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Abstract
Purpose of Review
In inherited
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…
syndromes…
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Key Points
Timely manufacture of VCAR33, a donor-derived CD33-directed CAR T, was feasible in patients with relapsed/refractory AML after alloHCT.
VCAR33 had an acceptable safety profile at doses up to 3 × 106 cells/kg and demonstrated antileukemic activity with a response rate of 20%.
Abstract
VCAR33, a donor-derived CD33-directed chimeric antigen receptor T-cell (CAR T) product, was developed…
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Abstract
Acute leukemia of ambiguous lineage (ALAL) is a rare, high-risk form of acute leukemia. It is characterized by the inability to assign a single lineage of differentiation to the leukemia and can manifest with more than one lineage-defining marker, called mixed phenotype acute leukemia (MPAL), or the complete absence of such markers, defined as acute undifferentiated leukemia (AUL).…
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Correspondence
The Myelodysplastic/Myeloproliferative neoplasm (MDS/MPN) overlap syndromes are a heterogeneous group of rare hematologic malignancies characterized by features of both MDS such as morphologic dysplasia and cytopenias and MPNs including elevated blood counts and often splenomegaly [1]. These syndromes frequently harbor recurrent mutations, underscoring their interrelated biological…
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Key Points:
Many patients and caregivers associate early palliative care (EPC) with hospice or death, a misperception that may persist even after gaining a better understanding of the role of EPC and may prevent them from experiencing the benefits of EPC.
Cultural or religious beliefs about illness and death may further influence views of EPC, and clinicians must engage in culturally sensitive…
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Abstract
Chimeric antigen receptor (CAR)-T cell therapy has transformed the treatment of hematologic malignancies, yet its application 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…
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Abstract
Background
Clonal
hematopoiesis
hematopoiesis:
(hi-mat-uh-poy-EE-suss) The process of making blood cells in the bone marrow.
(CH) is a feature of 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 platelets. The term…
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Key Takeaways
Approval targets older/unfit newly diagnosed AML, introducing an all-oral HMA/venetoclax backbone that may reduce infusion burden and expand outpatient feasibility.
ASCERTAIN‑V part B delivered DEC‑C days 1–5 q28d with
venetoclax
venetoclax:
Venetoclax is used to treat chronic lymphocytic leukemia or small lymphocytic leukemia in adults. Venetoclax is used alone or in…
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Abstract
Allogeneic hematopoietic cell transplantation (ASCT) is the only curative option for 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 marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means abnormal…
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Key Points
Frontline
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:
…
plus IST is cost-effective compared with IST…
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Abstract
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…
resistance remains a critical challenge in the treatment…
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ABSTRACT
Allogeneic hematopoietic cell transplantation (allo-HCT) is an effective treatment for patients with high-risk hematologic malignancies. Over the last decade, gut microbiota composition during allo-HCT has been associated with patients' outcomes. Treatment-related factors, in particular the use of broad-spectrum
antibiotics
antibiotics:
The most common white blood cells are…
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On May 13, 2026, the U.S. Food and Drug Administration (FDA) approved an all-oral regimen of
decitabine
decitabine:
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.…
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Key Points
For patients with AML treated with LIT, ELN 2024 and Beat AML 2024 best identify a favorable group.
Allogeneic stem cell transplant
Allogeneic stem cell transplant:
A procedure where bone marrow stem cells are taken from a genetically matched donor (a brother, sister, or unrelated donor) and given to the patient through an intravenous (IV) line. In time, donated stem cells…
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In this issue of Blood, Muffly et al1 demonstrate in a phase 1 trial the feasibility and safety of CD117-targeted, precision preconditioning for allogeneic hematopoietic cell transplantation (allo-HCT) in
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…
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Session Recordings Available:
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Recorded Sessions:
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Here are links to the conference sessions from the 2025 Kansas City Patient and Family Conference:
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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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