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Table 1 Baseline demographic and clinical characteristics at birth in infants included in the case series

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

  1. CPAP Continuous positive airway pressure, HPP Hypophosphatasia