Baseline demographic and clinical characteristics
| Parameter . | Double-blind, dose-escalation cohorts . | Expansion cohort/matched controls . | |||||
|---|---|---|---|---|---|---|---|
| Placebo (n = 6) . | CD24Fc 240 mg (n = 6) . | CD24Fc 480 mg (n = 6) . | CD24Fc 480/240/240 mg∗ (n = 6) . | CD24Fc total (n = 18) . | Matched controls (n = 92) . | CD24Fc 480/240/240 mg∗ (n = 26†) . | |
| Age, mean (SD), y‡ | 53.8 (11.67) | 54.2 (16.82) | 53.3 (19.76) | 57.3 (11.15) | 54.9 (15.42) | 50.1 (12.92) | 51.3 (13.28) |
| Age group, y | |||||||
| <65§ | 5 (83.3) | 5 (83.3) | 3 (50.0) | 3 (50.0) | 11 (61.1) | 79 (85.9) | 21 (80.8) |
| ≥65§ | 1 (16.7) | 1 (16.7) | 3 (50.0) | 3 (50.0) | 7 (38.9) | 13 (14.1) | 5 (19.2) |
| Sex | |||||||
| Male | 4 (66.7) | 4 (66.7) | 4 (66.7) | 3 (50.0) | 11 (61.1) | 64 (69.6) | 14 (53.8) |
| Female | 2 (33.3) | 2 (33.3) | 2 (33.3) | 3 (50.0) | 7 (38.9) | 28 (30.4) | 12 (46.2) |
| Race | |||||||
| White | 6 (100.0) | 5 (83.3) | 6 (100.0) | 6 (100.0) | 17 (94.4) | 79 (85.9) | 25 (96.2) |
| African American | 0 | 0 | 0 | 0 | 0 | 4 (4.3) | 1 (3.8) |
| Pacific Islander/Asian | 0 | 0 | 0 | 0 | 0 | 7 (7.6) | 0 |
| American Indian | 0 | 0 | 0 | 0 | 0 | 1 (1.1) | 0 |
| Not reported | 0 | 1 (16.7) | 0 | 0 | 1 (5.6) | 1 (1.1) | 0 |
| Ethnicity | |||||||
| Hispanic or Latino | 0 | 1 (16.7) | 0 | 0 | 1 (5.6) | 2 (2.2) | 0 |
| Not Hispanic or Latino | 6 (100.0) | 5 (83.3) | 6 (100.0) | 6 (100.0) | 17 (94.4) | 89 (96.7) | 26 (100.0) |
| NA (non-US resident) | 0 | 0 | 0 | 0 | 0 | 1 (1.1) | 0 |
| Primary diagnosis‖ | |||||||
| AML | 2 (33.3) | 3 (50.0) | 3 (50.0) | 1 (16.7) | 7 (38.9) | 54 (58.7) | 12 (46.2) |
| ALL | 0 | 1 (16.7) | 1 (16.7) | 1 (16.7) | 3 (16.7) | 17 (18.5) | 7 (26.9) |
| CML | 0 | 1 (16.7) | 1 (16.7) | 0 | 2 (11.1) | 2 (2.2) | 1 (3.8) |
| MDS | 3 (50.0) | 1 (16.7) | 0 | 4 (66.7) | 5 (27.8) | 19 (20.7) | 6 (23.1) |
| CMML | 1 (16.7) | 0 | 1 (16.7) | 0 | 1 (5.6) | 0 | 0 |
| Refined disease risk index | |||||||
| Low/intermediate | NA | NA | NA | NA | NA | 65 (70.7) | 21 (80.8) |
| High/very high | NA | NA | NA | NA | NA | 27 (29.3) | 5 (19.2) |
| Karnofsky performance status at baseline | |||||||
| 90%-100% | 1 (16.7) | 1 (16.7) | 1 (16.7) | 3 (50.0) | 5 (27.8) | 62 (67.4) | 17 (65.4) |
| 70%-80% | 5 (83.3) | 5 (83.3) | 5 (83.3) | 3 (50.0) | 13 (72.2) | 30 (32.6) | 9 (34.6) |
| Comorbidity index risk category at baseline | |||||||
| Low (0) | 3 (50.0) | 1 (16.7) | 2 (33.3) | 2 (33.3) | 5 (27.8) | 19 (20.7) | 6 (23.1) |
| Intermediate (1-2) | 2 (33.3) | 5 (83.3) | 2 (33.3) | 2 (33.3) | 9 (50.0) | 42 (45.7) | 13 (50.0) |
| High (3-4)¶ | 1 (16.7) | 0 | 2 (33.3) | 2 (33.3) | 4 (22.2) | 31 (33.7) | 7 (26.9) |
| Graft type | |||||||
| PBSCs | 4 (66.7) | 4 (66.7) | 5 (83.3) | 5 (83.3) | 14 (77.8) | 86 (93.5) | 23 (88.5) |
| BM | 2 (33.3) | 2 (33.3) | 1 (16.7) | 1 (16.7) | 4 (22.2) | 6 (6.5) | 3 (11.5) |
| Donor-recipient sex | |||||||
| Male-male | 3 (50.0) | 2 (33.3) | 3 (50.0) | 2 (33.3) | 7 (38.9) | 48 (52.2) | 9 (34.6) |
| Male-female | 1 (16.7) | 2 (33.3) | 2 (33.3) | 2 (33.3) | 6 (33.3) | 18 (19.6) | 7 (26.9) |
| Female-male | 1 (16.7) | 2 (33.3) | 1 (16.7) | 1 (16.7) | 4 (22.2) | 16 (17.4) | 3 (11.5) |
| Female-female | 1 (16.7) | 0 | 0 | 1 (16.7) | 1 (5.6) | 10 (10.9) | 5 (19.2) |
| Unknown-male | 0 | 0 | 0 | 0 | 0 | 0 | 2 (7.7) |
| Conditioning regimen | |||||||
| TBI/Cy | 0 | 1 (16.7) | 2 (33.3) | 1 (16.7) | 4 (22.2) | 20 (21.7) | 3 (11.5) |
| TBI/Cy/etoposide | 0 | 0 | 0 | 0 | 0 | 1 (1.1) | 0 |
| TBI/etoposide | 0 | 0 | 0 | 0 | 0 | 1 (1.1) | 0 |
| Bu/Cy | 0 | 0 | 0 | 0 | 0 | 41 (44.6) | 0 |
| Bu/Flu | 6 (100.0) | 5 (83.3) | 4 (66.7) | 5 (83.3) | 14 (77.8) | 28 (30.4) | 16 (61.5) |
| Flu/melphalan | 0 | 0 | 0 | 0 | 0 | 1 (1.1) | 0 |
| Cy/thiotepa | 0 | 0 | 0 | 0 | 0 | 0 | 7 (26.9) |
| Parameter . | Double-blind, dose-escalation cohorts . | Expansion cohort/matched controls . | |||||
|---|---|---|---|---|---|---|---|
| Placebo (n = 6) . | CD24Fc 240 mg (n = 6) . | CD24Fc 480 mg (n = 6) . | CD24Fc 480/240/240 mg∗ (n = 6) . | CD24Fc total (n = 18) . | Matched controls (n = 92) . | CD24Fc 480/240/240 mg∗ (n = 26†) . | |
| Age, mean (SD), y‡ | 53.8 (11.67) | 54.2 (16.82) | 53.3 (19.76) | 57.3 (11.15) | 54.9 (15.42) | 50.1 (12.92) | 51.3 (13.28) |
| Age group, y | |||||||
| <65§ | 5 (83.3) | 5 (83.3) | 3 (50.0) | 3 (50.0) | 11 (61.1) | 79 (85.9) | 21 (80.8) |
| ≥65§ | 1 (16.7) | 1 (16.7) | 3 (50.0) | 3 (50.0) | 7 (38.9) | 13 (14.1) | 5 (19.2) |
| Sex | |||||||
| Male | 4 (66.7) | 4 (66.7) | 4 (66.7) | 3 (50.0) | 11 (61.1) | 64 (69.6) | 14 (53.8) |
| Female | 2 (33.3) | 2 (33.3) | 2 (33.3) | 3 (50.0) | 7 (38.9) | 28 (30.4) | 12 (46.2) |
| Race | |||||||
| White | 6 (100.0) | 5 (83.3) | 6 (100.0) | 6 (100.0) | 17 (94.4) | 79 (85.9) | 25 (96.2) |
| African American | 0 | 0 | 0 | 0 | 0 | 4 (4.3) | 1 (3.8) |
| Pacific Islander/Asian | 0 | 0 | 0 | 0 | 0 | 7 (7.6) | 0 |
| American Indian | 0 | 0 | 0 | 0 | 0 | 1 (1.1) | 0 |
| Not reported | 0 | 1 (16.7) | 0 | 0 | 1 (5.6) | 1 (1.1) | 0 |
| Ethnicity | |||||||
| Hispanic or Latino | 0 | 1 (16.7) | 0 | 0 | 1 (5.6) | 2 (2.2) | 0 |
| Not Hispanic or Latino | 6 (100.0) | 5 (83.3) | 6 (100.0) | 6 (100.0) | 17 (94.4) | 89 (96.7) | 26 (100.0) |
| NA (non-US resident) | 0 | 0 | 0 | 0 | 0 | 1 (1.1) | 0 |
| Primary diagnosis‖ | |||||||
| AML | 2 (33.3) | 3 (50.0) | 3 (50.0) | 1 (16.7) | 7 (38.9) | 54 (58.7) | 12 (46.2) |
| ALL | 0 | 1 (16.7) | 1 (16.7) | 1 (16.7) | 3 (16.7) | 17 (18.5) | 7 (26.9) |
| CML | 0 | 1 (16.7) | 1 (16.7) | 0 | 2 (11.1) | 2 (2.2) | 1 (3.8) |
| MDS | 3 (50.0) | 1 (16.7) | 0 | 4 (66.7) | 5 (27.8) | 19 (20.7) | 6 (23.1) |
| CMML | 1 (16.7) | 0 | 1 (16.7) | 0 | 1 (5.6) | 0 | 0 |
| Refined disease risk index | |||||||
| Low/intermediate | NA | NA | NA | NA | NA | 65 (70.7) | 21 (80.8) |
| High/very high | NA | NA | NA | NA | NA | 27 (29.3) | 5 (19.2) |
| Karnofsky performance status at baseline | |||||||
| 90%-100% | 1 (16.7) | 1 (16.7) | 1 (16.7) | 3 (50.0) | 5 (27.8) | 62 (67.4) | 17 (65.4) |
| 70%-80% | 5 (83.3) | 5 (83.3) | 5 (83.3) | 3 (50.0) | 13 (72.2) | 30 (32.6) | 9 (34.6) |
| Comorbidity index risk category at baseline | |||||||
| Low (0) | 3 (50.0) | 1 (16.7) | 2 (33.3) | 2 (33.3) | 5 (27.8) | 19 (20.7) | 6 (23.1) |
| Intermediate (1-2) | 2 (33.3) | 5 (83.3) | 2 (33.3) | 2 (33.3) | 9 (50.0) | 42 (45.7) | 13 (50.0) |
| High (3-4)¶ | 1 (16.7) | 0 | 2 (33.3) | 2 (33.3) | 4 (22.2) | 31 (33.7) | 7 (26.9) |
| Graft type | |||||||
| PBSCs | 4 (66.7) | 4 (66.7) | 5 (83.3) | 5 (83.3) | 14 (77.8) | 86 (93.5) | 23 (88.5) |
| BM | 2 (33.3) | 2 (33.3) | 1 (16.7) | 1 (16.7) | 4 (22.2) | 6 (6.5) | 3 (11.5) |
| Donor-recipient sex | |||||||
| Male-male | 3 (50.0) | 2 (33.3) | 3 (50.0) | 2 (33.3) | 7 (38.9) | 48 (52.2) | 9 (34.6) |
| Male-female | 1 (16.7) | 2 (33.3) | 2 (33.3) | 2 (33.3) | 6 (33.3) | 18 (19.6) | 7 (26.9) |
| Female-male | 1 (16.7) | 2 (33.3) | 1 (16.7) | 1 (16.7) | 4 (22.2) | 16 (17.4) | 3 (11.5) |
| Female-female | 1 (16.7) | 0 | 0 | 1 (16.7) | 1 (5.6) | 10 (10.9) | 5 (19.2) |
| Unknown-male | 0 | 0 | 0 | 0 | 0 | 0 | 2 (7.7) |
| Conditioning regimen | |||||||
| TBI/Cy | 0 | 1 (16.7) | 2 (33.3) | 1 (16.7) | 4 (22.2) | 20 (21.7) | 3 (11.5) |
| TBI/Cy/etoposide | 0 | 0 | 0 | 0 | 0 | 1 (1.1) | 0 |
| TBI/etoposide | 0 | 0 | 0 | 0 | 0 | 1 (1.1) | 0 |
| Bu/Cy | 0 | 0 | 0 | 0 | 0 | 41 (44.6) | 0 |
| Bu/Flu | 6 (100.0) | 5 (83.3) | 4 (66.7) | 5 (83.3) | 14 (77.8) | 28 (30.4) | 16 (61.5) |
| Flu/melphalan | 0 | 0 | 0 | 0 | 0 | 1 (1.1) | 0 |
| Cy/thiotepa | 0 | 0 | 0 | 0 | 0 | 0 | 7 (26.9) |
Except for mean (SD) age, all values are reported as n (%); percentages were calculated using the number of participants in the column heading as the denominator.
ALL, acute lymphoblastic leukemia; AML, acute myeloid leukemia; BM, bone marrow; Bu, busulfan; CML, chronic myelogenous leukemia; CMML, chronic myelomonocytic leukemia; Cy, cyclophosphamide; Flu, fludarabine; MDS, myelodysplastic syndrome; NA, not applicable; PBSC, peripheral blood stem cell; SD, standard deviation.
Regimen (infusion): 480, 240, and 240 mg on days −1, 14, and 28, respectively.
Expansion cohort includes n = 6 participants from the double-blind, dose-escalation cohort and n = 20 additional participants from the open-label expansion phase.
Age for double-blind, dose-escalation cohorts reported as mean age at time of informed consent; age for expansion cohort/matched controls reported as mean age at time of HSCT.
For expansion cohort/matched controls, values are ≤65 and >65 years, respectively.
None of the participants in the expansion cohort had CMML, thus, the selection criteria for matched controls only included AML, ALL, CML, and MDS.
Comorbidity index risk category of high ranges from 3 to 5 for expansion cohort/matched controls.