Patients With CLL Are Willing to Trade Efficacy For Less Severe Side Effects


To offset an increased risk for serious side effects, patients with chronic lymphocytic leukemia say they prefer a substantial increase in progression-free survival in order to accept this negative treatment attribute.

Patients with chronic lymphocytic leukemia (CLL) are willing to accept a shorter duration of progression-free survival (PFS) if it means they will avoid serious side effects from their treatment, according to a study published online in Blood Advances, a Journal of the American Society of Hematology.

Researchers from across the country also found that out-of-pocket costs played a deciding factor, too.

“People have preferences based on what they value the most,” Carol Mansfield, Ph.D., senior research economist at RTI Health Solutions, said in an interview with CURE. “As medicine moves toward a more patient-involved paradigm, we are learning that people have different preferences and circumstances, so there may not be a ‘one size fits all’ medicine choice.”

More than 4,000 patients with CLL were recruited by the Leukemia & Lymphoma Society to fill out an email survey. Only 384 patients consented to and completed it. Patients were on average 65 years old, approximately half were men and the majority were white.

They were asked to choose between hypothetical treatment options, each of which was defined by five variable attributes — PFS, mode of administration, typical severity of diarrhea, chance of serious infection and chance of organ damage.

Although patients placed the highest importance on longer PFS, reductions in the risk for side effects also appeared to be of importance. For example, patients said they would need, on average, an additional 36 months of PFS in order to accept an increased risk for serious infection. In turn, this amount of added efficacy would offset a change in side effects, in this case, patients going from having no risk for side effects to a 30 percent chance of an adverse event occurring, according to the study authors.

“Patients value both efficacy and quality of life during treatment, and they are often willing to make trade-offs,” said Mansfield.

In addition, cost of care plays a significant role in treatment decision-making. When cost was not a factor, 91 percent of patients responded that they would choose the medicine with the longest PFS. But when the choice changed and patients had to choose between two medicines where out-of-pocket costs were indicated, patients chose the cheaper medicine despite the lower PFS. In the scenario where out-of-pocket costs amounted to a $400 difference between medicines, 75 percent of patients chose the cheaper medicine. In the case where there was a $75 difference in out-of-pocket cost per month, half (50 percent) chose the lower-cost medicine.

“Cost is complicated. It varies from patient to patient based on insurance and access to other sources of financial aid,” said Mansfield. “The reality is that the costs we presented in the survey were high enough that some patients couldn’t afford them.”

The study authors hope that these findings will help health care providers have frank and open discussions with patients about how different treatments will affect their lives, said Mansfield.

By having providers help patients manage side effects they can make a big difference, and may even help convince them to stay on drugs that have higher efficacy and worse side effects, she added. “Choosing a treatment is more than a go or no-go decision,” said Mansfield. “Patients don’t always understand the tradeoffs involved with different treatment plans. Once they understand the impact of their choices, they can make decisions based on what they are willing to tolerate and what they are willing to risk.”

Related Videos
Andrew McMahon, wearing a white sweater and a hat, in an interview with CURE
Experts on chronic lymphocytic leukemia
Experts on chronic lymphocytic leukemia
Experts on chronic lymphocytic leukemia
Related Content