Table 2 Transplantation outcomes

From: A multi-center clinical trial of allogeneic hematopoietic stem cell transplantation in transfusion-dependent thalassemia

Variable

Matched sibling donor

Total alternative donora

Matched unrelated donor

Haploidentical related donor

 

N = 331

N = 492

HR/OR (95% CI)

N = 352

HR/OR (95% CI)

N = 140

HR/OR (95% CI)

Overall survival, n

322

463

–

329

–

134

–

2-year OS (95% CI)

97.2% (95.4–99.0)

93.8% (91.6–96.0) *

2.35 (1.11–5.00)

93.1% (90.5–95.9) **

2.74 (1.25–5.99)

95.4% (91.9–99.1)

1.61 (0.57–4.55)

Event-free survival, n

322

461

–

328

–

133

–

2-year EFS (95% CI)

97.2% (95.4–99.0)

93.4%(91.1–95.7) *

2.57 (1.21–5.42)

92.9% (90.1–95.7) **

2.90 (1.33–6.32)

94.7% (90.9–98.6)

1.90 (0.70–5.14)

GRFS survival, n

302

382

–

274

–

108

–

2-year GRFS (95% CI)

91.4% (88.4–94.6)

76.6% (72.8–80.6) ***

2.88 (1.90, 4.37)

77.0% (72.6–81.7) ***

2.96 (1.91, 4.58)

75.6% (68.5–83.4) ***

2.72 (1.64, 4.54)

Transplant related mortality, n

8

27

–

21

–

6

–

2-year TRM (95% CI)

2.5% (0.8–4.2)

5.8% (3.7–7.9) *

2.42 (1.09, 5.34)

6.3% (3.7–9.9) *

2.74(1.20, 6.26)

4.6% (0.9–8.1)

1.81 (0.63, 5.24)

Mix chimerism

41 (12.4%)

13 (2.6%) ***

0.19 (0.10–0.35)

11 (3.1%) ***

0.23 (0.12–0.45)

2 (1.4%) **

0.10 (0.02–0.43)

GF

0 (0.0%)

4 (0.8%)

–

3 (0.9%)

–

1 (0.7%)

–

Neutrophil graft, days, median (IQR)

12 (11–14)

12 (11–14)

–

12 (11–13)

–

12 (11–14)

–

Platelet graft, days, median (IQR)

14 (12–20)

13 (11–16) ***

–

12.00 (11–15) ***##

–

14 (12–17)

–

Infection

243 (73.4%)

420 (85.4%) ***

2.11 (1.49–3.00)

291 (82.7%) **##

1.73 (1.19–2.50)

129 (92.1%) ***

4.24 (2.19–8.24)

Bacterial infection

94 (28.4%)

158 (32.1%)

1.19 (0.88–1.62)

114 (32.4%)

1.21 (0.87–1.68)

44 (31.4%)

1.16 (0.75–1.78)

Fungal infection

45 (13.6%)

65 (13.2%)

0.96 (0.64–1.46)

41 (11.6%)

0.84 (0.53–1.32)

24 (17.1%)

1.32 (0.77–2.26)

Pneumonia

98 (29.6%)

198 (40.2%) **

1.60 (1.19–2.16)

130 (36.9%) *#

1.39 (1.01–1.92)

68 (48.6%) ***

2.25 (1.50–3.37)

Septicemia

42 (12.7%)

57 (11.6%)

0.90 (0.59–1.38)

37 (10.5%)

0.81 (0.51–1.29)

20 (14.3%)

1.15 (0.65–2.04)

CMV reactivation

90 (27.1%)

220 (44.7%) ***

2.17 (1.60–2.93)

147 (41.8%) ***#

1.92 (1.39–2.65)

73 (52.1%) ***

2.92 (1.94–4.40)

EBV reactivation

57 (17.2%)

132 (26.8%) **

1.76 (1.24–2.51)

86 (24.4%) *

1.55 (1.07–2.26)

46 (32.9%) ***

2.35 (1.49–3.70)

VOD/SOS

8 (2.4%)

19 (3.9%)

1.62 (0.73–3.97)

15 (4.3%)

1.80 (0.75–4.30)

4 (2.9%)

1.19 (0.35–4.01)

HC

48 (14.5%)

142 (28.9%) ***

2.39 (1.67–3.47)

99 (28.1%) ***

2.31 (1.57–3.39)

43 (30.7%) ***

2.61 (1.63–4.19)

PRES

18 (5.4%)

32 (6.5%)

1.21 (0.67–2.19)

21(6.0%)

1.10 (0.68–2.11)

11 (7.9%)

1.48 (0.68–3.23)

aGvHDb, n

41

211

–

142

–

69

–

Cumulative incidence(95% CI)

12.4% (9.1–16.2)

42.9% (38.5–47.2) ***

4.20 (3.03–5.83)

40.4% (35.2–45.4) ***

3.90 (2.77–5.48)

49.3% (40.7–57.3) ***

5.02 (3.44–7.34)

Grades 2–4 aGvHDb, n

24

129

–

88

–

41

–

Cumulative incidence (95% CI)

7.5% (4.9–10.7)

28.9% (24.7–33.2) ***

4.34 (2.85–6.61)

27.6% (22.8–32.6)***

4.08 (2.64–6.32)

32.3% (24.2–40.6) ***

5.03 (3.07–8.24)

Grades 3–4 aGvHDb, n

6

60

–

43

–

17

–

Cumulative incidence (95% CI)

1.9% (0.8–3.9)

14.6% (11.4–18.2) ***

7.09 (3.25–15.40)

14.6% (10.8–18.9) ***

6.99 (3.15–15.50)

14.7% (8.9–21.9) ***

7.36 (3.06–17.70)

cGvHDc, n

26

83

–

57

–

26

–

Cumulative incidence (95% CI)

8.5% (5.7–12.0)

20.1% (16.3–24.2) ***

2.23(1.45–3.43)

19.1% (14.8–23.9) **

2.14 (1.36–3.36)

22.6% (15.2–30.8) ***

2.48 (1.46–4.23)

Moderate-severe cGvHDc, n

15

50

–

34

–

16

–

Cumulative incidence (95% CI)

5.0% (2.9–7.9)

12.3% (9.2–15.7) **

2.23(1.29–3.86)

11.2% (7.9–15.2)*

2.12 (1.19–3.78)

15.1% (8.9–22.9) **

2.52 (1.28–4.96)

  1. For the OS, EFS, GRFS, and TRM, time-to-event rates were estimated using the Kaplan-Meier method, and hazard ratios were estimated using the Cox proportional-hazards model with centers as a random-effect. For GF, comparisons were made using Fisher’s exact test. For neutrophil and platelet graft, data are summarized as medians and interquartile ranges, and made comparisons with Kruskal–Wallis test or Wilcoxon rank-sum test. For complications such as mixed chimerism, VOD/SOS, HC, PRES and infection events, the odds ratio was estimated using the Logistic model. In the presence of competing risks, the Fine and Gray model was used for GvHD. All statistical tests are two-sided.
  2. *denotes comparison with Matched Sibling Donor, #denotes comparison with Haploidentical Related Donor: */#p <0.05, **/##p < 0.01, ***/###p < 0.001.
  3. OS overall survival, HR hazard ratio, CI confidence interval, EFS event-free survival, TRM transplant related mortality, OR odds ratio, GF graft failure, IQR Interquartile range, CMV cytomegalovirus, EBV Epstein-Barr virus, VOD/SOS hepatic veno-occlusive disease/sinusoidal obstruction syndrome, HC hemorrhagic cystitis, PRES posterior reversible encephalopathy syndrome, aGvHD acute graft versus host disease, cGvHD chronic graft versus host disease.
  4. aAlternative Donors includes Matched Unrelated Donors and Haploidentical Donors.
  5. baGVHD at 100 days post transplantation. For grades aGvHD, GF, death and relapse without corresponding grade aGvHD were considered as competing risks.
  6. ccGVHD at 2 years post transplantation. For cGvHD and moderate-severe cGvHD, GF, death, relapse and loss to follow-up without corresponding grade cGvHD were considered as competing risks.
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