Table 5.3.
Haploidentical (and mismatched related) transplantation for patients with sickle cell disease
| Refs. | Transplant regimen | Graft Type | No. of patients (age) | Alive without SCD | Acute GvHD (Gr 2–4) | Chronic GvHD (extensive) | Death (cause) |
|---|---|---|---|---|---|---|---|
| Raj et al. [57] | Flu 90 mg/m2, 200 cGy TBI, CSA + MMF | BM | 1(14) | 0 | 0 | 0 | 0 |
| Bolanos- Meade et al. [58] | Cy 29 mg/kg, Flu 150 mg/m2, 200 cGy TBI, ATG 4.5 mg/kg, PT-Cy 100 mg/kg, tac or sir + MMF | BM ± GCSF | 14(15–42) | 7a | 0 | 0 | 0 |
| Dallas et al. [61] | Flu 150–200 mg/m3 or Cy 200 mg/kg, TT 10 mg/kg, Bu, muromonab-CD3 ± ATG 30 mg/kg, MMF | PBSCb | 8(4.2–17.1) | 3 | 2 | 2 | 2(GvHD) |
| Total | Mixed | - | 23 | 43% | 9%(of total) | 9%(of total) | 9% of total |
| 20%(of ensrafted) | 20%(of ensrafted) |
Abbreviations: SCD sickle cell disease, GvHD graft-versus-host disease, Flu ftudarabine, Gy gray, TBI total body irradiation, CSA cyclosporine A, MMF mycophenolate mofetil, BM bone marrow, N/A not applicable, Cy cyclophosphamide, ATG anti-thymocyte globulin, PT post-transplant, tac tacrolimus, sir sirolirnus, GCSF granulocyte colony-stimulating factor, TT thiotepa, Bu busulfan, PBSC peripheral blood stem cell, Gr grade
a
One additional patient with mixed chimerism is transfusion-independent and free from SCD crises, but remains anemic
b
Patients received a CD34+-selected product on day 0 and a CD3+-depleted product on day +1 post-transplant