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 |