Table 2A.
Summary of recent publications on Haploidentical hematopoietic cell transplant for sickle cell disease (2017–2022)
| Reference | Size | Median Age years (range) | Graft Source | Conditioning Regimen | Median follow-up, months (range) | GVHD | Graft Failure | EFS | OS |
|---|---|---|---|---|---|---|---|---|---|
| Gilman et al. 2017 | 8 | 16 (8–23) | ex vivo CD34+ cell selected CD3+ depleted PBSC | ATG/Flu/Thio/Mel | 21.5 (9.9–60) | 25% acute, 13% chronic | 13% | 100% | 87% |
| Frangoul et al. 2018 | 4 | 14(12–23) | BM/PBSC | ATG/Thio/Flu Cy/TBI 200 cGy/PT-Cy | 16 (13–34) | 100% acute, 0% chronic | 0% | 100% | 100% |
| Pawlowska et al 2018 | 4 | 19 (13–23) | BM/PBSC | ATG/Flu/Bu/PT-Cy | 9 (5–11) | 25% acute, 75% chronic | 0% | 100% | 100% |
| * Eapen et al 2019 | 137 | 19 (13–27) | BM/PBSC | varied | 25 (12–48) | 8.7% acute, 16% chronic | 26% | 67.2% | 90.5% |
| Cairo et al. 2020 | 19 | 13(3–20) | PBSC | ATG/Flu/Cy/Thio/Bu/TBI 500 cGy | 46.3 (1.9–73.3) | 5% acute, 5% chronic | 0% | 84% | 84% |
| Foell al 2020 | 25 | 13(3–31) | CD3+/CD19+ or TCR αβ/CD19+ depleted PBSC | ATG/Flu/Thio/Treo | 20.0 (NR) | 28% acute, 16% chronic | 8% | 100% | 88% |
| *Gluckman et al 2020 | 71 | 9.3 (2–43) | BM/PBSC | varied | 38 (2–154) | 23% acute, 23% chronic | 16% | 62% | 88% |
| Jaiswal et al 2020 | 5 | 8 (3–19) | PBSC | ATG/Flu/Treo/Mel/PT-Cy & Aba | 28 (13–54) | 0% acute, 0% chronic | 0% | 100% | 100% |
| Kharya et. al 2021 | 25 | 7 (1–27) | PBSC | (Flu/Cy/Dex)/ATG/Flu/Cy/Thio/Bu/TBI 200 cGy//PT-Cy, | 15.9 (6.5–26.3) | 20% acute, 12% chronic | 0% | 88% | 88% |
*
Registry studies, data include cases reported in the individual series.
Abbreviations: BM: bone marrow, PBSC: peripheral blood stem cell, Treo, treosulfan, Thio thiotepa, Flu: fludarabine, ATG: anti-thymocyte globulin, Bu: busulfan, Alem: alemtuzumab, Cy: cyclophosphamide, TBI: total body irradiation, cGY: centigray, Mel: melphalan, PT-Cy: post-transplant cyclophosphamide Aba: abatacept, Dex: dexamethasone NR: not reported