Later Lines of Therapy Increase Humanistic and Economic Burden in CML

As patients with chronic myeloid leukemia (CML) fail on treatment and move to later lines of therapy, the impact on their health-related quality of life and the economic burden significantly increase.

For patients with chronic myeloid leukemia (CML), the economic and health-related quality of life burden (HRQOL) increases as they move into later lines of therapy, according to a poster presented at Virtual ISPOR 2021.

Tyrosine kinase inhibitors (TKIs) are the standard treatment for chronic phase CML (CP-CML). The rate of treatment failure increases with the subsequent lines of therapy from 50% of patients on second-line treatments to up to 80% of patients receiving third-line treatment.

After patients fail on the first line therapy, they may receive an increased dose of the same TKI, be switched to a different TKI, switch to interferon therapy or chemotherapy, or stem cell transplantation.

The researchers conducted a literature review to understand the humanistic and economic burden in patients with CML receiving different lines of TKIs. They noted that “there is an unmet need for novel treatments for heavily pretreated CP-CML patients.”

They searched for literature published from 2001 to July 2020 that included humanistic and economic data for adult patients with CML. A total of 1601 records were identified, but ultimately only 33 publications were included in the review. Eleven were journal articles and 22 were conference abstracts.

Most of the studies evaluated the humanistic burden: 23 reported on HRQOL and 5 reported on symptom burden. “Physical function and emotional function were the most severely impacted QOL domains in most of the studies,” the authors reported.

One study found that 58% of patients treated with first- or second-line TKIs had mild QOL impairments, 11% had moderate impairments, and 31% severe impairments. The most frequently reported symptoms were fatigue, musculoskeletal pain, and disturbed sleep. One of the studies reported 19 symptoms associated with CML.

Ten studies reported on cost data and 5 on resource use. The primary drivers of increased cost burden associated with treatment failure in later lines of therapy were inpatient services, emergency department (ED) visits, ambulatory care, and laboratory tests.

“Total costs per month increased significantly for patients who switched from first line to second line therapy,” the researchers wrote.

According to one study, for patients who failed first-line treatment, the average cost in 2012 US dollars was $78,677, which increased to $99,624 for patients who failed second-line treatment, and $181,029 for patients with failure of third-line treatment.

Patients who switched to later lines of therapy had more ED, hospital, and outpatient visits than patients who stayed on the initial line of TKI therapy.

“Overall resource utilization was higher for patients with failure of 2 lines of therapy compared with those who failed on one line of therapy,” the authors wrote.


Negi H, Agrawal R, Vieira J, Ryan J, Thakur D, Viana R. Humanistic and economic burden in patients with chronic myeloid leukemia – a review of the literature. Presented at: Virtual ISPOR 2021; May 17-20, 2021. Poster PCN231.

Related Videos
Related Content
© 2023 MJH Life Sciences
All rights reserved.