Chemical information



Thrombopoietin is a naturally occurring colony-stimulating factor that regulates thrombopoiesis. Recombinant thrombopoietin, and a form of recombinant thrombopoietin conjugated with polyethylene glycol (pegacaristim, PEG-megakaryocyte growth and development factor, PEGrHuMGDF), are under investigation. They have been studied in the management of thrombocytopenia in patients receiving myelosuppressive or myeloablative chemotherapy, and in patients with myelodysplastic syndrome or HIV-associated thrombocytopenia. There is also some interest in the use of recombinant forms of thrombopoietin in stem cell mobilisation regimens and to increase platelet counts in healthy apheresis donors. However, some results have been disappointing and there are reports of neutralising antibody development.

General references.

Studies and reviews.
1. Vadhan-Raj S, et al. Safety and efficacy of transfusions of autologous cryopreserved platelets derived from recombinant human thrombopoietin to support chemotherapy-associated severe thrombocytopenia: a randomised cross-over study. Lancet 2002; 359: 2145–52
2. Nomura S, et al. Effects of pegylated recombinant human megakaryocyte growth and development factor in patients with idiopathic thrombocytopenic purpura. Blood 2002; 100: 728–30
3. Schuster MW, et al. The effects of pegylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF) on platelet recovery in breast cancer patients undergoing autologous bone marrow transplantation. Exp Hematol 2002; 30: 1044–50
4. Kuter DJ, Begley CG. Recombinant human thrombopoietin: basic biology and evaluation of clinical studies. Blood 2002; 100: 3457–69
5. Linker C, et al. Recombinant human thrombopoietin augments mobilization of peripheral blood progenitor cells for autologous transplantation. Biol Blood Marrow Transplant 2003; 9: 405–13
6. Vadhan-Raj S, et al. Importance of predosing of recombinant human thrombopoietin to reduce chemotherapy-induced early thrombocytopenia. J Clin Oncol 2003; 21: 3158–67
7. Geissler K, et al. Prior and concurrent administration of recombinant human megakaryocyte growth and development factor in patients receiving consolidation chemotherapy for de novo acute myeloid leukemia—a randomized, placebo-controlled, doubleblind safety and efficacy study. Ann Hematol 2003; 82: 677–83.
Published December 26, 2018.