Health state utilities for chronic lymphocytic leukemia: importance of prolonging progression-free survival

Abstract

Chronic lymphocytic leukemia (CLL) is a largely incurable disease which affects patients' health related quality of life (HRQL). Treatment is often initiated when symptoms affect HRQL, and patients can experience many rounds of treatment throughout their life. Therefore, the economic burden of CLL can be high. Utility or preference weights for health states reflect the value of HRQL of a given health state and range from 1 (full health) to 0 (dead) and below (negative values possible). Nine health states were developed representing different CLL treatment lines or disease stages. One hundred members of the UK general public valued each health state using the time trade-off methodology. Progression-free survival (PFS) without therapy (mean utility = 0.82) was the least burdensome, with relapsed lines of treatment (mean utility = 0.42) representing the greatest burden. The results underline the value in maintaining a state of PFS for as long as possible.

Differences in treatment goals and perception of symptom burden between patients with myeloproliferative neoplasms (MPNs) and hematologists/oncologists in the United States: Findings from the MPN Landmark survey

“…Finally, limited availability and access to effective treatment options that provide durable, clinically relevant improvements in QoL may also be a contributor to the observed discrepancies between patients and physicians. Among patients with chronic lymphocytic leukemia, the increased effectiveness of new treatment options is expected to correspond with improvements in health‐related QoL . It is likely that the use of more effective treatments in the MPN setting could yield similar improvements and enhance the alignment of patients and physicians with regard to MPN symptomatology and treatment goals.…”

“…Among patients with chronic lymphocytic leukemia, the increased effectiveness of new treatment options 20 is expected to correspond with improvements in healthrelated QoL. 21 It is likely that the use of more effective treatments in the MPN setting could yield similar improvements and enhance the alignment of patients and physicians with regard to MPN symptomatology and treatment goals.…”

Utility scores for vesicoureteral reflux and anti-reflux surgery

“…The prior elicitation effort using the Amazon Mechanical Turk platform found a utility score of 0.87, with a smaller sample [2]. For reference, published studies have elicited similar utility scores for other conditions including compensated hepatitis B-related cirrhosis (0.80) [5], progression-free survival with chronic lymphocytic leukemia (0.82) [6], and glaucoma (0.82) [7]. Conditions reported to have higher utility values include neonatal jaundice requiring phototherapy (0.99) [8] and transient neonatal neurological symptoms (0.95) [8].…”

Cost-effectiveness of First-line Chronic Lymphocytic Leukemia Treatments When Full-dose Fludarabine Is Unsuitable

“…Patient utility was based on a recent UK utility elicitation study with validated health states and EuroQoL-5D-3L (EQ-5D-3L) dimensions. 10 The impact of on/off treatment on quality of life (QoL) was accounted for. The UK utility values based on elicitation were adjusted to take into account the representative EQ-5D-3L tool-based generic QoL of 65-to 74-year-old Finns together with their symptoms/comorbidities (adjusting factor 0.776/0.820 based on the EQ-5D-3L QoL of 65-to 74-year-old Finns per utility with PFS off-treatment [43]/ [10], To avoid the underestimation of the intravenous drug administration costs, 42 the administration cost was approximated by using specialist health care visit/ administration (1 hour of time included) and additional time counted with nurse's employment cost (€24.50/hour 40,41 ).…”

“…CLL is more common in elderly people, with almost one half of the newly diagnosed CLL patients being at least 75 years of age. CLL causes significant humanistic [4][5][6][7][8][9][10] and economic 9,11,12 burdens.…”