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1.

CRISPR-Cas9 Gene Editing for Sickle Cell Disease and ß-Thalassemia.

Frangoul, Haydar; Altshuler, David; Cappellini, M Domenica; Chen, Yi-Shan; Domm, Jennifer; Eustace, Brenda K; Foell, Juergen; de la Fuente, Josu; Grupp, Stephan; Handgretinger, Rupert; Ho, Tony W; Kattamis, Antonis; Kernytsky, Andrew; Lekstrom-Himes, Julie; Li, Amanda M; Locatelli, Franco; Mapara, Markus Y; de Montalembert, Mariane; Rondelli, Damiano; Sharma, Akshay; Sheth, Sujit; Soni, Sandeep; Steinberg, Martin H; Wall, Donna; Yen, Angela; Corbacioglu, Selim.

N Engl J Med ; 384(3): 252-260, 2021 01 21.

Artículo en Inglés | MEDLINE | ID: mdl-33283989

2.

A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent ß-Thalassemia.

Cappellini, M Domenica; Viprakasit, Vip; Taher, Ali T; Georgiev, Pencho; Kuo, Kevin H M; Coates, Thomas; Voskaridou, Ersi; Liew, Hong-Keng; Pazgal-Kobrowski, Idit; Forni, G L; Perrotta, Silverio; Khelif, Abderrahim; Lal, Ashutosh; Kattamis, Antonis; Vlachaki, Efthymia; Origa, Raffaella; Aydinok, Yesim; Bejaoui, Mohamed; Ho, P Joy; Chew, Lee-Ping; Bee, Ping-Chong; Lim, Soo-Min; Lu, Meng-Yao; Tantiworawit, Adisak; Ganeva, Penka; Gercheva, Liana; Shah, Farrukh; Neufeld, Ellis J; Thompson, Alexis; Laadem, Abderrahmane; Shetty, Jeevan K; Zou, Jun; Zhang, Jennie; Miteva, Dimana; Zinger, Tatiana; Linde, Peter G; Sherman, Matthew L; Hermine, Olivier; Porter, John; Piga, Antonio.

N Engl J Med ; 382(13): 1219-1231, 2020 03 26.

Artículo en Inglés | MEDLINE | ID: mdl-32212518

3.

ß-Thalassemias. Reply.

Taher, Ali T; Musallam, Khaled M; Cappellini, M Domenica.

N Engl J Med ; 384(22): 2166, 2021 06 03.

Artículo en Inglés | MEDLINE | ID: mdl-34077657

4.

ß-Thalassemias.

Taher, Ali T; Musallam, Khaled M; Cappellini, M Domenica.

N Engl J Med ; 384(8): 727-743, 2021 02 25.

Artículo en Inglés | MEDLINE | ID: mdl-33626255

5.

Effects of deferasirox-deferoxamine on myocardial and liver iron in patients with severe transfusional iron overload.

Aydinok, Yesim; Kattamis, Antonis; Cappellini, M Domenica; El-Beshlawy, Amal; Origa, Raffaella; Elalfy, Mohsen; Kilinç, Yurdanur; Perrotta, Silverio; Karakas, Zeynep; Viprakasit, Vip; Habr, Dany; Constantinovici, Niculae; Shen, Junwu; Porter, John B.

Blood ; 125(25): 3868-77, 2015 Jun 18.

Artículo en Inglés | MEDLINE | ID: mdl-25934475

6.

Optimising iron chelation therapy with deferasirox for non-transfusion-dependent thalassaemia patients: 1-year results from the THETIS study.

Taher, Ali T; Cappellini, M Domenica; Aydinok, Yesim; Porter, John B; Karakas, Zeynep; Viprakasit, Vip; Siritanaratkul, Noppadol; Kattamis, Antonis; Wang, Candace; Zhu, Zewen; Joaquin, Victor; Uwamahoro, Marie José; Lai, Yong-Rong.

Blood Cells Mol Dis ; 57: 23-9, 2016 Mar.

Artículo en Inglés | MEDLINE | ID: mdl-26852651

7.

Approaching low liver iron burden in chelated patients with non-transfusion-dependent thalassemia: the safety profile of deferasirox.

Taher, Ali T; Porter, John B; Viprakasit, Vip; Kattamis, Antonis; Chuncharunee, Suporn; Sutcharitchan, Pranee; Siritanaratkul, Noppadol; Origa, Raffaella; Karakas, Zeynep; Habr, Dany; Zhu, Zewen; Cappellini, M Domenica.

Eur J Haematol ; 92(6): 521-6, 2014 Jun.

Artículo en Inglés | MEDLINE | ID: mdl-24460655

8.

Αlpha-thalassemia: A practical overview.

Musallam, Khaled M; Cappellini, M Domenica; Coates, Thomas D; Kuo, Kevin H M; Al-Samkari, Hanny; Sheth, Sujit; Viprakasit, Vip; Taher, Ali T.

Blood Rev ; 64: 101165, 2024 Mar.

Artículo en Inglés | MEDLINE | ID: mdl-38182489

9.

Iron chelation with deferasirox in adult and pediatric patients with thalassemia major: efficacy and safety during 5 years' follow-up.

Cappellini, M Domenica; Bejaoui, Mohamed; Agaoglu, Leyla; Canatan, Duran; Capra, Marcello; Cohen, Alan; Drelichman, Guillermo; Economou, Marina; Fattoum, Slaheddine; Kattamis, Antonis; Kilinc, Yurdanur; Perrotta, Silverio; Piga, Antonio; Porter, John B; Griffel, Louis; Dong, Victor; Clark, Joan; Aydinok, Yesim.

Blood ; 118(4): 884-93, 2011 Jul 28.

Artículo en Inglés | MEDLINE | ID: mdl-21628399

10.

Deferasirox effectively reduces iron overload in non-transfusion-dependent thalassemia (NTDT) patients: 1-year extension results from the THALASSA study.

Taher, Ali T; Porter, John B; Viprakasit, Vip; Kattamis, Antonis; Chuncharunee, Suporn; Sutcharitchan, Pranee; Siritanaratkul, Noppadol; Galanello, Renzo; Karakas, Zeynep; Lawniczek, Tomasz; Habr, Dany; Ros, Jacqueline; Zhu, Zewen; Cappellini, M Domenica.

Ann Hematol ; 92(11): 1485-93, 2013 Nov.

Artículo en Inglés | MEDLINE | ID: mdl-23775581

11.

Treating iron overload in patients with non-transfusion-dependent thalassemia.

Taher, Ali T; Viprakasit, Vip; Musallam, Khaled M; Cappellini, M Domenica.

Am J Hematol ; 88(5): 409-15, 2013 May.

Artículo en Inglés | MEDLINE | ID: mdl-23475638

12.

Deferasirox demonstrates a dose-dependent reduction in liver iron concentration and consistent efficacy across subgroups of non-transfusion-dependent thalassemia patients.

Taher, Ali T; Porter, John B; Viprakasit, Vip; Kattamis, Antonis; Chuncharunee, Suporn; Sutcharitchan, Pranee; Siritanaratkul, Noppadol; Galanello, Renzo; Karakas, Zeynep; Lawniczek, Tomasz; Habr, Dany; Ros, Jacqueline; Zhang, Yiyun; Cappellini, M Domenica.

Am J Hematol ; 88(6): 503-6, 2013 Jun.

Artículo en Inglés | MEDLINE | ID: mdl-23553596

13.

Improvement in liver pathology of patients with ß-thalassemia treated with deferasirox for at least 3 years.

Deugnier, Yves; Turlin, Bruno; Ropert, Martine; Cappellini, M Domenica; Porter, John B; Giannone, Vanessa; Zhang, Yiyun; Griffel, Louis; Brissot, Pierre.

Gastroenterology ; 141(4): 1202-11, 1211.e1-3, 2011 Oct.

Artículo en Inglés | MEDLINE | ID: mdl-21741344

14.

Hematologic malignancies in thalassemia: Adding new cases to the repertoire.

Halawi, Racha; Beydoun, Hassan; Cappellini, M Domenica; Ferla, Valeria; Taher, Ali.

Am J Hematol ; 92(5): E68-E70, 2017 May.

Artículo en Inglés | MEDLINE | ID: mdl-28195422

15.

Oral ferroportin inhibitor vamifeport for improving iron homeostasis and erythropoiesis in ß-thalassemia: current evidence and future clinical development.

Porter, John; Taher, Ali; Viprakasit, Vip; Kattamis, Antonis; Coates, Thomas D; Garbowski, Maciej; Dürrenberger, Franz; Manolova, Vania; Richard, Frank; Cappellini, M Domenica.

Expert Rev Hematol ; 14(7): 633-644, 2021 07.

Artículo en Inglés | MEDLINE | ID: mdl-34324404

16.

Decreased differentiation of erythroid cells exacerbates ineffective erythropoiesis in beta-thalassemia.

Libani, Ilaria V; Guy, Ella C; Melchiori, Luca; Schiro, Raffaella; Ramos, Pedro; Breda, Laura; Scholzen, Thomas; Chadburn, Amy; Liu, YiFang; Kernbach, Margrit; Baron-Lühr, Bettina; Porotto, Matteo; de Sousa, Maria; Rachmilewitz, Eliezer A; Hood, John D; Cappellini, M Domenica; Giardina, Patricia J; Grady, Robert W; Gerdes, Johannes; Rivella, Stefano.

Blood ; 112(3): 875-85, 2008 Aug 01.

Artículo en Inglés | MEDLINE | ID: mdl-18480424

17.

The autophagy-activating kinase ULK1 mediates clearance of free α-globin in ß-thalassemia.

Lechauve, Christophe; Keith, Julia; Khandros, Eugene; Fowler, Stephanie; Mayberry, Kalin; Freiwan, Abdullah; Thom, Christopher S; Delbini, Paola; Romero, Emilio Boada; Zhang, Jingjing; Motta, Irene; Tillman, Heather; Cappellini, M Domenica; Kundu, Mondira; Weiss, Mitchell J.

Sci Transl Med ; 11(506)2019 08 21.

Artículo en Inglés | MEDLINE | ID: mdl-31434755

18.

New therapeutic targets in transfusion-dependent and -independent thalassemia.

Cappellini, M Domenica; Motta, Irene.

Hematology Am Soc Hematol Educ Program ; 2017(1): 278-283, 2017 12 08.

Artículo en Inglés | MEDLINE | ID: mdl-29222267

19.

New insights into transfusion-related iron toxicity: Implications for the oncologist.

Porter, John B; de Witte, Theo; Cappellini, M Domenica; Gattermann, Norbert.

Crit Rev Oncol Hematol ; 99: 261-71, 2016 Mar.

Artículo en Inglés | MEDLINE | ID: mdl-26806144

20.

Development of a new disease severity scoring system for patients with non-transfusion-dependent thalassemia.

Cappellini, M Domenica; Porter, John B; Musallam, Khaled M; Kattamis, Antonis; Viprakasit, Vip; Galanello, Renzo; Taher, Ali T.

Eur J Intern Med ; 28: 91-6, 2016 Mar.

Artículo en Inglés | MEDLINE | ID: mdl-26545830

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