Patient and transplant characteristics
. | . | . | Regimen . | ||||
---|---|---|---|---|---|---|---|
Characteristic . | Entire cohort . | %* . | BuFlu±TT . | % . | TreoFlu±TT . | % . | P . |
Patients | 197 | 100 | 103 | 52.3 | 94 | 47.7 | NA |
Follow-up, median (IQR), mo | 44.9 (25.5-70.4) | NA | 44.8 (24.0-60.2) | NA | 46.4 (26.0-80.1) | NA | NA |
Age at HSCT, median (IQR), y | 1.6 (1.0-3.1) | NA | 1.6 (0.9-3.9) | NA | 1.6 (1.1-2.5) | NA | .833† |
<5 y | 165 | 83.8 | 84 | 81.6 | 81 | 86.2 | .404 |
≥5 to <18 y | 24 | 12.2 | 13 | 12.6 | 11 | 11.7 | |
≥18 y | 8 | 4.1 | 6 | 5.8 | 2 | 2.1 | |
WAS score at HSCT‡ | |||||||
1-2 | 24 | 17.8 | 6 | 8.7 | 18 | 27.3 | .011 |
3-4 | 71 | 52.6 | 43 | 62.3 | 28 | 42.4 | |
5 | 40 | 29.6 | 20 | 29.0 | 20 | 30.3 | |
Missing | 62 | ||||||
Pre-HSCT autoimmunity | |||||||
Yes | 61 | 38.9 | 31 | 36.9 | 30 | 41.1 | .625 |
No | 96 | 61.1 | 53 | 63.1 | 43 | 58.9 | |
Missing | 40 | ||||||
Conditioning regimen | |||||||
No thiotepa | 155 | 78.7 | 86 | 83.5 | 69 | 73.4 | .12 |
Thiotepa | 42 | 21.3 | 17 | 16.5 | 25 | 26.6 | |
Serotherapy | |||||||
Yes | 161 | 81.7 | 77 | 74.8 | 84 | 89.4 | .014 |
Antithymocyte globulin Alemtuzumab | 96 65 | 48.7 33.0 | 53 24 | 51.5 23.3 | 43 41 | 45.7 43.6 | |
No | 36 | 18.3 | 26 | 25.2 | 10 | 10.6 | |
Donor | |||||||
MSD/MFD | 39 | 20.0 | 26 | 25.5 | 13 | 14.0 | .093 |
10/10 MUD | 86 | 44.1 | 39 | 38.2 | 47 | 50.5 | |
9/10 MUD | 28 | 14.4 | 15 | 14.7 | 13 | 14 | |
<9/10 MMUD | 14 | 7.2 | 10 | 9.8 | 4 | 4.3 | |
MMFD§ | 28 | 14.4 | 12 | 11.8 | 16 | 17.2 | |
Missing | 2 | ||||||
Stem cell source | |||||||
Bone marrow | 103 | 52.6 | 61 | 59.2 | 42 | 45.2 | .031 |
Peripheral blood | 72 | 36.7 | 29 | 28.2 | 43 | 46.2 | |
Cord blood | 21 | 10.7 | 13 | 12.6 | 8 | 8.6 | |
Missing | 1 | ||||||
Cytomegalovirus(recipient/donor) | |||||||
–/– | 49 | 26.8 | 20 | 20.8 | 29 | 33.3 | .244 |
–/+ | 26 | 14.2 | 16 | 16.7 | 10 | 11.5 | |
+/– | 29 | 15.8 | 15 | 15.6 | 14 | 16.1 | |
+/+ | 79 | 43.2 | 45 | 46.9 | 34 | 39.1 | |
Missing | 14 | ||||||
Period of HSCT | |||||||
2014-2017 | 86 | 43.7 | 55 | 53.4 | 31 | 32.9 | .006 |
2006-2013 | 111 | 56.3 | 48 | 46.6 | 63 | 67.1 | |
Graft manipulation | |||||||
No | 149 | 76.0 | 79 | 76.7 | 70 | 75.3 | .947 |
Yes | 47 | 24.0 | 24 | 23.3 | 23 | 24.7 | |
Missing | 1 | ||||||
GVHD prophylaxis | |||||||
CNI/mycophenolate mofetil | 87 | 44.2 | 37 | 35.9 | 50 | 53.2 | .087 |
CNI/methotrexate | 52 | 26.4 | 33 | 32.0 | 19 | 20.2 | |
Other | 45 | 22.8 | 25 | 24.3 | 20 | 21.3 | |
None | 13 | 6.6 | 8 | 7.8 | 5 | 5.3 |
. | . | . | Regimen . | ||||
---|---|---|---|---|---|---|---|
Characteristic . | Entire cohort . | %* . | BuFlu±TT . | % . | TreoFlu±TT . | % . | P . |
Patients | 197 | 100 | 103 | 52.3 | 94 | 47.7 | NA |
Follow-up, median (IQR), mo | 44.9 (25.5-70.4) | NA | 44.8 (24.0-60.2) | NA | 46.4 (26.0-80.1) | NA | NA |
Age at HSCT, median (IQR), y | 1.6 (1.0-3.1) | NA | 1.6 (0.9-3.9) | NA | 1.6 (1.1-2.5) | NA | .833† |
<5 y | 165 | 83.8 | 84 | 81.6 | 81 | 86.2 | .404 |
≥5 to <18 y | 24 | 12.2 | 13 | 12.6 | 11 | 11.7 | |
≥18 y | 8 | 4.1 | 6 | 5.8 | 2 | 2.1 | |
WAS score at HSCT‡ | |||||||
1-2 | 24 | 17.8 | 6 | 8.7 | 18 | 27.3 | .011 |
3-4 | 71 | 52.6 | 43 | 62.3 | 28 | 42.4 | |
5 | 40 | 29.6 | 20 | 29.0 | 20 | 30.3 | |
Missing | 62 | ||||||
Pre-HSCT autoimmunity | |||||||
Yes | 61 | 38.9 | 31 | 36.9 | 30 | 41.1 | .625 |
No | 96 | 61.1 | 53 | 63.1 | 43 | 58.9 | |
Missing | 40 | ||||||
Conditioning regimen | |||||||
No thiotepa | 155 | 78.7 | 86 | 83.5 | 69 | 73.4 | .12 |
Thiotepa | 42 | 21.3 | 17 | 16.5 | 25 | 26.6 | |
Serotherapy | |||||||
Yes | 161 | 81.7 | 77 | 74.8 | 84 | 89.4 | .014 |
Antithymocyte globulin Alemtuzumab | 96 65 | 48.7 33.0 | 53 24 | 51.5 23.3 | 43 41 | 45.7 43.6 | |
No | 36 | 18.3 | 26 | 25.2 | 10 | 10.6 | |
Donor | |||||||
MSD/MFD | 39 | 20.0 | 26 | 25.5 | 13 | 14.0 | .093 |
10/10 MUD | 86 | 44.1 | 39 | 38.2 | 47 | 50.5 | |
9/10 MUD | 28 | 14.4 | 15 | 14.7 | 13 | 14 | |
<9/10 MMUD | 14 | 7.2 | 10 | 9.8 | 4 | 4.3 | |
MMFD§ | 28 | 14.4 | 12 | 11.8 | 16 | 17.2 | |
Missing | 2 | ||||||
Stem cell source | |||||||
Bone marrow | 103 | 52.6 | 61 | 59.2 | 42 | 45.2 | .031 |
Peripheral blood | 72 | 36.7 | 29 | 28.2 | 43 | 46.2 | |
Cord blood | 21 | 10.7 | 13 | 12.6 | 8 | 8.6 | |
Missing | 1 | ||||||
Cytomegalovirus(recipient/donor) | |||||||
–/– | 49 | 26.8 | 20 | 20.8 | 29 | 33.3 | .244 |
–/+ | 26 | 14.2 | 16 | 16.7 | 10 | 11.5 | |
+/– | 29 | 15.8 | 15 | 15.6 | 14 | 16.1 | |
+/+ | 79 | 43.2 | 45 | 46.9 | 34 | 39.1 | |
Missing | 14 | ||||||
Period of HSCT | |||||||
2014-2017 | 86 | 43.7 | 55 | 53.4 | 31 | 32.9 | .006 |
2006-2013 | 111 | 56.3 | 48 | 46.6 | 63 | 67.1 | |
Graft manipulation | |||||||
No | 149 | 76.0 | 79 | 76.7 | 70 | 75.3 | .947 |
Yes | 47 | 24.0 | 24 | 23.3 | 23 | 24.7 | |
Missing | 1 | ||||||
GVHD prophylaxis | |||||||
CNI/mycophenolate mofetil | 87 | 44.2 | 37 | 35.9 | 50 | 53.2 | .087 |
CNI/methotrexate | 52 | 26.4 | 33 | 32.0 | 19 | 20.2 | |
Other | 45 | 22.8 | 25 | 24.3 | 20 | 21.3 | |
None | 13 | 6.6 | 8 | 7.8 | 5 | 5.3 |
CNI, calcineurin inhibitor (cyclosporin A or tacrolimus); IQR, interquartile range; MMUD, mismatched unrelated donor; MUD, matched unrelated donor; NA, not applicable.
Percentages are calculated within the nonmissing.
Mann-Whitney U test.
Definition of WAS score is provided in Albert et al.1
Details on these patients are provided in supplemental Table 1.