Laboratory evaluations of the intention-to-treat population at final assessment
| Laboratory parameter . | Final assessment . | Change from baseline . | ||||
|---|---|---|---|---|---|---|
| Transfusions and chelation group, N = 66 . | Hydroxyurea and phlebotomy group, N = 67 . | P* . | Transfusions and chelation group, N = 66 . | Hydroxyurea and phlebotomy group, N = 67 . | P† . | |
| Hemoglobin, g/dL | 9.0 (8.7-9.6) | 9.0 (8.4-9.6) | .934 | 0.0 (−0.5-0.6) | 0.0 (−0.7-0.7) | .898 |
| MCV, fL | 86.1 (83.3-88.5) | 103.3 (93.5-114.9) | < .001 | 0.1 (−2.0-2.5) | 19.5 (7.5-28.5) | < .001 |
| HbA, % | 63.6 (56.4-69.1) | 1.7 (0.0-31.4) | < .001 | 0.0 (−12.7-6.7) | −50.9 (-66.8-−33.7) | < .001 |
| HbF, % | 1.3 (0.8-2.7) | 19.5 (10.0-24.3) | < .001 | −0.2 (−0.8-0.4) | 17.9 (9.2-22.9) | < .001 |
| HbS, % | 32.3 (25.0-38.3) | 64.1 (52.6-76.4) | < .001 | 0.3 (−7.5-12.3) | 35.0 (21.7-46.2) | < .001 |
| ARC, ×109/L | 312.4 (226.3-419.6) | 176.7 (115.1-303.4) | < .001 | −11.8 (−88.2-93.2) | −149.1 (−231.0-−19.0) | < .001 |
| WBC, ×109/L | 13.2 (10.4-17.0) | 7.2 (5.6-11.6) | < .001 | 0.2 (−2.0-2.3) | −5.4 (−8.1-−2.2) | < .001 |
| ANC, ×109/L | 7.8 (6.5-10.0) | 3.8 (2.6-5.5) | < .001 | 0.8 (−1.3-2.4) | −3.3 (−5.1-−1.4) | < .001 |
| Platelets, ×109/L | 368.0 (315.0-437.0) | 315.0 (242.0-435.0) | .0183 | −28.0 (−70.0-18.0) | −83.0 (−171.0-−8.0) | .0022 |
| Total bilirubin, mg/dL | 3.3 (2.2-4.2) | 1.6 (1.1-2.5) | < .001 | 0.4 (−0.3-1.2) | −1.1 (−1.9-−0.6) | < .001 |
| LIC, mg Fe/g dw liver‡ | 17.3 (8.8-30.7) | 17.2 (10.0-30.6) | .7920 | −2.2 (−5.5-4.9) | −1.2 (−2.8-7.2) | .4888 |
| Serum ferritin, ng/mL | 4064.0 (2330.0-7126.0) | 1994.0 (998.0-3475.0) | < .001 | 1159.5 (−662.0-2724.0) | −966.0 (−1629.0-49.0) | < .001 |
| LDH, U/L | 434.0 (350.0-533.0) | 311.0 (274.0-434.0) | < .001 | −8.5 (−74.0-74.0) | −67.0 (−143.0-7.0) | .0015 |
| Laboratory parameter . | Final assessment . | Change from baseline . | ||||
|---|---|---|---|---|---|---|
| Transfusions and chelation group, N = 66 . | Hydroxyurea and phlebotomy group, N = 67 . | P* . | Transfusions and chelation group, N = 66 . | Hydroxyurea and phlebotomy group, N = 67 . | P† . | |
| Hemoglobin, g/dL | 9.0 (8.7-9.6) | 9.0 (8.4-9.6) | .934 | 0.0 (−0.5-0.6) | 0.0 (−0.7-0.7) | .898 |
| MCV, fL | 86.1 (83.3-88.5) | 103.3 (93.5-114.9) | < .001 | 0.1 (−2.0-2.5) | 19.5 (7.5-28.5) | < .001 |
| HbA, % | 63.6 (56.4-69.1) | 1.7 (0.0-31.4) | < .001 | 0.0 (−12.7-6.7) | −50.9 (-66.8-−33.7) | < .001 |
| HbF, % | 1.3 (0.8-2.7) | 19.5 (10.0-24.3) | < .001 | −0.2 (−0.8-0.4) | 17.9 (9.2-22.9) | < .001 |
| HbS, % | 32.3 (25.0-38.3) | 64.1 (52.6-76.4) | < .001 | 0.3 (−7.5-12.3) | 35.0 (21.7-46.2) | < .001 |
| ARC, ×109/L | 312.4 (226.3-419.6) | 176.7 (115.1-303.4) | < .001 | −11.8 (−88.2-93.2) | −149.1 (−231.0-−19.0) | < .001 |
| WBC, ×109/L | 13.2 (10.4-17.0) | 7.2 (5.6-11.6) | < .001 | 0.2 (−2.0-2.3) | −5.4 (−8.1-−2.2) | < .001 |
| ANC, ×109/L | 7.8 (6.5-10.0) | 3.8 (2.6-5.5) | < .001 | 0.8 (−1.3-2.4) | −3.3 (−5.1-−1.4) | < .001 |
| Platelets, ×109/L | 368.0 (315.0-437.0) | 315.0 (242.0-435.0) | .0183 | −28.0 (−70.0-18.0) | −83.0 (−171.0-−8.0) | .0022 |
| Total bilirubin, mg/dL | 3.3 (2.2-4.2) | 1.6 (1.1-2.5) | < .001 | 0.4 (−0.3-1.2) | −1.1 (−1.9-−0.6) | < .001 |
| LIC, mg Fe/g dw liver‡ | 17.3 (8.8-30.7) | 17.2 (10.0-30.6) | .7920 | −2.2 (−5.5-4.9) | −1.2 (−2.8-7.2) | .4888 |
| Serum ferritin, ng/mL | 4064.0 (2330.0-7126.0) | 1994.0 (998.0-3475.0) | < .001 | 1159.5 (−662.0-2724.0) | −966.0 (−1629.0-49.0) | < .001 |
| LDH, U/L | 434.0 (350.0-533.0) | 311.0 (274.0-434.0) | < .001 | −8.5 (−74.0-74.0) | −67.0 (−143.0-7.0) | .0015 |
Laboratory evaluations of the intention-to-treat population upon final assessment—either at study exit (N = 50), at the time of study closure (N = 62), or upon early termination for other reasons (N = 21). Median results are provided (interquartile range) unless indicated. Treatment group differences for laboratory parameters were assessed via Wilcoxon rank sum.
See Table 1 for abbreviation definitions.
P value based on final assessment.
P value based on change from baseline to final assessment.
Values for LIC represent 25 subjects in the standard treatment arm and 31 on the alternative treatment arm.