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New Drug Offers Better Quality of Life for Older Adults With Acute Myeloid Leukemia

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Key Takeaways

  • Older adults with acute myeloid leukemia have a high incidence of relapse and low survival rates.
  • A new medication provides an option for some patients who are not candidates for more aggressive measures during the maintenance phase of treatment.

A recently-FDA approved drug, Onureg, is now a suitable option for some older adults in maintenance therapy for acute myeloid leukemia (AML) who cannot tolerate more aggressive treatments.

AML patients often receive azacitidine, a chemotherapy drug, either intravenously or by injection. Onureg is an oral formulation of azacitidine, not previously used to treat AML or any other cancer.

After receiving chemotherapy and entering remission, AML patients begin a new care phase known as maintenance therapy. Unfortunately, many patients with AML are older and cannot tolerate the current treatment options available to them, including bone marrow transplants.

What Is Maintenance Therapy?

According to the American Cancer Society, after initial cancer treatment, a patient may receive maintenance therapy: low doses of chemotherapy over a prolonged period of time intended to prevent relapse. Maintenance therapy can last from months to years.

The QUAZAR study, published in the New England Journal of Medicine, examined the use of Onureg in AML patients who had entered the maintenance phase. Researchers followed 472 patients in 23 countries who were in complete remission for AML. All participants were 55 years of age or older, with an average age of 68, who were not candidates for bone marrow transplants.

One group of 238 patients received Onureg, also called CC-486, during the trial; the remaining 234 received a placebo. Participants who received CC-486 had an overall survival of 24.7 months, nearly ten months longer than those who received the placebo. The group that received CC-486 also had more prolonged survival without relapse: 10.2 months compared to 4.8 months in the placebo group.

The most remarkable adverse events, noted about equally in both groups, included gastrointestinal events and low platelets. Participants who received CC-486 experienced low white blood cell counts in 41% of cases compared to 24% in the placebo group.

Improving Quality of Life

Researchers also evaluated the effects of CC-486 on quality of life and found that recipients of the drug reported no negative impact.

"Our findings show that CC-486 significantly delays recurrence of the disease, thereby prolonging survival and without impacting on the quality of life. This is a very significant advance because the drug is easy to administer and means that adults with AML don't have to spend extra time in the hospital," study author Andrew Wei, PhD, said in a press release.

Andrew Wei, PhD

The drug is easy to administer and means that adults with AML don't have to spend extra time in the hospital.

— Andrew Wei, PhD

Lee Greenberger, PhD, Chief Scientific Officer for The Leukemia & Lymphoma Society, tells Verywell Health that the average age of a patient with AML is 68. Many of them cannot tolerate aggressive treatment measures, which are more successful in patients under 55.

“Oral azacitidine has been in the works for over a decade, Greenberger tells Verywell. "When the drug is given orally, the exposure is more spread out over time and the metabolism of the drug is different compared to subcutaneously (an injection) or intravenously administered azacitidine.”

Patients can also take Onureg at home instead of receiving it in a hospital or outpatient facility, another attractive feature.


Younger patients can take Onureg, but Greenberger suggests they explore more traditional measures first.

“Any patient with complete hematologic response can go onto maintenance therapy, but patients under 55 should consider a bone marrow transplant because we don’t know oral azacitidine will be as good as a bone marrow transplant,” he says.

While Onureg offers hope for some older patients with AML, more research is needed, Greenberger says.

“Patients who took this maintenance therapy got, on average, 10 months of relapse-free survival compared to approximately five months in the placebo-treated group," he says. "While oral azacitidine also increased overall survival by 10 months compared to the placebo-treated group, after four years, approximately 20% of AML patients survived, regardless of the treatment. Therefore, oral azacitidine extends overall survival, but it’s not a cure.”

Still, overall, this trial yielded valuable data in the fight against AML and other blood cancers.

“In this particular trial, the concept of maintenance therapy has beneficial effects for the patient,” Greenberger says.

Greenberger says the Leukemia and Lymphoma Society is exploring the use of Onureg in treating diffuse large B-cell lymphoma (DLBCL), the most common variant of non-Hodgkin’s lymphoma, and T-cell lymphoma.

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Article Sources
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  1. U.S. Food & Drug Administration. FDA approves Onureg (azactidine tablets) for acute myeloid leukemia. Updated September 1, 2020.

  2. American Cancer Society. Chemotherapy for acute myeloid leukemia. Updated September 3, 2020.

  3. Wei AH, Döhner H, Pocock C, et al. Oral azacitidine maintenance therapy for acute myeloid leukemia in first remissionN Engl J Med. 2020;383(26):2526-2537. doi:10.1056/NEJMoa2004444

  4. Wei A, Döhner H, Pocock C et al. Oral azacitidine maintenance therapy for acute myeloid leukemia in first remissionNew England Journal of Medicine. 2020;383(26):2526-2537. doi:10.1056/nejmoa2004444

  5. Monash University. Global trial reveals life saving drug for acute myeloid leukemia. Medical Xpress. Updated December 23, 2020.