First-line BMT for Severe Aplastic Anemia
NMDP℠ Explore Cell Therapy podcast season 2, episode 11
In this two-part "NMDP Explore Cell Therapy Podcast" episode, Amy DeZern, MD, MHS, and Christin DeStefano, MD, discuss the latest innovation in the treatment of severe aplastic anemia (SAA), using bone marrow transplantation (BMT) as a first-line treatment and moving beyond traditional immunosuppressive therapies (ISTs).
Experts discuss the benefits of haploidentical BMT for SAA, noting quicker recovery times and reduced complications compared to past methods. We explore how these advancements are particularly impactful for diverse populations, including military personnel, and underscore the critical importance of early referral and coordination between hematology/oncology and transplant centers. Listen to discover how first-line BMT is offering a potential cure to patients with SAA.
Listen to Part 1: Outcomes and Equity
Listen to Part 2: Future Research and Care Coordination
Key takeaways
- Transition to first-line BMT: The episode highlights a potential shift from IST to BMT as the first-line treatment for SAA, especially using haploidentical (haplo) donors. This change is credited with faster recovery and higher efficacy, evidenced by increased engraftment and reduced need for transfusions.
- Challenges with IST: IST, a common treatment for SAA, faces issues like high relapse rates, severe side effects, and potential development of myelodysplastic syndromes (MDS) upon withdrawal, highlighting the need for alternative treatments like BMT.
- Improving access and outcomes: There's a significant focus on increasing access to care for racially and ethnically diverse populations, and for patients in the military. Haplo BMT is viewed as a potential curative option for patients who previously had limited treatment choices.
- Importance of early referral: Guests emphasize the critical need for early referral to transplant centers, which enables quicker treatment and better coordination between hematology/oncology and transplant specialists, thus improving overall outcomes.
- Future directions and trials: Looking ahead, there's excitement about ongoing trials like the Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 2207, which aims to expand the use of mismatched related and unrelated donors across a multicenter trial, expanding a proactive approach to treatment and care coordination for SAA patients.
This episode's expert guests
Amy DeZern, MD, MHS
Professor of Oncology and
Medicine, Division of
Hematologic Malignancies
Johns Hopkins Medicine
Dr. DeZern’s primary clinical and research interests are focused on bone marrow failure disorders. She has expertise in the diagnosis and treatment of MDS, aplastic anemia (AA) and paroxysmal nocturnal hemoglobinuria (PNH) as well as acute leukemias. She has been the principal investigator for over 40 single center and multicenter clinical trials in MDS and AA.
Christin DeStefano, MD
Hematology/Oncology Division
Director, Department of Medicine
Uniformed Services University
Dr. DeStefano is a hematologist/oncologist at Walter Reed National Military Medical Center and a lieutenant colonel in the United States Air Force. She also serves as the Hematology/Oncology Fellowship Program director and hematology/oncology consultant to the Air Force surgeon general. Her research interests deal with myeloid and lymphoid malignancies and cancer epidemiology.
Disclaimer: The contents of this podcast are the sole responsibility of the author(s) and do not necessarily reflect the views, opinions or policies of Uniformed Services University of the Health Sciences (USUHS), the Department of Defense (DoD), or the Departments of the Army, Navy or Air Force. Mention of trade names, commercial products or organizations does not imply endorsement by the U.S. Government.
Listen to Part 1: Outcomes and Equity
Listen to Part 2: Future Research and Care Coordination
Access all "Explore Cell Therapy" episodes
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Featured articles and clinical trials discussed in this episode
- Alternative donor BMT with posttransplant cyclophosphamide as initial therapy for acquired severe aplastic anemia (Blood, June 2023)
- Two national trials poised to expand the role of bone marrow transplant in new diagnosed severe aplastic anemia (The Hematologist, October 2023)
- Haploidentical bone marrow transplantation in patients with relapsed or refractory severe aplastic anaemia in the USA (BMT CTN 1502): a multicentre, single-arm, phase 2 trial (The Lancet Haematology, July 2022)
Resources for hematology/oncology and transplant physicians
This new and improved app features our latest HCT Consultation Timing Guidelines, Post-Transplant Care Guidelines, tips to help identify GVHD, CME opportunities and more.
Transplant Center Directory for hematology/oncology physicians
This searchable database provides details on the transplant centers, such as center contact information, number of transplants by cell source, patient survival information and more.
CIBMTR® Summary Slides and Reports
CIBMTR data on outcomes and trends are summarized and made available for clinical decision-making, presentations, and research planning. CIBMTR is a research collaboration between NMDP and Medical College of Wisconsin®.
Resources for your patients [H2]
The NMDP Patient Support Center provides support, information and resources for transplant patients, caregivers and families. All our programs and resources are free.
Jason Carter Clinical Trials Search and Support
The Clinical Trials Search and Support program helps patients find and join clinical trials through one-on-one support, an easy-to-navigate website, and financial assistance to help patients pay for travel costs to participate in a clinical trial.