Development of a modular patient-reported outcome and experience measure on patient needs and benefits in CLL (PBI-CLL).

Abstract

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 guidelines is met, treatment is initiated. When choosing from the numerous new treatment options, individual patient needs should be considered. To date, no instrument exists to capture these needs.

Methodology: The ePROM was developed based on the Patient Benefit Index (PBI) methodology which captures the importance of treatment goals as well as the achievement of these goals. The development considered the COSMIN guidelines and included semi-structured interviews with 28 patients with CLL and free-text questionnaires (n = 15). Data were analysed via qualitative content analysis according to Kuckartz. The PBI-CLL was finalised through an expert consensus and cognitive debriefing interviews with 14 patients.

Results: The content elicitation showed that the individual treatment burden and treatment goals in CLL vary considerably between patients, underlining the heterogeneity of this patient group. Patients reported disease burden in their physical constitution as well as mental burden. Many patients' main goal was to live normally and with the lowest impact possible through the CLL and its therapy. The PBI-CLL developed based on these data consists of three modules: therapy outcomes, process quality and relative treatment preferences. The cognitive debriefing interviews showed that patients find the instrument relevant, comprehensive, and comprehensible.

Conclusions: The PBI-CLL is the first instrument to assess patients' needs and benefits in CLL. The heterogeneity we found in patient needs and preferences underlines the importance of a modular instrument which measures treatment goals and benefits in a standardized way. The PBI-CLL shall support both patient-centred therapeutic decision making and treatment evaluation in clinical practice, as well as patient-centred benefit assessment in clinical and health care research. It should therefore be tested for its psychometric properties in a subsequent validation study.