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Abstract
Improved ex vivo expansion of human haematopoietic
stem cells
stem cells:
Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem…
(HSCs)…
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Aged individuals with somatic TP53 mutations manifest clonal
hematopoiesis
hematopoiesis:
(hi-mat-uh-poy-EE-suss) The process of making blood cells in the bone marrow.
(CH) and are at high risk of developing myeloid neoplasms. However, the underlying mechanisms are not fully understood. Here we show that inflammatory stress confers a competitive advantage to p53 mutant hematopoietic…
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Abstract
NRAS and KRAS mutations, commonly identified alongside ancestral co-mutations, are generally regarded as pathogenic in adults presenting with monocytosis and/or
cytopenia
cytopenia:
(sie-tuh-PEE-nee-uh) A shortage of one or more blood cell types. Also called a low blood count.
(s). However, their significance in isolation is not well defined. We studied a multi-institutional…
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NIH
clinical trial
clinical trial:
A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a…
shows promise for people with treatment-limited, life-…
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Key Takeaways
The combination therapy achieved a 91% overall response rate, with 45% complete remission and 46% marrow complete remission rates.
Median overall survival was 30 months, with 1- and 3-year survival rates of 68.8% and 40.7%, respectively.
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Decitabine
Decitabine:
It works by reducing the amount of methylation in the body. Methylation is a process that acts like a…
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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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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
Conference Event last updated
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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…
Conference Event last updated
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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
Conference Event last updated
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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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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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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…
Podcast Episode last updated
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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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Abstract
The TP53 gene ensures genetic fidelity by regulating cell cycle kinetics and
apoptosis
apoptosis:
Programmed cell death.
. In myeloid neoplasms, TP53
mutation
mutation:
Any change or alteration in a gene. A mutation may cause disease or may be a normal variation. Paroxysmal nocturnal hemoglobinuria (PNH) occurs because of a mutation in the PIG-A gene of a single stem…
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Immunohistochemistry (IHC) could represent a biomarker for the early detection of TP53 mutations and for the prediction of a TP53 allelic state in patients with myelodysplastic neoplasms (MDS) and
acute myeloid leukemia
acute myeloid leukemia:
(uh-KYOOT my-uh-LOYD loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail…
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TP53 mutations are found in 10-15% of myeloid neoplasms and are one of its most important prognostic factors. Emerging data show that TP53 mutational allele status is a key determinant of clinical outcomes, with multi-hit TP53 mutant myeloid neoplasms having a very poor prognosis. Significant differences exist among the methods used in clinical and research settings to assess TP53 mutational…
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