TY - JOUR
T1 - Comparison of Characteristics and Outcomes of Trial Participants and Nonparticipants
T2 - Example of Blood and Marrow Transplant Clinical Trials Network 0201 Trial
AU - Khera, Nandita
AU - Majhail, Navneet S.
AU - Brazauskas, Ruta
AU - Wang, Zhiwei
AU - He, Naya
AU - Aljurf, Mahmoud D.
AU - Akpek, Görgün
AU - Atsuta, Yoshiko
AU - Beattie, Sara
AU - Bredeson, Christopher N.
AU - Burns, Linda J.
AU - Dalal, Jignesh D.
AU - Freytes, César O.
AU - Gupta, Vikas
AU - Inamoto, Yoshihiro
AU - Lazarus, Hillard M.
AU - LeMaistre, Charles F.
AU - Steinberg, Amir
AU - Szwajcer, David
AU - Wingard, John R.
AU - Wirk, Baldeep
AU - Wood, William A.
AU - Joffe, Steven
AU - Hahn, Theresa E.
AU - Loberiza, Fausto R.
AU - Anasetti, Claudio
AU - Horowitz, Mary M.
AU - Lee, Stephanie J.
N1 - Funding Information:
Support for the clinical trial BMT CTN 0201 was provided by grant U10HL069294 from the National Heart, Lung, and Blood Institute (NHLBI) and the National Cancer Institute (NCI), the Department of the Navy , Office of Naval Research , and the National Marrow Donor Program. Enrollment support was provided by DKMS Germany. Any views, opinions, findings, conclusions or recommendations expressed in this material are those of the author(s) and do not reflect the views or the official policy or position of the above mentioned parties.
Funding Information:
The CIBMTR is supported by Public Health Service grant/cooperative agreement U24-CA076518 from the NCI , the NHLBI , and the National Institute of Allergy and Infectious Diseases ; a grant/cooperative agreement 5U10HL069294 from NHLBI and NCI ; a contract HHSH250201200016C with Health Resources and Services Administration (HRSA/DHHS); 2 Grants N00014-12-1-0142 and N00014-13-1-0039 from the Office of Naval Research ; and grants from * Actinium Pharmaceuticals ; Allos Therapeutics, Inc. ; * Amgen ; anonymous donation to the Medical College of Wisconsin ; Ariad ; Be The Match Foundation ; * Blue Cross and Blue Shield Association ; * Celgene Corporation ; Chimerix, Inc. ; Fred Hutchinson Cancer Research Center ; Fresenius-Biotech North America, Inc. ; * Gamida Cell Teva Joint Venture Ltd. ; Genentech, Inc. ; * Gentium SpA ; Genzyme Corporation ; GlaxoSmithKline ; Health Research, Inc. ; Roswell Park Cancer Institute ; HistoGenetics ; Incyte Corporation ; Jeff Gordon Children's Foundation ; Kiadis Pharma ; The Leukemia & Lymphoma Society ; Medac GmbH ; The Medical College of Wisconsin ; Merck & Co., Inc. ; Millennium: The Takeda Oncology Co. ; * Milliman USA, Inc. ; * Miltenyi Biotec ; National Marrow Donor Program ; Onyx Pharmaceuticals ; Optum Healthcare Solutions, Inc. ; Osiris Therapeutics ; Otsuka America Pharmaceutical, Inc. ; Perkin Elmer, Inc. ; * Remedy Informatics ; * Sanofi US ; Seattle Genetics ; Sigma-Tau Pharmaceuticals ; Soligenix, Inc. ; St. Baldrick's Foundation ; StemCyte, A Global Cord Blood Therapeutics Co. ; Stemsoft Software, Inc. ; Swedish Orphan Biovitrum ; * Tarix Pharmaceuticals ; * Terumo BCT ; * Teva Neuroscience, Inc. ; * Therakos ; University of Minnesota ; University of Utah ; and * WellPoint . The views expressed in this article do not reflect the official policy or position of the National Institute of Health, the Department of the Navy, the Department of Defense, Health Resources and Services Administration or any other agency of the US Government.
Publisher Copyright:
© 2015 American Society for Blood and Marrow Transplantation.
PY - 2015/10/1
Y1 - 2015/10/1
N2 - Controversy surrounds the question of whether clinical trial participants have better outcomes than comparable patients who are not treated on a trial. We explored this question using a recent large, randomized, multicenter study comparing peripheral blood (PB) with bone marrow transplantation from unrelated donors, conducted by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). We compared characteristics and outcomes of study participants (n = 494) and nonparticipants (n = 1384) who appeared eligible and received similar treatment without enrolling on the BMT CTN trial at participating centers during the study time period. Data were obtained from the Center for International Blood and Marrow Transplant Research. Outcomes were compared between the 2 groups using Cox proportional hazards regression models. No significant differences in age, sex, disease distribution, race/ethnicity, HLA matching, comorbidities, and interval from diagnosis to hematopoietic cell transplantation were seen between the participants and nonparticipants. Nonparticipants were more likely to have lower performance status, lower risk disease, and older donors, and to receive myeloablative conditioning and antithymocyte globulin. Nonparticipants were also more likely to receive PB grafts, the intervention tested in the trial (66% versus 50%, P < .001). Overall survival, transplantation-related mortality, and incidences of acute or chronic graft-versus-host disease were comparable between the 2 groups though relapse was higher (hazard ratio, 1.22; 95% confidence interval, 1.02 to 1.46; P = .028) in nonparticipants. Despite differences in certain baseline characteristics, survival was comparable between study participants and nonparticipants. The results of the BMT CTN trial appear generalizable to the population of trial-eligible patients.
AB - Controversy surrounds the question of whether clinical trial participants have better outcomes than comparable patients who are not treated on a trial. We explored this question using a recent large, randomized, multicenter study comparing peripheral blood (PB) with bone marrow transplantation from unrelated donors, conducted by the Blood and Marrow Transplant Clinical Trials Network (BMT CTN). We compared characteristics and outcomes of study participants (n = 494) and nonparticipants (n = 1384) who appeared eligible and received similar treatment without enrolling on the BMT CTN trial at participating centers during the study time period. Data were obtained from the Center for International Blood and Marrow Transplant Research. Outcomes were compared between the 2 groups using Cox proportional hazards regression models. No significant differences in age, sex, disease distribution, race/ethnicity, HLA matching, comorbidities, and interval from diagnosis to hematopoietic cell transplantation were seen between the participants and nonparticipants. Nonparticipants were more likely to have lower performance status, lower risk disease, and older donors, and to receive myeloablative conditioning and antithymocyte globulin. Nonparticipants were also more likely to receive PB grafts, the intervention tested in the trial (66% versus 50%, P < .001). Overall survival, transplantation-related mortality, and incidences of acute or chronic graft-versus-host disease were comparable between the 2 groups though relapse was higher (hazard ratio, 1.22; 95% confidence interval, 1.02 to 1.46; P = .028) in nonparticipants. Despite differences in certain baseline characteristics, survival was comparable between study participants and nonparticipants. The results of the BMT CTN trial appear generalizable to the population of trial-eligible patients.
KW - Hematopoietic cell transplantation
KW - Trial participation
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U2 - 10.1016/j.bbmt.2015.06.004
DO - 10.1016/j.bbmt.2015.06.004
M3 - Article
C2 - 26071866
AN - SCOPUS:84941315341
VL - 21
SP - 1815
EP - 1822
JO - Biology of Blood and Marrow Transplantation
JF - Biology of Blood and Marrow Transplantation
SN - 1083-8791
IS - 10
ER -