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News and Treatment Updates

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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.

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
Article Source: Press Release
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
Article Source: External Web Content
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
Article Source: Foundation Update

COVID-19 vaccination for hematopoietic stem cell transplant patients

Originally Published: 05/14/2021
Article Source: External Web Content
The SARS-CoV-2 pandemic has changed the world over the last year and impacted almost every part of our lives. In particular, patients with hematologic malignancies have been significantly affected by this crisis at every stage of their treatment. Delays in diagnosis and increased waiting times for transplants and therapy have become common occurrences. Patients with hematologic malignancies are also more likely to suffer from severe COVID-19, leading to a greater mortality rate. A recent meta-analysis of 3,377 patients with hematologic malignancies and COVID-19 showed a mortality rate of 34...

Clinical effects of administering leukemia-specific donor T cells to patients with AML/MDS after allogeneic transplant

Originally Published: 05/13/2021
Article Source: External Web Content
Key Points Donor-derived T cells with native specificity for multiple myeloid leukemia antigens can be expanded ex vivo. Infusion of mLSTs after HCT is well tolerated and produces antileukemia effects. Abstract Relapse after allogeneic hematopoietic stem cell transplantation (HCT) is the leading cause of death in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS). Infusion of unselected donor lymphocytes (DLIs) enhances the graft-versus-leukemia (GVL) effect. However, because the infused lymphocytes are not selected for leukemia specificity, the GVL effect is often...

Participation of Black Americans in Cancer Clinical Trials: Current Challenges and Proposed Solutions

Originally Published: 05/11/2021
Article Source: External Web Content
ABSTRACT Low participation of Black Americans in cancer clinical trials is a well-established predicament. Many factors resulted in this current dilemma with racism being the fundamental unit. Here, we discuss some current challenges and proposed solutions to help in increasing the enrollment of Black Americans in cancer clinical trials. We suggest implementing the least acceptable race-specific percentage as a new bar that registrational clinical trials need to pass before cancer drugs approval. Clinical trials will continue to draw the future of cancer therapeutics in which we believe that...

Researchers Uncover How MN1 Overexpression Causes AML

Originally Published: 05/10/2021
Article Source: External Web Content
Meningioma-1 (MN1) is a protein whose overexpression has been linked to acute myeloid leukemia (AML) and brain tumors, and 70% to 80% of AML patients with high overexpression of MN1 die within two years. Yet the mechanism by which MN1 causes these diseases has largely remained a mystery. “Looking at the sequence, the protein has no obvious structure,” said Kathrin M. Bernt, MD, a pediatric oncologist in the Cancer Center’s Leukemia and Lymphoma Program at Children’s Hospital of Philadelphia (CHOP). “MN1 doesn’t resemble other typical oncogenes – or any other proteins – so it’s difficult to...

“If it’s the time, it’s the time”: Existential communication in naturally-occurring palliative care conversations with individuals with advanced cancer, their families, and clinicians

Originally Published: 05/10/2021
Article Source: External Web Content
Highlights We analyzed naturally-occurring inpatient palliative care conversations. Existential communication is pervasive and varied in palliative care conversations Patients, families, and clinicians talk about time, the self, and connectedness. Findings help develop a lexicon for everyday existential communication. (Article continues at link.)

Aplastic Anemia and MDS International Foundation and NORD Launch New Natural History Study of Paroxysmal Nocturnal Hemoglobinuria (PNH)

Originally Published: 05/06/2021
Article Source: Press Release

Mesothelin is a novel cell surface disease marker and potential therapeutic target in acute myeloid leukemia

Originally Published: 05/03/2021
Article Source: External Web Content
Key Points Mesothelin is aberrantly expressed in over one third of childhood and young adult AML and not expressed on normal hematopoietic cells. Mesothelin is successfully targeted in vitro and in xenograft models of MSLN+ AML with ADCs. Abstract In an effort to identify acute myeloid leukemia (AML)-restricted targets for therapeutic development in AML, we analyzed the transcriptomes of 2051 children and young adults with AML and compared the expression profile with normal marrow specimens. This analysis identified a large cohort of AML-restricted genes with high expression in AML, but low...