Mucosal toxicity in hematological malignancies: prevention, management, and novel therapeutic insights.

Introduction: Mucosal toxicities remain a longstanding and challenging concern in the treatment of hematopoietic malignancies (HM). In addition to the classic oral (OM) and gastrointestinal mucositis (GIM) induced by chemotherapy (CHT) and/or radiotherapy (RT), novel targeted treatments and immunotherapies may cause other forms of mucosal disorders.

Areas Covered: This overview provides updated insights into the pathobiology and management strategies for mucosal toxicities induced by treatments for HMs.

Palliative care interventions and their early integration in the management of older adults with acute myeloid leukemia: a narrative review.

Background And Objective: Palliative care (PC) interventions have become essential in treating patients with acute myeloid leukemia (AML). Studies indicate that the early PC (EPC) integration during the entire disease course, from the diagnosis and the start of treatment until the end-of-life (EOL), especially regarding the decision-making process and the relief of symptom burden, can significantly impact the patient's quality of life (QoL) of AML patients and comprehensively address their physical and psychosocial needs. This narrative review synthesizes quantitative and qualitative research, including observational and randomized clinical studies, as well as review articles, to place issues of EPC within the context of AML.

Correction: Niscola et al. Acute Myeloid Leukemia in Older Patients: From New Biological Insights to Targeted Therapies. 2024, , 6632-6658.

In the original publication [...

Assessing frailty in patients with relapsed/refractory multiple myeloma: A comparison between the patient-reported frailty phenotype and the International Myeloma Working Group frailty index.

Introduction: The International Myeloma Working Group Frailty Index (IMWG FI) is one of the most used frailty assessment tools in patients with multiple myeloma (MM). A patient-centered frailty tool based on patient-reported outcomes (PROs) has been recently proposed for patients with relapsed/refractory MM (RRMM): the Patient-Reported Frailty Phenotype (PRFP). This cross-sectional analysis aimed to replicate the PRFP within a real-world setting and to describe health-related quality of life (HRQoL) profiles based on this new patient-centered frailty classification.

Oral decitabine in acute myeloid leukemia: assessing efficacy, safety, and future implications for older patients.

Introduction: Older patients with acute myeloid leukemia (AML) are often unsuitable for standard treatments and traditionally have a dismal prognosis. For 20 years, hypomethylating agents (HMAs), as single agents and recently as a backbone with venetoclax, have been used in this setting. The oral combination of decitabine and cedazuridine (C-DEC), which is therapeutically and pharmacologically equivalent to the intravenous (IV) formulation (IV-DEC), has expanded the therapeutic arsenal for AML, allowing for better convenience of administration.

Long-term Sustained Response to Lenalidomide after Clearance of Bone Marrow Blasts by Azacytidine in High-risk Myelodysplastic Syndromes with Del5q: A Case Report.

Introduction: This report discusses a rare case involving a patient with high-risk (HR) Del(5q) myelodysplastic syndrome (MDS) who achieved a long-term response to lenalidomide after having received six cycles of azacytidine. The latter treatment led to the clearance of blast cells from the bone marrow (BM).

Case Representation: As per current clinical practice, patients with HR MDS receive azacytidine continuously until the disease progresses or the occurrence of unmanageable side effects.

Menin Inhibitors: New Targeted Therapies for Specific Genetic Subtypes of Difficult-to-Treat Acute Leukemias.

Menin (MEN1) is a well-recognized powerful tumor promoter in acute leukemias (AL) with KMT2A rearrangements (KMT2Ar, also known as MLL) and mutant nucleophosmin 1 (NPM1m) acute myeloid leukemia (AML). MEN1 is essential for sustaining leukemic transformation due to its interaction with wild-type KMT2A and KMT2A fusion proteins, leading to the dysregulation of KMT2A target genes. MEN1 inhibitors (MIs), such as revumenib, ziftomenib, and other active small molecules, represent a promising new class of therapies currently under clinical development.

Translational Research on Azacitidine Post-Remission Therapy of Acute Myeloid Leukemia in Elderly Patients (QOL-ONE Trans-2).

The achievement of complete remission (CR) is crucial for acute myeloid leukemia (AML) patients undertaking curative therapy, but relapse often occurs within months, highlighting the need for strategies to prolong disease-free survival (DFS). Our phase III study compared the efficacy and safety of azacitidine (AZA) to best supportive care (BSC) in elderly AML patients who achieved CR following intensive induction and consolidation therapy. This ancillary study (QOL-ONE Trans-2) evaluated biological changes in bone marrow using Next-Generation Sequencing (NGS).

Latest Insights and Therapeutic Advances in Myelodysplastic Neoplasms.

Myelodysplastic syndromes/neoplasms (MDSs) encompass a range of hematopoietic malignancies, commonly affecting elderly individuals. Molecular alterations in the hematopoietic stem cell compartment drive disease pathogenesis. Recent advancements in genomic profiling have provided valuable insights into the biological underpinnings of MDSs and have expanded therapeutic options, particularly for specific molecularly defined subgroups.

Impact on mental health, disease management, and socioeconomic modifications in hematological patients during the COVID-19 pandemic in Italy.

Background: Hematological patients are a highly vulnerable population with an increased risk of developing severe COVID-19 symptoms due to their immunocompromised status. COVID-19 has proven to cause serious mental health issues, such as stress, anxiety, and depression in the general population. However, data on the psycho-social impact of COVID-19 on hematological patients are lacking.

Health-related Quality of Life Profile of Newly Diagnosed Patients With Myelodysplastic Syndromes by Age, Sex, and Risk Group: A Real-world Study by the GIMEMA.

Health-related quality of life (HRQoL) is an important goal of therapy for patients with myelodysplastic syndromes (MDS); however, little is known about HRQoL of these patients at clinical presentation. We report HRQoL profile of newly diagnosed patients with MDS across both the the International Prognostic Scoring System (IPSS) and IPSS-Revised (IPSS-R) classifications, stratified by sex and age group categories, aiming to also establish European Organization for Research and Treatment of Cancer Quality of Life Questionnaire-Core30 (EORTC QLQ-C30) reference values for these patients. Analysis was based on 927 patients with a median age of 73.

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.

Azacitidine Post-Remission Therapy for Elderly Patients with AML: A Randomized Phase-3 Trial (QoLESS AZA-AMLE).

This phase-3 randomized multicenter trial evaluated the efficacy of subcutaneous azacitidine (AZA) post-remission therapy vs. best supportive care (BSC) in elderly acute myeloid leukemia (AML) patients. The primary endpoint was the difference in disease-free survival (DFS) from complete remission (CR) to relapse/death.

Identification of Predictive Factors for Overall Survival and Response during Hypomethylating Treatment in Very Elderly (≥75 Years) Acute Myeloid Leukemia Patients: A Multicenter Real-Life Experience.

Elderly patients represent the most challenging and hard-to-treat patient population due to dismal characteristics of the disease, such as secondary-acute myeloid leukemia (AML), enrichment of unfavorable molecular genes () and comorbidities. We conducted a multicentric retrospective study to evaluate activity and safety in a real-life setting of hypomethylating drugs (HMAs) in patients older than 75 years with AML. Between September 2010 and December 2021, 220 patients were treated, 164 (74.

Ascorbate Plus Buformin in AML: A Metabolic Targeted Treatment.

In the present study, we characterized the metabolic background of different Acute Myeloid Leukemias' (AMLs) cells and described a heterogeneous and highly flexible energetic metabolism. Using the Seahorse XF Agilent, we compared the metabolism of normal hematopoietic progenitors with that of primary AML blasts and five different AML cell lines. We assessed the efficacy and mechanism of action of the association of high doses of ascorbate, a powerful oxidant, with the metabolic inhibitor buformin, which inhibits mitochondrial complex I and completely shuts down mitochondrial contributions in ATP production.