News and Treatment Updates
Here's where you'll find a regularly updated, broad range of articles written by the AAMDSIF team, allied health organizations and news organizations. By staying well-informed, patients and families are practicing a form of self-support that will help them be more effective self-advocates when engaging with health care providers.
Aplastic Anemia and MDS International Foundation Announces 2021 Research Grant Recipients
Originally Published: 02/11/2022
Article Source: Press Release
FOR IMMEDIATE RELEASE
Aplastic Anemia and MDS International Foundation Announces 2021 Research Grant Recipients
(BETHESDA, MD) February 11, 2022. The Aplastic Anemia and MDS International Foundation announces the two recipients of the 2021 Research Awards. For over 30 years, AAMDSIF has provided financial support for research that leads to new insights into the causes of bone marrow failure diseases and to the development of new therapeutic approaches. Since 1989 we have awarded over $5 million in funding 98 grantees.
“Supporting early career investigators is an investment in the future,”...
Research Identifies How Gene Mechanisms Influence Pediatric MDS
Originally Published: 01/31/2022
Article Source: External Web Content
Each year, approximately 1 in every 1 million infants is diagnosed with pediatric myelodysplastic syndromes (MDS), a group of rare disorders characterized by the abnormal or deficient formation of blood cells in the bone marrow. However, researchers wanted to understand the underlying mechanisms behind this condition. In the past, both SAMD9 and SAMD9L mutations have been linked to pediatric MDS; in fact, approximately 8% of pediatric patients with MDS have one of these mutations. According to the University of Texas Health Science Center at San Antonio, a research team recently learned more...
Eltrombopag inhibits TET dioxygenase to contribute to hematopoietic stem cell expansion in aplastic anemia
Originally Published: 01/27/2022
Article Source: External Web Content
Eltrombopag, an FDA-approved non-peptidyl thrombopoietin receptor agonist is clinically used for the treatment of aplastic anemia, a disease characterized by hematopoietic stem cell failure and pancytopenia, to improve platelet counts and stem cell function. Eltrombopag treatment results in durable tri-lineage hematopoietic expansion in patients. Some of the eltrombopag hematopoietic activity has been attributed to its off-target effects including its iron chelation properties. However, eltrombopag mechanism of action is still poorly understood with respect to its full spectrum of clinical...
Structural Racism is a Mediator of Disparities in Acute Myeloid Leukemia Outcomes
Originally Published: 01/21/2022
Article Source: External Web Content
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 (AML) have higher mortality rates than non-Hispanic white (NHW) patients despite more favorable genetics and younger age. A discrete survival analysis was performed on 822 adult AML patients from six urban cancer centers and revealed...
Machine learning assisted real-time deformability cytometry of CD34+ cells allows to identify patients with myelodysplastic syndromes
Originally Published: 01/18/2022
Article Source: External Web Content
Abstract
Diagnosis of myelodysplastic syndrome (MDS) mainly relies on a manual assessment of the peripheral blood and bone marrow cell morphology. The WHO guidelines suggest a visual screening of 200 to 500 cells which inevitably turns the assessor blind to rare cell populations and leads to low reproducibility. Moreover, the human eye is not suited to detect shifts of cellular properties of entire populations. Hence, quantitative image analysis could improve the accuracy and reproducibility of MDS diagnosis. We used real-time deformability cytometry (RT-DC) to measure bone marrow biopsy...
Science Simplified: What is a Natural History Study?
Originally Published: 01/17/2022
Article Source: External Web Content
Want to learn about scientific topics without needing a PhD? Check out the Science Simplified blog from TESS Research Foundation! Dr. Tanya Brown, PhD, works with researchers to make science accessible and empower rare disease community members with scientific knowledge. Dr. Brown has over a decade of experience in neurodevelopmental research and is currently the Scientific Director for TESS Research Foundation. Please reach out to her at tanya@tessfoundation.org if you have questions or comments.
Thank you to Tanya Brown, PhD, Research Program Manager, for writing this article about natural...
Eunice S. Wang, MD, on FLT3-Mutated AML: Gilteritinib and Azacitidine for Intensive Induction Chemotherapy–Ineligible Patients
Originally Published: 01/03/2022
Article Source: External Web Content
Eunice S. Wang, MD, of Roswell Park Comprehensive Cancer Center, discusses phase III results showing that gilteritinib and azacitidine led to significantly higher composite complete response rates in patients with newly diagnosed FLT3-mutant acute myeloid leukemia who are ineligible for intensive induction chemotherapy. Overall survival was similar to that of azacitidine alone (Abstract 700).
Leslie S. Kean, MD, PhD, on Bone Marrow Transplantation: Using Abatacept to Prevent Graft-vs-Host Disease
Originally Published: 01/03/2022
Article Source: External Web Content
Leslie S. Kean, MD, PhD, of Dana-Farber/Boston Children's Cancer and Blood Disorders Center, discusses findings from her analysis of the International Blood and Marrow Transplant Research Database, which led to the recent FDA approval of abatacept for the prevention of acute graft-vs-host disease (GVHD) in adult and pediatric patients. The data suggest improved overall survival with the immunosuppressant abatacept in combination with a calcineurin inhibitor and methotrexate following 7/8 HLA–matched unrelated allogeneic hematopoietic stem cell transplantation (Abstract 3912).
Coronavirus (COVID-19) Update: FDA Authorizes First Oral Antiviral for Treatment of COVID-19
Originally Published: 12/22/2021
Article Source: External Web Content
Today, the U.S. Food and Drug Administration issued an emergency use authorization (EUA) for Pfizer’s Paxlovid (nirmatrelvir tablets and ritonavir tablets, co-packaged for oral use) for the treatment of mild-to-moderate coronavirus disease (COVID-19) in adults and pediatric patients (12 years of age and older weighing at least 40 kilograms or about 88 pounds) with positive results of direct SARS-CoV-2 testing, and who are at high risk for progression to severe COVID-19, including hospitalization or death. Paxlovid is available by prescription only and should be initiated as soon as possible...
MDS Update for Q4 2021 - online version
Originally Published: 12/22/2021
Article Source: MDS Update
Please click the link above to find the online version of the MDS Update for Q4 2021.