gms | German Medical Science

Background: Chronic lymphocytic leukaemia (CLL) is the most common form of leukaemia in adults in western countries. Asymptomatic patients are under clinical observation; when indication for treatment according to treatment guidelines is met, treatment is initiated. Numerous new treatment options underline the importance of considering individual patient needs in treatment decision making. To date, no instrument exists to capture these needs.

Objective: This project aims to develop an electronic Patient-Reported Outcome Measure (ePROM) that assesses treatment goals and benefits in adult patients with CLL.

Methods: The ePROM is being developed based on the Patient Benefit Index (PBI) methodology which captures importance and achievement of treatment goals. The PBI-CLL will introduce three modules on treatment preferences: general preferences, process quality, and therapy outcomes. As the tool will also measure patient’s experiences throughout the therapy, it will also include elements of a patient-reported experience measure (PREM). Qualitative interviews with patients with CLL were conducted to capture treatment goals and disease burden in CLL; in addition, free-text questionnaires were completed by patients. Based on qualitative content analysis according to Kuckartz, a draft version of the PBI-CLL was developed in an expert panel including oncologists and patients; this will be adapted in the final study phase through cognitive debriefing interviews with patients.

Results: We conducted 28 qualitative interviews, and 15 free-text questionnaires were completed. The draft version of the instrument is currently being tested in cognitive debriefing interviews. Preliminary results show that the individual burden of CLL varies widely; some patients reported high, others low impact on their mental and physical health as well as other aspects of their life, including social life and work life. Patients reported different treatment goals, for example preferences in the administration method and site. They also wished for empathic haematologists who provide them with ample information about their disease and available treatment options. Overall, CLL patients wanted a treatment that allows them to live normally with few limitations through their disease or treatment. The draft version of the PBI-CLL consists of three modules that can be used for different patient groups. In the first cognitive debriefing interviews, patients rated the tool as relevant and easy to understand, however, they had some suggestions for minor changes. The study is estimated to be completed in summer 2024.

Implication for research and/or (healthcare) practice: The preliminary results show that burdens and treatment goals in CLL vary considerably between patients, which underlines the heterogeneity of this patient group. The findings confirm the importance of a new instrument measuring treatment goals and benefits in a standardised way which can be utilized in both routine care and clinical studies and may improve the healthcare of patients with CLL.

Funding: Other funding; Project name: PBI-CLL; This study is conducted in cooperation with and funded by AstraZeneca.