Qualitative Assessment of Health-Related Quality of Life Impacts Associated with Sickle Cell Disease in the United States and United Kingdom - PubMed (original) (raw)

Jennifer Drahos et al. Adv Ther. 2025 Feb.

Abstract

Introduction: Individuals living with sickle cell disease (SCD) commonly report impaired health-related quality of life (HRQoL). However, impacts of SCD on HRQoL and the unmet needs of SCD treatment/management are under-researched. This study characterized the impact of SCD on HRQoL and identified the unmet needs of individuals with SCD.

Methods: Adults with SCD (aged ≥ 18 years) and caregivers of adolescents (aged 12‒17) with SCD in the United States (US) and United Kingdom (UK) participated in one-on-one virtual semi-structured interviews and focus group discussions (hereafter referred to as 'interviews'). Interviews were transcribed and thematically analyzed.

Results: Nineteen individuals participated in the study (across five interviews and three focus group discussions), including 18 adults with SCD (United States, n = 11; United Kingdom, n = 7) and one caregiver of an adolescent with SCD (United States). Most participants were female (n = 15). Participants reported negative impacts of SCD on their HRQoL, including the burden of structuring their lives around SCD, due to unpredictable symptoms. They reported negative impacts to psychological health (e.g., depression/low mood and anxiety) and physical health (e.g., chronic pain and fatigue) that affected their social and family life, work, and education, leading to feelings of isolation. Participants expressed concerns about the future, feelings of resentment, and the need for high resilience when facing the barriers/impacts associated with SCD. Many participants reported negative interactions with healthcare professionals, leading to trauma, anxiety, and routine care avoidance. Most participants reported perceived prejudice during routine SCD treatment/management, including being treated as drug-seekers.

Conclusion: Individuals with SCD experience negative HRQoL impacts, including impacts to daily activities, social and family life, work and education, psychological health, and prejudice/stigma. Our findings highlight significant unmet needs of individuals living with SCD, including alternative treatment options to reduce vaso-occlusive crisis (VOC) frequency and treat fatigue.

Keywords: Fatigue; Focus groups; Health-related quality of life; Hemoglobinopathy; Qualitative interviews; Sickle cell disease; Vaso-occlusive crises.

© 2024. The Author(s).

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Conflict of interest statement

Declarations. Conflict of Interest: Jennifer Drahos, Adriana A. Boateng-Kuffour, and Nanxin Li are employees of Vertex Pharmaceuticals Incorporated and may hold stock or stock options in the company. Melanie Calvert is Director of Birmingham Health Partners Centre for Regulatory Science and Innovation and Centre for Patient Reported Outcomes Research and a National Institute for Health and Care Research (NIHR) senior investigator; has received funding from Anthony Nolan, European Regional Development Fund-Demand Hub and Health Data Research UK, Gilead, GlaxoSmithKline, Janssen, Macmillan Cancer Support, Merck, NIHR, NIHR ARC WM, NIHR Birmingham BRC, NIHR BTRU Precision and Cellular Therapeutics, UCB Pharma, UKRI, and UK SPINE; and has received consultancy fees from Aparito, Astellas, CIS Oncology, Daiichi Sankyo, Gilead, Glaukos, GlaxoSmithKline, Halfloop, Merck, Patient-Centered Outcomes Research Institute, Pfizer, Takeda, and Vertex Pharmaceuticals Incorporated. Ashley Valentine has received consulting fees for Atlas Clarity Consulting Firm and Vertex Pharmaceuticals Incorporated; acted as Board of Director for IVI; and acted as an advisory board member for PCORI. Anthony Mason has received consulting fees for Vertex Pharmaceuticals Incorporated. Zahra Pakbaz has received research grants from Amgen, Forma Therapeutics, Global Blood Therapeutics, Novartis, NovoNordisk, and Pfizer; received consulting fees from Agio, Amgen, Dova, Global Blood Therapeutics, Guide point, Novartis, Sanofi, Sobi, and Vertex Pharmaceuticals Incorporated; received honoraria from Cayeene Wellness Center and Child Foundation, Dova, and Global Blood Therapeutics Inc; served as an advisory board or committee member for Alexion, Sanofi, and Sobi; and acted as a CME course director for the Cayenne Wellness Center and Child Foundation and planning committee member for their annual education symposium. Farrukh Shah has received research grants from IQVIA, Novartis Pharma AG, and Vertex Pharmaceuticals Incorporated; received honoraria from Biologix FZ co, Bristol Myers Squibb, Chiesi Ltd, and Novartis Pharma AG; served as an advisory board or committee member for Agios, bluebird bio, Bristol Myers Squibb, Silence Therapeutics Plc, and Vertex Pharmaceuticals Incorporated; and acted as Chair for the UK Forum on Haemoglobin Disorders. Antony P. Martin is a partner of QC Medica who was funded by Vertex Pharmaceuticals Incorporated to perform this research. Ethical Approval: This study was conducted in accordance with the ethical standards of the institutional and/or national research committee and the 1964 Helsinki Declaration and its later amendments, as well as with the Association of the British Pharmaceutical Industry, British Healthcare Business Intelligence Association, and European Pharmaceutical Market Research Association codes of conduct [22–24]. All participants provided informed consent for participation and publication of the results before participating in the study. Participant eligibility was determined using the study’s inclusion/exclusion criteria, fielded through an eligibility screening questionnaire. The WIRB-Copernicus Group® Institutional Review Board reviewed the study and granted exemption based on 45 CFR § 46.104(d)(2).

Figures

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Fig. 1

Key themes identified in participant interviews. HRQoL health-related quality of life, SCD sickle cell disease, VOC vaso-occlusive crisis

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