Burden of disease in patients with a history of status epilepticus and their caregivers.

OBJECTIVE: Status epilepticus (SE) is a life-threatening neurological emergency with the potential for wide-ranging impact on patients and caregivers. In this study, the burden of disease in patients with a history of SE and their caregivers was assessed. METHODS: Adult patients as well as caregivers of children, adolescents, and adults who had experienced ≥1 SE event in the past 24 months completed an online survey. Functional, social, emotional, and economic burden in patients and caregivers was assessed. Burden was measured through concept-targeted questionnaires, including the US Centers for Disease Control and Prevention (CDC) Health-Related Quality of Life 4 (HRQoL-4) and the Work Productivity and Activity Impairment (WPAI) instruments. RESULTS: The 198 respondents comprised 49 adult patients, 51 caregivers of children, 47 caregivers of adolescents, and 51 caregivers of adults. Most patients (93.9%) were diagnosed with epilepsy. Patients' daily activities were highly affected, and many respondents reported a substantial long-term physical and mental impact on patients. The mean CDC HRQoL-4 score for unhealthy days per month ranged from 11.1 for caregivers of adults to 16.9 for caregivers of children. WPAI scores demonstrated a substantial impact on the ability of adult patients and all caregivers to work. Among respondents, caregivers of children reported the highest absenteeism from work (20%) and the lowest employment rate (33%). Proportions of caregivers reporting that their daily social life was impacted at least 'some of the time' ranged from 80% to 92%, with nearly half (47%) of caregivers of children responding that their social life was impacted 'all the time'. CONCLUSIONS: Status epilepticus episodes place a high burden on patients and caregivers. Notably, the burden appeared high across a variety of domains. This study highlights that the burden of disease is pronounced and wide-reaching and goes beyond the immediate physical and medical impact of an SE episode.

Development of a Conceptual Model of Chronic Lymphocytic Leukemia to Better Understand the Patient Experience.

BACKGROUND AND OBJECTIVE: Understanding the patient experience is important for identifying the unmet need in chronic lymphocytic leukemia. The current study aimed to develop a comprehensive chronic lymphocytic leukemia conceptual model. METHODS: The conceptual model was based on literature searches, review of chronic lymphocytic leukemia patient blogs/forums, and interviews with five expert clinicians, with 20 patients who received at most one treatment (first line) for their chronic lymphocytic leukemia, and with 20 patients with relapsed or refractory chronic lymphocytic leukemia. De-identified interviews were transcribed, coded, and evaluated using qualitative data analysis software. RESULTS: Thirty-five prevalent chronic lymphocytic leukemia-related symptom and impact concepts were identified from literature searches, patient blogs/forums, and clinician interviews. Patient interviews confirmed the identified concepts and revealed five additional concepts. Fatigue-related sub-components were identified from how patients described their fatigue, covering symptoms (tiredness/need for sleep, lack of energy, weakness, cognitive fatigue), and impacts (decreased ability to maintain their social, familial, or professional role, decreased physical functioning, frustration). Three versions of the conceptual model were created: an overall model with all concepts; a model highlighting the most prominent concepts in first line; and a model highlighting the most prominent concepts in relapsed or refractory disease. Prominent concepts in both first line and relapsed or refractory disease were fatigue-related symptoms and impacts, muscle/joint aches, night sweats, bruising, fever, recurrent infections/illness, insomnia, decreased cognitive/emotional functioning, anxiety/worry, stress, depression, financial difficulty, and fear of death. Dyspnea and cough were prominent in first line only, and enlarged lymph nodes, headaches, pain/discomfort, weight loss, nausea/vomiting, and infusion reactions were prominent in relapsed or refractory disease only. CONCLUSIONS: The results show that fatigue is a dominant issue affecting patients with chronic lymphocytic leukemia. The three versions of the conceptual model can help researchers to understand patients' unmet needs and guide the patient-reported outcome strategy for clinical trials.