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1.

Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria.

Peffault de Latour, Régis; Röth, Alexander; Kulasekararaj, Austin G; Han, Bing; Scheinberg, Phillip; Maciejewski, Jaroslaw P; Ueda, Yasutaka; de Castro, Carlos M; Di Bona, Eros; Fu, Rong; Zhang, Li; Griffin, Morag; Langemeijer, Saskia M C; Panse, Jens; Schrezenmeier, Hubert; Barcellini, Wilma; Mauad, Vitor A Q; Schafhausen, Philippe; Tavitian, Suzanne; Beggiato, Eloise; Chew, Lee Ping; Gaya, Anna; Huang, Wei-Han; Jang, Jun Ho; Kitawaki, Toshio; Kutlar, Abdullah; Notaro, Rosario; Pullarkat, Vinod; Schubert, Jörg; Terriou, Louis; Uchiyama, Michihiro; Wong Lee Lee, Lily; Yap, Eng-Soo; Sicre de Fontbrune, Flore; Marano, Luana; Alashkar, Ferras; Gandhi, Shreyans; Trikha, Roochi; Yang, Chen; Liu, Hui; Kelly, Richard J; Höchsmann, Britta; Kerloeguen, Cécile; Banerjee, Partha; Levitch, Rafael; Kumar, Rakesh; Wang, Zhixin; Thorburn, Christine; Maitra, Samopriyo; Li, Shujie.

N Engl J Med ; 390(11): 994-1008, 2024 Mar 14.

Artículo en Inglés | MEDLINE | ID: mdl-38477987

2.

Treatment outcomes of complement protein C5 inhibition in 509 UK patients with paroxysmal nocturnal hemoglobinuria.

Kelly, Richard J; Holt, Matthew; Vidler, Jennifer; Arnold, Louise M; Large, Joanna; Forrest, Briony; Barnfield, Catherine; Pike, Alexandra; Griffin, Morag; Munir, Talha; Muus, Petra; Nagumantry, Sateesh K; Varghese, Abraham; Davies, John R; Trikha, Roochi; Kulasekararaj, Austin G; Mitchell, Lindsay; Gandhi, Shreyans.

Blood ; 143(12): 1157-1166, 2024 Mar 21.

Artículo en Inglés | MEDLINE | ID: mdl-38142401

3.

Alternative donor transplantation for severe aplastic anemia: a comparative study of the SAAWP EBMT.

Montoro, Juan; Eikema, Diderik-Jan; Tuffnell, Joe; Potter, Victoria; Kalwak, Krzysztof; Halkes, Constantijn Jm Jm; Kulagin, Alexander; Collin, Matthew; Wynn, Robert F; Robinson, Stephen; Nicholson, Emma; Sengeloev, Henrik; Halahleh, Khalid; Skorobogatova, Elena; Sanz, Jaime; Passweg, Jakob R; Mielke, Stephan; Ryhänen, Samppa Johannes; Carpenter, Ben; Gedde-Dahl, Tobias; Tholouli, Eleni; Fanin, Renato; Lewalle, Philippe; Kulasekararaj, Austin G; Risitano, Antonio Maria; Peffault de Latour, Régis.

Blood ; 2024 Apr 21.

Artículo en Inglés | MEDLINE | ID: mdl-38643511

4.

Consensus proposal for revised International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes.

Zeidan, Amer M; Platzbecker, Uwe; Bewersdorf, Jan Philipp; Stahl, Maximilian; Adès, Lionel; Borate, Uma; Bowen, David; Buckstein, Rena; Brunner, Andrew; Carraway, Hetty E; Daver, Naval; Díez-Campelo, Maria; de Witte, Theo; DeZern, Amy E; Efficace, Fabio; Garcia-Manero, Guillermo; Garcia, Jacqueline S; Germing, Ulrich; Giagounidis, Aristoteles; Griffiths, Elizabeth A; Hasserjian, Robert P; Hellström-Lindberg, Eva; Iastrebner, Marcelo; Komrokji, Rami; Kulasekararaj, Austin G; Malcovati, Luca; Miyazaki, Yasushi; Odenike, Olatoyosi; Santini, Valeria; Sanz, Guillermo; Scheinberg, Phillip; Stauder, Reinhard; van de Loosdrecht, Arjan A; Wei, Andrew H; Sekeres, Mikkael A; Fenaux, Pierre.

Blood ; 141(17): 2047-2061, 2023 04 27.

Artículo en Inglés | MEDLINE | ID: mdl-36724453

5.

Germ line DDX41 mutations define a unique subtype of myeloid neoplasms.

Makishima, Hideki; Saiki, Ryunosuke; Nannya, Yasuhito; Korotev, Sophia; Gurnari, Carmelo; Takeda, June; Momozawa, Yukihide; Best, Steve; Krishnamurthy, Pramila; Yoshizato, Tetsuichi; Atsuta, Yoshiko; Shiozawa, Yusuke; Iijima-Yamashita, Yuka; Yoshida, Kenichi; Shiraishi, Yuichi; Nagata, Yasunobu; Kakiuchi, Nobuyuki; Onizuka, Makoto; Chiba, Kenichi; Tanaka, Hiroko; Kon, Ayana; Ochi, Yotaro; Nakagawa, Masahiro M; Okuda, Rurika; Mori, Takuto; Yoda, Akinori; Itonaga, Hidehiro; Miyazaki, Yasushi; Sanada, Masashi; Ishikawa, Takayuki; Chiba, Shigeru; Tsurumi, Hisashi; Kasahara, Senji; Müller-Tidow, Carsten; Takaori-Kondo, Akifumi; Ohyashiki, Kazuma; Kiguchi, Toru; Matsuda, Fumihiko; Jansen, Joop H; Polprasert, Chantana; Blombery, Piers; Kamatani, Yoichiro; Miyano, Satoru; Malcovati, Luca; Haferlach, Torsten; Kubo, Michiaki; Cazzola, Mario; Kulasekararaj, Austin G; Godley, Lucy A; Maciejewski, Jaroslaw P.

Blood ; 141(5): 534-549, 2023 02 02.

Artículo en Inglés | MEDLINE | ID: mdl-36322930

6.

Current use of androgens in bone marrow failure disorders: a report from the Severe Aplastic Anemia Working Party of the European Society for Blood and Marrow Transplantation.

Pagliuca, Simona; Kulasekararaj, Austin G; Eikema, Dirk-Jan; Piepenbroek, Brian; Iftikhar, Raheel; Satti, Tariq Mahmood; Griffin, Morag; Laurino, Marica; Kupesiz, Alphan; Bertrand, Yves; Fattizzo, Bruno; Yakoub-Agha, Ibrahim; Aljurf, Mahmoud; Corti, Paola; Massaccesi, Erika; Lioure, Bruno; Calabuig, Marisa; Klammer, Matthias; Unal, Emel; Wu, Depei; Chevallier, Patrice; Forcade, Edouard; Snowden, John A; Ozdogu, Hakan; Risitano, Antonio; De Latour, Régis Peffault.

Haematologica ; 109(3): 765-776, 2024 Mar 01.

Artículo en Inglés | MEDLINE | ID: mdl-37199126

7.

Real-world experience of pegcetacoplan in paroxysmal nocturnal hemoglobinuria.

Griffin, Morag; Kelly, Richard; Brindel, Isabelle; Maafa, Lynda; Trikha, Roochi; Muus, Petra; Munir, Talha; Varghese, Abraham Mullasseril; Mitchell, Lindsay; Nagumantry, Sateesh; Gandhi, Shreyans; Pike, Alex; Kulasekararaj, Austin G; Peffault de Latour, Regis.

Am J Hematol ; 99(5): 816-823, 2024 May.

Artículo en Inglés | MEDLINE | ID: mdl-38348608

8.

Phase 2 study of danicopan in patients with paroxysmal nocturnal hemoglobinuria with an inadequate response to eculizumab.

Kulasekararaj, Austin G; Risitano, Antonio M; Maciejewski, Jaroslaw P; Notaro, Rosario; Browett, Peter; Lee, Jong Wook; Huang, Mingjun; Geffner, Michael; Brodsky, Robert A.

Blood ; 138(20): 1928-1938, 2021 11 18.

Artículo en Inglés | MEDLINE | ID: mdl-34314483

9.

Paroxysmal nocturnal hemoglobinuria: Where are we going.

Kulasekararaj, Austin G; Lazana, Ioanna.

Am J Hematol ; 98 Suppl 4: S33-S43, 2023 05.

Artículo en Inglés | MEDLINE | ID: mdl-36794458

10.

VEXAS syndrome (vacuoles, E1 enzyme, X-linked, autoinflammatory, somatic) for the dermatologist.

Sterling, David; Duncan, Mhairi E; Philippidou, Marianna; Salisbury, Jonathan R; Kulasekararaj, Austin G; Basu, Tanya N.

J Am Acad Dermatol ; 89(6): 1209-1214, 2023 12.

Artículo en Inglés | MEDLINE | ID: mdl-35121074

11.

Concomitant Immunosuppressive Therapy and Eculizumab Use in Patients with Paroxysmal Nocturnal Hemoglobinuria: An International PNH Registry Analysis.

Hill, Anita; de Latour, Régis Peffault; Kulasekararaj, Austin G; Griffin, Morag; Brodsky, Robert A; Maciejewski, Jaroslaw P; Marantz, Jing L; Gustovic, Philippe; Schrezenmeier, Hubert.

Acta Haematol ; 146(1): 1-13, 2023.

Artículo en Inglés | MEDLINE | ID: mdl-36108594

12.

The Effect of Respiratory Viral Infections on Breakthrough Hemolysis in Patients with Paroxysmal Nocturnal Hemoglobinuria.

Lazana, Ioanna; Apap Mangion, Sean; Babiker, Selma; Large, Joanna; Trikha, Roochi; Zuckerman, Mark; Gandhi, Shreyans; Kulasekararaj, Austin G.

Int J Mol Sci ; 24(11)2023 May 27.

Artículo en Inglés | MEDLINE | ID: mdl-37298309

13.

Long-term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2-year results from two pivotal phase 3 studies.

Kulasekararaj, Austin G; Griffin, Morag; Langemeijer, Saskia; Usuki, Kensuke; Kulagin, Alexander; Ogawa, Masayo; Yu, Ji; Mujeebuddin, Arshad; Nishimura, Jun-Ichi; Lee, Jong Wook; Peffault de Latour, Régis.

Eur J Haematol ; 109(3): 205-214, 2022 Sep.

Artículo en Inglés | MEDLINE | ID: mdl-35502600

14.

Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study.

Kulasekararaj, Austin G; Hill, Anita; Rottinghaus, Scott T; Langemeijer, Saskia; Wells, Richard; Gonzalez-Fernandez, F Ataulfo; Gaya, Anna; Lee, Jong Wook; Gutierrez, Emilio Ojeda; Piatek, Caroline I; Szer, Jeff; Risitano, Antonio; Nakao, Shinji; Bachman, Eric; Shafner, Lori; Damokosh, Andrew I; Ortiz, Stephan; Röth, Alexander; Peffault de Latour, Regis.

Blood ; 133(6): 540-549, 2019 02 07.

Artículo en Inglés | MEDLINE | ID: mdl-30510079

15.

Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria

Risitano, Antonio M; Kulasekararaj, Austin G; Lee, Jong Wook; Maciejewski, Jaroslaw P; Notaro, Rosario; Brodsky, Robert; Huang, Mingjun; Geffner, Michael; Browett, Peter.

Haematologica ; 106(12): 3188-3197, 2021 12 01.

Artículo en Inglés | MEDLINE | ID: mdl-33121236

16.

Characterization of breakthrough hemolysis events observed in the phase 3 randomized studies of ravulizumab versus eculizumab in adults with paroxysmal nocturnal hemoglobinuria.

Brodsky, Robert A; Peffault de Latour, Régis; Rottinghaus, Scott T; Röth, Alexander; Risitano, Antonio M; Weitz, Ilene C; Hillmen, Peter; Maciejewski, Jaroslaw P; Szer, Jeff; Lee, Jong Wook; Kulasekararaj, Austin G; Volles, Lori; Damokosh, Andrew I; Ortiz, Stephan; Shafner, Lori; Liu, Peng; Hill, Anita; Schrezenmeier, Hubert.

Haematologica ; 106(1): 230-237, 2021 01 01.

Artículo en Inglés | MEDLINE | ID: mdl-31949012

17.

Association between red blood cell transfusion dependence and burden in patients with myelodysplastic syndromes: A systematic literature review and meta-analysis.

Braga Lemos, Mayra; Rodrigues, Sarah Ramalho; Schroeder, Thomas; Kulasekararaj, Austin G; Matos, Joana E; Tang, Derek.

Eur J Haematol ; 107(1): 3-23, 2021 Jul.

Artículo en Inglés | MEDLINE | ID: mdl-33715214

18.

One-year outcomes from a phase 3 randomized trial of ravulizumab in adults with paroxysmal nocturnal hemoglobinuria who received prior eculizumab.

Kulasekararaj, Austin G; Hill, Anita; Langemeijer, Saskia; Wells, Richard; González Fernández, F Ataúlfo; Gaya, Anna; Ojeda Gutierrez, Emilio; Piatek, Caroline I; Mitchell, Lindsay; Usuki, Kensuke; Bosi, Alberto; Brodsky, Robert A; Ogawa, Masayo; Yu, Ji; Ortiz, Stephan; Röth, Alexander; Lee, Jong Wook; Peffault de Latour, Régis.

Eur J Haematol ; 106(3): 389-397, 2021 Mar.

Artículo en Inglés | MEDLINE | ID: mdl-33301613

19.

Pharmacokinetic and pharmacodynamic effects of ravulizumab and eculizumab on complement component 5 in adults with paroxysmal nocturnal haemoglobinuria: results of two phase 3 randomised, multicentre studies.

Peffault de Latour, Régis; Brodsky, Robert A; Ortiz, Stephan; Risitano, Antonio M; Jang, Jun H; Hillmen, Peter; Kulagin, Alexander D; Kulasekararaj, Austin G; Rottinghaus, Scott T; Aguzzi, Rasha; Gao, Xiang; Wells, Richard A; Szer, Jeff.

Br J Haematol ; 191(3): 476-485, 2020 11.

Artículo en Inglés | MEDLINE | ID: mdl-32449174

20.

AI for AA: machine learning makes an entry.

Kulasekararaj, Austin G.

Blood ; 141(17): 2040-2042, 2023 04 27.

Artículo en Inglés | MEDLINE | ID: mdl-37103950

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