Hemophilia is a rare lifelong condition which can be potentially life-threatening. Parents bare a significant responsibility for delivery of medical care because the treatment for hemophilia begins early in life for children within the home setting. As a result, parents frequently exhibit a heightened level of stress, anxiety, and subsequent trauma around the acceptance of the illness and the administration of medication management. To address the multifaceted nature of chronic illness for patients and their families, the ideal treatment utilizes a multidisciplinary team. Our proposed 3P Patient Parent Power Program aims to standardize care for families with patients of hemophilia using a tiered approach of psychosocial support. The necessary level of support will be provided to parents in order for them to successfully provide in-home prophylactic factor treatment. The goal of the program is to reduce parental stress and anxiety related to this chronic illness and increase feelings of empowerment for the parent and child.
Objective:
To evaluate changes in healthcare resource utilization and haemophilia related events among patients diagnosed with haemophilia A between 2008 and 2012.
Methods:
This retrospective study analyzed data from the Humedica de-identified electronic medical record database between January 2008 and December 2012. Male patients diagnosed with hemophilia A (ICD-9-CM 286.0) receiving treatment with a clotting factor were eligible if they 1) were ≥18 years of age 2) did not receive Factor IX therapy and 3) did not have a diagnosis of Von Willebrand while receiving factor VIII therapy containing von Willebrand factor. All patient level resource utilization was converted to utilization per patient year. Resource utilization was then compared across time periods using repeated measures analysis of variance (ANOVA). The annualized number of haemophilia related events (haemophilic arthropathy or other joint related events) was calculated for each year. McNemar’s chi-square test was used to compare the frequencies across years.
Summary:
136 patients contributing 375 patient-years were included in this study. Office/clinic visits accounted for the majority of healthcare encounters annually; 7.5 all-cause visits per year and 2.2 haemophilia related visits per year. The number of annual all-cause office/clinic visits for Haemophilia A patients decreased significantly over time from 12.5 visits in 2008 to 5.9 visits in 2012 (p=0.0404), while haemophilia A-specific annual visits decreased from 4.0 to 1.5 (p=0.1991) during the same period. On average haemophilia A patients had less than 1 inpatient and emergency room visits per year, which did not change significantly over time (p=0.6371 and p=0.4845, respectively). Over the 5-year period, haemophilic events occurred in 30.93% of patient years, changed from 23.81% in 2009 to 34.09% in 2011 (p=0.6658).
Conclusions:
Office/clinic outpatient visits among patients diagnosed with haemophilia A has decreased overtime. However, the rate of haemophilia related arthropathies and other associated events have remained high. Further analysis is needed to understand how to best manage patients diagnosed with haemophilia A and reduce the proportion of patients who develop reduced joint mobility due to bleeding into joints.
Objectives:
Factor VIII (FVIII) prophylaxis regimens for severe hemophilia A that allow more flexible dosing than the standard 3-times-weekly regimen while maintaining efficacy may improve adherence. This analysis compared the clinical efficacy of once- or twice-weekly versus ≥3-times-weekly prophylaxis dosing of Bayer’s sucrose-formulated recombinant FVIII (rFVIII-FS) in patients with severe hemophilia A.
Methods:
Data from 3 postmarketing studies were pooled. Patients with severe hemophilia A and no history of inhibitors who were receiving ≥1 prophylaxis infusion/wk of rFVIII-FS for ≥80% of a prophylaxis observation period (≥5 months) were included. Patients were categorized based on age (<18 and ≥18 years) and physician-assigned treatment regimens of 1–2 prophylaxis injections/wk (n=63) or ≥3 prophylaxis injections/wk (n=76). Descriptive statistics were determined for annualized bleeding rates (ABRs) by dosing group and age subgroups.
Summary:
Median (quartile 1; quartile 3) ABR for all bleeds was 2.0 (0; 4.0) in the group receiving 1–2 prophylaxis injections/wk and 3.9 (1.5; 9.3) in the group with ≥3 prophylaxis injections/wk. Similarly, median ABRs for joint, spontaneous, and trauma-related bleeds were numerically lower in the group receiving 1–2 prophylaxis injections/wk. The trend toward lower ABRs in the group with 1–2 prophylaxis injections/wk was observed in both age subgroups, although ABRs were somewhat higher in patients ≥18 vs <18 years. Zero annualized bleeds were reported by 30% and 7% of patients in the groups with 1–2 prophylaxis injections/wk and ≥3 prophylaxis injections/wk, respectively.
Conclusions:
These data demonstrate that bleeding control can be achieved in some patients with severe hemophilia A using a <3-times-weekly prophylaxis dosing regimen and that physicians’ judgment based on bleeding phenotype can successfully direct the frequency of prophylactic dosing.
