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

Abstract

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.

Administrative data identify sickle cell disease: A critical review of approaches in U.S. health services research

“…Some researchers use the presence of ≥1 claim in any setting with an SCD diagnosis code to identify putative SCD cases. [61][62][63][69][70][71][72][73] However, owing to the frequency of false-positive diagnoses in outpatient claims, that approach can lead to misclassification and result in underestimation of the use of services or costs among individuals with SCD. 20…”

In utero Therapy for the Treatment of Sickle Cell Disease: Taking Advantage of the Fetal Immune System

“…Although 63% of SCA patients in high-income countries receive hydroxyurea, due to lack of treatment guidelines, reduced clinical infrastructure and medication inaccessibility, this percentage can be as low as zero in under-resourced settings (McGann et al, 2016;Power-Hays and Ware, 2020). Treatment is also burdened by poor medication adherence, high patient discontinuation, or qualitative HbF level assessments every 3-6 months (Agrawal et al, 2014;Shah et al, 2019).…”

Primary caregiver decision‐making in hematopoietic cell transplantation and gene therapy for sickle cell disease

“…Hydroxyurea (HU), and the recently approved disease‐modifying therapies, including l ‐glutamine, 6 voxeltor, 7 crizanlizumab, 8 offer the possibility of long‐term amelioration of the disorder. Lack of access to high‐quality, comprehensive care, health disparities, and other sociodemographic factors all contribute to difficulties in adherence to, and the long‐term effectiveness of, these regimens 9 …”

“…Lack of access to high-quality, comprehensive care, health disparities, and other sociodemographic factors all contribute to difficulties in adherence to, and the long-term effectiveness of, these regimens. 9 …”

“…Lack of access to high-quality, comprehensive care, health disparities, and other sociodemographic factors all contribute to difficulties in adherence to, and the long-term effectiveness of, these regimens. 9 Hematopoietic stem cell transplantation (HCT) from allogeneic donors and, more recently, gene therapy (GT) with autologous genetically modified hematopoietic stem cells, offer the possibility of longterm disease amelioration. HCT/GT are associated with substantial risks of morbidity and mortality in the short term, as well as a risk of chronic, and even disabling, complications.…”