Table 2.
Clinical trials and outcomes after haplo-HSCT with CD45RA-depleted progenitor cell grafts.
| Author | Diagnosis/ Patient Number |
Donor | Conditioning | GvHD Prophylaxis |
Graft | Survival | CI of aGvHD | CI of cGvHD |
|---|---|---|---|---|---|---|---|---|
| Naik, S. et al. Blood 2021 [47] |
Children with hematologic malignancies (n = 72) | haplo | subMA 100% | MMF (n = 61) and/or sirolimus (n = 8) |
Day 0: CD34+ cells/kg: 9.85 × 106 (range: 1.96 × 106 to 44.64 × 106) day +1: CD45RA-depleted graft: CD34+ cells/kg: 5.82 × 106 (range: 0.58 × 106 to 39.43 × 106) CD3+ T cells/kg: 60.1 × 106 (range: 16.08 × 106 to 528.43 × 106) CD3+CD45RA+ cells/kg: median 0, range 0–0.2 × 10 6 cells/kg). day +6: NK cells (median: 11.7 × 10 6 cells/kg; range: 1.65–99.2) |
3-y OS: 68.9% 3-y DFS: 62.2% |
Grade II–IV: 36.1% Grade III–IV: 29.2% | 3y: 20.8% |
| Sisinni, L. et al. Biol. Blood Marrow Transpl. 2018 [43] |
Children with acute leukemias (n = 25) |
haplo | subMA 100% | CSA (n = 3), CSA+ MTX (n = 1), MMF (n = 21) |
CD34+ cells/kg: 6.29 × 106
(range: 4.04 × 106 to 18.1 × 106) CD45RA+ cells/kg: 0.6 × 104 (range: 0.2 × 104 to 1 × 104) |
30 months OS: 58% | Grade II–IV: 39% Grade III–IV: 33% |
30 months: 22% |
| Gasior Kabat, M. et al. Int. J. Hematol. 2021 [45] |
Children with hematologic malignancies (n = 17) Severe aplastic anemia (n = 1) | haplo MRD |
subMA 100% | MMF (n = 18) | CD34+ cells/kg: 6.5 × 106 (range: 5 × 106 to 11.2 × 106) CD3+CD45RA+ cells/kg: 3.6 × 104 (range: 0 to 23 × 104) day +7: NK cells/kg: 12.6 × 106 (range: 3.9 × 106 to 100 × 106) |
2yOS: 87.2% 2y DFS: 67.3% | d+180: III–IV: 34.8% |
1y: 23.1% |
MA: myeloablative conditioning; MMF: mycophenolate mofetile; CSA: cyclosporine; MRD: matched related donor.