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.
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...
Adenovirus infections after allogeneic hematopoietic cell transplantation in children and adults: a study from the Infectious Diseases Working Party of the European Society for Blood and Marrow Transplantation
Originally Published: 07/10/2024
Article Source: External Web Content
Abstract
The objective of the study was the analysis of clinical types, outcomes, and risk factors associated with the outcome of adenovirus (ADV) infection, in children and adults after allo-HCT. A total number of 2529 patients (43.9% children; 56.1% adults) transplanted between 2000 and 2022 reported to the EBMT database with diagnosis of ADV infection were analyzed. ADV infection manifested mainly as viremia (62.6%) or gastrointestinal infection (17.9%). The risk of 1-year mortality was higher in adults (p = 0.0001), and in patients with ADV infection developing before day +100 (p < 0....
NMDP and CIBMTR share new, promising stem cell transplantation trial data using mismatched, unrelated donors at the 2024 EHA Congress
Originally Published: 06/14/2024
Article Source: External Web Content
MINNEAPOLIS, June 14, 2024 — NMDPSM, a global nonprofit leader in cell therapy, and the CIBMTR® (Center for International Blood and Marrow Transplant Research®) announced interim results from the ACCESS trial as an oral abstract during the European Hematology Association (EHA) Annual Meeting in Madrid, Spain. The study demonstrated that adults with hematologic malignancies who received peripheral blood stem cell (PBSC) transplant from HLA-mismatched unrelated donors (MMUD) followed by post-transplant cyclophosphamide (PTCy) graft-versus-host-disease (GvHD) prophylaxis exhibited a 79% overall...
FDA approves imetelstat for low- to intermediate-1 risk myelodysplastic syndromes with transfusion-dependent anemia
Originally Published: 06/06/2024
Article Source: External Web Content
On June 6, 2024, the Food and Drug Administration approved imetelstat (Rytelo, Geron Corporation), an oligonucleotide telomerase inhibitor, for adults with low- to intermediate-1 risk myelodysplastic syndromes (MDS) with transfusion-dependent anemia requiring four or more red blood cell units over 8 weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs).
Full prescribing information for Rytelo will be posted here.
Efficacy was evaluated in IMerge (NCT02598661), a randomized (2:1), double-blind, placebo-controlled multicenter...
Blood and guts: how the intestinal microbiome shapes hematopoiesis and treatment of hematologic disease
Originally Published: 04/25/2024
Article Source: External Web Content
Abstract
Over the past 10 years, there has been a marked increase in recognition of the interplay between the intestinal microbiome and the hematopoietic system. Despite their apparent distance in the body, a large literature now supports the relevance of the normal intestinal microbiota to steady-state blood production, affecting both hematopoietic stem and progenitor cells as well as differentiated immune cells. Microbial metabolites enter the circulation where they can trigger cytokine signaling that influences hematopoiesis. Furthermore, the state of the microbiome is now recognized to...