Brenner H, Gondos A, Pulte D Recent major improvement in long-term survival of younger patients with multiple myeloma. Blood. 2008 Mar 1;111(5):2521-6. Epub 2007 Sep 27.
Cerrato C, Palumbo A Initial treatment of nontransplant patients with multiple myeloma. Semin Oncol. 2013 Oct;40(5):577-84. doi: 10.1053/j.seminoncol.2013.07.003.
Charlson ME, Pompei P, Ales KL, MacKenzie CR A new method of classifying prognostic comorbidity in longitudinal studies: development and validation. J Chronic Dis. 1987;40(5):373-83.
Moreau P, Avet-Loiseau H, Harousseau JL, Attal M Current trends in autologous stem-cell transplantation for myeloma in the era of novel therapies. J Clin Oncol. 2011 May 10;29(14):1898-906. doi: 10.1200/JCO.2010.32.5878. Epub 2011 Apr 11. Review.
Stewart AK, Richardson PG, San-Miguel JF How I treat multiple myeloma in younger patients. Blood. 2009 Dec 24;114(27):5436-43. doi: 10.1182/blood-2009-07-204651. Epub 2009 Oct 27. Erratum in: Blood. 2010 May 13;115(19):4006.
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.