Hemophilia B

Common Name(s)

Hemophilia B, Hereditary factor IX deficiency disease

Hemophilia B is a bleeding disorder that slows the blood clotting process. People with this condition experience prolonged bleeding or oozing following an injury or surgery. In severe cases of hemophilia, heavy bleeding occurs after minor trauma or even in the absence of injury. Serious complications can result from bleeding into the joints, muscles, brain, or other internal organs. Milder forms may not become apparent until abnormal bleeding occurs following surgery or a serious injury. People with an unusual form of hemophilia B, known as hemophilia B Leyden, experience episodes of excessive bleeding in childhood but have few bleeding problems after puberty. Hemophilia B is inherited in an X-linked recessive pattern and is caused by mutations in the F9 gene.
 

Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Hemophilia B" for support, advocacy or research.

Bleeding Disorders Alliance Illinois

The Bleeding Disorders Alliance Illinois exists to improve the quality of life for persons affected by hemophilia and other inherited bleeding disorders.

Last Updated: 25 Sep 2013

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Hemophilia Federation of America

The Hemophilia Federation of America is a national non-profit organization that assists and advocates for the bleeding disorders community.

Last Updated: 26 Nov 2012

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General Support Organizations

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Advocacy and Support Organizations

 

Condition Specific Organizations

Following organizations serve the condition "Hemophilia B" for support, advocacy or research.

Bleeding Disorders Alliance Illinois

The Bleeding Disorders Alliance Illinois exists to improve the quality of life for persons affected by hemophilia and other inherited bleeding disorders.

http://www.bdai.org

Last Updated: 25 Sep 2013

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Hemophilia Federation of America

The Hemophilia Federation of America is a national non-profit organization that assists and advocates for the bleeding disorders community.

http://www.hemophiliafed.org

Last Updated: 26 Nov 2012

View Details

 

General Support Organizations

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Scientific Literature

Articles from the PubMed Database

Research articles describe the outcome of a single study. They are the published results of original research.
The terms "Hemophilia B" returned 146 free, full-text research articles on human participants. First 3 results:

Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial.
 

Author(s): Peter W Collins, Guy Young, Karin Knobe, Faraizah Abdul Karim, Pantep Angchaisuksiri, Claus Banner, Türkiz Gürsel, Johnny Mahlangu, Tadashi Matsushita, Eveline P Mauser-Bunschoten, Johannes Oldenburg, Christopher E Walsh, Claude Negrier,

Journal: Blood. 2014 Dec;124(26):3880-6.

 

This multinational, randomized, single-blind trial investigated the safety and efficacy of nonacog beta pegol, a recombinant glycoPEGylated factor IX (FIX) with extended half-life, in 74 previously treated patients with hemophilia B (FIX activity ≤2 IU/dL). Patients received prophylaxis ...

Last Updated: 19 Dec 2014

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Phase I study of BAY 94-9027, a PEGylated B-domain-deleted recombinant factor VIII with an extended half-life, in subjects with hemophilia A.
 

Author(s): T E Coyle, M T Reding, J C Lin, L A Michaels, A Shah, J Powell

Journal: J. Thromb. Haemost.. 2014 Apr;12(4):488-96.

 

BAY 94-9027 is a B-domain-deleted recombinant factor VIII (rFVIII) with site-specific attachment of poly(ethylene glycol) that has shown an extended half-life in animal models of hemophilia.

Last Updated: 19 May 2014

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Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia B.
 

Author(s): Geoffrey L Rogers, Ashley T Martino, Irene Zolotukhin, Hildegund C J Ertl, Roland W Herzog

Journal:

 

Self-complementary adeno-associated virus (scAAV) vectors have become a desirable vector for therapeutic gene transfer due to their ability to produce greater levels of transgene than single-stranded AAV (ssAAV). However, recent reports have suggested that scAAV vectors are more immunogenic ...

Last Updated: 29 Jan 2014

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Reviews from the PubMed Database

Review articles summarize what is currently known about a disease. They discuss research previously published by others.
The terms "Hemophilia B" returned 3 free, full-text review articles on human participants. First 3 results:

[Gene therapy for hemophilia B].
 

Author(s): Zhang-biao Long, Guo-wei Zhang, Xiao-dong Xi

Journal: Zhonghua Xue Ye Xue Za Zhi. 2013 Sep;34(9):809-11.

 

Last Updated: 9 Oct 2013

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Self-complementary adeno-associated viral vectors for gene therapy of hemophilia B: progress and challenges.
 

Author(s): Deepak Raj, Andrew M Davidoff, Amit C Nathwani

Journal: Expert Rev Hematol. 2011 Oct;4(5):539-49.

 

Therapies currently used for hemophilia involve injection of protein concentrates that are expensive, invasive and associated with side effects such as development of neutralizing antibodies (inhibitors) that diminish therapeutic efficacy. Gene transfer is an attractive alternative ...

Last Updated: 23 Sep 2011

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[Extracorporeal shock wave lithotripsy in a patient with hemophilia B].
 

Author(s): T Yokoyama, Y Nasu

Journal: Nippon Hinyokika Gakkai Zasshi. 1993 Mar;84(3):566-9.

 

We report a successful treatment of extracorporeal shock wave lithotripsy (ESWL) on a renal stone in a patient with severe hemophilia B. The patient was 44 years old man who had suffered left flank colicky pain with gross hematuria. A radiographic study showed a left renal stone, ...

Last Updated: 21 Jul 1993

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Clinical Trial Information This information is provided by ClinicalTrials.gov

Hemophilia B Gene Therapy - Spark
 

Status: Recruiting

Condition Summary: Hemophilia B

 

Last Updated: 31 Oct 2014

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Global Hemostatic Methods in Hemophilia and Von Willebrand's Disease
 

Status: Recruiting

Condition Summary: Hemophilia A; Hemophilia B; Von Willebrand's Disease

 

Last Updated: 11 Feb 2014

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Last Updated: 15 Jan 2015

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