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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.

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

3.

Acquired severe aplastic anaemia: how medical therapy evolved in the 20th and 21st centuries.

Scheinberg, Phillip.

Br J Haematol ; 194(6): 954-969, 2021 09.

Artículo en Inglés | MEDLINE | ID: mdl-33855695

4.

Eltrombopag preferentially expands haematopoietic multipotent progenitors in human aplastic anaemia.

Quintino de Oliveira, Bruno; Catto, Luiz F B; Santana, Bárbara A A; Tellechea, M Florencia; Scheucher, Priscila S; Scheinberg, Phillip; Calado, Rodrigo T.

Br J Haematol ; 193(2): 410-414, 2021 04.

Artículo en Inglés | MEDLINE | ID: mdl-33216370

5.

Treatment optimization and genomic outcomes in refractory severe aplastic anemia treated with eltrombopag.

Winkler, Thomas; Fan, Xing; Cooper, James; Desmond, Ronan; Young, David J; Townsley, Danielle M; Scheinberg, Phillip; Grasmeder, Sophia; Larochelle, Andre; Desierto, Marie; Valdez, Janet; Lotter, Jennifer; Wu, Colin; Shalhoub, Ruba N; Calvo, Katherine R; Young, Neal S; Dunbar, Cynthia E.

Blood ; 133(24): 2575-2585, 2019 06 13.

Artículo en Inglés | MEDLINE | ID: mdl-30992268

6.

A New Standard Immunosuppression Regimen in Severe Aplastic Anemia.

Scheinberg, Phillip.

N Engl J Med ; 386(1): 89-90, 2022 01 06.

Artículo en Inglés | MEDLINE | ID: mdl-34986290

7.

Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia.

Townsley, Danielle M; Scheinberg, Phillip; Winkler, Thomas; Desmond, Ronan; Dumitriu, Bogdan; Rios, Olga; Weinstein, Barbara; Valdez, Janet; Lotter, Jennifer; Feng, Xingmin; Desierto, Marie; Leuva, Harshraj; Bevans, Margaret; Wu, Colin; Larochelle, Andre; Calvo, Katherine R; Dunbar, Cynthia E; Young, Neal S.

N Engl J Med ; 376(16): 1540-1550, 2017 04 20.

Artículo en Inglés | MEDLINE | ID: mdl-28423296

8.

Eltrombopag monotherapy can improve hematopoiesis in patients with low to intermediate risk-1 myelodysplastic syndrome.

Vicente, Alana; Patel, Bhavisha A; Gutierrez-Rodrigues, Fernanda; Groarke, Emma; Giudice, Valentina; Lotter, Jennifer; Feng, Xingmin; Kajigaya, Sachiko; Weinstein, Barbara; Barranta, Evette; Olnes, Matthew J; Parikh, Ankur R; Albitar, Maher; Wu, Colin O; Shalhoub, Ruba; Calvo, Katherine R; Townsley, Danielle M; Scheinberg, Phillip; Dunbar, Cynthia E; Young, Neal S; Winkler, Thomas.

Haematologica ; 105(12): 2785-2794, 2020 12 01.

Artículo en Inglés | MEDLINE | ID: mdl-33256377

9.

Danazol Treatment for Telomere Diseases.

Townsley, Danielle M; Dumitriu, Bogdan; Liu, Delong; Biancotto, Angélique; Weinstein, Barbara; Chen, Christina; Hardy, Nathan; Mihalek, Andrew D; Lingala, Shilpa; Kim, Yun Ju; Yao, Jianhua; Jones, Elizabeth; Gochuico, Bernadette R; Heller, Theo; Wu, Colin O; Calado, Rodrigo T; Scheinberg, Phillip; Young, Neal S.

N Engl J Med ; 374(20): 1922-31, 2016 May 19.

Artículo en Inglés | MEDLINE | ID: mdl-27192671

10.

Somatic Mutations and Clonal Hematopoiesis in Aplastic Anemia.

Yoshizato, Tetsuichi; Dumitriu, Bogdan; Hosokawa, Kohei; Makishima, Hideki; Yoshida, Kenichi; Townsley, Danielle; Sato-Otsubo, Aiko; Sato, Yusuke; Liu, Delong; Suzuki, Hiromichi; Wu, Colin O; Shiraishi, Yuichi; Clemente, Michael J; Kataoka, Keisuke; Shiozawa, Yusuke; Okuno, Yusuke; Chiba, Kenichi; Tanaka, Hiroko; Nagata, Yasunobu; Katagiri, Takamasa; Kon, Ayana; Sanada, Masashi; Scheinberg, Phillip; Miyano, Satoru; Maciejewski, Jaroslaw P; Nakao, Shinji; Young, Neal S; Ogawa, Seishi.

N Engl J Med ; 373(1): 35-47, 2015 Jul 02.

Artículo en Inglés | MEDLINE | ID: mdl-26132940

11.

Rabbit antithymocyte globulin dose does not affect response or survival as first-line therapy for acquired aplastic anemia: a multicenter retrospective study.

Clé, Diego V; Atta, Elias H; Dias, Danielle S P; Lima, Carlos B L; Bonduel, Mariana; Sciuccati, Gabriela; Medeiros, Larissa A; de Oliveira, Michel M; Blum Fonseca, Patricia B; Saad, Sara T O; Hamerschlak, Nelson; Salvino, Marco A; Garanito, Marlene P; Pazin-Filho, Antonio; Scheinberg, Phillip; Calado, Rodrigo T.

Ann Hematol ; 97(11): 2039-2046, 2018 Nov.

Artículo en Inglés | MEDLINE | ID: mdl-29978284

12.

Why do Tregs suddenly disappear in aplastic anemia?

Scheinberg, Phillip.

Blood ; 136(7): 779-780, 2020 08 13.

Artículo en Inglés | MEDLINE | ID: mdl-32790851

13.

A plasma microRNA signature as a biomarker for acquired aplastic anemia.

Hosokawa, Kohei; Kajigaya, Sachiko; Feng, Xingmin; Desierto, Marie J; Fernandez Ibanez, Maria Del Pilar; Rios, Olga; Weinstein, Barbara; Scheinberg, Phillip; Townsley, Danielle M; Young, Neal S.

Haematologica ; 102(1): 69-78, 2017 01.

Artículo en Inglés | MEDLINE | ID: mdl-27658437

14.

Predictors of early mortality after rabbit antithymocyte globulin as first-line treatment in severe aplastic anemia.

Atta, Elias H; Lima, Carlos B L; Dias, Danielle S P; Clé, Diego V; Bonduel, Mariana M; Sciuccati, Gabriela B; Medeiros, Larissa A; Oliveira, Michel M; Salvino, Marco A; Garanito, Marlene P; Blum Fonseca, Patricia B; Saad, Sara Teresinha O; Calado, Rodrigo T; Scheinberg, Phillip.

Ann Hematol ; 96(11): 1907-1914, 2017 Nov.

Artículo en Inglés | MEDLINE | ID: mdl-28815305

15.

Moderate-dose cyclophosphamide for severe aplastic anemia has significant toxicity and does not prevent relapse and clonal evolution.

Scheinberg, Phillip; Townsley, Danielle; Dumitriu, Bogdan; Scheinberg, Priscila; Weinstein, Barbara; Daphtary, Maithili; Rios, Olga; Wu, Colin O; Young, Neal S.

Blood ; 124(18): 2820-3, 2014 Oct 30.

Artículo en Inglés | MEDLINE | ID: mdl-25185712

16.

Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug.

Desmond, Ronan; Townsley, Danielle M; Dumitriu, Bogdan; Olnes, Matthew J; Scheinberg, Phillip; Bevans, Margaret; Parikh, Ankur R; Broder, Kinneret; Calvo, Katherine R; Wu, Colin O; Young, Neal S; Dunbar, Cynthia E.

Blood ; 123(12): 1818-25, 2014 Mar 20.

Artículo en Inglés | MEDLINE | ID: mdl-24345753

17.

In search for a haemoglobin threshold in myelodysplastic syndromes.

Scheinberg, Phillip.

Br J Haematol ; 189(5): 813-814, 2020 06.

Artículo en Inglés | MEDLINE | ID: mdl-32030730

18.

Eltrombopag and improved hematopoiesis in refractory aplastic anemia.

Olnes, Matthew J; Scheinberg, Phillip; Calvo, Katherine R; Desmond, Ronan; Tang, Yong; Dumitriu, Bogdan; Parikh, Ankur R; Soto, Susan; Biancotto, Angelique; Feng, Xingmin; Lozier, Jay; Wu, Colin O; Young, Neal S; Dunbar, Cynthia E.

N Engl J Med ; 367(1): 11-9, 2012 Jul 05.

Artículo en Inglés | MEDLINE | ID: mdl-22762314

19.

Long-term outcome of fludarabine-based reduced-intensity allogeneic hematopoietic cell transplantation for debilitating paroxysmal nocturnal hemoglobinuria.

Pantin, Jeremy; Tian, Xin; Geller, Nancy; Ramos, Catalina; Cook, Lisa; Cho, Elena; Scheinberg, Phillip; Vasu, Sumithira; Khuu, Hahn; Stroncek, David; Barrett, John; Young, Neal S; Donohue, Theresa; Childs, Richard W.

Biol Blood Marrow Transplant ; 20(9): 1435-9, 2014 Sep.

Artículo en Inglés | MEDLINE | ID: mdl-24844857

20.

Horse versus rabbit antithymocyte globulin in acquired aplastic anemia.

Scheinberg, Phillip; Nunez, Olga; Weinstein, Barbara; Scheinberg, Priscila; Biancotto, Angélique; Wu, Colin O; Young, Neal S.

N Engl J Med ; 365(5): 430-8, 2011 08 04.

Artículo en Inglés | MEDLINE | ID: mdl-21812672

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Oral Iptacopan Monotherapy in Paroxysmal Nocturnal Hemoglobinuria.

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

Acquired severe aplastic anaemia: how medical therapy evolved in the 20th and 21st centuries.

Eltrombopag preferentially expands haematopoietic multipotent progenitors in human aplastic anaemia.

Treatment optimization and genomic outcomes in refractory severe aplastic anemia treated with eltrombopag.

A New Standard Immunosuppression Regimen in Severe Aplastic Anemia.

Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia.

Eltrombopag monotherapy can improve hematopoiesis in patients with low to intermediate risk-1 myelodysplastic syndrome.

Danazol Treatment for Telomere Diseases.

Somatic Mutations and Clonal Hematopoiesis in Aplastic Anemia.

Rabbit antithymocyte globulin dose does not affect response or survival as first-line therapy for acquired aplastic anemia: a multicenter retrospective study.

Why do Tregs suddenly disappear in aplastic anemia?

A plasma microRNA signature as a biomarker for acquired aplastic anemia.

Predictors of early mortality after rabbit antithymocyte globulin as first-line treatment in severe aplastic anemia.

Moderate-dose cyclophosphamide for severe aplastic anemia has significant toxicity and does not prevent relapse and clonal evolution.

Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug.

In search for a haemoglobin threshold in myelodysplastic syndromes.

Eltrombopag and improved hematopoiesis in refractory aplastic anemia.

Long-term outcome of fludarabine-based reduced-intensity allogeneic hematopoietic cell transplantation for debilitating paroxysmal nocturnal hemoglobinuria.

Horse versus rabbit antithymocyte globulin in acquired aplastic anemia.