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- Title
Use of Disease-Modifying Treatments in Patients With Sickle Cell Disease.
- Authors
Newman, Terri Victoria; Yang, Jingye; Suh, Kangho; Jonassaint, Charles R.; Kane-Gill, Sandra L.; Novelli, Enrico M.
- Abstract
Key Points: Question: How has the uptake of disease-modifying treatments (DMTs) evolved from 2014 to 2021 with the introduction of new drug treatments for sickle cell disease (SCD), and what characteristics can be observed across different treatment use groups? Findings: In this cross-sectional study, a sample of 5022 adults and children with SCD found that inconsistent DMT users had a higher prevalence of severe SCD complications and more frequent health care visits, whereas non-DMT users were generally older with milder symptoms. In a second sample of 6387 participants, analysis of annual uptake of DMTs found a modest increase in DMT use from 2014 to 2021; notably, nearly 3 of 4 individuals with SCD did not receive any DMTs. Meaning: These findings suggest that uptake of DMTs in SCD is low; interventions to increase DMT uptake need to consider patient characteristics. This cross-sectional study describes patient characteristics associated with use of disease-modifying treatments and evaluates the observed annual patterns of individual and composite use among adults and children with sickle cell disease. Importance: Despite hydroxyurea being an established treatment for sickle cell disease (SCD), it remains underused. The recent approval of the disease-modifying treatments (DMTs) l-glutamine, crizanlizumab, and voxelotor underscores the need to understand the uptake of DMTs in the current treatment landscape. Objective: To explore characteristics that may be associated with DMT use and to describe observed patterns of yearly DMT use from 2014 to 2021. Design, Setting, and Participants: This cross-sectional study used administrative claims data from Optum's deidentified Clinformatics Data Mart Database from January 1, 2014, to September 30, 2021, to identify adults and children with SCD. Data were analyzed from August 1, 2022, to August 28, 2023. Exposure: Use of DMTs. Main Outcomes and Measures: Patient characteristics across groups with varying patterns of DMT use and yearly patterns of prescription fills for hydroxyurea, crizanlizumab, voxelotor, and l-glutamine. Results: A total of 5022 beneficiaries with SCD (2081 [41.4%] aged 18-45 years; 2929 [58.3%] female) were included in sample A (144 [2.9%] inconsistent users, 274 [5.5%] incident users, 892 [17.8%] consistent users, and 3712 [73.9%] non-DMT users). Inconsistent users had a higher prevalence of vaso-occlusive crises (mean [SD], 3.7 [4.7]), splenic complications (6 of 144 [4.2%]), pulmonary complications (36 of 144 [25.0%]), kidney disease (21 of 144 [14.6%]), acute chest syndrome (18 of 144 [12.5%]), and health care visits (eg, mean [SD] inpatient visits, 7.0 [10.7]) compared with the other use groups. Non-DMT users had the lowest prevalence of vaso-occlusive crises (mean [SD], 0.8 [2.4]), acute chest syndrome (109 of 3712 [2.9%]), and inpatient (mean [SD], 2.0 [6.6]) and emergency department (mean [SD], 0.7 [3.1]) visits and the highest proportion of adults 65 years and older (593 of 3712 [16.0%]). In sample B (6387 beneficiaries with SCD), hydroxyurea use modestly increased from 428 of 2188 participants (19.6%) in 2014 to 701 of 2880 (24.3%) in 2021. Use of l-glutamine increased briefly but gradually decreased throughout the study period. In 2021, out of 2880 participants, 102 (3.5%) had at least 1 fill for crizanlizumab and 131 (4.6%) had at least 1 fill for voxelotor. Overall, total DMT use increased from 428 of 2188 participants (19.6%) in 2014 to 815 of 2880 patients (28.3%) in 2021. Conclusions and Relevance: In this cross-sectional analysis of adults and children with SCD, uptake of DMTs remained low from 2014 to 2021, despite the approval of newer therapies. Notable differences in patient characteristics across varied DMT exposure types necessitate further exploration into factors that facilitate DMT use and the creation of strategies to enhance DMT uptake.
- Subjects
DRUG therapy for sickle cell anemia; ANTISICKLING agents; CROSS-sectional method; HYDROXYUREA; RETROSPECTIVE studies; MONOCLONAL antibodies; DESCRIPTIVE statistics; DATA analysis software; GLUTAMINE; DISEASE management
- Publication
JAMA Network Open, 2023, Vol 6, Issue 11, pe2344546
- ISSN
2574-3805
- Publication type
Article
- DOI
10.1001/jamanetworkopen.2023.44546