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Expert Review of Hematology Volume 7, 2014 - Issue 6
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Perspective

Design of the INHIBIT trial: preventing inhibitors by avoiding ‘danger’, prolonging half-life and promoting tolerance

Margaret V RagniDepartment of Medicine, Division Hematology/Oncology, University of Pittsburgh, 3636 Boulevard of the Allies, Pittsburgh, 15213-4306, PA, USA;Hemophilia Center of Western PA, Pittsburgh, PA, USACorrespondence[email protected]
&
Lynn M MalecHemophilia Center of Western PA, Pittsburgh, PA, USA;Children’s Hospital of Pittsburgh, Pittsburgh, PA, USA
Pages 747-755 | Published online: 05 Nov 2014

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Lindsey A George & Rodney M Camire. (2015) Profile of efraloctocog alfa and its potential in the treatment of hemophilia A. Journal of Blood Medicine 6, pages 131-141.
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Lynn Malec, An Van Damme, Anthony K. C. Chan, Mariya Spasova, Nisha Jain, Charlotte Sensinger, Jennifer Dumont, Stefan Lethagen, Manuel Carcao & Flora Peyvandi. (2023) Recombinant factor VIII Fc fusion protein for first-time immune tolerance induction: final results of the verITI-8 study. Blood 141:16, pages 1982-1989.
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Feixu Zhang, Xiaoying Zong, Xinyue Zhou, Shuming Sun, Xiao Xiao & Junjiang Sun. (2021) Naive haemophilia mice displayed different pattern of cytokine profiles of cytokine profiles changes might be associated with subclinical bleeding. Blood Coagulation & Fibrinolysis 32:8, pages 584-590.
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Omotola O Olasupo, Megan S Lowe, Ashma Krishan, Peter Collins, Alfonso Iorio & Davide Matino. (2021) Clotting factor concentrates for preventing bleeding and bleeding-related complications in previously treated individuals with haemophilia A or B. Cochrane Database of Systematic Reviews 2021:8.
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Marnie Bertolet, Maria M. Brooks & Margaret V. Ragni. (2020) The design of a Bayesian platform trial to prevent and eradicate inhibitors in patients with hemophilia. Blood Advances 4:21, pages 5433-5441.
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Shannon L. Meeks & Sébastien Lacroix‐Desmazes. (2020) Emerging benefits of Fc fusion technology in the context of recombinant factor VIII replacement therapy. Haemophilia 26:6, pages 958-965.
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Patrick T. Ebbert, Frederico Xavier, Lynn M. Malec, Craig D. Seaman & Margaret V. Ragni. (2020) Observational study of recombinant factor VIII-Fc, rFVIIIFc, in hemophilia A. Thrombosis Research 195, pages 51-54.
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Margaret V. Ragni & Lindsey A. George. (2019) The national blueprint for future factor VIII inhibitor clinical trials: NHLBI State of the Science (SOS) Workshop on factor VIII inhibitors. Haemophilia 25:4, pages 581-589.
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Courtney D. Thornburg. (2018) How I approach: Previously untreated patients with severe congenital hemophilia A. Pediatric Blood & Cancer 65:12, pages e27466.
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Richard S. Blumberg & David Lillicrap. (2018) Tolerogenic properties of the Fc portion of IgG and its relevance to the treatment and management of hemophilia. Blood 131:20, pages 2205-2214.
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David W. Scott. (2017) From IgG Fusion Proteins to Engineered-Specific Human Regulatory T Cells: A Life of Tolerance. Frontiers in Immunology 8.
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K. M. Lövgren, H. Søndergaard, S. Skov & B. Wiinberg. (2016) Non‐genetic risk factors in haemophilia A inhibitor management – the danger theory and the use of animal models. Haemophilia 22:5, pages 657-666.
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M. V. Ragni, M. Alabek & L. M. Malec. (2016) Inhibitor development in two cousins receiving full‐length factor VIII (FVIII) and FVIII‐Fc fusion protein. Haemophilia 22:5.
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Charles L. Groomes, David M. Gianferante, Gary D. Crouch, Dina S. Parekh, David W. Scott & Kenneth Lieuw. (2016) Reduction of Factor VIII Inhibitor Titers During Immune Tolerance Induction With Recombinant Factor VIII-Fc Fusion Protein. Pediatric Blood & Cancer 63:5, pages 922-924.
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Margaret V. Ragni. (2016) Platelet VIII pack evades immune detection. Blood 127:10, pages 1222-1224.
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Yingyu ChenJocelyn A. SchroederJuan ChenXiaofeng LuoChristina K. BaumgartnerRobert R. Montgomery, Jianda HuQizhen Shi. (2016) The immunogenicity of platelet-derived FVIII in hemophilia A mice with or without preexisting anti-FVIII immunity. Blood 127:10, pages 1346-1354.
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