Table 3.
Bone manifestations at first infusion of imiglucerasea.
| Age at first infusion of imiglucerase |
||||
|---|---|---|---|---|
| ≥5 to <12 years | ≥12 to <20 years | ≥20 to <30 years | ≥30 to ≤50 years | |
| Patients enrolled | n=156 | n=125 | n=185 | n=423 |
| Bone pain, n (%) | n=118 | n=78 | n=104 | n=258 |
| Absent | 71 (60) | 36 (46) | 43 (41) | 100 (39) |
| Present | 47 (40) | 42 (54) | 61 (59) | 158 (61) |
| Bone crisis, n (%) | n=115 | n=75 | n=97 | n=238 |
| Absent | 101 (88) | 59 (79) | 83 (86) | 213 (89) |
| Present | 14 (12) | 16 (21) | 14 (14) | 25 (11) |
| Radiologic bone disease, n (%) | n=104 | n=76 | n=112 | n=279 |
| Evidence of any bone disease | ||||
| Absent | 19 (18.3) | 8 (10.5) | 14 (12.5) | 34 (12.2) |
| Present | 85 (81.7) | 68 (89.5) | 98 (87.5) | 245 (87.8) |
| Type of bone disease reported | Any data available |
Abnormality present |
Any data available |
Abnormality present |
Any data available |
Abnormality present |
Any data available |
Abnormality present |
|---|---|---|---|---|---|---|---|---|
| n | n (%) | n | n (%) | N | n (%) | n | n (%) | |
| Avascular necrosis | 60 | 4 (6.7) | 44 | 12 (27.3) | 65 | 24 (36.9) | 175 | 47 (26.9) |
| Erlenmeyer flask deformity | 83 | 59 (71.1) | 46 | 37 (80.4) | 71 | 55 (77.5) | 171 | 120 (70.2) |
| Fractures | 43 | 0 (0) | 32 | 3 (9.4) | 37 | 3 (8.1) | 119 | 14 (11.8) |
| Infarction | 54 | 13 (24.1) | 44 | 20 (45.5) | 49 | 20 (40.8) | 164 | 73 (44.5) |
| Lytic lesions | 41 | 9 (22.0) | 30 | 6 (20.0) | 35 | 16 (45.7) | 117 | 41 (35.0) |
| Marrow infiltration | 60 | 49 (81.7) | 54 | 47 (87.0) | 75 | 70 (93.3) | 200 | 188 (94.0) |
| Age at first infusion of imiglucerase |
||||
|---|---|---|---|---|
| ≥5 to <12 years | ≥12 to <20 years | ≥20 to <30 years | ≥30 to ≤50 years | |
| Decreased bone mineral density (lumbar spine DXA Z-scoreb), n (%) | n=43 | n=41 | n=56 | n=171 |
| >−1 | 24 (55.8) | 10 (24.4) | 26 (46.4) | 82 (48.0) |
| ≤−1 to≥−2 | 14 (32.6) | 14 (34.1) | 18 (32.1) | 53 (31.0) |
| <−2 | 5 (11.6) | 17 (41.5) | 12 (21.4) | 36 (21.1) |
| Pediatric growth retardation, n (%) | n=122 | n=69 | n=0 | n=0 |
| Observed | 40 (33) | 22 (33) | 0 (0) | 0 (0) |
| Expectedc | 6 (5) | 3 (5) | 0 (5.0) | 0 (5.0) |
a
“At the time of first infusion” is defined as the data point closest to the first infusion date, no more than −2 years/+6 weeks (inclusive) from first infusion. Patients with no infusion date were excluded from the analysis for each bone assessment.
b
Standard deviations of age and gender-adjusted norms.
c
Pediatric growth retardation is defined as the number of patients who are below the 5th percentile for height based on age and gender of the normal healthy population and is calculated as 0.05× total number of patients [26].