Demographic details on HSCT recipients
| Demographics . | Patients . | |||
|---|---|---|---|---|
| 1 . | 2 . | 3 . | 4 . | |
| Age, y | 7 | 1.4 | 4 | 0.5 |
| Race | White | African American | White | White |
| FOXP3 mutation analysis | A>G splice junction mutation in intron 9 | 303_304 del TT | 1271 G>A, C424Y | 1226 A>G, D409G |
| Clinical indications for HSCT | Colitis, food allergies, eczema, FTT, AIHA, RAD | Colitis, food allergies, eczema, FTT, AIHA, TPN | Colitis, food allergies, eczema, MGN | Colitis, TPN, AIHA |
| Pre-HSCT IS | Imuran, CSA, prednisone | CSA, rituximab | FK506, MMF, prednisone | FK506, rituximab, prednisone, alemtuzumab |
| Stem-cell source* | 8 of 8 URD, BM | 7 of 8, A-antigen mismatched URD, BM | 8 of 8, MSD, BM | 8 of 8, URD, BM |
| Cell dose TNCs × 108/kg/CD34, ×106/kg | 12.6/34.7 | 6.0/5.8 | 3.1/5.04 | 5.3/12.7 |
| Melphalan dose, m2 | 70 | 140 | 70 | 140 |
| Day of myeloid engraftment | 12 | 16 | 13 | 12 |
| Day of platelet engraftment | 13 | 26 | 26 | 23 |
| Demographics . | Patients . | |||
|---|---|---|---|---|
| 1 . | 2 . | 3 . | 4 . | |
| Age, y | 7 | 1.4 | 4 | 0.5 |
| Race | White | African American | White | White |
| FOXP3 mutation analysis | A>G splice junction mutation in intron 9 | 303_304 del TT | 1271 G>A, C424Y | 1226 A>G, D409G |
| Clinical indications for HSCT | Colitis, food allergies, eczema, FTT, AIHA, RAD | Colitis, food allergies, eczema, FTT, AIHA, TPN | Colitis, food allergies, eczema, MGN | Colitis, TPN, AIHA |
| Pre-HSCT IS | Imuran, CSA, prednisone | CSA, rituximab | FK506, MMF, prednisone | FK506, rituximab, prednisone, alemtuzumab |
| Stem-cell source* | 8 of 8 URD, BM | 7 of 8, A-antigen mismatched URD, BM | 8 of 8, MSD, BM | 8 of 8, URD, BM |
| Cell dose TNCs × 108/kg/CD34, ×106/kg | 12.6/34.7 | 6.0/5.8 | 3.1/5.04 | 5.3/12.7 |
| Melphalan dose, m2 | 70 | 140 | 70 | 140 |
| Day of myeloid engraftment | 12 | 16 | 13 | 12 |
| Day of platelet engraftment | 13 | 26 | 26 | 23 |
Myeloid engraftment is defined as having an ANC greater than 0.5 × 109/L; platelet engraftment, as having a platelet count greater than 50 × 109/L.
URD indicates unrelated donor; MSD, matched sibling donor; TNC, total nucleated cell; IS, immunosuppression; FTT, failure to thrive; AIHA, autoimmune hemolytic anemia; TPN, total parental nutrition; RAD, reactive airway disease; CSA, cyclosporine; MMF, mycophenolate mofetil; MGN, membranous glomerulonephritis.
Allele matched by high-resolution typing.