The study was an open-label, phase 3, multicenter, randomized trial conducted by the Blood and Marrow Transplant Clinical Trials Network. Randomization was performed in a 1:1 ratio, with the use of random block sizes, and was stratified according to transplantation center and disease risk. The target enrollment was 550 donor–recipient pairs. The primary end point was 2-year survival as assessed by means of an intention-to-treat analysis. Prespecified secondary end points included post-transplantation incidences of neutrophil and platelet engraftment, graft failure, acute and chronic GVHD, relapse, and infections. Other end points included adverse events, immune reconstitution, time to discontinuation of immunosuppressive therapy, and quality of life. This article focuses on the primary end point and clinical secondary end points. Analyses of immune reconstitution and quality of life are ongoing.
Enrollment began on March 31, 2004, and ended on September 9, 2009. The analysis included data collected as of November 15, 2011. The median follow-up of surviving patients is 36 months (interquartile range, 30 to 37). Patients were followed in the study for 3 years, with a late analysis at 5 years planned with the use of data from the Center for International Blood and Marrow Transplant Research, which tracks the outcomes of all allogeneic transplantations in the United States.
Anasetti C., Logan B.R., Lee S.J., Waller E.K., Weisdorf D.J., Wingard J.R., Cutler C.S., Westervelt P., Woolfrey A., Couban S., Ehninger G., Johnston L., Maziarz R.T., Pulsipher M.A., Porter D.L., Mineishi S., McCarty J.M., Khan S.P., Anderlini P., Bensinger W.I., Leitman S.F., Rowley S.D., Bredeson C., Carter S.L., Horowitz M.M, & Confer D.L. (2012). Peripheral-Blood Stem Cells versus Bone Marrow from Unrelated Donors. The New England journal of medicine, 367(16), 10.1056/NEJMoa1203517.