Cost‐effectiveness of hydroxyurea in reducing the frequency of pain episodes and hospitalization in pediatric sickle cell disease

Abstract

In a cohort of children with sickle cell disease (SCD) and vaso-occlusive pain visits served through South Carolina's Medicaid system over a 6-year period (N 5 523), we compared the number of vaso-occlusive pain or acute chest syndrome (ACS)/pneumonia episodes, and outpatient or acute service costs in those treated or not treated with hydroxyurea (HU). HU may be an underused intervention for SCD in this practice setting, for a variety of reasons. Treatment with HU varied greatly, appears to have been administered to more severely ill children, but was associated with a reduction in vaso-occlusive pain episodes, hospitalizations, and total costs of care within the HU cohort during a 2-3 year period of active HU treatment. Those receiving care through specialized SCD clinics were less likely to have pain or acute care episodes (RR 5 0.79, P < 0.0001; RR 5 0.90, P 5 0.01). Compared with the non-HU cohort, the HU group evinced a significantly higher risk of experiencing vaso-occlusive pain episodes (RR 5 3.32, P < 0.0001) and ACS/pneumonia episodes (RR 5 2.66, P < 0.0001), and higher outpatient, inpatient/emergency, and total service costs (RR 5 1.85, 2.11, 2.10, and P < 0.0001, respectively) over time. HU is clinically effective in reducing pain episodes, hospitalizations, and total care costs, but those receiving it might be more severely ill.Erythrocytes in children with sickle cell disease (SCD) become deoxygenated, dehydrated, and crescent-shaped, and tend to aggregate or stick to blood vessel walls, blocking blood flow within limbs and organs, causing painful episodes. These patients are frequently seen in emergency departments and hospitalized for these severe pain episodes [1]. SCD poses an enormous personal burden to these young patients and is also costly to the family and third-party payer, especially for low-income children [2]. Interventions designed to control vaso-occlusive pain episodes, and avoid hospitalizations may reduce the significant personal and economic burdens of the disease [3,4].Hydroxyurea (HU), a myelosuppressive agent, which raises the levels of Hb F [5] and of hemoglobin [6][7][8], effectively decreases the rate of painful vaso-occlusive and acute chest syndrome (ACS) episodes by 50% in adults [9][10][11][12], and is generally safe and well-tolerated in children older than 5 years of age [13]. There is strong evidence that HU reduces the frequency of hospitalization in children with SCD and moderate evidence that it decreases the frequency of painful crises [14]. Given the short-term safety profile of HU in children and its established efficacy in adults, HU is commonly used, off-label, in children with multiple painful episodes (3 per year) to reduce episode frequency and acute services utilization [15][16][17], even in children as young as 9 months.Results from a multicenter study of HU in sickle-cell anemia demonstrated that adult patients treated with HU had a 44% decrease in hospitalizations compared with those taking placebo, which accounted for the majority of cost savings in ...

Hydroxycarbamide treatment in sickle cell disease: estimates of possible leukaemia risk and of hospitalization survival benefit

“…Physicians appear to prescribe HC primarily to SCD patients with high vaso-occulsive severity, as epidemiological studies show an association of HC use with more frequent hospitalizations (Lanzkron et al, 2006;Stallworth et al, 2010). Our results suggest that the same was true in the patient sample we analysed.…”

Clinical complications in severe pediatric sickle cell disease and the impact of hydroxyurea

“…Ourfindings are consistent with previous findings. Under usual-care conditions, 2-3 years of HU use is safe and effective in children and adolescents [27].…”

Hydroxyurea Is Associated With Lower Costs of Care of Young Children With Sickle Cell Anemia

“…Another study examined the cost and effectiveness of hydroxyurea in reducing the frequency of pain episodes and hospitalization in pediatric sickle cell disease in South Carolina. 12 Analysis of that state's Medicaid records indicated that those receiving the drug had a higher risk of experiencing vaso-occlusive pain episodes and ACS, as well as significantly higher medical costs than those children not receiving hydroxyurea, which was attributed to the hydroxyurea users having greater severity of disease. However, among those receiving hydroxyurea, average costs fell 31% from $12 842 to $8839 (P , .001) during the 2-3 year period of active treatment.…”

“…15,16 Although the MarketScan database does not identify the 12 states contributing data, those data may be more generalizable at the national level than cost estimates from individual states used in previous cost studies. 11,12,20 Of note, the claims data used to estimate hospitalization costs lacked sociodemographic information except for age (which was controlled in this analysis by restriction to 1-to 3-yearold children), gender, and, to a limited extent, race/ethnicity. 14 Another limitation of those data are the use of ICD-9 billing codes to identify children with sickle cell anemia; previous analyses of these data have found that the overall frequency of sickle cell disease among children reported to be black or African American is accurate but that information on subtypes of sickle cell disease is often lacking or imprecisely reported.…”

“…Previously published analyses of observational data have yielded mixed results regarding relative medical costs among patients using hydroxyurea, which may reflect differences in disease severity. [10][11][12] Because the BABY HUG study was a blinded randomized trial, it provided a unique opportunity to assess differences in medical costs, while controlling for differences in disease severity.…”