The Multidimensional Burden of Sickle Cell Disease on Quality of Life and the Impact of Treatment Adherence on Outcomes: Insights from a Real-World Study
Authors: Hey Felix , Wright Devon , Kyle Aine , Levent Ayse 
Published: December 2024

Background:
Sickle cell disease (SCD) is a chronic, debilitating blood disorder that significantly impacts patients' quality of life (QoL). Patients with SCD experience a range of symptoms that contribute to decreased QoL, including pain, fatigue, and acute complications such as vaso-occlusive crises (VOCs) and acute chest syndrome (ACS).
Aims:
This study aims to identify the most prevalent symptoms affecting QoL in SCD patients and to evaluate the impact of treatment adherence on improving QoL outcomes in real-world clinical practice.
Methods:
The Ipsos Sickle Cell Disease Monitor is a US-based quantitative online survey conducted bi-annually among 75 physicians specialized in treating patients with SCD (and pediatric hematologists and hematology oncologists). Physicians were screened for duration of practice in their specialty and SCD patient caseload. The study collected data from 7,474 SCD adult and adolescent patient records (aged 12+ years) and 967 pediatric SCD patient records (aged 4-11 years) from participating physicians from January 2020 - April 2024. Patient data was analyzed to determine the prevalence of key SCD symptoms and the effect of treatment on QoL measures, including fatigue, pain, and school/work absenteeism. Treatment adherence rates and reasons for non-adherence were also assessed.
Results:
Data from reported patient record forms show that fatigue and vaso-occlusive crises (VOCs) represent the top symptoms experienced by patients due to their SCD. Among the 12+ age group, 33% experience fatigue vs. 27% among the 4-11 age group. Among the 12+ age group, 24% experience VOCs vs. 30% among the 4-11 age group.
Treatment adherence rates were suboptimal, with 36% of patients aged 12+ years and 42% of patients aged 4-11 years reported as almost never missing a dose. In the 12+ age group, those patients adherent to treatment 90-100% of the time experienced significantly fewer VOCs (21% vs. 28%, p&lt;0.05), less fatigue (73% vs. 62%, p&lt;0.05), and were more likely not to exhibit symptoms (38% vs. 23%, p&lt;0.05) compared to non-adherent patients. Similarly, in the 4-11 age group, adherent patients had significantly less pain (76% vs. 63%, p&lt;0.05) and more were more likely not to exhibit symptoms (52% vs. 37%, p&lt;0.05) compared to non-adherent patients. The most common reasons for non-adherence were forgetfulness (30% in 12+ years; 51% in 4-11 years) and lack of a support system (28% in 12+ years; 36% in 4-11 years).
Summary/Conclusion:
This study highlights the impact of SCD symptoms on patients' QoL and the crucial role of treatment adherence in improving QoL outcomes. While treatment initiation led to reductions in symptoms overall, adherence rates remained suboptimal, limiting the potential benefits of therapy and the degree of effects on QoL. Forgetfulness and lack of support were identified as key barriers to adherence, underscoring the need for comprehensive patient support programs and personalized strategies to improve treatment compliance. By prioritizing adherence and addressing the unique challenges faced by SCD patients, healthcare providers can optimize QoL outcomes and reduce the overall burden of this chronic condition. Further research using comparator data is warranted.