Originally published by 2 Minute Medicine® (view original article). Reused on AccessMedicine with permission.

1. Overall survival in the quizartinib group was 31.9 months compared to 15.1 months in the placebo group.

2. Proportion of adverse events in both groups was comparable between groups.

Evidence Rating Level: 1 (Excellent)

Study Rundown:

Acute myeloid leukemia (AML) is an aggressive form of blood cancer with poor clinical outcomes. In patients with AML who have FLT3 internal tandem duplication (ITD) mutations, quizartinib, a potent FLT3 inhibitor has shown effectiveness. However, the extent to which it improves survival is unknown. This randomized controlled trial aimed to compare the safety and efficacy of quizartinib on overall survival in patients with AML. The primary outcome was median overall survival while key secondary outcomes included proportion of patients with grade 3 or 4 adverse events. According to study results, quizartinib resulted in substantially greater medial survival from randomization compared to placebo without increasing the occurrence of serious adverse events. A major strength of this study was that it assessed the effect of chemotherapy with or without allogenic cell transplantation, thus increasing its validity.

Relevant Reading: Azacitidine and Venetoclax in Previously Untreated Acute Myeloid Leukemia

In-depth [randomized-controlled trial]:

Between Sept 27, 2016, and Aug 14, 2019, 3468 patients were screened for eligibility across 193 hospitals in 26 countries. Included were patients ≥ 18 years with a diagnosis of AML, FLT3-ITD mutation, and those eligible for standard induction chemotherapy. Altogether, 539 patients (268 in quizartinib group and 271 in placebo group) were included in the final analysis. The primary outcome of median overall survival was greater in the quizartinib group (31.9 months) versus placebo (15.1 months; hazard ratio [HR] 0.78, 95% confidence interval [CI] 0.62-0.98, p=0.032). The proportion of adverse events was similar with 100% and 99% of patients having ≥ 1 adverse event in the quizartinib and placebo groups, respectively. Similarly, 92% of patients in the quizartinib group and 90% in the placebo group experienced a grade 3 or higher adverse event, the most common being febrile neutropenia and hypokalemia. Overall, findings from this study suggest that quizartinib chemotherapy improved overall survival in patients with new-onset AML.

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