Drahos Jennifer, Boateng-Kuffour Adriana, Calvert Melanie, Valentine Ashley, Mason Anthony, Li Nanxin, Pakbaz Zahra, Shah Farrukh, Martin Antony P
Vertex Pharmaceuticals Incorporated, Boston, MA, USA.
Health Economics and Outcomes Research, Vertex Pharmaceuticals Incorporated, 50 Northern Avenue, Boston, MA, 02210, USA.
Adv Ther. 2025 Feb;42(2):863-885. doi: 10.1007/s12325-024-03038-x. Epub 2024 Dec 16.
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.
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.
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.
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.
镰状细胞病（SCD）患者普遍报告其健康相关生活质量（HRQoL）受损。然而，SCD对HRQoL的影响以及SCD治疗/管理方面未满足的需求研究不足。本研究描述了SCD对HRQoL的影响，并确定了SCD患者未满足的需求。
美国（US）和英国（UK）的成年SCD患者（年龄≥18岁）以及青少年（12 - 17岁）SCD患者的照料者参加了一对一的虚拟半结构化访谈和焦点小组讨论（以下简称“访谈”）。访谈内容被转录并进行了主题分析。
19人参与了该研究（包括5次访谈和3次焦点小组讨论），其中有18名成年SCD患者（美国11名；英国7名）和1名青少年SCD患者的照料者（美国）。大多数参与者为女性（n = 15）。参与者报告SCD对其HRQoL有负面影响，包括因症状不可预测而围绕SCD安排生活的负担。他们报告了对心理健康（如抑郁/情绪低落和焦虑）和身体健康（如慢性疼痛和疲劳）的负面影响，这些影响了他们的社交和家庭生活、工作及教育，导致孤独感。参与者对未来表示担忧、感到怨恨，并且在面对与SCD相关的障碍/影响时需要具备高度的适应能力。许多参与者报告与医疗保健专业人员有负面互动，导致创伤、焦虑并避免常规护理。大多数参与者报告在常规SCD治疗/管理过程中感受到偏见，包括被视为药物寻求者。
SCD患者的HRQoL受到负面影响，包括对日常活动、社交和家庭生活、工作和教育、心理健康以及偏见/污名的影响。我们的研究结果突出了SCD患者存在的重大未满足需求，包括减少血管闭塞性危机（VOC）发作频率和治疗疲劳的替代治疗方案。
