Table 1

Patients’ characteristics

CharacteristicValue
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 
 PR 19 
 NR 14 
CharacteristicValue
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 
 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.

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