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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.

Clinical development of our investigational therapy, pegcetacoplan

Originally Published: 01/08/2021
Dear members of the PNH community,  We are excited to share important updates with you regarding the clinical development of our investigational therapy, pegcetacoplan, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). Since our last update, we have partnered with Sobi, a global biopharmaceutical company, to develop pegcetacoplan. Apellis will be responsible for bringing pegcetacoplan to individuals with PNH in the United States (U.S.) while Sobi will work to bring pegcetacoplan to people with PNH outside of the U.S.  The U.S. Food and Drug Administration (FDA) and European...

The metabolic reprogramming in acute myeloid leukemia patients depends on their genotype and is a prognostic marker

Originally Published: 01/06/2021
Key Points Different metabolic pathways are involved in intracellular metabolic reprogramming depending on the genetic characteristics of AML cells. Leukemic progenitors activate different programs of the metabolism, depending on their levels of differentiation blockade. (Article continues at link.)

Could Baking Soda Fight Leukemia Relapse After Stem Cell Transplant?

Originally Published: 01/05/2021
For patients with leukemia and other hematological malignancies, donor hematopoietic stem cells can recognize and attack remaining cancer cells after chemotherapy. But residual cancer cells’ immune-suppressing effects can thwart these transplants and lead to eventual relapse, requiring a donor lymphocyte infusion. A new study suggests that a treatment as simple as sodium bicarbonate—better known as baking soda—can potentially boost donor lymphocytes’ ability to overcome relapse after stem cell transplants. In a previous study, the same research team found that T cells from hematopoietic stem...

Imetelstat Improves Rate of Durable Transfusion Independence in Patients With Lower-Risk MDS

Originally Published: 01/01/2021
Treatment with imetelstat, a competitive inhibitor of telomerase enzymatic activity, was associated with a clinically meaningful rate of transfusion independence in heavily transfused patients with lower-risk myelodysplastic syndromes (MDS), according to study results published in the Journal of Clinical Oncology. In the open-label, phase II IMerge trial, David Steensma, MD, of Dana-Farber Cancer Institute, and colleagues evaluated the efficacy and safety of imetelstat in 57 patients with lower-risk MDS who were red blood cell (RBC) transfusion-dependent and ineligible for or relapsed/...

Ivosidenib and Azacitidine Combination Demonstrates Durable Activity in Newly Diagnosed AML

Originally Published: 01/01/2021
Ivosidenib, an inhibitor of the mutant isocitrate dehydrogenase 1 (IDH1) enzyme, was well tolerated and associated with deep and durable responses in combination with azacitidine in patients with newly diagnosed acute myeloid leukemia (AML), according to findings published in the Journal of Clinical Oncology. According to lead study author Courtney DiNardo, MD, of the University of Texas MD Anderson Cancer Center, the ivosidenib and azacitidine combination regimen studied in this small trial may be considered as an appropriate frontline combination approach for older patients with IDH1-...

Precision Medicine Approach Deemed Safe, Feasible for Older Patients With Untreated AML

Originally Published: 01/01/2021
A precision medicine treatment approach that incorporates genomic data into treatment decisions appears to be feasible in older patients with untreated acute myeloid leukemia (AML), according to a study published in Nature Medicine. Investigators also found that delaying treatment until mutational data were available did not seem to increase the risk of early death or adversely impact overall survival (OS). According to the investigators of this study, delaying therapy to conduct detailed molecular profiling was considered safe, except for in patients with rapid proliferative disease or...

Pegcetacoplan Bests Eculizumab for PNH Patients

Originally Published: 12/28/2020
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
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
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
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...
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