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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…
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…
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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
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
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…
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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
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…
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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)…
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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…
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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
.
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-…
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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…
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Summary
Many veterans and doctors have long questioned whether Agent Orange (AO) exposure during the Vietnam War contributed to cases of myelodysplastic syndrome (MDS), a type of blood cancer.
The contaminated chemical is linked to several cancers, but a link to MDS was unclear. This created real barriers for exposed veterans with MDS who are seeking care and disability benefits.
Now, a new…
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Abstract
Somatic mutations in hematopoietic stem/progenitor cells (HSPCs) can lead to clonal
hematopoiesis
hematopoiesis:
(hi-mat-uh-poy-EE-suss) The process of making blood cells in the bone marrow.
of indeterminate potential (CHIP) and progression to
myelodysplastic syndromes
myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the…
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Key Points
Ivosidenib
Ivosidenib:
A small molecule inhibitor of isocitrate dehydrogenase-1 (IDH1), which is mutated in several forms of cancer, including acute myeloid leukemia (AML).
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venetoclax
venetoclax:
Venetoclax is used to treat chronic lymphocytic leukemia or small lymphocytic leukemia in adults. Venetoclax is used alone or in combination with other cancer medicines…
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Recordings:
Aplastic Anemia
Low Risk MDS
High Risk MDS to AML
Transplant and GVHD
MDS/MPN Overlap
A Patient Story: Shauna McMillian
Managing PNH Complications
Conference Event last updated
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Session Recordings Available:
MDS to MPN Overlap from the 2025 Boston Patient and Family Conference
High Risk MDS from the 2025 Boston Patient and Family Conference
Low Risk MDS from the 2025 Boston Patient and Family Conference
What is Aplastic Anemia? From the 2025 Boston Patient and Family Conference
What is PNH? Webinar recorded at the 2025 Boston Patient and Family Conference…
Conference Event last updated
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Here are links to the conference sessions from the 2025 Kansas City Patient and Family Conference:
MDS/MPN Overlap
PNH and Current Therapies
Living Well with Bone Marrow Failure
Judy's Story of PNH
Transplant and GVHD
Aplastic Anemia
PNH Complications
High Risk MDS
Conference Event last updated
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