A Strategy for the Measurement of Sickle Cell Disease Symptoms from the Patient Perspective
24th European Hematology Association (EHA) Congress (2019)
This poster provides an overview of the patient and clinician interviews and the literature research that informed the development of Cyclerion’s patient-reported outcomes (PRO) tool. This PRO tool is designed to measure the symptoms that patients with sickle cell disease indicated are most important and relevant to their daily lives. Cyclerion is using this instrument in the ongoing STRONG SCD study.
Sickle cell disease (SCD) conceptual model synthesized from the perspective of patients, the literature, and clinical experts
A literature review and interviews with clinical experts (N = 5) and adolescents and adults with SCD (N = 20) were conducted to develop an SCD conceptual model summarizing important SCD signs, symptoms, and impacts. Patients reported 22 symptoms that they experienced regularly and/or during pain crises, which were largely endorsed by the literature and/or clinical experts. Pain was reported by all patients, and tiredness was reported by 80%; both were reported as the most important to improve with treatment. Patients reported impacts of SCD on their daily lives across 9 domains (activities of daily living, cognitive, emotional, financial, leisure, physical, sleep, social, and work/school). Ojo et al. (EHA 2019) poster presentation
Incorporating the patient voice into symptom measurement in sickle cell disease clinical research
Funke Ojo, Cyclerion’s Senior Manager of Study Endpoints and lead developer of our sickle cell disease patient-reported outcomes (PRO) tool, shares insights on the work conducted to understand the symptoms that matter most to sickle cell patients. Cyclerion is incorporating patients’ perspectives into our own method of measuring the symptoms of sickle cell disease in our clinical research program, including in the ongoing STRONG SCD Phase 2 study.