Table 1.
Patient and Transplantation Characteristics
| Characteristic | Controls (Noncryopreserved Graft) | Cases (Cryopreserved Graft) | P Value |
|---|---|---|---|
| Number | 195 | 52 | |
| Age, yr, median (range) | 22 (4-67) | 21 (5-64) | .96 |
| Age 1-17 yr, n (%) | 77 (40) | 21 (40) | .95 |
| Age 18-39 yr, n (%) | 72 (37) | 18 (35) | |
| Age ≥40 yr, n (%) | 46 (23) | 13 (25) | |
| Sex, male/female, n (%) | 115 (59)/80 (41) | 22 (42)/30 (58) | .03 |
| Performance score, n (%) | .72 | ||
| 90-100 | 128 (66) | 31 (60) | |
| ≤80 | 61 (31) | 19 (37) | |
| Not reported | 6 (3) | 2 (3) | |
| Comorbidity score, n (%) | .55 | ||
| ≤2 | 136 (70) | 34 (65) | |
| ≥3 | 59 (30) | 18 (35) | |
| Cytomegalovirus serostatus, n (%) | .13 | ||
| Negative | 58 (30) | 10 (19) | |
| Positive | 137 (70) | 42 (81) | |
| Donor type, n (%)* | .97 | ||
| HLA-matched sibling | 79 (41) | 21 (41) | |
| HLA-haploidentical | 25 (13) | 8 (15) | |
| HLA-matched unrelated | 64 (33) | 16 (31) | |
| HLA-mismatched unrelated | 27 (14) | 7 (14) | |
| Donor-recipient ABO match, n (%) | .86 | ||
| Matched | 63 (32) | 19 (37) | |
| Minor mismatch | 22 (11) | 6 (11) | |
| Major mismatch | 31 (16) | 6 (11) | |
| Not reported | 79 (41) | 21 (41) | |
| Conditioning regimen, n (%)† | .15 | ||
| Cy + ATG | 59 (30) | 11 (21) | |
| Flu + Cy + ATG | 18 (9) | 6 (12) | |
| Bu/Cy ± ATG | 4 (2) | 3 (6) | |
| Flu + TBI (200 cGy) | 1 (1) | 1 (2) | |
| Cy + ATG + TBI (200 cGy) | 38 (19) | 8 (15) | |
| Cy + ATG + TBI (1000 cGy) | 0 | 1 (2) | |
| Flu + Cy + ATG + TBI (200 cGy) | 55 (28) | 13 (25) | |
| Flu + Bu ± ATG | 8 (4) | 5 (9) | |
| Flu + melphalan + thiotepa + ATG | 2 (1) | 2 (4) | |
| Flu + melphalan | 10 (5) | 2 (4) | |
| Graft type, n (%) | .56 | ||
| Bone marrow | 132 (68) | 33 (64) | |
| Peripheral blood | 63 (32) | 19 (36) | |
| Bone marrow TNC dose (× 108/kg), median (IQR) | |||
| Pre-cryopreservation | Not applicable | 3.83 (2.70-5.07) (n = 19 of 33) | |
| Infusion | 3.40 (2.45-4.57) (n = 109 of 132) | 2.63 (1.49-3.05) (n = 23 of 33) | .004 |
| Peripheral blood CD34+ cell dose (× 106/kg), median (IQR) | |||
| Pre-cryopreservation | Not applicable | 7.90 (7.14-8.74) (n = 15 of 19) | |
| Infusion | 6.63 (4.78-10.97) (n = 62 of 63) | 5.38 (3.78-10.97) (n = 15 of 19) | .45 |
| GVHD prophylaxis, n (%) | .07 | ||
| Ex vivo T cell depletion or CD34+ | 18 (9) | 4 (7) | |
| Post-transplantation Cy + other | 22 (11) | 6 (12) | |
| Calcineurin inhibitor + MMF | 21 (11) | 14 (27) | |
| Calcineurin inhibitor + MTX | 110 (56) | 25 (48) | |
| Calcineurin inhibitor + other | 21 (11) | 2 (4) | |
| Other agents | 3 (2) | 1 (2) | |
| Interval from diagnosis to HCT, n (%) | .28 | ||
| ≤3 mo‡ | 44 (22) | 8 (15) | |
| 3-6 mo§ | 41 (21) | 15 (29) | |
| 7-12 mo║ | 42 (22) | 15 (29) | |
| >12 mo¶ | 68 (35) | 14 (27) | |
| Transplantation period, n (%) | .16 | ||
| 2013-2015 | 103 (53) | 20 (39) | |
| 2016-2019 | 92 (47) | 32 (61) |
Cy indicates cyclophosphamide; ATG, antithymocyte globulin; Flu, fludarabine; TBI, total body irradiation ; MMF, mycophenolate mofetil; MTX, methotrexate.
*Donor age, yr, median (range):
haploidentical: controls, 32 (10-65); cases, 36 (14-65); unrelated: controls, 27 (18-59); cases, 30 (19-43).
†Cyclophosphamide dosing:
Cy + ATG:
cases, 200 mg/kg (n = 11); controls, 200 mg/kg (n = 56), 120 mg/kg (n = 3);
Flu + Cy + ATG:
cases, 120 mg/kg (n = 5), 60 mg/kg (n = 1); controls, 120 mg/kg (n = 15), 60 mg/kg (n = 3);
Bu + Cy:
cases, 200 mg/kg (n = 1), 120 mg/kg (n = 2); controls, 200 mg/kg (n = 2), 120 mg/kg (n = 2);
Cy + ATG + TBI (200 cGy):
cases, 200 mg/kg (n = 6), 29 mg/kg (n = 2); controls, 200 mg/kg (n = 22), 120 mg/kg (n = 1), 100 mg/kg (n = 4), 50 mg/kg (n = 2), 29 mg/kg (n = 8), unknown (n = 1);
Cy + ATG + TBI (1000 cGy):
cases, 120 mg/kg (n = 1); Flu + Cy + ATG + TBI (200 cGy): cases, 100 mg/kg (n = 4), 50 mg/kg (n = 4), 29 mg/kg (n = 5); controls, 100 mg/kg (n = 19), 50 mg/kg (n = 15), 29 mg/kg (n = 19). Interval between diagnosis and HCT:
‡77% HLA-matched sibling transplant; 23% HLA-matched or mismatched unrelated donor transplant.
§55% HLA-matched sibling transplant; 30% HLA-matched or mismatched unrelated donor transplant; 14% HLA-haploidentical transplant.
║23% HLA-matched sibling transplant; 59% were HLA-matched or mismatched unrelated donor transplant; 19% HLA-haploidentical transplant.
¶20% HLA-matched sibling transplant; 63% HLA-matched or mismatched unrelated donor transplant; 17% HLA-haploidentical transplant.