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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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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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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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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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Link above leads to podcast recording.
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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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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…
Article last updated
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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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Guadecitabine (SGI-110), when given as maintenance therapy following allogeneic stem cell transplantation (allo-SCT), showed promising relapse-free survival rates (RFS) with manageable safety in high-risk patients with
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…
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TP53-mutant
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 count for at…
(MDS) and
acute…
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Maintenance treatment with single-agent
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…
at a dose of 32 mg/m2…
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December 5, 2020 - GTB-3550 TriKE was found to safely drive natural killer cell proliferation in patients with high-risk
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…
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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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– With this approval, ULTOMIRIS is the first and only medicine approved in the U.S. to treat children and adolescents with PNH –
– Approval based on interim results from Phase 3 study showing ULTOMIRIS demonstrated complete terminal complement inhibition through 26 weeks –
BOSTON--(BUSINESS WIRE)--Jun. 7, 2021-- Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today…
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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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Study achieved median overall survival of 31.7 months
Updated efficacy data demonstrated an overall response rate of 62%, with 22% of patients achieving a complete response
INQOVI is the only oral hypomethylating agent with equivalent exposure to its intravenous (IV) form
Pleasanton, CA, September 23, 2021. Astex Pharmaceuticals, Inc., a wholly owned subsidiary of Otsuka Pharmaceutical Co. Ltd.,…
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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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