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Table 3 Overview of treatment and patient outcomes in infants included in the case series

From: Characterization of tracheobronchomalacia in infants with hypophosphatasia

Treatment 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
Asfotase alfa dosage, mg/kg/week 1 month: 6
3 months: 9
Current: 7.5
5 weeks: 6
6 months: 7.8
9 months: 7.5
Current: 9
7.5 weeks: 6
Current: 12
1 month: 6
3.5 months
(post-cardiac arrest): 15
Current: 7.5
Birth: 6
2 months: 9
3 months: 15
Current: 7.5
Surgical treatments 5 weeks: tracheostomy 6 weeks: tracheostomy
4 months: gastrostomy
7 weeks: tracheostomy
3 months: gastrostomy
6 weeks: tracheostomy
1 year: gastrostomy
3 days: tracheostomy and central venous access
16 months: left ureteroscopy with laser lithotripsy for renal stone obstructing left ureter causing left hydronephrosis
19 months: closure of tracheostomy
2 years: surgical correction of craniosynostosis
Age at TBM diagnosis 2 months 5 months 5 months (suspected at 8 weeks) 5 months 3 months
Current age 5 years, 8 months 5 years, 9 months 5 years, 5 months 7 years, 2 months 3 years, 4 months
Long-term follow-up and current status 15 months: complete clinical resolution and self-ventilating in room air 17 months: normal-appearing lower airways, but profound TBM when coughing or bearing down; BiPAP: PIP 24 cm H2O, PEEP 10 cm H2O
27 months: remains on ventilator; respiratory issues (viral infections) requiring hospital readmission; BiPAP: PIP 22 cm H2O, PEEP 8 cm H2O
5 years, 9 months: off ventilator for 4 h three times daily; plan to completely wean from daytime ventilator by 6 years of age
18 months: significant TBM identified (severity difficult to assess owing to well-positioned custom tracheostomy tube in distal trachea); BiPAP: PIP 26 cm H2O, PEEP 12 cm H2O
23 months: tracheostomy in situ with ventilator support; BiPAP: PIP 25 cm H2O, PEEP 12 cm H2O
5 years, 5 months: off ventilator for 16–18 h daily; plan to rapidly completely discontinue ventilator support in the coming months
2 years: all ventilator support removed 9 months: all ventilator support removed
  1. BiPAP Bilevel positive airway pressure, PEEP Positive end-expiratory pressure, PIP Positive inspiratory pressure, TBM Tracheobronchomalacia