Aplastic Anemia — Mycophenolate Mofetil and Cyclosporine to Treat Relapsing Aplastic Anemia
Citation(s)
Rosenfeld SJ, Kimball J, Vining D, Young NS Intensive immunosuppression with antithymocyte globulin and cyclosporine as treatment for severe acquired aplastic anemia. Blood. 1995 Jun 1;85(11):3058-65.
Young NS, Barrett AJ The treatment of severe acquired aplastic anemia. Blood. 1995 Jun 15;85(12):3367-77. Review.
Young NS Autoimmunity and its treatment in aplastic anemia. Ann Intern Med. 1997 Jan 15;126(2):166-8. Erratum in: Ann Intern Med 1997 Oct 15;127(8 Pt 1):658.
A Randomized Trial for the Treatment of Relapsing Aplastic Anemia With Mycophenolate Mofetil (MMF) and Cyclosporine (CSA)
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.
