From: Characterization of tracheobronchomalacia in infants with hypophosphatasia
Characteristic | Patients Who Received Asfotase Alfa as Part of the ENB-010-10 Study | Patient Who Received Asfotase Alfa Through Compassionate-Use Program | Patient Who Received Asfotase Alfa as Licensed Medication Post-Authorization | ||
---|---|---|---|---|---|
Patient 1 | Patient 2 | Patient 3 | Patient 4 | Patient 5 | |
Sex | Male | Male | Female | Female | Female |
Ethnicity | White Caucasian | White Caucasian | White Caucasian | Asian-Pakistani | White Caucasian |
Gestational age | Term | Term | 35 weeks, 4 days | 34 weeks | Term |
Birth weight | 2.89 kg | 3.46 kg | 3.06 kg | 1.69 kg | 3.46 kg |
Post-birth respiratory support | CPAP ventilation at birth; intubation and ventilation starting at 4 weeks | Intubation and ventilation | Intubation and ventilation | Intubation and ventilation: surfactant | Intubation and ventilation at birth |
Presence of skeletal manifestations of HPP | Yes | Yes | Yes | Yes | Yes |
Presence of hypotonia | Yes | Yes | Yes | Yes | Yes |
Presence of feeding difficulty | Yes | Yes | Yes | Yes | Yes |