Table 1.
Characteristic | Number (n = 80) |
---|---|
Median age (range), y | 9.4 (0.5-37.9) |
Female | 28 |
Underlying diagnosis | |
Malignancy | 32 |
Primary immune deficiency | 17 |
Hemoglobinopathy | 4 |
Metabolic disorder | 4 |
Bone marrow failure | 22 |
Platelet function disorder | 1 |
Conditioning regimen | |
Myeloablative | 57 |
Reduced intensity | 23 |
HLA match | |
5/10 | 3 |
6/10 | 2 |
7/10 | 1 |
8/10 | 5 |
9/10 | 13 |
10/10 | 56 |
Donor relation | |
Related | 18 |
Unrelated | 62 |
Stem cell source | |
Bone marrow | 42 |
Peripheral blood stem cells | 33 |
Umbilical cord blood | 5 |
Acute GVHD prophylaxis | |
CNI + MMF | 20 |
CNI + MMF + abatacept | 27 |
T-cell depletion∗ | 25 |
CNI + methotrexate + abatacept | 2 |
CNI + MMF + posttransplant cyclophosphamide | 2 |
CNI + methylprednisolone | 3 |
Sirolimus + abatacept | 1 |
Clinical outcomes | |
Acute GVHD | 16 |
Skin GVHD | 13 |
GI GVHD | 6 |
BSI by day +30 | 21 |
Mucosal barrier injury–related BSI by day +30 | 14 |
Moderate/high-risk TA-TMA | 32 |
CNI, calcineurin inhibitor; MMF, mycophenolate mofetil.
In vitro T-cell depletion included either CD34+ selection or T -cell receptor alpha/beta/CD19+ depletion.