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News and Treatment Updates

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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.

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...

Hematological Response to Frontline Treatment in Lower Risk Myelodysplastic Syndromes (LRMDS) is Associated With Better Overall Survival

Originally Published: 02/23/2025
Article Source: External Web Content
Section snippets Background Myelodysplastic neoplasms (MDS) are a heterogenous group of hematopoietic stem cell malignancies characterized by ineffective hematopoiesis and tendency to progress to acute myeloid leukemia (AML).1 Patients (pts) are risk stratified to tailor treatment according to disease risk. Historically using International Prognostic Scoring System (IPSS), two thirds of pts are classified as lower risk MDS (LR-MDS).2 Utilizing modern risk stratification tools such as the revised IPSS (IPSS-R) or molecular  Methods We included LR-MDS pts (IPSS-R very low and low) from Moffitt...

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 (...

Jane Biehl: Moving Slower and Slower After Myelodysplastic Syndrome

Originally Published: 02/12/2025
Article Source: External Web Content
I have honestly been frustrated lately. I am slowing down increasingly, and it has even become noticeable to others. I wrote an article in November about how nice it was to slow down and smell the roses. But now it seems like too much. In my previous life (before cancer), I moved steadily and effortlessly from working all day to classes at night. I could hurry up by running late and still be on time. Now, I am frequently late to meetings with friends, church and even appointments. I finally realized that I was miscalculating how long it would take me to get dressed and out the door. When I...

Genome sequencing in the management of myelodysplastic syndromes and related disorders

Originally Published: 02/11/2025
Article Source: External Web Content
Abstract Myeloid neoplasms originate from the clonal proliferation of hematopoietic stem cells, which is driven by the acquisition of somatic genetic mutations. Within these disorders, myelodysplastic syndromes (MDS) are specifically characterized by morphological abnormalities (dysplasia) and impaired maturation of myeloid precursors (ineffective hematopoiesis), resulting in peripheral blood cytopenia. Several studies have advanced the field of MDS, with a few landmark papers leading to a paradigm shift, opening new avenues of research and enabling a molecular revolution. These seminal...