ASCO 2015 Highlights

An updated safety and efficacy analysis of a phase 1b study demonstrated a promising benefit–risk profile with the combination of the BCL-2 inhibitor venetoclax with either decitabine or azacitidine in elderly, treatment-naïve patients with acute myeloid leukemia (AML) who are not candidates for intensive standard induction therapy.

The results of a post-hoc analysis of the RATIFY trial suggest that midostaurin decreases the cumulative incidence of relapse (CIR) in patients with FLT3 mutation–positive, newly diagnosed acute myeloid leukemia (AML), with no negative impact on survival.

This analysis showed that ivosidenib-treated patients with IDH1-mutated (mIDH1), relapsed/refractory, and untreated acute myeloid leukemia (AML) whose best response is a complete remission (CR) or CR with partial hematologic recovery achieve mIDH1 clearance ≤0.04%.

In this subanalysis of the AG221-C-001 phase 1 study, enasidenib monotherapy was found to be well-tolerated, and yielded hematologic responses in previously untreated older patients with IDH2-mutated (mIDH2) acute myeloid leukemia (AML) who are not fit to receive standard cytotoxic chemotherapy.

The initial results of a phase 1 study suggest that combination therapy with ivosidenib or enasidenib plus standard induction chemotherapy is well-tolerated and effective in newly diagnosed patients with IDH-mutated (mIDH), relapsed/refractory acute myeloid leukemia (AML).

In this analysis, the hedgehog pathway inhibitor sonidegib plus azacitidine combination therapy showed encouraging antileukemia activity in terms of high rate of disease stabilization, particularly in the relapsed/refractory acute myeloid leukemia patient population.

The results of the Radius-X expanded treatment protocol showed a safety profile that was consistent with that previously described in patients with FLT3 mutation–positive, newly diagnosed acute myeloid leukemia (AML).

Ivosidenib monotherapy is well-tolerated and produced durable remissions in high-risk patients with relapsed/refractory acute myeloid leukemia (AML).

Interim results of an ongoing phase 1 study showed that the addition of the selective FLT3 inhibitor quizartinib to azacitidine or low-dose cytarabine produced high overall response rates in patients with FLT3-ITD–mutated myeloid leukemias.

The preliminary results of a multicenter phase 1b dose-escalation study evaluated the safety, tolerability, and efficacy of 2 novel venetoclax-based combination therapies, in combination with the MEK inhibitor cobimetinib or the MDM2 inhibitor idasanutlin, in relapsed/refractory (R/R) acute myeloid leukemia (AML).