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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.

Overcoming Clinical Challenges Associated With TP53-Mutant MDS, AML

Originally Published: 12/15/2020
Article Source: External Web Content
TP53-mutant myelodysplastic syndromes (MDS) and acute myeloid leukemia (AML) represent a molecularly distinct, poor-risk patient subgroup frequently associated with complex karyotypes, high propensity for relapse, and inferior overall survival. Across TP53-altered MDS and AML, standard therapies for wild-type disease have been of minimal benefit, but a wave of investigational agents currently in development offers promise for patients with TP53-altered malignancies, which affect 8% to 12% and 5% to 10% of patients with MDS and AML, respectively. In contrast with wild-type, FLT3-, and IDH1/2-...

Clinical Challenges: Paroxysmal Nocturnal Hemoglobinuria — Complement inhibitors a major advance but not a cure

Originally Published: 12/09/2020
Article Source: External Web Content
Paroxysmal nocturnal hemoglobinuria (PNH) is an extremely rare, acquired, life-threatening disease of the blood. The name comes from one of the clinical consequences of the disease -- the breakdown of red blood cells and consequent release of hemoglobin into the urine. It often results in dark-colored urine most prominently seen in the morning. PNH is caused by deficiency in glycophosphatidylinositol-anchored biosynthesis, leaving red blood cells susceptible to destruction -- i.e., hemolysis -- by the body's complement system. Though the condition is rare, its diagnosis is straightforward,...

Guadecitabine Maintenance Therapy Shows Promise in High Risk MDS/AML Patients

Originally Published: 12/08/2020
Article Source: External Web Content
Guadecitabine (SGI-110), when given as maintenance therapy following allogeneic stem cell transplantation (allo-SCT), showed promising relapse-free survival rates (RFS) with manageable safety in high-risk patients with myelodysplastic syndromes/acute myeloid leukemia (MDS/AML), according to data presented by Betul Oran, MD, MS, Stem Cell Transplantation and Cellular Therapy, The University of Texas, MD Anderson Cancer Center, Houston, TX, at the virtual 62nd American Society of Hematology (ASH) Annual Meeting and Exposition.  “Disease relapse remains to be one of the major reasons [for[...

Oral Azacitidine Improves, Sustains Health-Related QoL in AML

Originally Published: 12/06/2020
Article Source: External Web Content
Treatment with the oral agent showed sustained health-related quality of life compared with placebo in patients with acute myeloid leukemia, according to results of the phase 3 QUAZAR AML-001 trial. Treatment with oral azacitidine (CC-486) showed sustained health-related quality of life (HRQoL), compared with placebo, in patients with acute myeloid leukemia (AML), according to results of the phase 3 QUAZAR AML-001 trial.1 Results from HRQoL analysis of the QUAZAR AML-001 trial were presented by Gail J. Roboz, MD, professor of Medicine, Weill Cornell Medical College, during the American...

Significant Racial, Ethnic Related Socioeconomic Disparities Influence Survival in AML

Originally Published: 12/06/2020
Article Source: External Web Content
Census tract socioeconomic status information demonstrated significant disparities between survival outcomes of non-Hispanic white, non-Hispanic black, and Hispanic patients with acute myeloid leukemia AML in the Chicago metropolitan area. Significant disparities were found between survival outcomes of non-Hispanic white, non-Hispanic black, and Hispanic patients with acute myeloid leukemia (AML) in the Chicago metropolitan area , according to findings from a multilevel analysis presented virtually during the 2020 ASH Annual Meeting. Moreover, socioeconomic tract information, including...

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...