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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.

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...

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-naïve 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%),...

Natural killer cells’ functional impairment drives the immune escape of pre-malignant clones in early-stage myelodysplastic syndromes

Originally Published: 04/11/2025
Abstract Dissecting the preneoplastic disease states’ biological mechanisms that precede tumorigenesis can lead to interventions that can slow down disease progression and/or mitigate disease-related comorbidities. Myelodysplastic syndromes (MDS) cannot be cured by currently available pharmacological therapies, which fail to eradicate aberrant hematopoietic stem cells (HSCs), most of which are mutated by the time of diagnosis. Here, we sought to elucidate how MDS HSCs evade immune surveillance and expand in patients with clonal cytopenias of undetermined significance (CCUS), the pre-...

Cell-autonomous dysregulation of interferon signaling drives clonal expansion of SRSF2-mutant MDS stem/progenitor cells

Originally Published: 03/31/2025
Key Points SRSF2 mutations blunt responsiveness of MDS cells to IFN stimulation and promote their clonal fitness by downregulating STAT1 expression Proteasome inhibition restores STAT1 protein level and sensitivity of SRSF2-mutant MDS cells to IFN, suggesting a new therapeutic strategy Myelodysplastic syndromes (MDS) are myeloid malignancies often driven by mutations in genes encoding splicing factors (SFs). How these mutations drive the clonal expansion of MDS stem/progenitor cells to outcompete normal hematopoietic stem/progenitor cells (HSPCs) remains unexplained. Although a role for...

TP53-Mutated Acute Myeloid Leukemia and Blast Phase Myeloproliferative Neoplasm: Distinct Mutation Leads to Poorer Prognosis

Originally Published: 03/28/2025
ABSTRACT Patients with TP53 mutations in acute myeloid leukemia (AML) and blast phase myeloproliferative neoplasms (MPN-BP) experience similar poor clinical outcomes. A retrospective analysis of 39 patients with TP53 mutations (23 with AML and 16 with MPN-BP) revealed comparable mutation patterns associated with prognostic significance. A total of 47 distinct TP53 mutations were identified, including seven patients with multiple mutations. Based on clinical outcomes, we propose a two-tiered risk stratification for TP53-mutated AML and MPN-BP. The high-risk group mutations, such as splice...