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.
In this issue of Blood, Mushtaq et al1 report on a phase 1/2 trial (NCT05984199, known as Vor Biopharma trial VBP301) utilizing donor-derived anti-CD33 chimeric antigen receptor–modified (CAR) T cells (VCAR33) in patients with relapsed acute myeloid leukemia (AML) following…
Abstract Selection of a hematopoietic progenitor cell donor for allogeneic hematopoietic cell transplantation (HCT) is essential for treatment planning; however, the parameters that define an “optimal” donor in the modern era are not well defined. Historically, donor-recipient…
ABSTRACT Regimens combining calcineurin inhibitors with methotrexate or mycophenolate have been the backbone for graft-versus-host disease (GVHD) prophylaxis. Early studies highlighted a delicate dose–response balance. The inclusion criteria comprised randomized clinical trials…
Abstract Paroxysmal nocturnal hemoglobinuria (PNH) originates from hematopoietic stem cells (HSCs) harboring somatic mutations in the phosphatidylinositol glycan class A (PIGA) gene. Clonal expansion of PIGA-mutated cells occurs uniquely in the setting of bone marrow (BM) failure…
Background Paroxysmal nocturnal hemoglobinuria (PNH) clones are detected in up to 60% of patients with aplastic anemia (AA); however, their prognostic impact remains incompletely defined, particularly in the context of frontline immunosuppressive therapy (IST) or allogeneic…
Abstract Myelodysplastic neoplasms, also known as myelodysplastic syndromes (MDS), are a heterogeneous group of myeloid malignancies characterized by ineffective haematopoiesis, cytopenias and a variably increased risk of progression to acute myeloid leukaemia. MDS primarily…
ABSTRACT The prognostic value of NPM1-mut Measurable Residual Disease (MRD) has been increasingly recognized. However, real-world data validating the prognostic impact of NPM1-mut MRD remains limited. The aim of this study was to assess the prognostic value of NPM1-mut MRD and to…
Key Points aGVHD causes loss of gastric stem cells, G-cells, and PCs, leading to increased gastric pH. Pentagastrin therapy repairs stem cell and PC deficits, reverses pH changes, and reduces aGVHD damage, improving survival. Abstract Acute graft-versus-host disease (aGVHD) is a…
Patients who receive hematopoietic stem cell transplants are at risk of developing graft-versus-host disease (GVHD), where the immune cells descended from the transplanted stem cells attack the transplant recipient’s own tissues. Acute GVHD (aGVHD) is often treated with…
Myelodysplastic syndromes/neoplasms (MDS) are myeloid malignancies with ineffective hematopoiesis, resulting in variable cytopenias and increased risk of progression to acute myeloid leukemia (AML) [1, 2]. Imetelstat, a first-in-class competitive telomerase inhibitor, is an…