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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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UBTF-TD has been reported in a significant percentage of childhood MDS-EB and has been associated with inferior survival compared to that of patients with the wild-type gene. We treated three consecutive pediatric patients affected by UBTF-TD MDS-EB with
venetoclax
venetoclax:
Venetoclax is used to treat chronic lymphocytic leukemia or small lymphocytic leukemia in adults. Venetoclax is…
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Abstract
Pediatric
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 with MDS have low blood cell count for at…
(MDS)…
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Abstract
GATA2 deficiency is an
autosomal dominant
autosomal dominant:
Describes one of several ways that a trait or disorder can be inherited, or passed down through families. "Autosomal" means that the mutated, or abnormal, gene is located on one of the numbered, or non-sex, chromosomes. "Dominant" means that only one copy of the mutated gene is……
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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…
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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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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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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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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
In this explorative study we aimed to identify inflammatory serum proteins measured before allogeneic hematopoietic stem cell transplantation (HSCT) that are associated with acute Graft-versus-Host Disease (aGvHD) and mortality in pediatric HSCT recipients. We measured 28 inflammatory serum proteins in 384 pediatric patients (2010–2022) with malignant (30%) and non-malignant (70%)…
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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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AAMDSIF Supports the Accelerating Kids' Access to Care Act.
On March 26, 2025, AAMDSIF joined with 212 additional patient advocacy and medical center organizations in urging members of the US Congress to pass the Accelerating Kids’ Access to Care Act.
Passage of this bill will permit faster, more thorough care, for children with complex medical conditions.
Please view the PDF below.
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Veronica learned that delaying treatment was not a good idea.
That delay nearly ended her life.
In 2018, Veronica was a busy mom, working full time, leaving the house at 5:30 a.m., returning at 5:30 p.m. She stayed healthy by working and staying active. She is a busy mom of two young children, and all the normal activities of modern life. One day, she thought she caught a bad flu, so…
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Findings refute former consensus that pregnancies post-transplant are nearly impossible, highlight need for increased fertility counseling
(WASHINGTON, July 15, 2024) — Despite treatment-related fertility challenges, female patients can become pregnant and give birth to healthy children after undergoing allogeneic hematopoietic cell transplantation (alloHCT), according to a…
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The conference was held over two days - Friday, July 19 from 2-7 PM ET and Saturday, July 20 from 8AM - 4 PM ET.
Watch these recordings from conference sessions:
High-Risk MDS
A Patient Story
Aplastic Anemia
GVHD - Graft VS Host Disease
Low-Risk MDS
PNH
Nutrition and Bone Marrow Failure
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Abstract
The objective of the study was the analysis of clinical types, outcomes, and risk factors associated with the outcome of adenovirus (ADV) infection, in children and adults after allo-HCT. A total number of 2529 patients (43.9% children; 56.1% adults) transplanted between 2000 and 2022 reported to the EBMT database with diagnosis of ADV infection were analyzed. ADV infection manifested…
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Key Points
The number of autoHCT and alloHCT grew faster in Non-Hispanic African Americans and Hispanics compared to Non-Hispanic Whites.
Survival after autoHCT and alloHCT improved over time for all racial/ ethnic groups, though African Americans have worse outcomes.
There has been an increase in volume as well as improvement in overall survival (OS) after hematopoietic cell transplantation (…
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Dr. Mikkael Sekeres describes why patients should request a second opinion about their
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…
Podcast Episode last updated
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Thank you for attending the 2023 Salt Lake City Patient and Family Conference. We will keep uploading presentations as we receive them, please keep checking back for updates.
Presentation Materials:
Predisposition to Inherited Bone Marrow Failure Syndromes
Aplastic Anemia and Non-Transplant Therapies
MDS and Non-Transplant Therapies
PNH and Non-Transplant Therapies
Ask the…
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Key Points
Structural racism (SR) assessed by census tract variables accounts for nearly all Black-white and Hispanic-white disparity in AML survival.
Structural Racism is a stronger mediator of survival disparities than molecular features, co-morbidities, healthcare access and treatment.
Non-Hispanic Black (NHB) and Hispanic patients with
acute myeloid leukemia
acute myeloid leukemia…
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