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Quality of life in ...
Quality of life in a large multinational haemophilia B cohort (The B-Natural study) – Unmet needs remain
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- Berntorp, Erik (author)
- Lund University,Lunds universitet,Klinisk koagulationsmedicin, Malmö,Forskargrupper vid Lunds universitet,Clinical Coagulation, Malmö,Lund University Research Groups
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- LeBeau, Petra (author)
- Rho, Inc.
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- Ragni, Margaret V. (author)
- University of Pittsburgh
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Borhany, Munira (author)
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- Abajas, Yasmina L. (author)
- University of North Carolina
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Tarantino, Michael D. (author)
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- Holstein, Katharina (author)
- University Medical Center Hamburg-Eppendorf
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- Croteau, Stacy E. (author)
- Boston Children's Hospital
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- Liesner, Raina (author)
- Great Ormond Street Hospital
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- Tarango, Cristina (author)
- University of Cincinnati College of Medicine
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Carvalho, Manuela (author)
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- McGuinn, Catherine (author)
- Weill Cornell Medical College
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- Funding, Eva (author)
- Copenhagen University Hospital
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- Kempton, Christine L. (author)
- Emory University
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- Bidlingmaier, Christoph (author)
- University Hospital Munich
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Cohen, Alice (author)
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- Oldenburg, Johannes (author)
- University Hospital Bonn
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Kearney, Susan (author)
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- Knoll, Christine (author)
- Phoenix Children’s Hospital
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- Kuriakose, Philip (author)
- Henry Ford Health System
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Acharya, Suchitra (author)
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- Reiss, Ulrike M. (author)
- St Jude Children´s Research Hospital, Memphis
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- Kulkarni, Roshni (author)
- Michigan State University
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- Witkop, Michelle (author)
- National Hemophilia Foundation,Munson Medical Center
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- Lethagen, Stefan (author)
- Swedish Orphan Biovitrum
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- Krouse, Rebecca (author)
- Rho, Inc.
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Shapiro, Amy D. (author)
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- Astermark, Jan (author)
- 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)
- 2022-03-08
- 2022
- English.
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In: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516. ; 28:3, s. 453-461
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http://dx.doi.org/10... (free)
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https://lup.lub.lu.s...
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https://doi.org/10.1...
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Abstract
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- Introduction: The B-Natural study is a multicentre, multinational, observational study of haemophilia B (HB) designed to increase understanding of clinical manifestations, treatment and quality of life (QoL). Aim: To characterise and compare QoL in HB across disease severity groups and individuals with inhibitors to identify gaps in treatment. Methods: A total of 224 individuals from 107 families were enrolled from a total of 24 centres in North America (n = 16), Europe (n = 7) and Asia (n = 1). Of these, 68 (30.4%) subjects had severe (<1 IU/dL), median age 15.6 years, 114 (50.9%) moderate (1–5 IU/dL), age 13.3 years, and 42 (18.8%) mild (>5–< 40 IU/dL), age 12.1 years, disease. Twenty-nine participants had inhibitors or a history of inhibitors. Three versions of the EQ-5D instrument were used as a measure of QoL: proxy (ages 4–7), youth (ages 8–15) and self (age 16+). Each instrument included a visual analogue scale ranging from 100 (best health) to 0 (worst health) to assess current day's health (EQ VAS). Range-of-motion (ROM) for elbows, knees and ankles was assessed using a four-point scale, from which a composite score was calculated. Results: In all severity groups, a proportion of subjects showed less than optimal QoL. The majority of the mild and moderate severe participants reported a normal EQ-5D health profile (79% and 72%, respectively), whereas about half (47%) of the severe participants and only 13% of the inhibitor participants reported this profile. Conclusion: The B-Natural study reveals impacted QoL in all disease severities of HB including those with inhibitors. Unmet needs remain and include nonsevere HB.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Kardiologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Cardiac and Cardiovascular Systems (hsv//eng)
Keyword
- EQ-5D
- FIX
- haemophilia B
- inhibitor
- prophylaxis
- QoL
Publication and Content Type
- art (subject category)
- ref (subject category)
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- By the author/editor
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Berntorp, Erik
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LeBeau, Petra
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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, Raina
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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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Krouse, Rebecca
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Shapiro, Amy D.
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Astermark, Jan
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- About the subject
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- MEDICAL AND HEALTH SCIENCES
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MEDICAL AND HEAL ...
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and Clinical Medicin ...
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and Cardiac and Card ...
- Articles in the publication
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Haemophilia
- By the university
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Lund University