Sorafenib

Sorafenib was shown to improve EFS in the COG AAML1031 (NCT01371981) study of pediatric patients with de novo AML and high-allelic ratio (HAR) (i.e., >0.4) FLT3 ITD variants. Seventy-two patients who received sorafenib were evaluable for response. The patients in this study were compared with patients with AML and HAR FLT3 ITD (N = 76) in the AAML1031 and the COG AAML0531 trials who did not receive sorafenib.[31]

Sorafenib was shown to improve EFS in the COG AAML1031 (NCT01371981) study of pediatric patients with de novo AML and high-allelic ratio (HAR) (i.e., >0.4) FLT3 ITD variants. Seventy-two patients who received sorafenib were evaluable for response. The patients in this study were compared with patients with AML and HAR FLT3 ITD (N = 76) in the AAML1031 and the COG AAML0531 trials who did not receive sorafenib.[31]

The morphological CR rate after induction cycle I was significantly improved for patients who received sorafenib (75% vs. 57%; P = .028).

Sorafenib was shown to improve EFS in the COG AAML1031 (NCT01371981) study of pediatric patients with de novo AML and high-allelic ratio (HAR) (i.e., >0.4) FLT3 ITD variants. Seventy-two patients who received sorafenib were evaluable for response. The patients in this study were compared with patients with AML and HAR FLT3 ITD (N = 76) in the AAML1031 and the COG AAML0531 trials who did not receive sorafenib.[31]

However, there was similar prevalence of MRD in both groups of patients (48% vs. 45%; P = .724).

Sorafenib was shown to improve EFS in the COG AAML1031 (NCT01371981) study of pediatric patients with de novo AML and high-allelic ratio (HAR) (i.e., >0.4) FLT3 ITD variants. Seventy-two patients who received sorafenib were evaluable for response. The patients in this study were compared with patients with AML and HAR FLT3 ITD (N = 76) in the AAML1031 and the COG AAML0531 trials who did not receive sorafenib.[31]

Patients who received sorafenib had significantly improved 3-year EFS rates (55.9% vs. 31.9%, P = .001), DFS rates (70.9% vs. 49.4%, P = .032), and relapses after CR (17.6% vs. 44.1%, P = .012).

Sorafenib was shown to improve EFS in the COG AAML1031 (NCT01371981) study of pediatric patients with de novo AML and high-allelic ratio (HAR) (i.e., >0.4) FLT3 ITD variants. Seventy-two patients who received sorafenib were evaluable for response. The patients in this study were compared with patients with AML and HAR FLT3 ITD (N = 76) in the AAML1031 and the COG AAML0531 trials who did not receive sorafenib.[31]

The OS rate did not improve after treatment with sorafenib (65.8% vs. 55.3%, P = .244).

Sorafenib was shown to improve EFS in the COG AAML1031 (NCT01371981) study of pediatric patients with de novo AML and high-allelic ratio (HAR) (i.e., >0.4) FLT3 ITD variants. Seventy-two patients who received sorafenib were evaluable for response. The patients in this study were compared with patients with AML and HAR FLT3 ITD (N = 76) in the AAML1031 and the COG AAML0531 trials who did not receive sorafenib.[31]

Although similar trends were seen in patients with AML harboring both HAR FLT3 ITD variants and NPM1 variants, they did not approach a significant level of benefit.

Sorafenib was shown to improve EFS in the COG AAML1031 (NCT01371981) study of pediatric patients with de novo AML and high-allelic ratio (HAR) (i.e., >0.4) FLT3 ITD variants. Seventy-two patients who received sorafenib were evaluable for response. The patients in this study were compared with patients with AML and HAR FLT3 ITD (N = 76) in the AAML1031 and the COG AAML0531 trials who did not receive sorafenib.[31]

Statistics showed that a benefit of sorafenib treatment remained in multivariable analyses controlling for both NPM1 status and HSCT, a time-varying covariate.