Year of transplant | Age at transplant | Donor | Conditioning | GVHD prophylaxis | Engraftment | Outcome | Latest chimerism |
---|---|---|---|---|---|---|---|
1 2011 | 12y M | 8/10 MMUD PBSC (allelic A and C mismatches) | Fludarabine 180mg/m2, alemtuzumab 1mg/kg, treosulfan 14g/m2, thiotepa 10mg/kg | CSA + MMF | Neutrophil D+10, Platelet NA | Died D+46 (Multisystem organ failure from D+7. CVVH, intubation, inotropes. Encephalopathic. Sudden deterioration D+45 ?sepsis) | 100% |
2 2012 | 2y M | 10/10 MUD PBSC | Fludarabine 150mg/m2, Treosulfan 42g/m2, Alemtuzumab 1mg/kg | CSA + MMF | Neutrophil D+15, Platelet D+13 | Alive and well | 100% 5years 7m post |
3 2019 | 5y F | 10/10 MUD PBSC | Fludarabine 150mg/m2, Treosulfan 42g/m2, Alemtuzumab 1mg/kg | CSA + MMF | Neutrophil D+12, Platelet D+14 | Alive and well | CD15 100%, CD19 94%, T cell 89% D+124 |
4 2019 | 2y F | Maternal TCRα β/CD19 depleted haploidentical PBSC | Fludarabine 160mg/m2, Treosulfan 42g/m2, Thiotepa 10mg/kg, ATG (grafalon) 15mg/m2), Rituximab 200mg/m2 | Nil | Neutrophil D+10, Platelets D+16 | Alive and well | 100% D+62 |
[Patient and transplant characteristics]