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DISCUSSION PAPER: BASIC SCIENCE AND PRACTICAL MANAGEMENT

There is a continued dichotomy between basic research and clinical management in a variety of diseased states. The rapidity with which basic concepts change makes it impossible to rely on information learned during basic medical training. In addition, when therapy is semiempirical, clearly improved modes of therapy can be devised. However, it is rare that basic scientists and practical clinicians talk to each other to the degree that a mutually useful understanding is reached. Rather, at best the clinician is impressed by the need for further development in research. Consequently, he attempts to undertake that job himself. The result is clinical research to answer simple management problems in such a way that the answers are reliable and scientifically sound. Far too often the clinical protocols are designed to answer questions that do not need answering by the time the protocol is brought to completion. The need of the knowledge of basic research, and the ability to extrapolate into the future on the basis of such knowledge, is greater than ever to avoid obsolescence in the design of improved clinical management before the experiment is begun. This paper is meant to describe some reflections on biochemical concepts in relation to the management of hemophilia. There exists at the present time sufficient information about the basic biochemistry of this disease that fragments of knowledge exist, which appear enticing enough to be promoted to the role of the foundation on which a clinical interpretation can be based. Yet there are enough analogies in our knowledge of basic biochemistry to allow US to remain appropriately critical of individual findings and to avoid promoting them to fundamental axioms when they are only correlaries.