Table 3.

Measures of normal or genetically corrected RBCs proposed to assess for cure in SCD

End pointMeasuresGrade of cure
Anemia and hemolysis Hb; reticulocyte counts; normal Epo, sTfR, LDH, haptoglobin, hemopexin; cell free Hb; RBC microparticles 1: All parameters normal 
2: Compensated anemia (Hb near normal, high reticulocyte counts, sTFR, Epo) 
3: Improved anemia with ongoing hemolysis (high reticulocyte count, bilirubin, LDH) 
4: Mildly improved Hb and persistent intra- and extravascular hemolysis 
5: No improvement 
RBCs, erythropoiesis, and genetically modified RBCs RBC count; erythropoiesis: sTFR, Epo; Hb electrophoresis/HPLC, for HbS, anti-sickling Hb%; cellular distribution of anti-sickling Hb (eg, % F cells); research: deformability, osmoscan 1: Normal 
2: 75% improvement 
3: 50% improvement 
4: 25% improvement 
5: No improvement 
End pointMeasuresGrade of cure
Anemia and hemolysis Hb; reticulocyte counts; normal Epo, sTfR, LDH, haptoglobin, hemopexin; cell free Hb; RBC microparticles 1: All parameters normal 
2: Compensated anemia (Hb near normal, high reticulocyte counts, sTFR, Epo) 
3: Improved anemia with ongoing hemolysis (high reticulocyte count, bilirubin, LDH) 
4: Mildly improved Hb and persistent intra- and extravascular hemolysis 
5: No improvement 
RBCs, erythropoiesis, and genetically modified RBCs RBC count; erythropoiesis: sTFR, Epo; Hb electrophoresis/HPLC, for HbS, anti-sickling Hb%; cellular distribution of anti-sickling Hb (eg, % F cells); research: deformability, osmoscan 1: Normal 
2: 75% improvement 
3: 50% improvement 
4: 25% improvement 
5: No improvement 

Epo, erythropoietin; HPLC, high-performance liquid chromatography; LDH, lactate dehydrogenase; sTFR, soluble transferrin receptor.

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