Table 2.
Univariate predictors of survival and being alive and engrafted
| A&E | Alive | ||||||||
|---|---|---|---|---|---|---|---|---|---|
| n | % | OR | 95% CI | P-value | % | OR | 95% CI | P-value | |
| Overall | 146 | 56 | 85 | ||||||
| Age | 146 | 0.98 | 0.96–1.01 | 0.23 | 1.02 | 0.99–1.05 | 0.23 | ||
| Gender | 146 | 1.15 | 0.60–2.21 | 0.67 | 0.87 | 0.45–1.72 | 0.70 | ||
| Heterozygote donora | 146 | 1.46 | 0.41–5.2 | 0.90 | 0.49 | 0.055–4.32 | 0.52 | ||
| HLA disparity b | |||||||||
| Matched | 96 | 64 | 1 | 85 | 1 | ||||
| Mismatched | 50 | 42 | 0.41 | 0.21–0.84 | 0.014 | 84 | 0.78 | 0.31–1.95 | 0.59 |
| Conditioning c | |||||||||
| Bu/Cy | 68 | 53 | 1 | 87 | 1 | ||||
| Bu/Cyhi | 30 | 67 | 1.78 | 0.72–4.36 | 0.21 | 80 | 0.55 | 0.57–5.79 | 0.32 |
| Bu target | 15 | 87 | 5.76 | 1.20–27.54 | 0.028 | 87 | 0.89 | 0.17–4.73 | 0.90 |
| Flud-MA | 17 | 70 | 2.13 | 0.67–6.71 | 0.19 | 88 | 0.97 | 0.19–5.04 | 0.97 |
| RIC | 18 | 11 | 0.11 | 0.02–0.52 | 0.005 | 78 | 0.48 | 0.13–1.83 | 0.29 |
| TCD d | |||||||||
| No | 118 | 64 | 1 | 84 | 1 | ||||
| Yes | 28 | 25 | 0.19 | 0.08–0.49 | 0.001 | 89 | 1.15 | 0.36–1.95 | 0.81 |
| Donor | |||||||||
| Family | 52 | 58 | 1 | 90 | 1 | ||||
| Unrelatede | 94 | 55 | 0.91 | 0.46–1.80 | 0.78 | 82 | 2.08 | 0.72–6.01 | 0.18 |
| Source | |||||||||
| BM | 103 | 55 | 1 | 84 | 1 | ||||
| PBSC | 20 | 50 | 0.81 | 0.31–2.11 | 0.67 | 95 | 4.02 | 0.51–32.02 | 0.19 |
| CBf | 23 | 65 | 1.51 | 0.59–3.88 | 0.39 | 83 | 1.00 | 0.31–3.31 | 0.99 |
| ERT g | |||||||||
| No | 127 | 55.9 | 1 | 82.7 | 1 | ||||
| Yes | 19 | 57.9 | 1.09 | 0.41–2.88 | 0.87 | 94.7 | 3.77 | 0.48–29.75 | 0.21 |
Abbreviations: A&E=alive and engrafted; BM=bone marrow; CB=cord blood; CI=confidence interval; ERT, enzyme replacement therapy; Flud-MA=fludarabine-based myeloablative; HLA=human leukocyte antigen; OR=odds ratios; RIC=reduced-intensity conditioning; TCD=T-cell depletion.
aUnrelated donors were regarded as not carrying the α-L-iduronidase mutation.
bFrom the matched donors, 46 were family members and 50 were unrelated. From the mismatched donors, 6 were family members and 44 unrelated: 32 had 1 mismatch, 6 had 2 mismatches and 11 more than 2 mismatches. From one patient, the mismatch grade is unclear.
cBusulfan was given in the regular myeloablative doses (16 or 20 mg/kg) p.o., unless otherwise indicated. Groups were subdivided: Bu/Cy=busulfan+cyclophosphamide 200 mg/kg including one patient receiving+10 mg/kg thiothepa, Bu/Cyhi=busulfan+cyclophosphamide 240 or 260 mg/kg, Bu target=Bu/Cy (4) or Bu/Cyhi (9) or Bu/Cy+fludarabine 150 mg/m2 (2), Flud-based myeloablation (MA)=Bu/Cy+fludarabine 150 mg/m2 (9) or busulfan+fludarabine 180 mg/m2 (4) or busulfan+melphalan 4 mg/kg+fludarabine 150 mg/m2 (4) and reduced-intensity conditioning (RIC)=melphalan 140 mg/m2+fludarabine 150 mg/m2 (8) or melphalan 140 mg/m2+TLI 2 Gy+fludarabine 150 mg/m2 (2) or busulfan 10 mg/kg+fludarabine 150 mg/m2 (2) and treosulfan 36 or 42 g/m2+fludarabine 150 mg/m2 (6). Busulfan target: either steady state 600–900 ng/ml (n=10) or daily areas under the curves (AUCs) of 17 500–25 000 μg/l × h (n=5). Twelve received an adjusted dose on the second day. Busulfan was given either p.o. or i.v. No VOD was seen in this group.
dCD3+ ranging from <5 × 104/kg to 107/kg.
eFor unrelated donors, serotherapy was given: either ATG or Campath-1H depending on institutional protocols.
f Median cell dose of the CBs used was as follows: in NC/kg 7.8 (range 2.7–20.0) × 107 and in CD34+/kg (n=13) 2.5 (1.1–10.0) × 105. Three of the 23 patients received a CB from an HLA-identical sibling donor and the rest was unrelated.
gERT=enzyme replacement therapy, pre-SCT.
P-values <0.1 were selected for multivariate analysis. Bold and italic indicates the P-value, bold alone for the OR.