Change in hemolysis measures from baseline to 90 days or more
| Change from baseline to end of treatment . | Dosing duration ≥90 d . | P* for pooled voxelotor vs placebo . | |||
|---|---|---|---|---|---|
| Voxelotor/placebo, mg/d | 700† | 900‡ | 700/900 | Placebo | |
| N | 6 | 7§ | 13 | 4 | |
| Hb, median (25th, 75th percentile), g/dL | 1.1 (0.6, 1.3) | 0.8 (0.5, 1.3) | 1.0 (0.6, 1.3) | −0.1 (−0.2, 0.1) | <.05 |
| Unconjugated bilirubin, median (25th, 75th percentile), % change | −37.2 (−43.4, −23.7) | −42.9 (−58.4, −30.5) | −39.7 (−49.9, −28.8) | 14.8 (1.8, 18.5) | <.05 |
| Percentage of reticulocytes, median (25th, 75th percentile), % change | −21.0 (−32.9, −18.1) | −18.9 (−35.4, −6.2) | −18.9 (−32.9, −15.0) | 8.9 (2.5, 25.5) | <.05 |
| LDH, median (25th, 75th percentile), % change | 0.8 (−14.7, 1.1) | −47.7 (−63.4, −12.5) | −12.9 (−47.7, 0.9) | 0.5 (−0.7, 7.2) | NS |
| Dense RBC, median (25th, 75th percentile), % change | −35.5|| (−63.1, 18.1) | −21.0¶ (−60.5, 11.3) | −31.4# (−62.5, 11.3) | 3.8** (−21.3, 4.3) | NS |
| Sickled red cell, median (25th, 75th percentile), % change | −72.6 (−79.0, −60.6) | −79.2¶ (−91.3, −57.7) | −74.0†† (−88.6, −57.0) | 6.9 (3.9, 10.3) | <.05 |
| Change from baseline to end of treatment . | Dosing duration ≥90 d . | P* for pooled voxelotor vs placebo . | |||
|---|---|---|---|---|---|
| Voxelotor/placebo, mg/d | 700† | 900‡ | 700/900 | Placebo | |
| N | 6 | 7§ | 13 | 4 | |
| Hb, median (25th, 75th percentile), g/dL | 1.1 (0.6, 1.3) | 0.8 (0.5, 1.3) | 1.0 (0.6, 1.3) | −0.1 (−0.2, 0.1) | <.05 |
| Unconjugated bilirubin, median (25th, 75th percentile), % change | −37.2 (−43.4, −23.7) | −42.9 (−58.4, −30.5) | −39.7 (−49.9, −28.8) | 14.8 (1.8, 18.5) | <.05 |
| Percentage of reticulocytes, median (25th, 75th percentile), % change | −21.0 (−32.9, −18.1) | −18.9 (−35.4, −6.2) | −18.9 (−32.9, −15.0) | 8.9 (2.5, 25.5) | <.05 |
| LDH, median (25th, 75th percentile), % change | 0.8 (−14.7, 1.1) | −47.7 (−63.4, −12.5) | −12.9 (−47.7, 0.9) | 0.5 (−0.7, 7.2) | NS |
| Dense RBC, median (25th, 75th percentile), % change | −35.5|| (−63.1, 18.1) | −21.0¶ (−60.5, 11.3) | −31.4# (−62.5, 11.3) | 3.8** (−21.3, 4.3) | NS |
| Sickled red cell, median (25th, 75th percentile), % change | −72.6 (−79.0, −60.6) | −79.2¶ (−91.3, −57.7) | −74.0†† (−88.6, −57.0) | 6.9 (3.9, 10.3) | <.05 |
NS, not significant.
Wilcoxon rank-sum test.
Ninety days of dosing.
Ninety days to 6 months of dosing (2 patients had 90 days of dosing, 1 patient had 118 days of dosing, and 4 patients had 6 months of dosing).
Includes 1 patient who received placebo in GBT440-001 and voxelotor 900 mg for 6 months in GBT440-024.
n = 4.
n = 6.
n = 10.
n = 3.
n = 12.