Table 1.
Characteristics of patient and donor (graft) and clinical outcomes between the two groups
| Variables | MRD-HSCT (n = 108) | IST + EPAG (n = 104) | P |
|---|---|---|---|
| Median age, yr (range) | 29 (6–56) | 34.5 (4–69) | 0.024 |
| Age, no. (%) | < 0.001 | ||
| < 20 yr | 17 (15.7) | 24 (23.1) | |
| 20–40 yr | 67 (62.0) | 32 (30.8) | |
| ≥ 40 yr | 24 (22.2) | 48 (46.2) | |
| Sex, no. (%) | 0.767 | ||
| Male | 57 (52.8) | 57 (54.8) | |
| Female | 51 (47.2) | 47 (45.2) | |
| Disease status, no. (%) | 0.831 | ||
| SAA | 68 (66.7) | 64 (61.5) | |
| vSAA | 40 (33.3) | 40 (38.5) | |
| With PNH clone, no. (%) | 26 (24.1) | 19 (10.2) | 0.301 |
| ECOG score, median (range) | 1 (0–2) | 1 (0–2) | 0.537 |
| Median time from diagnosis to treatment, mth (range) | 3 (1.0–200) | 2 (0.5–240) | 0.011 |
| Median time to an ANC ≥ 1.0 × 109/L, d (range) | 15 (11–35) | 30 (4–58) | 0.002 |
| Median time to transfusion independence for RBCs, d (range) | 22 (13–32) | 63 (6–302) | < 0.001 |
| Median time to transfusion independence for platelets, d (range) | 12 (8–52) | 52 (11–287) | < 0.001 |
| Normal blood routine at 6-mth, no. (%) | 83 (86.5) | 23 (23.7) | < 0.001 |
| Early death, no (%) | 7 (6.5) | 2 (1.9) | 0.192 |
| Secondary clonal disease, no (%) | 1 (0.9) | 2 (2.9) | 0.587 |
| Relapsed, no (%) | 0 (0.0) | 1 (0.9) | – |
| Alternative donor transplantation, no (%) | 0 (0.0) | 10 (9.6) | – |
| TRM, no (%) | 17 (15.7) | 10 (9.6) | 0.181 |
| Secondary GF, no (% of TRM) | 1 (5.9) | – | |
| aGVHD, no (% of TRM) | 1 (5.9) | – | |
| cGVHD, no (% of TRM) | 1 (5.9) | – | |
| TMA, no (% of TRM) | 2 (11.8) | – | |
| Poor graft function, no (% of TRM) | 1 (5.9) | – | |
| Infection, no (% of TRM) | 9 (52.8) | 5 (50.0) | |
| Intracranial hemorrhage, no (% of TRM) | 1 (5.9) | 2 (20.0) | |
| Heart failure, no (% of TRM) | – | 1 (10.0) | |
| Other, no (% of TRM) | 1 (5.9) | 2 (20.0) | |
| Median follow-up time among living patients, mth (range) | 31.5 (13.0–69.0) | 30.5 (14.0–66.0) | 0.589 |
| Conditioning regimen | Flu + CY + ATG | rATG (pALG) + CsA + EPAG | |
| Donor median age, yr (range) | 30 (10–55) | – | |
| Donor sex, no. (%) | – | ||
| Male | 54 (50.0) | – | |
| Female | 54 (50.0) | – | |
| Blood types of donor to recipient, no. (%) | |||
| Matched | 65 (60.2) | – | |
| Major mismatched | 15 (13.9) | – | |
| Minor mismatched | 19 (17.6) | – | |
| Major and minor mismatched | 9 (8.3) | – | |
| Source of graft, no. (%) | |||
| BM | 4 (3.7) | – | |
| PB | 30 (27.8) | – | |
| BM + PB | 74 (68.5) | – | |
| Median MNC, × 108/kg (range) | 11.6 (3.2–24.4) | – | |
| Median CD34+ cells, × 106/kg (range) | 3.7 (1.1–8.6) | – | |
| Median time to ANC > 0.5 × 109/L, d (range) | 11 (7–21) | – | |
| Median time to PLT > 20.0 × 109/L, d (range) | 12 (8–52) | – | |
| Primary GF, no. (%) | 0 (0.0) | – | |
| Secondary GF, no. (%) | 2 (1.9) | – | |
| GF of platelet, no. (%) | 3 (2.9) | – | |
| Delayed platelet recovery, no. (%) | 4 (3.8) | – | |
| Poor graft function, no. (%) | 1 (0.9) | – | |
| Adverse events of attributed to EPAG, no. (%) | |||
| Skin (maculopapular and/or rash pruritus) | – | 3 (2.9) | |
| Abdominal pain | – | 2 (1.9) | |
| Joint pain | – | 2 (1.9) | |
| Liver test abnormality | |||
| Increased aminotransferase level | – | 42 (40.4) | |
| Increased blood bilirubin level | – | 19 (18.3) |
The bold values were statistically significant
MRD-HSCT matched related donor hematopoietic stem cell transplantation, IST immunosuppressive therapy, EPAG eltrombopag, SAA severe aplastic anemia, vSAA very SAA, PNH paroxysmal nocturnal hemoglobinuria, ECOG Eastern Cooperative Oncology Group Scale, BM bone marrow, PB peripheral blood, MNC mononuclear cell, ANC absolute neutrophil count, PLT platelet, GF graft failure, TRM treatment related mortality