Symptom burden in chronic myeloid leukemia (CML).

Abstract

6133 Background: Symptom burden is the combined impact of symptoms from disease and treatment on daily functioning. Lack of symptom recognition may result in failure to address symptoms and maximize patient functioning. Patient-reported outcomes (PROs) can be endpoints in clinical trials to establish treatment benefits. The purpose of this study was to define the content domain for a PRO measure of chronic myeloid leukemia (CML) symptom burden. Methods: This is stage I of a study to develop a PRO CML symptom-burden measure. Thirty-five patients with Philadelphia-chromosome-positive CML described symptoms over the disease course in single qualitative interviews. Content analysis was used to identify symptoms and define the content domain. After the interview, patients reported symptoms in the last 24 hours on the M. D. Anderson Symptom Inventory (MDASI) to provide context for analysis. Results: Mean patient age was 52.7 years (standard deviation [sd] = 13.8), 54.3% female, and 94.3% chronic phase receiving kinase inhibitors. Content analysis found a total of 44 symptoms, 26 reported by 20% or more patients, occurring over the disease course. Fatigue (followed by pain and nausea) was the symptom reported most often over the disease course. During the interviews, patients volunteered ways in which symptoms interfered with daily activities and how symptoms were managed to maintain acceptable quality of life. Symptoms led some patients to stop or consider stopping treatment or to decrease the dose or frequency of treatment. On the MDASI, fatigue (followed by trouble remembering and disturbed sleep) was the symptom reported most often in the last 24 hours. Fatigue had the highest mean severity rating (3.37, sd = 2.77, 0-10 scale) on the MDASI. Conclusions: Patients with CML experience numerous symptoms related to disease and treatment, with fatigue being the most prominent throughout the disease course. These symptoms can cause significant interference with daily activities, leading to therapy reductions. Accurate assessment and management of symptoms is critical to ensure optimal treatment with the best quality of life. The content domain for a PRO symptom-burden measure of CML encompasses the severity and activity interference of common symptoms of CML and its treatment. Author Disclosure Employment or Leadership Position Consultant or Advisory Role Stock Ownership Honoraria Research Funding Expert Testimony Other Remuneration Bristol-Myers Squibb, Novartis Bristol-Myers Squibb, Novartis Bristol-Myers Squibb, Novartis, Wyeth