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Year Number of Results
1999 1
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2004 4
2005 4
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2007 4
2008 10
2009 5
2010 10
2011 14
2012 10
2013 26
2014 18
2015 24
2016 36
2017 34
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442 results

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Page 1
Caplacizumab Treatment for Acquired Thrombotic Thrombocytopenic Purpura.
Scully M, Cataland SR, Peyvandi F, Coppo P, Knöbl P, Kremer Hovinga JA, Metjian A, de la Rubia J, Pavenski K, Callewaert F, Biswas D, De Winter H, Zeldin RK; HERCULES Investigators. Scully M, et al. N Engl J Med. 2019 Jan 24;380(4):335-346. doi: 10.1056/NEJMoa1806311. Epub 2019 Jan 9. N Engl J Med. 2019. PMID: 30625070 Free article. Clinical Trial.
Caplacizumab, an anti-von Willebrand factor humanized, bivalent variable-domain-only immunoglobulin fragment, inhibits interaction between von Willebrand factor multimers and platelets. METHODS: In this double-blind, controlled trial, we randomly assigned 145 patients with …
Caplacizumab, an anti-von Willebrand factor humanized, bivalent variable-domain-only immunoglobulin fragment, inhibits interaction between v …
ISTH guidelines for treatment of thrombotic thrombocytopenic purpura.
Zheng XL, Vesely SK, Cataland SR, Coppo P, Geldziler B, Iorio A, Matsumoto M, Mustafa RA, Pai M, Rock G, Russell L, Tarawneh R, Valdes J, Peyvandi F. Zheng XL, et al. J Thromb Haemost. 2020 Oct;18(10):2496-2502. doi: 10.1111/jth.15010. Epub 2020 Sep 11. J Thromb Haemost. 2020. PMID: 32914526 Free PMC article.
BACKGROUND: Despite advances in treatment options for thrombotic thrombocytopenic purpura (TTP), there are still limited high quality data to inform clinicians regarding its appropriate treatment. METHODS: In June 2018, the ISTH formed a multidisciplinary guideline panel t …
BACKGROUND: Despite advances in treatment options for thrombotic thrombocytopenic purpura (TTP), there are still limited high quality data t …
Efanesoctocog Alfa Prophylaxis for Patients with Severe Hemophilia A.
von Drygalski A, Chowdary P, Kulkarni R, Susen S, Konkle BA, Oldenburg J, Matino D, Klamroth R, Weyand AC, Jimenez-Yuste V, Nogami K, Poloskey S, Winding B, Willemze A, Knobe K; XTEND-1 Trial Group. von Drygalski A, et al. N Engl J Med. 2023 Jan 26;388(4):310-318. doi: 10.1056/NEJMoa2209226. N Engl J Med. 2023. PMID: 36720133 Clinical Trial.
The efficacy, safety, and pharmacokinetics of efanesoctocog alfa for prophylaxis and treatment of bleeding episodes in previously treated patients with severe hemophilia A are unclear. METHODS: We conducted a phase 3 study involving patients 12 years of age or older with s …
The efficacy, safety, and pharmacokinetics of efanesoctocog alfa for prophylaxis and treatment of bleeding episodes in previously treated pa …
ISTH guidelines for the diagnosis of thrombotic thrombocytopenic purpura.
Zheng XL, Vesely SK, Cataland SR, Coppo P, Geldziler B, Iorio A, Matsumoto M, Mustafa RA, Pai M, Rock G, Russell L, Tarawneh R, Valdes J, Peyvandi F. Zheng XL, et al. J Thromb Haemost. 2020 Oct;18(10):2486-2495. doi: 10.1111/jth.15006. Epub 2020 Sep 11. J Thromb Haemost. 2020. PMID: 32914582 Free PMC article.
OBJECTIVE: The evidence-based guidelines of the International Society on Thrombosis and Haemostasis (ISTH) are intended to support patients, clinicians, and other health care professionals in their decisions about the initial diagnosis and management of acute TTP. METHODS: …
OBJECTIVE: The evidence-based guidelines of the International Society on Thrombosis and Haemostasis (ISTH) are intended to support patients, …
Caplacizumab for Acquired Thrombotic Thrombocytopenic Purpura.
Peyvandi F, Scully M, Kremer Hovinga JA, Cataland S, Knöbl P, Wu H, Artoni A, Westwood JP, Mansouri Taleghani M, Jilma B, Callewaert F, Ulrichts H, Duby C, Tersago D; TITAN Investigators. Peyvandi F, et al. N Engl J Med. 2016 Feb 11;374(6):511-22. doi: 10.1056/NEJMoa1505533. N Engl J Med. 2016. PMID: 26863353 Free article. Clinical Trial.
Daily plasma exchange and immunosuppressive therapies induce remission, but mortality and morbidity due to microthrombosis remain high. METHODS: Caplacizumab, an anti-von Willebrand factor humanized single-variable-domain immunoglobulin (Nanobody), inhibits the interaction …
Daily plasma exchange and immunosuppressive therapies induce remission, but mortality and morbidity due to microthrombosis remain high. M …
Valoctocogene Roxaparvovec Gene Therapy for Hemophilia A.
Ozelo MC, Mahlangu J, Pasi KJ, Giermasz A, Leavitt AD, Laffan M, Symington E, Quon DV, Wang JD, Peerlinck K, Pipe SW, Madan B, Key NS, Pierce GF, O'Mahony B, Kaczmarek R, Henshaw J, Lawal A, Jayaram K, Huang M, Yang X, Wong WY, Kim B; GENEr8-1 Trial Group. Ozelo MC, et al. N Engl J Med. 2022 Mar 17;386(11):1013-1025. doi: 10.1056/NEJMoa2113708. N Engl J Med. 2022. PMID: 35294811 Clinical Trial.
The efficacy and safety of the therapy were previously evaluated in men with severe hemophilia A in a phase 1-2 dose-escalation study. METHODS: We conducted an open-label, single-group, multicenter, phase 3 study to evaluate the efficacy and safety of valoctocogene roxapar …
The efficacy and safety of the therapy were previously evaluated in men with severe hemophilia A in a phase 1-2 dose-escalation study. ME …
Thrombotic thrombocytopenic purpura: early diagnosis and effective treatment in 2025.
Azoulay E, Veyradier A, Cataland S, Joly BS, Knoebl P, Zafrani L, Wendon J, Fakhouri F, Lämmle B, Pavenski K, Biebuyck N, Vanhoorelbeke K, Chaturvedi S, Pishko AM, Donadello K, Scully M, Matsumoto M, Peyvandi F, Mingot-Castellano ME, Zheng XL, Mariotte E, Benhamou Y, Coppo P. Azoulay E, et al. Intensive Care Med. 2025 Jul;51(7):1225-1239. doi: 10.1007/s00134-025-07981-3. Epub 2025 Jul 3. Intensive Care Med. 2025. PMID: 40608084 Review.
Efanesoctocog Alfa Prophylaxis for Children with Severe Hemophilia A.
Malec L, Peyvandi F, Chan AKC, Königs C, Zulfikar B, Yuan H, Simpson M, Álvarez Román MT, Carcao M, Staber JM, Dunn AL, Chou SC, d'Oiron R, Albisetti M, Demissie M, Santagostino E, Yarramaneni A, Wong N, Abad-Franch L, Gunawardena S, Fijnvandraat K; XTEND-Kids Trial Group. Malec L, et al. N Engl J Med. 2024 Jul 18;391(3):235-246. doi: 10.1056/NEJMoa2312611. N Engl J Med. 2024. PMID: 39018533 Clinical Trial.
Data on outcomes of efanesoctocog alfa treatment in children younger than 12 years of age with severe hemophilia A are limited. METHODS: We conducted a phase 3, open-label study involving previously treated patients younger than 12 years of age with severe hemophilia A. .. …
Data on outcomes of efanesoctocog alfa treatment in children younger than 12 years of age with severe hemophilia A are limited. METHODS …
Three-year outcomes of valoctocogene roxaparvovec gene therapy for hemophilia A.
Madan B, Ozelo MC, Raheja P, Symington E, Quon DV, Leavitt AD, Pipe SW, Lowe G, Kenet G, Reding MT, Mason J, Wang M, von Drygalski A, Klamroth R, Shapiro S, Chambost H, Dunn AL, Oldenburg J, Chou SC, Peyvandi F, Millar CM, Osmond D, Yu H, Dashiell-Aje E, Robinson TM, Mahlangu J. Madan B, et al. J Thromb Haemost. 2024 Jul;22(7):1880-1893. doi: 10.1016/j.jtha.2024.04.001. Epub 2024 Apr 12. J Thromb Haemost. 2024. PMID: 38614387 Free article. Clinical Trial.
OBJECTIVES: To present 3-year efficacy and safety in the multicenter, open-label, single-arm, phase 3 GENEr8-1 trial. METHODS: GENEr8-1 enrolled 134 adult males with severe hemophilia A who were receiving FVIII prophylaxis. ...
OBJECTIVES: To present 3-year efficacy and safety in the multicenter, open-label, single-arm, phase 3 GENEr8-1 trial. METHODS: GENEr8 …
Consensus on the standardization of terminology in thrombotic thrombocytopenic purpura and related thrombotic microangiopathies.
Scully M, Cataland S, Coppo P, de la Rubia J, Friedman KD, Kremer Hovinga J, Lämmle B, Matsumoto M, Pavenski K, Sadler E, Sarode R, Wu H; International Working Group for Thrombotic Thrombocytopenic Purpura. Scully M, et al. J Thromb Haemost. 2017 Feb;15(2):312-322. doi: 10.1111/jth.13571. Epub 2017 Jan 30. J Thromb Haemost. 2017. PMID: 27868334 Free article.
Objectives To present a consensus document from an International Working Group on TTP and associated thrombotic microangiopathies (TMAs). Methods The International Working Group has proposed definitions and terminology based on published information and consensus-based rec …
Objectives To present a consensus document from an International Working Group on TTP and associated thrombotic microangiopathies (TMAs). …
442 results