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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…
Conference Event last updated
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Abstract
QuANTUM-First was a randomized trial that demonstrated that the addition of quizartinib, a potent and selective FMS-like tyrosine kinase 3 (FLT3) inhibitor, to induction and consolidation
chemotherapy
chemotherapy:
(kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People with high-risk or intermediate-2 risk myelodysplastic syndrome (MDS) may be…
Article last updated
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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)…
Article last updated
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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…
Article last updated
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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)…
Article last updated
.
Abstract
The intensity of the conditioning regimen in hematopoietic stem cell transplantation (HSCT) correlates with the risk of relapse, however its potential benefit may be outweighed by the associated risk of toxicity. The addition of total marrow irradiation (TMI) to myeloablative conditioning provides an opportunity to increase intensity with minimal additional toxicity. In this phase II…
Article last updated
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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…
Article last updated
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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
Conference Event last updated
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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…
Article last updated
.
To the Editor,
We present the first prospective, multicenter, randomized controlled trial (RCT) evaluating Basiliximab prophylaxis for acute
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'…
Article last updated
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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)…
Article last updated
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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…
Article last updated
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The 10th International
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…
Disease Scientific…
Webinar last updated
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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…
Article last updated
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Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative option for
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…
Article last updated
.
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…
Article last updated
.
To the Editor,
We present the first prospective, multicenter, randomized controlled trial (RCT) evaluating Basiliximab prophylaxis for acute
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'…
Article last updated
.
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-…
Article last updated
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Chemotherapy
Chemotherapy:
(kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People with high-risk or intermediate-2 risk myelodysplastic syndrome (MDS) may be given chemotherapy to kill bone marrow cells that have an abnormal size, shape, or look. Chemotherapy hurts healthy cells along with…
-free approaches could offer comparable efficacy with less…
Article last updated
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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…
Article last updated
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