Measures of normal or genetically corrected RBCs proposed to assess for cure in SCD
| End point . | Measures . | Grade 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 point . | Measures . | Grade 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.