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AbstractCalreticulin (CALR) frameshift mutations drive the majority of JAK2/MPL-wild-type cases of essential thrombocythemia and myelofibrosis, producing a shared novel C-terminus that activates the thrombopoietin receptor and leads to constitutive Janus kinase (JAK)/signal transducer and activator of transcription (STAT) signaling. CALR mutations transform the multifunctional…
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Relapse is the leading cause of allogeneic hematopoietic stem cell transplant (allo-HSCT) failure in myeloid neoplasms [1]. Patients receiving reduced-intensity conditioning (RIC) regimens have a higher risk of post-transplant relapse compared to those undergoing myeloablative conditioning (MAC) [2]. However, many patients are not candidates for MAC due to age, comorbidities, or frailty.…
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Abstract
Hypocellular
Hypocellular:
A condition in which there are too few cells, for example, within the bone marrow. Patients with aplastic anemia have hypocellular bone marrow.
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 aplastic…
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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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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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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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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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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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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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AbstractPurpose: To evaluate the efficacy and safety of the cluster of differentiation 47-targeted antibody magrolimab plus
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,…
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In a single-center phase I to II trial (SAVE) reported in the Journal of Clinical Oncology, Issa et al found that the all-oral combination of revumenib,
decitabine
decitabine:
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…
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TO THE EDITOR:In older patients with
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 cells and platelets. Some cases of MDS become…
(…
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Patients with
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…
,
paroxysmal nocturnal hemoglobinuria…
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