Comparison of Characteristics and Outcomes of Trial Participants and Nonparticipants

Example of Blood and Marrow Transplant Clinical Trials Network 0201 Trial

Nandita Khera, Navneet S. Majhail, Ruta Brazauskas, Zhiwei Wang, Naya He, Mahmoud D. Aljurf, Görgün Akpek, Yoshiko Atsuta, Sara Beattie, Christopher N. Bredeson, Linda J. Burns, Jignesh D. Dalal, César O. Freytes, Vikas Gupta, Yoshihiro Inamoto, Hillard M. Lazarus, Charles F. LeMaistre, Amir Steinberg, David Szwajcer, John R. Wingard & 8 others Baldeep Wirk, William A. Wood, Steven Joffe, Theresa E. Hahn, Fausto R. Loberiza, Claudio Anasetti, Mary M. Horowitz, Stephanie J. Lee

Research output: Contribution to journalArticle

5 Citations (Scopus)

Abstract

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.

Original languageEnglish (US)
Pages (from-to)1815-1822
Number of pages8
JournalBiology of Blood and Marrow Transplantation
Volume21
Issue number10
DOIs
StatePublished - Oct 1 2015

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Bone Marrow
Clinical Trials
Transplants
Unrelated Donors
Sex Distribution
Antilymphocyte Serum
Survival
Cell Transplantation
Graft vs Host Disease
Bone Marrow Transplantation
Proportional Hazards Models
Multicenter Studies
Comorbidity
Transplantation
Outcome Assessment (Health Care)
Tissue Donors
Confidence Intervals
Recurrence
Mortality
Incidence

All Science Journal Classification (ASJC) codes

  • Hematology
  • Transplantation

Cite this

Khera, Nandita ; Majhail, Navneet S. ; Brazauskas, Ruta ; Wang, Zhiwei ; He, Naya ; Aljurf, Mahmoud D. ; Akpek, Görgün ; Atsuta, Yoshiko ; Beattie, Sara ; Bredeson, Christopher N. ; Burns, Linda J. ; Dalal, Jignesh D. ; Freytes, César O. ; Gupta, Vikas ; Inamoto, Yoshihiro ; Lazarus, Hillard M. ; LeMaistre, Charles F. ; Steinberg, Amir ; Szwajcer, David ; Wingard, John R. ; Wirk, Baldeep ; Wood, William A. ; Joffe, Steven ; Hahn, Theresa E. ; Loberiza, Fausto R. ; Anasetti, Claudio ; Horowitz, Mary M. ; Lee, Stephanie J. / Comparison of Characteristics and Outcomes of Trial Participants and Nonparticipants : Example of Blood and Marrow Transplant Clinical Trials Network 0201 Trial. In: Biology of Blood and Marrow Transplantation. 2015 ; Vol. 21, No. 10. pp. 1815-1822.
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abstract = "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.",
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Khera, N, Majhail, NS, Brazauskas, R, Wang, Z, He, N, Aljurf, MD, Akpek, G, Atsuta, Y, Beattie, S, Bredeson, CN, Burns, LJ, Dalal, JD, Freytes, CO, Gupta, V, Inamoto, Y, Lazarus, HM, LeMaistre, CF, Steinberg, A, Szwajcer, D, Wingard, JR, Wirk, B, Wood, WA, Joffe, S, Hahn, TE, Loberiza, FR, Anasetti, C, Horowitz, MM & Lee, SJ 2015, 'Comparison of Characteristics and Outcomes of Trial Participants and Nonparticipants: Example of Blood and Marrow Transplant Clinical Trials Network 0201 Trial', Biology of Blood and Marrow Transplantation, vol. 21, no. 10, pp. 1815-1822. https://doi.org/10.1016/j.bbmt.2015.06.004

Comparison of Characteristics and Outcomes of Trial Participants and Nonparticipants : Example of Blood and Marrow Transplant Clinical Trials Network 0201 Trial. / Khera, Nandita; Majhail, Navneet S.; Brazauskas, Ruta; Wang, Zhiwei; He, Naya; Aljurf, Mahmoud D.; Akpek, Görgün; Atsuta, Yoshiko; Beattie, Sara; Bredeson, Christopher N.; Burns, Linda J.; Dalal, Jignesh D.; Freytes, César O.; Gupta, Vikas; Inamoto, Yoshihiro; Lazarus, Hillard M.; LeMaistre, Charles F.; Steinberg, Amir; Szwajcer, David; Wingard, John R.; Wirk, Baldeep; Wood, William A.; Joffe, Steven; Hahn, Theresa E.; Loberiza, Fausto R.; Anasetti, Claudio; Horowitz, Mary M.; Lee, Stephanie J.

In: Biology of Blood and Marrow Transplantation, Vol. 21, No. 10, 01.10.2015, p. 1815-1822.

Research output: Contribution to journalArticle

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.

PY - 2015/10/1

Y1 - 2015/10/1

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