米他匹伐，一种丙酮酸激酶激活剂，可减轻β地中海贫血小鼠的输血负担并减少铁过载。 - Suppr

Mitapivat, a pyruvate kinase activator, improves transfusion burden and reduces iron overload in β-thalassemic mice.

作者信息

Mattè Alessandro, Kosinski Penelope A, Federti Enrica, Dang Lenny, Recchiuti Antonio, Russo Roberta, Siciliano Angela, Riccardi Veronica, Janin Anne, Mucci Matteo, Leboeuf Christophe, Iolascon Achille, Brugnara Carlo, De Franceschi Lucia

机构信息

University of Verona and AOUI Verona, Verona.

Agios Pharmaceuticals, Inc., Cambridge, MA.

出版信息

Haematologica. 2023 Sep 1;108(9):2535-2541. doi: 10.3324/haematol.2022.282614.

DOI:10.3324/haematol.2022.282614

PMID:36794508

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10483370/

Abstract

摘要

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/dac2/10483370/f6a8d50dc8dd/1082535.fig1.jpg

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Mitapivat, a pyruvate kinase activator, improves transfusion burden and reduces iron overload in β-thalassemic mice.

Haematologica. 2023 Sep 1;108(9):2535-2541. doi: 10.3324/haematol.2022.282614.

The pyruvate kinase activator mitapivat reduces hemolysis and improves anemia in a β-thalassemia mouse model.

J Clin Invest. 2021 May 17;131(10). doi: 10.1172/JCI144206.

Mitapivat improves ineffective erythropoiesis and iron overload in adult patients with pyruvate kinase deficiency.

Blood Adv. 2024 May 28;8(10):2433-2441. doi: 10.1182/bloodadvances.2023011743.

Mitapivat for sickle cell disease and thalassemia.

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Cardiac complications in beta-thalassemia: From mice to men.

Exp Biol Med (Maywood). 2017 Jun;242(11):1126-1135. doi: 10.1177/1535370217708977. Epub 2017 May 9.

Combined therapy with desferrioxamine and deferiprone in thalassemic patients: effect on urinary iron excretion.

Haematologica. 2003 Dec;88(12):1423-5.

The influence of hemochromatosis mutations on iron overload of thalassemia major.

Haematologica. 1999 Sep;84(9):799-803.

A decisional algorithm to start iron chelation in patients with beta thalassemia.

Haematologica. 2014 Mar;99(3):e38-40. doi: 10.3324/haematol.2013.098202.

Non-transferrin-bound labile plasma iron and iron overload in sickle-cell disease: a comparative study between sickle-cell disease and beta-thalassemic patients.

Eur J Haematol. 2010 Jan 1;84(1):72-8. doi: 10.1111/j.1600-0609.2009.01342.x.

Iron-chelating therapy for transfusional iron overload.

N Engl J Med. 2011 Apr 14;364(15):1476; author reply 1477. doi: 10.1056/NEJMc1101838.

引用本文的文献

Luspatercept versus mitapivat for non-transfusion-dependent β-thalassemia: Dare to compare?

Hemasphere. 2025 Jul 13;9(7):e70165. doi: 10.1002/hem3.70165. eCollection 2025 Jul.

Long-term efficacy and safety of mitapivat in non-transfusion-dependent α- or β-thalassaemia: An open-label phase 2 study.

Br J Haematol. 2025 Jun;206(6):1764-1773. doi: 10.1111/bjh.20058. Epub 2025 May 20.

Mitapivat metabolically reprograms human β-thalassemic erythroblasts, increasing their responsiveness to oxidation.

Blood Adv. 2025 Jun 10;9(11):2818-2830. doi: 10.1182/bloodadvances.2024013591.

Pyruvate kinase activators: targeting red cell metabolism in thalassemia.

Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):114-120. doi: 10.1182/hematology.2023000468.

The mechanism of ferroptosis and its related diseases.

Mol Biomed. 2023 Oct 16;4(1):33. doi: 10.1186/s43556-023-00142-2.

Mitapivat reprograms the RBC metabolome and improves anemia in a mouse model of hereditary spherocytosis.

JCI Insight. 2023 Oct 23;8(20):e172656. doi: 10.1172/jci.insight.172656.

Therapeutic perspective for children and young adults living with thalassemia and sickle cell disease.

Eur J Pediatr. 2023 Jun;182(6):2509-2519. doi: 10.1007/s00431-023-04900-w. Epub 2023 Mar 31.

本文引用的文献

Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or β-thalassaemia: an open-label, multicentre, phase 2 study.

Lancet. 2022 Aug 13;400(10351):493-501. doi: 10.1016/S0140-6736(22)01337-X.

Betibeglogene Autotemcel Gene Therapy for Non-β/β Genotype β-Thalassemia.

N Engl J Med. 2022 Feb 3;386(5):415-427. doi: 10.1056/NEJMoa2113206. Epub 2021 Dec 11.

The pyruvate kinase activator mitapivat reduces hemolysis and improves anemia in a β-thalassemia mouse model.

J Clin Invest. 2021 May 17;131(10). doi: 10.1172/JCI144206.

β-Thalassemias.

N Engl J Med. 2021 Feb 25;384(8):727-743. doi: 10.1056/NEJMra2021838.

Methylation in pericytes after acute injury promotes chronic kidney disease.

J Clin Invest. 2020 Sep 1;130(9):4845-4857. doi: 10.1172/JCI135773.

Pathologic angiogenesis in the bone marrow of humanized sickle cell mice is reversed by blood transfusion.

Blood. 2020 Jun 4;135(23):2071-2084. doi: 10.1182/blood.2019002227.

Beta-thalassemia: renal complications and mechanisms: a narrative review.

Hematology. 2019 Dec;24(1):426-438. doi: 10.1080/16078454.2019.1599096.

Combination of Tmprss6- ASO and the iron chelator deferiprone improves erythropoiesis and reduces iron overload in a mouse model of beta-thalassemia intermedia.

Haematologica. 2016 Jan;101(1):e8-e11. doi: 10.3324/haematol.2015.133348. Epub 2015 Sep 24.

β-thalassemias: paradigmatic diseases for scientific discoveries and development of innovative therapies.

Haematologica. 2015 Apr;100(4):418-30. doi: 10.3324/haematol.2014.114827.

Lin28/let-7 axis regulates aerobic glycolysis and cancer progression via PDK1.

Nat Commun. 2014 Oct 10;5:5212. doi: 10.1038/ncomms6212.

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Mitapivat, a pyruvate kinase activator, improves transfusion burden and reduces iron overload in β-thalassemic mice.

作者信息

机构信息

University of Verona and AOUI Verona, Verona.

Agios Pharmaceuticals, Inc., Cambridge, MA.

出版信息

Haematologica. 2023 Sep 1;108(9):2535-2541. doi: 10.3324/haematol.2022.282614.

DOI:10.3324/haematol.2022.282614

PMID:36794508

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10483370/

Abstract

摘要

相似文献

Mitapivat, a pyruvate kinase activator, improves transfusion burden and reduces iron overload in β-thalassemic mice.

Haematologica. 2023 Sep 1;108(9):2535-2541. doi: 10.3324/haematol.2022.282614.

The pyruvate kinase activator mitapivat reduces hemolysis and improves anemia in a β-thalassemia mouse model.

J Clin Invest. 2021 May 17;131(10). doi: 10.1172/JCI144206.

Mitapivat improves ineffective erythropoiesis and iron overload in adult patients with pyruvate kinase deficiency.

Blood Adv. 2024 May 28;8(10):2433-2441. doi: 10.1182/bloodadvances.2023011743.

Mitapivat for sickle cell disease and thalassemia.

Drugs Today (Barc). 2023 Mar;59(3):125-134. doi: 10.1358/dot.2023.59.3.3521880.

Cardiac complications in beta-thalassemia: From mice to men.

Exp Biol Med (Maywood). 2017 Jun;242(11):1126-1135. doi: 10.1177/1535370217708977. Epub 2017 May 9.

Combined therapy with desferrioxamine and deferiprone in thalassemic patients: effect on urinary iron excretion.

Haematologica. 2003 Dec;88(12):1423-5.

The influence of hemochromatosis mutations on iron overload of thalassemia major.

Haematologica. 1999 Sep;84(9):799-803.

A decisional algorithm to start iron chelation in patients with beta thalassemia.

Haematologica. 2014 Mar;99(3):e38-40. doi: 10.3324/haematol.2013.098202.

Non-transferrin-bound labile plasma iron and iron overload in sickle-cell disease: a comparative study between sickle-cell disease and beta-thalassemic patients.

Eur J Haematol. 2010 Jan 1;84(1):72-8. doi: 10.1111/j.1600-0609.2009.01342.x.

Iron-chelating therapy for transfusional iron overload.

N Engl J Med. 2011 Apr 14;364(15):1476; author reply 1477. doi: 10.1056/NEJMc1101838.

引用本文的文献

Luspatercept versus mitapivat for non-transfusion-dependent β-thalassemia: Dare to compare?

Hemasphere. 2025 Jul 13;9(7):e70165. doi: 10.1002/hem3.70165. eCollection 2025 Jul.

Long-term efficacy and safety of mitapivat in non-transfusion-dependent α- or β-thalassaemia: An open-label phase 2 study.

Br J Haematol. 2025 Jun;206(6):1764-1773. doi: 10.1111/bjh.20058. Epub 2025 May 20.

Mitapivat metabolically reprograms human β-thalassemic erythroblasts, increasing their responsiveness to oxidation.

Blood Adv. 2025 Jun 10;9(11):2818-2830. doi: 10.1182/bloodadvances.2024013591.

Pyruvate kinase activators: targeting red cell metabolism in thalassemia.

Hematology Am Soc Hematol Educ Program. 2023 Dec 8;2023(1):114-120. doi: 10.1182/hematology.2023000468.

The mechanism of ferroptosis and its related diseases.

Mol Biomed. 2023 Oct 16;4(1):33. doi: 10.1186/s43556-023-00142-2.

Mitapivat reprograms the RBC metabolome and improves anemia in a mouse model of hereditary spherocytosis.

JCI Insight. 2023 Oct 23;8(20):e172656. doi: 10.1172/jci.insight.172656.

Therapeutic perspective for children and young adults living with thalassemia and sickle cell disease.

Eur J Pediatr. 2023 Jun;182(6):2509-2519. doi: 10.1007/s00431-023-04900-w. Epub 2023 Mar 31.

本文引用的文献

Safety and efficacy of mitapivat, an oral pyruvate kinase activator, in adults with non-transfusion dependent α-thalassaemia or β-thalassaemia: an open-label, multicentre, phase 2 study.

Lancet. 2022 Aug 13;400(10351):493-501. doi: 10.1016/S0140-6736(22)01337-X.

Betibeglogene Autotemcel Gene Therapy for Non-β/β Genotype β-Thalassemia.

N Engl J Med. 2022 Feb 3;386(5):415-427. doi: 10.1056/NEJMoa2113206. Epub 2021 Dec 11.

The pyruvate kinase activator mitapivat reduces hemolysis and improves anemia in a β-thalassemia mouse model.

J Clin Invest. 2021 May 17;131(10). doi: 10.1172/JCI144206.

β-Thalassemias.

N Engl J Med. 2021 Feb 25;384(8):727-743. doi: 10.1056/NEJMra2021838.

Methylation in pericytes after acute injury promotes chronic kidney disease.

J Clin Invest. 2020 Sep 1;130(9):4845-4857. doi: 10.1172/JCI135773.

Pathologic angiogenesis in the bone marrow of humanized sickle cell mice is reversed by blood transfusion.

Blood. 2020 Jun 4;135(23):2071-2084. doi: 10.1182/blood.2019002227.

Beta-thalassemia: renal complications and mechanisms: a narrative review.

Hematology. 2019 Dec;24(1):426-438. doi: 10.1080/16078454.2019.1599096.

Combination of Tmprss6- ASO and the iron chelator deferiprone improves erythropoiesis and reduces iron overload in a mouse model of beta-thalassemia intermedia.

Haematologica. 2016 Jan;101(1):e8-e11. doi: 10.3324/haematol.2015.133348. Epub 2015 Sep 24.

β-thalassemias: paradigmatic diseases for scientific discoveries and development of innovative therapies.

Haematologica. 2015 Apr;100(4):418-30. doi: 10.3324/haematol.2014.114827.

Lin28/let-7 axis regulates aerobic glycolysis and cancer progression via PDK1.

Nat Commun. 2014 Oct 10;5:5212. doi: 10.1038/ncomms6212.

Mitapivat, a pyruvate kinase activator, improves transfusion burden and reduces iron overload in β-thalassemic mice.

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Mitapivat, a pyruvate kinase activator, improves transfusion burden and reduces iron overload in β-thalassemic mice.

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