Literature DB >> 34694225

Advancing the care of β-thalassaemia patients with novel therapies.

Rayan Bou-Fakhredin1, Irene Motta2, Maria Domenica Cappellini1,2.   

Abstract

The β-thalassaemias are a group of inherited disorders of haemoglobin synthesis characterised by chronic anaemia of varying severity. Currently available conventional therapies in thalassaemia have many challenges and limitations. A better understanding of the pathology of β-thalassaemia has led to the development of new treatment options, most of which are currently in clinical trials. These could have the potential of reducing red blood cell transfusion burden, raising haemoglobin levels, and improving patients' overall quality of life. In this review, we will provide an overview of the novel therapeutic approaches that are currently under development to advance the care of β-thalassaemia patients.

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Year:  2021        PMID： 34694225      PMCID： PMC8796844          DOI： 10.2450/2021.0265-21

Source DB:  PubMed          Journal:  Blood Transfus        ISSN： 1723-2007            Impact factor:   3.443


  82 in total

1.  Human fetal hemoglobin expression is regulated by the developmental stage-specific repressor BCL11A.

Authors:  Vijay G Sankaran; Tobias F Menne; Jian Xu; Thomas E Akie; Guillaume Lettre; Ben Van Handel; Hanna K A Mikkola; Joel N Hirschhorn; Alan B Cantor; Stuart H Orkin
Journal:  Science       Date:  2008-12-04       Impact factor: 47.728

2.  Hematopoietic stem cell transplantation for homozygous β-thalassemia and β-thalassemia/hemoglobin E patients from haploidentical donors.

Authors:  U Anurathapan; S Hongeng; S Pakakasama; N Sirachainan; D Songdej; A Chuansumrit; P Charoenkwan; A Jetsrisuparb; K Sanpakit; P Rujkijyanont; A Meekaewkunchorn; Y Lektrakul; P Iamsirirak; P Surapolchai; W Satayasai; S Sirireung; R Sruamsiri; P A Wahidiyat; A Ungkanont; S Issaragrisil; B S Andersson
Journal:  Bone Marrow Transplant       Date:  2016-02-15       Impact factor: 5.483

Review 3.  Gene Therapy for β-Hemoglobinopathies.

Authors:  Marina Cavazzana; Chiara Antoniani; Annarita Miccio
Journal:  Mol Ther       Date:  2017-04-01       Impact factor: 11.454

Review 4.  Luspatercept for β-thalassemia: beyond red blood cell transfusions.

Authors:  Ali T Taher; Maria Domenica Cappellini
Journal:  Expert Opin Biol Ther       Date:  2021-08-23       Impact factor: 4.388

Review 5.  The potential of gene therapy approaches for the treatment of hemoglobinopathies: achievements and challenges.

Authors:  Michael A Goodman; Punam Malik
Journal:  Ther Adv Hematol       Date:  2016-06-25

6.  Secondary solid cancer following hematopoietic cell transplantation in patients with thalassemia major.

Authors:  S Santarone; A Pepe; A Meloni; A Natale; L Pistoia; P Olioso; G Papalinetti; L Cuccia; A Spasiano; R Lisi; M Di Ianni; T Bonfini; P Accorsi; S Salvadori; F Papola; S Angelini; P Di Bartolomeo
Journal:  Bone Marrow Transplant       Date:  2017-10-09       Impact factor: 5.483

7.  Pretransplant immunosuppression followed by reduced-toxicity conditioning and stem cell transplantation in high-risk thalassemia: a safe approach to disease control.

Authors:  Usanarat Anurathapan; Samart Pakakasama; Piya Rujkijyanont; Nongnuch Sirachainan; Duantida Songdej; Ampaiwan Chuansumrit; Somtawin Sirireung; Pimlak Charoenkwan; Arunee Jetsrisuparb; Surapol Issaragrisil; Artit Ungkanont; Rosarin Sruamsiri; Supanart Srisala; Borje S Andersson; Suradej Hongeng
Journal:  Biol Blood Marrow Transplant       Date:  2013-05-03       Impact factor: 5.742

8.  The Oral Ferroportin Inhibitor VIT-2763 Improves Erythropoiesis without Interfering with Iron Chelation Therapy in a Mouse Model of β-Thalassemia.

Authors:  Naja Nyffenegger; Anna Flace; Cédric Doucerain; Franz Dürrenberger; Vania Manolova
Journal:  Int J Mol Sci       Date:  2021-01-16       Impact factor: 5.923

Review 9.  Selecting β-thalassemia Patients for Gene Therapy: A Decision-making Algorithm.

Authors:  Donatella Baronciani; Maddalena Casale; Lucia De Franceschi; Giovanna Graziadei; Filomena Longo; Raffaella Origa; Paolo Rigano; Valeria Pinto; Monia Marchetti; Antonia Gigante; Gian Luca Forni
Journal:  Hemasphere       Date:  2021-04-29

10.  Sotatercept, a novel transforming growth factor β ligand trap, improves anemia in β-thalassemia: a phase II, open-label, dose-finding study.

Authors:  Maria Domenica Cappellini; John Porter; Raffaella Origa; Gian Luca Forni; Ersi Voskaridou; Frédéric Galactéros; Ali T Taher; Jean-Benoît Arlet; Jean-Antoine Ribeil; Maciej Garbowski; Giovanna Graziadei; Chantal Brouzes; Michaela Semeraro; Abderrahmane Laadem; Dimana Miteva; Jun Zou; Victoria Sung; Tatiana Zinger; Kenneth M Attie; Olivier Hermine
Journal:  Haematologica       Date:  2018-10-18       Impact factor: 9.941
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