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Natural history stu...
Natural history study of factor IX deficiency with focus on treatment and complications (B-Natural)
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- Shapiro, Amy D. (författare)
- Indiana Hemophilia and Thrombosis Center
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- Ragni, Margaret V. (författare)
- University of Pittsburgh
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- Borhany, Munira (författare)
- National Institute of Blood Disease and Bone Marrow Transplantation
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- Abajas, Yasmina L. (författare)
- University of North Carolina
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- Tarantino, Michael D. (författare)
- Bleeding and Clotting Disorders Institute, Peoria
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- Holstein, Katharina (författare)
- University Medical Center Hamburg-Eppendorf
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- Croteau, Stacy E. (författare)
- Boston Children's Hospital
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- Liesner, Riana (författare)
- Great Ormond Street Hospital
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- Tarango, Cristina (författare)
- University of Cincinnati College of Medicine
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- Carvalho, Manuela (författare)
- Sao Joao Hospital
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- McGuinn, Catherine (författare)
- Weill Cornell Medical College
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- Funding, Eva (författare)
- Copenhagen University Hospital
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- Kempton, Christine L. (författare)
- Emory University
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- Bidlingmaier, Christoph (författare)
- University Hospital Munich
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- Cohen, Alice (författare)
- Newark Beth Israel Medical Center
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- Oldenburg, Johannes (författare)
- University Hospital Bonn
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- Kearney, Susan (författare)
- Children's Minnesota Center for Bleeding and Clotting Disorders
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- Knoll, Christine (författare)
- Phoenix Children’s Hospital
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- Kuriakose, Philip (författare)
- Henry Ford Health System
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- Acharya, Suchitra (författare)
- Northwell Haemostasis and Thrombosis Centre
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- Reiss, Ulrike M. (författare)
- St Jude Children´s Research Hospital, Memphis
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- Kulkarni, Roshni (författare)
- Michigan State University
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- Witkop, Michelle (författare)
- Munson Medical Center
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- Lethagen, Stefan (författare)
- Swedish Orphan Biovitrum
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- Donfield, Sharyne (författare)
- Rho, Inc.
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- LeBeau, Petra (författare)
- Rho, Inc.
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- Berntorp, Erik (författare)
- Lund University,Lunds universitet,Klinisk koagulationsmedicin, Malmö,Forskargrupper vid Lunds universitet,Clinical Coagulation, Malmö,Lund University Research Groups
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- Astermark, Jan (författare)
- Lund University,Lunds universitet,Klinisk koagulationsmedicin, Malmö,Forskargrupper vid Lunds universitet,Clinical Coagulation, Malmö,Lund University Research Groups,Skåne University Hospital
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(creator_code:org_t)
- 2020-12-05
- 2021
- Engelska 11 s.
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Ingår i: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516. ; 27:1, s. 49-59
- Relaterad länk:
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http://dx.doi.org/10...
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https://lup.lub.lu.s...
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https://doi.org/10.1...
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Abstract
Ämnesord
Stäng
- 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.
Ämnesord
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Hematologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Hematology (hsv//eng)
Nyckelord
- factor IX deficiency
- FIX inhibitors
- haemophilia B
- joint range-of-motion
- quality-of-life
- treatment
Publikations- och innehållstyp
- art (ämneskategori)
- ref (ämneskategori)
Hitta via bibliotek
Till lärosätets databas
- Av författaren/redakt...
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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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visa fler...
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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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visa färre...
- Om ämnet
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- MEDICIN OCH HÄLSOVETENSKAP
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MEDICIN OCH HÄLS ...
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och Klinisk medicin
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och Hematologi
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Haemophilia
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Lunds universitet