Michelle Perugini¹, Herman Lee², Simon McRae³ and Richard J. D’Andrea^1,2,4

¹The Division of Haematology and the Centre for Cancer Biology, Hanson Institute and SA Pathology, Adelaide, South Australia. ²The Division of Haematology and Oncology, The Queen Elizabeth Hospital, Woodville, South Australia. ³Department of Haematology, SA Pathology, The Royal Adelaide Hospital, Adelaide, South Australia. ⁴Women’s and Children’s Health Research Institute, North Adelaide, South Australia.

Abstract

Immune Thrombocytopenic Purpura (ITP) has long been thought to result predominantly from immune mediated acceleration of platelet destruction. As a result, immunosuppressive therapy has traditionally played a central role in the treatment of the disorder. Despite many advances in treatment options chronic ITP that is refractory to available therapy remains a difficult clinical problem. Impaired platelet production is now accepted as a hallmark of ITP providing alternate avenues for therapeutic development. Romiplostim is a second generation Thrombopoietin (TPO) mimetic that was approved in 2008 by the US Food and Drug Administration (FDA) for the treatment of patients with chronic ITP that have an insufficient response to corticosteroids, immunoglobulins, or splenectomy, and an increased risk of bleeding. FDA approval was based on pivotal clinical trials that demonstrated romiplostim to be effective in the treatment of chronic ITP in adults, with limited serious side effects reported. Thus romiplostim now provides an option for management of ITP patients refractory to first-line treatments.

Presentation

• Pages: 7

• References: 27
  

• Tables and figures: 2