Table 1.
Patient baseline characteristics
| Variable | Malignancies | Nonmalignant | ||||
|---|---|---|---|---|---|---|
| Bu + Cy | Bu + Flu | P | Bu + Cy | Bu + Flu | P | |
| Number of enrolled patients | 773 | 205 | 627 | 176 | ||
| Number of centers | 105 | 64 | 66 | 45 | ||
| Age at transplant, y | .43 | .95 | ||||
| Median (range) | 10 (<1-18) | 11 (<1-18) | 5 (<1-18) | 3 (<1-18) | ||
| 1-9 | 371 (48) | 92 (45) | 501 (80) | 141 (80) | ||
| 10-20 | 402 (52) | 113 (55) | 126 (20) | 35 (20) | ||
| Sex | .06 | .15 | ||||
| Male | 414 (54) | 125 (61) | 361 (58) | 112 (64) | ||
| Female | 359 (46) | 80 (39) | 266 (42) | 64 (36) | ||
| Lansky performance score | .41 | .02 | ||||
| ≥90% | 672 (87) | 173 (84) | 499 (80) | 150 (85) | ||
| <90% | 89 (12) | 30 (15) | 119 (19) | 20 (11) | ||
| Missing | 12 (2) | 2 | 9 (1) | 6 (3) | ||
| Primary disease | .10 | .10 | ||||
| AML | 515 (67) | 138 (67) | ||||
| ALL | 37 (5) | 15 (7) | ||||
| CML | 77 (10) | 13 (6) | ||||
| MDS | 117 (15) | 33 (16) | ||||
| MPN | 14 (2) | 0 | ||||
| Other acute leukemia | 13 (2) | 6 (3) | ||||
| Hemoglobinopathies | 322 (51) | 48 (27) | ||||
| Primary Immunodeficiencies | 195 (31) | 86 (49) | ||||
| Inherited abnormalities of platelets | 10 (2) | 2 (1) | ||||
| Inherited disorder of metabolism | 65 (10) | 24 (14) | ||||
| Histiocytic disorders | 35 (6) | 16 (9) | ||||
| HCT-CI | <.001 | <.001 | ||||
| 0 | 511 (66) | 109 (53) | 347 (55) | 98 (56) | ||
| 1-2 | 107 (14) | 35 (17) | 60 (10) | 29 (16) | ||
| ≥3 | 61 (8) | 36 (18) | 37 (6) | 22 (13) | ||
| Missing | 94 (12) | 25 (12) | 183 (29) | 27 (15) | ||
| Disease status* | .036 | |||||
| Early | 433 (56) | 95 (46) | ||||
| Intermediate | 125 (16) | 49 (24) | ||||
| Advanced | 106 (14) | 29 (14) | ||||
| Unknown | 109 (16) | 32 (16) | ||||
| Donor type | .004 | <.001 | ||||
| HLA-matched related | 444 (57) | 95 (46) | 503 (80) | 82 (47) | ||
| 8/8 URD | 239 (31) | 71 (35) | 96 (15) | 72 (41) | ||
| 7/8 URD | 90 (12) | 39 (19) | 28 (4) | 22 (13) | ||
| Graft type | <.001 | .004 | ||||
| Bone marrow | 686 (89) | 136 (66) | 614 (98) | 165 (94) | ||
| Peripheral blood stem cells | 87 (11) | 69 (34) | 13 (2) | 11 (6) | ||
| Route of busulfan administration | .35 | <.001 | ||||
| Oral | 63 (8) | 11 (5) | 73 (12) | 1 (<1) | ||
| IV | 709 (92) | 194 (95) | 552 (88) | 175 (99) | ||
| Unknown | 1 (<1) | 0 | 2 (<1) | 0 | ||
| GVHD prophylaxis | <.001 | <.001 | ||||
| TAC+MMF | 27 (3) | 21 (10) | 30 (5) | 10 (6) | ||
| TAC+MTX | 180 (23) | 94 (46) | 67 (11) | 33 (19) | ||
| CSA+MMF | 26 (3) | 16 (8) | 33 (5) | 27 (15) | ||
| CSA+MTX | 455 (59) | 65 (32) | 418 (67) | 85 (48) | ||
| Others | 85 (11) | 9 (4) | 79 (13) | 21 (12) | ||
| Antithymocyte globulin/alemtuzumab | <.001 | <.001 | ||||
| No | 529 (68) | 90 (44) | 194 (31) | 41 (23) | ||
| Yes | 219 (28) | 106 (52) | 396 (63) | 94 (53) | ||
| Missing | 25 (3) | 9 (4) | 37 (6) | 41 (23) | ||
| Year of transplant | <.001 | <.001 | ||||
| 2008 | 130 (17) | 8 (4) | 100 (16) | 7 (4) | ||
| 2009 | 128 (17) | 10 (5) | 103 (16) | 7 (4) | ||
| 2010 | 129 (17) | 16 (8) | 89 (14) | 17 (10) | ||
| 2011 | 107 (14) | 27 (13) | 102 (16) | 19 (11) | ||
| 2012 | 98 (13) | 38 (19) | 106 (17) | 27 (15) | ||
| 2013 | 88 (11) | 40 (20) | 67 (11) | 46 (26) | ||
| 2014 | 93 (12) | 66 (32) | 60 (10) | 53 (30) | ||
| Median follow-up of survivors, months (range) | 60 (3-94) | 26 (6-86) | 62 (6-79) | 24 (3-96) | ||
Values are n (%) unless otherwise noted.
ALL, acute lymphoblastic leukemia; CML, chronic myelogenous leukemia; CSA, cyclosporine A; MDS, myelodysplasia; MMF, mycophenolate mofetil; MPN, myeloproliferative neoplasm; MTX, methotrexate; URD, unrelated donor.
*
Disease status definition: early, AML/ALL in first complete remission, CML in chronic phase or MDS excluding refractory anemia with excess blasts; intermediate, AML/ALL in any complete remission beyond first or CML in accelerated phase any time before transplant; and advanced, AML/ALL with primary induction failure or relapse, CML in blastic crisis any time before transplant or MDS with refractory anemia with excess blasts.