Patients’ characteristics
| Characteristic . | Value . |
|---|---|
| Number of patients | 39 |
| Median age, y (range) | 45 (20-72) |
| Sex | |
| M | 19 |
| F | 20 |
| Disease severity at diagnosis | |
| VSAA | 11 |
| SAA | 16 |
| NSAA | 12 |
| PNH clone at the time of study | |
| Yes | 28 |
| No | 11 |
| Size of PNH clone, % (range) | |
| Red cells | 0.402 (0-87.3) |
| Granulocytes | 3.86 (0-96.7) |
| Monocytes | 4.517 (0-91.8) |
| Etiology | |
| Idiopathic | 39 |
| Response to treatment | |
| CR | 6 |
| PR | 19 |
| NR | 14 |
| Characteristic . | Value . |
|---|---|
| Number of patients | 39 |
| Median age, y (range) | 45 (20-72) |
| Sex | |
| M | 19 |
| F | 20 |
| Disease severity at diagnosis | |
| VSAA | 11 |
| SAA | 16 |
| NSAA | 12 |
| PNH clone at the time of study | |
| Yes | 28 |
| No | 11 |
| Size of PNH clone, % (range) | |
| Red cells | 0.402 (0-87.3) |
| Granulocytes | 3.86 (0-96.7) |
| Monocytes | 4.517 (0-91.8) |
| Etiology | |
| Idiopathic | 39 |
| Response to treatment | |
| CR | 6 |
| PR | 19 |
| NR | 14 |
Thirty-nine AA patients were recruited in this study, and PB samples were used for mass cytometry and/or in vitro experiments. AA patients who were eligible for IST were randomly selected and invited to participate in this study. A small to moderate PNH clone was detected in 28 patients, with no cases of hemolytic PNH. PBMCs from 16 patients (12 IST responders and 4 nonresponders) were used for initial CyTOF analysis; samples from another 15 patients (11 IST responders and 4 nonresponders) were used as a validation cohort. PBMCs from additional 8 patients were used for functional assays. Among nonresponder patients (n = 14), 7 underwent transplant later and the remaining were treated with cyclosporine A and supportive care.
CR, complete response; F, female; M, male; NR, non-response; NSAA, nonsevere aplastic anemia; PNH, paroxysmal nocturnal hemoglobinuria; PR: partial response; VSAA: very severe AA.