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) |