Patient demographics
| . | Vitamin A (n = 40) . | Placebo (n = 40) . |
|---|---|---|
| Median age (range), y | 6.9 (2-20) | 8.5 (1.1-28) |
| Underlying diagnosis | ||
| Malignancy | 8 | 9 |
| Primary immune deficiency | 8 | 6 |
| Bone marrow failure | 14 | 17 |
| Hemoglobinopathy | 8 | 8 |
| Metabolic disorder | 2 | 0 |
| Conditioning regimen | ||
| Myeloablative | 25 | 27 |
| Reduced intensity | 15 | 13 |
| Serotherapy in conditioning regimen | 24 | 28 |
| Radiation in conditioning regimen | 4 | 2 |
| HLA match | ||
| ≥2 antigen mismatch | 5 | 2 |
| 9 of 10 | 11 | 6 |
| 10 of 10 | 24 | 32 |
| Donor relation | ||
| Related (excluding ≥2 antigen mismatch) | 8 | 15 |
| Related (≥2 antigen mismatch) | 4 | 1 |
| Unrelated | 28 | 24 |
| Stem cell source | ||
| Unmanipulated PBSCs | 5 | 1 |
| PBSCs with stem cell manipulation | 11 | 11 |
| Cord blood | 1 | 0 |
| Bone marrow | 23 | 28 |
| GVHD prophylaxis | ||
| Calcineurin inhibitor + MMF | 9 | 6 |
| Calcineurin inhibitor + MTX | 1 | 5 |
| Calcineurin inhibitor + prednisone | 2 | 2 |
| T-cell depletion | 12 | 10 |
| Abatacept to standard prophylaxis | 15 | 14 |
| αβ-CD19+ TCR depletion | 0 | 2 |
| Posttransplant cyclophosphamide | 1 | 1 |
| . | Vitamin A (n = 40) . | Placebo (n = 40) . |
|---|---|---|
| Median age (range), y | 6.9 (2-20) | 8.5 (1.1-28) |
| Underlying diagnosis | ||
| Malignancy | 8 | 9 |
| Primary immune deficiency | 8 | 6 |
| Bone marrow failure | 14 | 17 |
| Hemoglobinopathy | 8 | 8 |
| Metabolic disorder | 2 | 0 |
| Conditioning regimen | ||
| Myeloablative | 25 | 27 |
| Reduced intensity | 15 | 13 |
| Serotherapy in conditioning regimen | 24 | 28 |
| Radiation in conditioning regimen | 4 | 2 |
| HLA match | ||
| ≥2 antigen mismatch | 5 | 2 |
| 9 of 10 | 11 | 6 |
| 10 of 10 | 24 | 32 |
| Donor relation | ||
| Related (excluding ≥2 antigen mismatch) | 8 | 15 |
| Related (≥2 antigen mismatch) | 4 | 1 |
| Unrelated | 28 | 24 |
| Stem cell source | ||
| Unmanipulated PBSCs | 5 | 1 |
| PBSCs with stem cell manipulation | 11 | 11 |
| Cord blood | 1 | 0 |
| Bone marrow | 23 | 28 |
| GVHD prophylaxis | ||
| Calcineurin inhibitor + MMF | 9 | 6 |
| Calcineurin inhibitor + MTX | 1 | 5 |
| Calcineurin inhibitor + prednisone | 2 | 2 |
| T-cell depletion | 12 | 10 |
| Abatacept to standard prophylaxis | 15 | 14 |
| αβ-CD19+ TCR depletion | 0 | 2 |
| Posttransplant cyclophosphamide | 1 | 1 |
MMF, mycophenolate mofetil; MTX, methotrexate; PBSCs, peripheral blood stem cells; TCR, T-cell receptor.