Multivariable Cox regression analysis of clinical, cytogenetic, and molecular predictors of adverse outcome in patients with AA
| Variable . | HR (95% CI) . | P value . |
|---|---|---|
| DNMT3A mutation | 2.01 (0.04-104.74) | .73 |
| Age at diagnosis | 1.02 (1.00-1.05) | .094 |
| PIGA mutation | 0.56 (0.20-1.59) | .274 |
| CUX1 mutation | 8.18 (0.74-89.99) | .086 |
| Total number of mutations | 1.02 (0.67-1.56) | .916 |
| U2AF1 mutation | 0.58 (0.04-7.68) | .682 |
| RUNX1 mutation | 1.06 (0.06-19.70) | .969 |
| TP53 mutation | 1.60 (0.06-46.32) | .785 |
| PNH clone size at diagnosis (%) | 1.00 (0.97-1.04) | .948 |
| TET2 mutation | 0.81 (0.08-7.81) | .854 |
| ZRSR2 mutation | 0.58 (0.02-20.84) | .768 |
| ASXL1 mutation | 1.03 (0.10-10.20) | .981 |
| BCOR/L1 mutation | 0.77 (0.08-7.26) | .82 |
| EZH2 mutation | 0.93 (0.06-15.49) | .959 |
| SETBP1 mutation | 1.42 (0.06-35.24) | .832 |
| PNH clone size of >0% | 0.86 (0.38-1.96) | .727 |
| Abnormal karyotype at AA diagnosis | 0.59 (0.02-14.74) | .747 |
| Karyotype del(13q) | 0.45 (0.01-17.95) | .674 |
| SAA or VSAA diagnosis | 1.71 (0.64-4.52) | .284 |
| Male sex | 1.21 (0.54-2.70) | .637 |
| AA-PNH overlap syndrome diagnosis | 1.50 (0.29-7.92) | .63 |
| Variable . | HR (95% CI) . | P value . |
|---|---|---|
| DNMT3A mutation | 2.01 (0.04-104.74) | .73 |
| Age at diagnosis | 1.02 (1.00-1.05) | .094 |
| PIGA mutation | 0.56 (0.20-1.59) | .274 |
| CUX1 mutation | 8.18 (0.74-89.99) | .086 |
| Total number of mutations | 1.02 (0.67-1.56) | .916 |
| U2AF1 mutation | 0.58 (0.04-7.68) | .682 |
| RUNX1 mutation | 1.06 (0.06-19.70) | .969 |
| TP53 mutation | 1.60 (0.06-46.32) | .785 |
| PNH clone size at diagnosis (%) | 1.00 (0.97-1.04) | .948 |
| TET2 mutation | 0.81 (0.08-7.81) | .854 |
| ZRSR2 mutation | 0.58 (0.02-20.84) | .768 |
| ASXL1 mutation | 1.03 (0.10-10.20) | .981 |
| BCOR/L1 mutation | 0.77 (0.08-7.26) | .82 |
| EZH2 mutation | 0.93 (0.06-15.49) | .959 |
| SETBP1 mutation | 1.42 (0.06-35.24) | .832 |
| PNH clone size of >0% | 0.86 (0.38-1.96) | .727 |
| Abnormal karyotype at AA diagnosis | 0.59 (0.02-14.74) | .747 |
| Karyotype del(13q) | 0.45 (0.01-17.95) | .674 |
| SAA or VSAA diagnosis | 1.71 (0.64-4.52) | .284 |
| Male sex | 1.21 (0.54-2.70) | .637 |
| AA-PNH overlap syndrome diagnosis | 1.50 (0.29-7.92) | .63 |
A multivariable Cox proportional hazards model was constructed to assess the association between baseline clinical characteristics, somatic mutations, karyotypic abnormalities, and PNH clone size with the risk of adverse outcomes in patients with aplastic anemia. HRs with 95% CIs and P values are shown for each variable. Among the top-ranked features by model-derived impact scores, CUX1 and DNMT3A mutations demonstrated the highest HRs (HR of 8.18 and 2.01, respectively), although neither reached statistical significance. Age at diagnosis trended toward significance (HR, 1.02 per year; P = .094). Other mutations, including TP53, RUNX1, TET2, and ASXL1, as well as clinical features such as PNH clone size, abnormal karyotype, and AA-PNH overlap, were not significantly associated with outcome in this cohort.
CI, confidence interval; HR, hazard ratio.