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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.

Bone Marrow Transplant Recipients During Childhood at Increased Risk for Severe Chronic Health Conditions

Originally Published: 12/05/2020
Article Source: External Web Content
Patients who underwent allogeneic bone marrow transplantation (BMT) as children were at increased risk for severe or life-threatening chronic health conditions. These findings were presented during the American Society of Hematology (ASH) 62nd Annual Meeting and Exposition. For this study, 848 patients who survived at least 2 years after a BMT during early adulthood or adolescence (aged 22 years or younger) and their siblings or parents (n=515) were recruited. Participants were assessed by questionnaire for sociodemographic characteristics and health status. At the time of study, 563 of the...

GTB-3550 TriKE Stimulates Natural Killer Cell Function in MDS and AML

Originally Published: 12/05/2020
Article Source: External Web Content
December 5, 2020 - GTB-3550 TriKE was found to safely drive natural killer cell proliferation in patients with high-risk myelodysplastic syndromes and acute myeloid leukemia. GTB-3550 TriKE was found to safely drive natural killer cell proliferation in patients with high-risk myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML), according to data from initial dose cohorts of a phase 1/2 trial (NCT03214666) presented during the 2020 ASH Annual Meeting & Exposition. Results showed that the GTB-3550 TriKE, when administered at a daily dose of 5 mcg/kg in a patient with therapy-...

Survival following relapse after allogeneic hematopoietic cell transplantation for acute leukemia and myelodysplastic syndromes in the contemporary era

Originally Published: 12/05/2020
Article Source: External Web Content
Abstract Objective/Background Relapse is the most common cause of treatment failure after allogeneic hematopoietic cell transplantation (alloHCT). No standard of care exists, and a wide range of treatments are used for post-alloHCT relapse. In the recent era, several novel therapies including targeted agents are available for acute lymphocytic leukemia (ALL), acute myeloid leukemia (AML), and myelodysplastic syndrome (MDS). Methods We reviewed outcomes after alloHCT relapse, with or without use of these newer agents for ALL, AML, and MDS. In total, 115 adults with relapsed or refractory ALL...

Targeted therapy venetoclax improving acute myeloid leukemia treatment options

Originally Published: 12/05/2020
Article Source: External Web Content
Targeted therapies are designed to work against specific vulnerabilities in a cancer cell. The targeted therapy drug venetoclax is designed to block the function of the B-cell lymphoma-2 (Bcl-2) protein, which normally works to prevent cell death. Venetoclax was first approved in 2016 for treating certain patients with chronic lymphocytic leukemia, which has high levels of Bcl-2. More recently, venetoclax has begun to show promise for treating acute myeloid leukemia (AML), which also has elevated Bcl-2. The results of several promising clinical trials were presented this week at the virtual...

The Aplastic Anemia and MDS International Foundation (AAMDSIF) and Acceleron Pharma Inc. Announce Partnership Exploring the Mental Health Implications of Myelodysplastic Syndromes (MDS) Among Adult MDS Patients and Their Caregivers in the U.S.

Originally Published: 11/25/2020
Article Source: Press Release
FOR IMMEDIATE RELEASE November 25, 2020 The Aplastic Anemia and MDS International Foundation (AAMDSIF) and Acceleron Pharma Inc. Announce Partnership Exploring the Mental Health Implications of Myelodysplastic Syndromes (MDS) Among Adult MDS Patients and Their Caregivers in the U.S. (Bethesda, MD, November 25, 2020) The Aplastic Anemia and MDS International Foundation (AAMDSIF) and Acceleron Pharma Inc. are partnering in an effort to better understand the mental health implications of myelodysplastic syndromes (MDS) among adult MDS patients and their caregivers in the U.S. An online survey...

APLASTIC ANEMIA and MDS INTERNATIONAL FOUNDATION ANNOUNCES NEW EXECUTIVE DIRECTOR - Nonprofit Executive Janice Frey-Angel Assumes Role December 1, 2020

Originally Published: 11/24/2020
Article Source: Press Release
FOR IMMEDIATE RELEASE November 24, 2020 APLASTIC ANEMIA and MDS INTERNATIONAL FOUNDATION ANNOUNCES NEW EXECUTIVE DIRECTOR Nonprofit Executive Janice Frey-Angel Assumes Role December 1, 2020 November 24, 2020; BETHESDA, MARYLAND –  The Aplastic Anemia and MDS International Foundation (AAMDSIF), the world's leading nonprofit health organization dedicated to supporting patients and families living with bone marrow failure diseases, announced today that Janice Frey-Angel has been named the Foundation's new Chief Executive Officer and Executive Director. Ms. Frey-Angel succeeds Neil Horikoshi,...

NCCN Grants Category 2A Recommendation to Decitabine/Cedazuridine Combo for MDS

Originally Published: 11/23/2020
Article Source: External Web Content
The National Comprehensive Cancer Network has updated its Clinical Practice Guidelines in Oncology for Myelodysplastic Syndromes (MDS) to include the combination of decitabine and cedazuridine (Inqovi) as a category 2a recommendation for the treatment of adult patients with intermediate- and high-risk MDS.1 Oral decitabine and cedazuridine, given at 35-mg and 100-mg tablets, can be considered a substitution for intravenous decitabine in the treatment of adult patients with MDS who are experiencing clinically significant cytopenias or increased marrow blasts, significant anemia, or are...

Benefits of Hematopoietic Stem Cell Transplantation in Advanced Myelodysplastic Syndromes

Originally Published: 11/20/2020
Article Source: External Web Content
Among fit older adults with advanced myelodysplastic syndromes (MDS) undergoing hematopoietic stem cell transplantation (HSCT), survival is significantly improved when transplantation is performed early or for adverse risk disease but not for patients with standard risk disease with severe cytopenia, according to study results published in Leukemia. For patients with MDS, the median age at the time of diagnosis is approximately 70 years, and patients typically have an overall survival of 1 year in advanced disease. Although the only known curative therapy is HSCT, the study authors noted...

Seventh International Bone Marrow Failure Scientific Symposium - Summary for Patients

Originally Published: 11/18/2020
Article Source: AAMDSIF Article
We are pleased to present this Summary for Patients of the Seventh AAMDSIF International Bone Marrow Failure Scientific Symposium held virtually on July 15, 16 & 17, 2020. Our Symposium brought together many of the worldʼs leading experts on the biology and treatment of aplastic anemia, myelodysplastic syndromes, paroxysmal nocturnal hemoglobinuria, acute myeloid leukemia and related disorders. Despite the virtual format due to the coronavirus pandemic, our registration was higher than ever, with over 750 participants from 38 nations. The Aplastic Anemia and MDS International Foundation...

Human and artificial intelligence to illuminate MDS

Originally Published: 11/12/2020
Article Source: External Web Content
In this issue of Blood, Nagata et al show for the first time that machine learning (ML) algorithms can discern patterns and identify diagnostically and prognostically relevant associations between genetic variants and cytomorphological changes in myelodysplastic syndromes (MDS), correlations that are too subtle or complex for the human eye to observe. We know about many phenotype-genotype correlations in hematology, including PML-RARA and the classic hypergranular abnormal promyelocytes of acute promyelocytic leukemia, germline GATA2 mutations, and myelokathexis. In MDS, examples include the...