Hematopoietic Cell Transplantation (HCT): State of the Science

December 5, 2014

Almost 18,000 HCTs are performed in the United States (US) annually, and the number increases by approximately 5% per year. This increase reflects the utility of HCT in treating both malignant and non-malignant diseases, higher donor availability, and treatment advances that allow HCT to be performed in older and sicker patients. However, this number reflects less than half of number of the patients who could benefit from transplantation if the safety and efficacy of the procedure and the ability to successfully perform transplants across histocompatibility barriers were improved.  Such improvements require development and testing of new transplant strategies.

Yet, HCT clinical trials face unique challenges, including the relatively small number of transplantations performed at any single center, the diverse indications for HCT, the complexities of the procedure, and multiple post-transplant complications. 

The BMT CTN was established to address these challenges and execute multicenter HCT trials with broad national participation. The Network:

  • Effectively fosters development of innovative and important concepts into well-designed trials that answer questions in the most efficient manner;
  • Supports timely implementation and completion of those trials;
  • Ensures protection of subjects (both donors and recipients);
  • Provides high-quality data and adherence to regulatory requirements in an increasingly complex environment.

The BMT CTN has answered relevant research questions in both common and rare diseases and critical issues such as optimal graft sources, conditioning intensity, regimen-related toxicity, engraftment, GVHD, infection, disease control, and quality of life.

The BMT CTN has held two State of the Science Symposia, the most recent in 2007.  That meeting resulted in very specific recommendations for high priority clinical trials to be implemented by the US transplant community using the resources of the BMT CTN, NCI Cooperative Groups, and other programs.  Most of these trials have been launched and some have already been successfully completed.

In 2014, BMT CTN will host a third State of the Science Symposium at which 13 scientific committees will survey the HCT landscape and identify areas with the greatest need for high-impact Phase II and III clinical trials. The results of this Symposium are expected to guide national multicenter trial efforts in BMT over the next 5-6 years.

Physicians who refer for transplant need to be aware of the current state of the art in transplant, key transplant research, best practices and future research direction that aid in their clinical decision-making.

ACCME Accreditation Statement:
This activity has been planned and implemented in accordance with the accreditation requirements and policies of the Accreditation Council for Continuing Medical Education through the joint providership of the Medical College of Wisconsin and National Marrow Donor Program. The Medical College of Wisconsin is accredited by the ACCME to provide continuing medical education for physicians.
AMA Credit Designation Statement:
The Medical College of Wisconsin designates this Enduring material for a maximum of 3.0 AMA PRA Category 1 Credits™.  Physicians should claim only the credit commensurate with the extent of their participation in the activity.

Hours of Participation for Allied Health Care Professionals
The Medical College of Wisconsin designates this activity for up to 3.0 hours of participation for continuing education for allied health professionals.  

Target Audience

This course is intended for hematology, oncology, and transplant physicians.

Learning Objectives

At the conclusion of this activity, I will be able to

1. Identify biologic and clinical factors and current recommendations in selecting risk-adapted treatment for CLL.

2. Determine timing of transplant for CLL based on patient risk, disease and molecular factors.

3. Evaluate outcomes for current therapeutic approaches to MCL.

4. Compare the risks and benefits of MCL treatment approach, and how timing of  transplant and clinical patient characteristics affects outcomes.

Additional information


Ellyce Hayes
Phone Number: 
+1 (612) 460-4231
Course summary
Available credit: 
  • 3.00 AMA PRA Category 1 Credit(s)™
    AMA PRA Category 1 Credit(s)™
  • 3.00 Hours of Participation
    Hours of Participation credit.
Course opens: 
Course expires: 
Event starts: 
12/05/2014 - 8:00am
Event ends: 
12/05/2014 - 11:30am
Moscone Center West, 3rd Floor, Room 3007
San Francisco, CA
United States
Planning Committee:
Mary Horowitz, MD, MS
Dennis Confer, MD
Willis Navarro, MD
Amy Foley
Darlene Haven
Ellyce Hayes
Renee Ryan
Mary Horowitz, M.D., M.S. (moderator)
Medical College of Wisconsin, The Center for International Blood and Bone Marrow Transplant Research, Milwaukee, Wis. 

Frederick Appelbaum, M.D.
Fred Hutchinson Cancer Research Center, Seattle, Wash. 

Stella Davies, M.B.B.S., Ph.D.
Cincinnati Children’s Hospital Medical Center, Cincinnati, Ohio 

Steven Devine, M.D.
The Ohio State University Comprehensive Cancer Center, Columbus, Ohio 

John Wingard, M.D.
University of Florida Shands Cancer Center, Gainesville, Fla.
All persons in control of content have NO relevant financial relationships to disclose with the exception of the following:  
Fred Appelbaum, MD - Celegene, Celator, Igenica, Merk, Fate Therapeutics Inc.
John Wingard, MD - Pfizer, Astellas, Ansum, New England Research Institute

Available Credit

  • 3.00 AMA PRA Category 1 Credit(s)™
    AMA PRA Category 1 Credit(s)™
  • 3.00 Hours of Participation
    Hours of Participation credit.


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