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Key PointsZiftomenib shows pronounced preclinical activity in the menin-KMT2A–dependent leukemia models, including patient-derived xenografts.Ziftomenib demonstrates potent activity against a subset of the drug-induced MEN1 mutations found in patients with
acute myeloid leukemia
acute myeloid leukemia:
(uh-KYOOT my-uh-LOYD loo-KEE-mee-uh) A cancer of the blood cells. It…
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HighlightsReduced cumulative post-transplant
cyclophosphamide
cyclophosphamide:
Cyclophosphamide is in a class of medications called alkylating agents. When used to treat cancer, it works by slowing or stopping the growth of cancer cells in your body. When cyclophosphamide is used to treat bone marrow failure, it works by suppressing your body's immune system.
(<…
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Key PointsMLL3 and MLL4 act redundantly in HSCs to sustain transcription factor and enhancer networks that support multipotency.Simultaneous loss of MLL3 and MLL4 drives hematopoietic progenitors into a uniform B-cell–like default state.AbstractHematopoietic
stem cells
stem cells:
Cells in the body that develop into other cells. There are two main…
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Key PointsPosttransplant
cyclophosphamide
cyclophosphamide:
Cyclophosphamide is in a class of medications called alkylating agents. When used to treat cancer, it works by slowing or stopping the growth of cancer cells in your body. When cyclophosphamide is used to treat bone marrow failure, it works by suppressing your body's immune system.
significantly reduced GVHD…
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ABSTRACTIntroductionCentral venous catheter (CVC) insertion is frequently required in patients with haematological diseases. Despite limited evidence,
thrombocytopenia
thrombocytopenia:
(throm-buh-sie-tuh-PEE-nee-uh) A condition in which there is a shortage of platelets in the bloodstream. This results in a low platelet count. Bleeding and bruising…
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AbstractMyeloproliferative neoplasms (MPNs) were previously thought to result solely from somatic mutations; however, familial clustering and germline genetic testing have revealed hereditary MPNs. These hereditary MPNs are one type of hereditary hematopoietic malignancy (HHM) syndrome, or hereditary blood cancer, and generally display an autosomal-dominant inheritance pattern…
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Key PointscHSPCTrack uses circulated human HSPCs to reconstruct clonal differentiation dynamicsHuman HSPCs clones differentiation varies stochastically in lineage output and differentiation rate even in controlled isolated environmentsHematopoietic stem and progenitor cells (HSPCs) balance self-renewal with on-demand differentiation of blood lineages. Modern
bone marrow…
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Key PointsChronic inflammation promotes transient myeloid differentiation in Tet2-deficient HSPCs, but accelerates HSC exhaustion and MDS development.NLRC4 inflammasome activation drives this process; Nlrc4 deletion delays MDS development and improves survival in Tet2-deficient mice.
Myelodysplastic syndromes
Myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-…
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AbstractPOEMS syndrome is a rare multisystem
plasma
plasma:
The fluid part of the blood. Plasma is mostly made of water with chemicals in it. These chemicals include proteins, hormones, minerals, and vitamins.
cell disorder for which evidence guiding management is largely derived from single-center retrospective series. We conducted a retrospective multicenter study…
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IntroductionPayers employ health care utilization–management strategies, including prior authorization (PA), to limit costs and promote evidence-based, guideline-concordant care. However, PA may be inconsistently applied and associated with patient confusion and frustration.1-3 Patient engagement in PA processes can add to administrative burden and may be associated with adverse…
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Hypomethylating agents–venetoclax as salvage therapy for relapse of AML and MDS after allogeneic HCT
Key PointsHMA-VEN is well tolerated in patients with AML/MDS who relapsed after alloHCT.HMA-VEN is effective for relapsed AML/MDS after alloHCT, even in patients who have failed prior VEN-based regimens.AbstractRelapse after allogeneic hematopoietic cell transplantation (alloHCT) remains a challenge for patients with
acute myeloid leukemia
acute myeloid…
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Background: Philadelphia-negative myeloproliferative neoplasms (MPNs), including essential thrombocythaemia (ET), polycythaemia vera (PV), and myelofibrosis (MF), usually affect older adults, but a subset occurs in adolescents and young adults (AYA). Regional age-stratified data remain limited.
Methods: We retrospectively studied Philadelphia-negative MPN patients managed at the National Center…
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Highlights -Considerable heterogeneity exists in germline testing implementation across hematopoietic stem cell transplantation centers in the United States.-Lack of standardized protocols affects the timing of testing and inconsistent sample collection workflows.-Barriers to consistent care include insurance challenges, logistical constraints, and…
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CONFERENCE AGENDA - November 7, 20268:00 - 2:45 (Central Time Zone)
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…
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Saturday, October 24, 2026Conference Co-ChairsUma Borate, MBBSAlice Mims, MDAplastic Anemia/PNH Agenda 7:30 - 8:15 Breakfast and Registration8:15 - 8:45 Welcome 8:45 - 9:15
Bone Marrow Failure
Bone Marrow Failure:
A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are…
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Weill Cornell Medicine investigators have developed a new AI-based method to assess patients with a form of blood cancer called myelodysplastic neoplasms. The method, published June 30 in Leukemia, compares the physical locations and morphometric (size and shape) characteristics of every hematopoietic cell in the patient’s
bone marrow
bone marrow:
The soft, spongy tissue inside most…
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AbstractIntroduction
Azacitidine
Azacitidine:
It works by reducing the amount of methylation in the body. Methylation is a process that acts like a switch to turn off or “silence” genes in certain cells. When these genes (called tumor suppressor genes) are turned off, MDS cells and cancer cells can grow freely. Azacitidine is approved by the U…
-venetoclax (AZA-VEN) has become a standard…
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ABSTRACTThe RACE study (NCT02009747) compared horse
antithymocyte globulin
antithymocyte globulin:
ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the first-line immunosuppressive therapy (IS) to treat patients with acquired aplastic anemia. In some cases it is used as a treatment for patients with MDS and PNH.
(hATG…
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SummaryReduced-intensity conditioning (RIC) is associated with a high relapse rate in high-risk acute myeloid leukaemia (AML) and
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.…
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For most of the last 50 years, the conversation with a patient with newly diagnosed
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 to mature. The blast cells stay in the bone marrow and become to numerous. This slows production of red blood…
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