PRO102 CHARACTERISTICS AND OUTCOMES OF PEDIATRIC HEMOPHILIA-A PATIENTS IN THE INPATIENT SETTING

Abstract

Hemophilia-A is a rare X-linked genetic blood disorder that deforms the factor VIII clotting protein and reduces the ability for blood to clot. Hemophilia-A affects 1 in 5,000 live male births, with an estimated 20,000 patients living in the United States. There are multiple clinical trials assessing a gene therapy to treat Hemophilia-A. The goal of this study was to characterize pediatric patients with Hemophilia-A and compare them to a simple random sample (SRS) of hospital discharges to characterize the treatable patient population for new gene therapy treatments. The 2016 AHRQ Kids Inpatient Database was used to identify any patient discharge with a primary ICD-10 CM diagnosis of ‘D66’. Hemophilia-A discharges were compared to a SRS of 1000 discharges for patients between the ages of 1 and 20. The two populations were compared on clinical and economic characteristics using t-tests for continuous variables and chi-square for categorical variables. Overall, 468 pediatric Hemophilia-A discharges were analyzed. The average age of hemophilia patients was significantly lower than the SRS (Hem 6.86, SD 6.53 vs SRS 12.85, SD 6.56 p<0.0001). Payer representation was significantly different between the two groups. Specifically, the hemophilia group had a significantly higher rate of Medicaid participation (Hem 62.82% vs SRS 50.8% p<0.0001). Median length of stay was not significantly different between the two groups (Hem 2.5 days vs SRS 3 days p=0.65) however, median charges were significantly different (Hem $57,804 vs SRS $18,731; p<0.0001). Patients with Hemophilia-A do have some differences with the general pediatric inpatient population. Hemophilia patients are younger and require significantly more resources over the same length of stay with a median charge difference of $16,878 per day. This increase in resource utilization is likely tied to the burden of the disease as four of the top five secondary diagnoses were directly associated with Hemophilia.