Literature DB >> 29018078

Novel approaches to hemophilia therapy: successes and challenges.

Valder R Arruda1,2,3, Bhavya S Doshi1, Benjamin J Samelson-Jones1,2.   

Abstract

New therapies for hemophilia A and hemophilia B will likely continue to change clinical practice. Ranging from extended half-life to nonfactor products and gene therapy, these innovative approaches have the potential to enhance the standard of care by decreasing infusion frequency to increase compliance, promoting prophylaxis, offering alternatives to inhibitor patients, and easing route of administration. Each category has intrinsic challenges that may limit the broader application of these promising therapies. To date, none specifically address the challenge of dispersing treatment to the developing world.
© 2017 by The American Society of Hematology.

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Year:  2017        PMID: 29018078      PMCID: PMC5813735          DOI: 10.1182/blood-2017-08-742312

Source DB:  PubMed          Journal:  Blood        ISSN: 0006-4971            Impact factor:   22.113


  63 in total

1.  Post hoc assessment of the immunogenicity of bioengineered factor VIIa demonstrates the use of preclinical tools.

Authors:  Kasper Lamberth; Stine Louise Reedtz-Runge; Jonathan Simon; Ksenia Klementyeva; Gouri Shankar Pandey; Søren Berg Padkjær; Véronique Pascal; Ileana R León; Charlotte Nini Gudme; Søren Buus; Zuben E Sauna
Journal:  Sci Transl Med       Date:  2017-01-11       Impact factor: 17.956

Review 2.  Half-life extension technologies for haemostatic agents.

Authors:  Pier Mannuccio Mannucci
Journal:  Thromb Haemost       Date:  2014-10-02       Impact factor: 5.249

Review 3.  Rethinking events in the haemostatic process: role of factor V and TFPI.

Authors:  R M Camire
Journal:  Haemophilia       Date:  2016-07       Impact factor: 4.287

Review 4.  Life in the shadow of a dominant partner: the FVIII-VWF association and its clinical implications for hemophilia A.

Authors:  Steven W Pipe; Robert R Montgomery; Kathleen P Pratt; Peter J Lenting; David Lillicrap
Journal:  Blood       Date:  2016-09-01       Impact factor: 22.113

5.  Concizumab, an anti-tissue factor pathway inhibitor antibody, induces increased thrombin generation in plasma from haemophilia patients and healthy subjects measured by the thrombin generation assay.

Authors:  E K Waters; J Sigh; U Friedrich; I Hilden; B B Sørensen
Journal:  Haemophilia       Date:  2017-06-08       Impact factor: 4.287

6.  The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy.

Authors:  Jonathan D Finn; Timothy C Nichols; Nikolaos Svoronos; Elizabeth P Merricks; Dwight A Bellenger; Shangshen Zhou; Paolo Simioni; Katherine A High; Valder R Arruda
Journal:  Blood       Date:  2012-08-23       Impact factor: 22.113

7.  Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A.

Authors:  Johnny Mahlangu; Jerry S Powell; Margaret V Ragni; Pratima Chowdary; Neil C Josephson; Ingrid Pabinger; Hideji Hanabusa; Naresh Gupta; Roshni Kulkarni; Patrick Fogarty; David Perry; Amy Shapiro; K John Pasi; Shashikant Apte; Ivan Nestorov; Haiyan Jiang; Shuanglian Li; Srividya Neelakantan; Lynda M Cristiano; Jaya Goyal; Jurg M Sommer; Jennifer A Dumont; Nigel Dodd; Karen Nugent; Gloria Vigliani; Alvin Luk; Aoife Brennan; Glenn F Pierce
Journal:  Blood       Date:  2013-11-13       Impact factor: 22.113

8.  Safety and pharmacokinetics of anti-TFPI antibody (concizumab) in healthy volunteers and patients with hemophilia: a randomized first human dose trial.

Authors:  P Chowdary; S Lethagen; U Friedrich; B Brand; C Hay; F Abdul Karim; R Klamroth; P Knoebl; M Laffan; J Mahlangu; W Miesbach; J Dalsgaard Nielsen; M Martín-Salces; P Angchaisuksiri
Journal:  J Thromb Haemost       Date:  2015-04-06       Impact factor: 5.824

Review 9.  Gene therapy for hemophilia.

Authors:  Geoffrey L Rogers; Roland W Herzog
Journal:  Front Biosci (Landmark Ed)       Date:  2015-01-01

Review 10.  Targeting activated protein C to treat hemophilia.

Authors:  Stéphanie G I Polderdijk; Trevor P Baglin; James A Huntington
Journal:  Curr Opin Hematol       Date:  2017-09       Impact factor: 3.284

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  25 in total

Review 1.  Gene therapy for hemophilia: what does the future hold?

Authors:  Bhavya S Doshi; Valder R Arruda
Journal:  Ther Adv Hematol       Date:  2018-08-27

2.  Hyperactivity of factor IX Padua (R338L) depends on factor VIIIa cofactor activity.

Authors:  Benjamin J Samelson-Jones; Jonathan D Finn; Lindsey A George; Rodney M Camire; Valder R Arruda
Journal:  JCI Insight       Date:  2019-06-20

Review 3.  CRISPR System: A High-throughput Toolbox for Research and Treatment of Parkinson's Disease.

Authors:  Fatemeh Safari; Gholamreza Hatam; Abbas Behzad Behbahani; Vahid Rezaei; Mazyar Barekati-Mowahed; Peyman Petramfar; Farzaneh Khademi
Journal:  Cell Mol Neurobiol       Date:  2019-11-26       Impact factor: 5.046

4.  Novel Piperazino-Enaminones Decrease Pro-inflammatory Cytokines Following Hemarthrosis in a Hemophilia Mouse Model.

Authors:  Chen Zhong; Doreen Szollosi; Junjiang Sun; Baolai Hua; Ola Ghoneim; Ashley Bill; Yingping Zhuang; Ivan Edafiogho
Journal:  Inflammation       Date:  2019-10       Impact factor: 4.092

Review 5.  Non-factor replacement therapy for haemophilia: a current update.

Authors:  Massimo Franchini; Pier Mannuccio Mannucci
Journal:  Blood Transfus       Date:  2018-02-14       Impact factor: 3.443

6.  Complete correction of hemophilia B phenotype by FIX-Padua skeletal muscle gene therapy in an inhibitor-prone dog model.

Authors:  Robert A French; Benjamin J Samelson-Jones; Glenn P Niemeyer; Clinton D Lothrop; Elizabeth P Merricks; Timothy C Nichols; Valder R Arruda
Journal:  Blood Adv       Date:  2018-03-13

Review 7.  Extended Half-Life Factor VIII and Factor IX Preparations.

Authors:  Lukas Graf
Journal:  Transfus Med Hemother       Date:  2018-03-21       Impact factor: 3.747

8.  Safety of intra-articular transplantation of lentivirally transduced mesenchymal stromal cells for haemophilic arthropathy in a non-human primate.

Authors:  Tsukasa Ohmori; Hiroaki Mizukami; Yuko Katakai; Sho Kawai; Hitoyasu Nakamura; Makoto Inoue; Tsugumine Shu; Hideharu Sugimoto; Yoichi Sakata
Journal:  Int J Hematol       Date:  2018-05-08       Impact factor: 2.490

9.  Peptides identified on monocyte-derived dendritic cells: a marker for clinical immunogenicity to FVIII products.

Authors:  Wojciech Jankowski; Yara Park; Joseph McGill; Eugene Maraskovsky; Marco Hofmann; Vincent P Diego; Bernadette W Luu; Tom E Howard; Roberta Kellerman; Nigel S Key; Zuben E Sauna
Journal:  Blood Adv       Date:  2019-05-14

10.  Gene-based FVIIa prophylaxis modulates the spontaneous bleeding phenotype of hemophilia A rats.

Authors:  Shannon M Zintner; Juliana C Small; Giulia Pavani; Lynn Dankner; Oscar A Marcos-Contreras; Phyllis A Gimotty; Mads Kjelgaard-Hansen; Bo Wiinberg; Paris Margaritis
Journal:  Blood Adv       Date:  2019-02-12
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