Demographic and HSCT data of study participants
| 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 |
| 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.