Results 31 to 40 of about 405 (131)

Rise of the planet of rare anemias: An update on emerging treatment strategies

Frontiers in Medicine, 2023
Therapeutic options for rare congenital (hemoglobinopathies, membrane and enzyme defects, congenital dyserythropoietic anemia) and acquired anemias [warm autoimmune hemolytic anemia (wAIHA), cold agglutinin disease CAD, paroxysmal nocturnal ...
Bruno Fattizzo, Bruno Fattizzo, Irene Motta, Irene Motta   +3 more
doaj   +1 more source

Mitapivat-Associated Rib Fracture in a Hemolytic Anemia Patient. [PDF]

Cureus
Hereditary hemolytic anemia associated with pyruvate kinase deficiency is a rare hematological disorder that affects the glycolic pathway within red blood cells. The standard of care includes splenectomy, transfusions, and hematopoietic stem cell transplantation.
Abouelkheer Y, Ladel L, Boxer D, Shafique S.   +3 more
europepmc   +3 more sources

Long-term mitapivat treatment is safe and efficacious in patients with sickle cell disease. [PDF]

Blood Red Cells Iron
In a phase 1 open-label study (ClinicalTrials.gov identifier: NCT04000165), mitapivat, a pyruvate kinase (PK) activator that is approved by the US Food and Drug Administration for treating anemia of PK deficiency, showed promise as a disease-modifying therapy for sickle cell disease (SCD).
Conrey A, Asomaning N, Frey I, Charles RP, Lovins D, Xu JZ, Mendez-Marti S, Le K, Kruah B, Li Q, Dunkelberger E, Cellmer T, Yates A, Wind-Rotolo M, Huston C, Jeffries N, Eaton WA, Thein SL.   +17 more
europepmc   +4 more sources

P1473: CLINICALLY RELEVANT HEMOGLOBIN RESPONSE IN ADULTS WITH PYRUVATE KINASE DEFICIENCY TREATED WITH MITAPIVAT – A SUB-ANALYSIS OF THE ACTIVATE TRIAL [PDF]

HemaSphere, 2023
Hanny Al-Samkari, Rachael F. Grace, Andreas Glenthøj, Wilma Barcellini, Madeleine Verhovsek, Jennifer A. Rothman, Marta Morado, Mark Layton, Oliver Andres, Frédéric Galactéros, Koichi Onodera, Satheesh Chonat, Rengyi Xu, Bryan Mcgee, Melissa Dibacco, Jaime Morales, Eduard van Beers   +16 more
doaj   +2 more sources

Efficacy and Safety of Mitapivat in Pyruvate Kinase Deficiency: A Systematic Review and Meta-analysis of Clinical Trials [PDF]

Indian Journal of Hematology and Blood Transfusion
Hazem Eslam, Mohamed E Haseeb, Mohamed Nasser   +2 more
exaly   +2 more sources

Right in time: Mitapivat for the treatment of anemia in α- and β-thalassemia

Cell Reports Medicine, 2022
Kuo and colleagues1 evaluated the safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion-dependent α-thalassemia or β-thalassemia. The high rate of hemoglobin response and good tolerability encourages further development in thalassemia.
Musallam, Khaled M., Taher, Ali T., Cappellini, Maria Domenica   +2 more
openaire   +2 more sources

P1424: ONE-YEAR FOLLOW-UP OF A PHASE 2 STUDY OF MITAPIVAT, AN ORAL PYRUVATE KINASE ACTIVATOR, FOR THE TREATMENT OF SICKLE CELL DISEASE [PDF]

HemaSphere, 2023
Myrthe van Dijk, Minke Rab, Brigitte van Oirschot, Jennifer Bos, Cleo Derichs, Anita Rijneveld, Marjon Cnossen, Erfan Nur, Bart Biemond, Marije Bartels, Judith Jans, Wouter van Solinge, Roger Schutgens, Richard Van Wijk, Eduard van Beers   +14 more
doaj   +2 more sources

Pyruvate Kinase Deficiency: Current Challenges and Future Prospects

Journal of Blood Medicine, 2022
Bruno Fattizzo,1,2 Francesca Cavallaro,1,2 Anna Paola Maria Luisa Marcello,1 Cristina Vercellati,1 Wilma Barcellini1 1Hematology Unit, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milan, Italy; 2Department of Oncology and Hemato-Oncology ...
Fattizzo B, Cavallaro F, Marcello APML, Vercellati C, Barcellini W   +4 more
doaj  

ENERGIZE: A GLOBAL PHASE 3 STUDY OF MITAPIVAT DEMONSTRATING EFFICACY AND SAFETY IN ADULTS WITH ALPHA- OR BETA-NON–TRANSFUSION-DEPENDENT THALASSEMIA

Hematology, Transfusion and Cell Therapy
Background: In thalassemia, ATP production in erythroid cells is too low to meet the demand of oxidative stress and ensuing cellular damage; this leads to ineffective erythropoiesis (IE) and chronic hemolytic anemia.
AT Taher, H Al-Samkari, Y Aydinok, M Besser, G Luna, S Gheuens, A Glenthj, AS Goh, A Kattamis, SR Loggetto, KM Musallam, P Ricchi, E Salido-Fiérrez, S Sheth, V Viprakasit, MD Cappellini, KH Kuo   +16 more
doaj   +1 more source

Red lines and green lights: Gene therapy for inherited erythroid disorders beyond the haemoglobinopathies

British Journal of Haematology, EarlyView.
Gene therapy is revolutionizing treatment paradigms for haemoglobinopathies, establishing a translational framework for disorders that impact red blood cell development. In their paper, Joshi et al. describe the preclinical and early clinical landscape of gene therapies for non‐haemoglobinopathy erythroid disorders and highlight common thematic ...
Gaurav Joshi, Nandakumar Natarajan, Kathryn E. Dickerson, Richard A. Voit   +3 more
wiley   +1 more source