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Summary
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 anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be…
syndromes (BMFS) are a diverse group of…
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Key PointsInfection risk in MDS is dynamic and biologically driven, influenced by
neutropenia
neutropenia:
(noo-truh-PEE-nee-uh) A condition in which there is a shortage of neutrophils in the bloodstream. Neutrophils are a type of white blood cell. This results in a low white blood cell count.
, therapy and immune reserveTreatment with
azacitidine
azacitidine:
It works by…
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AbstractPurpose
Neutropenic diet
Neutropenic diet:
(noo-truh-PEE-nik) A diet for a patient with very low white blood cell count. A neutropenic diet avoids the use of certain foods that can may contain germs, such as raw meats, aged cheeses, fermented drinks, and unwashed fruits and vegetables.
(ND) has historically been recommended to reduce infectious complications in patients with…
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AbstractPost-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.
(PTCy) has transformed
graft-versus-host…
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Valeria Santini, MD, discusses advances in the treatment of
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…
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AbstractInflammation accelerates evolutionary dynamics of hematopoietic
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…
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Patients with erythropoiesis-stimulating agent (ESA)-refractory lower-risk myelodysplastic syndrome (LR-MDS) continued to benefit from the first-in-class allosteric NEK7/NLRP3 inhibitor ofirnoflast, according to results from a phase 2 study. With its favorable safety profile, ofirnoflast may represent a novel, effective mechanism of action in LR-MDS.NLRP3 inflammasome hyperactivation is a core…
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Join AAMDSIF for a virtual Patient and Family Conference, an educational program designed for patients and caregivers impacted by
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.…
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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…
Conference Event last updated
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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…
Article last updated
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Key PointsStag2 reprograms Gata1 cistrome specificity via chromatin accessibility, a key determinant of transcription factor lineage discrimination.Loss of the Stag2 rheostat causes accessibility-driven Gata1 retargeting, providing a mechanistic framework for dyserythropoiesis in MDS.AbstractThe transcription factor GATA1 has pleiotropic hematopoietic functions,…
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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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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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