Photopheresis and Hematopoietic Stem Cell Transplantation: Mechanisms and Clinical Application
Pitfalls in the Design of Clinical Trials for Prevention or Treatment of Acute Graft-versus-Host Disease

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Abstract

This review addresses pitfalls of clinical trials to evaluate new approaches for prevention or treatment of graft-versus-host disease. Determination of end points poses a difficult challenge in the design of clinical trials, and examples from previous studies are used to illustrate some of the pitfalls. Also discussed is the need for a new conceptual approach for evaluation of graft-versus-host disease after nonmyeloablative conditioning regimens, because the donor antirecipient alloimmune reaction is the primary mechanism of benefit with this type of treatment. Finally, investigators should be aware of regulatory and socioeconomic pitfalls that apply to all clinical trials.

Key words

Hematopoietic cell transplantation
Graft versus host disease
Clinical trials

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This research was supported by grants CA18029 from the Public Health Service, Department of Health and Human Services. Clinical trials comparing cyclosporine and tacrolimus for prevention of acute graft-versus-host disease were supported in part by grants from Fujisawa Healthcare.