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  1. Article ; Online: Cost-Effectiveness of Recombinant Factor IX Fc Prophylaxis and Recombinant Factor IX On-Demand Treatment in Patients with Haemophilia B Without Inhibitors.

    Pochopien, Michal / Tytuła, Anna / Toumi, Mondher / Falk, Aletta / Martone, Nicoletta / Hakimi, Zalmai / Eriksson, Daniel

    Advances in therapy

    2024  

    Abstract: Introduction: Recombinant factor IX (rFIX) and recombinant FIX Fc fusion protein (rFIXFc) are standard half-life and extended half-life FIX replacement therapies, respectively, and represent established treatment options indicated for adults and ... ...

    Abstract Introduction: Recombinant factor IX (rFIX) and recombinant FIX Fc fusion protein (rFIXFc) are standard half-life and extended half-life FIX replacement therapies, respectively, and represent established treatment options indicated for adults and children with haemophilia B. These FIX replacement therapies can be administered as prophylaxis (to prevent bleeding) or 'on-demand' (to stop bleeding). This analysis aimed to estimate the cost-effectiveness of once-weekly prophylaxis with rFIXFc versus on-demand treatment with rFIX in patients with haemophilia B without inhibitors in the Italian healthcare setting.
    Methods: A Markov model was developed to assess a hypothetical cohort of adolescent or adult male patients (≥ 12 years) with haemophilia B (FIX level of ≤ 2 IU/dL) without inhibitors. Model inputs were derived from the pivotal phase 3 clinical studies for rFIXFc and rFIX, published literature and assumptions when published data were unavailable. The model employed a lifelong time horizon with 6-monthly transitions between health states, and it estimated total costs, total quality-adjusted life years (QALYs), number of bleeds, number of surgeries and incremental cost-effectiveness ratio.
    Results: rFIXFc prophylaxis was associated with lower total costs per patient (€5,308,625 versus €6,564,510) and greater total QALYs per patient (15.936 versus 11.943) compared with rFIX on-demand; rFIXFc prophylaxis was therefore the dominant treatment strategy. The model also demonstrated that rFIXFc prophylaxis was associated with fewer incremental bleeds (- 682.29) and surgeries (- 0.39) compared with rFIX on-demand.
    Conclusions: rFIXFc prophylaxis provides improved health outcomes and lower costs, and represents a cost-effective treatment option compared with rFIX on-demand for adolescent and adult male patients with haemophilia B. This comparative assessment of cost-effectiveness should help to inform both clinicians and healthcare policy makers when making treatment decisions for patients with haemophilia B.
    Language English
    Publishing date 2024-04-23
    Publishing country United States
    Document type Journal Article
    ZDB-ID 632651-1
    ISSN 1865-8652 ; 0741-238X
    ISSN (online) 1865-8652
    ISSN 0741-238X
    DOI 10.1007/s12325-024-02841-w
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

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  2. Article ; Online: Direct, precise, enzyme-free detection of miR-103-3p in real samples by microgels with highly specific molecular beacons

    Napoletano, Sabrina / Battista, Edmondo / Martone, Nicoletta / Netti, Paolo Antonio / Causa, Filippo

    Talanta. 2023 July, v. 259 p.124468-

    2023  

    Abstract: Low abundance, small size, and sequence similarities render microRNA (miRNAs) detection challenging, particularly in real samples, where quantifying weakly expressed miRNAs can be arduous due to interference of more abundant molecules. The standard ... ...

    Abstract Low abundance, small size, and sequence similarities render microRNA (miRNAs) detection challenging, particularly in real samples, where quantifying weakly expressed miRNAs can be arduous due to interference of more abundant molecules. The standard quantitative reverse transcription polymerase chain reaction (qRT-PCR) requires multiple steps, thermal cycles, and costly enzymatic reactions that can negatively affect results. Here we present a direct, precise, enzyme-free assay based on microgels particles conjugating molecular beacons (MB) capable of optically detecting low abundant miRNAs in real samples. We validate the applicability of microgels assay using qRT-PCR as a reference technology. As a relevant case, we chose miR-103-3p, a valuable diagnostic biomarker for breast cancer, both in serum samples and MCF7 cells. As a result, microgels assay quantifies miRNA molecules at room temperature in a single step, 1 h (vs. 4 hrs for qRT-PCR) without complementary DNA synthesis, amplification, or expensive reagents. Microgels assay exhibits femtomolar sensitivity, single nucleotide specificity, and a wide linear range (10²-10⁷ fM) (wider than qRT-PCR), with low sample consumption (2 μL) and excellent linearity (R²= 0.98). To test the selectivity of the microgel assay in real samples, MCF7 cells were considered where the pool of 8 other miRNAs were further upregulated with respect to miRNA 103-3p. In such complex environments, microgels assay selectively detects the miRNA target, mainly due to MB advanced stability and specificity as well as high microgel antifouling properties. These results show the reliability of microgels assay to detect miRNAs in real samples.
    Keywords DNA replication ; ambient temperature ; biomarkers ; blood serum ; breast neoplasms ; complementary DNA ; microRNA ; microgels ; reverse transcriptase polymerase chain reaction ; miRNAs detection ; Biosensor ; Microgel ; Sensitivity ; Real-sample
    Language English
    Dates of publication 2023-07
    Publishing place Elsevier B.V.
    Document type Article ; Online
    Note Use and reproduction
    ZDB-ID 1500969-5
    ISSN 1873-3573 ; 0039-9140
    ISSN (online) 1873-3573
    ISSN 0039-9140
    DOI 10.1016/j.talanta.2023.124468
    Database NAL-Catalogue (AGRICOLA)

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  3. Article: Cost-Utility Analysis Comparing Pegcetacoplan to Anti-C5 Monoclonal Antibodies in the Treatment of Paroxysmal Nocturnal Hemoglobinuria.

    Di Matteo, Sergio / Freilone, Roberto / Bruno, Giacomo Matteo / Notaro, Rosario / Moumene, Sabrin / Martone, Nicoletta / Teruzzi, Cristina / Ciccarone, Antonio / Colombo, Giorgio Lorenzo

    ClinicoEconomics and outcomes research : CEOR

    2024  Volume 16, Page(s) 225–232

    Abstract: Background: Paroxysmal nocturnal hemoglobinuria is a rare, acquired disease characterized by hemolytic episodes and associated with significant clinical burden. The introduction of C5 inhibitory monoclonal antibodies (C5i) represented a major ... ...

    Abstract Background: Paroxysmal nocturnal hemoglobinuria is a rare, acquired disease characterized by hemolytic episodes and associated with significant clinical burden. The introduction of C5 inhibitory monoclonal antibodies (C5i) represented a major breakthrough in PNH treatment, effectively reducing intravascular hemolysis (IVH) but showing limited impact on extravascular hemolysis (EVH). In 2021, the C3 inhibitor pegcetacoplan was approved by EMA and recently reimbursed in Italy, which also has the advantages in the reduction of both IVH and EVH, increasing hemoglobin values and simultaneously improving the quality of life and fatigue of patients. A cost-utility analysis was developed to compare pegcetacoplan to C5i (eculizumab and ravulizumab) in the PNH population who remain anemic after treatment with C5i for at least 3 months.
    Materials and methods: The analysis employed a Markov model with a 5-year time horizon whereby patients can transition among 3 PNH health states, adopting the perspective of the Italian NHS. Efficacy data were sourced from the PEGASUS study, with drug prices reflecting ex-factory costs. Additionally, costs associated with resource utilization, adverse events, and complications were estimated based on outpatient and hospital care rates, excluding indirect expenses. Utility and disutility values related to transfusions were also considered, with pegcetacoplan allowing for dose escalation.
    Results: The cumulative cost of treatment per individual patient at 5 years was estimated to be €1,483,454 for pegcetacoplan, €1,585,763 for eculizumab, and €1,574,826 for ravulizumab. Pegcetacoplan demonstrated a superior increase in quality-adjusted life years (QALYs) compared to both eculizumab (0.51 increase) and ravulizumab (0.27 increase). Furthermore, pegcetacoplan showed a reduction in complication management costs (€22,891 less compared to eculizumab and €22,611 less compared to ravulizumab) and lower transfusion-related expenses (€14,147 less than both C5i treatments).
    Conclusion: Pegcetacoplan emerged as the dominant strategy in this analysis, being more effective, less expensive and improves quality of life in the analyzed population affected by PNH.
    Language English
    Publishing date 2024-04-11
    Publishing country New Zealand
    Document type Journal Article
    ZDB-ID 2520698-9
    ISSN 1178-6981
    ISSN 1178-6981
    DOI 10.2147/CEOR.S442906
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  4. Article ; Online: Direct, precise, enzyme-free detection of miR-103-3p in real samples by microgels with highly specific molecular beacons.

    Napoletano, Sabrina / Battista, Edmondo / Martone, Nicoletta / Netti, Paolo Antonio / Causa, Filippo

    Talanta

    2023  Volume 259, Page(s) 124468

    Abstract: Low abundance, small size, and sequence similarities render microRNA (miRNAs) detection challenging, particularly in real samples, where quantifying weakly expressed miRNAs can be arduous due to interference of more abundant molecules. The standard ... ...

    Abstract Low abundance, small size, and sequence similarities render microRNA (miRNAs) detection challenging, particularly in real samples, where quantifying weakly expressed miRNAs can be arduous due to interference of more abundant molecules. The standard quantitative reverse transcription polymerase chain reaction (qRT-PCR) requires multiple steps, thermal cycles, and costly enzymatic reactions that can negatively affect results. Here we present a direct, precise, enzyme-free assay based on microgels particles conjugating molecular beacons (MB) capable of optically detecting low abundant miRNAs in real samples. We validate the applicability of microgels assay using qRT-PCR as a reference technology. As a relevant case, we chose miR-103-3p, a valuable diagnostic biomarker for breast cancer, both in serum samples and MCF7 cells. As a result, microgels assay quantifies miRNA molecules at room temperature in a single step, 1 h (vs. 4 hrs for qRT-PCR) without complementary DNA synthesis, amplification, or expensive reagents. Microgels assay exhibits femtomolar sensitivity, single nucleotide specificity, and a wide linear range (10
    MeSH term(s) Microgels ; Reproducibility of Results ; MicroRNAs/analysis ; Real-Time Polymerase Chain Reaction
    Chemical Substances Microgels ; MicroRNAs
    Language English
    Publishing date 2023-03-23
    Publishing country Netherlands
    Document type Journal Article
    ZDB-ID 1500969-5
    ISSN 1873-3573 ; 0039-9140
    ISSN (online) 1873-3573
    ISSN 0039-9140
    DOI 10.1016/j.talanta.2023.124468
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  5. Article: Budget impact analysis for avatrombopag in the treatment of chronic primary immune thrombocytopenia in adult patients refractory to other treatments.

    Aiello, Andrea / Mariano, Elisa Elena / Prada, Mariangela / Teruzzi, Cristina / Martone, Nicoletta / Capri, Stefano / Carli, Giuseppe / Siragusa, Sergio

    Journal of market access & health policy

    2023  Volume 11, Issue 1, Page(s) 2230663

    Abstract: Introduction: ...

    Abstract Introduction:
    Language English
    Publishing date 2023-06-30
    Publishing country United States
    Document type Journal Article
    ISSN 2001-6689
    ISSN 2001-6689
    DOI 10.1080/20016689.2023.2230663
    Database MEDical Literature Analysis and Retrieval System OnLINE

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  6. Article ; Online: Early Palliative Home Care versus Hospital Care for Patients with Hematologic Malignancies: A Cost-Effectiveness Study.

    Cartoni, Claudio / Breccia, Massimo / Giesinger, Johannes Maria / Baldacci, Erminia / Carmosino, Ida / Annechini, Giorgia / Palumbo, Giovanna / Armiento, Daniele / Niscola, Pasquale / Tendas, Andrea / Brunetti, Gregorio Antonio / Minotti, Clara / Marini, Maria Giulia / Reale, Luigi / Martone, Nicoletta / Martelli, Maurizio / Efficace, Fabio

    Journal of palliative medicine

    2020  Volume 24, Issue 6, Page(s) 887–893

    Abstract: Background: ...

    Abstract Background:
    MeSH term(s) Cost-Benefit Analysis ; Hematologic Neoplasms ; Home Care Services ; Hospitals ; Humans ; Palliative Care
    Language English
    Publishing date 2020-12-02
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 1427361-5
    ISSN 1557-7740 ; 1096-6218
    ISSN (online) 1557-7740
    ISSN 1096-6218
    DOI 10.1089/jpm.2020.0396
    Database MEDical Literature Analysis and Retrieval System OnLINE

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    In stock of ZB MED Cologne/Königswinter

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