Skip to main content

Table 1 Characteristics of patient and donor (graft) and clinical outcomes between the two groups

From: Matched related transplantation versus immunosuppressive therapy plus eltrombopag for first-line treatment of severe aplastic anemia: a multicenter, prospective study

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)

 
  1. The bold values were statistically significant
  2. 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