Baseline patient, donor, and transplantation characteristics
| . | Baseline characteristics* . | |
|---|---|---|
| Characteristics . | n . | % . |
| Patient | ||
| Overall | 169 | |
| MLD | 66 | 39.1 |
| GLD | 47 | 27.8 |
| X-ALD | 56 | 33.1 |
| Child (<18 y)/adult | 161/8 | 95.3/4.7 |
| Sex (male/female) | 118/51 | 69.8/30.2 |
| Cytomegalovirus serology (negative) | 127 | 77.9 |
| Median | Range | |
| Weight, kg | 19.5 | 2.74-75.0 |
| Age at SCT, y | ||
| Overall | 5.1 | 0.1-43.3 |
| MLD | 4.3 | 0.1-22.7 |
| GLD | 0.6 | 0.1-16.7 |
| X-ALD | 8.2 | 2.4-43.3 |
| Interval diagnosis-transplant, mo | ||
| Overall | 2.8 | 0.6-147.9 |
| MLD | 3.0 | 0.6-70.3 |
| GLD | 1.6 | 0.6-60.0 |
| X-ALD | 5.4 | 0.6-147.9 |
| Performance score pre-SCT | ||
| <60 | 29 | 19.6 |
| 60-80 | 29 | 19.6 |
| >80 | 90 | 60.8 |
| Donor | n | % |
| sCB | 156 | 92.3 |
| dCB | 13 | 7.7 |
| Unrelated/related | 168/1 | 99.4/0.6 |
| HLA matching | ||
| 6/6 | 27 | 18.1 |
| 5/6 | 56 | 37.6 |
| 4/6 | 65 | 43.6 |
| 3/6 | 1 | 0.7 |
| CB cell dose | Median | Range |
| Collected NC, ×107/kg | 8.0 | 2.1-67.2 |
| Collected CD34+, ×105/kg | 2.7 | 0.01-28.5 |
| Infused NC, ×107/kg | 5.7 | 1.2-50.3 |
| Infused CD34+, ×105/kg | 2.1 | 0.01-32.4 |
| Transplantation | n | % |
| Conditioning regimen | ||
| MAC | 163 | 97 |
| Bu/Cy/(TT) | 135 (1) | |
| Bu/Fluda/(Cy)(Mel)(TT) | 9 (4)(3)(3) | |
| Fluda/TT/Mel | 3 | |
| Bu/Mel/other | 1 | |
| TBI/Cy | 4 | |
| RIC | 5 | 3 |
| Fluda/Mel | 1 | |
| Mel/other | 1 | |
| Fluda/other | 2 | |
| Cy/Fluda/TBI | 1 | |
| GVHD prophylaxes | ||
| CsA based | 156 | 96.9 |
| +steroids | 34 | |
| +MMF | 29 | |
| +Mtx | 4 | |
| Tacrolimus-based | 5 | 3.1 |
| +MMF | 4 | |
| +Mtx | 1 | |
| Serotherapy (ATG before day 0) | 162 | 96.4 |
| Median | Range | |
| Year of HCT | 2006 | 1996-2013 |
| Follow-up post SCT,† mo | 76.3 | 3.3-211 |
| . | Baseline characteristics* . | |
|---|---|---|
| Characteristics . | n . | % . |
| Patient | ||
| Overall | 169 | |
| MLD | 66 | 39.1 |
| GLD | 47 | 27.8 |
| X-ALD | 56 | 33.1 |
| Child (<18 y)/adult | 161/8 | 95.3/4.7 |
| Sex (male/female) | 118/51 | 69.8/30.2 |
| Cytomegalovirus serology (negative) | 127 | 77.9 |
| Median | Range | |
| Weight, kg | 19.5 | 2.74-75.0 |
| Age at SCT, y | ||
| Overall | 5.1 | 0.1-43.3 |
| MLD | 4.3 | 0.1-22.7 |
| GLD | 0.6 | 0.1-16.7 |
| X-ALD | 8.2 | 2.4-43.3 |
| Interval diagnosis-transplant, mo | ||
| Overall | 2.8 | 0.6-147.9 |
| MLD | 3.0 | 0.6-70.3 |
| GLD | 1.6 | 0.6-60.0 |
| X-ALD | 5.4 | 0.6-147.9 |
| Performance score pre-SCT | ||
| <60 | 29 | 19.6 |
| 60-80 | 29 | 19.6 |
| >80 | 90 | 60.8 |
| Donor | n | % |
| sCB | 156 | 92.3 |
| dCB | 13 | 7.7 |
| Unrelated/related | 168/1 | 99.4/0.6 |
| HLA matching | ||
| 6/6 | 27 | 18.1 |
| 5/6 | 56 | 37.6 |
| 4/6 | 65 | 43.6 |
| 3/6 | 1 | 0.7 |
| CB cell dose | Median | Range |
| Collected NC, ×107/kg | 8.0 | 2.1-67.2 |
| Collected CD34+, ×105/kg | 2.7 | 0.01-28.5 |
| Infused NC, ×107/kg | 5.7 | 1.2-50.3 |
| Infused CD34+, ×105/kg | 2.1 | 0.01-32.4 |
| Transplantation | n | % |
| Conditioning regimen | ||
| MAC | 163 | 97 |
| Bu/Cy/(TT) | 135 (1) | |
| Bu/Fluda/(Cy)(Mel)(TT) | 9 (4)(3)(3) | |
| Fluda/TT/Mel | 3 | |
| Bu/Mel/other | 1 | |
| TBI/Cy | 4 | |
| RIC | 5 | 3 |
| Fluda/Mel | 1 | |
| Mel/other | 1 | |
| Fluda/other | 2 | |
| Cy/Fluda/TBI | 1 | |
| GVHD prophylaxes | ||
| CsA based | 156 | 96.9 |
| +steroids | 34 | |
| +MMF | 29 | |
| +Mtx | 4 | |
| Tacrolimus-based | 5 | 3.1 |
| +MMF | 4 | |
| +Mtx | 1 | |
| Serotherapy (ATG before day 0) | 162 | 96.4 |
| Median | Range | |
| Year of HCT | 2006 | 1996-2013 |
| Follow-up post SCT,† mo | 76.3 | 3.3-211 |
ATG, anti-thymocyte globulin; Bu, busalfan; CsA, cyclosporine; Cy, cyclophosphamide; dCB, double cord blood; Fluda, fludarabine; MAC, myoablative conditioning; Mel, melphalan; MMF, mofetil; Mtx, methotrexate; NC, neutrophil count; RIC, reduced intensity conditioning; sCD, single cord blood; SCT, stem cell transplantation; TBI, total body irradiation; TT, thiotepa.
CB transplantation was performed in the following centers: Duke (102), Utrecht (11), Marseille (6), Australia (6), Saudi Arabia (5), Madrid (4), Gent (4), Brussels (4), Paris (4), Milano (2), Murcia (2), Barcelona (2), Montréal (2), Budapest (2), Leiden (1), Leuven (1), Jerusalem (1), Porto (1), Hannover (1), Lisboa (1), Berlin (1), Manchester (1), Antalya (1), Poland (1), Nancy (1), Israel (1), and Bratislava (1).
Median follow-up of survivors.