Faderl S, Gandhi V, Kantarjian HM Potential role of novel nucleoside analogs in the treatment of acute myeloid leukemia. Curr Opin Hematol. 2008 Mar;15(2):101-7. doi: 10.1097/MOH.0b013e3282f46e94. Review.
Leung AY, Tse E, Hwang YY, Chan TS, Gill H, Chim CS, Lie AK, Kwong YL Primary treatment of leukemia relapses after allogeneic hematopoietic stem cell transplantation with reduced-intensity conditioning second transplantation from the original donor. Am J Hematol. 2013 Jun;88(6):485-91. doi: 10.1002/ajh.23439. Epub 2013 May 2.
Sampat K, Kantarjian H, Borthakur G Clofarabine: emerging role in leukemias. Expert Opin Investig Drugs. 2009 Oct;18(10):1559-64. doi: 10.1517/13543780903173222.
Tse E, Leung AY, Sim J, Lee HK, Liu HS, Yip SF, Kwong YL Clofarabine and high-dose cytosine arabinoside in the treatment of refractory or relapsed acute myeloid leukaemia. Ann Hematol. 2011 Nov;90(11):1277-81. doi: 10.1007/s00277-011-1223-2. Epub 2011 Apr 1.
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.
