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.

Finding His Way Through: Evan’s Story of PNH

Original Publication Date

September 14, 2025

Article Source

External Web Content

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…

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

Original Publication Date

September 4, 2025

Article Source

External Web Content

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…

Germline and somatic genetic landscape of pediatric myelodysplastic syndromes

Original Publication Date

September 1, 2025

Article Source

External Web Content

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…

A clinical guide to TP53 mutations in myeloid neoplasms

Original Publication Date

August 29, 2025

Article Source

External Web Content

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…

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

Original Publication Date

August 28, 2025

Article Source

External Web Content

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…

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

Original Publication Date

August 28, 2025

Article Source

External Web Content

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…

Factors affecting treatment decisions for lower-risk myelodysplastic syndromes across practice settings

Original Publication Date

August 25, 2025

Article Source

External Web Content

Please find all content at the link.

Pivotal results of SELECT-MDS-1 phase 3 study of tamibarotene with azacitidine in newly diagnosed higher-risk MDS

Original Publication Date

August 13, 2025

Article Source

External Web Content

Abstract Higher-risk myelodysplastic syndrome (HR-MDS) with RARA gene overexpression is a subset of patients (pts) with an actionable target for tamibarotene, an oral and a selective retinoic acid receptor-α (RAR-α) agonist. Tamibarotene with azacitidine (AZA) showed complete…

Impaired cytotoxic function and exhausted phenotype of natural killer cells in VEXAS syndrome

Original Publication Date

July 30, 2025

Article Source

External Web Content

Key Points NK cells phenotyping reveals impaired cytotoxicity, chronic activation and exhaustion in VEXAS syndrome. Decreased circulating NK cells were independently associated with an increased risk of severe infections in VEXAS syndrome. VEXAS (vacuoles, E1 enzyme, X-linked…

IHC Testing Could Serve as Surrogate to NGS for Early Detection of TP53-Mutant MDS/AML

Original Publication Date

July 29, 2025

Article Source

External Web Content

Immunohistochemistry (IHC) could represent a biomarker for the early detection of TP53 mutations and for the prediction of a TP53 allelic state in patients with myelodysplastic neoplasms (MDS) and acute myeloid leukemia (AML), according to data from an observational trial…

NFL Player Teams Up with AAMDSIF

News and Treatment Updates

Finding His Way Through: Evan’s Story of PNH

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

Germline and somatic genetic landscape of pediatric myelodysplastic syndromes

A clinical guide to TP53 mutations in myeloid neoplasms

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

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

Factors affecting treatment decisions for lower-risk myelodysplastic syndromes across practice settings

Pivotal results of SELECT-MDS-1 phase 3 study of tamibarotene with azacitidine in newly diagnosed higher-risk MDS

Impaired cytotoxic function and exhausted phenotype of natural killer cells in VEXAS syndrome

IHC Testing Could Serve as Surrogate to NGS for Early Detection of TP53-Mutant MDS/AML

We Recommend

For Patients and Families

Website Feedback

We Recommend

For Patients and Families