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Amer Zeidan, MBBS, MHS, is professor of internal medicine at Yale School of Medicine in New Haven, Connecticut. He is also the chief of the Division of Hematologic Malignancies at Yale Cancer Center, New Haven.
Dr. Zeidan spoke with Blood Cancers Today about the phase 1/2 BEXMAB study, which was presented at the 2025 American Society of Clinical Oncology (ASCO) Annual Meeting, recently held in…
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VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is a newly identified monogenic disorder with symptoms including recurrent fever, skin involvement, pulmonary infiltrates, systemic vasculitis, and chondritis.1 Notably, 25-50% of patients also have myelodysplastic syndrome (MDS). VEXAS syndrome often requires high-dose steroids and is resistant to standard…
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Shane Pope talks about living with PNH, or Paroxysmal Nocturnal Hemglobinuria, in this recorded conversation with Leigh Clark. Listen or read the transcript below.
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Shaquilla Gordon explains her journey with the
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……
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Dr. Carlos DeCastro reviews how PiaSky can be used to treat patients with PNH, or
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…
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Donna Gilbride explains her journey through diagnosis and treatment with 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 development.…
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Dr. Satheesh Chonat explains how pediatric patients with PNH, or
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…
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Pediatric nurse to medical student to patient? This was Claire's life. Listen to her story here.
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Thanks to the efforts of our AAMDSIF
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…
community and…
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Key Points
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:
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combined with IST demonstrated a trend towards a favorable…
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Key Points
Treg-engineered donor graft prevents acute and chronic GVHD.
Patients treated with Treg-engineered graft who develop GVHD respond to primary corticosteroid therapy at a high rate.
Abstract
Allogeneic hematopoietic cell transplantation is a curative therapy limited by
graft-versus-host disease (GVHD)
graft-versus-host disease (GVHD):
Also called GVHD, it is a common…
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After a long battle with illness and seeking many specialists, I emphasize the need to self-advocate for ongoing care following my cancer diagnosis.
Almost every cancer survivor will agree that we end up going to too many doctors. A friend of mine with both cancer and diabetes has added up that he has 14 of them! I wrote an article for CURE titled “Doctors and More Doctors – A Way of Life for…
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Key Points
Flow cytometry
Flow cytometry:
(sy-TOM-uh-tree) A laboratory test that gives information about cells, such as size, shape, and percentage of live cells. Flow cytometry is the test doctors use to see if there are any proteins missing from the surface of blood cells. It is the standard test for confirming a diagnosis of paroxysmal…
enhances morphological analysis to detect AML…
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Abstract
The role of ABO blood group system mismatch on allogeneic hematopoietic cell transplantation (allo-HCT) outcomes is controversial since current publications of large datasets are lacking. We retrospectively analyzed 30,487 patients transplanted between 2010 and 2021 using the EBMT registry to assess ABO incompatibility’s effect on non-relapse mortality (NRM), overall survival (OS),…
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The
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…
and MDS International Foundation joined 156 other…
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Join us in Verona Park, NJ on Sunday, September 28, 2025 for a day of remembrance and awareness in celebration of the 19th Annual Walk for PNH.
The Walk for PNH is the nation's largest event to raise awareness and funds for PNH education and research. This inspiring event calls on participants of all ages and abilities to join in the fight against PNH.
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Abstract
Hypomethylating agent (HMA) plus
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.
(VEN) regimens are standard of care in patients with
acute myeloid leukemia
acute myeloid leukemia:
(uh-KYOOT my-uh-…
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Key Points
SRSF2 mutations blunt responsiveness of MDS cells to IFN stimulation and promote their clonal fitness by downregulating STAT1 expression
Proteasome inhibition restores STAT1 protein level and sensitivity of SRSF2-mutant MDS cells to IFN, suggesting a new therapeutic strategy
Myelodysplastic syndromes
Myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A group…
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Abstract
Dissecting the preneoplastic disease states’ biological mechanisms that precede tumorigenesis can lead to interventions that can slow down disease progression and/or mitigate disease-related comorbidities.
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…
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AAMDSIF Joins Coalition Urging the Administration to Preserve our Nation’s Newborn Screening Program
A coalition of 272 organizations signed a public letter to the Secretary, Department of Health and Human Services, addressing the decision to eliminate the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC). While each state may add on additional screenings, this Committee ensured standardized measurements and procedures, to endure early detection of newborn…
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