Table 2.
Patient | Sex/Age (yrs) | Initial Diagnosis | Follow-up (mos) | PNH size (%) (initial/current)* | LDH (mg/dl) (initial/current) | Therapy | Post-treatment AA status |
---|---|---|---|---|---|---|---|
1 | F/41 | SAA | 114 | 0.5 /0.2 | 87/ 98 | HiCy | Remission |
2 | F/22 | SAA | 129 | 0.16/50 | 186/628 | HiCy | Remission for 108 mos, then transformed to PNH |
3 | F/68 | vSAA | 86 | 22/0.18 | 178/172 | HiCy | Remission |
4 | F/15 | SAA | 87 | 24 /31 | 382/527 | HiCy | Remission for 84 mos, then transformed to PNH |
5 | M/37 | SAA | 78 | 12.3 / 14 | 390/501 | HiCy | Remission |
6 | M/9 | SAA | 109 | 66.5/96.7 | 536/507 | HiCy | Remission for 60 mos, then asymptomatic PNH expansion |
7 | F/19 | SAA | 63 | 0.1/38 | 181/447 | HiCy | Remission |
8 | F/19 | SAA | 52 | 0.12/23 | 193/285 | HiCy | Remission for 52 mos, then AA relapse |
9 | M/22 | SAA | 50 | 5/0.8 | 198/162 | HiCy | Remission |
10 | M/36 | SAA | 49 | 31/3.9 | 171/190 | HiCy | Remission |
11 | M/20 | SAA | 37 | 0.06/1 | 200/ 269 | HiCy | Remission |
12 | F/32 | SAA | 38 | 51.67/40.7 | 237/191 | HiCy | Remission with asymptomatic PNH expansion |
13 | M/20 | SAA | 18 | 0.34/ 0.46 | 180/ 172 | HiCy | Remission |
14 | M/24 | SAA | 30 | 17/ 84 | 302/ 912 | HiCy | AA relapse after 3 mos remission, then transformed to PNH after 18 mos |
15 | F/52 | SAA | 39 | 23/72 | 314/229/188 | HiCy, then BMT | Refractory, then remission post BMT |
16 | F/34 | SAA | 138 | 0.14/3 | 235/182 | CSA, then HiCy | AA relapse after 6 mos remission, then remission again post HiCy |
17 | F/18 | vSAA | 123 | 6/ 26 | 147/ 266 | Prednisone, then HiCy | Refractory, then remission post HiCy |
18 | F/10 | SAA | 134 | 0.02 / 0 | 178/208 | ATG/CSA, then HiCy | AA relapse after 30 mos remission, then remission again post HiCy |
19 | F/31 | SAA | 82 | 57 / 62.2 | 267/ 395 | ATG/CSA, then HiCy | Refractory, then remission post HiCy |
20 | M/41 | SAA | 81 | 0.12/1 | 174/ 171 | ATG/CSA, then HiCy twice | AA relapse after 7 mos remission, then relapse again 72 mos after HiCy, then remission again post second HiCy |
21 | F/62 | SAA | 60 | 15/6.2 | 189/189 | CSA, then HiCy | Refractory, then remission post HiCy |
22 | F/19 | SAA | 49 | 60/1 | 353/ 306 | CSA, then HiCy | Refractory, then remission post HiCy |
23 | F/46 | NSAA | 131 | 0.5 /6 | 223/ 235 | CSA | Remission |
24 | F/27 | SAA | 38 | 17/50 | 307/ 600 | ATG/CSA | Remission for 30 mos, then asymptomatic PNH expansion |
25 | M/39 | NSAA | 84 | 0.3/1.5 | 206/231 | No treatment | Self recovery |
26 | F/17 | NSAA | 56 | 15/4.2 | 419/ 189 | No treatment | Self recovery |
27 | F/48 | NSAA | 36 | 25/51 | 512/ 606 | No treatment | Asymptomatic PNH expansion |
Remission was classified as HCT ≥30%/independence of transfusion/ANC≥500/cumm/Platelet count≥20K/cumm. PNH transformation was classified as presenting classical PNH symptoms and developing apparent hemolysis with LDH value >1.5x of normal upper limit.
initial PNH size means the PNH size before treatment, and current PNH size means the PNH size in the most recent visit.