1B-02 - Prevalence and Event Rates of Acute Events and Prevalence of Long-term Organ Damage in Individuals with Sickle Cell Disease – A Retrospective Cohort Study Using Medicaid Claims Database

Background: Sickle cell disease (SCD) is a common hereditary red blood cell disorder. SCD may cause acute events and long-term end organ damage. Updated evidence on these acute events and long-term organ damage rates is important for characterizing disease progression, particularly from Medicaid claims where many US SCD patients are enrolled.

Objectives: The objective of this study was to assess prevalence and event rates of acute events associated with SCD as well as prevalence of long-term organ damage using recent US claims data.

Methods: This is a non-interventional, retrospective study using Medicaid claims data from January 2016 to December 2021. Patients were included if they had ≥1 inpatient or 2 outpatient SCD ICD-10 codes (all D57 except for D57.3) at least 30 days apart. Follow up for SCD related events (identified by diagnosis codes) started at the index date (first documented SCD diagnosis date) and continued until a patient was censored (i.e., lost database activity). For SCD acute events, period prevalence (patients with events/total patients) and event rates (total number of acute events among all patients/total person-time at-risk) were calculated. For SCD long-term organ damage, period prevalence was calculated. All rates were stratified by SCD genotype, age group, and sex. Healthcare resource utilization burden was also assessed.

Results: A total of 75,343 SCD patients were included in this study, of which the median age was 23.1 years (range 0-103.7), 59% were women, 87% were non-Hispanic Black among those with reported race. The period prevalence for selected SCD acute events were acute chest syndrome [(ACS), 24.5%], seizure (8.4%), ischemic stroke (6.7%); period prevalence for long-term organ damage were neurocognitive deficits (13%), heart failure (9.6%), CKD (6.9%). The event rates (per 100 person-years) for SCD acute events were: ACS (27.0), seizure (21.0), ischemic stroke (12.4). Both acute events and long-term organ damage were more frequent and/or prevalent among patients with Hb-SS genotype and adults. Among the total SCD population, the median visits per patient year was 26.5 for outpatient, 2.1 for emergency, 1.4 for inpatient, and the median length of inpatient stays was 3.5 days.

Conclusions: This is one of the largest studies using RWD providing the most recent SCD acute event and long-term organ damage rates. This study highlights the significant burden of acute events and long-term organ damage in US SCD patients on Medicaid, and demonstrates the critical need for improved therapeutic strategies to alleviate complications and enhance the quality of life for individuals with SCD. These results may serve as an epidemiologic reference for future SCD research and drug development.