Use of Patient-Reported Outcome Measures in Clinical Studies of Chronic Myeloid Leukemia: A Scoping Literature Review

Management of chronic myeloid leukemia (CML) with tyrosine kinase inhibitors has improved patient survival. However, patient quality of life (QOL) continues to be impacted by disease symptoms and treatment-related adverse events. Patient-reported outcome measures (PROMs) provide evidence of the patient experience. A scoping literature review was conducted to identify and summarize the evidence on PROMs used for patients with CML.

Embase and MEDLINE databases were searched for publications from 2001 to 2023 that reported PROMs. Ongoing and completed trials listed on ClinicalTrials.gov were also reviewed. Results were summarized according to the PROMs used and the information collected in these PROMs. After screening 6337 records, 208 unique studies were identified with published PRO evidence reporting data from 92 unique PROMs. The most commonly reported PROMs (in ≥5% of publications) were used in 115 studies, of which 45 were exclusively in the frontline setting. The most commonly used PROMs in studies in the frontline setting were variations of European Organisation for Research and Treatment of Cancer QLQ, Functional Assessment of Chronic Illness Therapy Measurement System/Functional Assessment of Cancer Therapy, and 36-Item Short Form Survey. This scoping literature review highlighted that a variety of PROMs are used in CML studies, including studies in the frontline setting. Different QOL aspects are measured by commonly used PROMs, and the choice of PROM is dependent on the study setting and objectives. A more comprehensive understanding of QOL gained by using appropriate PROMs will help optimize patient-centered treatment selection in CML.