Patient, disease, and transplant characteristics of recipients presenting stable mixed chimerism more than 6 months after CBT for either thalassemia or SCD
| UPN . | Age at CBT . | Diagnosis . | Clinical status at CBT . | HLA . | Conditioning . | GVHD prophylaxis . | Nucleated cell dose × 107/kg . | Chimerism at last follow-up (time after CBT); method of detection . | Outcome and survival . |
|---|---|---|---|---|---|---|---|---|---|
| 196 | 19 y 7 mo | Thal | Pesaro class 2 | Matched | FLU (40 mg/m2), BU (14 mg/kg), TT (6 mg/kg) | CsA | 2.0 | 80% of donor cell (2 y); STR | Alive without disease at 2 y 0 mo |
| 591 | 10 y 2 mo | SCD | Vaso-occlusive disease + chest syndrome | Matched | BU (16 mg/kg), CY (200 mg/kg), ATG | CsA | 7.6 | 93% of donor cells (1 y); VNTR | Alive without disease at 1 y 4 mo |
| 620 | 12 y 9 mo | SCD | Splenic sequestration + acute chest syndrome | Matched | BU (16 mg/kg), CY (200 mg/kg), ATG | CsA | 2.1 | 89% of donor cells (1 y 6 mo); VNTR | Alive without disease at 1 y 8 mo |
| 802 | 2 y 0 mo | SCD | Recurrent acute chest syndrome | Matched | BU (14 mg/kg) CY (200 mg/kg) | CsA + MTX | 7.6 | 45% of donor cells (1 y 6 mo); VNTR | Alive without disease at 1 y 6 mo |
| 817 | 6 y 5 mo | Thal | Pesaro class 1 | Matched | FLU (90 mg/m2), BU (14 mg/kg), CY (200 mg/kg) | MMF + tacrolimus | 6.6 | ∼80%-90% of donor cells (1 y 3 mo); VNTR | Alive without disease at 1 y 5 mo |
| 818 | 2 y 8 mo | Thal | Pesaro class 1 | Matched | BU (20 mg/kg), CY (200 mg/kg), ATG | CsA | 4.5 | 89% of donor cells (6 mo); FISH | Alive without disease at 1 y 1 mo |
| 916 | 3 y 2 mo | SCD | Vaso-occlusive diseases + osteomyelitis | Matched | BU (16 mg/kg), CY (200 mg/kg), ATG | CsA | 4.6 | 81% of donor cells (1 y); VNTR | Alive without disease at 1 y 2 mo |
| UPN . | Age at CBT . | Diagnosis . | Clinical status at CBT . | HLA . | Conditioning . | GVHD prophylaxis . | Nucleated cell dose × 107/kg . | Chimerism at last follow-up (time after CBT); method of detection . | Outcome and survival . |
|---|---|---|---|---|---|---|---|---|---|
| 196 | 19 y 7 mo | Thal | Pesaro class 2 | Matched | FLU (40 mg/m2), BU (14 mg/kg), TT (6 mg/kg) | CsA | 2.0 | 80% of donor cell (2 y); STR | Alive without disease at 2 y 0 mo |
| 591 | 10 y 2 mo | SCD | Vaso-occlusive disease + chest syndrome | Matched | BU (16 mg/kg), CY (200 mg/kg), ATG | CsA | 7.6 | 93% of donor cells (1 y); VNTR | Alive without disease at 1 y 4 mo |
| 620 | 12 y 9 mo | SCD | Splenic sequestration + acute chest syndrome | Matched | BU (16 mg/kg), CY (200 mg/kg), ATG | CsA | 2.1 | 89% of donor cells (1 y 6 mo); VNTR | Alive without disease at 1 y 8 mo |
| 802 | 2 y 0 mo | SCD | Recurrent acute chest syndrome | Matched | BU (14 mg/kg) CY (200 mg/kg) | CsA + MTX | 7.6 | 45% of donor cells (1 y 6 mo); VNTR | Alive without disease at 1 y 6 mo |
| 817 | 6 y 5 mo | Thal | Pesaro class 1 | Matched | FLU (90 mg/m2), BU (14 mg/kg), CY (200 mg/kg) | MMF + tacrolimus | 6.6 | ∼80%-90% of donor cells (1 y 3 mo); VNTR | Alive without disease at 1 y 5 mo |
| 818 | 2 y 8 mo | Thal | Pesaro class 1 | Matched | BU (20 mg/kg), CY (200 mg/kg), ATG | CsA | 4.5 | 89% of donor cells (6 mo); FISH | Alive without disease at 1 y 1 mo |
| 916 | 3 y 2 mo | SCD | Vaso-occlusive diseases + osteomyelitis | Matched | BU (16 mg/kg), CY (200 mg/kg), ATG | CsA | 4.6 | 81% of donor cells (1 y); VNTR | Alive without disease at 1 y 2 mo |
STR indicates short tandem repeats; VNTR, variable number tandem repeats; FISH, fluorescent in situ hybridization.