Table 5.
Haploidentical Hematopoietic Stem Cell Transplantation in Patients With SCD.
| No. | Age range (years) |
Graft source (N) |
Donor | Conditioning regimen | GvHD prophylaxis | Acute GvHD (grade II–IV) |
Chronic GvHD | OS % | EFS % | |
|---|---|---|---|---|---|---|---|---|---|---|
| Pawlowska et al. 45 | 4 | 12–24 | BM: 3 PB: 1 |
Haploidentical | BU+CY+ATG | PTCy+ Tacrolimus+ MMF | grade I GI in 1 patient. | Limited skin in 3 patients. | 100% | 100% |
| Foell et al. 46 | 9 | 3–31 | PB (CD3±CD19+ depleted) |
Haploidentical | Flu+Thio+Treo+ATG | CSA+MMF | grade I–II: 56% | Moderate in 1 patient. | 88% | 88% |
| Cairo et al. 44 | 19 | 3.3–20 | PB(CD34+ selected) | Haploidentical | BU+CY+Thio+Flu+ATG+TLI | - | 6.2% | 6.7% | 84% | 84% |
| Wiebking et al. 47 | 3 | 5–20 | BM | Haploidentical | Alemtuzumab+Treo+Thio+Flu+CY | PTCy+ Tacrolimus+ MMF | grade I in 1 patient. | No | 100% | 100% |
| Foell et al. 12 | 25 | 3–31 | PB: CD3/CD19-depleted (n = 19), TCRaß/CD19-depleted (n = 6) | Haploidentical | Treo+Thio+Flu+ATG | CSA+MMF (12 patients) |
grade I–II: 28% | 16% | 88% | 88% |
| Frangoul et al. 48 | 4 | 12.1–23.5 | BM: 3 PB: 1 |
Haploidentical | Thio+Flu+ATG+CY+TBI | Sirolimus+ MMF |
grade II: 100% |
No | 100% | 100% |
| Bolaños-Meade et al. 43 | 14 | 15–42 | BM | Haploidentical | Flu+CY +ATG +TBI | PTCy+ Tacrolimus+ MMF | No | No | 100% | 57% |
| Fitzhugh et al. 13 | 12 | 20–56 | PB | Haploidentical | Alemtuzumab +TBI | PTCy+ Sirolimus | grade I in 1 patient. | Limited ocular in 1 patient. | 92% | 50% |
| de la Fuente et al. 49 | 15 | 7–40 | BM | Haploidentical | NMA: Flu+CY+ Thio+ATG+TBI |
PTCy+ Sirolimus+ MMF |
grade III–IV in 2 patients. | Mild in 1 patient. | 100% | 93% |
| Saraf et al. 39 | 8 | 20–38 | PB | Haploidentical | Flu+CY+ Thio+ATG+TBI | PTCy+ Sirolimus+ MMF |
grade II–IV in 2 patients. | Moderate in 1 patient. | 87.% | 75% |
| Gluckman et al. 11 | 144 | Children (<16 y): 114 Adults (>16 y): 30 |
BM: 100 PB:38 UCB: 6 |
MUD:70 UCB: 6 Haploidentical: 68 |
Flu+Thio+Treo Flu+CY+Thio+TBI Bu+CY Flu+Mel+Thio ATG Alemtuzumab Ex vivo T-cell depleted or PTCy (Haploidentical) |
CSA + MTX MMF + Sirolimus CSA + MMF Tacrolimus + MMF |
24% | 24%; Limited: 13 patients Extensive: 18 patients. |
86% ± 3%; | 72% ± 4% |
| Kharya et al. 50 | 25 | 1–27 | PB | MRD | PTIS AND Thio+Flu+ATG+CY+TBI | PTCy+ Sirolimus+ MMF |
20% | Limited in 3 patients (skin, oral cavity and Liver) | 88% | 88% |
Alk: alkeran(melphalan); ATG: anti-thymocyte globulin BM: bone marrow; BU: busulfan; CNI: calcineurin inhibitor; CSA: cyclosporine; CY: cyclophosphamide; EFS: event-free survival; Flu: fludarabine; GI: gastrointestinal; GvHD: graft versus host disease; MAC: myeloablative conditioning; MMF: mycophenolate mofetil; MRD: matched related donor; MUD: matched unrelated donor; MTX: methotrexate; NMA: nonmyeloablative conditioning; OS: overall survival; PB: peripheral blood; PTCy: post transplant clophosphamide; TBI: total body irradiation; Thio: thiotepa; Treo: treosulfan; TRM: transplant related mortality; UCB: umblical cord blood; PTIS: pre-transplant immune suppression.