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  1. Article ; Online: Rainer Prohaska (1943-2022).

    Salzer, Ulrich / Mairhofer, Mario / De Franceschi, Lucia

    American journal of hematology

    2023  Volume 99, Issue 1, Page(s) 144–145

    Language English
    Publishing date 2023-10-23
    Publishing country United States
    Document type Journal Article
    ZDB-ID 196767-8
    ISSN 1096-8652 ; 0361-8609
    ISSN (online) 1096-8652
    ISSN 0361-8609
    DOI 10.1002/ajh.27139
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    Zs.A 1251: Show issues Location:
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  2. Article ; Online: Erythrocyte pyruvate kinase activation in red cell disorders.

    Matte, Alessandro / Federti, Enrica / De Franceschi, Lucia

    Current opinion in hematology

    2023  Volume 30, Issue 3, Page(s) 93–98

    Abstract: Purpose of review: In red cells, pyruvate kinase is a key enzyme in the final step of glycolytic degradative process, which generates a constant energy supply via ATP production. This commentary discusses recent findings on pyruvate kinase activators as ...

    Abstract Purpose of review: In red cells, pyruvate kinase is a key enzyme in the final step of glycolytic degradative process, which generates a constant energy supply via ATP production. This commentary discusses recent findings on pyruvate kinase activators as new therapeutic option in hereditary red cell disorders such as thalassemic syndromes or sickle cell disease (SCD).
    Recent findings: Mitapivat and etavopivat are two oral pyruvate kinase activators. Studies in a mouse model for β thalassemia have shown beneficial effects of mitapivat on both red cell survival and ineffective erythropoiesis, with an amelioration of iron homeostasis. This was confirmed in a proof-of-concept study in patients with nontransfusion-dependent thalassemias. Both mitapivat and etavopivat have been evaluated in mouse models for SCD, showing an increased 2-3DPG/ATP ratio and a reduction in haemolysis as well as in sickling. These data were confirmed in proof-of-concept clinical studies with both molecules carried in patients with SCD.
    Summary: Preclinical and clinical evidence indicate that pyruvate kinase activators represent new therapeutic option in hemoglobinopathies or SCD. Other red cell disorders such as hereditary spherocytosis or hereditary anaemias characterized by defective erythropoiesis might represent additional areas to investigate the therapeutic impact of pyruvate kinase activators.
    MeSH term(s) Mice ; Animals ; Humans ; Pyruvate Kinase ; Erythrocytes ; Hematologic Diseases ; Anemia, Sickle Cell ; Adenosine Triphosphate
    Chemical Substances Pyruvate Kinase (EC 2.7.1.40) ; Adenosine Triphosphate (8L70Q75FXE)
    Language English
    Publishing date 2023-02-09
    Publishing country United States
    Document type Review ; Journal Article ; Research Support, Non-U.S. Gov't
    ZDB-ID 1153887-9
    ISSN 1531-7048 ; 1065-6251
    ISSN (online) 1531-7048
    ISSN 1065-6251
    DOI 10.1097/MOH.0000000000000758
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  3. Article ; Online: Iron restriction in sickle cell disease: When less is more.

    Castro, Oswaldo L / De Franceschi, Lucia / Ganz, Tomas / Kanter, Julie / Kato, Gregory J / Pasricha, Sant-Rayn / Rivella, Stefano / Wood, John C

    American journal of hematology

    2024  

    Abstract: Primum non nocere! Can iron deficiency, an abnormality that causes anemia, benefit people with sickle cell disease (SCD) who already have an anemia? The published literature we review appears to answer this question in the affirmative: basic science ... ...

    Abstract Primum non nocere! Can iron deficiency, an abnormality that causes anemia, benefit people with sickle cell disease (SCD) who already have an anemia? The published literature we review appears to answer this question in the affirmative: basic science considerations, animal model experiments, and noncontrolled clinical observations all suggest a therapeutic potential of iron restriction in SCD. This is because SCD's clinical manifestations are ultimately attributable to the polymerization of hemoglobin S (HbS), a process strongly influenced by intracellular HbS concentration. Even small decrements in HbS concentration greatly reduce polymerization, and iron deficiency lowers erythrocyte hemoglobin concentration. Thus, iron deficiency could improve SCD by changing its clinical features to those of a more benign anemia (i.e., a condition with fewer or no vaso-occlusive events). We propose that well-designed clinical studies be implemented to definitively determine whether iron restriction is a safe and effective option in SCD. These investigations are particularly timely now that pharmacologic agents are being developed, which may directly reduce red cell hemoglobin concentrations without the need for phlebotomies to deplete total body iron.
    Language English
    Publishing date 2024-02-23
    Publishing country United States
    Document type Journal Article ; Review
    ZDB-ID 196767-8
    ISSN 1096-8652 ; 0361-8609
    ISSN (online) 1096-8652
    ISSN 0361-8609
    DOI 10.1002/ajh.27267
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  4. Article ; Online: SUCCESSES AND PITFALLS IN ORPHAN DRUG DEVELOPMENT FOR SICKLE CELL DISEASE.

    Costa, Enrico / Isgrò, Antonella / de Montalembert, Mariane / Leufkens, Hubert G M / Ware, Russell E / De Franceschi, Lucia

    Blood advances

    2024  

    Abstract: Sickle cell disease (SCD) is a hereditary red cell disorder with large global burden problem. In the United States (US) and Europe, medicines may qualify for orphan designation (OD), a regulatory status that provides incentives to boost development. We ... ...

    Abstract Sickle cell disease (SCD) is a hereditary red cell disorder with large global burden problem. In the United States (US) and Europe, medicines may qualify for orphan designation (OD), a regulatory status that provides incentives to boost development. We evaluated the development of new therapies for SCD using data for OD granted in the US and Europe over the last two decades (2000-2021). We analyzed their characteristics, pathophysiological targets, trends, and OD sponsors. We then investigated the approval outcomes, including the phase success rate and reasons for discontinuation across different variables. We identified 57 OD for SCD: 43 (75.4%) small molecules, 32 (56.1%) for oral administration, and 36 (63.1%) for chronic use to prevent SCD complications. At the end of the study (2021) development of 34/57 ODs was completed. Four OD were approved with a success rate of 11.8%. Products targeting upstream causative events of SCD pathophysiology had a 1.8 higher success rate compared to products targeting disease consequences. Large companies showed a fourfold higher success rate compared to small-medium enterprises. Failures in clinical development were mainly seen in Phase 3 for a lack of efficacy on vaso-occlusive crisis as the primary study endpoint, likely related to variable definitions and heterogeneity of pain scoring and treatment. Both advances in SCD knowledge and regulatory incentives paved the way for new therapies for SCD. Our finding of high failure rates in late-stage clinical development signals the need for better early-stage predictive models, also in the context of meaningful clinical endpoints.
    Language English
    Publishing date 2024-03-24
    Publishing country United States
    Document type Journal Article
    ZDB-ID 2915908-8
    ISSN 2473-9537 ; 2473-9529
    ISSN (online) 2473-9537
    ISSN 2473-9529
    DOI 10.1182/bloodadvances.2023011730
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  5. Article: Clinical Implications of

    Nista, Enrico Celestino / Pellegrino, Antonio / Giuli, Lucia / Candelli, Marcello / Schepis, Tommaso / De Lucia, Sara Sofia / Ojetti, Veronica / Franceschi, Francesco / Gasbarrini, Antonio

    Antibiotics (Basel, Switzerland)

    2022  Volume 11, Issue 10

    Abstract: Helicobacter ... ...

    Abstract Helicobacter pylori
    Language English
    Publishing date 2022-10-21
    Publishing country Switzerland
    Document type Journal Article ; Review
    ZDB-ID 2681345-2
    ISSN 2079-6382
    ISSN 2079-6382
    DOI 10.3390/antibiotics11101452
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  6. Article ; Online: The Biological and Clinical Role of the Telomerase Reverse Transcriptase Gene in Glioblastoma: A Potential Therapeutic Target?

    Di Nunno, Vincenzo / Aprile, Marta / Bartolini, Stefania / Gatto, Lidia / Tosoni, Alicia / Ranieri, Lucia / De Biase, Dario / Asioli, Sofia / Franceschi, Enrico

    Cells

    2023  Volume 13, Issue 1

    Abstract: ... ...

    Abstract Glioblastoma
    MeSH term(s) Humans ; Glioblastoma/drug therapy ; Glioblastoma/genetics ; Telomerase/genetics ; Central Nervous System ; Combined Modality Therapy ; Aggression
    Chemical Substances Telomerase (EC 2.7.7.49)
    Language English
    Publishing date 2023-12-25
    Publishing country Switzerland
    Document type Journal Article ; Review ; Research Support, Non-U.S. Gov't
    ZDB-ID 2661518-6
    ISSN 2073-4409 ; 2073-4409
    ISSN (online) 2073-4409
    ISSN 2073-4409
    DOI 10.3390/cells13010044
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  7. Article ; Online: Oxidation and erythropoiesis.

    Matte, Alessandro / De Franceschi, Lucia

    Current opinion in hematology

    2019  Volume 26, Issue 3, Page(s) 145–151

    Abstract: Purpose of review: Erythropoiesis is a complex multistep process going from committed erythroid progenitors to mature red cells. Although recent advances allow the characterization of some components of erythropoiesis, much still remains to be ... ...

    Abstract Purpose of review: Erythropoiesis is a complex multistep process going from committed erythroid progenitors to mature red cells. Although recent advances allow the characterization of some components of erythropoiesis, much still remains to be investigated particularly on stress erythropoiesis. This review summarizes recent progresses made to understand the impact of oxidative stress on normal and pathologic erythropoiesis.
    Recent findings: During erythroid maturation, reactive oxygen species might function as second messenger through either transient oxidation of cysteine residues on signaling targets or modulation of intracellular signaling pathways. Thus, in erythropoiesis, efficient cytoprotective systems are required to limit possible reactive oxygen species-related toxic effects especially in stress erythropoiesis characterized by severe oxidation such as β-thalassemia. In addition, prolonged or severe oxidative stress impairs autophagy, which might contribute to the block of erythroid maturation in stress erythropoiesis. Understanding the functional role of cytoprotective systems such as peroxiredoxin-2 or classical molecular chaperones such as the heat shock proteins will contribute to develop innovative therapeutic strategies for ineffective erythropoiesis.
    Summary: We provide an update on cytoprotective mechanisms against oxidation in normal and stress erythropoiesis. We discuss the role of oxidative sensors involved in modulation of intracellular signaling during erythroid maturation process in normal and stress erythropoiesis.
    MeSH term(s) Animals ; Erythroid Precursor Cells/metabolism ; Erythropoiesis ; Humans ; Oxidation-Reduction ; Oxidative Stress ; Peroxiredoxins/metabolism ; Second Messenger Systems
    Chemical Substances PRDX2 protein, human (EC 1.11.1.15) ; Peroxiredoxins (EC 1.11.1.15)
    Language English
    Publishing date 2019-03-11
    Publishing country United States
    Document type Journal Article ; Research Support, Non-U.S. Gov't ; Review
    ZDB-ID 1153887-9
    ISSN 1531-7048 ; 1065-6251
    ISSN (online) 1531-7048
    ISSN 1065-6251
    DOI 10.1097/MOH.0000000000000495
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  8. Article: Pharmacological Induction of Fetal Hemoglobin in β-Thalassemia and Sickle Cell Disease: An Updated Perspective.

    Bou-Fakhredin, Rayan / De Franceschi, Lucia / Motta, Irene / Cappellini, Maria Domenica / Taher, Ali T

    Pharmaceuticals (Basel, Switzerland)

    2022  Volume 15, Issue 6

    Abstract: A significant amount of attention has recently been devoted to the mechanisms involved in hemoglobin (Hb) switching, as it has previously been established that the induction of fetal hemoglobin (HbF) production in significant amounts can reduce the ... ...

    Abstract A significant amount of attention has recently been devoted to the mechanisms involved in hemoglobin (Hb) switching, as it has previously been established that the induction of fetal hemoglobin (HbF) production in significant amounts can reduce the severity of the clinical course in diseases such as β-thalassemia and sickle cell disease (SCD). While the induction of HbF using lentiviral and genome-editing strategies has been made possible, they present limitations. Meanwhile, progress in the use of pharmacologic agents for HbF induction and the identification of novel HbF-inducing strategies has been made possible as a result of a better understanding of γ-globin regulation. In this review, we will provide an update on all current pharmacological inducer agents of HbF in β-thalassemia and SCD in addition to the ongoing research into other novel, and potentially therapeutic, HbF-inducing agents.
    Language English
    Publishing date 2022-06-16
    Publishing country Switzerland
    Document type Journal Article ; Review
    ZDB-ID 2193542-7
    ISSN 1424-8247
    ISSN 1424-8247
    DOI 10.3390/ph15060753
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  9. Article ; Online: An Oral Carbon Monoxide-Releasing Molecule Protects against Acute Hyper-hemolysis in Sickle Cell Disease.

    Nguyen, Kim Anh / Matte, Alessandro / Foresti, Roberta / Federti, Enrica / Kiger, Laurent / Lefebvre, Cecile / Hocini, Hakim / Pelinski, Yanis / Kitagishi, Hiroaki / Bencheikh, Laura / Pirenne, France / De Franceschi, Lucia / Motterlini, Roberto / Bartolucci, Pablo

    Blood

    2024  

    Abstract: Acute hyper-hemolysis is a severe life-threatening complication in patients with sickle cell disease (SCD) that may occur during delayed hemolytic transfusion reaction (DHTR), or vaso-occlusive crises associated with multi-organ failure. Here, we ... ...

    Abstract Acute hyper-hemolysis is a severe life-threatening complication in patients with sickle cell disease (SCD) that may occur during delayed hemolytic transfusion reaction (DHTR), or vaso-occlusive crises associated with multi-organ failure. Here, we developed in vitro and in vivo animal models to mimic endothelial damage during the early phase of hyper-hemolysis in SCD. We then used the carbon monoxide (CO)-releasing molecule CORM-401 and examined its effects against endothelial activation, damage, and inflammation inflicted by hemolysates containing red blood cell membrane-derived particles. The in vitro results revealed that CORM-401: 1) prevented the up-regulation of relevant pro-inflammatory, and pro-adhesion controlled by the nuclear factor kappa-light-chain-enhancer of activated B cells (NF-κB), and 2) abolished the expression of the nuclear factor erythroid-2-related factor 2 (Nrf2) that regulates the inducible antioxidant cell machinery. We also show in SCD mice that CORM-401 protects against hemolysate-induced acute damage of target organs such as the lung, liver, and kidney through modulation of NF-kB pro-inflammatory and Nrf2 antioxidant pathways. Our data demonstrate the efficacy of CORM-401 as a novel therapeutic agent to counteract hemolysate-induced organ damage during hyper-hemolysis in SCD. This approach might be considered as possible preventive treatment in high-risk situations such as SCD patients with history of DHTR.
    Language English
    Publishing date 2024-03-22
    Publishing country United States
    Document type Journal Article
    ZDB-ID 80069-7
    ISSN 1528-0020 ; 0006-4971
    ISSN (online) 1528-0020
    ISSN 0006-4971
    DOI 10.1182/blood.2023023165
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  10. Article ; Online: Morbidity and mortality of sickle cell disease patients is unaffected by splenectomy: evidence from three decades of follow-up in a high-income setting.

    Pinto, Valeria Maria / Gianesin, Barbara / Piel, Frédéric B / Longo, Filomena / Rigano, Paolo / Quota, Alessandra / Spadola, Vincenzo / Graziadei, Giovanna / Mazzi, Filippo / Cappellini, Maria Domenica / Maggio, Aurelio / Piga, Antonio / De Franceschi, Lucia / Forni, Gian Luca

    Haematologica

    2023  Volume 108, Issue 4, Page(s) 1158–1162

    MeSH term(s) Humans ; Follow-Up Studies ; Splenectomy/adverse effects ; Anemia, Sickle Cell/epidemiology ; Splenic Diseases ; Morbidity
    Language English
    Publishing date 2023-04-01
    Publishing country Italy
    Document type Letter ; Research Support, Non-U.S. Gov't
    ZDB-ID 2333-4
    ISSN 1592-8721 ; 0017-6567 ; 0390-6078
    ISSN (online) 1592-8721
    ISSN 0017-6567 ; 0390-6078
    DOI 10.3324/haematol.2022.280815
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