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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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Key PointsZiftomenib shows pronounced preclinical activity in the menin-KMT2A–dependent leukemia models, including patient-derived xenografts.Ziftomenib demonstrates potent activity against a subset of the drug-induced MEN1 mutations found in patients with
acute myeloid leukemia
acute myeloid leukemia:
(uh-KYOOT my-uh-LOYD loo-KEE-mee-uh) A cancer of the blood cells. It…
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AbstractMyeloproliferative neoplasms (MPNs) were previously thought to result solely from somatic mutations; however, familial clustering and germline genetic testing have revealed hereditary MPNs. These hereditary MPNs are one type of hereditary hematopoietic malignancy (HHM) syndrome, or hereditary blood cancer, and generally display an autosomal-dominant inheritance pattern…
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AbstractPurposeThe Children's Oncology Group phase III
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…
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AbstractPOEMS syndrome is a rare multisystem
plasma
plasma:
The fluid part of the blood. Plasma is mostly made of water with chemicals in it. These chemicals include proteins, hormones, minerals, and vitamins.
cell disorder for which evidence guiding management is largely derived from single-center retrospective series. We conducted a retrospective multicenter study…
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Background: Philadelphia-negative myeloproliferative neoplasms (MPNs), including essential thrombocythaemia (ET), polycythaemia vera (PV), and myelofibrosis (MF), usually affect older adults, but a subset occurs in adolescents and young adults (AYA). Regional age-stratified data remain limited.
Methods: We retrospectively studied Philadelphia-negative MPN patients managed at the National Center…
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Highlights -Considerable heterogeneity exists in germline testing implementation across hematopoietic stem cell transplantation centers in the United States.-Lack of standardized protocols affects the timing of testing and inconsistent sample collection workflows.-Barriers to consistent care include insurance challenges, logistical constraints, and…
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Key PointsPRCA shows marked diagnostic heterogeneity, where molecular data can help identify challenging secondary forms, such as MDS-associated cases.Early initiation of CyA improves outcomes; mTORi are valuable second-line treatment. Infection related mortality remains non negligible.Abstract
Pure red cell aplasia (PRCA)
Pure red cell aplasia (PRCA):
A…
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CONFERENCE AGENDA - November 7, 20268:00 - 2:45 (Central Time Zone)
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…
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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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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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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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Abstract
The diagnosis and treatment of hematologic malignancies has undergone significant advancements over the past few decades, resulting in enhanced outcomes. For hematologic malignancies diagnosed during pregnancy, this poses new questions. As possibilities continue to expand and the rising global incidence results in an increasing number of diagnoses within the pregnant population, there is…
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