Table 2.
Characteristics of patients who were alive 2-years after transplantation.
| Characteristic | Number (percent) |
|---|---|
| Number | 950 |
| Age at transplant, years | |
| ≤10 | 458 (48%) |
| 11 – 17 | 276 (29%) |
| 18 – 29 | 142 (15%) |
| 30 – 39 | 47 (5%) |
| 40 – 49 | 21 (2%) |
| ≥ 50 | 6 (1%) |
| Sex, male/female | 531(56%)/419(44%) |
| Disease type, Hemoglobin SS/SP-thalassemia | 917(97%)/33(3%) |
| Performance score | |
| ≤ 80 | 187 (20%) |
| 90 – 100 | 711 (75%) |
| Not reported | 52 (5%) |
| Donor type | |
| HLA-matched sibling | 627 (66%) |
| Haploidentical relative | 124 (13%) |
| HLA-matched unrelated | 95 (10%) |
| HLA-mismatched unrelated | 104 (11%) |
| Graft type | |
| Bone marrow | 669 (70%) |
| Peripheral blood | 167 (18%) |
| Umbilical cord blood | 114 (12%) |
| Conditioning regimen intensity | |
| Myeloablative | 496 (52%) |
| Reduced-intensity | 237 (25%) |
| Non-myeloablative | 173 (18%) |
| Not reported | 44 (5%) |
| In vivo T-cell depletion | |
| None | 67 (7%) |
| Yes | 864 (91%) |
| GVHD prophylaxis | |
| Ex vivo T-cell depletion | 15 (2%) |
| CD 34 selection | 34 (4%) |
| Post-transplant cyclophosphamide/CNI/other | 92 (10%) |
| CNI/mycophenolate or methotrexate | 643 (68%) |
| CNI/prednisone | 63 (7%) |
| Other | 103 (11%) |
| History of grade II-IV acute GVHD | |
| None | 777 (82%) |
| Yes | 146 (15%) |
| Not reported | 27 (3%) |
| History of chronic GVHD | |
| None | 727 (77%) |
| Yes | 217 (23%) |
| Not reported | 6 (1%) |
| Transplant period | |
| 2000 – 2005 | 101 (11%) |
| 2006 – 2010 | 182 (19%) |
| 2011 – 2015 | 443 (47%) |
| 2016 – 2017 | 224 (24%) |
| Follow up, median (range), months | 62 (24 – 244) |
Abbreviation:
CNI = calcineurin inhibitor
GVHD = graft versus host disease
Conditioning regimen intensity
Myeloablative: busulfan was administered orally at a concentration greater than 8 mg/kg or intravenously at a concentration greater than 6 mg/kg, or melphalan was administered at concentrations greater than 150 mg/m2
Reduced intensity: lower doses of busulfan or melphalan
Non-myeloablative: Total body irradiation regimens (dose 200–400 cGy) alone or with cyclophosphamide and/or fludarabine