Table 1.
Baseline Characteristics of the Patients.
| Characteristic | All Patients (N = 717) | Midostaurin Group (N = 360) | Placebo Group (N = 357) | P Value* |
|---|---|---|---|---|
| Age at trial entry — yr | 0.22 | |||
| Median | 47.9 | 47.1 | 48.6 | |
| Range | 18.0–60.9 | 19.0–59.8 | 18.0–60.9 | |
| Female sex — no. (%) | 398 (55.5) | 186 (51.7) | 212 (59.4) | 0.04 |
| Race — no./total no. (%)† | 0.74 | |||
| White | 275/309 (89.0) | 147/165 (89.1) | 128/144 (88.9) | |
| Other | 34/309 (11.0) | 18/165 (10.9) | 16/144 (11.1) | |
| Subtype of FLT3 mutation — no. (%)‡ | 1.00 | |||
| TKD | 162 (22.6) | 81 (22.5) | 81 (22.7) | |
| ITD with low allelic ratio | 341 (47.6) | 171 (47.5) | 170 (47.6) | |
| ITD with high allelic ratio | 214 (29.8) | 108 (30.0) | 106 (29.7) | |
| Modified European LeukemiaNet classification — no./total no. (%)§ | 0.15 | |||
| Favorable | 29/547 (5.3) | 16/269 (5.9) | 13/278 (4.7) | |
| Normal | 375/547 (68.6) | 172/269 (63.9) | 203/278 (73.0) | |
| Intermediate II | 104/547 (19.0) | 59/269 (21.9) | 45/278 (16.2) | |
| Adverse | 39/547 (7.1) | 22/269 (8.2) | 17/278 (6.1) | |
| White-cell count per μl¶ | 0.72 | |||
| Median | 34,900 | 35,600 | 33,000 | |
| Range | 600–421,800 | 600–421,800 | 800–329,800 | |
| Platelet count per μl‖ | 0.58 | |||
| Median | 50,000 | 50,000 | 50,000 | |
| Range | 2000–461,000 | 2000–461,000 | 8000–444,000 | |
| Absolute neutrophil count per mm3** | 0.65 | |||
| Median | 2.2 | 2.2 | 2.3 | |
| Range | 0–55.9 | 0–55.9 | 0–55.9 |
All P values are two-sided. P values for continuous variables were calculated with the use of Kruskal–Wallis tests, and P values for categorical variables were calculated with the use of chi-square tests.
Race was reported by the patients. Race was not reported for European patients (195 in the midostaurin group, and 213 in the placebo group); the P value excludes these patients.
The subtypes of the FLT3 mutation are point mutation in the tyrosine kinase domain (TKD) or internal tandem duplication (ITD) mutation with either a high ratio (>0.7) or a low ratio (0.05 to 0.7) of mutant to wild-type alleles.
Cytogenetic data according to a modified European LeukemiaNet classification were available for 547 patients (269 in the midostaurin group, and 278 in the placebo group). Data on mutations in the nucleophosmin gene (NPM1) or the CCAAT/enhancer binding protein alpha gene (CEBPα) are not included. A classification of favorable indicated the presence of t(8;21) and inv(16) or t(16;16), normal the presence of a normal karyotype, intermediate II the presence of cytogenetic abnormalities that were not classified as favorable or adverse, and adverse the presence of adverse-risk cytogenetic abnormalities.
Data were available for 707 patients (355 in the midostaurin group, and 352 in the placebo group).
Data were available for 702 patients (351 in the midostaurin group, and 351 in the placebo group).
Data were available for 673 patients (339 in the midostaurin group, and 334 in the placebo group).