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.
Study Connects Agent Orange Exposure to Earlier and More Severe Cases of Myelodysplastic Syndrome
Originally Published: 12/08/2025
Summary
Many veterans and doctors have long questioned whether Agent Orange (AO) exposure during the Vietnam War contributed to cases of myelodysplastic syndrome (MDS), a type of blood cancer.
The contaminated chemical is linked to several cancers, but a link to MDS was unclear. This created real barriers for exposed veterans with MDS who are seeking care and disability benefits.
Now, a new national study shows for the first time that people exposed to Agent Orange face a higher risk of developing MDS, tend to develop it earlier and often have more aggressive disease that is more likely to...
Studies Point to a Growing Role for New Therapies and Immunotherapies in Treating Blood Cancers
Originally Published: 12/07/2025
Chemotherapy-free approaches could offer comparable efficacy with less toxicity
(ORLANDO, Dec. 7, 2025) — Targeted therapies and immunotherapies are increasingly offering viable alternatives to the chemotherapies that have stood for decades as a mainstay of treatment for individuals living with blood cancers, according to studies presented at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition.
“These studies point to the departure of traditional chemotherapy and shedding traditional approaches,” said Laura Michaelis, MD, professor of medicine at the Medical College...
Inhibition of heme biosynthesis triggers cuproptosis in acute myeloid leukemia
Originally Published: 11/19/2025
Highlights
Heme biosynthesis enzymes are variably suppressed in AML
Heme levels are linked with altered leukemic transcriptional programs via BACH1
Heme biosynthesis is a selective dependency in AML, both in vitro and in vivo
Heme starvation disrupts complex IV, inducing copper accumulation and cuproptosis
Summary
The ubiquitous metabolite heme has diverse enzymatic and signaling functions in most mammalian cells. Through integrated analyses of mouse models, human cell lines, and primary patient samples, we identify de novo heme biosynthesis as a selective dependency in...
Inflammatory stromal and T cells mediate human bone marrow niche remodeling in clonal hematopoiesis and myelodysplasia
Originally Published: 11/18/2025
Abstract
Somatic mutations in hematopoietic stem/progenitor cells (HSPCs) can lead to clonal hematopoiesis of indeterminate potential (CHIP) and progression to myelodysplastic syndromes (MDS). Using single-cell and anatomical profiling of a large cohort of human bone marrow (BM), we show that the HSPC BM niche in CHIP and MDS is undergoing inflammatory remodeling. This includes loss of CXCL12⁺ adipogenic stromal cells and the emergence of a distinct population of inflammatory mesenchymal stromal cells (iMSCs), which arise in CHIP and become more prevalent in MDS. Functional studies in...
Outpatient Low-Dose Venetoclax-Azacitidine vs. Intensive Therapy in Newly Diagnosed AML in Resource-Limited Settings
Originally Published: 11/06/2025
Key points
Outpatient low-dose VenAza plus itraconazole reduced early mortality and hospitalizations while maintaining treatment efficacy.
VenAza offers a safer, cost-effective alternative to intensive chemotherapy in newly diagnosed AML.
Abstract
Acute myeloid leukemia (AML) treatment in fit adults traditionally relies on intensive chemotherapy, but access to supportive care and inpatient capacity limits its feasibility in low-resource settings. Venetoclax, a BCL-2 inhibitor, has shown promise in unfit AML patients, but its role in induction-eligible adults remains unclear. We...
Rapid clonal selection within early hematopoietic cell compartments presages outcome to ivosidenib combination therapy
Originally Published: 11/05/2025
Key Points
Ivosidenib, venetoclax ± azacitidine either rapidly selects evolved, resistant leukemic clones or eradicates leukemic clones.
Resistant clones express RNA signatures of stemness, branched-chain amino acid metabolism, and menin targets.
Subjects:Myeloid Neoplasia
Acquired resistance to targeted, non-intensive therapies is common in myeloid malignancies. However, the kinetics of selection, the hematopoietic cell compartments where selection occurs, and the molecular mechanisms underlying selection remain open questions. To address this, we studied the kinetics of clonal and...
Cyclophosphamide and Cyclosporin for GVHD Prevention
Originally Published: 10/01/2025
Curtis et al. (July 17 issue)1 report evidence of longer graft-versus-host disease (GVHD)–free, relapse-free survival with post-transplantation cyclophosphamide–based prophylaxis than with cyclosporin–methotrexate among patients receiving a stem-cell transplant from a matched related donor after myeloablative conditioning or reduced-intensity conditioning.
We recently published real-world data with respect to post-transplantation cyclophosphamide–based prophylaxis in patients receiving a transplant from a matched sibling donor and similarly observed longer GVHD-free, relapse-free survival...
Azacitidine as therapy for VEXAS syndrome
Originally Published: 09/18/2025
How should patients with VEXAS (an acronym for vacuoles [in myeloid and erythroid precursors], E1 enzyme, X-linked, autoinflammatory, somatic) syndrome be treated? Until now, we have only had case reports and small series to guide us. In this issue of Blood, Jachiet and colleagues from the French VEXAS group FRENVEX retrospectively describe their experience with azacitidine therapy in 88 patients with genetically confirmed VEXAS syndrome.1 Improvements in inflammatory signs and symptoms, blood counts, or both disease markers were observed in more than 60% of patients, and the median duration...
Luspatercept for Patients with Lower-Risk Myelodysplastic Syndromes/Neoplasms: A Systematic Review and Meta-Analysis
Originally Published: 09/18/2025
Luspatercept has emerged as a novel therapy for anemia in transfusion-dependent (TD) lower-risk myelodysplastic syndromes (LR-MDS). This systematic review and meta-analysis aims to evaluate the efficacy and safety of luspatercept in LR-MDS. We conducted a systematic review and meta-analysis to evaluate the efficacy and safety of luspatercept in TD LR-MDS. Six databases were searched through March 2025 to find relevant material. Studies were screened and extracted by two independent authors. A total of 20 studies encompassing 3,455 patients were included in the analysis. The pooled 8-week...
A 30-gene classifier distinguishes low-risk MDS HSPCs from healthy HSPCs
Originally Published: 09/17/2025
Highlights
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Single-cell RNA-sequencing performed on 16 lower-risk myelodysplastic syndrome (LR-MDS) bone marrow-derived mononuclear cells (BMMCs)
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A 30-gene signature distinguishes LR-MDS hematopoietic stem and progenitor cells (HSPCs) from Healthy HSPCs
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Vesicular trafficking-related gene modules are enriched in MDS
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Certain trafficking genes such as LRBA are expressed exclusively in HSPCs
Myelodysplastic syndromes (MDSs) are a group of malignant clonal disorders that are characterized by functional impairment of hematopoiesis, morphologic dysplasia, and genetic...
