Baseline patient demographics and disease characteristics for the full analysis set
| Characteristic . | (N = 19) . |
|---|---|
| Sex, n (%) | |
| Female | 4 (21.1) |
| Male | 15 (78.9) |
| Age, median (min, max), y | 73.0 (52, 82) |
| Race | |
| White | 15 (78.9) |
| Black or African American | 1 (5.3) |
| Not reported | 3 (15.8) |
| Ethnicity | |
| Hispanic or Latino | 1 (5.3) |
| Not Hispanic or Latino | 12 (63.2) |
| Not reported | 6 (31.6) |
| ECOG PS | |
| 0 | 5 (26.3) |
| 1 | 11 (57.9) |
| 2 | 3 (15.8) |
| Prior therapy, n (%) | |
| Intensive chemotherapy | 3 (15.8) |
| One line of HMA-based therapy | 14 (73.7) |
| Two lines of HMA-based therapy | 1 (5.3) |
| Investigational | 3 (15.8) |
| Other∗ | 1 (5.3) |
| IPSS-R score at initial diagnosis, n (%)† | |
| ≤1.5 (very low) | 0 |
| >1.5-3 (low) | 2 (10.5) |
| >3-4.5 (intermediate) | 6 (31.6) |
| >4.5-6 (high) | 2 (10.5) |
| >6 (very high) | 2 (10.5) |
| Unknown | 7 (36.8) |
| IPSS-R score at screening, n (%) | |
| ≤1.5 (very low) | 0 |
| >1.5-3 (low)‡ | 4 (21.1) |
| >3-4.5 (intermediate)§ | 8 (42.1) |
| >4.5-6 (high)‖ | 3 (15.8) |
| >6 (very high)¶ | 3 (15.8) |
| Unknown# | 1 (5.3) |
| IPSS-M score at screening, n (%)∗∗ | |
| Low | 3 (15.8) |
| Moderate low | 1 (5.3) |
| Moderate high | 4 (21.1) |
| High | 3 (15.8) |
| Very high | 8 (42.1) |
| WHO classification at screening, n (%) | |
| MDS with excess blasts (MDS-EB1)†† | 4 (21.1) |
| MDS with excess blasts (MDS-EB2)‡‡ | 1 (5.3) |
| MDS, with MLD (MDS-MLD) | 4 (21.1) |
| MDS, unclassifiable | 10 (52.6) |
| Cytogenetic result, n (%) | |
| Normal | 9 (47.4) |
| Abnormal | 9 (47.4) |
| Missing | 1 (5.3) |
| Characteristic . | (N = 19) . |
|---|---|
| Sex, n (%) | |
| Female | 4 (21.1) |
| Male | 15 (78.9) |
| Age, median (min, max), y | 73.0 (52, 82) |
| Race | |
| White | 15 (78.9) |
| Black or African American | 1 (5.3) |
| Not reported | 3 (15.8) |
| Ethnicity | |
| Hispanic or Latino | 1 (5.3) |
| Not Hispanic or Latino | 12 (63.2) |
| Not reported | 6 (31.6) |
| ECOG PS | |
| 0 | 5 (26.3) |
| 1 | 11 (57.9) |
| 2 | 3 (15.8) |
| Prior therapy, n (%) | |
| Intensive chemotherapy | 3 (15.8) |
| One line of HMA-based therapy | 14 (73.7) |
| Two lines of HMA-based therapy | 1 (5.3) |
| Investigational | 3 (15.8) |
| Other∗ | 1 (5.3) |
| IPSS-R score at initial diagnosis, n (%)† | |
| ≤1.5 (very low) | 0 |
| >1.5-3 (low) | 2 (10.5) |
| >3-4.5 (intermediate) | 6 (31.6) |
| >4.5-6 (high) | 2 (10.5) |
| >6 (very high) | 2 (10.5) |
| Unknown | 7 (36.8) |
| IPSS-R score at screening, n (%) | |
| ≤1.5 (very low) | 0 |
| >1.5-3 (low)‡ | 4 (21.1) |
| >3-4.5 (intermediate)§ | 8 (42.1) |
| >4.5-6 (high)‖ | 3 (15.8) |
| >6 (very high)¶ | 3 (15.8) |
| Unknown# | 1 (5.3) |
| IPSS-M score at screening, n (%)∗∗ | |
| Low | 3 (15.8) |
| Moderate low | 1 (5.3) |
| Moderate high | 4 (21.1) |
| High | 3 (15.8) |
| Very high | 8 (42.1) |
| WHO classification at screening, n (%) | |
| MDS with excess blasts (MDS-EB1)†† | 4 (21.1) |
| MDS with excess blasts (MDS-EB2)‡‡ | 1 (5.3) |
| MDS, with MLD (MDS-MLD) | 4 (21.1) |
| MDS, unclassifiable | 10 (52.6) |
| Cytogenetic result, n (%) | |
| Normal | 9 (47.4) |
| Abnormal | 9 (47.4) |
| Missing | 1 (5.3) |
EB, excess blasts; ECOG PS, Eastern Cooperative Oncology Group performance status; max, maximum; min, minimum; MLD, multilineage dysplasia; n, number of patients; N, total number of patients; SD, standard deviation; WHO, World Health Organization.
Patient received lenalidomide.
Six patients did not have an IPSS-R score at diagnosis due to being treated locally before being referred to the clinical trial site.
Karyotypes in the IPSS-R low-risk group were: 46, XX[20]; 46, XY[20]; 46 XY[20]; and normal male karyotype.
Karyotypes in the IPSS-R intermediate-risk group were: 46, XX [20]; 47,XY,+21[1]/46,XY,DEL(7)(Q22)[1]/46,XY[18]; 43,XY,+1,DER(1;15)(Q10;Q10),-5,-9,-12 (1)/46,XY,DUP(1)[19]; 46, XX[20]; unknown; 46,X,DEL(X)(Q26),T(1;17)(Q12;Q25),T(12;16)(Q15;P11.2)[4]/46,XX,T(3;16)(P21;P13.1)[2]/46,XX[7]//46,XY[2]; and normal male karyotype; 46,XY[4].
Karyotypes in the IPSS-R high-risk group were: 47,XY,+8[3]/46,XY[17] (20 male metaphases [3 abnormal and 17 normal] were analyzed; the previously reported clone remained in the bone marrow, indicating persistent disease); 47,XY,+8[6]/46,XY[14]; and 46, XY[20].
Karyotypes in the IPSS-R very-high-risk group were: 45,XY,−7[17]/46,XY,DER(7;18)(P10;Q10),+11[3] (20 abnormal male metaphases were analyzed; the previously reported abnormal clones remain fully predominant in the bone marrow indicating persistent disease); 46,XY,+1,DER(1;7)(Q10;P10)[3]; and 47,XX,+21[13]/46,XX[7] (abnormal result showing previously unreported abnormality).
The karyotype in the patient whose IPSS-R score was unknown at screening was 46,XY[20].
Derived retrospectively by internal sponsor review.
MDS-EB1: 5% to 9% of bone marrow cells, or 2% to 4% of blood cells, are blasts.
MDS-EB2: 10% to 19% of bone marrow cells, or 5% to 19% of blood cells, are blasts.