Table 2. Characteristics of 45 Patients with MPS VI Undergoing Hematopoietic Stem Cell Transplantation at 19 Transplant Centers.
| Patient characteristic | N | (Median, range) (%) |
|---|---|---|
| Median Age at diagnosis (years) | 44 | (1, <1-9) |
| 0 – 2 | 30 | 68% |
| 3 – 5 | 10 | 23% |
| 6 – 9 | 4 | 9% |
| Median Age at Transplant (years) | 45 | (5, 1-22) |
| 0 – 9 | 39 | 87% |
| 10 – 19 | 4 | 9% |
| 20 – 29 | 2 | 4% |
| Gender | 45 | |
| Male | 24 | 53% |
| Female | 21 | 47% |
| Year of transplant | 45 | |
| 1982-1989 | 3 | 7% |
| 1990-1994 | 5 | 11% |
| 1995-1999 | 21 | 47% |
| 2000-2007 | 16 | 36% |
| Graft type | 45 | |
| Bone marrow | 34 | 76% |
| Peripheral blood | 1 | 2% |
| Cord blood | 10 | 22% |
| Graft type, Unrelated donors | 27 | |
| Bone marrow | 18 | 67% |
| Cord blood | 9 | 33% |
| Conditioning regimen | 44 | |
| Cy + TBI* ± other | 6 | 13% |
| Bu + Cy ± other | 30 | 67% |
| Flu + Mel** | 1 | 2% |
| Other | 7 | 16% |
| Donor | 45 | |
| HLA-identical sibling | 15 | 33% |
| Other related | 3 | 7% |
| Unrelated | 27 | 60% |
| Country | 45 | |
| United States | 27 | 60% |
| Australia | 1 | 2% |
| Brazil | 3 | 7% |
| England | 3 | 7% |
| Japan | 1 | 2% |
| Saudi Arabia | 8 | 18% |
| China | 2 | 4% |
| Median Follow-up of Survivors, Months (range), | 29 | (52, 3-267) |
*
TBI, Total Body Irradiation.
**
Fludarabine + Melphalan.