from matched-related donors without any higher-risk features were considered at lower risk of CMV disease progression.