Conceptual model of chronic lymphocytic leukemia.

Abstract

e19001 Background: The study aimed to create a conceptual model (CM) of disease- and treatment-related signs, symptoms and impacts in patients with first-line (1L) or relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL). Methods: The CM was based on literature searches, review of CLL patient blogs/forums, interviews with expert hematologists, and semi-structured concept elicitation interviews with 40 patients with CLL (20 1L, 20 R/R; with fatigue, weight loss, fever and/or night sweats in previous week). To identify salient concepts, the number of patients mentioning each concept and the concept’s mean disturbance rating (from 0 [not at all] to 10 [very]) were mapped. Results: Literature searches identified 32 prevalent concepts, with the most common being fatigue/tiredness. Patient blogs/forums revealed further symptoms (muscle/joint aches, headaches, weakness) and impacts (stress, fear of death, uncertainty). Clinicians reported recurrent infections and swollen lymph nodes as key signs of CLL and noted that fatigue is related to both symptoms (e.g. tiredness) and impacts (e.g. needing to rest and limit daily activities). In patient interviews, 50 concepts were revealed, with 42 present in both 1L and R/R; fatigue was the most prominent and impactful. Fatigue-related sub-concepts were identified, covering symptoms (tiredness/need to sleep, lack of energy, weakness, cognitive fatigue) and impacts (decreased ability to maintain social/familial/professional role, decreased physical functioning, frustration). Three versions of the CM were created: an overall model with all identified concepts, and two models highlighting the most salient concepts in 1L and R/R. Muscle/joint aches, night sweats, bruising, fatigue-related symptoms, fever and recurrent infections/illness were salient symptoms in both 1L and R/R, as were impacts such as anxiety/worry, stress, depression, financial difficulty, and fear of death. Shortness of breath and coughing were salient in 1L only, and swollen lymph nodes, headaches, pain/discomfort, weight loss, nausea/vomiting, and infusion reactions were salient in R/R only. Conclusions: The three versions of the CM can help to inform patient-centered end points to evaluate new treatments for 1L and/or R/R CLL.