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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.

The development and clinical use of oral hypomethylating agents in acute myeloid leukemia and myelodysplastic syndromes: dawn of the total oral therapy era

Originally Published: 04/14/2021
Article Source: External Web Content
Abstract Introduction Intravenous and subcutaneous hypomethylating agents have held a key role in myelodysplastic syndrome, chronic myelomonocytic leukemia and acute myeloid leukemia treatment. Following the approval of the cedazuridine/decitabine combination, ASTX727, as well as development of an oral formulation of azacitidine, CC-486, in the USA in 2020, these agents could gradually replace their injectable counterparts. Areas covered ASTX727 is approved for the treatment of adult patients with intermediate 1 or high risk MDS as well as those with chronic myelomonocytic leukemia based on...

HLA Haploidentical versus Matched Unrelated Donor Transplants with Post-Transplant Cyclophosphamide based prophylaxis

Originally Published: 04/13/2021
Article Source: External Web Content
Key Points Higher mortality with reduced intensity regimens after haploidentical relative compared to matched unrelated donor transplantation Higher grade III-IV acute graft versus host disease after haploidentical relative compared to matched unrelated donor transplantation Post transplant cyclophosphamide (PTCy) graft-versus-host disease (GVHD) prophylaxis has allowed haploidentical (Haplo) transplantation to be performed with results similar to that after matched unrelated donor (MUD) transplantation with traditional prophylaxis. The relative value of transplantation with MUD versus Haplo...

Yoga in Oncologic Care: An Evidence-Based Treatment to Improve Outcomes

Originally Published: 04/10/2021
Article Source: External Web Content
The ASCO Post’s Integrative Oncology series is intended to facilitate the availability of evidence-based information on integrative and complementary therapies sometimes used by patients with cancer. Increasingly, patients with cancer desire nonpharmacologic strategies for managing their symptoms. Yoga is a time-honored mind-body practice that has been shown to improve both physical and psychological well-being. In this article, Lorenzo Cohen, PhD, and Santosshi Narayanan, MD, summarize the current evidence surrounding the use of yoga in oncology settings and highlight the need for increased...

Aprea Therapeutics Receives FDA Orphan Drug Designation for Eprenetapopt for the Treatment of Acute Myeloid Leukemia (AML)

Originally Published: 04/08/2021
Article Source: External Web Content
BOSTON, April 08, 2021 (GLOBE NEWSWIRE) -- Aprea Therapeutics, Inc. (Nasdaq: APRE), a biopharmaceutical company focused on developing and commercializing novel cancer therapeutics that reactivate the mutant tumor suppressor protein, p53, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to eprenetapopt for treatment of AML. “We are pleased to have been granted Orphan Drug designation by FDA for eprenetapopt in AML, building on the Fast Track designation in AML that was granted in November 2020,” said Christian S. Schade, Chairman and Chief...

Pegcetacoplan Superior to Eculizumab for Paroxysmal Nocturnal Hemoglobinuria

Originally Published: 04/08/2021
Article Source: External Web Content
Pegcetacoplan was superior to eculizumab in improving outcomes in patients with paroxysmal nocturnal hemoglobinuria (PNH) according to a recent study published in the New England Journal of Medicine. PNH is a rare, acquired hematologic disease that causes fatigue, increased risk of thrombosis, and bone marrow dysfunction. People with PNH often have chronic hemolysis. C5 inhibitors control intravascular hemolysis and have reduced life-threatening complications of PNH, transforming outcomes for these patients. However, extravascular hemolysis still occurs in patients being treated with C5...

Blood cancer risk higher than expected in kids with Down syndrome

Originally Published: 04/07/2021
Article Source: External Web Content
New York, April 7 The risks of acute myeloid leukaemia (AML)—a type of blood cancer—in children with Down syndrome is stronger than expected, according to a new study. The study led by researchers from the University of Chicago, Davis Health and San Francisco, examined medical data of more than 3.9 million children born between 1996-2016 in seven US healthcare systems or in Ontario, Canada.  It showed that 2.8 per cent of children with Down syndrome were diagnosed with leukaemia, compared to 0.05 per cent of other children. Compared to other children, kids with Down syndrome had a higher...

MDS Update Q1 2021

Originally Published: 04/07/2021
Article Source: MDS Update

Impact of Conditioning Intensity and Genomics on Relapse After Allogeneic Transplantation for Patients With Myelodysplastic Syndrome

Originally Published: 03/25/2021
Article Source: External Web Content
ABSTRACT PURPOSE Patients with myelodysplastic syndrome (MDS) are at risk of relapse after allogeneic hematopoietic cell transplantation. The utility of ultra-deep genomic testing to predict and the impact of conditioning intensity to prevent MDS relapse are unknown. METHODS Targeted error-corrected DNA sequencing was performed on preconditioning blood samples from patients with MDS (n = 48) from the Blood and Marrow Transplant Clinical Trials Network 0901 phase III randomized clinical trial, which compared outcomes by allogeneic hematopoietic cell transplantation conditioning intensity in...

What's New? Foundation Update Newsletter March 2021

Originally Published: 03/16/2021
Article Source: Foundation Update

Clinical and molecular predictors of response and survival following venetoclax therapy in relapsed/refractory AML

Originally Published: 03/09/2021
Article Source: External Web Content
Key Points In patients with RR-AML, venetoclax combination therapy resulted in responses in 31% of patients and a median OS of 6.1 months. NPM1 mutations predicted higher response rates; adverse cytogenetics and mutations in TP53, KRAS/NRAS, and SF3B1 predicted worse OS. Abstract Azacitidine + venetoclax, decitabine + venetoclax, and low-dose cytarabine + venetoclax are now standard treatments for newly diagnosed older or unfit patients with acute myeloid leukemia (AML). Although these combinations are also commonly used in relapsed or refractory AML (RR-AML), clinical and molecular...