PF715 THE IMPACT OF CRIZANLIZUMAB ON VOC‐RELATED MEDICAL FACILITY VISITS

Abstract

Background:A major cause of morbidity among patients with sickle cell disease (SCD) is the intense, recurrent, unpredictable attacks, known as vaso‐occlusive crises (VOCs), which are responsible for >90% of hospital admissions in SCD. An important goal of pharmacological therapy is to prevent the occurrence of these painful episodes, and in turn, reduce the clinical and economic burden of the disease.In December 2018, the United States Food and Drug Administration granted crizanlizumab (SEG101) Breakthrough Therapy designation for the prevention of VOCs in patients with SCD. This designation was granted based on positive results from the Phase II SUSTAIN trial, in which 5 mg/kg crizanlizumab was associated with a 45.3% reduction in the median annual rate of VOCs leading to healthcare visits compared with placebo.Aims:The aim of the current analysis was to investigate the impact of crizanlizumab on VOC‐related medical facility visits.Methods:sing data from the SUSTAIN trial, analyses were conducted to determine the cumulative number of VOC events across all patients, medical facility visits and by medical facility type in the 5 mg/kg crizanlizumab and placebo arms. The total cumulative number of patient days in the study was considered in determining the event rate per year, which was calculated as follows:“[Total cumulative number of VOCs across all patients]/[Total number of days observed across all patients]” ∗ 365Incidence rate ratios (IRR) were derived using a Poisson regression model. Maximum likelihood parameter estimates were generated with standard errors to produce confidence intervals and p‐values. Effect modification over time by group was assessed by maximum likelihood.Results:The analyses showed a reduction in the overall rate of VOC events requiring a medical facility visit in the 5 mg/kg crizanlizumab group compared with the placebo group. Further examination of the impact of crizanlizumab on specific sites of care demonstrated a reduction in visits to outpatient care sites, largely driven by reduced visits to emergency care units and SCD crisis centers (Table 1). A trend to reduced inpatient admissions was also observed.Summary/Conclusion:Payers aim to reimburse treatments for SCD that reduce both the clinical and economic burden of the disease. In the SUSTAIN trial, patients treated with 5 mg/kg crizanlizumab experienced a 45.3% reduction in the median annual rate of VOCs leading to healthcare visits compared with those receiving placebo. The current analysis demonstrates that this reduction in VOCs leading to a healthcare visit was seen in most medical facility visits, largely driven by reduced visits to emergency care units and specialized SCD crisis centers. These findings suggest that patients treated with crizanlizumab will experience a substantial reduction in health care resource utilization and associated costs.image