Table 1.
Transplantation Outcomes for CGD
| Reference | No. of patients | Patient age in years (median) | Donor source | Conditioning regimen | GVHD prophylaxis | aGVHDa (no. of patients) | cGVHD (no. of patients) | Overall survival | Disease-free survival |
|---|---|---|---|---|---|---|---|---|---|
| Horwitz et al. [64] | 10 | 5–36 (15) | MSD | RIC: Cy/Flu/ATG and DLI | CSA | 1 | 2 | 7/10 (70%) | 6/10 (70%) |
| Seger et al. [65] | 27 | 0.8–38.7 (8.5) | MSD (25) MUD (2) | MAC (17): Bu/Cy MAC (1): Bu/Mel/alemtuzumab MAC (1): Bu/Cy/ATG MAC (1): Bu/Cy/TNI MAC (1): Bu/Cy/TT/ATG MAC (1): Bu/Flu/ATG RIC (2): Bu/Flu/ATG RIC (1): Flu/Cy/ATG RIC (1): Flu/TBI | CSA (27) MTX (13) prednisone (4) | 4 | 3 | 23/27 (85%) | 22/27 (81%) |
| Soncini et al. [66] | 20 | 1.25–21 (6.25) | MSD (9) Sib UCB (1) 10/10 MUD (8) 9/10 MUD (1) UCB (1) | MAC (16): Bu/Cy ± alemtuzumab MAC (1): Bu/Mel/alemtuzumab RIC (2): Flu/Mel/alemtuzumab RIC (1): Bu/Flu/alemtuzumab | CSA | 0 | 3 | 18/20 (90%) | 18/20 (90%) |
| Schuetz et al. [67] | 12 | 4–20 (9.5) | MSD (3) MUD (9) | MAC (3): Bu/Cy MAC (6): Bu/Cy/Flu + alemtuzumab or ATG RIC (2): Flu/Mel/RIT RIC (1): Flu/TBI | Not reported | 0 | 2 | 9/12 (75%) | 7/12 (58%) |
| Goździk et al. [68] | 6 | 1.5–13 (3) | MSD (2) 10/10 MUD (3) 9/10 MUD (1) | MAC: Bu/Cy ± ATG | CSA ± MTX | 1 | 1 | 6/6 (100%) | 6/6 (100%) |
| Martinez et al. [69] | 11 | 1–13 (3.8) | MSD (4) MUD (7) | MAC: Bu/Cy/Cytarabine or Flu/alemtuzumab | MSD: CSA + prednisone MUD: CSA + MTX | 0 | 0 | 11/11 (100%) | 11/11 (100%) |
| Tewari et al. [70] | 12 | 0.67–11.6 (4.95) | MSD (5) Sib CB (1) UCB (6) | MAC (5): Bu/Cy ± ATG MAC (6): Bu/Flu/Cy/ATGRIC (1): Flu/Cy/ATG | CSA/MTX (4) CSA/MMF (5) CSA/steroid (2) CSA/TCD (1) | 1 | 4 | 12/12 (100%) | 10/12 (83.3%) |
| Gungor et al. [78] | 56 | 0.8–40 (12.7) | MSD (18) MRD (3) 10/10 MUD (25) 9/10 MUD (10) | RIC: Bu/Flu + ATG or alemtuzumab | CSA or TAC + MMF | 2 | 4 | 52/56 (93%) | 50/56 (89%) |
| Mehta et al. [79] | 4 | 2–15 (10) | 10/10 MUD (2) 9/10 MUD (2) | RIC: Flu/TBI 200 cGy/alemtuzumab | CSA or TAC + MMF | 1 | 1 | 4/4 (100%) | 3/4 (75%) |
| Oshrine et al. [81] | 3 | 1.1–4 (4) | MUD | RIC: Bu/Flu/alemtuzumab | Not reported | 0 | 0 | 3/3 (100%) | 1/3 (33%) |
| Khandelwal et al. [80] | 18 | 0.45–19.39 (3.18) | MSD (2) MRD (1) 10/10 MUD (10) 9/10 MUD (5) | MAC (14): Bu/Cy/ATG RIC (4): Flu/Mel/alemtuzumab | Cy/pred (15) Cy/pred ± Maraviroc (2) Sirolimus/pred (1) | 5 | 4 | 15/18 (83%) | 15/18 (83%) |
|
Morillo-Gutierrez et al. [73] |
70 | 3.8–19.3 (8.9) | MSD (12) MRD (1) Haplo (1) 10/10 MUD (44) 9/10 MUD (11) UCB (1) | MAC (46): Treo/Flu ± ATG or alemtuzumab MAC (15): Treo/Flu/TT ± ATG or alemtuzumab Other (9): undefined treosulfan based regimen | CSA (4) CSA + MMF (44) CSA/MMF/pred (1) CSA + MTX (13) TAC + MTX (8) | 8 | 9 | 64/70 (91.4%) | 59/70 (84%) |
MSD Matched sibling donor, MRD matched related donor, MUD matched unrelated donor, UCB umbilical cord blood, Haplo haplo-identical donor, MAC myeloablative conditioning, RIC reduced intensity conditioning, Bu busulfan, Cy cyclophosphamide, Flu fludarabine, Mel melphalan, TT thiotepa, TBI total body irradiation, ATG anti-thymoglobulin, Treo treosulfan, CSA cyclosporine, TAC tacrolimus, MTX methotrexate, MMF mycophenolate mofetil
aGrade III–IV aGVHD