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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.

Cyclophosphamide and Cyclosporin for GVHD Prevention

Originally Published: 10/01/2025
Curtis et al. (July 17 issue)1 report evidence of longer graft-versus-host disease (GVHD)–free, relapse-free survival with post-transplantation cyclophosphamide–based prophylaxis than with cyclosporin–methotrexate among patients receiving a stem-cell transplant from a matched related donor after myeloablative conditioning or reduced-intensity conditioning. We recently published real-world data with respect to post-transplantation cyclophosphamide–based prophylaxis in patients receiving a transplant from a matched sibling donor and similarly observed longer GVHD-free, relapse-free survival...

Azacitidine as therapy for VEXAS syndrome

Originally Published: 09/18/2025
How should patients with VEXAS (an acronym for vacuoles [in myeloid and erythroid precursors], E1 enzyme, X-linked, autoinflammatory, somatic) syndrome be treated? Until now, we have only had case reports and small series to guide us. In this issue of Blood, Jachiet and colleagues from the French VEXAS group FRENVEX retrospectively describe their experience with azacitidine therapy in 88 patients with genetically confirmed VEXAS syndrome.1 Improvements in inflammatory signs and symptoms, blood counts, or both disease markers were observed in more than 60% of patients, and the median duration...

Luspatercept for Patients with Lower-Risk Myelodysplastic Syndromes/Neoplasms: A Systematic Review and Meta-Analysis

Originally Published: 09/18/2025
Luspatercept has emerged as a novel therapy for anemia in transfusion-dependent (TD) lower-risk myelodysplastic syndromes (LR-MDS). This systematic review and meta-analysis aims to evaluate the efficacy and safety of luspatercept in LR-MDS. We conducted a systematic review and meta-analysis to evaluate the efficacy and safety of luspatercept in TD LR-MDS. Six databases were searched through March 2025 to find relevant material. Studies were screened and extracted by two independent authors. A total of 20 studies encompassing 3,455 patients were included in the analysis. The pooled 8-week...

A 30-gene classifier distinguishes low-risk MDS HSPCs from healthy HSPCs

Originally Published: 09/17/2025
Highlights •     Single-cell RNA-sequencing performed on 16 lower-risk myelodysplastic syndrome (LR-MDS) bone marrow-derived mononuclear cells (BMMCs) •     A 30-gene signature distinguishes LR-MDS hematopoietic stem and progenitor cells (HSPCs) from Healthy HSPCs •     Vesicular trafficking-related gene modules are enriched in MDS •     Certain trafficking genes such as LRBA are expressed exclusively in HSPCs Myelodysplastic syndromes (MDSs) are a group of malignant clonal disorders that are characterized by functional impairment of hematopoiesis, morphologic dysplasia, and genetic...

Finding His Way Through: Evan’s Story of PNH

Originally Published: 09/14/2025
Meet Evan. In a recent conversation with the Aplastic Anemia and MDS International Foundation, he shared his journey from paroxysmal nocturnal hemoglobinuria (PNH) diagnosis through treatment and survival. Evan, please tell us a little about yourself. I’m 31 years old. I’m based in New York City, and I’ve been living here for around eight years, after graduating from college. I’m working, enjoying life, spending time with friends, and just moving along. What was your life like before the diagnosis of PNH? I was biking a lot and extremely active. I had career goals, friends, and was planning...

Sensitivity of Pediatric Myelodysplastic Syndromes With Excess of Blasts With UBTF–TD to Venetoclax/Azacitidine

Originally Published: 09/04/2025
UBTF-TD has been reported in a significant percentage of childhood MDS-EB and has been associated with inferior survival compared to that of patients with the wild-type gene. We treated three consecutive pediatric patients affected by UBTF-TD MDS-EB with venetoclax and azacitidine (ven/aza) in combination as 28-day cycles on a compassionate use basis three consecutive pediatric patients affected by UBTF-TD MDS-EB as a bridge to allogeneic HSCT. Treatment with ven/aza was well-tolerated, and all patients responded to the ven/aza course, achieving CR with flow-cytometry negativity. All three...

Germline and somatic genetic landscape of pediatric myelodysplastic syndromes

Originally Published: 09/01/2025
Abstract Pediatric myelodysplastic syndromes (MDS) represent a rare group of clonal hematopoietic stem cell disorders accounting for approximately 5% of pediatric hematologic malignancies. They are characterized by ineffective hematopoiesis, cytopenia, and dysplastic changes in the bone marrow with variable risk of progression to acute myeloid leukemia. Unlike adult MDS, pediatric cases predominantly present with hypocellular bone marrow, with monosomy 7 and trisomy 8 as the most common cytogenetic aberrations. Pediatric MDS can manifest as primary disease or arise secondary to classical...

A clinical guide to TP53 mutations in myeloid neoplasms

Originally Published: 08/29/2025
TP53 mutations are found in 10-15% of myeloid neoplasms and are one of its most important prognostic factors. Emerging data show that TP53 mutational allele status is a key determinant of clinical outcomes, with multi-hit TP53 mutant myeloid neoplasms having a very poor prognosis. Significant differences exist among the methods used in clinical and research settings to assess TP53 mutational status, leading to variability in reported patient characteristics, response to therapy, and survival. Indeed, differences in the criteria used to define TP53 mutational states among professional...

Correlates and consequences of clonal hematopoiesis expansion rate: a 16-year longitudinal study of 6976 women

Originally Published: 08/28/2025
Abstract Clonal hematopoiesis of indeterminate potential (CHIP) is associated with increased mortality and malignancy risk, yet the determinants of clonal expansion remain poorly understood. We performed sequencing at a depth of coverage of >4000× for CHIP mutations in 6976 postmenopausal women from the Women’s Health Initiative (WHI) at 2 time points: the WHI baseline examination and ∼16 years later at the Long Life Study (LLS) visit. Among 3685 CH mutations detected at baseline (variant allele fraction [VAF] of ≥0.5%), 24% were not detected at LLS, 26% were micro-CH at LLS (0.5% ≤ VAF...

Outcomes and Patterns of Relapse of NPM1 Mutated Acute Myeloid Leukemia Treated With Venetoclax Based Therapies

Originally Published: 08/28/2025
To the editor, Mutations in nucleophosmin 1 (NPM1-mut) are detected in approximately 30% of newly diagnosed (ND) acute myeloid leukemia (AML), and are considered founder events due to their persistence at relapse [1]. While treatment with both intensive chemotherapy (IC) and low-intensity therapy (LIT) is associated with favorable responses in NPM1-mutated AML, relapses are common, occurring in approximately 50% of IC-treated and more frequently with LIT-treated patients [1, 2]. Recent data suggests that NPM1 wildtype (NPM1-wt) relapses occur in up to 5%–10% of NPM1-mut AML treated with...