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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.

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...

Gene expression signature predicts relapse in adult patients with cytogenetically normal acute myeloid leukemia

Originally Published: 03/08/2021
Article Source: External Web Content
Key Points The weighted expressions of 7 coding and 3 noncoding genes is strongly associated with relapse in CN-AML patients. The 10-gene signature is independent from mutations known to associate with outcome in AML patients. Abstract Although ∼80% of adult patients with cytogenetically normal acute myeloid leukemia (CN-AML) achieve a complete remission (CR), more than half of them relapse. Better identification of patients who are likely to relapse can help to inform clinical decisions. We performed RNA sequencing on pretreatment samples from 268 adults with de novo CN-AML who were younger...

Myeloablative vs. Reduced Intensity Conditioning for Hematopoietic Cell Transplantation in Acute Myeloid Leukemia and Myelodysplastic Syndromes—Long Term Follow up of BMT CTN 0901 Clinical Trial

Originally Published: 02/26/2021
Article Source: External Web Content
Abstract Background : Several prospective randomized trials comparing conditioning intensity prior to allogeneic hematopoietic cell transplantation (HCT) have been performed with conflicting results. While reduced intensity conditioning (RIC) leads to lower treatment-related mortality (TRM) this is offset by higher rates of relapse. Long term follow-up of randomized comparative trials are limited. Here we present long term follow up of a randomized comparison of myeloablative conditioning (MAC) compared to RIC prior to HCT for acute myeloid leukemia (AML) or myelodysplasia (MDS). Objective...

Single cord blood transplantation for acute myeloid leukemia patients aged 60 years or older: a retrospective study in Japan

Originally Published: 02/24/2021
Article Source: External Web Content
This article was originally published here Ann Hematol. 2021 Feb 23. doi: 10.1007/s00277-021-04464-5. Online ahead of print. ABSTRACT The availability of alternative donor sources could allow elderly patients to receive allogeneic hematopoietic cell transplantation (HCT). We retrospectively evaluated the outcomes of single-unit cord blood transplantation (CBT) in 1577 patients aged ≥60 years with acute myeloid leukemia (AML) in Japan between 2002 and 2017. In total, 990 (63%) patients were not in complete remission (CR) at the time of CBT. A myeloablative conditioning regimen (52%) and...

Subcutaneous immunoglobulin in allogeneic hematopoietic cell transplant patients: a prospective study of feasibility, safety, and healthcare resource use

Originally Published: 02/23/2021
Article Source: External Web Content
Abstract Background We evaluated feasibility, safety, and total resource use of subcutaneous immunoglobulin (SCIG) in a pilot study of patients who underwent allogeneic hematopoietic cell transplant (HCT) over a 6-month period. Methods A total of 20 eligible patients were treated with SCIG at 0.1 g/kg/week for up to 6 months. Patients were matched to 20 concurrent intravenous immunoglobulin (IVIG) controls. Clinical outcomes measured included adverse reactions, healthcare resource use, patient satisfaction, and quality of life (QOL). (ClinicalTrials.gov Identifier: NCT03401268.) Results...

Scientists discover two unique subtypes of a prominent mutation in patients with AML

Originally Published: 02/16/2021
Article Source: External Web Content
Using advanced RNA sequencing, scientists have identified two unique subtypes of a prominent mutation present in many patients with Acute Myeloid Leukemia (AML) – called NPM1 – that could help predict survival and improve treatment response for patients whose leukemic cells bear the mutation. In research published Feb. 16, in Nature Communications, a team led by Princess Margaret Cancer Centre Senior Scientists, Drs. Benjamin Haibe-Kains, Aaron Schimmer and Mark Minden, have discovered that within the NPM1 mutation of AML there exists two unique subtypes, one of which can be effectively...