Abstract
Due to the increasingly strict word/character restrictions enforced by most scientific publications, we identified a need for a master document illustrating the “Scientific Methods” that may be applicable to the majority of observational and registry-based studies in hematopoietic cell transplantation (HCT). The purpose of this study is to serve as a reference document that describes the data source, study endpoints and statistical analyses utilized most commonly in retrospective studies in HCT. To this end we compile, define and reference the methodology commonly used in HCT research. While it is recognized that the scientific methodology may periodically require updates due to the evolving landscape of transplant research, such as newer study endpoints and statistical methods, this manuscript describes frequently used methodologies.
Article PDF
Avoid common mistakes on your manuscript.
References
LeMaistre, CF, Farnia, S, Crawford, S, McGuirk, J, Maziarz, RT, Coates, J, et al. Standardization of terminology for episodes of hematopoietic stem cell patient transplant care. Biol Blood Marrow Transplant 2013;19;851–7.
Majhail, NS, Giralt, S, Bonagura, A, Crawford, S, Farnia, S, Omel, JL, et al. Guidelines for defining and implementing standard episode of care for hematopoietic stem cell transplantation within the context of clinical trials. Biol Blood Marrow Transplant 2015;21;583–8.
Logan, BR, Zhang, M-J, Klein, JP. Regression models for hazard rates versus cumulative incidence probabilities in hematopoietic cell transplantation data. Biol Blood Marrow Transplant 2006;12;107–12.
Kim, HT, Armand, P. Clinical endpoints in allogeneic hematopoietic stem cell transplantation studies: the cost of freedom. Biol Blood Marrow Transplant 2013;19;860–6.
Iacobelli, S, On behalf of the EBMT Statistical Committee. Suggestions on the use of statistical methodologies in studies of the European Group for Blood and Marrow Transplantation. Bone Marrow Transplant 2013;48;S1–S37.
Baron, F, Sandmaier, BM. Chimerism and outcomes after allogeneic hematopoietic cell transplantation following nonmyeloablative conditioning. Leukemia 2006;20;1690–700.
Filipovich, AH, Weisdorf, D, Pavletic, S, Socie, G, Wingard, JR, Lee, SJ, et al. National Institutes of Health consensus development project on criteria for clinical trials in chronic graft-versus-host disease: I. Diagnosis and staging working group report. Biol Blood Marrow Transplant 2005;11;945–56.
Przepiorka, D, Weisdorf, D, Martin, P, Klingemann, H, Beatty, P, Hows, J, et al. 1994 consensus conference on acute GVHD grading. Bone Marrow Transplant 1995;15;825–8.
Lee, SJ, Vogelsang, G, Flowers, M Chronic graft-versus-host disease. Biol Blood Marrow Transplant 2003;9;215–33.
Holtan, SG, DeFor, TE, Lazaryan, A, Bejanyan, N, Arora, M, Brunstein, CG, et al. Composite end point of graft-versus-host disease-free, relapse-free survival after allogeneic hematopoietic cell transplantation. Blood 2015;125;1333–8.
Ruggeri, A, Labopin, M, Ciceri, F, Mohty, M, Nagler, A. Definition of GvHD-free, relapse-free survival for registry-based studies: an ALWP–EBMT analysis on patients with AML in remission. Bone Marrow Transplant 2015;51;610.
Pasquini, MC, Logan, B, Jones, RJ, Alousi, AM, Appelbaum, FR, Bolaños-Meade, J, et al. Blood and marrow transplant clinical trials network report on the development of novel endpoints and selection of promising approaches for graft-versus-host disease prevention trials. Biol Blood Marrow Transplant 2018;24; 1274–80.
Author information
Authors and Affiliations
Corresponding author
Additional information
Peer review under responsibility of the International Academy for Clinical Hematology
Rights and permissions
This is an open access article distributed under the CC BY-NC 4.0 license (http://creativecommons.org/licenses/by-nc/4.0/).
About this article
Cite this article
Kanate, A.S., Nagler, A. & Savani, B. Summary of Scientific and Statistical Methods, Study Endpoints and Definitions for Observational and Registry-Based Studies in Hematopoietic Cell Transplantation. Clin Hematol Int 2, 2–4 (2020). https://doi.org/10.2991/chi.d.191207.001
Received:
Accepted:
Published:
Issue Date:
DOI: https://doi.org/10.2991/chi.d.191207.001