Milestones in Hemophilia and Concepts in Future Clinical Trial Design

Hemophilia has long been documented as a bleeding disorder that afflicts males from early childhood. While some early societies set guidelines or laws to protect affected children, true advances in the understanding of the underlying deficiency and in the treatment of hemophilia have been relatively recent and continue today. This paper presents some of the key milestones that mark the path to current knowledge on hemophilia. Given that further studies are needed to explore issues such as optimal treatment and dosage, particularly in patients with inhibitors to factor VIII or factor IX, this paper also considers potential pitfalls in the design and conduct of clinical trials in hemophilia and suggests how careful forward planning may help to avoid them.