Table 2.
Summary of recent publications HCT for SDS
| Conditioning Regimen (n) | Donor Source(n) | Stem Cell Source (n) | Median Age at HCT (yrs) | Engraftment (n) | GVHD Prophylaxis (n) +/− | Acute GVHD Grade (n) | cGVHD | TRM | OS | Median f/u | Ref |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Bu based (14) TBI based (6) Flu (4) Others (2) |
Sibling (6) URD (19) Other family (1) T-cell depleted (21) |
BM (21) PBSC (3) CB (2) |
10.3 (1.2–26.8) | 21/26 (81%) | CSP/MTX (14) Other (6) Not specified (6) |
I–IV: 15 (71%) III–IV: 5/21 (24%) |
4/14 (29%)eligible | 35.5% (1 yr) | 64.5 % | 1.1 (0.05–16.2) yrs | [72] |
| Bu/CY (3) + ATG (3) TBI/CY (3) TBI/Mel (1) |
Sibling (4) URD (6) T-cell depleted (2) |
BM (10) | 11.2 (1.1–27.7) | 8/10 * | CSP/MTX (5) CSP/MTX/Steroids (2) Other (3) |
II (3)/IV (3) | 2/10 | 3/10 | 60% (5 yr) EFS | 7.6 (3.9–16.9) yrs | [39] |
| Flu/Treo/Mel + Campath-1H (2) + ATG (1) |
Sibling (1) URD (2) |
BM (2) CB (1) |
9.6 (1.5–17) | 3/3 | CSP/MTX (2) CSP/MMF (1) |
II (1) | NR | 1/3 | 2/3 | 2 yrs, 1.3 yrs | [73] |
| Campath-1H/Flu/Mel (7) | Sibling (4) URD (3) |
BM (4) PBSC (2) BM+CB (1) |
8.0 (1.0–29) | 7/7 | CSP/MTX (6) CSP/steroids (1) |
II (1) | NR | 0/7 | 100% | 1.5 (0.3–2.5) yrs | [74] |
ATG, anti-thymocyte globulin; Bu, busulfan; BM, bone marrow; CB, cord blood; cGVHD, chronic graft-versus-host-disease; CSP, cyclosporine; CY, cyclophosphamide; EFS, event free survival; Flu, fludarabine; f/u, follow up; HCT, hematopoietic cell transplantation; Mel, melphalan; MMF, mycophenolate mofetil; MTX, methotrexate; n, number; OS, overall survival; PBSC, peripheral blood stem cells; Pt, patient; Ref, reference; TBI, total body irradiation; Treo, treosulfan; TRM, transplant related mortality; URD, unrelated donor; yrs, years;
*
Two patients died before engraftment