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.
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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…
Inflammatory stromal and T cells mediate human bone marrow niche remodeling in clonal hematopoiesis and myelodysplasia
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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)…
Inhibiting ferroptosis enhances ex vivo expansion of human haematopoietic stem cells
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Abstract Improved ex vivo expansion of human haematopoietic stem cells (HSCs) would considerably advance transplantation and genome-engineered therapies, yet existing culture methods still allow substantial HSC loss. Here we show that this attrition is driven largely by…
Outpatient Low-Dose Venetoclax-Azacitidine vs. Intensive Therapy in Newly Diagnosed AML in Resource-Limited Settings
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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)…
Rapid clonal selection within early hematopoietic cell compartments presages outcome to ivosidenib combination therapy
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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…
Cyclophosphamide and Cyclosporin for GVHD Prevention
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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…
Azacitidine as therapy for VEXAS syndrome
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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…
Luspatercept for Patients with Lower-Risk Myelodysplastic Syndromes/Neoplasms: A Systematic Review and Meta-Analysis
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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…
A 30-gene classifier distinguishes low-risk MDS HSPCs from healthy HSPCs
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Highlights • Single-cell RNA-sequencing performed on 16 lower-risk myelodysplastic syndrome (LR-MDS) bone marrow-derived mononuclear cells (BMMCs) • A 30-gene signature distinguishes LR-MDS hematopoietic stem and progenitor cells (HSPCs) from Healthy HSPCs • Vesicular trafficking…
Finding His Way Through: Evan’s Story of PNH
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Meet Evan. In a recent conversation with the Aplastic Anemia and MDS International Foundation, he shared his journey from paroxysmal nocturnal hemoglobinuria (PNH) diagnosis through treatment and survival. Evan, please tell us a little about yourself. I’m 31 years old. I’m based…
