Means and proportions of selected treatment variables, defined by mean maximally tolerated dose, and percentage of fetal hemoglobin, divided into quartiles
| . | %HbF at MTD, quartiles . | P . | |||
|---|---|---|---|---|---|
| First . | Second . | Third . | Fourth . | ||
| No. of patients | 14 | 13 | 13 | 13 | — |
| Mean age, y | 10.2 | 7.9 | 9.1 | 10.8 | .12 |
| Sex, % male | 57.1 | 61.5 | 69.2 | 53.9 | .87 |
| Mean MTD, mg/kg/d | 21.4 | 28.8 | 27.9 | 26.9 | .001 |
| Toxicities, no more than 3, % patients | 35.7 | 61.5 | 61.5 | 46.2 | .47 |
| Fetal hemoglobin, % | |||||
| MTD value | 8.4 | 15.2 | 19.6 | 25.7 | * |
| Change from baseline | +4.2 | +9.4 | +11.9 | +16.4 | <.0001 |
| Hemoglobin, g/dL | |||||
| MTD value | 7.7 | 8.5 | 9.4 | 10.2 | <.0001 |
| Change from baseline | +0.5 | +0.9 | +1.6 | +2.0 | <.0001 |
| Mean cellular volume, fL | |||||
| MTD value | 96 | 102 | 104 | 107 | .02 |
| Change from baseline | +12 | +15 | +16 | +21 | .01 |
| Reticulocytes, ×109/L | |||||
| MTD value | 245 | 201 | 195 | 135 | .0004 |
| Change from baseline | −114 | −172 | −125 | −272 | .01 |
| White blood cells, ×109/L | |||||
| MTD value | 11.1 | 9.4 | 8.7 | 7.4 | .0005 |
| Change from baseline | −2.7 | −3.9 | −5.3 | −7.4 | .006 |
| Neutrophils, ×109/L | |||||
| MTD value | 5.2 | 4.6 | 4.5 | 3.8 | .09 |
| Change from baseline | −1.8 | −2.0 | −3.3 | −3.9 | .22 |
| Platelets, ×109/L | |||||
| MTD value | 407 | 362 | 355 | 330 | .47 |
| Change | −41 | −102 | −154 | −121 | .21 |
| . | %HbF at MTD, quartiles . | P . | |||
|---|---|---|---|---|---|
| First . | Second . | Third . | Fourth . | ||
| No. of patients | 14 | 13 | 13 | 13 | — |
| Mean age, y | 10.2 | 7.9 | 9.1 | 10.8 | .12 |
| Sex, % male | 57.1 | 61.5 | 69.2 | 53.9 | .87 |
| Mean MTD, mg/kg/d | 21.4 | 28.8 | 27.9 | 26.9 | .001 |
| Toxicities, no more than 3, % patients | 35.7 | 61.5 | 61.5 | 46.2 | .47 |
| Fetal hemoglobin, % | |||||
| MTD value | 8.4 | 15.2 | 19.6 | 25.7 | * |
| Change from baseline | +4.2 | +9.4 | +11.9 | +16.4 | <.0001 |
| Hemoglobin, g/dL | |||||
| MTD value | 7.7 | 8.5 | 9.4 | 10.2 | <.0001 |
| Change from baseline | +0.5 | +0.9 | +1.6 | +2.0 | <.0001 |
| Mean cellular volume, fL | |||||
| MTD value | 96 | 102 | 104 | 107 | .02 |
| Change from baseline | +12 | +15 | +16 | +21 | .01 |
| Reticulocytes, ×109/L | |||||
| MTD value | 245 | 201 | 195 | 135 | .0004 |
| Change from baseline | −114 | −172 | −125 | −272 | .01 |
| White blood cells, ×109/L | |||||
| MTD value | 11.1 | 9.4 | 8.7 | 7.4 | .0005 |
| Change from baseline | −2.7 | −3.9 | −5.3 | −7.4 | .006 |
| Neutrophils, ×109/L | |||||
| MTD value | 5.2 | 4.6 | 4.5 | 3.8 | .09 |
| Change from baseline | −1.8 | −2.0 | −3.3 | −3.9 | .22 |
| Platelets, ×109/L | |||||
| MTD value | 407 | 362 | 355 | 330 | .47 |
| Change | −41 | −102 | −154 | −121 | .21 |
First quartile includes the lowest percentage of fetal hemoglobin (%HbF) values and the fourth quartile includes the highest %HbF values.
Each P value derives from an analysis of variance test of the null hypothesis that the 4 group means are equal versus the alternative hypothesis that the means are not all equal.
MTD indicates maximally tolerated dose.
Test result is artificial because the groups are based on quartiles of mean HbF at MTD.