Table 4.

Laboratory and clinical biomarkers proposed for therapeutic monitoring

Therapeutic monitoring biomarkerSCT or GT/EDirect or surrogate biomarkerBiomarker value validation
Laboratory biomarkers    
 Donor chimerism (%) SCT/GT/E Surrogate Strong 
 Hb; reticulocyte (target; time to target) SCT/GT/E Surrogate Strong 
 Increase in HbF (α2γ2) GT/E Surrogate Strong 
 Increase in or HbA (α2β2) SCT/GT/E Surrogate Strong 
Clinical biomarkers    
  Change in TF requirements SCT/GT/E Direct Intermediate* 
  Change in VOC SCT/GT/E Direct Intermediate* 
  Change in all pain levels SCT/GT/E Direct Intermediate* 
  Change in HRQoL SCT/GT/E Direct Intermediate* 
  Change in organ-specific function SCT/GT/E Direct Intermediate* 
Therapeutic monitoring biomarkerSCT or GT/EDirect or surrogate biomarkerBiomarker value validation
Laboratory biomarkers    
 Donor chimerism (%) SCT/GT/E Surrogate Strong 
 Hb; reticulocyte (target; time to target) SCT/GT/E Surrogate Strong 
 Increase in HbF (α2γ2) GT/E Surrogate Strong 
 Increase in or HbA (α2β2) SCT/GT/E Surrogate Strong 
Clinical biomarkers    
  Change in TF requirements SCT/GT/E Direct Intermediate* 
  Change in VOC SCT/GT/E Direct Intermediate* 
  Change in all pain levels SCT/GT/E Direct Intermediate* 
  Change in HRQoL SCT/GT/E Direct Intermediate* 
  Change in organ-specific function SCT/GT/E Direct Intermediate* 

GT/E, gene therapy/editing; SCT, stem cell therapy.

*

Could be strengthened by choosing targets that meet the FDA guidance for rare disease trial end points (https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm613026.html).

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