The treatment of immune thrombocytopenia (ITP)—focus on thrombopoietin receptor
agonists
Davia J. Kuter
Hematology Division, Massachusetts General Hospital, Harvard Medical School, Boston, Massachusetts, USA
Correspondence to: Professor David J. Kuter, MD, DPhil, Hematology Division, Massachusetts General Hospital, Ste. 118, Room 110, Zero Emerson
Placa, Boston, MA 02114, USA. Email: dkuter@mgh harvard.edu
Abstract: Immune thrombocytopenia (/TP) is an autoimmune disease characterized by increased platelet destruction along with reduced
platelet production. All treatments attempt ether to reduce the rate of platelet production or increase the rate of platelet production. There is
no known cure but most patients attain 2 hemostatic platelet count New tieatment guidelines rave supported a shift trom corticosteroids
and splenectomy to newer medical treatments that mitigate the thrombocytopenia and avoid splenectomy. The thrombopoietin receptor
agonists (TPO-RA), romiplostim. eltrombopag, and avatrombonag. have markedly altered the treatment of ITP. Response rates of 80-90%
are routinely obtained and responses are usually maintained with continued therapy. Data shows that TPO-RA are just as ettecive In early
ITP as in chronic ITP and current guidelines encourage their use as early a3 3 months into the disease course, sometimes even earrier.
TPO-RA do nol naed to be continued forever, about a thid of patients in the frsi year and abou! another third after two years have a
remission. Whether TPO-RA affect the ITP pathophysiology and directly cause remission remains unclear. This review provides a personal
overview of the diagnosis and treatment of ITP with a focus on the mechanism of action of TFO-RA. their place in the treaiment algorithm,
unique aspects of thelr clinical use, aaverse emfects, and options should mney tall