View Item 

Show simple item record

Origins and organization of the NHLBI State of the Science Workshop: Generating a national blueprint for future research on factor VIII inhibitors
dc.contributor.authorSabatino, Denise E.
dc.contributor.authorPipe, Steven W.
dc.contributor.authorNugent, Diane J.
dc.contributor.authorSoucie, J. Michael
dc.contributor.authorHooper, W. Craig
dc.contributor.authorHoots, W. Keith
dc.contributor.authorDiMichele, Donna M.
dc.date.accessioned2019-08-09T17:15:48Z
dc.date.availableWITHHELD_12_MONTHS
dc.date.available2019-08-09T17:15:48Z
dc.date.issued2019-07
dc.identifier.citationSabatino, Denise E.; Pipe, Steven W.; Nugent, Diane J.; Soucie, J. Michael; Hooper, W. Craig; Hoots, W. Keith; DiMichele, Donna M. (2019). "Origins and organization of the NHLBI State of the Science Workshop: Generating a national blueprint for future research on factor VIII inhibitors." Haemophilia 25(4): 575-580.
dc.identifier.issn1351-8216
dc.identifier.issn1365-2516
dc.identifier.urihttps://hdl.handle.net/2027.42/150613
dc.publisherElsevier
dc.publisherWiley Periodicals, Inc.
dc.subject.otherfactor VIII
dc.subject.otherimmune response
dc.subject.otherinhibitor formation
dc.subject.otherhaemophilia
dc.titleOrigins and organization of the NHLBI State of the Science Workshop: Generating a national blueprint for future research on factor VIII inhibitors
dc.typeArticle
dc.rights.robotsIndexNoFollow
dc.subject.hlbsecondlevelOncology and Hematology
dc.subject.hlbtoplevelHealth Sciences
dc.description.peerreviewedPeer Reviewed
dc.description.bitstreamurlhttps://deepblue.lib.umich.edu/bitstream/2027.42/150613/1/hae13737_am.pdf
dc.description.bitstreamurlhttps://deepblue.lib.umich.edu/bitstream/2027.42/150613/2/hae13737.pdf
dc.identifier.doi10.1111/hae.13737
dc.identifier.sourceHaemophilia
dc.identifier.citedreferenceLe Quellec S, Négrier C. Emicizumab should be prescribed independent of immune tolerance induction. Blood Adv. 2018; 2 ( 20 ): 2783 ‐ 2786.
dc.identifier.citedreferenceLusher JM, Shapiro SS, Palascak JE, Rao AV, Levine PH, Blatt PM. Efficacy of prothrombin‐complex concentrates in hemophiliacs with antibodies to factor VIII: a multicenter therapeutic trial. N Engl J Med. 1980; 303 ( 8 ): 421 ‐ 425.
dc.identifier.citedreferenceHedner U, Kisiel W. Use of human factor VIIa in the treatment of two hemophilia A patients with high‐titer inhibitors. J Clin Invest. 1983; 71 ( 6 ): 1836 ‐ 1841.
dc.identifier.citedreferenceAstermark J, Donfield SM, DiMichele DM, et al. A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) study. Blood. 2007; 109 ( 2 ): 546 ‐ 551.
dc.identifier.citedreferenceKonkle BA, Ebbesen LS, Erhardtsen E, et al. Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitors. J Thromb Haemost. 2007; 5 ( 9 ): 1904 ‐ 1913.
dc.identifier.citedreferenceLeissinger C, Gringeri A, Antmen B, et al. Anti‐inhibitor coagulant complex prophylaxis in hemophilia with inhibitors. N Engl J Med. 2011; 365 ( 18 ): 1684 ‐ 1692.
dc.identifier.citedreferenceSchep SJ, Schutgens R, Fischer K, Boes ML. Review of immune tolerance induction in hemophilia A. Blood Rev. 2018; 32 ( 4 ): 326 ‐ 338.
dc.identifier.citedreferenceDiMichele DM. Immune tolerance in haemophilia: the long journey to the fork in the road. Br J Haematol. 2012; 159 ( 2 ): 123 ‐ 134.
dc.identifier.citedreferenceValentino LA, Kempton Cl, Kruse‐Jarres R, Mathew P, Meeks Sl, Reiss UM. US Guidelines for immune tolerance induction in patients with haemophilia a and inhibitors. Haemophilia. 2015; 21 ( 5 ): 559 ‐ 567.
dc.identifier.citedreferenceZhou Z‐Y, Koerper MA, Johnson KA, et al. Burden of illness: direct and indirect costs among persons with hemophilia A in the United States. J Med Econ. 2015; 18 ( 6 ): 457 ‐ 465.
dc.identifier.citedreferenceUchida N, Sambe T, Yoneyama K, et al. A first‐in‐human phase 1 study of ACE910, a novel factor VIII‐mimetic bispecific antibody, in healthy subjects. Blood. 2016; 127 ( 13 ): 1633 ‐ 1641.
dc.identifier.citedreferencePasi KJ, Rangarajan S, Georgiev P, et al. Targeting of antithrombin in hemophilia A or B with RNAi therapy. N Engl J Med. 2017; 377 ( 9 ): 819 ‐ 828.
dc.identifier.citedreferenceEichler H, Angchaisuksiri P, Kavakli K, et al. A randomized trial of safety, pharmacokinetics and pharmacodynamics of concizumab in people with hemophilia A. J Thromb Haemost. 2018; 16 ( 11 ): 2184 ‐ 2195.
dc.identifier.citedreferenceOldenburg J, Mahlangu JN, Bujan W, et al. The effect of emicizumab prophylaxis on health‐related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 study. Haemophilia. 2019; 25 ( 1 ): 33 ‐ 44.
dc.identifier.citedreferenceGeorge LA. Hemophilia gene therapy comes of age. Blood Adv. 2017; 1 ( 26 ): 2591 ‐ 2599.
dc.identifier.citedreferenceYoung G. Implementing emicizumab in hemophilia inhibitor management: emicizumab should be prescribed after tolerance. Blood Adv. 2018; 2 ( 20 ): 2780 ‐ 2782.
dc.identifier.citedreferencehttps://www.cdc.gov/ncbddd/hemophilia/inhibitors.html. Accessed February 5, 2019.
dc.identifier.citedreferenceSoucie JM, Miller CH, Kelly FM, et al. National surveillance for hemophilia inhibitors in the United States: summary report of an expert meeting. Am J Hematol. 2014; 89: 621 ‐ 625.
dc.identifier.citedreferenceState of the science inhibitors workshop: FVIII inhibitors: generating a national blueprint for future research (Day 1). https://videocast.nih.gov/Summary.asp?Live=27769&bhcp=1. Accessed February 5, 2019.
dc.identifier.citedreferenceState of the science inhibitors workshop: FVIII inhibitors: generating a national blueprint for future research (Day 2). https://videocast.nih.gov/summary.asp?Live=27775&bhcp=1. Accessed February 5, 2019.
dc.identifier.citedreferenceRagni M, George L; on behalf of the Members of Working Group 1. The national blueprint for future factor VIII inhibitor clinical trials: NHLBI State of the Science (SOS) Workshop on factor VIII inhibitors. Haemophilia. 2019; 25 ( 4 ): 581 ‐ 589.
dc.identifier.citedreferenceKonkle BA, Recht M; on behalf of the Members of Working Group 2. The national blueprint for 21st century data and specimen collection and observational cohort studies: NHLBI State of the Science (SOS) Workshop on factor VIII inhibitors. Haemophilia. 2019; 25 ( 4 ): 590 ‐ 594.
dc.identifier.citedreferenceMeeks SL, Herzog RW; on behalf of the Members of Working Group 3. The national blueprint for future basic and translational research to understand factor VIII immunogenicity: NHLBI State of the Science (SOS) Workshop on factor VIII inhibitors. Haemophilia. 2019; 25 ( 4 ): 595 ‐ 602.
dc.identifier.citedreferenceJohnsen JM, Brown DL; on behalf of the Members of Working Group 4. The national blueprint for pregnancy/birth longitudinal cohorts to study factor VIII immunogenicity: NHLBI State of the Science (SOS) Workshop on factor VIII inhibitors. Haemophilia. 2019; 25 ( 4 ): 603 ‐ 609.
dc.identifier.citedreferencePipe SW, Sabatino DE, Nugent DJ, et al. Executive summary of the NHLBI State of the Science (SOS) Workshop: Overview and next steps in generating a national blueprint for future research on factor VIII inhibitors. Haemophilia. 2019; 25 ( 4 ): 610 ‐ 615.
dc.identifier.citedreferenceSoucie JM, Evatt B, Jackson D. Occurrence of hemophilia in the United States. The hemophilia surveillance system project investigators. Am J Hematol. 1998; 59 ( 4 ): 288 ‐ 294.
dc.identifier.citedreferenceCarcao M, Moorehead P, Lillicrap D. Hemophilia A and B. In: Hoffman R, Benz EJ, Silberstein LE, et al., eds. Hematology: Basic Principles and Practice, 7 edn. Philadelphia, PA: Elsevier; 2017: 2001 ‐ 2022.
dc.identifier.citedreferenceManco‐Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. N Engl J Med. 2007; 357 ( 6 ): 535 ‐ 544.
dc.identifier.citedreferenceBlanchette VS, Key NS, Ljung LR, Manco‐Johnson MJ, van den Berg HM, Srivastava A. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2014; 12 ( 11 ): 1935 ‐ 1939.
dc.identifier.citedreferenceGouw SC, van den Berg HM, Fischer K, et al. Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study. Blood. 2013; 121 ( 20 ): 4046 ‐ 4055.
dc.identifier.citedreferenceGouw Sc, van der Bom Jg, Marijke van den Berg H. Treatment‐related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study. Blood. 2007; 109 ( 11 ): 4648 ‐ 4654.
dc.identifier.citedreferenceScalone L, Mantovani LG, Mannucci PM, Gringeri A, COCIS Study Investigators. Quality of life is associated to the orthopaedic status in haemophilic patients with inhibitors. Haemophilia. 2006; 12 ( 2 ): 154 ‐ 162.
dc.identifier.citedreferenceGringeri A, Mantovani LG, Scalone L, Mannucci PM, COCIS Study Group. Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group. Blood. 2003; 102 ( 7 ): 2358 ‐ 2363.
dc.identifier.citedreferenceWalsh CE, Soucie JM, Miller CH, United States Hemophilia Treatment Center Network. Impact of inhibitors on hemophilia A mortality in the United States. Am J Hematol. 2015; 90 ( 5 ): 400 ‐ 405.
dc.identifier.citedreferenceHashemi SM, Fischer K, Moons K, van den Berg HM. Improved prediction of inhibitor development in previously untreated patients with severe haemophilia A. Haemophilia. 2015; 21 ( 2 ): 227 ‐ 233.
dc.owningcollnameInterdisciplinary and Peer-Reviewed


Files in this item

Name:
hae13737_am.pdf
Size:
206.4KB
Format:
PDF
View/Open
Name:
hae13737.pdf
Size:
383.9KB
Format:
PDF
View/Open

Show simple item record

Remediation of Harmful Language

The University of Michigan Library aims to describe library materials in a way that respects the people and communities who create, use, and are represented in our collections. Report harmful or offensive language in catalog records, finding aids, or elsewhere in our collections anonymously through our metadata feedback form. More information at Remediation of Harmful Language.

Accessibility

If you are unable to use this file in its current format, please select the Contact Us link and we can modify it to make it more accessible to you.