Patients Report Improved Quality of Life with New Leukemia Drug

According to Dr. Mikkael Sekeres, patients on the drug live longer and better.

Patients often face trade-offs when they undergo cancer treatment. Their cancer may shrink, but side effects can reduce their quality of life.

In recent years, clinical studies have attempted to measure these trade-offs by surveying patients about their outcomes. Such surveys have given patients a voice in studies assessing a variety of new treatments.

One recently approved leukemia treatment, quizartinib, was similarly assessed for quality of life in patient surveys. The findings were revealed at the American Society of Hematology’s annual meeting on Monday, Dec. 11.

The results show that patients “not only live longer, they live better,” said Mikkael A. Sekeres, M.D., M.S., chief of the Division of Hematology at Sylvester Comprehensive Cancer Center at the University of Miami Miller School, senior author of the presentation.

A New, Targeted Drug for Acute Myeloid Leukemia

The new data are part of a larger clinical study that evaluated quizartinib in more than 500 patients with a certain form of acute myeloid leukemia (AML). AML is one of the most common types of leukemia in adults.

That study showed that adding quizartinib to standard chemotherapy, followed by long-term maintenance therapy with the new drug, improved patient survival. Patients treated with chemotherapy plus quizartinib lived, on average, 31.9 months, about twice as long as patients treated with chemotherapy plus a placebo.

The study involved patients at Sylvester and other cancer centers. And it led to the approval of quizartinib by the U.S. Food and Drug Administration this spring. The drug was green-lighted for patients with AML carrying a specific genetic mutation, which is present in about a quarter of AML patients. The drug targets this mutation, called FLT3-ITD.

The drug clearly extended the lifespan of these patients. But were there trade-offs?

“There are two aspects that I think about when I give a treatment to a patient,” said Dr. Sekeres. “The first is, will that treatment allow that person to live longer? The second is whether that treatment will allow that person to live better.”

To test this second question, he and his colleagues assessed questionnaires given to patients to track their quality of life throughout treatment. The new data reveal the results from these surveys.

The Patient View

When patients first enrolled in the study, they generally had a “very poor” quality of life, said Dr. Sekeres.

But after a month of treatment and after release from the hospital, they started to do better. “Quality of life took off and improved dramatically,” he said.

Patients reported improvements in physical and social functioning, emotional well-being and other factors throughout treatment.

Similar improvements generally occurred in patients treated with placebo or quizartinib. However, in one assessment, people treated with quizartinib showed a slower decline in quality of life with time, including cognitive function, appetite loss and constipation—though whether these changes were clinically meaningful is not clear.

The researchers concluded that quizartinib did not have a detrimental effect on patient-reported outcomes. “It turns out that those treated with the drug didn’t have to trade off on quality of life,” said Dr. Sekeres.

Treating the Whole Patient

Lessons from this and similar studies can help guide physicians as they navigate treatment for “the whole patient,” said Dr. Sekeres.

When he first meets a new patient, Dr. Sekeres assesses how he can tailor treatment to their form of leukemia. In addition, “I think about my patient’s goals and what they want to get out of treatment regimen. I am thinking about their quality of life from day one,” he said.

Researchers are getting better at defining the genetic underpinnings of cancers like AML and testing experimental drugs that take aim at the underlying biology, said Dr. Sekeres. New targeted drugs, like quizartinib, are under development.

“We’re also getting much better at paying attention to what our patients describe as their experience going through cancer,” said Dr. Sekeres. “And I think we’re getting better and better at truly assessing what’s meaningful to patients as they describe their quality of life.”

Tags: ASH2023, blood cancers, Dr. Mikkael Sekeres, Leukemia, Sylvester Comprehensive Cancer Center