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WFRF:(Tarantino Michael D.)
 

Sökning: WFRF:(Tarantino Michael D.) > Natural history stu...

LIBRIS Formathandbok  (Information om MARC21)
FältnamnIndikatorerMetadata
00005705naa a2200697 4500
001oai:lup.lub.lu.se:90c07a38-726c-4f2b-80b1-e417364fadbb
003SwePub
008220412s2021 | |||||||||||000 ||eng|
0247 a https://lup.lub.lu.se/record/90c07a38-726c-4f2b-80b1-e417364fadbb2 URI
0247 a https://doi.org/10.1111/hae.141392 DOI
040 a (SwePub)lu
041 a engb eng
042 9 SwePub
072 7a art2 swepub-publicationtype
072 7a ref2 swepub-contenttype
1001 a Shapiro, Amy D.u Indiana Hemophilia and Thrombosis Center4 aut
2451 0a Natural history study of factor IX deficiency with focus on treatment and complications (B-Natural)
264 c 2020-12-05
264 1b Wiley,c 2021
300 a 11 s.
520 a 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.
650 7a MEDICIN OCH HÄLSOVETENSKAPx Klinisk medicinx Hematologi0 (SwePub)302022 hsv//swe
650 7a MEDICAL AND HEALTH SCIENCESx Clinical Medicinex Hematology0 (SwePub)302022 hsv//eng
653 a factor IX deficiency
653 a FIX inhibitors
653 a haemophilia B
653 a joint range-of-motion
653 a quality-of-life
653 a treatment
7001 a Ragni, Margaret V.u University of Pittsburgh4 aut
7001 a Borhany, Munirau National Institute of Blood Disease and Bone Marrow Transplantation4 aut
7001 a Abajas, Yasmina L.u University of North Carolina4 aut
7001 a Tarantino, Michael D.u Bleeding and Clotting Disorders Institute, Peoria4 aut
7001 a Holstein, Katharinau University Medical Center Hamburg-Eppendorf4 aut
7001 a Croteau, Stacy E.u Boston Children's Hospital4 aut
7001 a Liesner, Rianau Great Ormond Street Hospital4 aut
7001 a Tarango, Cristinau University of Cincinnati College of Medicine4 aut
7001 a Carvalho, Manuelau Sao Joao Hospital4 aut
7001 a McGuinn, Catherineu Weill Cornell Medical College4 aut
7001 a Funding, Evau Copenhagen University Hospital4 aut0 (Swepub:lu)ev0001fu
7001 a Kempton, Christine L.u Emory University4 aut
7001 a Bidlingmaier, Christophu University Hospital Munich4 aut
7001 a Cohen, Aliceu Newark Beth Israel Medical Center4 aut
7001 a Oldenburg, Johannesu University Hospital Bonn4 aut
7001 a Kearney, Susanu Children's Minnesota Center for Bleeding and Clotting Disorders4 aut
7001 a Knoll, Christineu Phoenix Children’s Hospital4 aut
7001 a Kuriakose, Philipu Henry Ford Health System4 aut
7001 a Acharya, Suchitrau Northwell Haemostasis and Thrombosis Centre4 aut
7001 a Reiss, Ulrike M.u St Jude Children´s Research Hospital, Memphis4 aut
7001 a Kulkarni, Roshniu Michigan State University4 aut
7001 a Witkop, Michelleu Munson Medical Center4 aut
7001 a Lethagen, Stefanu Swedish Orphan Biovitrum4 aut0 (Swepub:lu)medf-sle
7001 a Donfield, Sharyneu Rho, Inc.4 aut
7001 a LeBeau, Petrau Rho, Inc.4 aut
7001 a Berntorp, Eriku Lund University,Lunds universitet,Klinisk koagulationsmedicin, Malmö,Forskargrupper vid Lunds universitet,Clinical Coagulation, Malmö,Lund University Research Groups4 aut0 (Swepub:lu)medf-ebe
7001 a Astermark, Janu Lund University,Lunds universitet,Klinisk koagulationsmedicin, Malmö,Forskargrupper vid Lunds universitet,Clinical Coagulation, Malmö,Lund University Research Groups,Skåne University Hospital4 aut0 (Swepub:lu)medf-jas
7102 a Indiana Hemophilia and Thrombosis Centerb University of Pittsburgh4 org
7730 t Haemophiliad : Wileyg 27:1, s. 49-59q 27:1<49-59x 1351-8216x 1365-2516
8564 u http://dx.doi.org/10.1111/hae.14139y FULLTEXT
8564 8u https://lup.lub.lu.se/record/90c07a38-726c-4f2b-80b1-e417364fadbb
8564 8u https://doi.org/10.1111/hae.14139

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