Protocols

A. Creation of a large research cohort, including new-born infants and adults

PI: R. Cooper et al.

Team Investigator by site:

Specific Aim:

  • Enroll and follow for 10 years 2,500 infants from birth, and 2,500 persons over the age of 14...Click for more

 

B. Genetic modifiers of clinical outcomes

PI: N. Hanchard, A. Wonkam, G. Lettre

Team Investigator by site:

Aims:

  • Genetics of HbF

  • Genetics of hemolysis

  • Genetics of kidney function

  • Population genetics...Click for more

 

C. Hemolysis and the pathophysiology of vascular complications  

PI: S. Ofori-Acquah

Team Investigator by site:

Aims:

  • Determine the spectrum of clinical outcomes and organ dysfunction associated with hemolysis in a longitudinal cohort of SCD patients.

  • Determine the activation of hemolysis and oxidative stress protective pathways in neonates and children with SCD and the deterioration of this protection with disease progression and aging longitudinally

  • Determine the genetic factors that attenuate and exacerbate hemolysis, and modulate the response to hemolysis, in SCD patients using a whole genome approach, and test for their association with clinical outcomes and organ dysfunction.

  • Determine the expression of putative hemolysis-modifying genes and transcripts...Click for more

 

D. Safety and efficacy of hydroxyurea (HU)

PI: B. Tayo, V. Gordeuk

Team Investigator by site:

Specific Aims:  

  • While a large-scale trial of HU in Africa is clearly a major priority, it is not clear that the necessary resources are available in clinical sites at this time

 

E. Influence of diet / obesity

PI: S. Saraf

Team Investigator by site:

Specific Aims: To be determined...Click for more

 

F. Psycholocial factors and HRQL of SCD patients

PI: A. Wonkam

Team Investigator by site:

Specific Aims: To be determined... Click for more