Real-world efficacy and safety of fostamatinib in ITP patients: Italian multicentre experience. GIMEMA ITP1122 study.

The efficacy and safety of fostamatinib in adult patients with persistent/chronic immune thrombocytopenia (p/c ITP) were demonstrated in the FIT-1/FIT-2 trials. This retrospective, multicentre observational study evaluated real-world outcomes in consecutive p/c ITP patients treated with fostamatinib in Italy. The primary end-point, serving as a surrogate for both efficacy and safety, was the proportion of patients receiving fostamatinib for at least 6 months.

Innovative Use of Fostamatinib in Managing Refractory Immune Thrombocytopenia in an HIV-Positive Patient: A Case Report.

Managing immune thrombocytopenia (ITP) in HIV patients remains challenging due to refractory cases and treatment limitations. This report highlights fostamatinib as a safe, effective, and antiretroviral therapy-compatible option, offering durable platelet stabilization in a difficult-to-treat population.

Chronic myeloid leukemia outcomes according to baseline risk and first-line treatment in real-world settings: Data from the Italian Network/CML Campus.

Background: Improved outcome has been reported in chronic myeloid leukemia (CML) patients treated with tyrosine kinase inhibitors (TKIs) in sponsored trials.

Methods: This is a multicenter prospective cohort study of consecutive patients with newly diagnosed chronic phase CML from 19 regions in Italy. Baseline treatments and prognostic factors on time to first optimal molecular response (≥ molecular response 3, MR3), time to disease progression, time to death from CML, and overall survival (OS) were analyzed using multivariable Fine and Gray models.

A critical review of conventional and emerging technologies for the detection of contaminants, allergens and adulterants in plant-based milk alternatives.

The increasing popularity of plant-based milk alternatives (PBMAs) necessitates effective safety and authentication measures to ensure food product integrity and maintain consumer trust. This review aims to offer a comprehensive overview of potential contaminants, allergens, and adulterants in PBMAs, and the analytical methodologies employed for their detection and quantitation. It details the advantages and limitations of widely employed testing techniques, such as chromatography, spectroscopy, immunoassays and PCR.

Insights into Alternaria in apple fruit causing mouldy core, external infection and mycotoxin production under retail and storage conditions.

Apple fruit is widely consumed worldwide, but fungal contamination in the postharvest stage presents a significant food safety concern. This study evaluates the production and accumulation of Alternaria mycotoxins, including alternariol (AOH), alternariol monomethyl-ether (AME), and the modified forms (AOH-3-S, AME-3-S, AOH-3-G, AME-3-G), altenuene (ALT), tenuazonic acid (TeA), tentoxin (TEN), altertoxin I and II (ATXI, ATX-II), in Red Delicious apples under simulated retail and post-harvest conditions. Three Alternaria tenuissima strains (isolates 02, 31 and 36) were inoculated in apple fruit at two sites separately (core and exterior) and incubated at two temperatures (25 °C and 4 °C) for 1 and 9 months.

Real-World Efficacy Profile of Compassionate Use of Asciminib in an Italian, Multi-Resistant Chronic-Phase Chronic Myeloid Leukemia (CML-CP) Patient Population.

Chronic myeloid leukemia (CML) patients who have experienced failure and/or intolerance to multiple lines of treatment have limited therapeutic possibilities. Asciminib is a first-in-class tyrosine kinase inhibitor (TKI) that inhibits the ABL Myristoyl Pocket (STAMP or Specifically Targeting the ABL Myristoyl Pocket) within the BCR::ABL1 oncoprotein. This retrospective Italian analysis reports the efficacy and safety outcomes of asciminib in treating 77 CML patients in chronic phase (CML-CP) within a compassionate use setting.

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

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 remission (CR) rates in myeloid leukemia. SELECT-MDS-1 was a phase 3 study comparing the activity of tamibarotene + AZA to placebo + AZA in these pts with newly diagnosed HR-MDS with RARA overexpression.

Therapeutic Vaccinations with p210 Peptides in Imatinib-Treated Chronic Myeloid Leukemia Patients: 10 Years Follow-Up of GIMEMA CML0206 and SI0207 Studies.

We previously showed that peptides encompassing the unique b3a2 or b2a2 breakpoint amino-acid sequence of oncogenic p210 induced peptide-specific T-cell responses in chronic myeloid leukemia (CML) patients. : From 2007 to 2011, two multicenter peptide vaccine phase II studies, GIMEMA CML0206 and SI0207, enrolling overall 109 CML patients (68 b3a2 and 41 b2a2) with persistence of molecular disease during imatinib treatment, were carried out. Peptide vaccination schedule included the following: "immunization phase" (six vaccinations every 2 weeks); "reinforcement" phase (three monthly boosts) and "maintenance" phase (two boosts at 3-month intervals).

Comparative Analysis of the Performance of Automated Digital Cell Morphology Analyzers for Leukocyte Differentiation in Hematologic Malignancies: Mindray MC-80 Versus West Medical Vision Hema.

Introduction: The use of artificial intelligence in hematology laboratories has improved the diagnostic evaluation of peripheral blood cells. The aim of this study is to compare the performance of two automated digital cell morphology analyzers, the Mindray MC-80 and the West Medical Vision Hema Pro, with manual microscopy, the gold standard, for leukocyte differentiation in patients with hematologic malignancies and infections.

Methods: Peripheral blood smears from 75 patients were analyzed, including cases of acute lymphoblastic leukemia (ALL, 4), chronic lymphocytic leukemia (CLL, 20), acute myeloid leukemia (AML, 20), chronic myeloid leukemia (CML, 5), other lymphoproliferative disorders (LPD, 20), and infections (6).

Pelabresib plus ruxolitinib for JAK inhibitor-naive myelofibrosis: a randomized phase 3 trial.

Janus kinase (JAK) inhibitors provide limited depth and durability of response in myelofibrosis. We evaluated pelabresib-a bromodomain and extraterminal domain (BET) inhibitor-plus ruxolitinib (a JAK inhibitor) compared with placebo plus ruxolitinib as first-line therapy. In this phase 3 study (MANIFEST-2), JAK inhibitor-naive patients with myelofibrosis were randomized 1:1 to pelabresib 125 mg once daily (QD; 50-175 mg QD permitted) for 14 days followed by a 7-day break (21-day cycle), or to placebo in combination with ruxolitinib 10 or 15 mg twice daily (BID; 5 mg QD-25 mg BID permitted).

Real-world evidence of pegcetacoplan in patients with paroxysmal nocturnal haemoglobinuria: A nationwide Italian study.

In this study, we collected real-world evidence on the use of pegcetacoplan among 22 Italian patients with paroxysmal nocturnal haemoglobinuria showing suboptimal response to anti-C5 treatments eculizumab and ravulizumab. Most patients exhibited a complete or good response as per the criteria of the European Bone Marrow Transplant group (Risitano et al. Front Immunol 2019) and median haemoglobin improvement from baseline was +3.

Clonal hematopoiesis in patients with autoimmune thrombocytopenia: an international multicenter study.

Diagnostic boundaries between immune thrombocytopenia (ITP) and other thrombocytopenic states, such as thrombocytopenic myelodysplastic syndromes, may be difficult to establish, and the detection of somatic mutations by next-generation sequencing (NGS) may be of aid. Here, we aimed at characterizing the prevalence and clinical significance of clonal hematopoiesis in ITP. In this multicentric retrospective observational study, we enrolled 167 adult patients with ITP, followed at 13 centers in Italy, United Kingdom, and the United States.

A Rare Case of Life-Threatening Jaundice Caused by Epstein-Barr Virus Infection and Secondary Cold Agglutinin Syndrome Successfully Treated with Rituximab.

Background: Jaundice and hyperbilirubinemia are common clinical problems characterized by the presence of bile pigments in the blood and their deposition in body tissues. This clinical condition can be associated with a broad spectrum of potential benign and malignant causes, including hepatic inflammation, biliary obstruction, impaired bilirubin conjugation and bilirubin overproduction Therefore, the hyperbilirubinemia diagnostic work-up sometimes can be highly challenging and its therapeutic management can require a multidisciplinary approach.

Case Report: We report on a unique case of life-threatening jaundice and hepatic failure in a 20-year-old female who presented to the emergency room with complaints of fever, constant left abdominal pain and generalized profuse fatigue.

Diagnosis and management of Evans syndrome in adults: first consensus recommendations.

Evans syndrome is a rare disease marked by a severe clinical course, high relapse rate, infectious and thrombotic complications, and sometimes fatal outcome. Management is highly heterogeneous. There are several case reports but few large retrospective studies and no prospective or randomised trials.

Ropeginterferon versus Standard Therapy for Low-Risk Patients with Polycythemia Vera.

BACKGROUND: Whether phlebotomy alone can adequately maintain target hematocrit in patients with low-risk polycythemia vera (PV) remains elusive. METHODS: In a phase 2 open-label randomized trial, we compared ropeginterferon alfa-2b (ropeg; 100 μg every 2 weeks) with phlebotomy only regarding maintenance of a median hematocrit level (≤45%) over 12 months in the absence of progressive disease (primary end point). In follow-up, crossover to the alternative treatment group was allowed if the primary end point was not met.

Ropeginterferon phase 2 randomized study in low-risk polycythemia vera: 5-year drug survival and efficacy outcomes.

In patients with low-risk polycythemia vera, exposure to low-dose Ropeginterferon alfa-2b (Ropeg) 100 µg every 2 weeks for 2 years was more effective than the standard treatment of therapeutic phlebotomy in maintaining target hematocrit (HCT) (< 45%) with a reduction in the need for phlebotomy without disease progression. In the present paper, we analyzed drug survival, defined as a surrogate measure of the efficacy, safety, adherence, and tolerability of Ropeg in patients followed up to 5 years. During the first 2 years, Ropeg and phlebotomy-only (Phl-O) were discontinued in 33% and 70% of patients, respectively, for lack of response (12 in the Ropeg arm vs.

Patient-reported symptom monitoring and adherence to therapy in patients with newly diagnosed chronic myeloid leukemia.

Background: The authors assessed the clinical utility of patient-reported symptom monitoring in the setting of newly diagnosed chronic myeloid leukemia (CML). The primary objective was to evaluate adherence to therapy.

Methods: The authors conducted an international prospective study that included patients with newly diagnosed, chronic-phase CML.

Venetoclax with Hypomethylating Agents in Newly Diagnosed Acute Myeloid Leukemia: A Systematic Review and Meta-Analysis of Survival Data from Real-World Studies.

In recent years, the association of venetoclax (VEN) with hypomethylating agents (HMAs) significantly improved the outcome of patients with newly diagnosed acute myeloid leukemia (AML) who were unfit for intensive chemotherapy and became the standard of care after the publication of the pivotal RCT VIALE-A. However, it is still not clear to what extent the results observed in the VIALE-A apply to a real-world setting. For this reason, we carried out a systematic review and meta-analysis of real-world studies on newly diagnosed patients with AML, ineligible for intensive induction chemotherapy, receiving first-line VEN+HMA.

High Expression Negatively Correlates with Expression and Predicts Better Prognosis in Diffuse Large B-Cell Lymphoma: Role of Autophagy.

Diffuse large B-cell lymphoma (DLBCL) is characterized by high molecular and clinical heterogeneity. Autophagy, a lysosome-driven catabolic process devoted to macromolecular turnover, is fundamental in maintaining normal hematopoietic stem cells and progenitors homeostasis, and its dysregulation plays a critical role in the initiation and progression of hematological malignancies. One main regulator of autophagy is BECLIN-1, which may interact alternatively with either BCL-2, thus allowing apoptosis, or PI3KC3, thus promoting autophagy.

Eltrombopag for Low-Risk Myelodysplastic Syndromes With Thrombocytopenia: Interim Results of a Phase II, Randomized, Placebo-Controlled Clinical Trial (EQOL-MDS).

Purpose: In myelodysplastic syndromes (MDS), severe thrombocytopenia is associated with poor prognosis. This multicenter trial presents the second-part long-term efficacy and safety results of eltrombopag in patients with low-risk MDS and severe thrombocytopenia.

Methods: In this single-blind, randomized, placebo-controlled, phase-II trial of adult patients with International Prognostic Scoring System low- or intermediate-1-risk MDS, patients with a stable platelet (PLT) count (<30 × 10/mm) received eltrombopag or placebo until disease progression.