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[Genetic analysis and targeted therapy for acute myeloid leukemia]
1Department of Hematology, Nippon Medical School.
Abstract:
Several chromosomal abnormalities and gene mutations involved in the onset and recurrence of acute myeloid leukemia (AML) were discovered with the recent progress of genome analysis technology. The founding not only have clinical application as prognostic factors and minimal residual disease markers but also contribute to the novel molecular-targeted drug development. Many new drugs such as first-generation FLT3 inhibitor, IDH1/2 inhibitor, and BCL2 inhibitor have been developed in Europe and the United States. In addition, second-generation FLT3 inhibitors, gilteritinib and quizartinib, were developed in Japan, and the treatment outcome of AML has been improved. However, a large disparity in the drug availability remains between Europe and the United States and Japan. As a result, treatment guidelines in Europe and the United States cannot be applied to the practical use in Japan. In this paper, molecular-targeted drug treatment by gene diagnosis will be considered for AML in Japan, and the future paradigm shift of gene diagnosis and treatment of AML will be outlined.
Insights
Recent genomic advances identify acute myeloid leukemia (AML) mutations for targeted therapies. However, drug availability disparities between regions necessitate tailored treatment guidelines for AML in Japan.
Area of Science:
- Genomics
- Oncology
- Pharmacology
Background:
- Genomic analysis has revealed chromosomal abnormalities and gene mutations crucial for acute myeloid leukemia (AML) development and relapse.
- These genetic findings serve as prognostic factors and minimal residual disease markers, guiding novel molecular-targeted drug development.
Purpose of the Study:
- To discuss molecular-targeted drug treatments for AML in Japan based on gene diagnosis.
- To outline the future paradigm shift in AML gene diagnosis and treatment strategies within the Japanese context.
Main Methods:
- Review of recent advancements in genome analysis technology for AML.
- Analysis of molecular-targeted drugs, including FLT3, IDH1/2, and BCL2 inhibitors.
- Comparison of drug availability and treatment guidelines between Europe/United States and Japan.
Main Results:
- Development of first-generation FLT3, IDH1/2, and BCL2 inhibitors in Europe and the US.
- Development of second-generation FLT3 inhibitors (gilteritinib, quizartinib) in Japan, improving AML outcomes.
- Significant disparities in drug availability between regions impact treatment guideline applicability.
Conclusions:
- Gene diagnosis is essential for personalized molecular-targeted therapy in AML.
- Current treatment guidelines from Europe and the US are not directly applicable to Japan due to drug availability differences.
- A paradigm shift towards region-specific gene diagnosis and treatment strategies is needed for AML in Japan.
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