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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.

Pegcetacoplan Bests Eculizumab for PNH Patients

Originally Published: 12/28/2020
Article Source: External Web Content
Results from the phase III PEGASUS trial suggest that the targeted C3 inhibitor pegcetacoplan improved hemoglobin levels better than eculizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH) who were still anemic despite at least three months of eculizumab therapy. The data were presented at December's American Society of Hematology virtual meeting. In this exclusive MedPage Today video, study co-author Ilene C. Weitz, MD, of Keck School of Medicine at the University of Southern California, discusses the trial's findings. (VIDEO at Link)

Naval G. Daver, MD, on the Past Year in Acute Myeloid Leukemia Research

Originally Published: 12/25/2020
Article Source: External Web Content
Despite only one new FDA approval in 2020 for treatment of patients with acute myeloid leukemia (AML), tremendous progress was still made. Several new combination regimens, including venetoclax (Venclexta) and azacitidine (Vidaza) for patients with previously untreated AML, showed positive results and improved outcomes. In an interview with CancerNetwork®, Naval G. Daver, MD, associate professor in the Department of Leukemia at MD Anderson Cancer Center, discussed the latest advancements in AML and what trial data he is looking forward to most in 2021. (VIDEO at Link)

Transplant May Improve Survival in Older Patients With High-Risk Myelodysplastic Syndrome, Study Reports

Originally Published: 12/25/2020
Article Source: External Web Content
Stem cell transplants are not frequently offered to older patients with high-risk myelodysplastic syndromes (MDS). According to a study from the Blood and Marrow Transplant Clinical Trials Network (BMTCTN 1102), these patients may indeed achieve a survival benefit from stem cell transplant. As reported by Corey Cutler, MD, MPH, FRCPC, of Dana-Farber Cancer Institute, Boston, at the 2020 American Society of Hematology (ASH) Annual Meeting & Exposition, subjects between the ages of 50 and 75 with HLA-compatible donors had nearly a doubled survival rate, without compromising quality of life...

Short telomere length predicts nonrelapse mortality after stem cell transplantation for myelodysplastic syndrome

Originally Published: 12/24/2020
Article Source: External Web Content
Abstract Allogeneic hematopoietic stem cell transplantation is the only potentially curative treatment for patients with myelodysplastic syndrome (MDS), but long-term survival is limited by the risk of transplant-related complications. Short telomere length, mediated by inherited or acquired factors, impairs cellular response to genotoxic and replicative stress and could identify patients at higher risk for toxicity after transplantation. We measured relative telomere length in pretransplant recipient blood samples in 1514 MDS patients and evaluated the association of telomere length with...

Short telomere length predicts nonrelapse mortality after stem cell transplantation for myelodysplastic syndrome

Originally Published: 12/24/2020
Article Source: External Web Content
Key Points Recipient telomere length is independently associated with overall survival and NRM after transplantation for MDS. The effect of telomere length on NRM was most prominent after higher intensity conditioning and in patients with severe acute graft-versus-host disease. Abstract Allogeneic hematopoietic stem cell transplantation is the only potentially curative treatment for patients with myelodysplastic syndrome (MDS), but long-term survival is limited by the risk of transplant-related complications. Short telomere length, mediated by inherited or acquired factors, impairs cellular...

Dr. Tony Lin Summarizes Venclexta Data Presented at ASH

Originally Published: 12/21/2020
Article Source: External Web Content
The all-virtual 62nd ASH Annual Meeting & Exposition highlighted some of the newest data about Venclexta. DocWire News interviewed Tony Lin, Pharm.D., a Senior Scientific Director with AbbVie US medical affairs, to discuss this more in-depth. Dr. Lin is a pharmacist by training and has been working in the oncology therapeutic area and biopharmaceutical industry for over 12 years. DocWire News: What patients is Venclexta indicated to treat? Dr. Lin: In the U.S., VENCLEXTA is approved for multiple blood cancers, including for adults with chronic lymphocytic leukemia (CLL) or small...

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...