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Ivosidenib
Ivosidenib:
A small molecule inhibitor of isocitrate dehydrogenase-1 (IDH1), which is mutated in several forms of cancer, including acute myeloid leukemia (AML).
, an inhibitor of the mutant isocitrate dehydrogenase 1 (IDH1) enzyme, was well tolerated and associated with deep and durable responses in combination with
azacitidine
azacitidine:
It works by reducing…
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Using advanced RNA sequencing, scientists have identified two unique subtypes of a prominent
mutation
mutation:
Any change or alteration in a gene. A mutation may cause disease or may be a normal variation. Paroxysmal nocturnal hemoglobinuria (PNH) occurs because of a mutation in the PIG-A gene of a single stem cell in the bone marrow.
present in many patients with
Acute Myeloid…
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Key Points
In patients with RR-AML,
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 to treat these conditions.
combination therapy resulted in responses in 31% of patients and a median OS of 6.1 months.
NPM1 mutations predicted higher…
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The novel agentSY-1425 combined with
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…
appears to be active in…
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Meningioma-1 (MN1) is a protein whose overexpression has been linked to
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…
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If you’re receiving treatment for
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…
(AML…
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Combination gemtuzumab ozogamicin (GO) and cytarabine is no more effective than standard
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…
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Link above leads to podcast recording.
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Eunice S. Wang, MD, of Roswell Park Comprehensive Cancer Center, discusses phase III results showing that gilteritinib and
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…
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Key Points
Donor-derived T cells with native specificity for multiple myeloid leukemia antigens can be expanded ex vivo.
Infusion of mLSTs after HCT is well tolerated and produces antileukemia effects.
Abstract
Relapse after allogeneic hematopoietic stem cell transplantation (HCT) is the leading cause of death in patients with
acute myeloid leukemia
acute myeloid leukemia:
(uh-KYOOT…
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A drug that targets a common
mutation
mutation:
Any change or alteration in a gene. A mutation may cause disease or may be a normal variation. Paroxysmal nocturnal hemoglobinuria (PNH) occurs because of a mutation in the PIG-A gene of a single stem cell in the bone marrow.
in
myelodysplastic syndromes
myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A group of…
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Results from the phase III PEGASUS trial suggest that the targeted C3 inhibitor
pegcetacoplan
pegcetacoplan:
EMPAVELI® is the first PNH treatment that binds to complement protein C3. It was approved by the Food and Drug Administration in May 2021 for treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH). EMPAVELI is given skin (subcutaneously) by using the Empaveli…
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Published: Dec 15, 2021
STOCKHOLM, Dec. 15, 2021 /PRNewswire/ --
The first targeted C3 therapy approved in the EU
Approval based on results from head-to-head PEGASUS phase 3 study where Aspaveli demonstrated superiority to
eculizumab
eculizumab:
Eculizumab (Soliris ®) is given as an IV into a vein at the doctor’s office or at a special center. The procedure…
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Key Points
Bone marrow
Bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
transplantation with posttransplantation CY in severe
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…
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Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science accessible and empower rare disease community members with scientific knowledge. Dr. Brown has over a decade of experience in neurodevelopmental research and is currently the Scientific Director for TESS…
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Abstract
Diagnosis of myelodysplastic syndrome (MDS) mainly relies on a manual assessment of the peripheral blood and
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
cell
morphology
morphology:
The study of the structure and form of an organism or one of its parts.
. The WHO guidelines suggest a visual screening…
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Beginning September 2012, I just wasn’t feeling quite right. I was increasingly fatigued and there were some perplexing bleeding incidents, as well. After many months I experienced partial vision – just little areas of vision that were blank. Internal bleeding in the eyes was causing this, and my eye doctor knew it was serious and even thought it could be leukemia.
Blood tests were done, and…
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September 16 through December 20 will stand out in Ashley’s mind.
Forever.
These are the days she spent in the hospital with her feisty three-year-old, Alayna, for conditioning and
bone marrow transplant
bone marrow transplant:
A bone marrow transplant (BMT) is also called a stem cell transplant (SCT) or hematopoietic stem cell transplant (HSCT).The procedure replaces unhealthy blood-…
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Each year, approximately 1 in every 1 million infants is diagnosed with pediatric
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.…
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Eltrombopag
Eltrombopag:
What are the possible side effects of eltrombopag (Promacta)?
Get emergency medical help if you have any of these signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.
Stop using eltrombopag and call your doctor at once if you have:
…
, an FDA-approved non-peptidyl thrombopoietin receptor agonist is…
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