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This page shows you the latest news and research items in this category.

Central venous access device‐related infections in patients with haemophilia
ConclusionCVAD‐related infection in this Australian population was comparable to rates described in the medical literature. Ongoing surveillance for infection rates is important to provide an up‐to‐date assessment of risks associated with CVAD use in this population. (Source: Journal of Paediatrics and Child Health)
Source: Journal of Paediatrics and Child Health - February 26, 2013 Category: Pediatrics Authors: Zhi Han Yeoh, Janine Furmedge, Julia Ekert, Joe Crameri, Nigel Curtis, Chris Barnes Tags: Original Article Source Type: research

Secondary prophylaxis in an adult patient with severe haemophilia A. Factor VIII consumption and effectiveness.
Conclusion: An optimal dosing schedule needs to be discussed and larger and longer prospective studies targeting the benefits of secondary prophylaxis with factor VIII started in adulthood are necessary. PMID: 23435748 [PubMed - as supplied by publisher] (Source: Hamostaseologie)
Source: Hamostaseologie - February 25, 2013 Category: Hematology Authors: Schlenkrich S, Schubert C Tags: Hamostaseologie Source Type: research

The effect of secondary prophylaxis versus episodic treatment on the range of motion of target joints in patients with haemophilia
This study prospectively compared the effect of secondary prophylaxis to episodic treatment on target joint (TJ) range of motion (ROM), number of joint haemorrhages and new TJ development in patients with moderate or severe haemophilia. Two‐hundred and eighty‐six males, 17% in prophylaxis, 83% in episodic treatment group, participating in the Centers for Disease Control and Prevention's Universal Data Collection project, fulfilled inclusion criteria: age >2 years at enrolment, free of TJs at enrolment, developed at least one TJ after enrolment, and received either prophylaxis or episodic treatment continuously for ...
Source: British Journal of Haematology - February 22, 2013 Category: Hematology Authors: Sweta Gupta, Azfar‐E‐Alam Siddiqi, J. Michael Soucie, Marilyn Manco‐Johnson, Roshni Kulkarni, Heidi Lane, Robina Ingram‐Rich, Joan C. Gill, Tags: Research Paper Source Type: research

Unraveling the genetics of inhibitors in hemophilia
(Source: Blood)
Source: Blood - February 21, 2013 Category: Hematology Authors: Gouw, S. C., Fijnvandraat, K. Tags: INSIDE BLOOD Source Type: research

The polygenic nature of inhibitors in hemophilia A: results from the Hemophilia Inhibitor Genetics Study (HIGS) Combined Cohort
Key Points The data demonstrate the complexity of the genetic contribution to inhibitor development in people with hemophilia A. Potentially decisive markers have been identified, indicating the importance of further evaluation of intracellular signaling pathways. (Source: Blood)
Source: Blood - February 21, 2013 Category: Hematology Authors: Astermark, J., Donfield, S. M., Gomperts, E. D., Schwarz, J., Menius, E. D., Pavlova, A., Oldenburg, J., Kessing, B., DiMichele, D. M., Shapiro, A. D., Winkler, C. A., Berntorp, E., on behalf of the Hemophilia Inhibitor Genetics Study (HIGS) Combined Coho Tags: Pediatric Hematology, Free Research Articles, Thrombosis and Hemostasis Source Type: research

Prophylactic Therapy Achieving Zero Annual Bleed Rates Gives Hemophilia Patients Normal Quality Of Life
Children with hemophilia A achieving annual bleed rates of zero on prophylactic therapy have similar quality of life and physical health to the healthy population, according to a study reported at the 6th Annual Congress of the European Association for Hemophilia and Allied Disorders (6-8 February 2013; Warsaw, Poland)... (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - February 16, 2013 Category: Consumer Health News Tags: Blood / Hematology Source Type: news

Alloantibodies against the B subunit of plasma factor XIII developed in its congenital deficiency.
Abstract Factor XIII (FXIII) is a fibrin-stabilising factor consisting of catalytic A subunits (FXIII-A) and carrier B subunits (FXIII-B). FXIII-B prevents the fast clearance of FXIII-A from the circulation. Congenital FXIII-A deficiency is a rare bleeding disorder, and congenital FXIII-B deficiency is even rarer. Through our recent nationwide survey on "acquired haemophilia-like disease due to anti-FXIII autoantibodies," we newly diagnosed severe congenital FXIII-B deficiency in a Japanese man. He developed thrombocytopenia and gingival bleedings at the age of 73, and his FXIII activity was as low as 10% of the no...
Source: Thrombosis and Haemostasis - February 14, 2013 Category: Hematology Authors: Wada H, Souri M, Matsumoto R, Sugihara T, Ichinose A Tags: Thromb Haemost Source Type: research

Enhancing the pharmacokinetic properties of recombinant factor VIII: First‐in‐man trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A
ConclusionsA single dose of up to 75 U/kg N8‐GP was well tolerated in patients with hemophilia A, with no safety concerns. N8‐GP had a prolonged half‐life and FVIII:C activity remained above 1% for a longer compared with the patient's previous product. These results indicate that N8–GP has the potential to reduce dosing frequency during prophylaxis.© 2013 International Society on Thrombosis and Haemostasis (Source: Journal of Thrombosis and Haemostasis)
Source: Journal of Thrombosis and Haemostasis - February 11, 2013 Category: Hematology Authors: A. Tiede, B. Brand, R. Fischer, K. Kavakli, S. R. Lentz, T. Matsushita, C. Rea, K. Knobe, D. Viuff Tags: Original Article ‐ Clinical Haemostasis and Thrombosis Source Type: research

Biogen, Sobi Phase III studies support recombinant Fc fusion proteins protection capability
Biogen Idec and Swedish Orphan Biovitrum (Sobi) have announced new data from Phase III registrational studies supporting recombinant Fc fusion proteins' ability to provide long-lasting protection from bleeding in patients with haemophilia A and (Source: Drug Development Technology)
Source: Drug Development Technology - February 10, 2013 Category: Pharmaceuticals Source Type: news

New hemophilia data could mean more jobs at Biogen
New data released this week for two hemophilia treatments could one day lead to Biogen Idec (Nasdaq: BIIB) adding jobs at its local facility. Pivotal Phase 3 data confirms that two Research Triangle Park-manufactured proteins - dubbed Factor VIII and Factor IX - can provide long-lasting protection from bleeding with fewer injections than what hemophilia patients currently face. It’s exciting - both for the hemophilia patients and to the Research Triangle Park facility that makes the products. The… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - February 8, 2013 Category: Pharmaceuticals Authors: Lauren K. Ohnesorge Source Type: research

Platelet activation and aggregation: the importance of thrombin activity. A laboratory model
This study introduces a new laboratory model of whole blood platelet aggregation stimulated by endogenously generated thrombin, and explores this aspect in haemophilia A in which impaired thrombin generation is a major hallmark. The method was established to measure platelet aggregation initiated by tissue factor evaluated by means of impedance aggregometry. Citrated whole blood from healthy volunteers and haemophilia A patients with the addition of inhibitors of the contact pathway and fibrin polymerization was evaluated. In healthy persons, a second wave of platelet aggregation was found to coincide with the thrombin bur...
Source: Haemophilia - February 7, 2013 Category: Hematology Authors: M. S. Jensen, O. H. Larsen, K. Christiansen, C. Fenger‐Eriksen, J. Ingerslev, B. Sørensen Tags: Original Article Source Type: research

Distinct characteristics of antibody responses against factor VIII in healthy individuals and in different cohorts of hemophilia A patients
Key Points Antibodies against factor VIII show distinct characteristics in healthy individuals and different cohorts of hemophilia A patients. IgG4 antibodies against FVIII are only found in patients with inhibitors but not in healthy individuals or patients without inhibitors. (Source: Blood)
Source: Blood - February 7, 2013 Category: Hematology Authors: Whelan, S. F. J., Hofbauer, C. J., Horling, F. M., Allacher, P., Wolfsegger, M. J., Oldenburg, J., Male, C., Windyga, J., Tiede, A., Schwarz, H. P., Scheiflinger, F., Reipert, B. M. Tags: Free Research Articles, Thrombosis and Hemostasis Source Type: research

Nuclear factor (NF)‐κB and its associated pathways are major molecular regulators of blood‐induced joint damage in a murine model of hemophilia
ConclusionsThese data suggest that NF‐κB‐associated signaling pathways are involved in the development of hemophilic arthropathy. (Source: Journal of Thrombosis and Haemostasis)
Source: Journal of Thrombosis and Haemostasis - February 7, 2013 Category: Hematology Authors: D. Sen, A. Chapla, N. Walter, V. Daniel, A. Srivastava, G. R. Jayandharan Tags: Original Article Source Type: research

LRP1/CD91 is up‐regulated in monocytes from patients with haemophilia A: a single‐centre analysis
In conclusion, our study documents for the first time that LRP1/CD91 is over‐expressed on monocytes from HA patients, with the intensity of expression varying according to the severity of the FVIII deficiency. Further studies are needed to assess the clinical implications of these findings. (Source: Haemophilia)
Source: Haemophilia - February 6, 2013 Category: Hematology Authors: M. Franchini, S. Urbani, B. Amadei, G. F. Rivolta, C. Di Perna, F. Riccardi, F. Frattini, S. Crestani, C. Bonfanti, A. Formentini, R. Quintavalla, A. Tagliaferri Tags: Original Article Source Type: research

Cytokine profile and FVIII inhibitors development in haemophilia A
This study investigated the immunological cytokine profile using plasma from HA patients which were either positive or negative for FVIII inhibitors and from healthy individuals. The results showed that healthy individuals and HA patients that do not develop FVIII inhibitors have a mixed immune response profile with high secretion of IFN‐γ, TNF‐α IL‐2 and IL‐5. In contrast, HA patients with FVIII inhibitors exhibited an anti‐inflammatory/regulatory immune response characterized by low levels of all measured cytokines except for IL‐4 and IL‐10. This profile may be related to the development and maintenance o...
Source: Haemophilia - February 6, 2013 Category: Hematology Authors: C. A. Oliveira, C. Velloso‐Rodrigues, F. C. J. Machado, B. N. Carvalho, S. H. L. Gentz, O. A. Martins‐Filho, D. G. Chaves Tags: Original Article Source Type: research

Novel coagulation factor concentrates: Issues relating to their clinical implementation and pharmacokinetic assessment for optimal prophylaxis in haemophilia patients
Summary Prophylaxis is considered the optimal treatment regimen for patients with severe haemophilia, and may be especially important in the prevention of joint disease. Novel coagulation factor concentrates with prolonged half‐lives promise to improve patient treatment by enabling prophylaxis with less frequent dosing. With the call to individualize therapy in haemophilia, there is growing awareness of the need to use pharmacokinetic (PK) assessments to tailor prophylaxis. However, for new factor concentrates, it is not yet known which PK values will be most informative for optimizing prophylaxis. This topic was explore...
Source: Haemophilia - February 6, 2013 Category: Hematology Authors: R. Ljung, G. Auerswald, G. Benson, A. Jetter, V. Jiménez‐Yuste, T. Lambert, M. Morfini, E. Remor, B. Sørensen, S. Z. Šalek Tags: Original Article Source Type: research

Third Åland islands conference on von Willebrand disease, 26–28 September 2012: meeting report
Summary The first meeting of international specialists in the field of von Willebrand disease (VWD) was held in the Åland islands in 1998 where Erik von Willebrand had first observed a bleeding disorder in some members of a family from Föglö and a summary of the meeting was published in 1999. The second meeting was held in 2010 and a report of the meeting was published in 2012. Topics covered included progress in understanding of VWD over the last 50 years; multimers; classification of VWD; pharmacokinetics and laboratory assays; genetics; treating the paediatric patient; prophylaxis; geriatrics; gene therapy and treat...
Source: Haemophilia - February 6, 2013 Category: Hematology Authors: E. Berntorp, B. Fuchs, M. Makris, R. Montgomery, V. Flood, J. S. O'Donnell, A. B. Federici, D. Lillicrap, P. James, U. Budde, M. Morfini, P. Petrini, S. Austin, C. Kannicht, V. Jiménez‐Yuste, C. Lee Tags: Review Article Source Type: research

The clot thickens for long-lasting drugs that stop hemophilia short
Nature Medicine 19, 121 (2013). doi:10.1038/nm0213-121 Author: Alisa Opar (Source: Nature Medicine)
Source: Nature Medicine - February 6, 2013 Category: Journals (General) Authors: Alisa Opar Tags: News Source Type: research

Treatment of refractory hemorrhage with factor XIII in a patient with hemophilia A with inhibitor
Abstract An 11‐year‐old male with hemophilia A and a known high‐titer Factor VIII inhibitor was admitted with retroperitoneal hemorrhage. The patient was receiving infusions of recombinant activated Factor VII (rFVIIa) for a recent elbow hemorrhage when retroperitoneal bleeding commenced. Despite increased dosing of rFVIIa and a dose of activated prothrombin complex concentrate (aPCC), he continued to hemorrhage and required several blood transfusions. Factor XIII was administered 1 hour after rFVIIa and the patient demonstrated cessation of bleeding and normalization of clot strength. Factor XIII may act as an adj...
Source: Pediatric Blood and Cancer - February 4, 2013 Category: Cancer & Oncology Authors: Christopher Ng, Christopher C. Silliman, Gabrielle Pearl, Whitney Smith, Marilyn Manco‐Johnson, Michael Wang Tags: Brief Report Source Type: research

Case studies in the management of refractory bleeding in patients with haemophilia A and inhibitors
Summary In haemophilia patients with well‐established high‐titer inhibitors, even seemingly minor acute bleeding episodes or surgical procedures may become refractory to treatment and transform into limb‐ or life‐threatening situations. In the absence of evidence‐based treatment guidelines, this article presents 10 cases of difficult to control acute and surgical bleeding and offers consensus opinions regarding their management from a panel of experienced haemophilia treaters. (Source: Haemophilia)
Source: Haemophilia - February 4, 2013 Category: Hematology Authors: L. A. Valentino, G. Allen, J. C. Gill, A. Hurlet, B. A. Konkle, C. A. Leissinger, L. Luchtman‐Jones, J. Powell, M. Reding, K. Stine Tags: Original Article Source Type: research

Functional limitations in Romanian children with haemophilia: further testing of psychometric properties of the Paediatric Haemophilia Activities List
The objective was to gain further insight into the psychometric properties of the PedHAL and to study the functional health status of Romanian children and adolescents with haemophilia. Children attending to the rehabilitation centre of Buzias in Romania were sampled consecutively. Construct validity of the PedHAL was evaluated by concurrent testing with objective and subjective measures of physical function and functional ability. Reproducibility was tested by a 3‐day test–retest by intraclass correlation coefficient (ICC) and limits of agreement (LOA). Responsiveness to rehabilitation was assessed by Haemophilia Join...
Source: Haemophilia - February 4, 2013 Category: Hematology Authors: W. Groen, J. Net, A. M. Lacatusu, M. Serban, P. J. M. Helders, K. Fischer Tags: Original Article Source Type: research

Incidence, mortality rates and causes of deaths in haemophilia patients in Sweden
Summary Sweden has been a pioneer in the treatment of haemophilia, with the first concentrate available in the 1950s. Treatment has improved over the years to its current state‐of‐the art. The aim of the current study was to evaluate the long‐term outcome of haemophilia in terms of incidence, morbidity and mortality. Patients diagnosed with haemophilia A or B registered at the national haemophilia centres and/or the Patient Registry and born before 2009 and alive in 1968 were enrolled and linked to the Cause of Death‐, Migration‐ and Medical Birth registries. Five age‐ and sex‐matched controls were selected f...
Source: Haemophilia - February 4, 2013 Category: Hematology Authors: S. Lövdahl, K. M. Henriksson, F. Baghaei, M. Holmström, J.‐Å. Nilsson, E. Berntorp, J. Astermark Tags: Original Article Source Type: research

Practical and cost‐effective measurement of B‐domain deleted and full‐length recombinant FVIII in the routine haemostasis laboratory
The objective of this study was to facilitate the measurement of rFVIII, taking into account the recent demonstration that a calibration curve does not have to be included in each run. FVIII:C was measured in patients' samples after infusion of different types of rFVIII with a one‐stage and a chromogenic assay calibrated either with pooled normal plasma or a product‐specific laboratory standard. Results obtained with the one‐stage coagulation assay were compared with these provided by a chromogenic assay. We confirmed that a calibration curve can be used for a prolonged period of time without loss of precision and ac...
Source: Haemophilia - February 4, 2013 Category: Hematology Authors: M. Cauchie, J. Toelen, K. Peerlinck, M. Jacquemin Tags: Original Article Source Type: research

Economic burden of high-responding inhibitors in patients with hemophilia a in taiwan.
Conclusion: Our data reveal higher medical expenditures burden for patients with HA and high-responding inhibitors in Taiwan. Future research is encouraged to evaluate the impact of this burden on patient quality of life. PMID: 23364968 [PubMed - in process] (Source: Yonsei Medical Journal)
Source: Yonsei Medical Journal - February 3, 2013 Category: Universities & Medical Training Authors: Tu TC, Cheng SN, Chen JD, Cham TM, Chung MI Tags: Yonsei Med J Source Type: research

Erratum to "Prevalence, Incidence, and Factor Concentrate Usage Trends of Hemophiliacs in Taiwan" by Tu TC, et al.(Yonsei Med J 2013;54:71-80.).
Authors: PMID: 23364996 [PubMed - in process] (Source: Yonsei Medical Journal)
Source: Yonsei Medical Journal - February 3, 2013 Category: Universities & Medical Training Tags: Yonsei Med J Source Type: research

First Person with Hemophilia to Climb Mt. Aconcagua in Argentina:...
Using an experiemental drug, a man from Denver becomes the first person with hemophilia to attempt to summit Mt. Aconcagua in Argentina. The climb is sponsored by LA Kelley Communications, Inc.(PRWeb February 02, 2013)Read the full story at http://www.prweb.com/releases/2013/2/prweb10371422.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - February 2, 2013 Category: Pharmaceuticals Source Type: news

Transfusion related complications in hemophilia
Neelam MarwahaAsian Journal of Transfusion Science 2013 7(1):6-7 (Source: Asian Journal of Transfusion Science)
Source: Asian Journal of Transfusion Science - February 2, 2013 Category: Hematology Authors: Neelam Marwaha Source Type: research

Evaluation of transfusion-related complications along with estimation of inhibitors in patients with hemophilia: A pilot study from a single center
Conclusion: This is probably first comprehensive study from our state on laboratory testing in PWH. The specialty of Transfusion Medicine can be a core part of hemophilia care. The overall prevalence of inhibitors in our hemophilia A patients was 5.1%, which is less as compared to majority of published studies. (Source: Asian Journal of Transfusion Science)
Source: Asian Journal of Transfusion Science - February 2, 2013 Category: Hematology Authors: Anju DubeyAnupam VermaPriti ElhencePrashant Agarwal Source Type: research

Seroprevalence of hepatitis C, hepatitis B and HIV viruses in hemophiliacs born 1985-2010 in west Azarbaijan of Iran
Conclusion: In this study prevalence of HCV infection was very smaller than similar studies in Iran and other countries. This study shows the safety of using viral inactivated factor concentrates and recombinant factors after year 1985.None of Hemophiliacs were seropositive for HIV Ab and HBs Ag. (Source: Asian Journal of Transfusion Science)
Source: Asian Journal of Transfusion Science - February 2, 2013 Category: Hematology Authors: Nasim ValizadehShahin NateghiMehran NorooziSasan HejaziFateme AghanezhadAfsaneh Akbarpoor Morad Ali Source Type: research

How I manage patients with acquired haemophilia A
Summary Acquired haemophilia A (AHA) is a potentially life‐threatening bleeding disorder occurring in patients without a previous personal or family history of bleeding. Development of immune‐mediated autoantibodies against coagulation factor VIII is associated with a wide range of clinical disorders including pregnancy, autoimmune disorders, malignancy, or with no apparent disease. There exists great potential for morbidity and mortality related to acute and recurrent bleeding episodes, making prompt diagnosis and treatment necessary. The two primary goals of treatment focus on cessation of bleeding and eradication of...
Source: British Journal of Haematology - February 1, 2013 Category: Hematology Authors: Douglas W. Sborov, George M. Rodgers Tags: review Source Type: research

Paediatric presentation and outcome of congenital protein C deficiency in Japan
Summary Severe heritable protein C (PC) deficiency is quite rare, although heterozygous PROC mutation is the second leading cause of genetic predisposition to thrombosis in Japanese adults. The aim of the study was to search the optimal management, the paediatric onset and outcomes of PC deficiency were characterized in Japan. The genetic study, postmarketing survey of activated PC (aPC) concentrate (Anact®C) and intensive review in Japan for 20 years enabled the analysis of the disease onset, genotype, treatment and prognosis. Symptomatic PC deficiency was determined in 27 Japanese children. All but two patients present...
Source: Haemophilia - February 1, 2013 Category: Hematology Authors: S. Ohga, D. Kang, T. Kinjo, M. Ochiai, T. Doi, M. Ishimura, Y. Kayamori, M. Urata, J. Yamamoto, S.‐I. Suenobu, H. Kanegane, T. Ikenoue, A. Shirahata, T. Hara Tags: Original Article Source Type: research

IL‐6 Receptor Antagonist as Adjunctive Therapy with Clotting Factor Replacement to Protect Against Bleeding‐Induced Arthropathy in Hemophilia
ConclusionsShort‐course specific inhibition of inflammatory cytokines as an adjunct to replacement hemostasis may be an approach to minimize hemophilic joint degeneration.© 2013 International Society on Thrombosis and Haemostasis (Source: Journal of Thrombosis and Haemostasis)
Source: Journal of Thrombosis and Haemostasis - February 1, 2013 Category: Hematology Authors: N Narkbunnam, J Sun, G Hu, F‐C Lin, T A. Bateman, M Mihara, P E. Monahan Tags: Original Article ‐ Coagulation Source Type: research

Genetic changes in severe haemophilia A: new contribution to the aetiology of a complex disease
Haemophilia A is an X-linked bleeding disorder caused by reduced or absent clotting factor VIII (FVIII) activity, determined by heterogeneous mutations in the F8 gene. Identification of these pathogenic mutations is important for genetic counseling and the assessment of clinical manifestations. Although more than 700 mutations of the F8 gene have been reported as responsible for severe haemophilia (FVIII: C (Source: Blood Coagulation and Fibrinolysis)
Source: Blood Coagulation and Fibrinolysis - January 31, 2013 Category: Hematology Tags: Original Articles Source Type: research

Polymyalgia rheumatica: a diagnostic challenge in the elderly with haemophilia and diffuse arthropathy
With the increased life expectancy and associated comorbidities in haemophilic patients, greater awareness is needed to avoid being misled by symptoms mimicking haemophilia-related conditions. Here, we discuss the case of a 77-year-old man with congenital moderate haemophilia complicated by diffuse haemophilic arthropathy. He presented with persistent pain and stiffness affecting both shoulder and pelvic girdles, unresponsive to factor VIII (FVIII) infusions. A blood test confirmed the high clinical suspicion of polymyalgia rheumatica (PMR), justifying a course of low-dose methylprednisolone that led to significant clinica...
Source: Blood Coagulation and Fibrinolysis - January 31, 2013 Category: Hematology Tags: Case Reports Source Type: research

Study of intron 22 inversion mutation in north India with review
The present study assessed the frequency of intron 22 inversion mutation (Inv 22) in north Indian population with a cost analysis of different methods used for Inv 22 detection. We assessed the frequency of intron 22 inversion mutation in a series of 181 cases with hemophilia A and also compared methods used for detection of the mutation including the long-distance PCR, Southern blot analysis, and inverse PCR in terms of cost, infrastructure, and technical input as well as turnaround time. The study group comprised 102 severe cases and 79 moderate cases of hemophilia A from a north Indian population of which 77 cases teste...
Source: Blood Coagulation and Fibrinolysis - January 31, 2013 Category: Hematology Tags: Original Articles Source Type: research

Bone disease in patients with haemophilia. What is true?
Ghoshy et al. in their recent review in European Journal of Haematology (1) tried to present data about the prevalence of low bone mineral density (BMD) in patients with haemophilia A and B and analyze the pathophysiology of this entity proposing appropriate management. The authors report data of small studies and refer mainly to the association of low BMD with hepatitis C (HCV) and human immunodeficiency virus (HIV) infections and their treatment (interferon‐α, anti‐retroviral therapy). They also refer to the possible role of vitamin K and the interaction between factor VIII‐von Willebrand and receptor activator of...
Source: European Journal of Haematology - January 31, 2013 Category: Hematology Authors: Panagiotis Anagnostis, Vasilia Garipirou, Eirini Kazantzidou, Sofia Vakalopoulou Tags: Letter to the editor Source Type: research

Survey of laboratory tests used in the diagnosis and evaluation of haemophilia A.
Abstract Although the incidence of haemophilia A is reportedly uniform across ethnic groups, the prevalence varies in different countries. This suggests variability in the effectiveness of diagnostic strategies which is of particular importance in the recognition of milder forms of the disease. To assess the different laboratory tests that are used in the diagnosis and subsequent management of haemophilia A we carried out a web-based survey of established haemophilia centres. This was sent to 13 haemophilia physicians from haemophilia-treatment centres in Germany, Italy, Spain, South Africa, Taiwan, Norway, Canada,...
Source: Thrombosis and Haemostasis - January 31, 2013 Category: Hematology Authors: Gomez K, Chitlur M, on behalf of the GEHEP panel Tags: Thromb Haemost Source Type: research

High number of CD56bright NK-cells and persistently low CD4+ T-cells in a hemophiliac HIV/HCV co-infected patient without opportunistic infections
Conclusions: These data suggest that the expansion and lytic capacity of the CD56bright NK subset may be involved in the protection of this [left-pointing double angle quotation mark] rare [right-pointing double angle quotation mark] HIV/HCV co-infected hemophiliac A patient from opportunistic infections and virus-related cancers despite very low CD4+ cell counts. (Source: Virology Journal)
Source: Virology Journal - January 26, 2013 Category: Virology Authors: Giulia FregniAnaenza MarescaValérie JalbertAnne CaignardDaniel Scott-AlgaraElisabeth CramerElisabeth RouveixMarie BénéClaude Capron Source Type: research

Perioperative management of a hemophilia B infant with intracranial hemorrhage
Sarika Juneja, Pragati Ganjoo, Monica S Tandon, Ajay SharmaJournal of Pediatric Neurosciences 2012 7(3):239-240 (Source: Journal of Pediatric Neurosciences)
Source: Journal of Pediatric Neurosciences - January 25, 2013 Category: Neuroscience Authors: Sarika JunejaPragati GanjooMonica S TandonAjay Sharma Source Type: research

Baxter Announces Agreement to Acquire Investigational Hemophilia Compound OBI-1 and Related Assets
DEERFIELD, Ill.--(BUSINESS WIRE)--Jan 24, 2013 - Baxter International Inc. (NYSE:BAX) announced today that it has agreed to acquire the investigational hemophilia compound OBI-1 and related assets from Inspiration BioPharmaceuticals, Inc. as well as... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - January 24, 2013 Category: Pharmaceuticals Source Type: news

Novel electric power-driven hydrodynamic injection system for gene delivery: safety and efficacy of human factor IX delivery in rats
Liu & Y Aoyagi (Source: Gene Therapy)
Source: Gene Therapy - January 24, 2013 Category: Genetics & Stem Cells Authors: T YokooK KamimuraT SudaT KanefujiM OdaG ZhangD LiuY Aoyagi Tags: gene delivery hydrodynamic gene delivery hemophilia human factor IX Source Type: research

Plasmatic tissue factor pathway inhibitor is a major determinant of clotting in factor VIII inhibited plasma or blood.
Abstract Tissue factor pathway inhibitor (TFPI) is a major inhibitor of coagulation. We therefore hypothesised that high plasmatic TFPI levels are associated with impaired ex vivo clotting in a model of acquired haemophilia. Blood samples were collected in a prospective clinical study from 30 healthy volunteers. Coagulation in normal or factor VIII (FVIII)-inhibited human blood or plasma was measured by the calibrated automated thrombogram (CAT) and rotational thromboelastometry (ROTEM). Both methods are global haemostatic assays that provide insight into the whole coagulation process. Monoclonal mouse antibodies r...
Source: Thrombosis and Haemostasis - January 24, 2013 Category: Hematology Authors: Knappe S, Gorczyca ME, Jilma B, Derhaschnig U, Hartmann R, Palige M, Scheiflinger F, Dockal M Tags: Thromb Haemost Source Type: research

Response to desmopressin is strongly dependent on F8 gene mutation type in mild and moderate haemophilia A.
Response to desmopressin is strongly dependent on F8 gene mutation type in mild and moderate haemophilia A. Thromb Haemost. 2013 Jan 24;109(3) Authors: Stoof SC, Sanders YV, Petrij F, Cnossen MH, de Maat MP, Leebeek FW, Kruip MJ Abstract Desmopressin causes two- to six-fold increase of factor VIII (FVIII) in mild or moderate haemophilia A patients. However, responses are variable and little is known whether this is associated with F8 gene mutation. The study objective was to assess the relationship between F8 gene mutation and desmopressin response in haemophilia A patients. Desmopressin response (absolute ...
Source: Thrombosis and Haemostasis - January 24, 2013 Category: Hematology Authors: Stoof SC, Sanders YV, Petrij F, Cnossen MH, de Maat MP, Leebeek FW, Kruip MJ Tags: Thromb Haemost Source Type: research

Haemophilia at various stages of life: design of new therapeutic strategies through an interactive course - the Kogeniale project.
This article describes a course to educate young physicians who were already engaged in the management of haemophilia on the emerging and unmet issues of haemophilia care and to implement existing guidelines. Physicians (n=53) already caring for patients with haemophilia in their haematology, internal medicine, or paediatric practices in Italy attended the course. Problem-solving group activity and open discussion were the methods chosen to formulate consensus statements. During the specifically designed interactive course, three clinical cases were simulated: a young child with congenital dislocation of the hip, an adoles...
Source: Blood Transfusion - January 23, 2013 Category: Hematology Authors: Santagostino E, Messina M, Tagliaferri A, Iorio A, Morfini M Tags: Blood Transfus Source Type: research

Programme
(Source: Haemophilia)
Source: Haemophilia - January 22, 2013 Category: Hematology Tags: Programme Source Type: research

Abstracts of the 6th Annual Congress of the European Association for Haemophilia and Allied Disorders
(Source: Haemophilia)
Source: Haemophilia - January 22, 2013 Category: Hematology Tags: Speaker Abstracts Source Type: research

Poster Presentation
(Source: Haemophilia)
Source: Haemophilia - January 22, 2013 Category: Hematology Tags: Poster Presentation Source Type: research

Author Index
(Source: Haemophilia)
Source: Haemophilia - January 22, 2013 Category: Hematology Tags: Author Index Source Type: research

Hemophilia clinical gene therapy-brief review.
Abstract Genetic correction of hemophilia A and B was long considered amenable to the available gene transfer technologies. This assumption has come to fruition with the recent results of a phase I/II trial for hemophilia B. Here we review the clinical application of gene therapy for the hemophilia's as a paradigm of the evolution of gene transfer science and technology. This review is not intended as comprehensive but rather to highlight current clinical developments of gene therapy for the hemophilias. PMID: 23352600 [PubMed - as supplied by publisher] (Source: Translational Research : the journal of laborato...
Source: Translational Research : the journal of laboratory and clinical medicine - January 22, 2013 Category: Laboratory Medicine Authors: Walsh CE, Batt KM Tags: Transl Res Source Type: research

CSL Behring commences patient enrolment in paediatric Phase III haemophilia B study
CSL Behring has commenced patient enrolment in its pivotal paediatric Phase III Haemophilia B study designed to assess the recombinant fusion protein linking coagulation factor IX with recombinant albumin (rIX-FP) for its safety, efficacy and pharmac… (Source: Drug Development Technology)
Source: Drug Development Technology - January 21, 2013 Category: Pharmaceuticals Source Type: news