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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.

Outcomes in patients with newly diagnosed TP53-mutated acute myeloid leukemia with or without venetoclax-based therapy

Originally Published: 06/28/2021
Abstract Background Venetoclax (VEN) in combination with a hypomethylating agent (HMA) has become the standard of care for patients aged >75 years and for those not eligible for intensive chemotherapy who have newly diagnosed acute myeloid leukemia (AML). The benefit of VEN-based therapy in patients who have newly diagnosed AML with mutations in the TP53 gene (TP53mut) over standard therapy is undefined. Methods In this single-institutional, retrospective analysis, the authors assessed the clinical outcomes of 238 patients with newly diagnosed TP53mut AML and compared the clinical...

Myeloproliferative Neoplasms Defined: What Are ET, PV, and MF?

Originally Published: 06/24/2021
What are essential thrombocythemia (ET) , polycythemia vera (PV), and myelofibrosis (MF) exactly? Dr. Naveen Pemmaraju explains how each of these blood disorders manifests along with the symptoms observed in these MPN patients. Dr. Naveen Pemmaraju is Director of the Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) Program in the Department of Leukemia at The University of Texas MD Anderson Cancer Center. Learn more about Dr. Pemmaraju, here. (Watch video at link above)

Pediatric Myelodysplastic Syndromes

Originally Published: 06/24/2021
Key points •Pediatric myelodysplastic syndrome (MDS) is rare and often develops in the setting of an underlying inherited bone marrow failure syndrome. •Refractory cytopenia of childhood and MDS with excess blasts comprise most cases. •Definitive diagnosis frequently requires repeat marrow sampling and correlation with ancillary laboratory testing. •Allogeneic hematopoietic stem cell transplant represents the only curative intervention, and should be considered in all patients based on clinical and genetic features. •Comprehensive next-generation sequencing–based studies are starting to...

How Immunity Generated from COVID-19 Vaccines Differs from an Infection

Originally Published: 06/22/2021
A key issue as we move closer to ending the pandemic is determining more precisely how long people exposed to SARS-CoV-2, the COVID-19 virus, will make neutralizing antibodies against this dangerous coronavirus. Finding the answer is also potentially complicated with new SARS-CoV-2 “variants of concern” appearing around the world that could find ways to evade acquired immunity, increasing the chances of new outbreaks. Now, a new NIH-supported study shows that the answer to this question will vary based on how an individual’s antibodies against SARS-CoV-2 were generated: over the course of a...

Core binding factor acute myelogenous leukemia-2021 treatment algorithm

Originally Published: 06/16/2021
Abstract Core binding factor acute myelogenous leukemia (CBF-AML), characterized by the presence of either t(8;21) (q22;q22) or inv(16) (p13q22)/t(16;16), is considered good-risk AML in the context of cytarabine based intensive chemotherapy. Still, outcome can be improved significantly through the effective implementation of available therapeutic measures and appropriate disease monitoring. The incorporation of gemtuzumab ozogamicin into frontline therapy should be standard. Cytarabine based induction/consolidation regimen may be combined with anthracycline (3 + 7 standard) or antimetabolite...

What's New? Foundation Update Newsletter June 2021

Originally Published: 06/15/2021

Alexion Announces FDA Approval of ULTOMIRIS® (ravulizumab-cwvz) for Children and Adolescents with Paroxysmal Nocturnal Hemoglobinuria (PNH)

Originally Published: 06/07/2021
 – With this approval, ULTOMIRIS is the first and only medicine approved in the U.S. to treat children and adolescents with PNH – – Approval based on interim results from Phase 3 study showing ULTOMIRIS demonstrated complete terminal complement inhibition through 26 weeks – BOSTON--(BUSINESS WIRE)--Jun. 7, 2021-- Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the U.S. Food and Drug Administration (FDA) has approved the expanded use of ULTOMIRIS® (ravulizumab-cwvz) to include children (one month of age and older) and adolescents with paroxysmal nocturnal hemoglobinuria (PNH)....

Apellis and Sobi Report Positive Top-line Results from the Phase 3 PRINCE Study of EMPAVELI™ (pegcetacoplan) in Treatment-naïve Patients with PNH

Originally Published: 05/25/2021
May 25, 2021 EMPAVELI demonstrated statistical superiority on the co-primary endpoints of hemoglobin stabilization (p<0.0001) and reduction in lactate dehydrogenase (LDH) (p<0.0001) compared to standard of care, which did not include complement inhibitors, at Week 26   Mean hemoglobin levels in the EMPAVELI group increased from 9.4 g/dL to 12.1 g/dL compared to an increase from 8.7 g/dL to 9.4 g/dL on standard of care (p=0.0019)   91% of patients on EMPAVELI were transfusion free compared to 22% on standard of care (p<0.0001)   The safety profile of EMPAVELI was consistent with...

Azacitidine Improves RBC-TI Rates in Patients with Lower-Risk MDS

Originally Published: 05/18/2021
CC-486—the oral formulation of azacitidine—significantly improved RBC transfusion independence (RBC-TI) rates and induced durable bilineage improvements in patients with lower-risk myelodysplastic syndromes (LR-MDS) and high-risk disease features, according to a phase 3 clinical trial published in the Journal of Clinical Oncology (2021 March 25. Epub ahead of print). Guillermo Garcia-Manero, MD, Department of Leukemia, University of Texas MD Anderson Cancer Center, Houston, TX, and colleagues, conducted this study to discover more treatment options for patients with LR-MDS by evaluating CC-...

What's New? Foundation Update Newsletter May 2021

Originally Published: 05/18/2021
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