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.
Very long-term remission with azacitidine in VEXAS syndrome
Originally Published: 06/01/2025
VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is a newly identified monogenic disorder with symptoms including recurrent fever, skin involvement, pulmonary infiltrates, systemic vasculitis, and chondritis.1Â Notably, 25-50% of patients also have myelodysplastic syndrome (MDS). VEXAS syndrome often requires high-dose steroids and is resistant to standard immunomodulatory agents. Emerging therapies such as JAK inhibitors, azacitidine (AZA), and, in severe selected cases, allogeneic stem cell transplantation (alloHSCT) show promise,2Â but optimal management remains...
Low rates of chronic graft-versus-host disease with ruxolitinib maintenance following allogeneic HCT
Originally Published: 05/15/2025
Key Points
Prolonged ruxolitinib administration after allogeneic HCT is associated with low rates of clinically significant chronic GVHD.
Abstract
Despite recent advances in graft-versus-host disease (GVHD) prophylaxis, novel approaches to effective prevention of chronic GVHD (cGVHD) remain of high importance. In this prospective, multicenter, phase 2 trial, ruxolitinib, an oral inhibitor of Janus kinase (JAK) 1 and 2, was administered as maintenance therapy after reduced-intensity allogeneic hematopoietic cell transplantation (HCT). GVHD prophylaxis consisted of tacrolimus and methotrexate...
Pre-transplant inflammation and its associations with acute GvHD and mortality in pediatric allogeneic hematopoietic stem cell transplantation patients
Originally Published: 05/12/2025
Abstract
In this explorative study we aimed to identify inflammatory serum proteins measured before allogeneic hematopoietic stem cell transplantation (HSCT) that are associated with acute Graft-versus-Host Disease (aGvHD) and mortality in pediatric HSCT recipients. We measured 28 inflammatory serum proteins in 384 pediatric patients (2010–2022) with malignant (30%) and non-malignant (70%) indications for allogeneic HSCT. A sample before the start of the conditioning (T1) was included, as well as a sample on the day of HSCT (T2). For patients who developed aGvHD we also included a sample at...
Measurable residual disease and posttransplantation gilteritinib maintenance for patients with FLT3-ITD–mutated AML
Originally Published: 05/08/2025
Abstract
BMT CTN (Blood and Marrow Transplant Clinical Trials Network) 1506 (“MORPHO”) was a randomized study of gilteritinib compared with placebo as maintenance therapy after hematopoietic cell transplantation (HCT) for patients with FLT3-ITD–mutated acute myeloid leukemia (AML). A key secondary end point was to determine the impact on survival of before and/or after HCT measurable residual disease (MRD), as determined using a highly sensitive assay for FLT3-ITD mutations. Generally, gilteritinib maintenance therapy was associated with improved relapse-free survival (RFS) for participants...
Donor regulatory T-cell therapy to prevent graft-versus-host disease
Originally Published: 05/02/2025
Key Points
Treg-engineered donor graft prevents acute and chronic GVHD.
Patients treated with Treg-engineered graft who develop GVHD respond to primary corticosteroid therapy at a high rate.
Abstract
Allogeneic hematopoietic cell transplantation is a curative therapy limited by graft-versus-host disease (GVHD). In preclinical studies and early-phase clinical studies, enrichment of donor regulatory T cells (Tregs) appears to prevent GVHD and promote healthy immunity. We enrolled 44 patients in an open-label, single-center, phase 2 efficacy study investigating if a precision selected and...
Eltrombopag in combination with immunosuppressive therapy in pediatric severe aplastic anemia: Phase 2 ESCALATE trial
Originally Published: 05/02/2025
Key Points
Eltrombopag combined with IST demonstrated a trend towards a favorable ORR in pediatric patients with r/r SAA.
The PK profile for eltrombopag was similar to that observed in patients with ITP, and no unexpected safety signals were observed in children
Severe aplastic anemia (SAA) is a rare, life-threatening disease with acquired pancytopenia and hypocellular bone marrow. ESCALATE evaluated eltrombopag in combination with immunosuppressive therapy (IST) in pediatric patients (aged 1 to <18 years) with relapsed or refractory (r/r) or treatment-naĂŻve SAA. The eltrombopag starting...
Taking Charge of My Care and Advocacy Following a Cancer Diagnosis
Originally Published: 04/24/2025
After a long battle with illness and seeking many specialists, I emphasize the need to self-advocate for ongoing care following my cancer diagnosis.
Almost every cancer survivor will agree that we end up going to too many doctors. A friend of mine with both cancer and diabetes has added up that he has 14 of them! I wrote an article for CURE titled “Doctors and More Doctors – A Way of Life for Cancer Survivors.” This blog described different conditions caused by cancer which force us to consult with other doctors, dentists and therapists to treat side effects caused by either the cancer or...
Prognostic Significance of Monocytic-like Phenotype in AML patients treated with Venetoclax and Azacytidine
Originally Published: 04/18/2025
Key Points
Flow cytometry enhances morphological analysis to detect AML blasts with monocytic differentiation (mono-blasts).
A high mono-blasts/CD45+ proportion cells predicts poor response and reduced survival in newly diagnosed AML patients receiving Ven-Aza
The prognostic impact of monocytic differentiation in AML patients receiving Venetoclax (Ven) and azacitidine (Aza) remains unclear. In a prospective cohort of 86 newly diagnosed AML patients treated with Ven-Aza, we used multiparametric flow cytometry (MFC) to define mono-blasts as AML blasts co-expressing ≥2 monocytic markers (CD4,...
Reduced venetoclax exposure to 7 days vs standard exposure with hypomethylating agents in newly diagnosed AML patients
Originally Published: 04/17/2025
Abstract
Hypomethylating agent (HMA) plus venetoclax (VEN) regimens are standard of care in patients with acute myeloid leukemia (AML) ineligible for intensive chemotherapy. While the VEN label recommends continuous 28-day cycles, shortened VEN durations may induce similar response rates and improve tolerability. It is unknown how a VEN exposure reduced to 7 days during cycles compares to standard HMA + VEN. We retrospectively compared newly diagnosed AML patients treated with azacitidine (AZA) x 7 days plus VEN x 7 days (“7 + 7” regimen) from the first cycle (n = 82) vs patients treated...
The impact of ABO compatibility on allogeneic hematopoietic cell transplantation outcomes: a contemporary and comprehensive study from the transplant complications working party of the EBMT
Originally Published: 04/17/2025
Abstract
The role of ABO blood group system mismatch on allogeneic hematopoietic cell transplantation (allo-HCT) outcomes is controversial since current publications of large datasets are lacking. We retrospectively analyzed 30,487 patients transplanted between 2010 and 2021 using the EBMT registry to assess ABO incompatibility’s effect on non-relapse mortality (NRM), overall survival (OS), progression-free survival (PFS), relapse incidence (RI), acute GvHD (aGvHD), chronic GvHD (cGvHD), and neutrophil engraftment. Transplantations were classified as ABO-compatible (56.3%), major (18.1%),...