Haematopoietic Stem and Progenitor Cell-targeted Therapies for Thrombocytopenia

This review discusses the present outlook for new thrombocytopenia therapies that induce haematopoietic stem and progenitor cells to proliferate, differentiate and produce functional platelets. A brief overview of megakaryopoiesis and its regulation by thrombopoietin (TPO) is followed by a discussion of how early experience with recombinant TPO therapies stimulated the search for novel TPO receptor ligands. A summary is then provided of the results of Phase I clinical trials with the new small molecule and peptide TPO mimetics that are in development at present. Finally, recent developments in the ex vivo expansion of primitive haematopoietic cells and the potential enhancement of cell-based therapies by haematopoietic growth factors in vivo are briefly summarised as part of a look towards the future.