Stephens Jennifer M, Joshi Ashish V, Sumner Michael, Botteman Marc F
Pharmerit North America LLC, 7272 Wisconsin Ave, Suite 300, Bethesda, MD 20814-4858, USA.
Expert Opin Pharmacother. 2007 Jun;8(8):1127-36. doi: 10.1517/14656566.8.8.1127.
Severe hemophilia with inhibitors is a rare disease with substantial clinical, humanistic and economic consequences. This review provides an overview of the role of recombinant activated factor VII (rFVIIa) versus plasma-derived bypassing agents for hemophilia with inhibitors and summarizes the 13 formal economic analyses (6 burden of illness and 7 comparative studies) that have been published in this indication. The findings suggest that the economic impact of rFVIIa has occurred primarily during hospitalization to manage major bleeding episodes and to allow for elective orthopedic surgeries that would not have been attempted prior to rFVIIa. Comparative analyses for on-demand treatment suggest that the total cost of treating a bleeding episode with rFVIIa may be lower than with plasma-based agents due to faster bleeding resolution, higher initial efficacy rates and avoidance of second and third lines of treatment.
伴有抑制物的重度血友病是一种罕见疾病，会产生重大的临床、人文和经济后果。本综述概述了重组活化凝血因子VII（rFVIIa）与血浆源性旁路制剂在伴有抑制物的血友病治疗中的作用，并总结了已发表的针对该适应症的13项正式经济分析（6项疾病负担分析和7项比较研究）。研究结果表明，rFVIIa的经济影响主要发生在住院期间，用于治疗严重出血事件以及开展择期骨科手术，而在rFVIIa出现之前这些手术是不会尝试的。按需治疗的比较分析表明，由于出血缓解更快、初始有效率更高以及避免二线和三线治疗，使用rFVIIa治疗出血事件的总成本可能低于使用血浆制剂。
