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Abstract
Improved ex vivo expansion of human haematopoietic
stem cells
stem cells:
Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body repair and maintain the organ or tissue in which they are found. Blood-forming (hemapoietic) stem…
(HSCs)…
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Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is a curative option for
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…
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NIH
clinical trial
clinical trial:
A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a…
shows promise for people with treatment-limited, life-…
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Jen had a great life. She was married with two children, and she’s just transitioned from teaching to school counseling. At 41, life was busy yet wonderful.Her symptoms were unusual, but not alarming. The leg pain was new, as was the bruising. Then fatigue set in, far beyond usual tiredness from her schedule. A visit to the doctor did not offer a definitive diagnosis. They suggested it was…
Patient Chronicle last updated
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Recordings:
Aplastic Anemia
Low Risk MDS
High Risk MDS to AML
Transplant and GVHD
MDS/MPN Overlap
A Patient Story: Shauna McMillian
Managing PNH Complications
Conference Event last updated
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Session Recordings Available:
MDS to MPN Overlap from the 2025 Boston Patient and Family Conference
High Risk MDS from the 2025 Boston Patient and Family Conference
Low Risk MDS from the 2025 Boston Patient and Family Conference
What is Aplastic Anemia? From the 2025 Boston Patient and Family Conference
What is PNH? Webinar recorded at the 2025 Boston Patient and Family Conference…
Conference Event last updated
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Here are links to the conference sessions from the 2025 Kansas City Patient and Family Conference:
MDS/MPN Overlap
PNH and Current Therapies
Living Well with Bone Marrow Failure
Judy's Story of PNH
Transplant and GVHD
Aplastic Anemia
PNH Complications
High Risk MDS
Conference Event last updated
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Meet Evan. In a recent conversation with 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…
Article last updated
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Abstract
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…
(AA) transformation into
myelodysplastic…
Article last updated
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Evan biked daily. He had no symptoms. The diagnosis was fast, but surprising. Evan shares his remarkable story in this episode.
Podcast Episode last updated
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Recorded Sessions:
Nutrition for Aplastic Anemia, MDS, or PNH
Managing PNH Treatments
Advances in Aplastic Anemia
What is PNH?
Transplant and GVHD: Aplastic Anemia and PNH
Inherited Bone Marrow Failure (focus on pediatrics)
Expert Panel: Pediatric Bone Marrow Failure
Life After Diagnosis - Pediatrics
Pediatric MDS
Transitioning to Adult Care
Pediatric PNH
Pediatric Aplastic…
Conference Event last updated
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Data from APPULSE-PNH may support oral
iptacopan
iptacopan:
FABHALTA, a complement factor B inhibitor, is the first oral medication approved to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). It was approved by the U.S. Food and Drug Administration in December 2023. Fabhalta is taken twice a day in a capsule form.
What is FABHALTA?
…
as a potentially practice-changing…
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ABSTRACT
Background
Elevated blood counts in polycythemia vera (PV) are associated with increased thrombotic risk, which contributes to morbidity and mortality.
Research design and methods
This retrospective study describes treatment patterns and blood count control in patients with PV managed at community oncology practices (January 2014–February 2023; Integra Precision Q database).
Results
Of…
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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.
Podcast Episode last updated
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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……
Podcast Episode last updated
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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…
Podcast Episode last updated
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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…
Podcast Episode last updated
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Pediatric nurse to medical student to patient? This was Claire's life. Listen to her story here.
Podcast Episode last updated
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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:
…
combined with IST demonstrated a trend towards a favorable…
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