Myeloablative HSCT for children with SCD with matched related donors
| Reference . | Transplantation regimen . | Number of patients (age range, y) . | Alive without SCD, n (%) . | Acute GVHD . | Chronic GVHD . | Death (cause) . | Comments . |
|---|---|---|---|---|---|---|---|
| Bernaudin et al, 200711 | Bu 16 mg/kg, Cy 200 mg/kg ± ATG, CSA ± MTX | 87 (2-22) | 80 (92) | 10 (Gr 2); 5 (Gr 3); 2 (Gr 4) | 9 (limited); 2 (extensive) | 6 (sepsis, CNS hemorrhage, and GVHD in 4) | At 6 y after HSCT; 25%-40% had mixed chimerism (5%-95%) |
| Panepinto et al, 200727 | Mostly Bu 16 mg/kg, Cy 200 mg/kg, MTX, CSA | 67 (2-27) | 55 (82) | 8 (Gr 2-4) | 13 (9 limited, 3 extensive) | 3 (CNS hemorrhage with GVHD, organ failure, unknown cause) | At 5 y after HSCT; CIBMTR review; 9 had mixed red cell chimerism (HbS > 50%) |
| Brachet et al, 200426 | Bu 14-16 mg/kg, Cy 200 mg/kg ± ATG, MTX, CSA | 24 (2-14) | 19 (79) | 3 (Gr 2); 3 (Gr 3) | 7 (limited) | 1 (unknown) | At 8.7 y after HSCT; 1 had mixed chimerism |
| Walters et al, 2001, 199612,25 | Bu 14 mg/kg, Cy 200 mg/kg, ATG or alemtuzumab, MTX, CSA | 59 (3-16) | 50 (85) | 11 (acute or chronic GVHD) | 3 (all GVHD) | At 3.5 years after HSCT; 13 had mixed chimerism (8 with 90%-99%, 5 with 11%-74%) | |
| Vermylen et al, 199824 | Bu 16 mg/kg, Cy 200 mg/kg ± TLI or ATG, CSA | 50 (0.3-11) | 45 (90) | 10 (Gr 1); 9 (Gr 2); 1 (Gr 3-4) | 7 (limited);3 (extensive) | 2 (acute GVHD with CMV and aspergillus; sudden death) | At 5 y after HSCT; 6 had mixed chimerism (3: > 90%, 3: 50%-70%) |
| Reference . | Transplantation regimen . | Number of patients (age range, y) . | Alive without SCD, n (%) . | Acute GVHD . | Chronic GVHD . | Death (cause) . | Comments . |
|---|---|---|---|---|---|---|---|
| Bernaudin et al, 200711 | Bu 16 mg/kg, Cy 200 mg/kg ± ATG, CSA ± MTX | 87 (2-22) | 80 (92) | 10 (Gr 2); 5 (Gr 3); 2 (Gr 4) | 9 (limited); 2 (extensive) | 6 (sepsis, CNS hemorrhage, and GVHD in 4) | At 6 y after HSCT; 25%-40% had mixed chimerism (5%-95%) |
| Panepinto et al, 200727 | Mostly Bu 16 mg/kg, Cy 200 mg/kg, MTX, CSA | 67 (2-27) | 55 (82) | 8 (Gr 2-4) | 13 (9 limited, 3 extensive) | 3 (CNS hemorrhage with GVHD, organ failure, unknown cause) | At 5 y after HSCT; CIBMTR review; 9 had mixed red cell chimerism (HbS > 50%) |
| Brachet et al, 200426 | Bu 14-16 mg/kg, Cy 200 mg/kg ± ATG, MTX, CSA | 24 (2-14) | 19 (79) | 3 (Gr 2); 3 (Gr 3) | 7 (limited) | 1 (unknown) | At 8.7 y after HSCT; 1 had mixed chimerism |
| Walters et al, 2001, 199612,25 | Bu 14 mg/kg, Cy 200 mg/kg, ATG or alemtuzumab, MTX, CSA | 59 (3-16) | 50 (85) | 11 (acute or chronic GVHD) | 3 (all GVHD) | At 3.5 years after HSCT; 13 had mixed chimerism (8 with 90%-99%, 5 with 11%-74%) | |
| Vermylen et al, 199824 | Bu 16 mg/kg, Cy 200 mg/kg ± TLI or ATG, CSA | 50 (0.3-11) | 45 (90) | 10 (Gr 1); 9 (Gr 2); 1 (Gr 3-4) | 7 (limited);3 (extensive) | 2 (acute GVHD with CMV and aspergillus; sudden death) | At 5 y after HSCT; 6 had mixed chimerism (3: > 90%, 3: 50%-70%) |
ATG indicates antithymocyte globulin; Bu, busulfan; CIBMTR, Center for International Blood and Marrow Transplant Research; CSA, cyclosporine A; Cy, cyclophosphamide; Gr, grade; MTX, methotrexate; and SCD, sickle cell disease.