NIH Blood & Marrow Transplant Late Effects Consensus Conference
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.
Nonniekaye Shelburne, C.R.N.P., M.S., A.O.C.N.
Program Director, Clinical and Translational Epidemiology Branch
nshelburne@mail.nih.gov
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.
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.
Agenda
View agenda for Tuesday, June 21| Time | Topic |
|---|---|
| 7:30 a.m. - 8:00 a.m. | Check-In |
| 8:00 a.m. - 8:30 a.m. |
Opening Remarks Richard Childs, M.D. Ronald Gress, M.D. |
| 8:30 a.m. - 8:40 a.m. |
Initiative Overview Bipin Savani, M.D. |
| 8:40 a.m. - 8:50 a.m. |
Conference Overview and Panel Activities |
| 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
|
| 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
|
| 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
|
| 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
|
| 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 |
| 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
|
| 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
|
| 11:10 a.m. - 11:40 a.m. |
NIH Funding Opportunities to Support Consensus Recommendations Nahed El Kassar, Ph.D., M.D. |
| 11:40 a.m. - 12:00 p.m. |
Meeting Summary & Next Steps Nonniekaye Shelburne, C.R.N.P., M.S., A.O.C.N |
| 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 |
Steering Committee
- Minoo Battiwalla, M.D., National Heart Lung and Blood Institute (NHLBI)
- Shahrukh Hashmi, M.D.
, Mayo Clinic - Navneet Majhail, M.D., M.S., Cleveland Clinic
- Steve Pavletic, M.D., M.S., National Cancer Institute
- Bipin Savani, M.D., Vanderbilt University School of Medicine
- Nonniekaye Shelburne, C.R.N.P., M.S., A.O.C.N., National Cancer Institute
Working Groups
Subsequent Neoplasms
Working Group Co-Chairs:
- Linda Burns, M.D., National Marrow Donor Program
- Lindsay Morton, Ph.D., National Cancer Institute
Working Group Members:
- Scott Baker, M.D., M.S., Fred Hutchinson Cancer Research Center
- John Barrett, M.D., National Heart, Lung and Blood Institute
- Smita Bhatia, M.D., M.P.H., University of Alabama Birmingham
- Eric Engels M.D., M.P.H., National Cancer Institute
- Shahinaz Gadalla, M.D., PhD., National Cancer Institute
- David Kleiner, M.D., Ph.D., National Cancer Institute
- Steven Pavletic, M.D., National Heart, Lung and Blood Institute
- Wael Saber, M.D., M.S., CIBMTR/Medical College of Wisconsin
Psychosocial and Quality of Life Outcomes
Working Group Co-Chairs:
- Margaret Bevans, R.N., Ph.D., NIH Clinical Center
- Karen Syrjala, Ph.D., Fred Hutchinson Cancer Research Center
Working Group Members:
- Ellen Denzen, M.S., National Marrow Donor Program
- Areej El-Jawahri, M.D., Massachusetts General Hospital
- Flora Hoodin, Ph.D., M.A., University of Michigan Ann Arbor
- Matt Hsieh, M.D., National Heart, Lung and Blood Institute
- Paul Jacobsen, Ph.D., Moffitt Cancer Center
- Erin Kent, Ph.D., M.S., National Cancer Institute
- Stephanie Lee, M.D., M.P.H., Fred Hutchinson Cancer Research Center
- Nonniekaye Shelburne, C.R.N.P., M.S., National Cancer Institute
- Kate Tierney, R.N., Ph.D., Stanford Cancer Institute
- Lori Wiener, Ph.D., National Cancer Institute
- Bill Wood, M.D., University of North Carolina
Immune Dysregulation and Pathobiology
Working Group Co-Chairs:
- Juan Gea-Banacloche, M.D., National Cancer Institute
- John Wingard, M.D., University of Florida
Working Group Members:
- Paul Carpenter, M.D., Fred Hutchinson Cancer Research Center
- Nahed El Kassar, Ph.D., M.D., National Heart, Lung and Blood Institute
- Linda Griffith, M.D., Ph.D., National Institute of Allergy and Infection Diseases
- Krishna Komanduri, M.D., University of Miami
- Robert Le, M.D., Ph.D., Food and Drug Administration
- Sophie Paczesny, M.D., Ph.D., Indiana University
- Stefanie Sarantopoulous, M.D., Ph.D., Duke Cancer Institute
- Sharon Savage, M.D., National Cancer Institute
- Bipin Savani, M.D., Vanderbilt University Medical Center
- Kirk Schultz, M.D., BC Children's Hospital
- Jo-Anne Young, M.D., University of Minnesota
Cardiovascular and Metabolic Impairment
Working Group Co-Chairs:
- Saro Armenian, D.O., M.P.H., City of Hope
- Minoo Battiwalla, M.D., National Heart, Lung and Blood Institute
Working Group Members:
- Wassim Chemaitilly, M.D., St. Jude's Children's Research Hospital
- Marcus Chen, M.D., National Heart, Lung and Blood Institute
- Eric Chow, M.D., Fred Hutchinson Cancer Research Center
- Christine Duncan, M.D., Dana Farber Cancer Institute
- Lee Jones, Ph.D., Memorial Sloan Kettering Cancer Center
- Mike Pulsipher, M.D., Children's Hospital of Los Angeles
- Alan Remaley, M.D., Ph.D., National Heart, Lung and Blood Institute
- Alicia Rovo, M.D., University Hospital Bern
- Nina Salooja, M.D., Hammersmith
Healthcare Delivery
Working Group Co-Chairs:
- Shahrukh Hashmi, M.D., Mayo Clinic
- Navneet Majhail, M.B.B.S., M.D., M.S., Cleveland Clinic
Working Group Members:
- Chris Bredeson, M.D., M.Sc., ASBMT / University of Ottawa
- Rafael Duarte, M.D., Ph.D., European Society for Blood and Marrow Transplantation
- Stephanie Farnia, M.P.H., National Marrow Donor Program
- Sue Ferrey, M.D., Moffitt Cancer Center
- Courtney Fitzhugh, M.D., National Heart, Lung and Blood Institute
- Mary Flowers, M.D., Fred Hutchinson Cancer Research Center
- Jim Gajewski, M.D., Ph.D., Oregon Health Sciences Center Portland
- Dennis Gastineau, M.D., Mayo Clinic
- Melissa Greenwald, M.D., Health Resources and Services Administration
- Madan Jagasia, M.D., M.B.B.S., M.S., Vanderbilt University Medical Center
- Patricia Martin, R.N., B.S.N., Anthem BlueCross BlueShield
- Scott Ramsey, M.D., Ph.D., Fred Hutchinson Cancer Research Center
- Doug Rizzo, M.D., M.S., Medical College of Wisconsin
- Kim Schmit-Pokorny, R.N., M.S.N., University of Nebraska
Research Methodology and Study Design
Working Group Co-Chairs:
- Smita Bhatia, M.D., M.P.H., University of Alabama Birmingham
- Bronwen Shaw, M.D., Ph.D., Medical College of Wisconsin
Working Group Members:
- Greg Armstrong, M.D., M.S.C.E., St Jude's Children's Research Hospital
- Terri Hahn, Ph.D., Roswell Park Cancer Institute
- Paul Martin, M.D., Fred Hutchinson Cancer Research Center
- Sandy Mitchell, Ph.D., C.R.N.P., National Cancer Institute
- Effie Petersdorf, M.D., Fred Hutchinson Cancer Research Center
- Nonniekaye Shelburne, C.R.N.P., M.S., National Cancer Institute
- Barry Storer, Ph.D., Fred Hutchinson Cancer Research Center
Conference-Related Publications
Battiwalla M, Hashmi S, Majhail N, Pavletic S, Savani BN, Shelburne N. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: Developing Recommendations to Improve Survivorship and Long-Term Outcomes. Biol Blood Marrow Transplant. 2017 Jan;23(1):6-9.
Morton LM, Saber W, Baker KS, Barrett AJ, Bhatia S, Engels EA, Gadalla SM, Kleiner DE, Pavletic S, Burns LJ. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: The Subsequent Neoplasms Working Group Report. Biol Blood Marrow Transplant. 2017 Mar;23(3):367-378.
Bevans M, El-Jawahri A, Tierney DK, Wiener L, Wood WA, Hoodin F, Kent EE, Jacobsen PB, Lee SJ, Hsieh MM, Denzen EM, Syrjala KL. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: The Patient-Centered Outcomes Working Group Report. Biol Blood Marrow Transplant. 2017 Apr;23(4):538-551.
Gea-Banacloche J, Komanduri KV, Carpenter P, Paczesny S, Sarantopoulos S, Young JA, El Kassar N, Le RQ, Schultz KR, Griffith LM, Savani BN, Wingard JR. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: The Immune Dysregulation and Pathobiology Working Group Report. Biol Blood Marrow Transplant. 2017 Jun;23(6):870-881.
Armenian SH, Chemaitilly W, Chen M, Chow EJ, Duncan CN, Jones LW, Pulsipher MA, Remaley AT, Rovo A, Salooja N, Battiwalla M. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: The Cardiovascular Disease and Associated Risk Factors Working Group Report. Biol Blood Marrow Transplant. 2017 Feb;23(2):201-210.
Hashmi SK, Bredeson C, Duarte RF, Farnia S, Ferrey S, Fitzhugh C, Flowers MED, Gajewski J, Gastineau D, Greenwald M, Jagasia M, Martin P, Rizzo JD, Schmit-Pokorny K, Majhail NS. National Institutes of Health Blood and Marrow Transplant Late Effects Initiative: The Healthcare Delivery Working Group Report. Biol Blood Marrow Transplant. 2017 May;23(5):717-725.
Shaw BE, Hahn T, Martin PJ, Mitchell SA, Petersdorf EW, Armstrong GT, Shelburne N, Storer BE, Bhatia S. National Institutes of Health Hematopoietic Cell Transplantation Late Effects Initiative: The Research Methodology and Study Design Working Group Report. Biol Blood Marrow Transplant. 2017 Jan;23(1):10-23.
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
- Improving Outcomes in Cancer Treatment-Related Cardiotoxicity (Clinical Trial Optional) - expires January 8, 2022
- Intervening with Cancer Caregivers to Improve Patient Health Outcomes and Optimize Health Care Utilization - expires April 12, 2019
- Dissemination and Implementation Research in Health - expires May 8, 2019
- Exploratory Grants in Cancer Epidemiology and Genomics Research - expires May 8, 2019
- Improving Outcomes for Pediatric, Adolescent and Young Adult Cancer Survivors (U01 Clinical Trial Required) – expires January 4, 2020
- NCI Small Grants Program for Cancer Research (NCI Omnibus R03 Clinical Trial Optional) - expires January 8, 2020
- Cancer Prevention and Control Clinical Trials Grant Program (R01 Clinical Trial Required) – expires January 8, 2021
- Modular R01s in Cancer Control and Population Science (R01 Clinical Trial Optional) – expires March 9, 2021
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