Table 1.
Demographic and Clinical Characteristics of IEM Patients Treated with BMT
| Characteristic | Entire Cohort (N = 154) | Alive Participants (N = 106) |
|---|---|---|
| Age at study participation, yr | ||
| Median (range) | 18.2 (2.7–56.5) | 19.8 (4.6–56.5) |
| Mean ± SD | 19.3 ± 11.6 | 21.4 ± 11.1 |
| Sex, n (%) | ||
| Male | 101 (65.6) | 78 (73.6) |
| Race/ethnicity, n (%) | ||
| Non-Hispanic white | 139 (90.3) | 98 (92.5) |
| Hispanic | 8 (5.2) | 4 (3.8) |
| Other | 7 (4.5) | 4 (3.8) |
| Education, n (%) | ||
| Less than high school | - | 37 (44.6) |
| High school and/or some college | - | 36 (43.4) |
| College graduate or higher education | - | 10 (12.0) |
| Annual household income, n (%) | ||
| ≤$20,000 | - | 26 (32.1) |
| >$20,000 | - | 55 (67.9) |
| Current health insurance, n (%) | ||
| Yes | - | 105 (99.0) |
| Age at BMT, yr | ||
| Median (range) | 3.0 (0.2–44.2) | 3.7 (0.2–42.3) |
| Time from BMT to study participation (or death), yr | ||
| Median (range) | 10.8 (2.1–38.1) | 15.2 (3.0–38.1) |
| Primary IEM diagnosis, n (%) | ||
| Leukodystrophies | 67 (43.5) | 50 (47.2) |
| Mucopolysaccharidoses | 63 (41.0) | 48 (45.2) |
| Other IEMs | 24 (15.5) | 8 (7.6) |
| Year of BMT, n (%) | ||
| <2000 | 77 (50.0) | 55 (51.9) |
| 2000–2009 | 51 (33.1) | 28 (26.4) |
| ≥2010 | 26 (16.9) | 23 (21.7) |
| Stem cell source, n (%) | ||
| Bone marrow | 100 (64.9) | 70 (66.0) |
| Cord blood | 52 (33.8) | 36 (33.0) |
| PBSCs | 2 (1.3) | 1 (1.0) |
| Type of donor, n (%) | ||
| Unrelated donor | 99 (64.3) | 66 (62.3) |
| Type of conditioning, n (%) | ||
| Myeloablative conditioning | 126 (81.8) | 89 (84.0) |
| Conditioning agents, n (%) | ||
| Busulfan + cyclophosphamide | 77 (50.0) | 54 (50.9) |
| Cyclophosphamide + TBI4 | 24 (15.6) | 19 (17.9) |
| Fludarabine + melphalan | 28 (18.2) | 17 (16.0) |
| Other conditioning | 25 (16.2) | 16 (15.1) |
| >1 BMT | ||
| Yes | 20 (13.0) | 12 (11.3) |
| Chronic GVHD, n (%) | ||
| Yes | 15 (12.2) | 6 (6.7) |
| No | 108 (87.8) | 84 (93.3) |
| GVHD prophylaxis, n (%) | ||
| Cyclosporine | 138 (93.2) | 99 (96.1) |
| Mycophenolate mofetil | 48 (32.4) | 34 (33.0) |
| T cell depletion | 45 (30.4) | 30 (29.1) |
Percent calculations exclude 23 participants who did not report education status and 25 participants who did not report household income. Chronic GVHD status was not available for 31 patients in the entire cohort and for 16 patients among those alive at study participation (excluded from percent calculations). Six patients were missing information regarding GVHD prophylaxis, among whom 3 were alive at study participation.