Treatment patterns and economic burden of sickle-cell disease patients prescribed hydroxyurea: a retrospective claims-based study

Nirmish Shah,Lin Xie,Jincy Paulose,Huseyin Yuce,Rashid Halloway,Steve Arcona,Menaka Bhor

doi:10.1186/s12955-019-1225-7

Abstract

BackgroundThis study aimed to evaluate sickle-cell disease (SCD) treatment patterns and economic burden among patients prescribed hydroxyurea (HU) in the US, through claims data.MethodsSCD 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.ResultsA 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).ConclusionsOverall, the study showed the patients had significant unmet needs manifest as poor medication adherence, high treatment discontinuation rates, and high economic burden.

Highlights

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
A 5-year study carried out using Florida Medicaid program data estimated that SCD patients had an average of 3.7 inpatient hospitalizations and 24.1 hospital days during the study period, with roughly 84% attributable to SCD-related diagnoses [16]
The FDA approved oral L-glutamine therapy for SCD patients aged 5 years and older to reduce the number of acute complications associated with the blood disorder [27]; while this medication’s mechanism of action (MOA) is not well understood, it is believed to result from a reduction of intracellular oxidation damage [28]

Summary

Introduction

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. Other MOAs include increasing the size of the reticulocytes and improving cellular deformability, which increases blood flow and reduces vaso-occlusion and its associated complications. Studies have shown that HU can be used as a substitute for chronic transfusions for the prevention of primary stroke among high-risk SCD pediatric patients with abnormal transcranial doppler (TCD) flow velocity [23, 24]. The FDA approved oral L-glutamine therapy for SCD patients aged 5 years and older to reduce the number of acute complications associated with the blood disorder [27]; while this medication’s MOA is not well understood, it is believed to result from a reduction of intracellular oxidation damage [28]. Adverse events associated with HU include GI symptoms such as loss of appetite, nausea, constipation and diarrhea; other symptoms include infection and bleeding [19]

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