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Table 1 Patient disposition in the taliglucerase alfa clinical studiesa

From: Taliglucerase alfa: safety and efficacy across 6 clinical studies in adults and children with Gaucher disease

Patient population and study Enrolled Completed Notes
Adult treatment-naive patients
 PB-06-001 [15] 33 29 • 1 patient withdrew consent before first dose
• 3 patients discontinued
 PB-06-003 [18] 26 23 • 3 patients not recruited to PB-06-003
• 3 patients discontinued
 PB-06-007 [21] 19 17 • 4 patients not recruited into PB-06-007
• 2 patients discontinued
Adult treatment-switched patients
 PB-06-002 [16] 26 25 • 1 patient discontinued
 PB-06-003 [19] 19 10 • 6 patients from PB-06-002 not recruited into PB-06-003,
5 of whom continued with compassionate use program
• 1 patient non-compliant
• 4 patients discontinued
• 4 patients treated for 30–33 total months but transitioned to commercial use before study completion
Paediatric treatment-naïve patients
 PB-06-005 [17] 11 11  
 PB-06-006 [20] 10 9 • 1 patient from PB-06-005 not recruited into PB-06-006 and continued with compassionate use program
• 1 patient dropped out during PB-06-006
Paediatric treatment-switched patients
 PB-06-002 [16] 5 5  
 PB-06-006 [20] 5 4 • 2 patients completed 27 months of treatment and continued with compassionate use program
• 2 patients completed 33 months of treatment
• 1 patient lost to follow-up
  1. aFor full patient disposition details, refer to the original studies. For some endpoints, data were not available for all patients; refer to the original studies for reasons