Table 1.
Clinical trials and outcomes after haplo-HSCT with depletion of alloreactive αβ T cells and CD19+ B cells.
| Author | Diagnosis/ Patient Number |
Donor | Conditioning | GvHD Prophylaxis |
Graft | Survival | CI of aGvHD | CI of cGvHD |
|---|---|---|---|---|---|---|---|---|
| Bertaina, A. et al. Blood 2014 [18] | Children with malignant and nonmalignant diseases (n = 23) | haplo | MAC 30%; NMA 70% |
ATG (n = 23) | CD34+ cells/kg: 15.8 × 106
(range: 10.4 × 106 to 40 × 106) TCR-αβ+CD3+ cells/kg: 4 × 104 (range: 1 × 104 to 9.5 × 104) |
2y DFS 91.1% 2y OS 91.1% |
Grade I–II: 13% Grade III–IV: 0% |
18 months: 0% |
| Gaziev, J. et al. Blood Adv. 2018 [20] | Children with nonmalignant diseases (n = 14) | haplo | MAC 100% | CSA+ steroids (n = 12); CSA + MMF (n = 2) |
CD34+ cells/kg: 15.7 × 106
(range: 8.1 × 106 to 39.2 × 106) TCR-αβ+CD3+ cells/kg: 4 × 104 (range: 1 × 104 to 10 × 104) |
5y DFS 69% 5y OS 84% |
Grade II–III: 28% | extensive cGvHD: 21% |
| Laberko, A. et al. Blood 2019 [21] | Children with nonmalignant diseases (n = 98) | MUD, haplo | MAC 74%, NMA 26%, |
ATG + CSA ± MTX/MMF (n = 96) | CD34+ cells/kg: 10.5 × 106
(range: 6.3 × 106 to 14.9 × 106) in haplo TCR-αβ+CD3+ cells/kg: 1.4 × 104 (range: 0.5 × 104 to 13 × 104) in haplo |
5y OS 86% in MUD; 5y OS 87% in haplo |
Grade II–IV 17% in MUD; Grade II–IV 22% in haplo |
limited cGvHD 9% in MUD; chronic GvHD 13% in haplo |
| Locatelli, F. et al. Blood 2017 [22] | Children with malignant diseases (n = 80) | haplo | MAC 100% | ATG (n = 80) | CD34+ cells/kg: 13.9 × 106
(range: 6 × 106 to 40.4 × 106) TCR-αβ+CD3+ cells/kg: 4.7 × 104 (range: 0.2 × 104 to 9.9 × 104) |
5y DFS 71% 5y OS 72% |
Grade I–II 30% | limited cGvHD 5% |
| Prezioso, L. et al. Bone Marrow Transpl. 2019 [27] | Adult with malignant diseases (n = 59) | haplo | MAC 100% | ATG (n = 59) | CD34+ cells/kg: 11 × 106
(range: 5 × 106 to 19 × 106) TCR-αβ+CD3+ cells/kg: 8.4 × 104 (range: 0.4 × 104 to 62 × 104) |
2y OS 51% | Grade II–IV: 17% Grade III–IV: 3% |
limited cGvHD 3% |
| Kaynar, L. et al. Hematology 2017 [28] |
Adult with malignant diseases (n = 34) | haplo | MAC 100% | ATG (n = 34) | CD34+ cells/kg: 12.7 × 106
(range: 10.3 × 106 to 16 × 106) TCR-αβ+CD3+ cells/kg: 1.8 × 104 (range: 0.7 × 104 to 2.5 × 104) |
2y DFS 33%; 2y OS 36% |
Grade I–IV: 30.3% Grade III–IV: 6.1% |
cGvHD 6.1% |
MAC: myeloablative conditioning regimen; NMA: non-myeloablative conditioning regimen; MUD: matched unrelated donor; ATG: anti-thymocyte globulin; CSA: cyclosporine; MMF: mycophenolate mofetile; MTX: methotrexate.