Hemophilia — A Study of Factor Inhibitors in Adult Patients With Hemophilia and Von Willebrand's Disease in Upper Egypt
Citation(s)
Duncan E, Collecutt M, Street A Nijmegen-Bethesda assay to measure factor VIII inhibitors. Methods Mol Biol. 2013;992:321-33. doi: 10.1007/978-1-62703-339-8_24.
Ghosh K, Shukla R Future of Haemophilia Research in India. Indian J Hematol Blood Transfus. 2017 Dec;33(4):451-452. doi: 10.1007/s12288-017-0862-4. Epub 2017 Aug 21.
Hay CR, DiMichele DM; International Immune Tolerance Study The principal results of the International Immune Tolerance Study: a randomized dose comparison. Blood. 2012 Feb 9;119(6):1335-44. doi: 10.1182/blood-2011-08-369132. Epub 2011 Nov 18.
Pokras SM, Petrilla AA, Weatherall J, Lee WC The economics of inpatient on-demand treatment for haemophilia with high-responding inhibitors: a US retrospective data analysis. Haemophilia. 2012 Mar;18(2):284-90. doi: 10.1111/j.1365-2516.2011.02623.x. Epub 2011 Aug 4.
Schep SJ, Boes M, Schutgens REG, van Vulpen LFD An update on the 'danger theory' in inhibitor development in hemophilia A. Expert Rev Hematol. 2019 May;12(5):335-344. doi: 10.1080/17474086.2019.1604213. Epub 2019 Apr 25.
Witmer C, Young G Factor VIII inhibitors in hemophilia A: rationale and latest evidence. Ther Adv Hematol. 2013 Feb;4(1):59-72. doi: 10.1177/2040620712464509.
A Study of Factor Inhibitors in Adult Patients With Hemophilia and Von Willebrand's Disease in Upper Egypt
Interventional studies are often prospective and are specifically tailored to evaluate direct impacts of treatment or preventive measures on disease.
Observational studies are often retrospective and are used to assess potential causation in exposure-outcome relationships and therefore influence preventive methods.
Expanded access is a means by which manufacturers make investigational new drugs available, under certain circumstances, to treat a patient(s) with a serious disease or condition who cannot participate in a controlled clinical trial.
Clinical trials are conducted in a series of steps, called phases - each phase is designed to answer a separate research question.
Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.
