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1965 1
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Page 1
Management of pyruvate kinase deficiency in children and adults.
Grace RF, Barcellini W. Grace RF, et al. Blood. 2020 Sep 10;136(11):1241-1249. doi: 10.1182/blood.2019000945. Blood. 2020. PMID: 32702739 Free article. Review.
Pyruvate kinase deficiency (PKD) is an autosomal-recessive enzyme defect of the glycolytic pathway that causes congenital nonspherocytic hemolytic anemia. The diagnosis and management of patients with PKD can be challenging due to difficulties in the d …
Pyruvate kinase deficiency (PKD) is an autosomal-recessive enzyme defect of the glycolytic pathway that causes congenital nonspher …
Red Blood Cell Enzyme Disorders.
Grace RF, Glader B. Grace RF, et al. Pediatr Clin North Am. 2018 Jun;65(3):579-595. doi: 10.1016/j.pcl.2018.02.005. Pediatr Clin North Am. 2018. PMID: 29803284 Review.
The most common red blood cell enzyme disorders are characterized by hemolysis but with wide clinical variability. Glucose-6-phosphate dehydrogenase deficiency is the most common red cell enzyme disorder worldwide. Frequent clinical presentations include neonatal ja …
The most common red blood cell enzyme disorders are characterized by hemolysis but with wide clinical variability. Glucose-6-phosphat …
Mitapivat versus Placebo for Pyruvate Kinase Deficiency.
Al-Samkari H, Galactéros F, Glenthøj A, Rothman JA, Andres O, Grace RF, Morado-Arias M, Layton DM, Onodera K, Verhovsek M, Barcellini W, Chonat S, Judge MP, Zagadailov E, Xu R, Hawkins P, Beynon V, Gheuens S, van Beers EJ; ACTIVATE Investigators. Al-Samkari H, et al. N Engl J Med. 2022 Apr 14;386(15):1432-1442. doi: 10.1056/NEJMoa2116634. N Engl J Med. 2022. PMID: 35417638 Clinical Trial.
In a phase 2 study, mitapivat, an oral, first-in-class activator of erythrocyte pyruvate kinase, increased the hemoglobin level in patients with pyruvate kinase deficiency. METHODS: In this global, phase 3, randomized, placebo-controlled trial, we evaluated the effi …
In a phase 2 study, mitapivat, an oral, first-in-class activator of erythrocyte pyruvate kinase, increased the hemoglobin level in patients …
Congenital non-spherocytic hemolytic anemia.
Zipursky A, Rowland M, Peters JC, Israels LG. Zipursky A, et al. Can Med Assoc J. 1965 Nov 27;93(22):1141-6. Can Med Assoc J. 1965. PMID: 5320918 Free PMC article. Clinical Trial.
Mitapivat: A Review in Pyruvate Kinase Deficiency in Adults.
Zhuang-Yan A, Shirley M. Zhuang-Yan A, et al. Drugs. 2023 Nov;83(17):1613-1620. doi: 10.1007/s40265-023-01961-x. Epub 2023 Nov 22. Drugs. 2023. PMID: 37991635 Review.
In the single-arm phase III ACTIVATE-T trial in adults with PK deficiency who were regularly RBC transfused, a reduction in RBC transfusion burden was observed with mitapivat. In both trials, mitapivat improved other clinical parameters of haemolysis and patient-rep …
In the single-arm phase III ACTIVATE-T trial in adults with PK deficiency who were regularly RBC transfused, a reduction in RBC trans …
An evaluation of mitapivat for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency.
Song AB, Al-Samkari H. Song AB, et al. Expert Rev Hematol. 2022 Oct;15(10):875-885. doi: 10.1080/17474086.2022.2125865. Epub 2022 Sep 22. Expert Rev Hematol. 2022. PMID: 36124781
INTRODUCTION: Pyruvate kinase deficiency (PKD) is the most common cause of congenital nonspherocytic hemolytic anemia. Until recently, treatment had been limited to supportive management including red blood cell transfusions, splenectomy, and managemen …
INTRODUCTION: Pyruvate kinase deficiency (PKD) is the most common cause of congenital nonspherocytic hemolytic anemi …
The Pyruvate Kinase Deficiency Global Longitudinal (Peak) Registry: rationale and study design.
Grace RF, van Beers EJ, Vives Corrons JL, Glader B, Glenthøj A, Kanno H, Kuo KHM, Lander C, Layton DM, Pospíŝilová D, Viprakasit V, Li J, Yan Y, Boscoe AN, Bowden C, Bianchi P. Grace RF, et al. BMJ Open. 2023 Mar 23;13(3):e063605. doi: 10.1136/bmjopen-2022-063605. BMJ Open. 2023. PMID: 36958777 Free PMC article.
The registry objective is to foster an understanding of the longitudinal clinical implications of PK deficiency, including its natural history, treatments and outcomes, and variability in clinical care. ...Registry data will be published in peer-reviewed journal art …
The registry objective is to foster an understanding of the longitudinal clinical implications of PK deficiency, including its natura …
Mitapivat: A Quinolone Sulfonamide to Manage Hemolytic Anemia in Adults With Pyruvate Kinase Deficiency.
Wills J, Horenstein M, Kim A, Silva MA, Dima L. Wills J, et al. Am J Ther. 2023 Sep-Oct 01;30(5):e433-e438. doi: 10.1097/MJT.0000000000001663. Am J Ther. 2023. PMID: 37713687 Clinical Trial.
The half-life of elimination is 3-5 hours, with 73% bioavailability, 98% plasma protein binding, and a median duration of response of 7 months. CLINICAL TRIALS: Mitapivat has been investigated through various clinical trials for different therapeutic indications. .. …
The half-life of elimination is 3-5 hours, with 73% bioavailability, 98% plasma protein binding, and a median duration of response of 7 mont …
Health-related quality of life and fatigue in children and adults with pyruvate kinase deficiency.
Al-Samkari H, van Beers EJ, Morton DH, Eber SW, Chonat S, Kuo KHM, Kollmar N, Wang H, Breakey VR, Sheth S, Sharma M, Forbes PW, Klaassen RJ, Grace RF. Al-Samkari H, et al. Blood Adv. 2022 Mar 22;6(6):1844-1853. doi: 10.1182/bloodadvances.2021004675. Blood Adv. 2022. PMID: 34470054 Free PMC article.
Pyruvate kinase deficiency (PKD) is the most common cause of congenital nonspherocytic hemolytic anemia. Although recognition of the disease spectrum has recently expanded, data describing its impact on health-related quality of life (HRQoL) are limite …
Pyruvate kinase deficiency (PKD) is the most common cause of congenital nonspherocytic hemolytic anemia. Althoug …
Mitapivat in adult patients with pyruvate kinase deficiency receiving regular transfusions (ACTIVATE-T): a multicentre, open-label, single-arm, phase 3 trial.
Glenthøj A, van Beers EJ, Al-Samkari H, Viprakasit V, Kuo KHM, Galactéros F, Chonat S, Porter J, Zagadailov E, Xu R, Oluyadi A, Hawkins P, Gheuens S, Beynon V, Barcellini W; ACTIVATE-T investigators. Glenthøj A, et al. Lancet Haematol. 2022 Oct;9(10):e724-e732. doi: 10.1016/S2352-3026(22)00214-9. Epub 2022 Aug 18. Lancet Haematol. 2022. PMID: 35988546 Clinical Trial.
Eligible participants were adults (aged 18 years) with a clinical laboratory confirmation of pyruvate kinase deficiency receiving regular transfusions (at least six episodes in the previous year). ...Efficacy and safety were assessed in all participants who received at lea …
Eligible participants were adults (aged 18 years) with a clinical laboratory confirmation of pyruvate kinase deficiency receiving reg …
38 results