Genetic Alterations Can Determine Optimal Treatment Options for Patients

Cancer Connect

A recent article published in the journal Blood indicates that the detection of specific genetic alterations may help to individualize treatment strategies for patients with acute myeloid leukemia (AML). Through individualized therapy, a patients treatment is tailored according to specific biological properties of their cancer in order to provide optimal chances of a cure.

AML is a cancer of the white blood cells. The bone marrow contains early blood-forming cells, called stem cells, which grow and mature into 3 blood cell types: red blood cells, which provide oxygen to tissues; platelets, which aid in blood clotting; and white blood cells, which fight infection. AML is characterized by the rapid, uncontrolled growth of immature white blood cells in the bone marrow, which suppresses the formation and function of other blood cells. Ultimately the cancer cells invade other parts of the body including the blood, lymph system, and vital organs. AML is a rapid growing cancer and treatment must be aggressive for an optimal chance of cure. Treatment options for patients with AML usually consist of chemotherapy, radiation and/or stem cell transplantation.

AML patients are divided into 3 groups according to the presence of different genetic alterations that have caused the cancer. According to specific identified genetic alterations, patients either have a favorable prognosis (overall survival) following therapy, an intermediate prognosis or an unfavorable prognosis.

A recent clinical trial involving AML patients assessed the responses of these patients to different treatment regimens. Over 600 AML patients from the 3 prognostic categories received different treatments while they were in their first complete remission (disappearance of cancer). Patients from each group received either intensive chemotherapy only, intensive chemotherapy followed by an autologous (self) stem cell transplantation (SCT), or intensive chemotherapy followed by an allogeneic (donor) SCT.

Five years following treatment, patients in the favorable prognostic group achieved a significantly higher survival rate when treated with chemotherapy followed by an autologous or allogeneic SCT compared to chemotherapy alone. Patients with an intermediate prognosis achieved a significantly higher rate of survival when treated with chemotherapy alone or chemotherapy followed by an allogeneic SCT compared to an autologous SCT. Patients in the unfavorable prognostic group achieved the highest survival rate when they were treated with chemotherapy followed by an allogeneic SCT.

These results represent a clear indication that the determination of genetic abnormalities should, at least in part, dictate the treatment regimen utilized for the treatment of AML patients. The overall medical condition of a patient must also be taken into consideration when determining optimal treatment strategies. Further evaluation in clinical trials is warranted involving the interpretation of genetic aberrations in AML patients, particularly in the group of patients considered to have an unfavorable prognosis.

Patients with AML may wish to discuss genetic testing and treatment options with their physician, or the risks and benefit of participating in a clinical trial further evaluating prognostic indicators of this disease. Two sources of information regarding ongoing trials that can be discussed with a doctor include comprehensive, easy-to-use listing services provided by the National Cancer Institute ( and also provides personalized clinical trial searches on behalf of patients. (Blood, Vol 96, No 13, 2000)

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