Major Research Findings

FLT3 inhibitors are effective in treating patients with acute myeloid leukemia (AML) who have FLT3 gene mutations. These inhibitors have recently gained prominence as AML treatment options in clinical trials. Recent studies have shown that midostaurin and gilteritinib have been approved by the United States Food and Drug Administration (FDA) as frontline treatment for AML and as therapeutic agents for recurrent disease. 1

Gilteritinib was approved in Japan in 2018 for the treatment of relapsed or refractory acute myeloid leukemia with FLT3 mutations. 2 A multicenter, observational surveillance study of gilteritinib use in Japan was conducted in accordance with regulatory requirements to assess the safety and efficacy of gilteritinib. 2

Oral medication adherence may be reduced in AML patients due to side effects associated with oral medication use. 4 A study investigating oral medication adherence in AML found that side effects associated with the use of oral medications, such as midostaurin, enasidenib, ivosidenib, gilteritinib, glasdegib, and venetoclax, contributed to reduced adherence. 4

Reasons for side effects

FLT3 inhibitors work by inhibiting the growth of AML cells with FLT3 gene mutations, but they can also affect normal cells. Therefore, there is a potential for various side effects.

Common Side Effects

Gastrointestinal

Gastrointestinal problems are common side effects of FLT3 inhibitors. 1 Specific symptoms include nausea, vomiting, diarrhea, and loss of appetite. 1

Hematologic

FLT3 inhibitors can inhibit the production of blood cells. 1 This can lead to side effects such as anemia, leukopenia, and thrombocytopenia. 1

Liver

FLT3 inhibitors can affect the liver. 1 Increased liver enzyme levels and jaundice can occur. 1

Side Effects Management

Gastrointestinal

Antiemetics and other medications are used to manage gastrointestinal side effects such as nausea and vomiting. 1 Nutritional support and appetite stimulants are used for loss of appetite. 1

Hematologic

Hematopoietic growth factors and other medications are used to manage side effects that inhibit blood cell production. 1 Treatments such as red blood cell transfusions are used for anemia. 1

Liver

Steroids and other medications are used to manage liver side effects. 1

Comparison between studies

Commonalities

Multiple studies have shown that FLT3 inhibitors are effective treatment options for patients with AML who have FLT3 mutations. 1 2 These studies have also shown that FLT3 inhibitors can cause a variety of side effects. 1 2

Differences

The drugs investigated and patient characteristics varied in each study, resulting in some differences in research findings. 1 2 For example, gilteritinib is a newer drug than midostaurin, so more research is needed. 1 2

Real-life application notes

Although FLT3 inhibitors are effective drugs for treating AML patients, it is important to understand the risks of side effects. 1 Consult your doctor if you experience any side effects. 1

Current research limitations

Research on FLT3 inhibitors is still in its early stages. Further research is needed to investigate the long-term safety and effectiveness of these drugs. 1

Future research directions

Research is needed on the long-term safety and effectiveness of FLT3 inhibitors, as well as on overcoming drug resistance. 1 These studies are expected to contribute to the development of safer and more effective treatments. 1

Conclusion

FLT3 inhibitors are effective drugs for treating patients with AML who have FLT3 mutations, but it is important to understand the risks of side effects. 1 Future research is expected to lead to the development of safer and more effective treatments. 1