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DEADLINE MAY 8Senators Roger Wicker (R-MS) and Dick Durbin (D-IL) are circulating a bipartisan letter to the Senate in support of increased funding for the Department of Defense’s medical research accounts, including CDMRP and BMFRP.You can help by asking Your Senators to Sign the LetterSenators Roger Wicker (R-MS) and Dick Durbin (D-IL) are circulating a…
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Listen to Barry describe the diagnosis, watch-and-wait, treatment, and survivorship for MDS, or
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…
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Background
Paroxysmal nocturnal hemoglobinuria
Paroxysmal nocturnal hemoglobinuria:
(par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria means hemoglobin in the urine. Hemoglobin is the red part of red blood cells. A…
(PNH…
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Key PointsA structured, home-based exercise intervention in MPN was feasible, well accepted, and showed initial benefits on disease-related markers.Physical activity is a potentially safe and accessible adjunct to standard treatments in this population, warranting further study.Abstract Myeloproliferative neoplasms (MPN) are associated with a high symptom burden and impaired quality of life…
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Patients who receive hematopoietic stem cell transplants are at risk of developing
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…
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ABSTRACT
The prognostic value of NPM1-mut Measurable Residual Disease (MRD) has been increasingly recognized. However, real-world data validating the prognostic impact of NPM1-mut MRD remains limited. The aim of this study was to assess the prognostic value of NPM1-mut MRD and to explore optimal MRD thresholds for relapse prediction. One hundred forty-one patients with newly diagnosed NPM1-mut…
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Key Points
aGVHD causes loss of gastric
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…
, G-cells, and PCs, leading…
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Abstract
Myelodysplastic neoplasms, also known as
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…
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Luspatercept generated favorable efficacy and safety outcomes in the treatment of
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 patients with transfusion-dependent, lower-risk MDS.
Data from a systematic review and meta-analysis of 20…
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Measurable residual disease (MRD) monitoring has become a critical component in the management of
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…
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Therapy-related myeloid neoplasm (tMN) is a fatal consequence of exposure to cytotoxic therapy administered in the treatment of cancer. Individuals with pre-existing TP53 clonal
hematopoiesis
hematopoiesis:
(hi-mat-uh-poy-EE-suss) The process of making blood cells in the bone marrow.
(CH) are at high risk of tMN, with avoidance of therapy being the only strategy to reduce tMN risk.…
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Key Points
MRD-guided
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…
therapy can effectively prevent or delay…
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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…
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paroxysmal nocturnal hemoglobinuria…
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Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) remains the only potential curative therapy for myelodysplastic syndrome (MDS), recommended in higher risk disease according to the
International Prognostic Scoring System
International Prognostic Scoring System:
A system that turns patient data into a score. The score tells how quickly a myelodysplastic syndrome (MDS)…
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Abstract
Health-related quality of life (HRQoL) of patients with myeloproliferative neoplasms (MPNs) may be impaired across several domains. In this multicenter observational study, we evaluated HRQoL and symptoms in a cohort of MPN patients with validated measures, including the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC QLQ-C30), the…
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ABSTRACT
The COMMANDS trial established luspatercept as a first-line treatment 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 lower-risk (LR)
myelodysplastic syndromes
myelodysplastic syndromes:
(my…
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2024 Patient and Family Conference in Los AngelesSession Recordings:Transplant for Aplastic Anemia and MDSEmerging Therapies in High-Risk MDSCardiovascular Concerns after HCTSupportive Care ManagementAdvances in Treating Lower-Risk MDSPalliative CareLiving with Chronic GVHDA Patient StoryInherited Bone Marrow Failure SyndromesAdvances in Treating Aplastic Anemia
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Rusfertide sustained
hematocrit
hematocrit:
(hi-MA-tuh-crit) A blood test that measures the percentage of the blood made up of red blood cells. This measurement depends on the number of red blood cells and their size. Hematocrit is part of a complete blood count. Also called HCT, packed cell volume, PCV.
control and sharply reduced phlebotomy need through week 52 in PV, showing durable…
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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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