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  1. Article ; Online: Can any new thalassemia therapy achieve a better than 97.4% cure rate?

    Angelucci, Emanuele

    American journal of hematology

    2020  Volume 95, Issue 11, Page(s) 1233–1234

    MeSH term(s) Humans ; Stem Cell Transplantation ; Thalassemia ; beta-Thalassemia
    Language English
    Publishing date 2020-08-24
    Publishing country United States
    Document type Journal Article ; Comment
    ZDB-ID 196767-8
    ISSN 1096-8652 ; 0361-8609
    ISSN (online) 1096-8652
    ISSN 0361-8609
    DOI 10.1002/ajh.25952
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    Zs.A 1251: Show issues Location:
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  2. Article: Mitapivat for sickle cell disease and thalassemia.

    Pilo, Federica / Angelucci, Emanuele

    Drugs of today (Barcelona, Spain : 1998)

    2023  Volume 59, Issue 3, Page(s) 125–134

    Abstract: Mitapivat, an oral first-in-class activator of erythrocyte pyruvate kinase (PKR), was first investigated in patients with pyruvate kinase deficiency (PKD), where it was found to improve hemoglobin (Hb) concentrations in patients who did not regularly ... ...

    Abstract Mitapivat, an oral first-in-class activator of erythrocyte pyruvate kinase (PKR), was first investigated in patients with pyruvate kinase deficiency (PKD), where it was found to improve hemoglobin (Hb) concentrations in patients who did not regularly receive transfusions and to reduce transfusion burden in patients who receive regular transfusions. It was approved in 2022 for the treatment of PKD and is being explored in other hereditary chronic conditions that are associated with hemolytic mechanisms of anemia, such as sickle cell disease (SCD) and thalassemia. In a proof-of-concept phase I study in SCD, treatment with mitapivat demonstrated efficacy in increasing Hb concentrations, but also restored the thermostability of PKR, increasing its activity and decreasing 2,3-diphosphoglycerate (2,3-DPG) levels in sickle erythrocytes, which decreases Hb polymerization by increasing the affinity of Hb to oxygen. In thalassemia, mitapivat is hypothesized to increase adenosine triphosphate (ATP) production and mitigate harmful effects on red blood cells. This hypothesis is supported by preclinical data showing that mitapivat ameliorated ineffective erythropoiesis, iron overload and anemia in the Hbb
    MeSH term(s) Humans ; Anemia, Sickle Cell/drug therapy ; beta-Thalassemia ; Piperazines ; Quinolines ; Thalassemia/drug therapy
    Chemical Substances mitapivat (2WTV10SIKH) ; Piperazines ; Quinolines
    Language English
    Publishing date 2023-02-18
    Publishing country Spain
    Document type Clinical Trial, Phase II ; Clinical Trial, Phase I ; Multicenter Study ; Journal Article
    ZDB-ID 2018441-4
    ISSN 1699-3993
    ISSN 1699-3993
    DOI 10.1358/dot.2023.59.3.3521880
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  3. Article ; Online: A new medical therapy for anemia in thalassemia.

    Angelucci, Emanuele

    Blood

    2019  Volume 133, Issue 12, Page(s) 1267–1268

    MeSH term(s) Activin Receptors, Type II ; Activins ; Blood Transfusion ; Hemoglobins ; Humans ; Immunoglobulin Fc Fragments ; Recombinant Fusion Proteins ; Thalassemia ; beta-Thalassemia
    Chemical Substances Hemoglobins ; Immunoglobulin Fc Fragments ; Recombinant Fusion Proteins ; Activins (104625-48-1) ; luspatercept (AQK7UBA1LS) ; Activin Receptors, Type II (EC 2.7.11.30)
    Language English
    Publishing date 2019-03-19
    Publishing country United States
    Document type Journal Article ; Comment
    ZDB-ID 80069-7
    ISSN 1528-0020 ; 0006-4971
    ISSN (online) 1528-0020
    ISSN 0006-4971
    DOI 10.1182/blood-2019-01-897587
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  4. Article ; Online: Complication free survival long-term after hemopoietic cell transplantation in thalassemia.

    Angelucci, Emanuele

    Haematologica

    2018  Volume 103, Issue 7, Page(s) 1094–1096

    MeSH term(s) Hematopoietic Stem Cell Transplantation ; Humans ; Thalassemia ; beta-Thalassemia
    Language English
    Publishing date 2018-07-03
    Publishing country Italy
    Document type Editorial ; Comment
    ZDB-ID 2333-4
    ISSN 1592-8721 ; 0017-6567 ; 0390-6078
    ISSN (online) 1592-8721
    ISSN 0017-6567 ; 0390-6078
    DOI 10.3324/haematol.2018.196071
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  5. Article: Hemophagocytic Lymphohistiocytosis Secondary to Refractory Acute Myeloid Leukemia Resolved after Second-Line Treatment with Azacitidine plus Venetoclax.

    Zorzetto, Francesco / Scalas, Alessandra / Longu, Francesco / Isoni, Maria Antonia / Angelucci, Emanuele / Fozza, Claudio

    Mediterranean journal of hematology and infectious diseases

    2024  Volume 16, Issue 1, Page(s) e2024011

    Language English
    Publishing date 2024-01-01
    Publishing country Italy
    Document type Journal Article ; Comment
    ZDB-ID 2674750-9
    ISSN 2035-3006
    ISSN 2035-3006
    DOI 10.4084/MJHID.2024.011
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  6. Article ; Online: A large co-operative biological and clinical study to better understand and improve treatment of the rare t(8;16)(p11;p13) acute myeloid leukaemia.

    Becker, Michael W / Angelucci, Emanuele

    British journal of haematology

    2021  Volume 192, Issue 5, Page(s) 800–802

    Language English
    Publishing date 2021-02-04
    Publishing country England
    Document type Journal Article ; Comment
    ZDB-ID 80077-6
    ISSN 1365-2141 ; 0007-1048
    ISSN (online) 1365-2141
    ISSN 0007-1048
    DOI 10.1111/bjh.17332
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  7. Article: Real-World Data Analysis of Patients Affected by Immune-Mediated Thrombotic Thrombocytopenic Purpura in Italy.

    Angelucci, Emanuele / Artoni, Andrea / Fianchi, Luana / Dovizio, Melania / Iacolare, Biagio / Saragoni, Stefania / Esposti, Luca Degli

    Journal of clinical medicine

    2024  Volume 13, Issue 5

    Abstract: Background: ...

    Abstract Background:
    Language English
    Publishing date 2024-02-27
    Publishing country Switzerland
    Document type Journal Article
    ZDB-ID 2662592-1
    ISSN 2077-0383
    ISSN 2077-0383
    DOI 10.3390/jcm13051342
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  8. Article ; Online: Another Step Forward in Iron Chelation Therapy.

    Angelucci, Emanuele

    Acta haematologica

    2015  Volume 134, Issue 4, Page(s) 231–232

    MeSH term(s) Anemia, Aplastic/drug therapy ; Benzoates/administration & dosage ; Humans ; Iron Overload/drug therapy ; Liver/metabolism ; Myelodysplastic Syndromes/drug therapy ; Triazoles/administration & dosage
    Chemical Substances Benzoates ; Triazoles
    Language English
    Publishing date 2015
    Publishing country Switzerland
    Document type Comment ; Journal Article
    ZDB-ID 80008-9
    ISSN 1421-9662 ; 0001-5792
    ISSN (online) 1421-9662
    ISSN 0001-5792
    DOI 10.1159/000431215
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  9. Article ; Online: Hurdles to the Adoption of Gene Therapy as a Curative Option for Transfusion-Dependent Thalassemia.

    Thuret, Isabelle / Ruggeri, Annalisa / Angelucci, Emanuele / Chabannon, Christian

    Stem cells translational medicine

    2022  Volume 11, Issue 4, Page(s) 407–414

    Abstract: Beta-thalassemia is one of the most common monogenic disorders. Standard treatment of the most severe forms, i.e., transfusion-dependent thalassemia (TDT) with long-term transfusion and iron chelation, represents a considerable medical, psychological, ... ...

    Abstract Beta-thalassemia is one of the most common monogenic disorders. Standard treatment of the most severe forms, i.e., transfusion-dependent thalassemia (TDT) with long-term transfusion and iron chelation, represents a considerable medical, psychological, and economic burden. Allogeneic hematopoietic stem cell transplantation from an HLA-identical donor is a curative treatment with excellent results in children. Recently, several gene therapy approaches were evaluated in academia or industry-sponsored clinical trials as alternative curative options for children and young adults without an HLA-identical donor. Gene therapy by addition of a functional beta-globin gene using self-inactivating lentiviral vectors in autologous stem cells resulted in transfusion independence for a majority of TDT patients across different age groups and genotypes, with a current follow-up of multiple years. More recently, promising results were reported in TDT patients treated with autologous hematopoietic stem cells edited with the clustered regularly interspaced short palindromic repeats-Cas9 technology targeting erythroid BCL11A expression, a key regulator of the normal switch from fetal to adult globin production. Patients achieved high levels of fetal hemoglobin allowing for discontinuation of transfusions. Despite remarkable clinical efficacy, 2 major hurdles to gene therapy access for TDT patients materialized in 2021: (1) a risk of secondary hematological malignancies that is complex and multifactorial in origin and not limited to the risk of insertional mutagenesis, (2) the cost-even in high-income countries-is leading to the arrest of commercialization in Europe of the first gene therapy medicinal product indicated for TDT despite conditional approval by the European Medicines Agency.
    MeSH term(s) Child ; Genetic Therapy/methods ; Hematopoietic Stem Cell Transplantation ; Humans ; Thalassemia/genetics ; Thalassemia/therapy ; Young Adult ; beta-Globins/genetics ; beta-Thalassemia/genetics ; beta-Thalassemia/therapy
    Chemical Substances beta-Globins
    Language English
    Publishing date 2022-03-10
    Publishing country England
    Document type Journal Article
    ZDB-ID 2642270-0
    ISSN 2157-6580 ; 2157-6580
    ISSN (online) 2157-6580
    ISSN 2157-6580
    DOI 10.1093/stcltm/szac007
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  10. Article ; Online: Histopathological Markers for Target Therapies in Primary Cutaneous Lymphomas.

    Sonego, Benedetta / Ibatici, Adalberto / Rivoli, Giulia / Angelucci, Emanuele / Sola, Simona / Massone, Cesare

    Cells

    2023  Volume 12, Issue 22

    Abstract: In recent years, targeted (biological) therapies have become available also for primary cutaneous T-cell lymphomas (PCTCLs) including anti-CD30 (brentuximab vedotin) in mycosis fungoides, primary cutaneous anaplastic large T-cell lymphoma, lymphomatoid ... ...

    Abstract In recent years, targeted (biological) therapies have become available also for primary cutaneous T-cell lymphomas (PCTCLs) including anti-CD30 (brentuximab vedotin) in mycosis fungoides, primary cutaneous anaplastic large T-cell lymphoma, lymphomatoid papulosis; anti-CCR4 (mogamulizumab) in Sezary syndrome; anti-CD123 (tagraxofusp) in blastic plasmocytoid cell neoplasm. Moreover, anti-PD1 (nivolumab), anti-PDL1 (pembrolizumab, atezolizumab), anti-CD52 (alemtuzumab), anti-KIR3DL2-CD158k (lacutamab), and anti-CD70 (cusatuzumab) have been tested or are under investigations in phase II trials. The expression of these epitopes on neoplastic cells in skin biopsies or blood samples plays a central role in the management of PCTCL patients. This narrative review aims to provide readers with an update on the latest advances in the newest therapeutic options for PCTCLs.
    MeSH term(s) Humans ; Skin Neoplasms/pathology ; Mycosis Fungoides/pathology ; Brentuximab Vedotin/therapeutic use ; Sezary Syndrome/pathology ; Antineoplastic Agents/therapeutic use ; Antibodies, Monoclonal/therapeutic use
    Chemical Substances Brentuximab Vedotin (7XL5ISS668) ; Antineoplastic Agents ; Antibodies, Monoclonal
    Language English
    Publishing date 2023-11-20
    Publishing country Switzerland
    Document type Journal Article ; Review
    ZDB-ID 2661518-6
    ISSN 2073-4409 ; 2073-4409
    ISSN (online) 2073-4409
    ISSN 2073-4409
    DOI 10.3390/cells12222656
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