Peak exercise oxygen uptake, ventilatory threshold, and erythropoietin with voxelotor treatment
| Voxelotor/placebo . | Placebo . | 700 mg/d . | 900 mg/d . | Voxelotor-treated pooled . | P* for pooled voxelotor vs placebo . |
|---|---|---|---|---|---|
| N | 4 | 6 | 6 | 12 | |
| Peak exercise oxygen uptake VO2max, mL/min/kg | |||||
| Median baseline (range) | 23.3 (15.7-23.8) | 16.7 (8.2-19.6) | 21.2 (17.1-23.4) | 18.9 (8.2-23.4) | |
| Median change at day 91 (range) | −2.4 (−7.5 to 0.3) | 1.0 (−6.6 to 2.1) | −1.9 (−5.0 to −0.4) | −0.4 (−6.6 to 2.1) | NS |
| Ventilatory threshold oxygen uptake, mL/min/kg | |||||
| Median baseline (range) | 11.9 (11.7-12.7) | 9.4 (6.4-12.4) | 12.8 (11.5-16.3) | 12.0 (6.4-16.3) | |
| Median change at day 91 (range) | −1.4 (−5.5 to 2.3) | 1.6 (−0.1 to 4.7) | −1.4 (−1.8 to 1.4) | 0.6 (−1.8 to 4.7) | NS |
| Erythropoietin | |||||
| Median baseline mU/mL (IQR 25%:75%) | 112.2 (46.5, 183.0) | 91.2 (77.6, 125.0) | 113.5 (42.4, 150.0) | 104.4 (68.6, 137.5) | |
| % Median change to day 90 (IQR 25%:75%) | −43.5 (−79.3, 19.7) | 0.4 (−32.9, 27.6) | −7.2 (−11.5, −4.7) | −5.2 (−22.2, 14.8) | NS |
| Vital signs: heart rate at rest | |||||
| Median baseline, beats per minute (range) | 83 (75-93) | 81 (65-101) | 87 (71-99) | 81 (65-101) | |
| Median change to day 91 (range) | 17 (17) | 11 (6-38) | 3 (0-5) | 6 (0-38) | NC |
| Vital signs: heart rate at peak exercise | |||||
| Median baseline, beats per minute (range) | 162 (160-162) | 150 (116-173) | 171 (146-184) | 164 (116-184) | |
| Median change to day 91 (range) | −12 (−28 to 4) | −2 (−20 to 16) | 1 (−7 to 8) | −2 (−20 to 16) | NC |
| Voxelotor/placebo . | Placebo . | 700 mg/d . | 900 mg/d . | Voxelotor-treated pooled . | P* for pooled voxelotor vs placebo . |
|---|---|---|---|---|---|
| N | 4 | 6 | 6 | 12 | |
| Peak exercise oxygen uptake VO2max, mL/min/kg | |||||
| Median baseline (range) | 23.3 (15.7-23.8) | 16.7 (8.2-19.6) | 21.2 (17.1-23.4) | 18.9 (8.2-23.4) | |
| Median change at day 91 (range) | −2.4 (−7.5 to 0.3) | 1.0 (−6.6 to 2.1) | −1.9 (−5.0 to −0.4) | −0.4 (−6.6 to 2.1) | NS |
| Ventilatory threshold oxygen uptake, mL/min/kg | |||||
| Median baseline (range) | 11.9 (11.7-12.7) | 9.4 (6.4-12.4) | 12.8 (11.5-16.3) | 12.0 (6.4-16.3) | |
| Median change at day 91 (range) | −1.4 (−5.5 to 2.3) | 1.6 (−0.1 to 4.7) | −1.4 (−1.8 to 1.4) | 0.6 (−1.8 to 4.7) | NS |
| Erythropoietin | |||||
| Median baseline mU/mL (IQR 25%:75%) | 112.2 (46.5, 183.0) | 91.2 (77.6, 125.0) | 113.5 (42.4, 150.0) | 104.4 (68.6, 137.5) | |
| % Median change to day 90 (IQR 25%:75%) | −43.5 (−79.3, 19.7) | 0.4 (−32.9, 27.6) | −7.2 (−11.5, −4.7) | −5.2 (−22.2, 14.8) | NS |
| Vital signs: heart rate at rest | |||||
| Median baseline, beats per minute (range) | 83 (75-93) | 81 (65-101) | 87 (71-99) | 81 (65-101) | |
| Median change to day 91 (range) | 17 (17) | 11 (6-38) | 3 (0-5) | 6 (0-38) | NC |
| Vital signs: heart rate at peak exercise | |||||
| Median baseline, beats per minute (range) | 162 (160-162) | 150 (116-173) | 171 (146-184) | 164 (116-184) | |
| Median change to day 91 (range) | −12 (−28 to 4) | −2 (−20 to 16) | 1 (−7 to 8) | −2 (−20 to 16) | NC |
CPET (peak exercise and ventilatory threshold) not available in all patients. CPET data available for n = 3 (placebo), n = 5 (900 mg), n = 11 (voxelotor-treated pooled).
IQR, interquartile range; NC, not calculated; NS, not significant.
Two-sample Student t test.