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.
Outcomes of Hematopoietic Cell Transplantation With Post-Transplant Cyclophosphamide-Based GVHD Prophylaxis in Acute Myeloid Leukemia and Myelodysplastic Syndromes Patients Over 70 Years of Age: A Comparative Analysis by Age Group
Abstract Background We aimed to study outcomes of allogeneic hematopoietic cell transplantation in acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) patients aged above 70 with using post-transplant cyclophosphamide (PTCy) graft-versus-host disease (GVHD)…
Expert Opinion on the Diagnosis and Treatment of Hematologic Malignancies During Pregnancy
Abstract The diagnosis and treatment of hematologic malignancies has undergone significant advancements over the past few decades, resulting in enhanced outcomes. For hematologic malignancies diagnosed during pregnancy, this poses new questions. As possibilities continue to…
Patients with myelodysplastic/myeloproliferative neoplasms have higher rates of relapse following allogeneic hematopoietic cell transplantation compared to those with MDS: A Brazilian SBTMO/CIBMTR registry analysis
Correspondence The Myelodysplastic/Myeloproliferative neoplasm (MDS/MPN) overlap syndromes are a heterogeneous group of rare hematologic malignancies characterized by features of both MDS such as morphologic dysplasia and cytopenias and MPNs including elevated blood counts and…
A phase 1/2 study of donor-derived anti-CD33 CAR T-cell therapy (VCAR33) for relapsed/refractory AML after allogeneic HCT
Key Points Timely manufacture of VCAR33, a donor-derived CD33-directed CAR T, was feasible in patients with relapsed/refractory AML after alloHCT. VCAR33 had an acceptable safety profile at doses up to 3 × 106 cells/kg and demonstrated antileukemic activity with a response rate…
Donor T cells on double duty: VCAR33 for AML after transplant
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…
How I select hematopoietic cell donors in the era of posttransplant cyclophosphamide
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…
Impact of Modern Strategies for Graft-Versus-Host Disease Prophylaxis on Relapse Risk in Matched-Sibling or Unrelated Donor Hematopoietic Cell Transplantation: A Systematic Review and Meta-Analysis of Randomized Trials
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…
Imputing causality and clonal dynamics from single-cell transcriptomics in paroxysmal nocturnal hemoglobinuria
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…
Prognostic significance of PNH clones in aplastic anemia treated with immunosuppression or allogeneic HSCT: a 20-year single-center experience
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…
