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Introduction <p>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.</p> Methods <p>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.</p> Results <p>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.</p> Conclusion <p>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.</p>

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

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2026年1月27日

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  • James Turnbull,
  • Betsy Williams,
  • Shane Myrick,
  • Stéphanie Bascle,
  • Ahmed Daak,
  • Kyran Jones,
  • Joshua Maher

摘要

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.