Table 1.
Patient characteristics, clinical features and transplant outcomes (SAA = severe aplastic anemia; MDS = myelodysplastic syndrome; TD = transfusion-dependent on red cells and/or platelets; ATG = antithymocyte globulin; CSA = cyclosporine; MMF = mycophenolate mofetil; MTX = methotrexate; SR = steroid-refractory; IST = immunosuppressive therapy; TRM = transplant related mortality; IPSS = International Prognostic Scoring System; Int-1 = Intermediate-1).
| Patient | Age at transplant |
Sex | Diagnosis | Clinical Features | Pre-transplant HLA- alloimmunization (%PRA) |
Pre- transplant %GPI- negative neutrophils |
Equine ATG with conditioning |
GVHD Prophylaxis |
Acute GVHD |
Chronic GVHD |
Follow- up (years) |
Discontinuation of IST (years) |
Survival Status |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 31 | M | PNH | Hemolysis and thrombosis (portal vein), TD | Yes (46%) | 92.0 | Yes | CSA | None | Extensive | 11.0 | 8.6 | Alive |
| 2 | 25 | F | PNH | Hemolysis and cytopenia, TD | Yes (68%) | 97.0 | Yes | CSA+MMF | None | Limited | 9.2 | 1.0 | Alive |
| 3 | 35 | M | PNH | Hemolysis and cytopenia, TD | No | 92.0 | Yes | CSA+MMF | Grade 3–4, SR | Extensive | 2.8 | On IST at last follow-up | Dead |
| 4 | 27 | F | PNH | Hemolysis and cytopenia, TD | Yes(45%) | 13.0 | Yes | CSA+MMF | Grade 3–4 | Limited | 8.2 | 3.1 | Alive |
| 5 | 35 | M | PNH | Hemolysis and thrombosis (portal vein) | No | 92.0 | Yes | CSA+MMF | None | None | 2.6 | Not applicable | Alive |
| 6 | 31 | M | PNH/SAA | Cytopenia, TD | No | 91.0 | Yes | CSA+MTX | Grade 3–4, SR | Extensive | 7.3 | 5.5 | Alive |
| 7 | 42 | F | PNH/MDS | Cytopenia and IPSS Int-1MDS t(1;13), TD | Yes (98%) | 86.2 | Yes | CSA+MTX | None | Extensive | 6.7 | 5.1 | Alive |
| 8 | 21 | M | PNH | Cytopenia, TD | Yes (95%) | 22.0 | Yes | CSA+MTX | Grade 3–4, SR | Limited | 6.0 | 2.2 | Alive |
| 9 | 34 | M | PNH | Hemolysis and thrombosis (cerebrovascular) | No | 99.0 | No | CSA+MTX | None | Extensive | 5.9 | On IST at last follow-up | Alive |
| 10 | 32 | M | PNH | Hemolysis | No | 74.0 | No | CSA+MTX | Grade 3–4, SR | Extensive | 5.9 | On IST at last follow-up | Alive |
| 11 | 34 | F | PNH | Hemolysis and cytopenia, TD | Yes (85%) | 56.0 | Yes | CSA+MTX | Grade 3–4, SR | Not applicable | 169 (days) | Not applicable | Dead(acute GVHD TRM) |
| 12 | 33 | M | PNH | Hemolysis, TD | Yes (100%) | 99.0 | Yes | CSA+MTX | None | None | 5.6 | Not applicable | Alive |
| 13 | 23 | M | PNH/MDS | Cytopenia and IPSS Int-1 MDS del13q, TD | Yes (55%) | 5.5 | Yes | CSA+MTX | Grade 3–4, SR | Extensive | 3.4 | On IST at last follow-up | Alive |
| 14 | 20 | M | PNH/SAA | Cytopenia, TD | No | 46.0 | Yes | CSA+MTX | None | None | 3.4 | Not applicable | Alive |
| 15 | 24 | M | PNH/SAA | Cytopenia, TD | No | 81.6 | Yes | CSA+MTX | None | Limited | 4.0 | 2.1 | Alive |
| 16 | 37 | M | PNH/SAA | Hemolysis, TD | No | 41.0 | Yes | CSA+MTX | None | None | 3.9 | Not applicable | Alive |
| 17 | 20 | M | PNH | Hemolysis | No | 71.2 | No | CSA+MTX | Grade 2 | None | 3.3 | Not applicable | Alive |