News and Treatment Updates
Here's where you'll find a regularly updated, broad range of articles written by the AAMDSIF team, allied health organizations and news organizations. By staying well-informed, patients and families are practicing a form of self-support that will help them be more effective self-advocates when engaging with health care providers.
Oppose 57% Cut to Congressionally Directed Medical Research Programs
Originally Published: 03/10/2025
Article Source: Community Impact
Oppose 57% Cut to Congressionally Directed Medical Research Programs
Take Action Before House Vote
Oppose 57% Cut to Congressionally Directed Medical Research Programs
Tell your Members of Congress to Vote NO on The Full-Year Continuing Appropriations and Extensions Act, 2025
As early as this Tuesday, the House will be voting on a continuing resolution to fund government programs through the end of fiscal year 2025 (FY25).
This legislation cuts FY25 funding for the Department of Defense’s Congressionally Directed Medical Research Program (CDMRP) from $1.509 billion to $650 million – a 57...
Real-World Outcomes of Relapsed/Refractory Core-Binding Factor Acute Myeloid Leukemia: A COMMAND Registry Study
Originally Published: 03/10/2025
Article Source: External Web Content
To the Editor,
Core-binding factor acute myeloid leukemia (CBF-AML) is characterized by the presence of inv(16)/t(16;16) or t(8;21) and is classified as favorable risk by the 2022 European LeukemiaNet (ELN) guidelines [1]. We have previously reported on outcomes of patients with newly diagnosed CBF-AML treated with intensive chemotherapy (IC) regimens [2] and despite the favorable risk status, approximately 50% of patients experienced relapse. Prior analyses have shown limited survival after relapse [3-5]. Khan et al. reported on 92 patients with relapsed/refractory (R/R) CBF-AML treated...
Standardization of Bone Marrow Reporting for Myelodysplastic Syndromes/Neoplasms on Behalf of the International Consortium for Myelodysplastic Syndromes/Neoplasms
Originally Published: 03/10/2025
Article Source: External Web Content
Abstract
Context.—: Standardized bone marrow reporting specifically for myelodysplastic syndromes/neoplasms (MDS) is currently lacking in the literature and much needed in practice.
Objective.—: To propose a standardized approach to MDS evaluation in bone marrow specimens by (1) enhancing interinstitutional and intrainstitutional collaborations and clinical decision-making among hematopathologists and clinical hematologists and (2) allowing for efficient data extraction for clinical trials, institutional databases, and registry templates. This suggested approach is summarized in a modifiable...
Letter on the Bipartisan Senate FY25 Labor-HHS-Education Appropriations Bill
Originally Published: 02/24/2025
Article Source: Coalition Action
AAMDSIF is Taking a Stand for NIH Research
Research programs led by the National Institutes of Health (NIH) forge the way for life-saving new treatments for rare diseases such as aplastic anemia, MDS, and PNH. With clinical research locations throughout the U.S., researchers and clinicians discover and refine treatments for these patients, bringing hope to the bone marrow failure community.
The recent cuts to funding through the NIH have real-life impact on researchers and clinicians as well as the patients enrolled in clinical trials and receiving treatment for these diseases.
With...
Clinical-genomic profiling of MDS to inform allo-HSCT:Recommendations from an international panel on behalf of the EBMT
Originally Published: 02/19/2025
Article Source: External Web Content
For patients with myelodysplastic neoplasm/syndrome (MDS), allogeneic hematopoietic cell transplantation (allo-HCT) represents the only potentially curative treatment, capable of eradicating disease-related mutant hematopoietic cells and establishing normal donor hematopoiesis. Biologic-assignment clinical trials have indicated that in eligible patients, allo-HCT is associated with superior clinical outcomes compared to non-transplant therapy. However, this therapeutic option is only available to a subset of patients, and the outcome is influenced by multiple factors inherent to the patient...
How is transfusion dependence evolving in anemia treatment in MDS?
Originally Published: 02/19/2025
Article Source: External Web Content
A roundtable discussion on anemia management in myelodysplastic syndromes. Featuring moderator Guillermo Garcia-Manero, MD, of the University of Texas MD Anderson Cancer Center; Amy DeZern, MD, MHS, Johns Hopkins Medicine; Tiffany Tanaka, MD, of the University of California San Diego; and Uma Borate, MBBS, of Ohio State University.
The following is an unedited transcript:
I’m going to go back to something that you started to mention that I think is where the field is going. You were talking about transfusion dependent versus transfusion independent, and then Uma is the world expert on CHIP...
What are some of the new therapies discussed at ASH 2024?
Originally Published: 02/18/2025
Article Source: External Web Content
A roundtable discussion on anemia management in myelodysplastic syndromes featuring moderator Guillermo Garcia-Manero, MD, of the University of Texas MD Anderson Cancer Center; Amy DeZern, MD, MHS, Johns Hopkins Medicine; Tiffany Tanaka, MD, of the University of California San Diego; and Uma Borate, MBBS, of Ohio State University.
The following is a transcript:
So let’s talk about some of the new drugs and some of the presentations at ASH to start. So what’s happening now? We have luspatercept, imetelstat we didn’t talk about the IDH1 inhibitor, but may not be 100% related to what we do....
Maintenance Therapy in AML: What Is the Future Potential?
Originally Published: 02/17/2025
Article Source: External Web Content
ABSTRACT
Over the last decade, there have been significant advancements in the treatment for patients with acute myeloid leukemia (AML) including the addition of novel, targeted agents to intensive or nonintensive chemotherapy regimens. However, despite this, the majority of patients will still ultimately relapse and long-term survival remains poor. While the use of maintenance therapy has emerged as a potential strategy to maintain more durable remissions and improve overall survival, the optimal use of these therapies has not yet been clearly defined. In this review, we provide a...
Therapeutic Advances and Future of Therapy in Acute Myeloid Leukemia
Originally Published: 02/17/2025
Article Source: External Web Content
Significant progress in the characterization of molecular pathogenic events in acute myeloid leukemia (AML) has led to better characterization of prognosis and identification of subsets that are more likely to benefit from currently available strategies. Furthermore, deciphering these molecular pathogenic events has led to the development of a number of effective molecularly targeted agents that have significantly improved our armamentarium in managing patients with AML. This has certainly provided us with opportunities for improving outcomes but at the same time has created new challenges...
ACROBAT interim results support AlloHeme for relapse prediction in AML, MDS
Originally Published: 02/12/2025
Article Source: External Web Content
Interim results of the ACROBAT study suggest that increasing microchimerism and loss of complete chimerism may indicate relapse risk in patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS). The findings were presented in an oral abstract1 at the 2025 Transportation and Cellular Therapy Tandem Meetings of ASTCT and CIBMTR in Honolulu, Hawaii.
The ACROBAT study is a prospective, multicenter, observational cohort study that enrolled patients 18 years and older who were eligible for allogeneic hematopoietic stem cell transplantation (HSCT) with acute myeloid leukemia (...