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https://www.readbyqxmd.com/read/27511890/recombinant-porcine-sequence-factor-viii-rpfviii-for-acquired-haemophilia-a-practical-clinical-experience-of-its-use-in-seven-patients
#1
M D Tarantino, A Cuker, B Hardesty, J C Roberts, M Sholzberg
INTRODUCTION: A recombinant porcine factor VIII B-domain-deleted product (rpFVIII; OBIZUR, Baxalta Incorporated, Deerfield, IL 60015, USA) was recently approved for treatment of bleeding episodes in adults with acquired haemophilia A (AHA) in the United States. To date, no clinical experience outside the registration study has been reported. AIM: To describe early clinical experience using rpFVIII for AHA. METHODS: A retrospective chart review of seven patients with AHA treated with rpFVIII at four institutions from November 2014 to October 2015...
August 10, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/27371116/preclinical-assessment-of-a-new-recombinant-adamts-13-drug-product-bax930-for-the-treatment-of-thrombotic-thrombocytopenic-purpura
#2
A Kopić, K Benamara, C Piskernik, B Plaimauer, F Horling, G Höbarth, T Ruthsatz, B Dietrich, E-M Muchitsch, F Scheiflinger, M Turecek, W Höllriegl
UNLABELLED: Essentials ADAMTS-13-deficiency is a cause of thrombotic thrombocytopenic purpura (TTP). Preclinical safety of recombinant human ADAMTS-13 (BAX930) was shown in animal models. Preclinical efficacy of BAX930 was shown in a mouse model of TTP. BAX930 showed advantageous efficacy over fresh frozen plasma, the current standard of care. Click to hear Dr Cataland and Prof. Lämmle present a seminar on Thrombotic Thrombocytopenic Purpura (TTP): new Insights in Pathogenesis and Treatment Modalities...
July 2016: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/27126341/10-liquid-human-immunoglobulin-kiovig-%C3%A2-for-immunomodulation-in-autoimmune-disorders
#3
Nikolai Nikolov, Jürgen Reisinger, Hans P Schwarz
Intravenous immunoglobulins have been used to treat autoimmune disorders (ADs) for over 50 years. The etiologies of various ADs are not fully understood and although intravenous immunoglobulin treatment has proved its immunomodulatory properties, the roles of proposed mechanisms of action also remain a matter of speculation. A systemic search of the literature regarding KIOVIG(®) (Baxalta US, Inc., MA, USA) use in clinical trials on patients with ADs and a detailed review of retrieved articles revealed eight relevant publications...
July 2016: Immunotherapy
https://www.readbyqxmd.com/read/27015254/a-budget-impact-model-of-hemophilia-bypassing-agent-prophylaxis-relative-to-recombinant-factor-viia-on-demand
#4
Darshan A Mehta, Abiola O Oladapo, Joshua D Epstein, Aaron R Novack, Ellis J Neufeld, Joel W Hay
BACKGROUND: Hemophilia patients use factor-clotting concentrates (factor VIII for hemophilia A and factor IX for hemophilia B) for improved blood clotting. These products are used to prevent or stop bleeding episodes. However, some hemophilia patients develop inhibitors (i.e., the patient's immune system develops antibodies against these factor concentrates). Hence, these patients do not respond well to the factor concentrates. A majority of hemophilia patients with inhibitors are managed on-demand with the following bypassing agents: recombinant factor VIIa (rFVIIa) and activated prothrombin complex concentrate (aPCC)...
February 2016: Journal of Managed Care & Specialty Pharmacy
https://www.readbyqxmd.com/read/26842562/spanish-consensus-guidelines-on-prophylaxis-with-bypassing-agents-in-patients-with-haemophilia-and-inhibitors
#5
Maria Fernanda López-Fernández, Carmen Altisent Roca, Maria Teresa Álvarez-Román, Mariana Isabel Canaro Hirnyk, Maria Eva Mingot-Castellano, Víctor Jiménez-Yuste, Ana Rosa Cid Haro, Rosario Pérez-Garrido, Carmen Sedano Balbas
Prophylaxis with the blood clotting factor, factor VIII (FVIII) is ineffective for individuals with haemophilia A and high-titre inhibitors to FVIII. Prophylaxis with the FVIII bypassing agents activated prothrombin complex concentrates (aPCC; FEIBA® Baxalta) or recombinant activated factor VII (rFVIIa; Novo-Seven®, Novo Nordisk) may be an effective alternative. It was our aim to develop evidence -and expert opinion- based guidelines for prophylactic therapy for patients with high-titre inhibitors to FVIII...
May 2, 2016: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/26157075/pegylated-full-length-recombinant-factor-viii-for-prophylactic-and-on-demand-treatment-of-severe-hemophilia-a
#6
Barbara A Konkle, Oleksandra Stasyshyn, Pratima Chowdary, David H Bevan, Tim Mant, Midori Shima, Werner Engl, Jacqueline Dyck-Jones, Monika Fuerlinger, Lisa Patrone, Bruce Ewenstein, Brigitt Abbuehl
Current management of hemophilia A includes prophylaxis with factor VIII (FVIII) replacement every 2 to 3 days. BAX 855, Baxalta's pegylated full-length recombinant FVIII (rFVIII), was designed to increase half-life and, thus, reduce the frequency of prophylactic infusions while maintaining hemostatic efficacy. BAX 855 was evaluated in previously treated patients with severe hemophilia A who were aged 12 to 65 years. A phase 1 study compared the pharmacokinetic (PK) profile of BAX 855 with that of licensed rFVIII (Advate)...
August 27, 2015: Blood
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