NIH Blood and Marrow Transplant Late Effects Consensus Conference

June 21-22, 2016 in Rockville, MD


Overview

Autologous and allogeneic blood and marrow transplantation (BMT) is a potentially curative treatment for a number of diseases, including cancer and immune or genetic disorders. Increasing indications for BMT, advances in transplantation practices, and the development of supportive multidisciplinary centers have led to more patients living longer after transplant than ever before. Currently more than 60,000 transplants are performed annually worldwide, and by the year 2030, it is projected that the number of survivors will increase five-fold, potentially resulting in half a million survivors in the United States alone.

As the majority of survivors are living beyond the first two years after transplantation, they are prone to a unique set of complications and late effects that reflect the complex interplay between their cancer diagnosis or immune/genetic disorder, prior treatment, comorbidities, conditioning therapy, and immune reconstitution. This leads to a multitude of changes in physical, psychological, financial, and social domains. The most common late-onset causes of death after BMT include recurrent disease, graft versus host disease, infection due to decreased immunity, and new malignancy, while treatment-related symptoms and toxicities such as chronic pain, fatigue, insomnia, sexual dysfunction, cardiac and respiratory complications, memory loss, mood changes, vision and dental problems, and social stressors cause significant distress in these patients.

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Purpose

This day and a half NCI-NHLBI- sponsored symposium brought together experts in blood and marrow transplantation, late effects, and health care delivery to discuss current evidence and knowledge gaps, develop consensus guidelines, and inform future research in the BMT survivor population. The symposium focused on six late effect categories: secondary malignancies, quality of life/psychosocial outcomes, immune dysregulation and pathobiology, health care delivery, vascular and metabolic outcomes, and research methodology/study design. Each session included a presentation on findings and recommendations followed by a panel discussion on consensus guidelines. The goal of these sessions was to identify knowledge gaps, develop evidence-based recommendations and identify important research questions to improve BMT survivor monitoring and management.

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Agenda


View agenda for Tuesday, June 21
NCI Shady Grove, Room TE406
Time Topic
7:30 a.m. - 8:00 a.m. Check-In
8:00 a.m. - 8:30 a.m.

Opening Remarks
Nonniekaye Shelburne, C.R.N.P., M.S., A.O.C.N
NCI

Richard Childs, M.D.
NHLBI

Ronald Gress, M.D.
NCI

8:30 a.m. - 8:40 a.m.

Initiative Overview
Minoo Battiwalla, M.D.
NHLBI

Bipin Savani, M.D.
Vanderbilt University Medical Center

8:40 a.m. - 8:50 a.m.

Conference Overview and Panel Activities
Steven Pavletic, M.D., M.S.
NCI

8:50 a.m. - 10:05 a.m. Session 1: New Malignancies
Gerard Socie, M.D., Ph.D. (Session Moderator)
Saint Louis Hospital/University of Paris
  • Presentation of Findings and Recommendations (20 minutes)
    Linda Burns, M.D. (Working Group Co-chair)
    National Marrow Donor Program

    Lindsay Morton, Ph.D. (Working Group Co-chair)
    NCI

  • Expert Commentary (10 minutes)
    Mary Flowers, M.D.
    Fred Hutchinson Cancer Research Center

    Gerard Socie, M.D., Ph.D.
    Saint Louis Hospital/University of Paris

  • Discussion Session (45 minutes)
    Working Group Panel:

    Steven Pavletic, M.D., M.S.
    NCI

    John Barrett, M.D.
    NHLBI

    Shahinaz Gadalla, M.D., Ph.D.
    NCI

    David Kleiner, M.D., Ph.D.
    NCI

    Wael Saber, M.D., M.S.
    CIBMTR / Medical College of Wisconsin

    Smita Bhatia, M.D., M.P.H.
    University of Alabama Birmingham

    Eric Engels, M.D., M.P.H.
    NCI

    Scott Baker, M.D., M.S.
    Fred Hutchinson Cancer Research Center

10:05 a.m. - 10:30 a.m. Break
10:30 a.m. - 11:45 a.m. Session 2: Psychosocial and Quality of Life Outcomes
Julia Rowland, Ph.D. (Session Moderator)
NCI
  • Presentation of Findings and Recommendations (20 minutes)
    Karen Syrjala, Ph.D. (Working Group Co-chair)
    Fred Hutchinson Cancer Research Center

    Margaret Bevans, R.N., Ph.D., A.O.C.N., F.A.A.N. (Working Group Co-chair)
    NIH Clinical Center

  • Expert Commentary (10 minutes)
    Helene Schoemans, M.D.
    UZ Leuven

    Julia Rowland, Ph.D.
    NCI

  • Discussion Session (45 minutes)
    Working Group Panel:

    Flora Hoodin, Ph.D., M.A.
    University of Michigan Ann Arbor

    Lori Wiener, Ph.D.
    NCI

    Matt Hsieh, M.D.
    NHLBI

    Paul Jacobsen, Ph.D.
    Moffitt Cancer Center

    Bill Wood, M.D.
    University of North Carolina

    Erin Kent, Ph.D., M.S.
    NCI

    Kate Tierney, R.N., Ph.D.
    Stanford Cancer Institute

    Areej El-Jawahri, M.D.
    Massachusetts General Hospital

    Ellen Denzen, M.S.
    National Marrow Donor Program

11:45 a.m. - 12:45 p.m. Lunch (Cafeteria on site)
12:45 p.m. - 2:00 p.m. Session 3: Immune Dysregulation and Pathobiology
John Barrett, M.D. (Session Moderator)
NHLBI
  • Presentation of Findings and Recommendations (20 minutes)
    Juan Gea-Banacloche, M.D. (Working Group Co-chair)
    NCI

    John Wingard, M.D. (Working Group Co-chair)
    University of Florida

  • Expert Commentary (10 minutes)
    Gerard Socie, M.D., Ph.D.
    Saint Louis Hospital/University of Paris

    John Barrett, M.D.
    NHLBI

  • Discussion Session (45 minutes)
    Working Group Panel:

    Sharon Savage, M.D.
    NCI

    Kirk Schultz, M.D.
    BC Children's Hospital

    Robert Le, M.D., Ph.D.
    Food and Drug Administration

    Linda Griffith, M.D., Ph.D.
    NIAID

    Krishna Komanduri, M.D.
    University of Miami

    Paul Carpenter, M.D.
    Fred Hutchinson Cancer Research Center

    Jo-Anne Young, M.D.
    University of Minnesota

    Sophie Paczesny, M.D., Ph.D.
    Indiana University

    Stefanie Sarantopoulous, M.D., Ph.D.
    Duke Cancer Institute

    Nahed El Kassar, Ph.D., M.D.
    NHLBI

    Bipin Savani, M.D.
    Vanderbilt University Medical Center

2:00 p.m. - 2:25 p.m. Break
2:25 p.m. - 3:40 p.m. Session 4: Cardiovascular and Metabolic Impairment
Andre Tichelli, M.D. (Session Moderator)
University Hospital of Basel
  • Presentation of Findings and Recommendations (20 minutes)
    Saro Armenian, D.O., M.P.H. (Working Group Co-chair)
    City of Hope

    Minoo Battiwalla, M.D. (Working Group Co-chair)
    NHLBI

  • Expert Commentary (10 minutes)
    Bipin Savani, M.D.
    Vanderbilt University Medical Center

    Andre Tichelli, M.D.
    University Hospital of Basel

  • Discussion Session (45 minutes)
    Working Group Panel:

    Marcus Chen, M.D.
    NHLBI

    Nina Salooja, M.D.
    Hammersmith

    Alicia Rovo, M.D.
    University Hospital of Basel

    Christine Duncan, M.D.
    Dana Farber Cancer Institute

    Alan Remaley, M.D., Ph.D.
    NHLBI

    Mike Pulsipher, M.D.
    Children's Hospital of Los Angeles

    Eric Chow, M.D.
    Fred Hutchinson Cancer Research Center

    Wassim Chemaitilly, M.D.
    St. Jude's Children's Research Hospital

3:40 p.m. - 4:50 p.m. The Patient's View on BMT Late Effects: A Panel Discussion
Sue Stewart, Executive Director (Session Moderator)
BMT InfoNet
4:50 p.m. - 5:00 p.m. Day 1 Summary
5:00 p.m. Adjourn

View agenda for Wednesday, June 22
NCI Shady Grove, Room TE406
Time Topic
8:00 a.m. - 8:15 a.m. Conference Overview
Steven Pavletic, M.D., M.S.
NCI
8:15 a.m. - 9:30 a.m. Session 5: Health Care Delivery
Krishna Komanduri, M.D. (Session Moderator)
University of Miami
  • Presentation of Findings and Recommendations (20 minutes)
    Shahrukh Hashmi, M.D. (Working Group Co-chair)
    Mayo Clinic

    Navneet Majhail, M.B.B.S., M.D., M.S. (Working Group Co-chair)
    Cleveland Clinic

  • Expert Commentary (10 minutes)
    David Jacobsohn, M.D., Sc.M.
    Children's National Health System

    Krishna Komanduri, M.D.
    University of Miami

  • Discussion Session (45 minutes)
    Working Group Panel:

    Mary Flowers, M.D.
    Fred Hutchinson Cancer Research Center

    Melissa Greenwald, M.D.
    Health Resources and Services Administration

    Madan Jagasia, M.D., M.B.B.S., M.S.
    Vanderbilt University Medical Center

    Stephanie Farnia, M.P.H.
    National Marrow Donor Program

    Rafael Duarte, M.D., Ph.D.
    European Society for Blood and Marrow Transplantation

    Doug Rizzo, M.D., M.S.
    Medical College of Wisconsin

    Courtney Fitzhugh, M.D.
    NHLBI

    Chris Bredeson, M.D., M.Sc.
    ASBMT / University of Ottawa

    Patricia Martin, R.N., B.S.N.
    Anthem BlueCross BlueShield

    Dennis Gastineau, M.D.
    Mayo Clinic

    Sue Ferrey, M.D.
    Moffitt Cancer Center

    Jim Gajewski, M.D., Ph.D.
    Oregon Health & Science University

9:30 a.m. - 9:55 a.m. Break
9:55 a.m. - 11:10 a.m. Session 6: Research Methodology and Study Design
Corey Cutler, M.D., M.P.H., F.R.C.P.C. (Session Moderator)
Dana Farber Cancer Institute
  • Presentation of Findings and Recommendations
    Smita Bhatia, M.D., M.P.H. (Working Group Co-chair)
    University of Alabama Birmingham

    Bronwen Shaw, M.D., Ph.D. (Working Group Co-chair)
    Medical College of Wisconsin

  • Expert Commentary (10 minutes)
    Dan Weisdorf, M.D.
    University of Minnesota

    Corey Cutler, M.D., M.P.H., F.R.C.P.C.
    Dana Farber Cancer Institute

  • Discussion Session (45 minutes)
    Working Group Panel:

    Terri Hahn, Ph.D.
    Roswell Park Cancer Institute

    Paul Martin, M.D.
    Fred Hutchinson Cancer Research Center

    Greg Armstrong, M.D., M.S.C.E.
    St Jude's Children's Research Hospital

    Sandy Mitchell, Ph.D., C.R.N.P., A.O.C.N.
    NCI

    Barry Storer, Ph.D.
    Fred Hutchinson Cancer Research Center

    Effie Petersdorf, M.D.
    ASBMT / Fred Hutchinson Cancer Research Center

11:10 a.m. - 11:40 a.m.

NIH Funding Opportunities to Support Consensus Recommendations
Nonniekaye Shelburne, C.R.N.P., M.S., A.O.C.N
NCI

Nahed El Kassar, Ph.D., M.D.
NHLBI

11:40 a.m. - 12:00 p.m.

Meeting Summary & Next Steps
Minoo Battiwalla, M.D.
NHLBI

Nonniekaye Shelburne, C.R.N.P., M.S., A.O.C.N
NCI

12:00 p.m. Adjournment
Post-Meeting Workshop
12:30 p.m. Starting Up a BMT Survivorship Program (Optional 1 hour session – not available via webinar)
Bipin Savani, M.D.
Vanderbilt University Medical Center

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Steering Committee

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Working Groups


Subsequent Neoplasms

Working Group Co-Chairs:

Working Group Members:


Psychosocial and Quality of Life Outcomes

Working Group Co-Chairs:

Working Group Members:


Immune Dysregulation and Pathobiology

Working Group Co-Chairs:

Working Group Members:


Cardiovascular and Metabolic Impairment

Working Group Co-Chairs:

Working Group Members:


Healthcare Delivery

Working Group Co-Chairs:

Working Group Members:


Research Methodology and Study Design

Working Group Co-Chairs:

Working Group Members:


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Related Funding Opportunities

NIH-sponsored Funding Opportunity Announcements (FOA’s) related to hematopoietic cell transplantation survivorship research include:

  • Mechanisms of Cancer and Treatment-Related Symptoms and Toxicities - expires September 8, 2019
  • NCI Pediatric Provocative Questions (PPQ) - expires November 25, 2017
    • PPQ #7 - How can predication models be developed and used to identify patients at highest risk of treatment-related complications
    • PPQ #9 - What are the underlying molecular mechanisms that cause accelerated aging seen in some pediatric cancer survivors?
    • PAR-16-218 (R01)External Web Site Policy
    • PAR-16-217 (R21)External Web Site Policy
  • Improving Outcomes in Cancer Treatment-Related Cardiotoxicity - expires January 8, 2019
  • Intervening with Cancer Caregivers to Improve Patient Health Outcomes and Optimize Health Care Utilization - expires April 12, 2019
  • Examination of Survivorship Care Planning Efficacy and Impact - expires January 8, 2019
  • Dissemination and Implementation Research in Health - expires May 8, 2019
  • NIH Research Project Grant (Parent R01) - expires September 6, 2018
  • Exploratory Grants in Cancer Epidemiology and Genomics Research - expires May 8, 2019
  • NCI Small Grants Program for Cancer Research (NCI Omnibus R03) - expires January 8, 2020

EGRP joins with other NCI Divisions, Offices, and Centers and other Institutes and Centers at the National Institutes of Health (NIH) to fund grant applications submitted in response to FOAs. View the full list of EGRP FOAs.

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Contact

For further information or questions, contact Nonniekaye Shelburne, C.R.N.P., M.S., A.O.C.N., Program Director, Clinical and Translational Epidemiology Branch, Epidemiology and Genomics Research Program, NCI.

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