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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.

What have we learned about TP53-mutated acute myeloid leukemia?

Originally Published: 11/19/2024
Article Source: External Web Content
Abstract TP53 is a tumor suppressor gene frequently mutated in human cancers and is generally associated with poor outcomes. TP53 mutations are found in approximately 5% to 10% of patients with de novo acute myeloid leukemia (AML), more frequently observed in elderly patients and those with therapy-related AML. Despite recent advances in molecular profiling and the emergence of targeted therapies, TP53-mutated AML remains a challenge to treat. Current treatment strategies, including conventional chemotherapy, hypomethylating agents, and venetoclax-based therapies, have shown limited efficacy...

Risk prediction for clonal cytopenia: multicenter real-world evidence

Originally Published: 11/07/2024
Article Source: External Web Content
Key Points A 3-parameter CCRS model was devised specifically for patients diagnosed with clonal cytopenia. The CCRS offers precise CCUS risk assessment for patient management and clinical trial eligibility. Abstract Clonal cytopenia of undetermined significance (CCUS) represents a distinct disease entity characterized by myeloid-related somatic mutations with a variant allele fraction of ≥2% in individuals with unexplained cytopenia(s) but without a myeloid neoplasm (MN). Notably, CCUS carries a risk of progressing to MN, particularly in cases featuring high-risk mutations. Understanding...

Abatacept for acute graft-versus-host disease prophylaxis after unrelated donor hematopoietic cell transplantation

Originally Published: 10/24/2024
Article Source: External Web Content
Key Points This study illustrates the survival benefits of abatacept combined with CNI/MTX in patients with hematologic malignancies undergoing HCT. The addition of abatacept may provide an approach for alternative donor pool expansion when HLA-identical sibling donors are unavailable. Abstract Abatacept plus calcineurin inhibitors/methotrexate (CNI/MTX) is the first US Food and Drug Administration (FDA)-approved regimen for acute graft-versus-host disease (aGVHD) prophylaxis during unrelated-donor hematopoietic cell transplantation (URD-HCT). Using Center for International Blood and Marrow...

Germ line ERG haploinsufficiency defines a new syndrome with cytopenia and hematological malignancy predisposition

Originally Published: 10/24/2024
Article Source: External Web Content
Abstract The genomics era has facilitated the discovery of new genes that predispose individuals to bone marrow failure (BMF) and hematological malignancy (HM). We report the discovery of ETS-related gene (ERG), a novel, autosomal dominant BMF/HM predisposition gene. ERG is a highly constrained transcription factor that is critical for definitive hematopoiesis, stem cell function, and platelet maintenance. ERG colocalizes with other transcription factors, including RUNX family transcription factor 1 (RUNX1) and GATA binding protein 2 (GATA2), on promoters or enhancers of genes that...

High-dose IV ascorbic acid therapy in CCUS patients with TET2 mutations

Originally Published: 10/01/2024
Article Source: External Web Content
This was a phase II trial, evaluating the safety/efficacy of high-dose IV-ascorbic acid in patients with TET2 mutant CCUS. Eight of 10 patients enrolled were eligible for response assessment. At week 20, there were no responses by IWG MDS criteria. NCT03418038 (pdf available at link)

Learn about PNH (Paroxysmal Nocturnal Hemoglobinuria)

Originally Published: 09/27/2024
Article Source: AAMDSIF Article
Recently, there has been increased mention of paroxysmal nocturnal hemoglobinuria (PNH) in the news and on social media.  As part of our mission, the Aplastic Anemia and MDS International Foundation (AAMDSIF) works with leading PNH specialists to provide patients, their support networks, and the public with trusted and up-to-date educational resources about PNH.   According to Dr. Carlos DeCastro, PNH expert and member of the AAMDSIF Medical Advisory Board, "PNH is a lifelong rare complex blood disorder that requires management by a PNH specialist.  It is important for patients and their...

Venetoclax plus decitabine as a bridge to allogeneic haematopoietic stem-cell transplantation in older patients with acute myeloid leukaemia (VEN-DEC GITMO): final report of a multicentre, single-arm, phase 2 trial

Originally Published: 09/20/2024
Article Source: External Web Content
Summary Background Access to allogeneic haematopoietic stem-cell transplantation (HSCT) remains challenging for older patients (aged >60 years) with acute myeloid leukaemia. We aimed to evaluate the efficacy of venetoclax plus decitabine as first-line therapy and bridge to transplantation in this patient population. Methods This multicentre, single-arm, phase 2 trial was conducted in 20 Gruppo Italiano Trapianto Midollo Osseo (GITMO) centres in Italy. Patients aged ≥60 and <75 years, with newly diagnosed acute myeloid leukaemia categorised as intermediate or high risk according to 2016...

Exercise Treatment as Part of Multidisciplinary Whole Person Care in Oncology

Originally Published: 08/21/2024
Article Source: External Web Content
Several national agencies, including the American Society of Clinical Oncology (ASCO), recommend the prescription of exercise for patients diagnosed with cancer.1,2 The evidence base for this recommendation is derived from studies that showed exercise-related improvements in patient-reported outcomes (PROs), treatment-associated adverse effects, cardiorespiratory fitness (CRF), and overall physical functioning.3-6 Additionally, exercise may have direct anticancer effects and indirect effects by synergizing standard cancer therapies.7,8 Despite these benefits, the implementation of exercise...

Navigating a Paradigm Shift Venetoclax Treatment Redefines Landscape of Acute Myeloid Leukemia

Originally Published: 08/19/2024
Article Source: External Web Content
Abstract Acute myeloid leukemia (AML) is a heterogeneous disease characterized by the accumulation of malignant myeloid progenitor hematopoietic cells in the bone marrow and peripheral blood. Recent studies have shown promising results with the use of small molecule inhibitors and targeted therapy in the treatment of patients with AML. One such molecule is venetoclax, which has been approved in AML by the FDA in combination with hypomethylating agents or low-dose cytarabine. We thoroughly searched electronic literature related to venetoclax and its role in AML, using databases such as...

Safe, Successful Pregnancies Possible After AlloHCT

Originally Published: 07/15/2024
Article Source: External Web Content
Findings refute former consensus that pregnancies post-transplant are nearly impossible, highlight need for increased fertility counseling   (WASHINGTON, July 15, 2024) — Despite treatment-related fertility challenges, female patients can become pregnant and give birth to healthy children after undergoing allogeneic hematopoietic cell transplantation (alloHCT), according to a study published in Blood. During alloHCT, stem cells from a healthy donor are transplanted to individuals with hematologic cancers or benign hematologic disorders such as leukemia and sickle cell disease. Procedural...