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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.

Azacitidine Maintenance Fails to Improve Post-Transplant Outcomes in High-Risk AML and MDS

Originally Published: 01/15/2021
Maintenance treatment with single-agent azacitidine at a dose of 32 mg/m2 daily for five days did not lead to improved survival in patients with high-risk myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML) who had undergone allogeneic hematopoietic cell transplantation (HCT), according to findings from a phase III randomized controlled trial published in Blood Advances. Despite the disappointing results, “this randomized trial with azacitidine maintenance showed that a prospective trial in the posttransplant setting was feasible and safe but challenging,” the authors, led by...

Outcomes of Adding a Fourth Chemotherapy Course in AML

Originally Published: 01/14/2021
Administering 2 additional courses of chemotherapy with high-dose cytarabine (Ara-C) after 2 courses of induction therapy improved relapse rates but did not improve overall survival for patients with acute myeloid leukemia (AML), according to research in the Journal of Clinical Oncology.1  For patients with AML who are younger than age 60 years, the optimal number of chemotherapy courses has been unclear. Previous studies found that 5 total courses offered no additional benefit over 4 courses of treatment.2,3 Therefore, a team of investigators conducted an analysis to determine whether 3 or...

Real-World Treatment Patterns, FLT3 Testing, and Outcomes in AML

Originally Published: 01/14/2021
Amer Zeidan, MBBS, MHS, Yale University School of Medicine and Yale Cancer Center, New Haven, Connecticut, discusses updated results from an ongoing retrospective study examining FLT3 testing trends, treatment patterns, and overall survival in patients with relapsed/refractory FLT3-positive acute myeloid leukemia (AML). Dr Zeidan presented these results at the virtual 62nd American Society of Hematology (ASH) Annual Meeting. Transcript Hello. My name is Amer Zeidan and I'm Associate Professor of Medicine at Yale University, in section of hematology, and the director of Hematology Early...

Eprenetapopt (APR-246) and Azacitidine in TP53-Mutant Myelodysplastic Syndromes

Originally Published: 01/11/2021
PURPOSE Approximately 20% of patients with TP53-mutant myelodysplastic syndromes (MDS) achieve complete remission (CR) with hypomethylating agents. Eprenetapopt (APR-246) is a novel, first-in-class, small molecule that restores wild-type p53 functions in TP53-mutant cells. METHODS This was a phase Ib/II study to determine the safety, recommended phase II dose, and efficacy of eprenetapopt administered in combination with azacitidine in patients with TP53-mutant MDS or acute myeloid leukemia (AML) with 20%-30% marrow blasts ( identifier: NCT03072043). RESULTS Fifty-five...

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...
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