Table 1.
Demographics and Baseline Characteristics of Study Patients
| Characteristic | Study Population (N = 190) |
|---|---|
| Sex, n (%) | |
| Female | 130 (68.4) |
| Male | 60 (31.6) |
| Age at diagnosis of HPP, years | |
| N | 159 |
| Median (min, max) | 37.1 (0, 77) |
| ≤ 18 years at diagnosis, n (%) | 32 (16.8) |
| > 18 years at diagnosis, n (%) | 127 (66.8) |
| Missing, n (%) | 31 (16.3) |
| Age at start of asfotase alfa treatment, years | |
| N | 190 |
| Median (min, max) | 45.5 (18.3, 77.9) |
| Time from diagnosis to start of asfotase alfa treatment, years | |
| N | 154 |
| Median (min, max) | 1.4 (0.01, 60.8) |
| White race, n (%) | 153 (80.5) |
| Not Hispanic or Latino, n (%) | 146 (76.8) |
| Number of signs and symptoms per patient | |
| N | 180 |
| Median (min, max) | 5 (1, 14) |
| Number of body systems impacted per patient | |
| N | 180 |
| Median (min, max) | 3 (1, 8) |
| History of signs and symptoms, n (%) | |
| Skeletal | 120 (63.2) |
| Neurologic | 30 (15.8) |
| Constitutional/metabolic | 88 (46.3) |
| Muscular | 71 (37.4) |
| Dental | 129 (67.9) |
| Respiratory | 7 (3.7) |
| Renal | 26 (13.7) |
| Rheumatic | 21 (11.1) |
| Pain | 138 (72.6) |
| HPP onset, n (%) | |
| Pediatric | 173 (91.1) |
| Perinatal/infantile onset | 18 (9.5) |
| Juvenile onset | 117 (61.6) |
| Pediatric onset, specific type unknown | 38 (20.0) |
| Adult-onset HPP | 17 (8.9) |
| Gene classification, n (%) | |
| Heterozygous | 113 (59.5) |
| Compound heterozygous | 55 (28.9) |
| Homozygous | 4 (2.1) |
| Unknown/missing | 18 (9.5) |
HPP hypophosphatasia