TABLE 1.
Demographic and baseline clinical characteristics
| Higher‐risk MDS after AZA failure (N = 42) | Other MDS (N = 5) | Total (N = 47) | |
|---|---|---|---|
| Sex, male, n (%) | 32 (76.2) | 3 (60.0) | 35 (74.5) |
| Age, years, median (range) | 74 (63‐93) | 76 (52‐89) | 74 (52‐93) |
| HLA Type (Class I), n (%) | |||
| A*02:01 | 4 (9.5) | 1 (20.0) | 5 (10.6) |
| A*02:01/A*24:02 | 5 (11.9) | 0 | 5 (10.6) |
| A*02:06 | 6 (14.3) | 1 (20.0) | 7 (14.9) |
| A*02:06/A*24:02 | 2 (4.8) | 0 | 2 (4.3) |
| A*24:02 | 25 (59.5) | 3 (60.0) | 28 (59.6) |
| ECOG PS, n (%) | |||
| 0 | 24 (57.1) | 4 (80.0) | 28 (59.6) |
| 1 | 16 (38.1) | 1 (20.0) | 17 (36.2) |
| 2 | 2 (4.8) | 0 | 2 (4.3) |
| WHO classification category, n (%) | |||
| RCUD (RA) | 0 | 1 (20.0) | 1 (2.1) |
| RCUD (RT) | 1 (2.4) | 0 | 1 (2.1) |
| RARS | 0 | 1 (20.0) | 1 (2.1) |
| RCMD | 3 (7.1) | 2 (40.0) | 5 (10.6) |
| RAEB‐1 | 16 (38.1) | 0 | 16 (34.0) |
| RAEB‐2 | 15 (35.7) | 1 (20.0) | 16 (34.0) |
| MDS‐U | 1 (2.4) | 0 | 1 (2.1) |
| N/A | 6 (14.3) | 0 | 6 (12.8) |
| FAB classification category, n (%) | |||
| RA | 5 (11.9) | 3 (60.0) | 8 (17.0) |
| RARS | 0 | 1 (20.0) | 1 (2.1) |
| RAEB | 30 (71.4) | 1 (20.0) | 31 (66.0) |
| RAEB‐t | 7 (16.7) | 0 | 7 (14.9) |
| IPSS risk category, n (%) | |||
| Low | 0 | 1 (20.0) | 1 (2.1) |
| Int‐1 | 5 (11.9) | 3 (60.0) | 8 (17.0) |
| Int‐2 | 27 (64.3) | 1 (20.0) | 28 (59.6) |
| High | 9 (21.4) | 0 | 9 (19.1) |
| N/A | 1 (2.4) | 0 | 1 (2.1) |
| IPSS Karyotype category, n (%) | |||
| Good | 12 (28.6) | 3 (60.0) | 15 (31.9) |
| Intermediate | 9 (21.4) | 2 (40.0) | 11 (23.4) |
| Poor | 20 (47.6) | 0 | 20 (42.6) |
| N/A | 1 (2.4) | 0 | 1 (2.1) |
| IPSS‐R risk category, n (%) | |||
| Low | 0 | 2 (40.0) | 2 (4.3) |
| Intermediate | 5 (11.9) | 2 (40.0) | 7 (14.9) |
| High | 13 (31.0) | 1 (20.0) | 14 (29.8) |
| Very high | 23 (54.8) | 0 | 23 (48.9) |
| N/A | 1 (2.4) | 0 | 1 (2.1) |
| IPSS‐R Karyotype risk category, n (%) | |||
| Good | 11 (26.2) | 3 (60.0) | 14 (29.8) |
| Intermediate | 13 (31.0) | 2 (40.0) | 15 (31.9) |
| Poor | 2 (4.8) | 0 | 2 (4.3) |
| Very poor | 15 (35.7) | 0 | 15 (31.9) |
| N/A | 1 (2.4) | 0 | 1 (2.1) |
| Bone marrow blasts, n (%) | |||
| ≤2% | 3 (7.1) | 2 (40.0) | 5 (10.6) |
| 3−<5% | 3 (7.1) | 2 (40.0) | 5 (10.6) |
| 5‐10% | 18 (42.9) | 0 | 18 (38.3) |
| >10% | 18 (42.9) | 1 (20.0) | 19 (40.4) |
| Hemoglobin, g/dL, n (%) | |||
| ≥10 | 8 (19.0) | 0 | 8 (17.0) |
| 8−<10 | 16 (38.1) | 2 (40.0) | 18 (38.3) |
| <8 | 18 (42.9) | 3 (60.0) | 21 (44.7) |
| Platelets, 1000/μL, n (%) | |||
| ≥100 | 5 (11.9) | 4 (80.0) | 9 (19.1) |
| 50−<100 | 16 (38.1) | 1 (20.0) | 17 (36.2) |
| <50 | 21 (50.0) | 0 | 21 (44.7) |
| Neutrophils, 1000/μL, n (%) | |||
| ≥0.8 | 15 (35.7) | 5 (100.0) | 20 (42.6) |
| <0.8 | 27 (64.3) | 0 | 27 (57.4) |
| Duration of prior AZA treatment, months, median (min‐max) | 9.4 (0.3‐29.3) | – | 9.4 (0.3‐29.3) |
| Duration from AZA treatment cessation to study drug administration, months, median (min‐max) | 1.6 (1.0‐49.4) | – | 1.6 (1.0‐49.4) |
| Type of AZA failure, n (%) | |||
| Primary failure a | 18 (42.9) | – | 18 (38.3) |
| Secondary failure b | 22 (52.4) | – | 22 (46.8) |
| AZA intolerance c | 2 (4.8) | – | 2 (4.3) |
AE, adverse event; AZA, azacitidine; CR, complete remission; ECOG, Eastern Cooperative Oncology Group; FAB, French‐American‐British, HI, hematologic improvement; HLA, human leukocyte antigen; IPSS, International Prognostic Scoring System; IPSS‐R, revised International Prognostic Scoring System; mCR, bone marrow complete remission; MDS‐U, MDS unclassifiable; PR, partial remission; RA, refractory anemia; RAEB, refractory anemia with excess blasts; RARS, refractory anemia with ringed sideroblasts; RCMD, refractory cytopenia with multilineage dysplasia; RCUD, refractory cytopenia with unilineage dysplasia; RN, refractory neutropenia; RT, refractory thrombocytopenia.
Defined as SD or PD.
Defined as failure after CR/mCR/PR/HI.
Defined as treatment discontinuation due to AE.