New Treatments Offer Promise to People with Acute Myeloid Leukemia

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Over the past 50 years, there’s been great news about leukemia: The five-year survival rate has doubled to about 70 percent.

But the good news for acute myeloid leukemia, or AML, has been harder to come by. This rare but aggressive type of leukemia tends to affect older people and the five-year survival rate was just 32 percent in the National Cancer Institute’s most recent figures.

But fortunately, that may be changing, says UNC Health hematologist/oncologist Joshua Zeidner, MD, who specializes in the management of acute leukemia.

“There has been very little progress in the management of AML from the 1970s until 2017, but since 2017, 11 new drugs have been approved,” Dr. Zeidner says. “AML was seen as a cancer with very poor outcomes and limited effective therapies. That’s no longer the case, as we have new treatments that can be very effective for some patients, particularly elderly patients.”

Dr. Zeidner explains some of these advances as well as what you need to know about AML.

What is AML?

Every day, your bone marrow—soft tissue within your bones—makes billions of new blood cells. These blood cells develop and then travel throughout the body, carrying oxygen, fighting infection and forming clots.

Leukemia begins with a genetic change in the bone marrow, which creates abnormal versions of your blood cells. These damaged blood cells make it difficult for the healthy blood cells to do their work, causing symptoms such as fatigue, fever, infections, shortness of breath, bruising and swollen lymph nodes.

Leukemia can be classified as acute (progressing quickly) or chronic (developing more slowly), and by the type of blood cell that’s affected—myeloids or lymphoids. AML can be further classified by the specific type of mutation that occurred in the bone marrow and blood cells.

Your risk for having one of these mutations occur increases as you age.

“The median age at diagnosis for AML is 68,” Dr. Zeidner says. “As you get older, it’s more common to have a random event that triggers the abnormal divisions in the bone marrow.”

When you’re diagnosed with most cancers, your doctor will tell you what stage it is, which is an indication of how much the disease has spread in your body, and that dictates your treatment options. AML doesn’t use that traditional staging system. Instead, your doctor will identify treatment options based on your age and specific chromosomal changes and genetic mutations that are present in your cancer cells.

Treatment Options for AML

Once you’re diagnosed with AML, you’ll undergo genetic testing via a blood test and bone marrow biopsy so that your doctor can understand which cells have been affected.

“AML behaves very differently for people because of that genetic makeup,” Dr. Zeidner says. “With the data from those tests, we can tailor the treatment to the individual’s leukemia. For specific mutations, we have certain treatments; for other mutations, some treatments won’t work as well.”

Because AML is aggressive, it’s important to order those tests as soon as possible. Dr. Zeidner says UNC’s Leukemia Expanded Access Program (LEAP) has the goal of seeing newly diagnosed AML patients within one to two days of referral so the testing process can begin and patients can be monitored while waiting for results. Depending on your type of AML, you may receive a targeted therapy drug that will work to address specific changes in your cells causing your disease.

In addition to considering the genetic makeup of the chromosomes, your doctor will look at your overall health and your age.

“For patients younger than 60, the standard of care is inpatient chemotherapy,” Dr. Zeidner says. “It is the most aggressive option. During that time, the bone marrow doesn’t make blood cells. If the chemotherapy works, the bone marrow regenerates and begins making healthy cells again.”

These younger patients may be eligible for a bone marrow transplant, which is typically done after a round of intensive chemotherapy. After existing bone marrow cells are killed, new cells from a donor can be implanted to form a new immune system in the patient. If effective, it can cure AML.

The success of these treatments has improved as doctors have learned more about the specific genetic mutations that can cause AML.

Treating AML in Older Adults

Standard AML treatments are aggressive, and older patients have typically not responded as well to the treatments or the side effects, which led some providers to offer only palliative care to people over 70 diagnosed with AML.

Dr. Zeidner says that the developments of the past few years offer new promise for this age group.

“For elderly patients, we don’t pursue the most intensive treatments, but there have been huge advances that can improve survival,” he says. “These patients should still receive a referral to a specialist and be offered treatment.”

Dr. Zeidner says the biggest development so far for older patients is the FDA approval of the prescription medication venetoclax in 2018. In a clinical study, patients treated with a combination of venetoclax and azacitidine (a chemotherapy drug) lived for five months longer than those who took azacitidine alone. About 66 percent of patients who had the combination therapy experienced complete remission, compared to 28 percent of patients who took only azacitidine.

For some patients, treatments available through clinical trials may prolong life. Dr. Zeidner is hopeful that clinical trials will yield new options for patients as researchers continue to make progress on understanding the distinct cellular changes that occur in AML.

“We’re not yet at a precision medicine platform, where every patient is getting something different, but we’re heading in that direction,” Dr. Zeidner says. “Clinical trials are continuing to make an impact and move the field forward for AML so that we can focus on each person receiving the best treatment and improve survival.”

If you’re concerned about your health, talk to your doctor. If you need a doctor, find one near you.The post New Treatments Offer Promise to People with Acute Myeloid Leukemia first appeared on UNC Health Talk.

Cancer, Hematology, Rare Cancers