Patient-, donor-, and transplantation-related characteristics
| . | n (%) . | Median (range) . |
|---|---|---|
| Patient-related characteristics | ||
| Overall | 63 | — |
| Sex (male) | 34 (54) | — |
| Ethnicity (white) | 60 (97) | — |
| Genotype (nonsense-nonsense*) | 28 (47) | — |
| Age at HSCT, mo | — | 15 (3-42) |
| Interval diagnosis, HSCT, mo | — | 6 (0-27) |
| Age at assessment, y | — | 8 (5-18) |
| Donor-related characteristics | ||
| Source (CB/BM/PBSC) | 15/40/8 (24/63/13) | — |
| Relation (related) | 23 (37) | — |
| Carrier status (carrier) | 11 (19) | — |
| Transplantation-related characteristics | ||
| No. of HSCTs (1/2/3) | 46/16/1 (73/25/2) | |
| Year of HSCT | 2002 (1987-2009) | |
| ERT pre-HSCT | 20 (32) | — |
| TBI | 2 (3) | — |
| Donor chimerism (<95%) | 15 (24) | — |
| IDUA level post-HSCT† (below reference‡) | 20 (32) | — |
| IDUA level post-HSCT† (% of mean) | — | 59 (13-190) |
| . | n (%) . | Median (range) . |
|---|---|---|
| Patient-related characteristics | ||
| Overall | 63 | — |
| Sex (male) | 34 (54) | — |
| Ethnicity (white) | 60 (97) | — |
| Genotype (nonsense-nonsense*) | 28 (47) | — |
| Age at HSCT, mo | — | 15 (3-42) |
| Interval diagnosis, HSCT, mo | — | 6 (0-27) |
| Age at assessment, y | — | 8 (5-18) |
| Donor-related characteristics | ||
| Source (CB/BM/PBSC) | 15/40/8 (24/63/13) | — |
| Relation (related) | 23 (37) | — |
| Carrier status (carrier) | 11 (19) | — |
| Transplantation-related characteristics | ||
| No. of HSCTs (1/2/3) | 46/16/1 (73/25/2) | |
| Year of HSCT | 2002 (1987-2009) | |
| ERT pre-HSCT | 20 (32) | — |
| TBI | 2 (3) | — |
| Donor chimerism (<95%) | 15 (24) | — |
| IDUA level post-HSCT† (below reference‡) | 20 (32) | — |
| IDUA level post-HSCT† (% of mean) | — | 59 (13-190) |