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Skipping Infusions? New Oral AML Treatment Makes it Possible

Skipping Infusions? New Oral AML Treatment Makes it Possible
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Published on January 26, 2021

Oral Acute Myeloid Leukemia Drug Offers New Treatment Option

Forget traveling to the clinic for costly infusions. Patients aged 55 and older with acute myeloid leukemia (AML) have a new treatment option: a once-daily tablet that can be taken at home.

In a global trial across 148 sites in 23 countries, those receiving the oral hypomethylating agent CC-486 (Onureg, azacitidine tablets) lived longer than those in the placebo group (median overall survival of almost 25 months vs. 15 months). CC-486 also extended how long patients lived without their cancer coming back by about five months (10 months vs. five months). The findings were published last month in the New England Journal of Medicine.

“…our findings show that CC-486 significantly delays recurrence of the disease, thereby prolonging survival and without impacting on quality-of-life,” said lead investigator Dr. Andrew Wei of Monash University's Australian Centre for Blood Diseases in a press release. “This is a very significant advance because the drug is easy to administer and it means that adults with AML don't have to spend extra time in [the] hospital.”

Onureg (CC-486) is an oral formulation of azacitidine (Vidaza), which is used to treat patients with some types of blood cancer and is given by injection or infusion. The FDA fast-tracked approval of Onureg in September based on Dr. Wei’s presentation at the American Society for Hematology (ASH) meeting in December 2019.

AML Treatment Regimen

AML is an extremely fast-moving cancer, with a poor prognosis. The average age at diagnosis is 68, according to the American Cancer Society. The five-year relative survival is 10.8%, and 2.7% for patients ages 65 to 74, and 75 or older, respectively. While many patients go into remission after initial therapy, the cancer typically returns.

“After intensive chemotherapy, the risk of AML relapse is high,” Dr. Wei said in the release. “Many older patients are not eligible to receive a stem cell transplant and so a less toxic option to reduce disease recurrence is desirable, rather than just being monitored and waiting for the disease to come back.”

The FDA has approved several drugs for patients ages 75 and older with AML, including venetoclax (Venclexta) and glasdegib (Daurismo) for use in combination with other drugs. However, none of them are curative.

“We have this, often, older patient population, where up until very recently we used lower intensity strategies, things like azacitidine (Vidaza), decitabine (Dacogen), low dose cytarabine (Cytosar-U),” said Dr. Courtney DiNardo in a recent Patient Power webinar. “They are effective for sure. But unfortunately, the majority of patients didn't respond well. It was not really seen as a durable curative treatment strategy.”

Dr. DiNardo, an associate professor of leukemia at the University of Texas MD Anderson Cancer Center in Houston, was the lead investigator for the phase III VIALE-A trial, a randomized study of patients ages 75 and older who were newly diagnosed with AML or ineligible for intensive chemotherapy because of co-morbidities, such as heart disease or poorly controlled diabetes. The findings were published in August in the New England Journal of Medicine.

The trial included 431 patients with a median age of 76; there were 286 in the venetoclax and azacitidine combination group and 145 in the azacytidine plus placebo group. The addition of venetoclax to azacitidine improved remission rates to 66.4% compared to 28.3% with azacitidine alone and resulted in a median overall survival of 14.7 months compared to 9.6 months.

The QUAZAR Clinical Trial

The QUAZAR trial involved 472 patients (median age 68) who were randomly assigned to receive Onureg 300 mg. or a placebo drug once daily for 14 days per 28-day cycle. Patients in the Onureg group were on the drug for 12 cycles, the placebo group for six cycles. All participants were enrolled within four months of achieving complete first remission or a form of remission called complete remission with incomplete hematologic recovery (CRi) following intensive chemotherapy. CRi is a risk factor for poor outcomes. The participants were not candidates for a stem cell transplant at the time of enrollment.

"Based on the results of the QUAZAR study, it is very exciting to think that, by taking a tablet that is relatively well-tolerated, we can help reduce relapse risk and improve survival,” Dr. Wei said.

~Megan Trusdell

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