|2.||Hemophilia A (Haemophilia)
|3.||Body Weight (Weight, Body)
|5.||Disease Susceptibility (Diathesis)
|1.||High, Katherine A: 34 articles (03/2015 - 04/2002)|
|2.||Herzog, Roland W: 25 articles (11/2015 - 04/2002)|
|3.||Nichols, Timothy C: 17 articles (03/2015 - 04/2002)|
|4.||Mingozzi, Federico: 16 articles (11/2014 - 01/2004)|
|5.||Arruda, Valder R: 13 articles (03/2015 - 04/2002)|
|6.||High, K A: 12 articles (08/2010 - 03/2000)|
|7.||Zhou, Shangzhen: 11 articles (01/2014 - 03/2006)|
|8.||Pierce, Glenn F: 10 articles (01/2015 - 01/2006)|
|9.||Jiang, Haiyan: 9 articles (01/2015 - 03/2006)|
|10.||Herzog, R W: 9 articles (10/2005 - 03/2000)|
|1.||Factor IX (PTC)FDA LinkGeneric
01/01/1993 - "This direct approach, which is based on computer-aided analysis of the whole coding, promoter and exon-flanking factor IX gene sequences, proved to be helpful for carrier detection and prenatal diagnosis in most hemophilia B families, including sporadic cases. "
10/01/2008 - "Investigation of factor IX (FIX) has benefited from excellent gene-deleted mouse models that recapitulate many of the features of human haemophilia B. "
10/01/2014 - "Hemophilia B management has improved considerably since the introduction of high-purity plasma-derived factor IX (pdFIX) products in the early 1990s. "
09/01/2013 - "The rapid clearance of factor IX (FIX) necessitates frequent intravenous administration to achieve effective prophylaxis for patients with hemophilia B. "
03/01/1992 - "The American Red Cross has developed an immunoaffinity chromatography method to purify human coagulation Factor IX to high levels of purity for therapeutic treatment of hemophilia B. "
|2.||prothrombin complex concentrates (PPSB)IBA
03/01/1999 - "Thus, continuous infusion with this highly purified factor IX concentrate with improved viral safety is effective for surgery in haemophilia B."
05/01/2008 - "OCTANINE F is a high-purity blood clotting factor IX concentrate that has been shown to be effective and safe in adults with haemophilia B. "
01/01/1996 - "The safety and efficacy of a monoclonal antibody purified factor IX concentrate were evaluated in two continuing trials of 32 previously untreated patients with mild, moderate, or severe hemophilia B. "
01/01/1996 - "Safety and efficacy of monoclonal antibody purified factor IX concentrate in previously untreated patients with hemophilia B."
07/01/2011 - "A clinical study assessing the pharmacokinetics, efficacy and safety of AlphaNine(®) , a high-purity factor IX concentrate, in patients with severe haemophilia B."
12/15/2003 - "The 7 of 12 tolerized hemophilia B dogs exhibited shortened whole blood clotting times (WBCTs), sustained detectable FIX antigen, undetectable Bethesda inhibitors, transient or no detectable antihuman FIX antibody titers by enzyme-linked immunosorbent assay (ELISA), and normal clearance of infused rhFIX. "
05/15/2003 - "Three newborn hemophilia B dogs that were injected intravenously with RV achieved 12% to 36% of normal cFIX antigen levels, which improved coagulation tests. "
08/01/2012 - "In hemophilia B dogs, human FIX antigen levels remained above 0.05 IU mL(-1) more than three times longer after rIX-FP (7.3 days) compared with rFIX (2.3 days), whereas respective calculations based on activity levels confirmed the observed superior profile. "
08/01/2010 - "Protein infusion of FIX-Triple into haemophilia B mice was not thrombogenic, even at a dose of 13-fold higher than FIX-WT. Gene knock-in to generate mice that constitutively produce FIX-WT or FIX-Triple protein revealed that all mice expressed equal antigen levels. "
01/01/2006 - "This mutation causes mild hemophilia B with approximately 25% FIX coagulant activity and FIX antigen levels of around 90% of normal. "
|4.||Factor VIII (Coagulation Factor VIII)IBA
10/01/1998 - "The use of dilution studies, chromogenic assays, a novel in-house enzyme-linked-immunosorbent-assay-based technique and phospholipid neutralization, demonstrated that Case 1 had a genuine factor VIII inactivator resulting in factor VIII levels of less than 1 IU/dl but no factor IX deficiency. "
05/01/2014 - "While the recent results in haemophilia B are extremely encouraging, there is, as yet, no similar data for factor VIII gene therapy. "
09/01/2011 - "The conditions result in various degrees of factor VIII or factor IX deficiency, respectively. "
12/29/1995 - "Transfected stromal cells may serve as suitable vehicles for gene delivery to correct single gene disorders in which the product of the target gene does not require stringent regulation as, for example, in the correction of Factor VIII and Factor IX deficiency. "
05/01/1995 - "Natural canine models of factor VIII and factor IX deficiency have been available for many years, and gene therapy attempts on these dogs have met with partial success. "
|5.||Complementary DNA (cDNA)IBA
03/01/2014 - "Overall, this study demonstrates that IDLVs carrying an improved human FIX cDNA safely and efficiently cure hemophilia B in a mouse model. "
08/01/1997 - "The regulation of hFIX cDNA expression in myoblasts was discussed and it was strongly suggested that a myoblast-mediated gene delivery system had the potential to be optimized as a safe and effective therapeutic modality for hemophilia B."
03/01/2003 - "It was found that while mutations responsible to account for all 2348 haemophilia B patients covered 20% of the FIX cDNA, only 1% of the cDNA involving mostly CpG dinucleotides accounted for mutation in 42.41% of the patient pool. "
12/01/1993 - "Injection of the vector Av1H9B, which encodes human factor IX cDNA, into the tail veins of mice resulted in efficient liver transduction and plasma levels of human factor IX that would be therapeutic for haemophilia B patients. "
06/01/2008 - "The two initial phase I/II AAV clinical trials for hemophilia B, delivering a factor IX cDNA to skeletal muscle or liver, showed no serious adverse events. "
01/01/1995 - "These studies indicate that Mononine is safe and effective in the treatment of hemorrhagic episodes in patients with hemophilia B."
02/01/1992 - "Mononine was evaluated for in vivo recovery, half-life, and for its safety and efficacy in 10 patients with hemophilia B. "
01/01/1995 - "Pharmacokinetic variables were studied in severe hemophilia B patients: Nanotiv was compared with Preconativ; Immunine was compared with Prothromplex TIM4 in crossover studies; and Mononine was tested in a single-drug study. "
11/15/1993 - "In vivo, Nanotiv was compared with Preconativ and Immunine with Prothromplex TIM4 in crossover studies in patients with severe hemophilia B, and Mononine was tested in a single drug study. "
05/01/1995 - "Monoclonal antibody purified factor IX concentrate, Mononine (Armour Pharmaceutical Company, Kankakee, Illinois, USA), is a recently developed replacement factor concentrate for the treatment of patients with hemophilia B. "
05/01/2012 - "Since FIX expression in platelets is effective for hemophilia B, efficacy in the presence of inhibitory antibodies to FIX was not achieved and emphasized the importance of VWF to the efficacy of platelet FVIII expression. "
11/30/1976 - "This paper presents the results of an immunological study of hemophilia B and its carriers which used two kinds of antibodies, a heterologous antibody of high specificity and a homologous antibody developed in a patient with severe hemophilia B. "
01/01/2015 - "Unfortunately, the overall reported success of immune tolerance induction in FIX deficiency with inhibitors is approximately 25-40%.We report the case of a 2-year-old boy with hemophilia B severe FIX deficiency (<1%), inhibitor antibodies to FIX development, and a history of adverse reactions to FIX infusions, who underwent a successful desensitization and immune tolerance induction with a daily FIX infusion. "
05/01/2012 - "Successful immune tolerance induction in two boys with haemophilia B and inhibitory antibodies."
05/01/2007 - "Achievement of immune tolerance in a patient with haemophilia B and inhibitory antibodies, complicated by an anaphylactoid reaction."
|8.||recombinant FVIIa (rFVIIa)IBA
01/01/2014 - "We present unique case of patient with hemophilia B and high titer inhibitors to coagulation FIX, who developed severe renal damage due to thromboembolic event during rFVIIa therapy, associated with unsuspected renovascular anomalies. "
11/01/2012 - "Arthroscopic synovectomy of the elbow covered with rFVIIa in a haemophilia B juvenile with inhibitor."
11/01/2011 - "Diagnosis of acquired haemophilia B confirmed, patient received recombinant factor VIIa and corticosteroid treatment. "
08/01/2011 - "The successful use of high dose FIX followed by recombinant FVIIa suggests that even major surgery could be safely performed in hemophilia B patients with a low titer of high responding inhibitors."
08/01/2011 - "The pGlcNAc nanofibers amplify rFVIIa activity in hemophilia B canine blood by activating platelets through integrin-dependent mechanisms."
|9.||Tranexamic Acid (AMCA)FDA Link
05/06/1972 - "In a double-blind trial tranexamic acid (AMCA, Cyclokapron), 1 g three times a day for five days, significantly reduced blood loss and transfusion requirements after dental extraction in patients with haemophilia and Christmas disease. "
05/06/1972 - "Use of tranexamic acid in control of haemorrhage after extraction of teeth in haemophilia and Christmas disease."
05/06/1972 - "Tranexamic acid in control of haemorrhage after dental extraction in haemophilia and Christmas disease."
05/06/1972 - "Bleeding after dental extraction was controlled with tranexamic acid in 19 patients with haemophilia and 3 with Christmas disease. "
04/30/1976 - "Re: tranexamic acid and bleeding: a double-blind cross-over study on three brothers with Christmas disease (factor IX deficiency)."
|10.||6-Aminocaproic Acid (6 Aminocaproic Acid)FDA LinkGeneric
05/01/1971 - "Epsilon-Aminocaproic acid therapy for dental extractions in haemophilia and Christmas disease: a double blind controlled trial."
01/02/1968 - "Epsilon-aminocaproic acid in the treatment of haemophilia and Christmas disease with special reference to the extraction of teeth."
02/01/1996 - "This study evaluated the safety and efficacy of combined treatment with epsilon-aminocaproic acid or tranexamic acid and monoclonal antibody purified factor IX (MAb factor IX) for prophylaxis against bleeding in eight hemophilia B patients undergoing nine dental extraction procedures. "
01/01/1995 - "Concomitant use of the monoclonal antibody-purified factor IX concentrate (Mononine, Armour Pharmaceutical Company, Collegeville, Pa.) and two antifibrinolytic agents, epsilon-aminocaproic acid (EACA; Amicar, Immunex, Seattle, Wash.) or tranexamic acid (AMCA; Cyklokapron, Kabi Pharmacia, Piscataway, N.J.) was examined for safety and efficacy in patients with hemophilia B. "
03/15/2005 - "Importantly, intramuscular injection of AAV-F.IX variants did not trigger antibody formation to F.IX in mice tolerant to F.IX-WT. These studies demonstrate that F.IX variants provide a promising strategy to improve the efficacy for a variety of gene-based therapies for hemophilia B."
04/15/2003 - "Previously, we established an experimental basis for gene transfer as a method of treating the disease in mice and hemophilic dogs through intramuscular injection of a recombinant adeno-associated viral (rAAV) vector expressing F.IX. In this study we investigated the safety of this approach in patients with hemophilia B. "
04/15/2000 - "Recent studies have shown that intramuscular injection of an adeno-associated viral (AAV) vector into mice and hemophilia B dogs results in vector dose-dependent, long-term expression of biologically active F.IX at therapeutic levels. "
03/01/2000 - "On the basis of these data and additional pre-clinical studies demonstrating an absence of vector-related toxicity, we initiated a clinical study of intramuscular injection of an AAV vector expressing human F.IX in adults with severe haemophilia B. "
05/01/2009 - "Direct intramuscular injection (IM) of adeno-associated virus (AAV) has been proven a safe and potentially efficient procedure for gene therapy of many genetic diseases including hemophilia B. "
|2.||Transplantation (Transplant Recipients)
07/15/2008 - "The aim of this study was to investigate the efficacy of hepatocyte transplantation in the mouse model of hemophilia B. "
12/01/2006 - "5. The results of the present study suggest that transplantation of HSC results in the persistant expression of hFIX in mice, which may be useful in haemophilia B gene therapy."
07/15/2008 - "These results demonstrate that hepatocyte transplantation can provide therapeutic benefits in the treatment of hemophilia B."
07/15/2008 - "Therapeutic effects of hepatocyte transplantation on hemophilia B."
04/01/2007 - "Here, we report the observation of six T-lymphoblastic leukemia cases arising during the course of a gene therapy study for hemophilia B after transplantation of ex vivo transduced hematopoietic stem cells (HSCs) by a lentivirus vector. "
|3.||Tissue Therapy (Cell Therapy)
05/01/2013 - "Human amniotic fluid derived progenitor cells (hAFPCs) may be multipotent and can be considered a potential tool in the field of cell therapy for haemophilia B. "
01/01/2010 - "The aim of this study was to evaluate therapeutic efficacy of cell therapy using hemophilia B (HB) as a disease model by transplanting FIX-Triple-secreting hepatocytes. "
08/01/2010 - "Currently, treatment of hemophilia B is performed by intravenous infusion of plasma-derived or recombinant FIX. "
09/01/2001 - "Here, we describe hemophilia B mice with a large F9 deletion that form inhibitors within 1 to 2 months after IM administration of an AAV vector expressing mouse F9 or after repeated intravenous infusion of mouse F9 concentrate. "
01/01/2014 - "Hemophilia B is an inherited coagulopathy caused by the partial or complete deficiency of factor (F) IX. Factor replacement therapy, involving the intravenous infusion of plasma-derived or recombinant (r) FIX concentrate, is the cornerstone of treatment, used to control and prevent bleeding episodes. "
08/01/1997 - "It involves intravenous infusion of 25-40 factor units per kg on alternate days (minimum 3 times a week) for boys with severe hemophilia A, and twice a week for boys with severe hemophilia B. "
10/01/2014 - "Current therapy requires frequent intravenous infusions of therapeutic recombinant or plasma-derived protein concentrates containing Factor IX. Alprolix™ (recombinant Factor IX Fc fusion protein), is a therapeutic Factor IX preparation that has been engineered for a prolonged half-life in circulation, has completed pivotal clinical trials and has been approved recently in the USA, Canada, Australia and Japan for use in the clinic for patients with hemophilia B. "
08/01/1995 - "Previous studies have shown that the delivery of a recombinant adenoviral vector expressing canine FIX (cFIX) resulted in a complete correction of hemophilia B in FIX-deficient dogs, but that cFIX expression decreased to only about 1-2% of normal levels 3 weeks after treatment. "
07/01/2011 - "We report a case of spontaneous spinal EDH in a 5-year-old male child with Hemophilia B, who was managed conservatively and was doing well at last follow-up, 2 years after treatment. "
05/01/1985 - "In a patient with severe haemophilia B and antibodies against factor IX in high titre, and known for many years to be a really high responder, it was possible to suppress the secondary antibody response after treatment with high doses of intravenous IgG (Gammonativ, KabiVitrum AB) combined with factor IX and cyclophosphamide. "