IMPROVEMENTS IN FATIGUE AND 6-MINUTE WALK TEST IN ADULTS WITH ALPHA- OR BETA-NON–TRANSFUSION-DEPENDENT THALASSEMIA: THE PHASE 3 ENERGIZE TRIAL OF MITAPIVAT

Autor: KH Kuo, H Al-Samkari, Y Aydinok, M Besser, S Gheuens, G Luna, A Glenthj, AS Goh, A Kattamis, SR Loggetto, KM Musallam, P Ricchi, E Salido-Fiérrez, S Sheth, V Viprakasit, MD Cappellini, AT Taher

Publikováno v: Hematology, Transfusion and Cell Therapy, Vol 46, Iss , Pp S34-S35 (2024)

Background: Thalassemia, a group of inherited disorders characterized by ineffective erythropoiesis and chronic hemolytic anemia, is associated with wide-ranging impacts on health-related quality of life (HRQoL), such as impaired physical functioning

Externí odkaz: https://doaj.org/article/50717b761b4d4b66bdbad276d68442b5

ENERGIZE AND ENERGIZE-T: TWO PHASE 3, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED STUDIES OF MITAPIVAT IN ADULTS WITH NON–TRANSFUSION-DEPENDENT OR TRANSFUSION-DEPENDENT ALPHA- OR BETA-THALASSEMIA

Autor: KH Kuo, DM Layton, H Al-Samkari, A Kattamis, S Sheth, A Taher, V Viprakasit, CX Chamberlain, L Czapla, S Gheuens, L Jiang, M Lynch, B Tong, K Uhlig, MD Cappellini

Publikováno v: Hematology, Transfusion and Cell Therapy, Vol 43, Iss , Pp S20- (2021)

Objectives: Thalassemias are characterized by imbalanced globin-chain production resulting in excess α- or β-globin precipitation, hemolytic anemia, and ineffective erythropoiesis. Adenosine triphosphate (ATP) levels are reduced in thalassemic red

Externí odkaz: https://doaj.org/article/9e31684761d54877af3244d42b7cdb74

Clinical and histological characterization of liver disease in patients with transfusion-dependent beta-thalassemia. A multicenter study of 117 cases

Autor: Prati D, Maggioni M, Milani S, Cerino M, Cianciulli P, Coggi G, Gl, Forni, Magnano C, Meo A, roberto gramignoli, Rebulla P, Fiorelli G, Md, Cappellini, Cooleycare Cooperative Group

Publikováno v: Europe PubMed Central
Karolinska Institutet
ResearcherID

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=dedup_wf_001::1eb27a34397680334689e75fdca1e8d4
http://europepmc.org/abstract/med/15477201

A glutamine metabolic switch supports erythropoiesis.

Autor: Lyu J; Center of Excellence for Leukemia Studies, Department of Pathology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Gu Z; Children's Medical Center Research Institute, Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA., Zhang Y; Center of Excellence for Leukemia Studies, Department of Pathology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Vu HS; Center of Excellence for Leukemia Studies, Department of Pathology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Lechauve C; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Cai F; Children's Medical Center Research Institute, Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA., Cao H; Center of Excellence for Leukemia Studies, Department of Pathology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Keith J; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Brancaleoni V; Unit of Medicine and Metabolic Disease, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, 20122 Milan, Italy., Granata F; Unit of Medicine and Metabolic Disease, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, 20122 Milan, Italy., Motta I; Unit of Medicine and Metabolic Disease, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, 20122 Milan, Italy.; Department of Clinical Sciences and Community Health, Università degli Studi di Milano, 20122 Milan, Italy., Cappellini MD; Unit of Medicine and Metabolic Disease, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, 20122 Milan, Italy., Huang LJ; Department of Cell Biology, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA., DeBerardinis RJ; Children's Medical Center Research Institute, Department of Pediatrics, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA.; Howard Hughes Medical Institute, University of Texas Southwestern Medical Center, Dallas, TX 75390, USA., Weiss MJ; Department of Hematology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Ni M; Division of Molecular Oncology, Department of Oncology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA., Xu J; Center of Excellence for Leukemia Studies, Department of Pathology, St. Jude Children's Research Hospital, Memphis, TN 38105, USA.

Publikováno v: Science (New York, N.Y.) [Science] 2024 Nov 15; Vol. 386 (6723), pp. eadh9215. Date of Electronic Publication: 2024 Nov 15.

Hypercoagulability in hemoglobinopathies: Decoding the thrombotic threat.

Autor: Bou-Fakhredin R; Department of Clinical Sciences and Community, University of Milan, Milan, Italy.; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, SC Medicina ad Indirizzo Metabolico, Milan, Italy., Cappellini MD; Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, SC Medicina ad Indirizzo Metabolico, Milan, Italy., Taher AT; Division of Hematology and Oncology, Department of Internal Medicine, American University of Beirut Medical Center, Beirut, Lebanon., De Franceschi L; Department of Medicine, University of Verona, and Azienda Ospedaliera Universitaria Verona, Verona, Italy.

Publikováno v: American journal of hematology [Am J Hematol] 2024 Oct 14. Date of Electronic Publication: 2024 Oct 14.