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Real-World Data of Crizanlizumab in Sickle Cell Disease: A Single-Center Analysis
Halle Cheplowitz1, Shanna Block1, Jessica Groesbeck2
1Department of Pharmacy, University of California San Diego Health, San Diego, CA, USA.
Insights
Crizanlizumab may reduce hospital visits for sickle cell disease (SCD) patients, especially frequent users. However, high discontinuation rates necessitate further study on its real-world effectiveness and barriers to sustained use.
Area of Science:
- Hematology
- Pharmacology
- Public Health
Background:
- Crizanlizumab, approved in 2019, targets vaso-occlusive events (VOEs) in sickle cell disease (SCD).
- Real-world data on crizanlizumab's effectiveness and usage patterns are limited.
- This study aimed to analyze crizanlizumab prescription patterns, benefits, and barriers in an SCD program.
Purpose of the Study:
- To evaluate the real-world utilization of crizanlizumab in a sickle cell disease (SCD) program.
- To assess the impact of crizanlizumab on acute care utilization among SCD patients.
- To identify barriers and discontinuation reasons for crizanlizumab therapy.
Main Methods:
- Retrospective analysis of 15 patients receiving crizanlizumab between July 2020 and January 2022.
- Comparison of acute care visits (emergency department, day infusion program) before and after crizanlizumab initiation.
- Assessment of treatment adherence, discontinuation rates, and reasons for discontinuation.
Main Results:
- A non-significant decrease in average acute care visits post-crizanlizumab initiation (20 vs. 10, P=0.07).
- A statistically significant reduction in acute care visits among high hospital service users (40 vs. 16, P=0.005).
- A high discontinuation rate, with only 5 of 15 patients remaining on therapy at 6 months.
Conclusions:
- Crizanlizumab may reduce acute care visits in SCD, particularly for high-utilizing patients.
- High discontinuation rates observed in this cohort warrant further investigation.
- Additional research in larger cohorts is needed to confirm efficacy and understand barriers to sustained crizanlizumab use.
Background:
Crizanlizumab was approved by the United States Food and Drug Administration agency in 2019 for decreasing vaso-occlusive events (VOEs) in sickle cell disease (SCD). Data regarding the use of crizanlizumab in the real-world setting are limited. Our goal was to identify patterns of crizanlizumab prescriptions in our SCD program and evaluate the benefits and identify barriers to its use in our SCD clinic.
Methods:
We conducted a retrospective analysis of patients who received crizanlizumab at our institution between July 2020 and January 2022. We compared acute care usage patterns before and after initiation of crizanlizumab, adherence to treatment, discontinuation and reasons for discontinuation. High utilizers of hospital-based services were defined as those with more than one visit to the emergency department (ED) per month or more than three visits to the day infusion program per month.
Results:
Fifteen patients received at least one dose of crizanlizumab 5 mg/kg of actual body weight during the study period. The average number of acute care visits decreased following crizanlizumab initiation but was not statistically significant (20 visits vs. 10 visits, P = 0.07). Among high users of hospital-based services, the average number of acute care visits decreased after initiation of crizanlizumab (40 vs. 16, P = 0.005). Only five patients included in this study remained on crizanlizumab 6 months after initiation.
Conclusion:
Our study suggests that crizanlizumab use may be helpful in decreasing acute care visits in SCD, particularly among high utilizers of hospital-based acute care services. However, the discontinuation rate in our cohort was extremely high, and further evaluation of efficacy and causes contributing to discontinuation in larger cohorts is warranted.
