TABLE 3.
Patient Characteristics
| Genotype | Sex | Age(y) | Duration of Enzyme Replacement Therapy (y) |
No Treatment (y) |
Liver Volume (cm3) |
Spleen Statusa |
Bone Marrow Burdenb |
Other | ||
|---|---|---|---|---|---|---|---|---|---|---|
| Lumbar Spine | Femur | Total | ||||||||
| N370S/N370S | F | 53 | 8 | 45 | 1,503 | 1 | 0 | 7 | 7 | Left hip replacement |
| N370S/N370S | F | 37 | 0 | 37 | 2,015 | 1 | 4 | 2 | 6 | |
| N370S/N370S | F | 50 | 8 | 42 | 1,342 | 2 | 0 | 0 | 0 | |
| N370S/N370S | F | 57 | 8 | 49 | 1,460 | 0 | 0 | 3 | 3 | |
| N370S/N370S | M | 54 | 1 | 53 | 1,912 | 1 | 5 | 3 | 8 | |
| N370S/N370S | F | 51 | 1 | 50 | 2,438 | 1 | 4 | 3 | 7 | |
| N370S/N370S | F | 68 | 8 | 60 | 1,700 | 2 | 3 | 5 | 8 | Right hip replacement |
| N370S/N370S | F | 26 | 0 | 26 | 1,798 | 1 | 0 | 3 | 3 | |
| N370S/N370S | F | 18 | 0 | 18 | 1,826 | 1 | 3 | 3 | 6 | |
| N370S/N370S | M | 22 | 7 | 15 | 1,072 | 1 | 3 | 3 | 6 | |
| N370S/N370S | M | 49 | 9 | 40 | 976 | 2 | 8 | 7 | 15 | Right knee replacement |
| N370S/N370S | F | 48 | 13 | 35 | 648 | 0 | 0 | 4 | 4 | |
| N370S/N370S | F | 55 | 7 | 48 | 713 | 0 | 3 | 5 | 8 | |
| N370S/N370S | F | 71 | 2 | 69 | 1,138 | 0 | 0 | 8 | 8 | |
| N370S/N370S | F | 37 | 6 | 31 | 1,623 | 1 | 0 | 3 | 3 | |
| N370S/N370S | F | 53 | 0 | 53 | 885 | 0 | 4 | 3 | 7 | |
| N370S/N370S | F | 43 | 6 | 37 | 1,563 | 0 | 0 | 3 | 3 | |
| N370S/N370S | F | 60 | 1 | 59 | 1,360 | 1 | 0 | 3 | 3 | |
| N370S/N370S | M | 32 | 0 | 32 | 1,720 | 1 | 7 | 3 | 10 | |
| N370S/L444P | M | 58 | 0 | 58 | 1,915 | 2 | 7 | 7 | 14 | |
| N370S/L444P | F | 33 | 4 | 29 | 2,297 | 2 | 7 | 4 | 11 | Left hip replacement |
| N370S/L444P | F | 33 | 5 | 28 | 1,403 | 2 | 3 | 7 | 10 | Right hip replacement, right knee replacement |
| N370S/L444P | M | 50 | 3 | 47 | 1,205 | 2 | 8 | 8 | 16 | Multiple vertebral compression fractures |
| N370S/L444P | M | 23 | 1 | 22 | 1,569 | 1 | 4 | 3 | 7 | |
| N370S/L444P | F | 47 | 11 | 36 | 823 | 2 | 8 | 8 | 16 | Bilateral hip replacement |
| N370S/L444P | F | 50 | 8 | 42 | 880 | 2 | 3 | 8 | 11 | Bilateral hip replacement, right knee replacement |
| N370S/L444P | M | 70 | 0 | 70 | 1,707 | 1 | 5 | 4 | 9 | |
| N370S/84GG | M | 37 | 8 | 29 | 1,170 | 2 | 3 | 6 | 9 | Multiple vertebral compression fractures |
| N370S/84GG | M | 35 | 10 | 25 | 1,587 | 1 | 0 | 3 | 3 | |
| N370S/84GG | F | 67 | 11 | 56 | 1,180 | 2 | 0 | 6 | 6 | |
| N370S/84GG | F | 46 | 5 | 41 | 1,780 | 2 | 6 | 6 | 12 | Multiple vertebral compression fractures |
| N370S/84GG | M | 70 | 9 | 61 | 1,063 | 2 | 0 | 2 | 2 | |
| N370S/84GG | M | 22 | 0 | 22 | 2,219 | 1 | 5 | 3 | 8 | |
| N370S/84GG | F | 51 | 13 | 38 | 1,294 | 2 | 8 | 8 | 16 | Sacral infarcts |
| N370S/IVS2+1 | M | 18 | 14 | 4 | 1,692 | 1 | 0 | 2 | 2 | |
| N370S/IVS2+1 | M | 25 | 21 | 4 | 1,266 | 1 | 4 | 5 | 9 | |
| N370S/IVS2+1 | F | 28 | 5 | 23 | 1,970 | 1 | 3 | 7 | 10 | |
| N370S/IVS2+1 | M | 50 | 12 | 38 | 842 | 1 | 4 | 6 | 10 | Left hip replacement |
| N370S/IVS2+1 | F | 41 | 1 | 40 | 1,068 | 2 | 5 | 7 | 12 | Bilateral hip replacement |
| N370S/IVS2+1 | M | 46 | 10 | 36 | 1,751 | 2 | 8 | 5 | 13 | |
| N370S/7 | F | 32 | 14 | 18 | 1,918 | 1 | 4 | 6 | 10 | |
| N370S/7 | M | 49 | 0 | 49 | 1,407 | 2 | 6 | 3 | 9 | |
| N370S/7 | M | 58 | 11 | 47 | 1,291 | 2 | 0 | 7 | 7 | |
| N370S/7 | F | 57 | 1 | 56 | 2,160 | 2 | 8 | 8 | 16 | |
| N370S/7 | M | 30 | 1 | 29 | 1,906 | 1 | 5 | 7 | 12 | Right hip replacement |
| N370S/V394L | M | 47 | 0 | 47 | 1,780 | 1 | 4 | 4 | 8 | |
| N370S/R131L | F | 41 | 9 | 32 | 2,334 | 2 | 8 | 8 | 16 | |