Search: onr:"swepub:oai:lup.lub.lu.se:90c07a38-726c-4f2b-80b1-e417364fadbb" >
Natural history stu...
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Shapiro, Amy D.Indiana Hemophilia and Thrombosis Center
(author)
Natural history study of factor IX deficiency with focus on treatment and complications (B-Natural)
- Article/chapterEnglish2021
Publisher, publication year, extent ...
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2020-12-05
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Wiley,2021
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11 s.
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LIBRIS-ID:oai:lup.lub.lu.se:90c07a38-726c-4f2b-80b1-e417364fadbb
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https://lup.lub.lu.se/record/90c07a38-726c-4f2b-80b1-e417364fadbbURI
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https://doi.org/10.1111/hae.14139DOI
Supplementary language notes
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Language:English
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Summary in:English
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Subject category:art swepub-publicationtype
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Subject category:ref swepub-contenttype
Notes
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Introduction: Haemophilia B (HB) is less well studied than haemophilia A (HA); despite similarities between the two inherited bleeding disorders, important differences remain that require further research. Aim: B-Natural is a multi-centre, prospective, observational study of HB, designed to increase understanding of clinical manifestations, treatment, quality-of-life (QoL), inhibitor development, immune tolerance induction (ITI) outcome, renal function and create a biorepository for future investigations. Methods: Participants include sibling pairs/groups without a current/history of inhibitors and singletons or siblings with a current/history of inhibitors followed for six months. Demographics, medical, social history and treatment were recorded. A physical examination including joint range of motion (ROM) was performed; QoL was assessed. Samples were collected for F9 gene mutation, HLA typing, non-inhibitory antibodies and renal function testing. Results: Twenty-four centres enrolled 224 individuals from 107 families including 29 with current/history of inhibitors. Of these, 68, 30.4%, had severe (<1% FIX level of normal); 114, 50.9%, moderate (1%-5%); and 42, 18.8%, mild (>5-<40%) disease. At enrolment, 53.1% had 50 + exposure days to exogenous FIX. Comparison of joint scores showed significant (P <.05) differences between those with severe (with/without inhibitors), and those with moderate/mild disease. The majority with severe disease, 80.0% with current/history of inhibitors and 64.3% of those without, were treated with prophylaxis. Conclusion: B-Natural provides data supporting an increased understanding of HB and its impact throughout life. The need for optimal disease control to normalize physical and psychosocial outcomes is underscored, and further analyses will contribute to an increased understanding of critical issues in HB.
Subject headings and genre
Added entries (persons, corporate bodies, meetings, titles ...)
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Ragni, Margaret V.University of Pittsburgh
(author)
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Borhany, MuniraNational Institute of Blood Disease and Bone Marrow Transplantation
(author)
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Abajas, Yasmina L.University of North Carolina
(author)
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Tarantino, Michael D.Bleeding and Clotting Disorders Institute, Peoria
(author)
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Holstein, KatharinaUniversity Medical Center Hamburg-Eppendorf
(author)
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Croteau, Stacy E.Boston Children's Hospital
(author)
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Liesner, RianaGreat Ormond Street Hospital
(author)
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Tarango, CristinaUniversity of Cincinnati College of Medicine
(author)
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Carvalho, ManuelaSao Joao Hospital
(author)
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McGuinn, CatherineWeill Cornell Medical College
(author)
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Funding, EvaCopenhagen University Hospital(Swepub:lu)ev0001fu
(author)
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Kempton, Christine L.Emory University
(author)
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Bidlingmaier, ChristophUniversity Hospital Munich
(author)
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Cohen, AliceNewark Beth Israel Medical Center
(author)
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Oldenburg, JohannesUniversity Hospital Bonn
(author)
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Kearney, SusanChildren's Minnesota Center for Bleeding and Clotting Disorders
(author)
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Knoll, ChristinePhoenix Children’s Hospital
(author)
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Kuriakose, PhilipHenry Ford Health System
(author)
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Acharya, SuchitraNorthwell Haemostasis and Thrombosis Centre
(author)
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Reiss, Ulrike M.St Jude Children´s Research Hospital, Memphis
(author)
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Kulkarni, RoshniMichigan State University
(author)
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Witkop, MichelleMunson Medical Center
(author)
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Lethagen, StefanSwedish Orphan Biovitrum(Swepub:lu)medf-sle
(author)
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Donfield, SharyneRho, Inc.
(author)
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LeBeau, PetraRho, Inc.
(author)
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Berntorp, ErikLund University,Lunds universitet,Klinisk koagulationsmedicin, Malmö,Forskargrupper vid Lunds universitet,Clinical Coagulation, Malmö,Lund University Research Groups(Swepub:lu)medf-ebe
(author)
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Astermark, JanLund University,Lunds universitet,Klinisk koagulationsmedicin, Malmö,Forskargrupper vid Lunds universitet,Clinical Coagulation, Malmö,Lund University Research Groups,Skåne University Hospital(Swepub:lu)medf-jas
(author)
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Indiana Hemophilia and Thrombosis CenterUniversity of Pittsburgh
(creator_code:org_t)
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In:Haemophilia: Wiley27:1, s. 49-591351-82161365-2516
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Shapiro, Amy D.
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Ragni, Margaret ...
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Borhany, Munira
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Abajas, Yasmina ...
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Tarantino, Micha ...
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Holstein, Kathar ...
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Croteau, Stacy E ...
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Liesner, Riana
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Tarango, Cristin ...
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Carvalho, Manuel ...
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McGuinn, Catheri ...
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Funding, Eva
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Kempton, Christi ...
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Bidlingmaier, Ch ...
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Cohen, Alice
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Oldenburg, Johan ...
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Kearney, Susan
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Knoll, Christine
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Kuriakose, Phili ...
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Acharya, Suchitr ...
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Reiss, Ulrike M.
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Kulkarni, Roshni
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Witkop, Michelle
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Lethagen, Stefan
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Donfield, Sharyn ...
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LeBeau, Petra
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Berntorp, Erik
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Astermark, Jan
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Hematology
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Haemophilia
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Lund University