Publications by authors named "PANE F"

Splenomegaly is a quite common clinical feature of Philadelphia (Ph) negative chronic myeloproliferative neoplasms (MPNs) and its presence may, in some cases, drives treatment decision. Most importantly, palpable splenomegaly is a minor criterion for both pre-fibrotic/early primary myelofibrosis and primary myelofibrosis (PMF) diagnosis, even if clinical assessment by physical examination is poorly reliable and accurate. On the other hand, despite the International Working Group-Myeloproliferative Neoplasms Research and Treatment and European LeukemiaNet guidelines defined spleen response criteria by palpation, they also recognized the highly subjective nature of spleen size assessment by physical examination, and recommended objective confirmation of volume reduction via computed tomography or magnetic resonance imaging (MRI).

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Calreticulin (CALR) mutations are detected in around 20% of patients with primary and post-essential thrombocythemia myelofibrosis (MF). Regardless of driver mutations, patients with splenomegaly and symptoms are generally treated with JAK2-inhibitors, most commonly ruxolitinib. Recently, new therapies specifically targeting the CALR mutant clone have entered clinical investigation.

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In elderly patients with high-risk classic Hodgkin lymphoma (c-HL), we evaluated the impact of a new modality treatment without bleomycin, that is, liposomal doxorubicin (NPLD)-based regimen plus consolidation radiotherapy of residual nodal masses (RNMs), on overall survival (OS) and progression free survival (PFS). In this retrospective study (2013-2023) conducted in tertiary hospitals in the bay of Naples (Italy), 50 older adults (median age, 69 years; range, 60-89) with advanced stage c-HL received frontline treatment with MVD ± irradiation. MVD consisted of 25 mg/m of NPLD along with standard Vinblastine and Dacarbazine for a total of 6 cycles (twelve iv administrations, every 2 weeks) followed by radiation of RNMs with size ≥ 2.

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Vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic (VEXAS) is a haemato-inflammatory syndrome genetically defined by somatic mutations in the X-linked UBA1 gene, typically Val/Thr/Leu substitutions at the Met41 hotspot. Clinical manifestations are heterogeneous and refractory to most haemato-rheumatological treatments. To date, no guidelines exist for the management of VEXAS, and scarce is the evidence on methodology and clinical significance of longitudinal UBA1 clonal burden evaluation upon therapy.

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  • The study found that 64% of the 25 patients had either a partial or complete increase in hemoglobin levels, with some experiencing improvements as early as week 1.
  • Although most patients reported some side effects (like diarrhea and fever), parsaclisib was generally well tolerated and showed sustained benefits throughout the study.
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Isatuximab, a novel anti-CD38 monoclonal antibody, is approved in combination with carfilzomib and dexamethasone (Isa-Kd) in relapsed/refractory multiple myeloma (RRMM) patients. Because of its recent introduction, real-world efficacy and safety are poorly reported. In this Italian multicenter real-life observational retrospective study, efficacy and safety of the Isa-Kd regimen were evaluated in a cohort of 103 RRMM patients.

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  • * A study of 3,494 Italian patients showed that a significant portion (66.9%) started treatment at a reduced dose, often due to older age and more severe disease symptoms.
  • * Patients starting on a full dose had a better overall survival rate (78.2 months) compared to those on a reduced dose (52.6 months), highlighting the impact of initial dosing on treatment outcomes.
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Introduction: Neoadjuvant therapy (NAT) should increase the rate of breast-conserving surgery (BCS) in non-metastatic breast cancer (BC) patients, especially in those achieving tumor shrinkage. Still, the conversion from a pre-planned mastectomy to BCS in patients responding to NAT is not a widespread standard. We aimed to identify factors influencing surgical choices in this setting.

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  • In the past decade, targeted kidney inhibitors (TKIs) have significantly improved the survival rates of chronic myeloid leukemia (CML) patients, particularly those achieving deep and sustained molecular response (DMR), allowing some to consider stopping treatment.
  • A study analyzed responses from 1,777 CML patients in Italy, focusing on the impact of different TKIs (imatinib, dasatinib, nilotinib) and timing of responses (3, 6, and 12 months).
  • Results showed that patients achieving certain molecular response benchmarks at 3 months were more likely to achieve DMR later, with significant differences in outcomes based on the specific TKI used.
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We report the long-term results of a randomized trial (GITMO, AML-R2), comparing 1:1 the combination of busulfan and cyclophosphamide (BuCy2, n = 125) and the combination of busulfan and fludarabine (BuFlu, n = 127) as conditioning regimen in acute myeloid leukemia patients (median age 51 years, range 40-65) undergoing allogeneic hematopoietic stem cell transplantation. With a median follow-up of 6 years, significantly better non-relapse mortality (NRM) was confirmed in BuFlu recipients, which is sustained up to 4 years after transplant (10% vs. 20%, p = 0.

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  • Immunological therapies, including immune checkpoint inhibitors (ICIs) and cell-based therapies like CAR-T, have transformed cancer treatment by enabling the immune system to target cancer cells.
  • While these therapies are generally effective, they can cause immune-related adverse events (irAEs) that vary in severity and timing, from mild skin rashes to serious complications such as myocarditis or cytokine release syndrome.
  • The statement discusses the growing understanding of cardiovascular toxicities associated with these therapies, outlines their diagnosis and management, and identifies gaps in current research that need further exploration.
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  • Ruxolitinib (RUX) is a treatment for myelofibrosis, mainly studied in high-risk patients, but is often given to intermediate-1 patients with limited data on its effects.
  • In a study of 1,055 myelofibrosis patients, over half were classified as intermediate-1 risk, with notable symptoms and some having high-molecular-risk mutations.
  • The study found that after 6 months of RUX treatment, a significant proportion of patients experienced improvements in spleen size and symptoms, with certain factors like the absence of high-molecular-risk mutations being linked to better treatment responses.
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  • - Hypereosinophilic syndrome (HES) is a group of diverse disorders characterized by high levels of eosinophils, with ongoing challenges in diagnosis, understanding, and personalized treatment still needing attention.
  • - There is an urgent need to reduce the time it takes to diagnose and begin treatment for HES, as the condition significantly affects patients' quality of life.
  • - The Italian Society of Allergy, Asthma, and Clinical Immunology (SIAAIC) has launched a national initiative, the InHES network, to unify medical efforts and improve communication about HES, including sharing recent research findings and treatment recommendations.
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In multiple myeloma (MM) bone marrow infiltration by monoclonal plasma cells can occur in both focal and diffuse manner, making staging and prognosis rather difficult. The aim of our study was to test whether texture analysis of 18 F-2-deoxy-d-glucose positron emission tomography/computed tomography (18F-FDG PET/CT) images can predict survival in MM patients. Forty-six patients underwent 18 F-FDG-PET/CT before treatment.

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Background: Gemtuzumab-ozogamycin (GO) is approved in combination with high-dose chemotherapy for treatment-naïve low- and intermediate-risk acute myeloid leukemia (AML).

Aims: In this retrospective real-life multicenter study, we reported efficacy and safety of GO plus high-dose chemotherapy in newly diagnosed AML patients.

Methods And Results: A total of 31 fit low- and intermediate-risk AML patients treated with GO-based regimens were retrospectively included in this real-life multicenter study, and results were compared with a control cohort treated with 3 + 7 alone.

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Real-world data in clinical practice are needed to confirm the efficacy and safety that ibrutinib has demonstrated in clinical trials of patients with chronic lymphocytic leukemia (CLL). We described the real-world persistence rate, patterns of use, and clinical outcomes in 309 patients with CLL receiving single-agent ibrutinib in first line (1L, = 118), 2L ( = 127) and ≥3L ( = 64) in the prospective, real-world, Italian EVIdeNCE study. After a median follow-up of 23.

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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.

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Background: This retrospective multicentric study aims to report on technical safety and effectiveness of pseudoaneurysms embolization with glue (N-butyl cyanoacrylate) adopting a percutaneous direct puncture approach.

Results: Fifty-four patients data were collected from five centers. All patients at the time of treatment presented with unruptured PAs and were hemodynamically stable.

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  • Anemia is common in patients with myelofibrosis (MF) and is worsened by the treatment drug ruxolitinib (RUX), with new cases of blast phase (BP) emerging in anemic patients not previously on this treatment.
  • A study of 886 MF patients treated with RUX found a BP incidence rate of 3.74 per 100 patient-years, with higher rates in patients who had varying levels of anemia; the most severe cases were in those dependent on transfusions.
  • The findings suggest that both pre-existing and treatment-induced anemia significantly increase the risk of BP development, indicating a need for better anemia treatments alongside MF therapies.
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  • VEXAS is a unique disease that combines symptoms of rheumatologic and hematologic disorders, and this study aimed to better understand its diagnosis and genetic features while tracking changes over time with different treatments.
  • Researchers gathered data from various centers in Italy, finding that 41 male patients had significant mutations in the UBA1 gene, mostly diagnosed around age 67, all presenting with anemia and common rheumatologic issues like polychondritis.
  • A high percentage of these patients also had myelodysplastic syndrome (MDS), showcasing diverse genetic mutations, and the study noted that after treatment like hematopoietic cell transplants, some mutations were cleared.
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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.

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  • A significant number of myelofibrosis patients stop using ruxolitinib within the first 5 years due to treatment failure, highlighting the need for early identification of those at risk.* -
  • A study analyzed data from 889 patients and found that factors like low platelet count, low hemoglobin, and certain disease types increase the likelihood of stopping ruxolitinib treatment early.* -
  • A new prognostic model called STR-PM was developed to categorize patients into low, intermediate, and high-risk groups for early treatment failure, suggesting that those in higher risk categories might benefit from alternative treatments.*
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