Clinical outcomes of patients with aplastic anemia stratified by the presence or absence of HLA risk alleles
| Clinical characteristic . | Total patients (n = 66) . | HLA risk allele present* (n = 24) . | HLA risk allele absent (n = 42) . | P . |
|---|---|---|---|---|
| Response to first-line therapy, patients | ||||
| OR (CR+PR), n (%) | 35 (76.1) | 12 (70.6) | 23 (79.3) | .722 |
| CR, n (%) | 17 (40.0) | 7 (41.2) | 10 (34.5) | |
| PR, n (%) | 18 (39.1) | 5 (29.4) | 13 (44.8) | |
| None, n (%) | 11 (23.9) | 5 (29.4) | 6 (20.7) | |
| Not evaluable, n | 20 | 7 | 13 | |
| Complications, patients | ||||
| Multiple lines of therapy or disease-related death, n (%) | 19 (38.0) | 11 (57.9) | 8 (25.8) | .036 |
| Required additional therapy, n | 18 | 10 | 8 | |
| Disease-related death after fist- line therapy, n | 1 | 1 | 0 | |
| No second-line therapy or disease-related death, n (%) | 31 (62.0) | 8 (42.1) | 23 (74.2) | |
| Not evaluable, n | 16 | 5 | 11 | |
| Patients with acquired structural chromosomal rearrangements | .005 | |||
| Abnormal, n (%) | 15 (26.8) | 11 (47.8) | 4 (12.1) | |
| Normal, n (%) | 41 (73.2) | 12 (52.2) | 29 (87.9) | |
| Not evaluable, n | 10 | 1 | 9 | |
| Patients with acquired cytogenetic abnormalities | .071 | |||
| Abnormal, n (%) | 6 (10.7) | 5 (21.7) | 1 (3.0) | |
| Normal, n (%) | 50 (89.3) | 18 (78.3) | 32 (97.0) | |
| Not evaluable, n | 10 | 1 | 9 | |
| Patients with acquired CN-LOH, n (%) | .029 | |||
| Acquired CN-LOH present | 10 (15.2) | 7 (29.2) | 3 (7.1) | |
| 6p CN-LOH | 8 (12.1) | 5 (20.8) | 3 (7.1) | |
| No CN-LOH | 56 (84.8) | 17 (70.8) | 39 (92.9) | |
| HLA loss, patients, n (%) | .013 | |||
| HLA loss present | 11 (16.7) | 8 (33.3) | 3 (7.1) | |
| No HLA loss | 55 (83.3) | 16 (66.7) | 39 (92.9) | |
| MDS transformation, patients | .013 | |||
| MDS transformation, n (%) | 4 (8.0) | 4 (22.2) | 0 (0) | |
| No MDS transformation, n (%) | 46 (92.0) | 14 (77.8) | 32 (100) | |
| Not evaluable, n | 16 | 6 | 10 |
| Clinical characteristic . | Total patients (n = 66) . | HLA risk allele present* (n = 24) . | HLA risk allele absent (n = 42) . | P . |
|---|---|---|---|---|
| Response to first-line therapy, patients | ||||
| OR (CR+PR), n (%) | 35 (76.1) | 12 (70.6) | 23 (79.3) | .722 |
| CR, n (%) | 17 (40.0) | 7 (41.2) | 10 (34.5) | |
| PR, n (%) | 18 (39.1) | 5 (29.4) | 13 (44.8) | |
| None, n (%) | 11 (23.9) | 5 (29.4) | 6 (20.7) | |
| Not evaluable, n | 20 | 7 | 13 | |
| Complications, patients | ||||
| Multiple lines of therapy or disease-related death, n (%) | 19 (38.0) | 11 (57.9) | 8 (25.8) | .036 |
| Required additional therapy, n | 18 | 10 | 8 | |
| Disease-related death after fist- line therapy, n | 1 | 1 | 0 | |
| No second-line therapy or disease-related death, n (%) | 31 (62.0) | 8 (42.1) | 23 (74.2) | |
| Not evaluable, n | 16 | 5 | 11 | |
| Patients with acquired structural chromosomal rearrangements | .005 | |||
| Abnormal, n (%) | 15 (26.8) | 11 (47.8) | 4 (12.1) | |
| Normal, n (%) | 41 (73.2) | 12 (52.2) | 29 (87.9) | |
| Not evaluable, n | 10 | 1 | 9 | |
| Patients with acquired cytogenetic abnormalities | .071 | |||
| Abnormal, n (%) | 6 (10.7) | 5 (21.7) | 1 (3.0) | |
| Normal, n (%) | 50 (89.3) | 18 (78.3) | 32 (97.0) | |
| Not evaluable, n | 10 | 1 | 9 | |
| Patients with acquired CN-LOH, n (%) | .029 | |||
| Acquired CN-LOH present | 10 (15.2) | 7 (29.2) | 3 (7.1) | |
| 6p CN-LOH | 8 (12.1) | 5 (20.8) | 3 (7.1) | |
| No CN-LOH | 56 (84.8) | 17 (70.8) | 39 (92.9) | |
| HLA loss, patients, n (%) | .013 | |||
| HLA loss present | 11 (16.7) | 8 (33.3) | 3 (7.1) | |
| No HLA loss | 55 (83.3) | 16 (66.7) | 39 (92.9) | |
| MDS transformation, patients | .013 | |||
| MDS transformation, n (%) | 4 (8.0) | 4 (22.2) | 0 (0) | |
| No MDS transformation, n (%) | 46 (92.0) | 14 (77.8) | 32 (100) | |
| Not evaluable, n | 16 | 6 | 10 |
CN-LOH, copy number-neutral loss of heterozygosity; CR, complete response; MDS, myelodysplastic syndrome (patients were diagnosed with MDS-EB-1 [n = 1], MDS-U [single lineage dysplasia and pancytopenia; n = 1], MDS-U [based on defining cytogenetic abnormality (monosomy 7) and cytopenia]; n = 2); OR, overall response; PR, partial response.
P value pertains to the comparison of the 24 patients who carry at least 1 of the 4 HLA risk alleles* (HLA-A*33:03, HLA-A*68:01, HLA-B*14:02, or HLA-B*40:02) to the 42 patients who carry none of the HLA risk alleles.