Baseline patient, donor, and transplantation characteristics
| Patient characteristics . | n . | % . | Missing . | Median . | Range . |
|---|---|---|---|---|---|
| Overall | 258 | ||||
| Gender (male/female) | 133/121 | 52 | 4 | ||
| IV ERT (before HCT; at least 4 infusions) | 48 | 18.6 | 1 | ||
| Age at SCT (mo) | 0 | 16.7 | 2.1-228 | ||
| Interval diagnosis-transplant (mo) | 32 | 5.2 | 1-63.6 | ||
| Follow-up post SCT (mo) | 0 | 59 | 1.3-159 | ||
| Donor characteristics | |||||
| MSD (all BM) | 37 | 14 | 0 | ||
| UD (95BM/15PBSC) | 105 | 41 | 0 | ||
| UCB | 116 | 45 | 0 | ||
| UD* | 3 | ||||
| 10/10 (HR) | 47 | 47 | |||
| 7-9/10 (HR) | 27 | 26 | |||
| 6-8/8.5-6/6 (LR/IR) | 28 | 27 | |||
| uCB HLA-matching | 3 | ||||
| 6/6 | 22 | 19 | |||
| 5/6 | 66 | 58 | |||
| 4/6 | 25 | 22 | |||
| 3/6 | 3 | 3 | |||
| CB cell dose | |||||
| Collected NC (·107/kg) | 2 | 8.8 | 1.2-32 | ||
| Collected CD34+ (·105/kg) | 3 | 3.0 | 0.21-105 | ||
| Transplantation characteristics | |||||
| Year of HCT | 0 | 2004 | 1995-2007 | ||
| Overall | 258 | ||||
| Conditioning regimen | 6 | ||||
| Bu/Cy | 181 | 70.2 | |||
| Bu/Cy (high) | 34 | 13.2 | |||
| Bu/Cy/Flud | 12 | 4.7 | |||
| Bu/Cy/TBI (200 cGy) | 12 | 4.7 | |||
| Bu + other combinations | 11 | 4.3 | |||
| TBI/Cy | 2 | 0.8 | |||
| GVHD prophylaxis | 29 | ||||
| CsA | 21 | 8.1 | |||
| CsA/Pred | 78 | 30.2 | |||
| CsA/MTX | 77 | 29.8 | |||
| CsA/MMF | 22 | 8.5 | |||
| CsA/MTX/Pred | 1 | 0.4 | |||
| None given (in TCD grafts only) | 30 | 11.6 | |||
| Serotherapy (ATG: 221 or Campath: 21) | 228 | 90 | 4 |
| Patient characteristics . | n . | % . | Missing . | Median . | Range . |
|---|---|---|---|---|---|
| Overall | 258 | ||||
| Gender (male/female) | 133/121 | 52 | 4 | ||
| IV ERT (before HCT; at least 4 infusions) | 48 | 18.6 | 1 | ||
| Age at SCT (mo) | 0 | 16.7 | 2.1-228 | ||
| Interval diagnosis-transplant (mo) | 32 | 5.2 | 1-63.6 | ||
| Follow-up post SCT (mo) | 0 | 59 | 1.3-159 | ||
| Donor characteristics | |||||
| MSD (all BM) | 37 | 14 | 0 | ||
| UD (95BM/15PBSC) | 105 | 41 | 0 | ||
| UCB | 116 | 45 | 0 | ||
| UD* | 3 | ||||
| 10/10 (HR) | 47 | 47 | |||
| 7-9/10 (HR) | 27 | 26 | |||
| 6-8/8.5-6/6 (LR/IR) | 28 | 27 | |||
| uCB HLA-matching | 3 | ||||
| 6/6 | 22 | 19 | |||
| 5/6 | 66 | 58 | |||
| 4/6 | 25 | 22 | |||
| 3/6 | 3 | 3 | |||
| CB cell dose | |||||
| Collected NC (·107/kg) | 2 | 8.8 | 1.2-32 | ||
| Collected CD34+ (·105/kg) | 3 | 3.0 | 0.21-105 | ||
| Transplantation characteristics | |||||
| Year of HCT | 0 | 2004 | 1995-2007 | ||
| Overall | 258 | ||||
| Conditioning regimen | 6 | ||||
| Bu/Cy | 181 | 70.2 | |||
| Bu/Cy (high) | 34 | 13.2 | |||
| Bu/Cy/Flud | 12 | 4.7 | |||
| Bu/Cy/TBI (200 cGy) | 12 | 4.7 | |||
| Bu + other combinations | 11 | 4.3 | |||
| TBI/Cy | 2 | 0.8 | |||
| GVHD prophylaxis | 29 | ||||
| CsA | 21 | 8.1 | |||
| CsA/Pred | 78 | 30.2 | |||
| CsA/MTX | 77 | 29.8 | |||
| CsA/MMF | 22 | 8.5 | |||
| CsA/MTX/Pred | 1 | 0.4 | |||
| None given (in TCD grafts only) | 30 | 11.6 | |||
| Serotherapy (ATG: 221 or Campath: 21) | 228 | 90 | 4 |
Bu, busulfan; CsA, cyclosporine; Cy, cyclophosphamide (high was defined as either 240 or 260 mg); Flud, fludarabine; Pred, prednisolone; SCT, stem cell transplant(ation).
HCT was performed in the following centers: Minneapolis (57), Duke (47), Manchester (29), Dublin (23), Lyon (15), Utrecht, (12), London (11), Marseille (3), Barcelona (4), Madrid (2), Israel (1), Argentina (1), Japan (2), Ghent (4), Nancy (1), Paris (11), Padua (1), Monza (9) Prague (4), Helsinki (1), Australia (1), New Zealand (1), Hanover (7), Stockholm (1), Zurich (1), Moscow (2), Cincinnati (2), Vienna (1), Jena (1), Leiden (1), Swansea (1), and Houston (1).
Of these 105 patients, 53 were T-cell–depleted. According to the HLA, T-cell–depleted HCT was performed in 10/47 (HLA 10/10) and 43 of 55 patients with HLA-mismatched UD.