Hematopoietic cell transplantation for sickle cell disease.
| . | Minimal-Toxicity Conditioning Regimen . | Reduced-Intensity Conditioning Regimen . | Conventional Myeloablative Conditioning Regimen . |
|---|---|---|---|
| †Includes 2 patients with thalassemia major. | |||
| ‡Includes 1 patient with thalassemia major. | |||
| Abbreviations: ATG, antithymocyte globulin; BU, busulfan; CY, cyclophosphamide; Flu, fludarabine; Gr, grade; GVHD, graft-versus-host disease; Mel, melphalan; No., number; PBHC, peripheral blood hematopoietic cells; TBI, total body irradiation; TLI, total lymphoid irradiation; CB, umbilical cord blood | |||
| No. of patients | 11† | 12‡ | 201 |
| Patient age (median, yrs) | 11 (range, 3–30) | 22 (range, 5–56) | – (range, 0.9–22) |
| Conditioning regimen (dose) [no. of patients] | Flu (90–150 mg/m2)/TBI (200 cGy) [5]; Flu (125–150 mg/m2)/ATG/TBI (200 cGy)[6] | Flu (175 mg/m2)/BU (8 mgkg)/ATG/TLI (500 cGy) [5]; Flu (120 mg/m2)/Mel(140 mg/m2)/ATG[2]; Flu (120 mg/m2)/CY (120 mg/kg) [1]; Flu (120 mg/m2)/Mel (140 mg/m2)/Campath [2]; Flu (120 mg/m2)/BU (3.2 mg/kg) [2] | BU/CY/ATG [133] BU/CY [59] BU/CY/TLI [6] CY/TBI [3] |
| Source of stem cells (no. of patients) | Marrow (9); PBHC (2) | Marrow (9); PBHC (6); CB (2) | Marrow |
| Induction of mixed chimerism | Yes (transient in 10) | Yes | Yes, in 11% |
| No. with graft rejection/disease recurrence | 10 | 3 | 16 (8%) |
| No. with GVHD | Acute 1 (Gr I), chronic, none | Acute 4 (Gr II–IV), chronic, 3 (2 fatal) | acute 25%, chronic 12% |
| No. of deaths | None | 2 | 20 (10%) |
| No. with event-free survival | 1 (9%) | 7 (58%) | 166 (83%) |
| . | Minimal-Toxicity Conditioning Regimen . | Reduced-Intensity Conditioning Regimen . | Conventional Myeloablative Conditioning Regimen . |
|---|---|---|---|
| †Includes 2 patients with thalassemia major. | |||
| ‡Includes 1 patient with thalassemia major. | |||
| Abbreviations: ATG, antithymocyte globulin; BU, busulfan; CY, cyclophosphamide; Flu, fludarabine; Gr, grade; GVHD, graft-versus-host disease; Mel, melphalan; No., number; PBHC, peripheral blood hematopoietic cells; TBI, total body irradiation; TLI, total lymphoid irradiation; CB, umbilical cord blood | |||
| No. of patients | 11† | 12‡ | 201 |
| Patient age (median, yrs) | 11 (range, 3–30) | 22 (range, 5–56) | – (range, 0.9–22) |
| Conditioning regimen (dose) [no. of patients] | Flu (90–150 mg/m2)/TBI (200 cGy) [5]; Flu (125–150 mg/m2)/ATG/TBI (200 cGy)[6] | Flu (175 mg/m2)/BU (8 mgkg)/ATG/TLI (500 cGy) [5]; Flu (120 mg/m2)/Mel(140 mg/m2)/ATG[2]; Flu (120 mg/m2)/CY (120 mg/kg) [1]; Flu (120 mg/m2)/Mel (140 mg/m2)/Campath [2]; Flu (120 mg/m2)/BU (3.2 mg/kg) [2] | BU/CY/ATG [133] BU/CY [59] BU/CY/TLI [6] CY/TBI [3] |
| Source of stem cells (no. of patients) | Marrow (9); PBHC (2) | Marrow (9); PBHC (6); CB (2) | Marrow |
| Induction of mixed chimerism | Yes (transient in 10) | Yes | Yes, in 11% |
| No. with graft rejection/disease recurrence | 10 | 3 | 16 (8%) |
| No. with GVHD | Acute 1 (Gr I), chronic, none | Acute 4 (Gr II–IV), chronic, 3 (2 fatal) | acute 25%, chronic 12% |
| No. of deaths | None | 2 | 20 (10%) |
| No. with event-free survival | 1 (9%) | 7 (58%) | 166 (83%) |