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Treatment patterns and economic burden of sickle-cell disease patients prescribed hydroxyurea: a retrospective
Nirmish Shah1, Menaka Bhor2, Lin Xie3,4
1Duke University, Durham, NC, USA.
Insights
Sickle cell disease patients on hydroxyurea (HU) show poor adherence and high discontinuation rates. This leads to significant unmet needs and a substantial economic burden for treatment in the US.
Area of Science:
- Hematology
- Pharmacoeconomics
- Public Health
Background:
- Sickle cell disease (SCD) is a genetic blood disorder with significant health implications.
- Hydroxyurea (HU) is a primary treatment for SCD, aiming to reduce complications.
- Understanding treatment patterns and economic burden is crucial for optimizing SCD care.
Purpose of the Study:
- To evaluate treatment patterns of hydroxyurea (HU) in US patients with sickle cell disease (SCD).
- To assess the economic burden associated with HU treatment for SCD.
- To identify unmet needs in SCD management.
Main Methods:
- Retrospective analysis of Medicaid Analytic Extracts (MAX) claims data from 2009-2013.
- Inclusion criteria: SCD patients with HU prescription, continuous enrollment for 6 months pre- and 12 months post-index date.
- Analysis of demographics, comorbidities, HU treatment patterns (adherence, discontinuation), healthcare utilization, and costs.
Main Results:
- 3999 SCD patients prescribed HU were analyzed; mean age 19.24 years, 73.3% African American.
- High HU discontinuation rate (58.9%) and low medication possession ratio (MPR) (mean 0.52, only 22.3% with MPR ≥80%).
- High SCD-related healthcare utilization and costs, with mean annual total costs of $27,779 per patient, predominantly inpatient costs.
Conclusions:
- Patients with SCD prescribed HU experience significant unmet needs.
- Poor medication adherence and high treatment discontinuation rates are prevalent.
- The economic burden of SCD remains substantial, highlighting the need for improved management strategies.
Background:
This study aimed to evaluate sickle-cell disease (SCD) treatment patterns and economic burden among patients prescribed hydroxyurea (HU) in the US, through claims data.
Methods:
SCD patients with pharmacy claims for HU were selected from the Medicaid Analytic Extracts (MAX) from January 1, 2009 - December 31, 2013. The first HU prescription during the identification period was defined as the index date and patients were required to have had continuous medical and pharmacy benefits for ≥6 months baseline and 12 months follow-up periods. Patient demographics, clinical characteristics, treatment patterns, health care utilization, and costs were examined, and variables were analyzed descriptively.
Results:
A total of 3999 SCD patients prescribed HU were included; the mean age was 19.24 years, most patients were African American (73.3%), and the mean Charlson comorbidity index (CCI) score was 0.6. Asthma (20.3%), acute chest syndrome (15.6%), and infectious and parasitic diseases (20%) were the most prevalent comorbidities. During the 12-month follow-up period, 58.9% (N = 2357) of patients discontinued HU medication. The mean medication possession ratio (MPR) was 0.52, and 22.3% of patients had MPR ≥80%. The average length of stay (LOS) for SCD-related hospitalization was 13.35 days; 64% of patients had ≥1 SCD-related hospitalization. The mean annual total SCD-related costs per patient were $27,779, mostly inpatient costs ($20,128).
Conclusions:
Overall, the study showed the patients had significant unmet needs manifest as poor medication adherence, high treatment discontinuation rates, and high economic burden.
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