Promacta (eltrombopag) shown to increase platelet counts and reduce bleeding in chronic immune thrombocytopenic
A study assessing the quality of care for patients with sickle cell disease in a variety of hospital settings was presented Saturday, December 6 during the 50th Annual Meeting of the American Society of Hematology in San Francisco, CA. Two other studies on screening methods that help determine stroke risk in children with sickle cell
Full Post: Studies examine quality of care for hospitalized sickle cell disease patients
GlaxoSmithKline has announced positive safety and efficacy results from RAISE (RAndomized placebo- controlled ITP Study with Eltrombopag), a Phase III study of Promacta (eltrombopag) in adults with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who had received one or more prior ITP therapies.
Patients receiving Promacta were eight times more likely than those on placebo to maintain platelet counts between 50,000 to 400,000/microliters during a six- month treatment period, thereby reducing patients’ bleeding symptoms and their need for concomitant and rescue ITP treatments. These data were presented at the 50th Annual Meeting of the American Society of Hematology (ASH), December 6-9, 2008, in San Francisco, CA.
“Promacta is the first approved agent to show that generating platelets can be achieved and maintained with an oral therapy,” said Paolo Paoletti, M.D., Senior Vice President of Oncology R&D, GSK. “With the continued emergence of GSK in oncology, we want patients and physicians to continuously benefit from our dedication to developing truly innovative treatments that can help improve patients’ lives. Promacta is a great example of this commitment.”
Promacta received accelerated approval from the FDA on November 20 as a thrombopoietin receptor treatment for patients with chronic ITP who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy. Promacta should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increase the risk for bleeding. Promacta should not be used in an attempt to normalize platelet counts.
Chronic ITP is a disorder marked by increased platelet destruction and/or inadequate platelet production in the blood, which causes an increased risk of bleeding.(1) Approximately 60,000 individuals in the U.S. have the disorder.(2)
“Patients with chronic ITP often have a difficult time managing their disease. They may experience excessive bruising, bleeding and sometimes more serious hemorrhages that can rarely be fatal. Until recently, ITP patients have had few options well demonstrated to be effective in the long term,” said James Bussel, M.D., director of the Platelet Disorders Center, Children’s Cancer and Blood Foundation Division of New York Presbyterian/Weill Cornell Medical Center. “As the RAISE study demonstrates, Promacta is an important new oral treatment option for ITP patients that is effective in maintaining a hemostatic platelet response.”
RAISE, a global, six-month, double-blind, placebo-controlled, Phase III study was designed to evaluate the safety and efficacy of Promacta in previously treated adults with chronic ITP and with platelet counts less than 30,000/microliters. The study enrolled 197 patients (Promacta: n=135; placebo: n=62) and, of these, approximately 50 percent had platelet counts less than or equal to 15,000/microliters; about 50 percent were receiving simultaneous ITP therapies at randomization; around 35 percent were splenectomized, and more than 50 percent had received at least three prior ITP medications. Patients began once daily treatment with Promacta at 50 mg (or matching placebo) with doses individualized based upon each patient’s platelet response, ranging from once-daily doses of 25 mg to 75 mg, or less frequently. The baseline median platelet count in both the placebo and the Promacta groups was 16,000/microliters.
Throughout the study, the median platelet count in the placebo group never exceeded 30,000/microliters. By contrast, after just one week, patients in the Promacta arm experienced a rise in their median platelet count to 36,000/microliters, with median platelet levels subsequently ranging from 52,000 to 91,000/microliters for the remainder of the study, meeting the study’s primary endpoint of odds of responding (platelets 50,000 to 400,000/microliters) during the six month treatment period. Patients receiving Promacta were eight times more likely to achieve an overall response of increased platelet counts of 50,000 to 400,000/microliters than those taking placebo (Odds ratio = 8.2; 99 percent CI [3.59, 18.73]; p < 0.001). In comparison to the placebo group, significantly fewer patients treated with Promacta had any bleeding or clinically significant (WHO Grades 2-4; p < 0.001) bleeding throughout the trial and more patients in the Promacta group (59 percent) stopped or reduced their simultaneous ITP medications than in the placebo group (32 percent; p = 0.016). In addition, during the treatment phase of the study, fewer patients in the Promacta arm (19 percent) required rescue therapy compared with those in the placebo arm (40 percent, p = 0.001).
The overall incidence of adverse events was similar between the Promacta (87 percent) and placebo groups (92 percent), which were mostly mild to moderate in severity. Headache was the most common adverse event in both groups (greater than or equal to 30 percent). Two corticosteroid-associated adverse events (dyspepsia and peripheral edema) were significantly less likely to occur in the Promacta group compared to the placebo group; however, a higher incidence of hepatobiliary laboratory abnormalities were reported in patients taking Promacta (13 percent) compared with those in the placebo group (7 percent). These abnormalities were not predictive of serious, drug-induced liver injury. One death was reported in the placebo group. There were no clinical or laboratory symptoms suggestive of bone marrow fibrosis in patients taking Promacta.
Four studies that highlight significant advances in treatment and survival outcomes for patients with various forms of thrombocytopenia, a group of bleeding disorders characterized by a low number of platelets in the blood, were presented December 6 during the 50th Annual Meeting of the American Society of Hematology in San Francisco, CA. The studies featured in
Full Post: Novel therapeutic approaches for various forms of thrombocytopenia
GlaxoSmithKline has announced that the United States Food and Drug Administration (FDA) granted accelerated approval for PROMACTA (eltrombopag) for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy. The approval is a significant milestone for PROMACTA and the ITP community,
Full Post: FDA approves GSK’s Promacta (Eltrombopag)
Amgen has announced that the European Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending marketing authorisation for Nplate (romiplostim) in the European Union (EU). The CHMP recommends Nplate for adult chronic immune (idiopathic) thrombocytopenia purpura (ITP) splenectomised patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins). Nplate may
Full Post: Nplate receives positive opinion for marketing authorisation in Europe
Researchers reported on a study of a new oral anti-clotting agent - rivaroxaban - designed to identify doses that would be safe to test in subsequent Phase III efficacy and safety trials. The results of ATLAS ACS-TIMI 46 were presented as a late-breaking clinical trial at the American Heart Association’s Scientific Sessions 2008. “The
Full Post: Rivaroxaban shows promise in patients with acute coronary syndrome
Individually tailored anti-clotting medication dosing significantly cut the rate of adverse events after non-emergent percutaneous coronary intervention (PCI) with stenting, researchers reported at the American Heart Association’s Scientific Sessions 2008. Results of the Tailored Clopidogrel Loading Dose According to Platelet Reactivity Monitoring to Prevent Stent Thrombosis study were presented as a late-breaking clinical trial.
Full Post: Tailored anti-clotting medications reduce adverse effects after percutaneous coronary intervention