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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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Saturday, October 24, 2026Conference Co-ChairsUma Borate, MBBSAlice Mims, MDAplastic Anemia/PNH Agenda 7:30 - 8:15 Breakfast and Registration8:15 - 8:45 Welcome 8:45 - 9:15
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…
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Weill Cornell Medicine investigators have developed a new AI-based method to assess patients with a form of blood cancer called myelodysplastic neoplasms. The method, published June 30 in Leukemia, compares the physical locations and morphometric (size and shape) characteristics of every hematopoietic cell in the patient’s
bone marrow
bone marrow:
The soft, spongy tissue inside most…
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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…
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CONFERENCE LOCATION:Heldrich Hotel 10 Livingston AvenueNew Brunswick, NJ 08901Parking: Complimentary hotel valet parking CONFERENCE AGENDA
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…
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HighlightsIn lower-risk MDS subgroup, trisomy 8 and 9 were more frequent in MDS-AID.MDS-AID had fewer tier 1 or 2 mutations than MDS-control or MDS-ALE.ASXL1 mutations were enriched in MDS-ALE within lower-risk MDS.No significant differences in OS or PFS versus MDS-control.IPSS-M was the only variable independently associated with outcomes. AbstractBackgroundAutoimmune features are…
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The timing of TP53
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.
acquisition carries important prognostic implications for patients with myeloproliferative neoplasms (MPNs) and…
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Abstract
Expression levels of
HLA
HLA:
See human leukocyte antigen.
are associated with susceptibility to various diseases and outcomes after allogeneic hematopoietic cell transplantation (HCT). However, there has been no comprehensive analysis of the effect of the expression levels of the HLA-A, -B, -C, and -DRB1 alleles in unrelated HCT (UR-HCT). Using the capture RNA-sequencing…
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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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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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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…
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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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