Development of a Novel Patient-Reported Outcome Diary to Capture and Monitor Home-Managed Pain Crises in Patients with Sickle Cell Disease

Sickle cell pain crises (SCPCs) are debilitating pain events that significantly impact quality of life in patients with sickle cell disease (SCD), but no fit-for-purpose tool exists to capture patients’ experiences of them. We developed a patient-reported outcome (PRO) tool to capture patients’ SCPC experiences and ensured its content validity through cognitive debriefing (CD) interviews with adolescents and adults with SCD. In this non-interventional, qualitative research study, a targeted literature review (TLR) was conducted to create preliminary items and a preliminary conceptual model (CM) of SCPCs in patients with SCD. Next, an item-generation session was held with clinical experts, followed by hybrid concept elicitation (CE) and CD interviews with patients over three waves; the preliminary items and CM were refined iteratively after each wave. SCPC and impact disturbance ratings were summarised using descriptive statistics. CD responses were analysed on the basis of a semi-structured discussion guide. The TLR identified 12 articles describing 47 concepts related to pain, anatomical sites, duration, frequency, and impacts of SCPCs. In CE interviews, patients described various aspects of their SCPC experience, including anatomical sites affected, pain characteristics, and duration. CD interviews confirmed the content validity of the eDiary, with patients finding most questions and response options clear and useful. The diary was modified on the basis of patient feedback per wave. The SCPC eDiary is a novel PRO instrument that captures the frequency, severity, and impact of home-managed pain crises. Future studies should assess its real-world use and performance in clinical settings. Sickle cell disease (SCD) is a genetic disease characterised by abnormal haemoglobin and red blood cells. Sickle cell pain crises (SCPCs), which are hallmarks of SCD, are highly painful events that affect the lives of people with SCD. People with SCD often manage SCPCs at home rather than going to hospital or seeing a doctor. Because of this, it can be hard to measure how often these crises happen or how severe they are. As no established tool was available to reliably assess SCPCs for people with SCD, the authors created such a tool and tested it with people with SCD. First, the authors gathered published scientific research on the topic and identified features of SCPCs, including the location of pain, how long it lasts, and how often it happens. Next, the authors interviewed medical professionals and people with SCD to refine the understanding of SCPCs. The authors used this information to create an electronic diary (eDiary) for patients to record details of their SCPCs. The clarity and usefulness of this diary were then tested through interviews with teenagers and adults with SCD. Diary questions were updated on the basis of feedback from these interviews, and during the last interviews, the final eDiary was reported to be mostly clear and helpful. The SCPC eDiary is a new tool that helps record how often, for how long, and by how much pain crises affect patients at home. Future studies can check how useful this diary is in clinics or clinical research.