Subsequent lines of therapy increase humanistic and economic burden of CML

After first-line treatment has failed, patients may receive an increased dose of the same TKI, switch to another TKI, switch to interferon or chemotherapy, or to have a stem cell transplant.

Researchers performed a literature review to understand the humanistic and economic burden in patients with CML receiving different lines of ITK. They noted that “there is an unmet need for new treatments for heavily pretreated CML-CP 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 studies assessed humanistic burden: 23 reported on HRQL and 5 reported on symptom burden. “Physical function and emotional function were the most severely affected quality of life areas in most studies,” the authors reported.

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

Ten studies reported data on costs and 5 on resource use. The main contributors to the increased costs associated with treatment failure in subsequent treatment lines were inpatient services, emergency department visits, outpatient care, and laboratory tests.

“Total costs per month increased dramatically for patients who switched from first-line treatment to second-line treatment,” 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 rose to $ 99,624 for patients who failed second-line treatment and $ 181,029 for patients whose third-line treatment has failed.

Patients who switched to subsequent lines of treatment had more emergency room, hospital and outpatient visits than patients who remained on the initial line of TKI treatment.

“Overall resource use was higher for patients who failed 2 lines of therapy compared to those who failed 1 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 myelogenous leukemia – a review of the literature. Presented at: Virtual ISPOR 2021; May 17-20, 2021. Poster PCN231.

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