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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:
Conference Event last updated
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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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DEADLINE MAY 8Senators Roger Wicker (R-MS) and Dick Durbin (D-IL) are circulating a bipartisan letter to the Senate in support of increased funding for the Department of Defense’s medical research accounts, including CDMRP and BMFRP.You can help by asking Your Senators to Sign the LetterSenators Roger Wicker (R-MS) and Dick Durbin (D-IL) are circulating a…
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Listen to Barry describe the diagnosis, watch-and-wait, treatment, and survivorship for MDS, or
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…
Podcast Episode last updated
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Learn from Dr. Richard Childs, RADM, as he explains the FDA approval of Omnisirge for treating patients with 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 aplastic is a Greek word meaning not to form. Anemia is a…
Podcast Episode last updated
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Background:
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…
is a rare, life-threatening condition marked…
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Background
Paroxysmal nocturnal hemoglobinuria
Paroxysmal nocturnal hemoglobinuria:
(par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria means hemoglobin in the urine. Hemoglobin is the red part of red blood cells. A…
(PNH…
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In this issue of Blood, Mushtaq et al1 report on a phase 1/2 trial (NCT05984199, known as Vor Biopharma trial VBP301) utilizing donor-derived anti-CD33 chimeric antigen receptor–modified (CAR) T cells (VCAR33) in patients with relapsed
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…
Article last updated
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Abstract
Selection of a hematopoietic progenitor cell donor for allogeneic hematopoietic cell transplantation (HCT) is essential for treatment planning; however, the parameters that define an “optimal” donor in the modern era are not well defined. Historically, donor-recipient
HLA
HLA:
See human leukocyte antigen.
mismatching correlated strongly with risk for
graft-versus-host…
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Abstract
Allogeneic stem cell transplantation (SCT) is a curative treatment in
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…
Article last updated
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ABSTRACT
Regimens combining calcineurin inhibitors with methotrexate or mycophenolate have been the backbone for
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's body as foreign and mount…
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