Barker JN, Davies SM, DeFor T, Ramsay NK, Weisdorf DJ, Wagner JE Survival after transplantation of unrelated donor umbilical cord blood is comparable to that of human leukocyte antigen-matched unrelated donor bone marrow: results of a matched-pair analysis. Blood. 2001 May 15;97(10):2957-61.
Hwang WY, Samuel M, Tan D, Koh LP, Lim W, Linn YC A meta-analysis of unrelated donor umbilical cord blood transplantation versus unrelated donor bone marrow transplantation in adult and pediatric patients. Biol Blood Marrow Transplant. 2007 Apr;13(4):444-53. Epub 2007 Feb 15. Review.
Merindol N, Charrier E, Duval M, Soudeyns H Complementary and contrasting roles of NK cells and T cells in pediatric umbilical cord blood transplantation. J Leukoc Biol. 2011 Jul;90(1):49-60. doi: 10.1189/jlb.0111007. Epub 2011 Mar 2. Review.
Messner HA How good is allogeneic transplantation for high-risk patients with AML? Best Pract Res Clin Haematol. 2006;19(2):329-32. Review.
van Rood JJ, Scaradavou A, Stevens CE Indirect evidence that maternal microchimerism in cord blood mediates a graft-versus-leukemia effect in cord blood transplantation. Proc Natl Acad Sci U S A. 2012 Feb 14;109(7):2509-14. doi: 10.1073/pnas.1119541109. Epub 2012 Jan 9.
van Rood JJ, Stevens CE, Smits J, Carrier C, Carpenter C, Scaradavou A Reexposure of cord blood to noninherited maternal HLA antigens improves transplant outcome in hematological malignancies. Proc Natl Acad Sci U S A. 2009 Nov 24;106(47):19952-7. doi: 10.1073/pnas.0910310106. Epub 2009 Nov 9.
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.