Development of a Novel Patient-Reported Outcome Diary to Capture and Monitor Home-Managed Pain Crises in Patients with Sickle Cell Disease.
| Title: | Development of a Novel Patient-Reported Outcome Diary to Capture and Monitor Home-Managed Pain Crises in Patients with Sickle Cell Disease. |
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| Authors: | Turnbull J; IQVIA, 600 Lexington Ave, New York, NY, 10022, USA. james.turnbull@iqvia.com.; Williams B; IQVIA, 600 Lexington Ave, New York, NY, 10022, USA.; Myrick S; IQVIA, 600 Lexington Ave, New York, NY, 10022, USA.; Bascle S; Sanofi, Gentilly, France.; Daak A; Sanofi, Cambridge, MA, USA.; Jones K; Sanofi, Cambridge, MA, USA.; Maher J; Sanofi, Reading, UK. |
| Source: | Advances in therapy [Adv Ther] 2026 Mar; Vol. 43 (3), pp. 1255-1273. Date of Electronic Publication: 2026 Jan 27. |
| Publication Type: | Journal Article |
| Language: | English |
| Journal Info: | Publisher: Health Communications Inc Country of Publication: United States NLM ID: 8611864 Publication Model: Print-Electronic Cited Medium: Internet ISSN: 1865-8652 (Electronic) Linking ISSN: 0741238X NLM ISO Abbreviation: Adv Ther Subsets: MEDLINE |
| Imprint Name(s): | Publication: New York : Springer Healthcare Communications, 2008- : Health Communications Inc.; Original Publication: Metuchen, N.J. : Health Communications Inc., c1984- |
| MeSH Terms: | Anemia, Sickle Cell*/complications ; Pain Management*/methods ; Pain*/etiology ; Patient Reported Outcome Measures*; Humans ; Adult ; Male ; Female ; Adolescent ; Quality of Life ; Qualitative Research ; Young Adult ; Home Care Services ; Pain Measurement |
| Abstract: | Introduction: 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.; Methods: 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.; Results: 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.; Conclusion: 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.; (© 2026. The Author(s).) |
| Competing Interests: | Declarations. Conflict of Interest: James Turnbull, Betsy Williams, and Shane Myrick are employees of IQVIA, which received funding from Sanofi to conduct this study. Stéphanie Bascle, Ahmed Daak, Kyran Jones, and Joshua Maher are employees or former employees of Sanofi, and may hold stock options in the company. Joshua Maher is currently Director, Patient Centred Outcomes, at Kyowa Kirin. Kyran Jones is currently at Biogen. Ethical Approval: This study was performed in accordance with the Helsinki Declaration of 1964 and its later amendments. The study protocol was approved by the WCG institutional review board (IRB Tracking Number 20216266). All patients provided a completed informed consent form (adolescent patients provided assent to the study, with parents/caregivers providing informed consent) before participating in the interviews, per the inclusion criteria. In the informed consent form, patients were informed that if this study were to be published, no identifiable information would be included. |
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| Contributed Indexing: | Keywords: Cognitive debriefing; Concept elicitation; Conceptual model; Patient-reported outcomes; Sickle cell disease; Sickle cell pain crisis; eDiary; Local Abstract: [plain-language-summary] 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. |
| Entry Date(s): | Date Created: 20260127 Date Completed: 20260318 Latest Revision: 20260321 |
| Update Code: | 20260321 |
| PubMed Central ID: | PMC12999680 |
| DOI: | 10.1007/s12325-025-03473-4 |
| PMID: | 41591645 |
| Database: | MEDLINE |
Journal Article