Prospective, multicenter HCT trials for IBMFS
| Disorder(s) . | Trial . | Regimen . | Donors . | Graft/GVHD ppx . | N (IBMFS) . | 1° graft . | OS . | Ref . |
|---|---|---|---|---|---|---|---|---|
| Fanconi anemia | NCT00987480 | PK-targeted busulfan; FLU 140 mg/m2; CY 40 mg/kg; rATG 10 mg/kg | HLA 7/8 or better URD; HLA 4/8 to 7/8 RD | CD34-selected PBSC CSA | 34 (non-MDS) | 100% | 85% | 21 |
| SDS, GATA2, DBA, other IBMFS | NCT00919503 (completed) NCT04965597 (ongoing) | Treosulfan 42 g/m2 FLU 150 mg/m2 | MRD or MUD | Unmanipulated BM or PBSC TAC/MTX | 10 (non-PNH) | 100% | 100% | 27 |
| Dyskeratosis congenita/telomere biology disorders | NCT01659606 | FLU 180 mg/m2 C1H 1 mg/kg | HLA 7/8 or better URD or RD | Unmanipulated BM CSA or TAC/MMF | 20 | 95% | 90% | 26 |
| Disorder(s) . | Trial . | Regimen . | Donors . | Graft/GVHD ppx . | N (IBMFS) . | 1° graft . | OS . | Ref . |
|---|---|---|---|---|---|---|---|---|
| Fanconi anemia | NCT00987480 | PK-targeted busulfan; FLU 140 mg/m2; CY 40 mg/kg; rATG 10 mg/kg | HLA 7/8 or better URD; HLA 4/8 to 7/8 RD | CD34-selected PBSC CSA | 34 (non-MDS) | 100% | 85% | 21 |
| SDS, GATA2, DBA, other IBMFS | NCT00919503 (completed) NCT04965597 (ongoing) | Treosulfan 42 g/m2 FLU 150 mg/m2 | MRD or MUD | Unmanipulated BM or PBSC TAC/MTX | 10 (non-PNH) | 100% | 100% | 27 |
| Dyskeratosis congenita/telomere biology disorders | NCT01659606 | FLU 180 mg/m2 C1H 1 mg/kg | HLA 7/8 or better URD or RD | Unmanipulated BM CSA or TAC/MMF | 20 | 95% | 90% | 26 |
1° graft, primary engraftment rate; BM, bone marrow; BU, busulfan; C1H, alemtuzumab; CSA, cyclosporine A; CY, cyclophosphamide; FLU, fludarabine; HLA, human leukocyte antigen; MMF, mycophenolate mofetil; MRD, matched related donor; MTX, methotrexate; N, number treated; OS, overall survival; PBSC, peripheral blood stem cell; PK, pharmacokinetic; PNH, paroxysmal nocturnal hemoglobinuria; ppx, prophylaxis; rATG, rabbit antithymocyte globulin; RD, related donor; TAC, tacrolimus; URD, unrelated donor.