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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…
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Chemotherapy
Chemotherapy:
(kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People with high-risk or intermediate-2 risk myelodysplastic syndrome (MDS) may be given chemotherapy to kill bone marrow cells that have an abnormal size, shape, or look. Chemotherapy hurts healthy cells along with…
-free approaches could offer comparable efficacy with less…
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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
chemotherapy:
(kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People with high-risk or intermediate-2 risk myelodysplastic syndrome (MDS) may…
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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 accumulation and cuproptosis
Summary…
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Abstract
Somatic mutations in hematopoietic stem/progenitor cells (HSPCs) can lead to clonal
hematopoiesis
hematopoiesis:
(hi-mat-uh-poy-EE-suss) The process of making blood cells in the bone marrow.
of indeterminate potential (CHIP) and progression to
myelodysplastic syndromes
myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the…
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Key Points
Ivosidenib
Ivosidenib:
A small molecule inhibitor of isocitrate dehydrogenase-1 (IDH1), which is mutated in several forms of cancer, including acute myeloid leukemia (AML).
,
venetoclax
venetoclax:
Venetoclax is used to treat chronic lymphocytic leukemia or small lymphocytic leukemia in adults. Venetoclax is used alone or in combination with other cancer medicines…
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Recordings:
Aplastic Anemia
Low Risk MDS
High Risk MDS to AML
Transplant and GVHD
MDS/MPN Overlap
A Patient Story: Shauna McMillian
Managing PNH Complications
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Session Recordings Available:
MDS to MPN Overlap from the 2025 Boston Patient and Family Conference
High Risk MDS from the 2025 Boston Patient and Family Conference
Low Risk MDS from the 2025 Boston Patient and Family Conference
What is Aplastic Anemia? From the 2025 Boston Patient and Family Conference
What is PNH? Webinar recorded at the 2025 Boston Patient and Family Conference…
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Here are links to the conference sessions from the 2025 Kansas City Patient and Family Conference:
MDS/MPN Overlap
PNH and Current Therapies
Living Well with Bone Marrow Failure
Judy's Story of PNH
Transplant and GVHD
Aplastic Anemia
PNH Complications
High Risk MDS
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Highlights
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Single-cell RNA-sequencing performed on 16 lower-risk myelodysplastic syndrome (LR-MDS)
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
-derived mononuclear cells (BMMCs)
•
A 30-gene signature distinguishes LR-MDS hematopoietic stem and progenitor cells (HSPCs) from Healthy…
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Curtis et al. (July 17 issue)1 report evidence of longer
graft-versus-host disease (GVHD)
graft-versus-host disease (GVHD):
Also called GVHD, it is a common complication of bone marrow/stem cell transplantation. It is caused when the donor's immune cells, now in the patient, begin to see the the patient's body as foreign and mount an immune response. GVHD most commonly effects the…
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UBTF-TD has been reported in a significant percentage of childhood MDS-EB and has been associated with inferior survival compared to that of patients with the wild-type gene. We treated three consecutive pediatric patients affected by UBTF-TD MDS-EB with
venetoclax
venetoclax:
Venetoclax is used to treat chronic lymphocytic leukemia or small lymphocytic leukemia in adults. Venetoclax is…
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Luspatercept has emerged as a novel therapy for
anemia
anemia:
(uh-NEE-mee-uh) A condition in which there is a shortage of red blood cells in the bloodstream. This causes a low red blood cell count. Symptoms of anemia are fatigue and tiredness.
in transfusion-dependent (TD) lower-risk
myelodysplastic syndromes
myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A…
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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 colleagues from the French VEXAS group FRENVEX retrospectively describe their experience with
azacitidine
azacitidine…
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Abstract
Pediatric
myelodysplastic syndromes
myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal growth or development. People with MDS have low blood cell count for at…
(MDS)…
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Abstract
Higher-risk myelodysplastic syndrome (HR-MDS) with RARA gene overexpression is a subset of patients (pts) with an actionable target for
tamibarotene
tamibarotene:
Tamibarotene (brand name: Amnolake), also called retinobenzoic acid, is orally active, synthetic retinoid, developed to overcome all-trans retinoic acid (ATRA) resistance, with potential antineoplastic activity…
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Dr. Rory Shallis explains the treatment,
Imetelstat
Imetelstat:
Who should not take RYTELO?
Before taking RYTELO, tell your healthcare provider about all your medical conditions, including if you:
are pregnant or plan to become pregnant. RYTELO may harm your unborn baby and may cause loss of pregnancy. Tell your healthcare provider right away if you…
, for patients with Low Risk MDS…
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Abstract
Clonal
hematopoiesis
hematopoiesis:
(hi-mat-uh-poy-EE-suss) The process of making blood cells in the bone marrow.
of indeterminate potential (CHIP) is associated with increased mortality and malignancy risk, yet the determinants of clonal expansion remain poorly understood. We performed sequencing at a depth of coverage of >4000× for CHIP mutations in 6976 postmenopausal…
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Meet Evan. In a recent conversation with the
Aplastic Anemia
Aplastic Anemia:
(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most…
and MDS…
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Please find all content at the link.
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