Economic Burden of High-Responding Inhibitors in Patients with Hemophilia A in Taiwan

PURPOSE: Hemophilia A (HA) is the most common X-linked inherited bleeding disorder. In some patients with HA, particularly those with severe HA, replacement therapy results in the production of high-responding clotting factor VIII inhibitors. The economic burden of this complication is the highest reported for a chronic disease. Our aim was to investigate the direct medical expenditure burden of high-responding inhibitors in patients with HA. MATERIALS AND METHODS: A retrospective study was conducted using the National Health Insurance Research Database, utilizing data covering the period of 2004-2007. RESULTS: In total, 638 males with HA, including 37 patients with high-responding inhibitors were evaluated. Over 99% of the annual median medical expenditure was attributable to the cost of clotting factor concentrates (CFCs) in patients with high-responding inhibitors. The annual median expenditure related to CFCs of the total medical care and outpatient care were US$170611 and US$141982, respectively, and were 4.6- and 4.3-fold higher in these patients during the study period, respectively. In patients with high-responding inhibitors, the median hospitalization expenditure and daily hospitalization cost with or without surgical procedures were 3.0- and 2.4-fold higher, respectively, and 4.3 and 5.6-fold higher, respectively. CONCLUSION: Our data reveal higher medical expenditures burden for patients with HA and high-responding inhibitors in Taiwan. Future research is encouraged to evaluate the impact of this burden on patient quality of life.

Successful Treatment of Infantile Fibrosarcoma Spinal Metastasis by Chemotherapy and Stereotactic Hypofractionated Radiotherapy

We report a case of infantile fibrosarcoma in an 8-month-old boy manifested as a right-sided lower leg mass. Repeated local recurrence and distant metastasis were noted during the following three-year period. Whole body fluoro-deoxyglucose positron emission tomography scan revealed an asymptomatic metastasis involving the fourth lumbar vertebrae. The patient received chemotherapy (VAC regimen) with Cyberknife(R) stereotactic hypofractionated radiotherapy (26 Gy; 4 fractions). This treatment reduced tumor size by 23% without acute radiation toxicity even after 33 months. This case suggests that combining chemotherapy and this form of radiotherapy may be safe and effective against childhood spinal metastasis.