Hemophilia B

Common Name(s)

Hemophilia B, Hereditary factor IX deficiency disease, Christmas disease

Hemophilia B is a bleeding disorder that slows the blood clotting process. People with this disorder 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: 6 Jul 2015

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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

View Details
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: 6 Jul 2015

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 151 free, full-text research articles on human participants. First 3 results:

Renal thromboembolism during treatment with recombinant activated factor VII (rFVIIa) in a child with hemophilia B with factor IX inhibitors.
 

Author(s): Danko Milošević, Ernest Bilić, Danica Batinić, Mirjana Poropat, Ranka Štern-Padovan, Slobodan Galić, Daniel Turudić

Journal:

 

Serious thromboembolic events connected with rFVIIa therapy in hemophilia patients are rare. Only three cases are reported in children, all of them with hemophilia A.

Last Updated: 21 Feb 2015

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Long-term safety and efficacy of factor IX gene therapy in hemophilia B.
 

Author(s): Amit C Nathwani, Ulreke M Reiss, Edward G D Tuddenham, Cecilia Rosales, Pratima Chowdary, Jenny McIntosh, Marco Della Peruta, Elsa Lheriteau, Nishal Patel, Deepak Raj, Anne Riddell, Jun Pie, Savita Rangarajan, David Bevan, Michael Recht, Yu-Min Shen, Kathleen G Halka, Etiena Basner-Tschakarjan, Federico Mingozzi, Katherine A High, James Allay, Mark A Kay, Catherine Y C Ng, Junfang Zhou, Maria Cancio, Christopher L Morton, John T Gray, Deokumar Srivastava, Arthur W Nienhuis, Andrew M Davidoff

Journal: N. Engl. J. Med.. 2014 Nov;371(21):1994-2004.

 

In patients with severe hemophilia B, gene therapy that is mediated by a novel self-complementary adeno-associated virus serotype 8 (AAV8) vector has been shown to raise factor IX levels for periods of up to 16 months. We wanted to determine the durability of transgene expression, ...

Last Updated: 21 Nov 2014

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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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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

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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GOLD-B: A Gene Therapy Study for Hemophilia B
 

Status: Not yet recruiting

Condition Summary: Hemophilia B

 

Last Updated: 24 Jun 2015

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Last Updated: 15 Jan 2015

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