Select recent reports of HLA-matched sibling and unrelated donor hematopoietic cell transplantation for severe aplastic anemia
| Reference (year) . | Year of transplant . | Patients, n . | Age range (median), y . | Conditioning program . | Hematopoietic source (n) . | Prevention of GVHD . | Graft rejection/failure, % . | GVHD, % . | Survival, % . | Follow-up, range (median), y . | |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Acute . | Chronic . | ||||||||||
| HLA-identical sibling donor transplantation | |||||||||||
| 30 (2012) | 1971-1984 1981-1988 1989-2010 | 98 19 31 | 1.8-19 (12.8) | CY CY CY+ATG | BM | MTX CSP+MTX CSP+MTX | 22 32 7 | 21 11 39 | 21 21 10 | 66 95 100 | 11-37.2 (31.1) 15.2-27.1 (23) 0.3-21.5 (6.1) |
| 49 (2012) | 1999-2009 | 1886 | 1-68 (18) | CY+ATG (41%) and various | BM (1163) | CSP+MTX (41%) or other | 9 | 11 | 11 | Age ≤20 y: 90 Age >20 y: 74 | (2.1) |
| 1-69 (24) | PBSCs (723) | 10 | 17 | 22 | Age ≤20 y: 76 Age >20 y: 64 | (2.0) | |||||
| 52 (2009) | 1998-2007 | 30 | 31-66 (46) | FLU+CY+ATG | BM (20) PBSCs (10) | CSP+MTX or other | 3 | 10 | 13 | 77 | 1.1-6.8 (4.1) |
| 82 (2016) | 1999-2014 | BMT: 1732 IST: 802 | 0-20 21-40 >40 | BMT various | Compare BMT vs IST | Various for BMT | N/A | N/A | N/A | Age 0-20 y: 86 (BMT); 84 (IST) Age 21-40 y: 76 (BMT); 65 (IST) Age >40 y: 56 (BMT); 58 (IST) | 0.2-10 |
| IST various | |||||||||||
| 50 (2015) | 2005-2009 | 940 | Age >20 y: 50% | Various | BM (566) PBSCs (374) | Various | 9 | 13 | 14 | Low risk: 93 Int. risk: 78 High risk: 67 | 0.1-9.1 (3.1) |
| 31 (2016) | 2006-2015 | 21 | 3-52 (15) | CY+ATG | BM <2.5 × 108 TNC/kg | CSP+MTX | 0 | 47 | 16 (24) | 100 | 1-8 (4.0) |
| Unrelated donor transplantation | |||||||||||
| 57 (2012) | 2000-2010 | 44 | 3-19 (8) | FLU+CY+Alem various doses | BM (26) PBSCs (18) | CSP or CSP+MMF or MTX | 0 | 38 | 12 | 95 | 1-6.3 (2.9) |
| 50 (2015) | 2005-2009 | 508 | Age >20 y: 53% | Various | BM (264) PBSCs (244) | Various | 9 | 25 | 26 | Low risk: 83 Int. risk: 77 High risk: 64 | 0.1-9.1 (3.1) |
| 58 (2015) | 2005-2014 | 29 Upfront MUD HCT (no prior IST) | 1.7-19.1 (8.4) | FLU+CY+Alem (+2-3-Gy TBI for 1 Ag-MM) | BM (21) PBSCs (8) | CSP or CSP/MMF | 4 | 10 | 19 | 96 OS 92 2-y EFS | 0.2-8.5 (1.7) |
| 56 (2015) | 2006-2013 | 79 | 0.5-66 (24) | ATG+2-Gy TBI+CY-100 (n = 41) vs CY-50 (n = 38) | BM | CSP/MTX | CY-100: 15 CY-50: 12 | 27 24 | 32 23 | 81 at 1 y 97 at 1 y | 1.0-4.2 (2.0) 0.3-2.2 (1.4) |
| 53 (2014) | 1999-2009 | 55 URD | 1-67 (18) | FLU+CY+Alem | BM (36) PBSCs (19) | Tacrolimus/MMF | 9 | 38 | 13 | 88 97 BM 70 PBSCs | 0.5-10 |
| Reference (year) . | Year of transplant . | Patients, n . | Age range (median), y . | Conditioning program . | Hematopoietic source (n) . | Prevention of GVHD . | Graft rejection/failure, % . | GVHD, % . | Survival, % . | Follow-up, range (median), y . | |
|---|---|---|---|---|---|---|---|---|---|---|---|
| Acute . | Chronic . | ||||||||||
| HLA-identical sibling donor transplantation | |||||||||||
| 30 (2012) | 1971-1984 1981-1988 1989-2010 | 98 19 31 | 1.8-19 (12.8) | CY CY CY+ATG | BM | MTX CSP+MTX CSP+MTX | 22 32 7 | 21 11 39 | 21 21 10 | 66 95 100 | 11-37.2 (31.1) 15.2-27.1 (23) 0.3-21.5 (6.1) |
| 49 (2012) | 1999-2009 | 1886 | 1-68 (18) | CY+ATG (41%) and various | BM (1163) | CSP+MTX (41%) or other | 9 | 11 | 11 | Age ≤20 y: 90 Age >20 y: 74 | (2.1) |
| 1-69 (24) | PBSCs (723) | 10 | 17 | 22 | Age ≤20 y: 76 Age >20 y: 64 | (2.0) | |||||
| 52 (2009) | 1998-2007 | 30 | 31-66 (46) | FLU+CY+ATG | BM (20) PBSCs (10) | CSP+MTX or other | 3 | 10 | 13 | 77 | 1.1-6.8 (4.1) |
| 82 (2016) | 1999-2014 | BMT: 1732 IST: 802 | 0-20 21-40 >40 | BMT various | Compare BMT vs IST | Various for BMT | N/A | N/A | N/A | Age 0-20 y: 86 (BMT); 84 (IST) Age 21-40 y: 76 (BMT); 65 (IST) Age >40 y: 56 (BMT); 58 (IST) | 0.2-10 |
| IST various | |||||||||||
| 50 (2015) | 2005-2009 | 940 | Age >20 y: 50% | Various | BM (566) PBSCs (374) | Various | 9 | 13 | 14 | Low risk: 93 Int. risk: 78 High risk: 67 | 0.1-9.1 (3.1) |
| 31 (2016) | 2006-2015 | 21 | 3-52 (15) | CY+ATG | BM <2.5 × 108 TNC/kg | CSP+MTX | 0 | 47 | 16 (24) | 100 | 1-8 (4.0) |
| Unrelated donor transplantation | |||||||||||
| 57 (2012) | 2000-2010 | 44 | 3-19 (8) | FLU+CY+Alem various doses | BM (26) PBSCs (18) | CSP or CSP+MMF or MTX | 0 | 38 | 12 | 95 | 1-6.3 (2.9) |
| 50 (2015) | 2005-2009 | 508 | Age >20 y: 53% | Various | BM (264) PBSCs (244) | Various | 9 | 25 | 26 | Low risk: 83 Int. risk: 77 High risk: 64 | 0.1-9.1 (3.1) |
| 58 (2015) | 2005-2014 | 29 Upfront MUD HCT (no prior IST) | 1.7-19.1 (8.4) | FLU+CY+Alem (+2-3-Gy TBI for 1 Ag-MM) | BM (21) PBSCs (8) | CSP or CSP/MMF | 4 | 10 | 19 | 96 OS 92 2-y EFS | 0.2-8.5 (1.7) |
| 56 (2015) | 2006-2013 | 79 | 0.5-66 (24) | ATG+2-Gy TBI+CY-100 (n = 41) vs CY-50 (n = 38) | BM | CSP/MTX | CY-100: 15 CY-50: 12 | 27 24 | 32 23 | 81 at 1 y 97 at 1 y | 1.0-4.2 (2.0) 0.3-2.2 (1.4) |
| 53 (2014) | 1999-2009 | 55 URD | 1-67 (18) | FLU+CY+Alem | BM (36) PBSCs (19) | Tacrolimus/MMF | 9 | 38 | 13 | 88 97 BM 70 PBSCs | 0.5-10 |
Alem, alemtuzumab; BM, bone marrow; CY-50, 50 mg/kg CY; CY-100, 100 mg/kg CY; EFS, event-free survival; HCT, hematopoietic cell transplantation; Int., intermediate; MMF, mycophenolate mofetil; MUD, matched unrelated donor; N/A, not available; OS, overall survival; TBI, total body irradiation; TNC, total nucleated cell; URD, unrelated donor; 1 Ag-MM, 1 HLA-antigen mismatch.