Sustained Humanistic Burden and Work Impact in Adults with Sickle Cell Disease with Recurrent Vaso-Occlusive Crises: Results from a Global Longitudinal Survey
Author(s)
Drahos J1, Boateng-Kuffour A1, Calvert M2, Valentine A3, Mason A4, Li N1, Pakbaz Z5, Shah F6, Ainsworth N7, Martin A7
1Vertex Pharmaceuticals Incorporated, Boston, MA, USA, 2University of Birmingham, Birmingham, UK, 3Sick Cells, Washington, DC, USA, 4Sickle Cell Care Manchester, Manchester, UK, 5University of California Irvine School of Medicine, Orange, CA, USA, 6NHS Blood and Transplant, London, UK, 7QC Medica, Liverpool, UK
Presentation Documents
OBJECTIVES: Sickle cell disease (SCD) is characterized by painful vaso-occlusive crises (VOCs). This longitudinal study examined the impact of SCD on participants’ health-related quality of life (HRQoL) and work productivity.
METHODS: A longitudinal survey was administered at three timepoints (month 0 [M0], 3 months [M3], 6 months [M6]) in the US, the UK, France, Germany, and Italy and included multiple patient-reported outcome (PRO) measures: FACT-G, EQ-5D-5L, ASCQ-Me, FACIT-Fatigue (only M3), pain 11-point NRS, and WPAI. Key eligibility criteria included experiencing severe, recurrent VOCs, defined as ≥2 VOCs/year for 2 years that required an interaction with a medical facility and administration of pain medication or red blood cell transfusion. Descriptive analyses were conducted at M3. These data were compared with M0 data (previously reported), where available.
RESULTS: The survey was completed by 118 adult participants with SCD in the US (n=73), the UK, France, Germany, and Italy (n=45) at M3. Overall, 72.9% were female, their mean age was 34.7 years (standard deviation [SD]=11.2), and only 17.8% had full-time employment. Annual mean number of severe VOCs was 6.0 (SD=4.9). At M3, the mean FACT-G score was 59.8 (SD=19.6). Participants had a mean EQ-5D-5L utility score of 0.54 (SD=0.31). On the ASCQ-Me, participants reported worse pain, stiffness, emotional, and social impact compared with the US general SCD adult population. The mean FACIT-Fatigue score was 24.2 (SD=10.2) and comparable with that of patients with anemia and cancer (mean=23.9 [SD=12.6]). The average pain 11-point NRS score was 4.5 (SD=2.4). According to the WPAI, employed participants had 31.9% (SD=32.6%) absenteeism, 44.8% (SD=29.0%) presenteeism, and 63.7% (SD=28.1%) reduction in work productivity in the past week. PRO findings were comparable between M0 and M3.
CONCLUSIONS: Despite receiving currently available treatments, adults with SCD and recurrent VOCs continue to experience substantial and sustained humanistic burden and indirect impacts.
Conference/Value in Health Info
Value in Health, Volume 26, Issue 6, S2 (June 2023)
Code
PCR85
Topic
Patient-Centered Research
Topic Subcategory
Patient-reported Outcomes & Quality of Life Outcomes
Disease
Genetic, Regenerative & Curative Therapies