title: National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: The Research Methodology and Study Design Working Group Report.
abstract: The increasing numbers of hematopoietic cell transplantations (HCTs) performed each year, the changing demographics of HCT recipients, the introduction of new transplantation strategies, incremental improvement in survival, and the growing population of HCT survivors demand a comprehensive approach to examining the health and well-being of patients throughout life after HCT. This report summarizes strategies for the conduct of research on late effects after transplantation, including consideration of the study design and analytic approaches; methodologic challenges in handling complex phenotype data; an appreciation of the changing trends in the practice of transplantation; and the availability of biospecimens to support laboratory-based research. It is hoped that these concepts will promote continued research and facilitate the development of new approaches to address fundamental questions in transplantation outcomes.
authors: Department of Medicine, Medical College of Wisconsin, Center for International Blood and Marrow Transplant Research, Milwaukee, Wisconsin.
authors: Bronwen E
authors: BE
authors: Shaw
authors: Department of Medicine, Roswell Park Cancer Institute, Buffalo, New York.
authors: Theresa
authors: T
authors: Hahn
authors: Department of Medicine, Division of Clinical Research, Fred Hutchinson Cancer Research Center, University of Washington, Seattle, Washington.
authors: Paul J
authors: PJ
authors: Martin
authors: Outcomes Research Branch, Division of Cancer Control and Population Sciences, National Cancer Institute, National Institutes of Health, Rockville, Maryland.
authors: Sandra A
authors: SA
authors: Mitchell
authors: Department of Medicine, Division of Clinical Research, Fred Hutchinson Cancer Research Center, University of Washington, Seattle, Washington.
authors: Effie W
authors: EW
authors: Petersdorf
authors: Epidemiology and Cancer Control, St. Jude Children's Research Hospital, Memphis, Tennessee.
authors: Gregory T
authors: GT
authors: Armstrong
authors: Division of Cancer Control and Population Sciences, National Cancer Institute, Bethesda, Maryland.
authors: Nonniekaye
authors: N
authors: Shelburne
authors: Department of Medicine, Division of Clinical Research, Fred Hutchinson Cancer Research Center, Seattle, Washington.
authors: Barry E
authors: BE
authors: Storer
authors: Institute for Cancer Outcomes and Survivorship, School of Medicine, University of Alabama at Birmingham, Birmingham, Alabama. Electronic address: sbhatia@peds.uab.edu.
authors: Smita
authors: S
authors: Bhatia
mesh_terms: D000328:Adult; D003695:Delivery of Health Care / Q000592:standards; D006287:Health Planning Guidelines; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects / Q000379:methods / Q000639:trends; D006801:Humans; D000069451:Long Term Adverse Effects; D008875:Middle Aged; D009316:National Institutes of Health (U.S.); D012107:Research Design / Q000592:standards / Q000639:trends*; D013997:Time Factors; D014481:United States; D055815:Young Adult
keywords: Hematopoietic cell transplantation; Late effects; National Institutes of Health consensus