Avatrombopag (Doptelet), an oral thrombopoietin receptor agonist (TPO-RA) by Dova Pharmaceuticals has been FDA-approved for thrombocytopenia in adults with chronic liver disease prior to undergoing a medical procedure. This is the first type of therapy to be approved for this indication, and is delivered as a five day course between 5-8 days prior to major dental or medical surgery to prevent and control bleeding. The drug would eliminate the need for platelet transfusions, and is estimated to be eligible for use in around 70,000 patients in the US.
The treatment has not been approved yet for use in ITP, but has already completed one phase 3 clinical trial (examines safety and efficacy in thousands of patients in a randomized or blind trial), and two phase 2 clinical trials (studies efficacy in hundreds of patients in a placebo-controlled trial). Dova has proclaimed Avatrombopag has fewer liver-related side effects than other TPO agents, and does not pose the concern of food interactions. The company plans to submit a New Drug Application (NDA) to the FDA later this year for use of Avatrombopag in adults with chronic ITP.
Walsh, Sandy. U.S. Food and Drug Administration. “FDA approves new drug for patients with chronic liver disease who have low blood platelets and are undergoing a medical procedure.” May 21 2018. https://bit.ly/2rWVqHx.
NIH Clinical Trials: https://clinicaltrials.gov/ct2/show/NCT03363334
Do all primary ITP patients need treatment and which treatments are the most commonly used? Those were two of the questions answered when top ITP experts in the United Kingdom reviewed the UK Adult ITP Registry, which contains clinical data from the cases of 2920 primary ITP patients from 2010-2018 from 89 sites. The aim of the review was to characterize the ITP patient population and evaluate the current management of ITP. The data demonstrated that around one-third of patients do not receive treatment; unsurprisingly, steroids and IVIG are the most common treatments used. One-fifth of patients need additional treatment beyond these first-line therapies, typically immunosuppressives and thrombopoeitin agonists (Promacta and Nplate). Ten percent of patients were splenectomized. Over 80% of patients were able to elevate their platelet counts above 30,000 in the five years following diagnosis. Comprehensive results will be presented at the European Hematology Association annual congress this June in Stockholm.
Zaidi, Abbas, et.al. “Epidemiology and Management of Primary Immune Thrombocytopenia: Real World Data from the UK ITP Registry” European Hematology Association. Jun 15, 2018. https://bit.ly/2GRswgD