Humanistic and economic burden among caregivers of adults and children with sickle cell disease and recurrent vaso-occlusive crises

Abstract Introduction: Sickle cell disease (SCD) is a chronic, inherited blood disorder characterized by recurrent pain episodes, frequent hospitalizations, and multi-system complications. While the patient burden of SCD is well-documented, there is limited evidence describing the experiences of those providing informal care for patients with SCD. Caregivers of patients with SCD, particularly of those experiencing multiple vaso-occlusive crises (VOCs), play a crucial role, frequently supporting medical, emotional, and daily living needs over many years. This role can exact a considerable toll on caregivers' physical and mental health, financial stability, and quality of life. This study aims to evaluate the humanistic and economic impact of caregiving for patients living with SCD and recurrent VOCs across North America and Europe. Methods: A quantitative online survey collected self-reported demographic, clinical, and health-related quality of life data from caregivers of patients with SCD and recurrent VOCs living in the US, Canada, UK, France, Germany, Italy, Spain, and the Netherlands. The survey included the CarerQOL-7D and CarerQOL-VAS; the Zarit Burden Interview short form (ZBI-12); the Work Productivity and Activity Impairment (WPAI) questionnaire; and the EuroQol 5-Dimension 5-Level (EQ-5D-5L) and EQ-5D VAS. Caregiver characteristics, out-of-pocket expenses, and health inequity experiences were also evaluated. Participants were required to provide consent and meet the eligibility criteria of ≥18 years of age and caring for a patient with a SCD diagnosis experiencing ≥2 VOCs/year for the last two years that required interaction with a medical facility and pain medication or red blood cell transfusion. Descriptive analyses were conducted for caregiver-reported data and validated measures. Results across North America and Europe were pooled for reporting. Monetary values were standardized to U.S. dollars (USD) using contemporaneous exchange rates. Results: The survey was completed by 173 caregivers from North America (53.8%) and Europe (46.2%). Caregivers were predominately female (76.9%), married or living with a domestic partner (47.4%), with an average age of 40.5 (SD: 11.1) years. Most were employed (66.5%) with 53.8% working full-time. Caregivers had been providing care for an average of 12.4 (SD: 7.8) years, most commonly for a child or dependent young adult (mean patient age: 23.5 (SD: 15.0) years), with an average of 5.1 (SD: 3.2) VOCs in the past year and considerable clinical complications. CarerQOL-7D (mean 68.2; SD: 21.2) (0-100 scale; 100=best QOL) and CarerQOL-VAS (6.5; SD: 1.9) (0-10 scale; 10=best QOL) scores, along with the ZBI-12 burden score (17.0; SD: 8.6) (0-48 scale; 48=the most burden), indicate substantial quality of life impacts due to caregiver burden. EQ-5D-5L index (0.84; SD: 0.18) (0-1 scale; 1 = best QOL) and VAS (75.5; SD: 16.6) (0-100 scale; 100=best QOL) suggest moderate health status impacts. Among employed caregivers, WPAI outcomes indicated substantial work impacts: 20% (SD: 24%) absenteeism, 41% (SD: 29%) presenteeism, and 49% (SD: 30%) overall work impairment. On average, caregivers reported spending approximately 46 hours weekly on caregiving activities for the patient. Most caregivers (63.6%) reported distress due to financial burden and incurred out-of-pocket costs totaling $8,652 annually (US: $13,306; ex-US: $4,052) for caregiver-related expenses while the median annual income was just $36,600 (IQR: $22,000-$51,190). Frequent healthcare encounters amplified caregiver exposure to systemic challenges in the healthcare system. In the past year, patients averaged 5.0 (SD: 4.2) emergency department visits, 4.0 (SD: 4.5) hospitalizations, and 25.9 (SD: 38.1) inpatient days. Three-quarters of caregivers (74.0%) reported advocating for the patient to get needed care some or all of the time, with 46.8% reporting stigma related to pain medication requests. Conclusion: Caregivers of patients with SCD and recurrent VOCs experience a substantial impact on their health, quality of life, and productivity. These findings highlight the urgent need for tailored support systems, financial assistance, and healthcare policies that recognize and address the distinct challenges faced by caregivers of patients with SCD and recurrent VOCs. Moreover, future initiatives aimed at improving access to emerging therapies may help ease the substantial burden experienced by caregivers.

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