Conditioning intensity in myelodysplastic patients aged ≥ 50 years undergoing allogeneic hematopoietic cell transplantation (allo-HCT): a study on behalf of the chronic malignancies working party of the EBMT.

Reduced intensity conditioning (RIC) is usually used for patients with myelodysplastic syndrome (MDS) undergoing allogeneic hematopoietic cell transplantation (allo-HCT), particularly in the elderly or those with comorbidities. The impact of conditioning intensity on patients' outcome remains controversial with clinicians' subjective opinion/ experience remaining a major guide in choosing the intensity. Here, we compare RIC versus MAC in a large EBMT retrospective study in MDS patients aged ≥50 years undergoing allo-HCT between 2014 and 2018.

American College of Rheumatology Guidance Statement for Diagnosis and Management of VEXAS Developed by the International VEXAS Working Group Expert Panel.

Objective: Vacuoles E1 enzyme X-linked autoinflammatory somatic syndrome (VEXAS) is a recently identified rare genetic disorder associated with somatic mutations in the UBA1 gene. VEXAS presents with a combination of inflammatory and hematologic manifestations, leading to increased morbidity and mortality.

Methods: Given the variability in disease presentation and the limited number of studies to date, no clinical documents currently exist to provide guidance to health care providers about the management of VEXAS.

A cross-sectional survey on VEXAS syndrome: insights from a global expert panel.

Background: Vacuolization, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome is recently described, for which the diagnosis and management lack official guidelines.

Aims: To assess the diagnostic capabilities and disease management of VEXAS syndrome among physicians in the global context.

Methods: An electronic survey was sent to clinicians with expertise in VEXAS syndrome between January and February 2025 to gather real-life data on the management of VEXAS.

A precision medicine approach to the myelodysplastic syndrome with isolated deletion 5q 50 years after its discovery.

In 1974, Vanden Berghe et al described a distinct hematologic disorder associated with acquired, interstitial deletion of part of the long arm of chromosome 5. This condition is now classified as myelodysplastic syndrome (MDS) with isolated deletion 5q, or MDS-del(5q). The common deletion region 5q32-5q33 contains several genes and microRNAs whose expression levels are reduced in hematopoietic cells, consistent with the loss of one allele.

Exploring patient-reported outcomes and morbidity burden of patients with VEXAS syndrome: a scoping review.

Background: VEXAS (vacuoles, E1 enzyme, X-linked, auto-inflammatory, somatic) is a recently discovered syndrome of autoinflammatory origin affecting mainly older patients with a clinical picture encompassing a variety of hematological and rheumatological conditions. We aimed to gather current knowledge on patient-reported outcomes (PRO) and morbidity burden of VEXAS patients.

Methods: A scoping review conducted via PubMed (last updated September 2024) identified studies involving VEXAS patients, excluding studies primarily addressing hematologic adverse events or laboratory parameters, with no lower date restriction.

Impact of Time to Treatment Initiation on Quality of Response in Patients with Acute Myeloid Leukemia Receiving Intensive Chemotherapy.

Background: Acute myeloid leukemia (AML) necessitates timely treatment, yet the impact of prolonged time to treatment (TTT) on clinical outcomes remains debated, especially its impact on achieving measurable residual disease (MRD) negativity, a powerful prognostic indicator in AML.

Methods: This retrospective study analyzed 196 adult AML patients treated with intensive chemotherapy, evaluating the effect of TTT on outcome measures and quality of response. TTT was categorized arbitrarily into <8, 8-14, and >14 days.

Infections in VEXAS syndrome: a systematic review of the literature.

Vacuolation, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) syndrome is a multisystem disease for which chronic immunosuppression is needed. Opportunistic infections are common; however, a clear prophylaxis regimen is not defined. A systematic review of the literature was undertaken.

Anti-thymocyte globulin in immune-mediated cytopenias: A monocentric experience and literature review.

Anti-thymocyte globulin in multi-refractory immune-mediated cytopenia is an effective treatment, with response rate as high as 65%. Younger patients, presenting with thrombocytopenia or neutropenia, and those with LGL and STAT3 mutations appear to be more likely responsive.

Inborn errors of immunity underlie clonal T cell expansions in large granular lymphocyte leukemia.

BACKGROUNDT cell large granular lymphocyte leukemia (T-LGLL) is a lymphoproliferative disorder of cytotoxic T lymphocytes (CTLs), often with gain-of-function STAT3 mutations. T-LGLL represents a unique model for the study of persistent CTL expansions. Albeit autoimmunity is implied, various paradoxical observations led us to investigate whether immunodeficiency traits underpin T-LGLL.

The role of morphology in diagnosing MDS with Non-clonal cytopenias.

Myelodysplastic syndromes/neoplasms (MDS) are a heterogeneous group of blood disorders characterized by distinctive biological and clinical features. Diagnosis typically relies on bone marrow examination and cytogenetic/molecular analysis. However, a small subset of patients lacks detectable cytogenetic or genetic abnormalities, making diagnosis challenging and reliant solely on marrow dysplasia and cytopenia.

Comprehensive Age-Stratified Impact of Mutation in Acute Myeloid Leukemia: A Real-World Experience.

While -mutated AML in the absence of -ITD generally carries a favorable prognosis, large registry studies suggest the positive prognostic benefit may not extend to patients > 65 years of age. We examined this preferential, age-dependent prognostic impact through a real-world analysis of 2811 adult AML patients. The median overall survival (OS) was significantly better in compared to patients [20.

Efficacy and safety of conventional disease-modifying antirheumatic drugs in VEXAS syndrome: real-world data from the international AIDA network.

Background: VEXAS (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) syndrome is an adult-onset autoinflammatory condition resulting in severe, often treatment-refractory inflammation. Currently, there are no established treatment guidelines for VEXAS syndrome.

Objectives: To assess the efficacy and safety of conventional disease-modifying antirheumatic drugs (cDMARDs) in a cohort of VEXAS patients.

Role of allo-HCT in "nonclassical" MPNs and MDS/MPNs: recommendations from the PH&G Committee and the CMWP of the EBMT.

"Nonclassical" myeloproliferative neoplasms (MPNs) and myelodysplastic/myeloproliferative neoplasms (MDS/MPNs) represent a heterogeneous group of malignancies characterized by a wide range of clinical manifestations. Unlike classical MPNs, there is no standardized management approach for these conditions, particularly concerning the indications for and management of allogeneic hematopoietic cell transplantation. To address this gap, the European Society for Blood and Marrow Transplantation (EBMT) Practice Harmonization and Guidelines (PH&G) Committee and the Chronic Malignancies Working Party (CMWP) have collaborated to develop shared guidelines aimed at optimizing the selection and management of patients with these rare forms of neoplasms.

Leukemia Cutis in a Patient with Acute Myeloid Leukemia Undergoing Azacitidine-Venetoclax: Case Presentation and Review of the Literature.

Leukemia Cutis (LC) in acute myeloid leukemia (AML) is typically managed within the context of systemic AML therapy, namely intensive chemotherapy (IC). In frail patients, though, viable options are hypomethylating agents (HMAs) associated with Venetoclax (VEN), but data on the efficacy of this approach in this specific setting is scarce. Here, we report our experience and provide a short review of the previous cases of LC treated with HMAs plus VEN to underline the efficacy of such treatment in LC patients who are unsuitable for IC.

Efficacy and safety profile of biotechnological agents and Janus kinase inhibitors in VEXAS syndrome: data from the international AIDA Network VEXAS registry.

Background: VEXAS syndrome, a recently identified systemic autoinflammatory disorder, poses new diagnostic and management challenges. Based on experience with other autoinflammatory diseases, anti-interleukin (IL)-1, anti-IL-6, anti-tumor necrosis factor (TNF) biotechnological agents, and Janus kinase inhibitors (JAKis) have been widely employed in VEXAS patients. The aim of this study is to evaluate the global effectiveness and safety of biotechnological agents and JAKis using data from the real-world context.

Use of Primary Prophylaxis with G-CSF in Acute Myeloid Leukemia Patients Undergoing Intensive Chemotherapy Does Not Affect Quality of Response.

: The objective of our study was to evaluate the safety and efficacy of granulocyte colony-stimulating factor (G-CSF) as primary prophylaxis in adult patients with acute myeloid leukemia (AML) undergoing intensive chemotherapy. : We retrospectively analyzed 112 AML patients treated with intensive chemotherapy at Fondazione Policlinico Tor Vergata in Rome between January 2014 and March 2024. Patients were divided into G-CSF and non-G-CSF (nG-CSF) groups.

Clinical-genomic profiling of MDS to inform allo-HCT: recommendations from an international panel on behalf of the EBMT.

For patients with myelodysplastic neoplasm/syndrome (MDS), allogeneic hematopoietic cell transplantation (allo-HCT) represents the only potentially curative treatment, capable of eradicating disease-related mutant hematopoietic cells and establishing normal donor hematopoiesis. Biologic-assignment clinical trials have indicated that in eligible patients, allo-HCT is associated with superior clinical outcomes compared with nontransplant therapy. However, this therapeutic option is only available to a subset of patients, and the outcome is influenced by multiple factors inherent to the patient, the MDS subtype, and the allo-HCT procedure itself.

Elderly Patients With Aplastic Anemia: Treatment Patterns and Outcomes in the Real World.

We retrospectively analyzed a large international cohort of 1113 patients with aplastic anemia to evaluate treatment choice and outcome in elderly patients as compared with a younger population. Overall, 319 (29%) patients were > 60 years old at diagnosis (60-64 years (n = 85), 106 65-69 years (n = 106), and 128 > 70 years (n = 128)). Elderly patients showed a more severe thrombocytopenia at onset and a significantly lower overall response (complete plus partial) to first-line therapy at 6 months as compared to younger patients (47% vs.

A fatal case of peritonitis due to colonic localization of acute myeloid leukemia.

Myeloid sarcoma (MS) is an extramedullary localization of immature granulocyte cells that can occur in association with acute myeloid leukemia (AML). Gastrointestinal involvement is relatively common in MS, but exclusive colonic localization is a rare occurrence. Here, we report on a 53-year-old male patient affected by AML developing a severe abdominal pain caused by intestinal perforation requiring surgical intervention.