Table 2.
Current published experience for haploidentical transplantation in sickle cell disease.
| Study | N | Age Range (years) | Conditioning Regimen | Stem Cell Source | aGVHD | cGVHD | Stable Engraftment | Overall Survival |
|---|---|---|---|---|---|---|---|---|
| Bolanos-Meade et al, 20128 | 14 | 15–42 | Flu 30mg/m2/day, Cy 14.5mg/kg/day, ATG, TBI 2Gy, 50mg/kg/day PT-Cy | Bone marrow | 0 | 0 | 8 | 14 |
| Dallas et al, 201328 | 8 | 4 – 17 | 1) Flu 150–200mg/m2, Thiotepa 10mg/kg, Bu (target 900ng/mL), ATG (10mg/kg), muromonab-CD3 (0.1mg/kg) 2) HU/azathioprine 3 months pre-transplant; Bu (target 900ng/mL), thiotepa, Cy (200mg/kg), muomonab-CD3 (0.1mg/kg) |
Bone marrow | 4 | 3 | 5 | 6 |
| Dhedin et al, 20169 | 5 | 12 – 50 | 1) Flu 30mg/m2/day, Cy 14.5mg/kg/day, ATG, TBI 2Gy, PT-Cy 50mg/kg/day | Bone marrow | 0 | 0 | 2 | 5 |
| 8 | 7 – 26 | 2) Thiotepa 10mg/kg/day, Flu 30mg/m2/day, Cy 14.5mg/kg/day, ATG, TBI 2Gy, PT-Cy 50mg/kg/day | 1 | 0 | 7 | 8 | ||
| 23 | 3 – 18 | 3) Pre-conditioning for 3 months with azathioprine 3mg/kg/day and HU 30mg/kg/day; Thiotepa 10mg/kg/day, Flu 30mg/m2/day, Cy 14.5mg/kg/day, ATG, TBI 2Gy, PT-Cy 50mg/kg/day | 4 | 0 | 21 | 18 | ||
| Fitzhugh et al, 201714 | 12 | 20 – 56 | Alemtuzumab 1mg/kg, TBI 4 Gy, PT-Cy 50mg/kg/day | PBSC | 1 | 1 | 6 | 11 |
| Pawlowska et al, 201829 | 4 | 13 – 23 | Flu 40mg/m2/day, dexamethasone 25mg/m2/day × 2 cycles pre-HSCT Rabbit ATG 1.5mg/kg/day, Flu 35mg/m2/day, Bu 130mg/m2/day, PT-Cy 50mg/kg/day | 3 Bone Marrow, 1 PBSC | 1 | 3 | 4 | 4 |
| Current Study | 8 | 20 – 38 | Flu 30mg/m2/day, Cy 14.5mg/kg/day, ATG, TBI 3Gy, PT-Cy 50mg/kg/day | PBSC | 2 | 1 | 7 | 7 |
| Summary | 82 | 3 – 51 | — | — | 13 (16%) | 8 (10%) | 60 (73%) | 73 (89%) |
aGVHD, acute graft versus host disease; cGVHD, chronic graft versus host disease; Flu, fludarabine, Cy, cyclophosphamide, ATG, antithymocyte globulin; TBI, total body irradiation; PT-Cy, post-transplantation cyclophosphamide; Bu, busulfan; PBSC, peripheral blood stem cell