Patient demographics and transplant-related characteristics of patients with FA who underwent HSCT
| . | HSCT, PLI (n = 21) . | HSCT, no PLI (n = 29) . | P value . |
|---|---|---|---|
| Sex, n (%) | |||
| Female | 12 (57.1) | 19 (65.5) | 1.00 |
| Male | 9 (42.9) | 10 (34.5) | |
| Race, n (%) | |||
| Caucasian | 17 (81) | 23 (79.3) | .57 |
| African American | 3 (14.3) | 4 (13.8) | |
| Hispanic | 0 | 2 (6.9) | |
| Asian | 1 (4.7) | 0 | |
| Age (y) at HSCT, median (IQR) | 6.7 (5.5-10.3) | 10.1 (6.2-12.8) | .32 |
| FANC complementation group, n (%) | |||
| FANCA | 5 (23.8) | 12 (41.4) | .47 |
| Non-FANCA | 12 (57.1) | 10 (34.5) | |
| Unknown | 4 (19.1) | 7 (24.1) | |
| Androgen use before HSCT, n (%) | 7 (33.3) | 6 (20.7) | .35 |
| Year of HSCT, n (%) | |||
| 2006-2010 | 10 (47.6) | 5 (17.2) | .03 |
| 2011-2014 | 11 (52.4) | 24 (82.8) | |
| Stem cell source, n (%) | |||
| Bone marrow | 2 (9.5) | 5 (17.2) | .19 |
| PBSC | 17 (81) | 24 (82.8) | |
| Cord | 2 (9.5) | 0 | |
| Degree of match, n (%) | |||
| Fully matched | 18 (85.7) | 26 (89.7) | 1.00 |
| Mismatched | 3 (14.3) | 3 (10.3) | |
| Donor type, n (%) | |||
| Related | 2 (9.5) | 8 (27.6) | .16 |
| Unrelated | 19 (90.5) | 21 (72.4) | |
| Conditioning regimen, n (%) | |||
| ATG/Bu/Cy/Flu | 12 (57.1) | 24 (82.8) | .09 |
| ATG/Cy/Flu | 2 (9.5) | 3 (10.4) | |
| ATG/Cy/Flu/TBI | 7 (33.2) | 1 (3.4) | |
| ATG/Flu | 0 | 1 (3.4) | |
| GVHD prophylaxis, n (%) | |||
| CNI-based only | 11 (52.4) | 12 (41.4) | .52 |
| TCD only | 2 (9.5) | 4 (13.8) | |
| CNI/TCD | 8 (38.1) | 13 (44.8) | |
| VOD, n (%) | 1 (4.8) | 2 (6.9) | 1.0 |
| TA-TMA, n (%) | 4 (19) | 13 (44.8) | .009 |
| Day-100 acute GVHD, n (%) | |||
| Grades 2-4 | 0 | 2 (6.9)∗ | .13 |
| Grades 3-4 | 0 | 2 (6.9) | |
| Acute GVHD with liver involvement | 0 | 0 | |
| cGVHD, n (%) | 6 (28.6) | 0 | .0034 |
| cGVHD with liver involvement | 1 (16.7) | 0 | |
| Survival, n (%) | |||
| Alive | 18 (85.7) | 19 (65.5) | .19 |
| . | HSCT, PLI (n = 21) . | HSCT, no PLI (n = 29) . | P value . |
|---|---|---|---|
| Sex, n (%) | |||
| Female | 12 (57.1) | 19 (65.5) | 1.00 |
| Male | 9 (42.9) | 10 (34.5) | |
| Race, n (%) | |||
| Caucasian | 17 (81) | 23 (79.3) | .57 |
| African American | 3 (14.3) | 4 (13.8) | |
| Hispanic | 0 | 2 (6.9) | |
| Asian | 1 (4.7) | 0 | |
| Age (y) at HSCT, median (IQR) | 6.7 (5.5-10.3) | 10.1 (6.2-12.8) | .32 |
| FANC complementation group, n (%) | |||
| FANCA | 5 (23.8) | 12 (41.4) | .47 |
| Non-FANCA | 12 (57.1) | 10 (34.5) | |
| Unknown | 4 (19.1) | 7 (24.1) | |
| Androgen use before HSCT, n (%) | 7 (33.3) | 6 (20.7) | .35 |
| Year of HSCT, n (%) | |||
| 2006-2010 | 10 (47.6) | 5 (17.2) | .03 |
| 2011-2014 | 11 (52.4) | 24 (82.8) | |
| Stem cell source, n (%) | |||
| Bone marrow | 2 (9.5) | 5 (17.2) | .19 |
| PBSC | 17 (81) | 24 (82.8) | |
| Cord | 2 (9.5) | 0 | |
| Degree of match, n (%) | |||
| Fully matched | 18 (85.7) | 26 (89.7) | 1.00 |
| Mismatched | 3 (14.3) | 3 (10.3) | |
| Donor type, n (%) | |||
| Related | 2 (9.5) | 8 (27.6) | .16 |
| Unrelated | 19 (90.5) | 21 (72.4) | |
| Conditioning regimen, n (%) | |||
| ATG/Bu/Cy/Flu | 12 (57.1) | 24 (82.8) | .09 |
| ATG/Cy/Flu | 2 (9.5) | 3 (10.4) | |
| ATG/Cy/Flu/TBI | 7 (33.2) | 1 (3.4) | |
| ATG/Flu | 0 | 1 (3.4) | |
| GVHD prophylaxis, n (%) | |||
| CNI-based only | 11 (52.4) | 12 (41.4) | .52 |
| TCD only | 2 (9.5) | 4 (13.8) | |
| CNI/TCD | 8 (38.1) | 13 (44.8) | |
| VOD, n (%) | 1 (4.8) | 2 (6.9) | 1.0 |
| TA-TMA, n (%) | 4 (19) | 13 (44.8) | .009 |
| Day-100 acute GVHD, n (%) | |||
| Grades 2-4 | 0 | 2 (6.9)∗ | .13 |
| Grades 3-4 | 0 | 2 (6.9) | |
| Acute GVHD with liver involvement | 0 | 0 | |
| cGVHD, n (%) | 6 (28.6) | 0 | .0034 |
| cGVHD with liver involvement | 1 (16.7) | 0 | |
| Survival, n (%) | |||
| Alive | 18 (85.7) | 19 (65.5) | .19 |
ATG, antithymocyte globulin; Bu, busulfan; CNI, calcineurin inhibitor; Cy, cyclophosphamide; Flu, fludarabine; PBSC, peripheral blood stem cell; TCD, T-cell depleted; VOD, veno-occlusive disease;
Both cases of grade 3 GVHD with gut and gut/skin but no liver involvement.