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Hemophilia A (Haemophilia)

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The classic hemophilia resulting from a deficiency of factor VIII. It is an inherited disorder of blood coagulation characterized by a permanent tendency to hemorrhage.

Also Known As:

Haemophilia; Hemophilia; Factor VIII Deficiency; Classic Hemophilia; Deficiency, Factor VIII; Factor 8 Deficiency, Congenital; Factor VIII Deficiency, Congenital; Hemophilia A, Congenital; Hemophilia, Classic; A, Hemophilia; As, Hemophilia; Classic Hemophilias; Congenital Hemophilia A; Congenital Hemophilia As; Deficiencies, Factor VIII; Factor VIII Deficiencies; Hemophilia As; Hemophilia As, Congenital; Hemophilias, Classic

Networked: 6254 relevant articles (365 outcomes, 626 trials/studies)

Relationship Network

Disease Context: Research Results

Related Diseases

1.	Hemorrhage
2.	Hemophilia A (Haemophilia)
3.	von Willebrand Diseases (von Willebrand's Disease)
4.	Hemophilia B (Haemophilia B)
5.	Infection

Experts

1.	Oldenburg, J: 54 articles (10/2015 - 01/2000)
2.	Franchini, Massimo: 52 articles (07/2015 - 11/2003)
3.	Shima, Midori: 34 articles (12/2015 - 03/2002)
4.	Santagostino, E: 30 articles (03/2015 - 10/2001)
5.	Shima, M: 29 articles (10/2015 - 07/2000)
6.	Berntorp, E: 29 articles (07/2015 - 10/2000)
7.	Mannucci, P M: 29 articles (09/2014 - 01/2000)
8.	Oldenburg, Johannes: 26 articles (10/2015 - 06/2002)
9.	Berntorp, Erik: 25 articles (06/2015 - 11/2002)
10.	Lillicrap, David: 24 articles (05/2015 - 09/2002)

Drugs and Biologics

Drugs and Important Biological Agents (IBA) related to Hemophilia A:

1.	Factor VIII (Coagulation Factor VIII)IBA 03/01/2013 - "In patients with hemophilia A, outcomes have improved dramatically over the last few decades due to several advances in care, including the availability of factor VIII (FVIII) concentrates. " 01/01/2009 - "Because factor VIII (FVIII) inhibitor formation is the most serious treatment-related complication for hemophilia A patients, we tested the efficacy of anti-CD3 to prevent FVIII inhibitor formation in hemophilia A BALB/c and C57BL/6 mice. " 05/15/1989 - "Factor VIII-CVP therapy is highly effective in the eradication of factor VIII inhibitor in nonhemophiliac patients but not in patients with hemophilia." 11/01/1999 - "These results suggest that PE is very effective in treating factor VIII inhibitor-positive acquired hemophilia." 01/01/2013 - "Prophylaxis may be beneficial for patients with severe haemophilia A who have developed inhibitors to factor VIII. The aim of this study was to determine physicians' preferences for medication attributes in the prophylactic treatment of this patient population. "
2.	recombinant FVIIa (rFVIIa)IBA 01/11/2011 - "Clinically significant benefits of recombinant factor VIIa as a general hemostatic agent in patients without hemophilia remain unproven. " 03/01/2008 - "rFVIIa has been shown effective for prophylaxis during elective surgery and has therefore improved the feasibility of orthopedic surgery in hemophilia patients with high-titer inhibitors. " 10/01/2003 - "In conclusion, should a TF-independent mechanism be involved in the efficacy of rFVIIa in patients with hemophilia, the superactive rFVIIa variants studied here might be clinically advantageous, as both procoagulant and antifibrinolytic potencies are significantly enhanced compared with those of wild-type rFVIIa. " 01/01/2012 - "Regarding patients with inhibitors, the advent of APCCs and rFVIIa has made major orthopaedic surgery possible, leading to an improved quality of life for haemophilia patients. " 09/01/2002 - "We have prospectively monitored treatment of haemophilia patients with inhibitors by recombinant factor VIIa (rFVIIa) administered by continuous infusion to obtain more insight in the underlying factors of the clinical efficacy of this administration method. "
3.	Factor VIIa (Activated Factor VII)IBA 04/01/2011 - "Platelet binding and activity play important roles in the efficacy of factor VIIa (FVIIa) as a bypassing agent for hemophilia treatment. " 10/26/2001 - "Factor VIIa is also an important therapeutic agent for hemophilia where its function may include TF-independent as well as TF-dependent mechanisms. " 05/01/2013 - "Safety and efficacy of recombinant activated factor VII for long-term secondary prophylaxis in a haemophilia boy with high-titre inhibitors." 08/01/1998 - "[Recombinant activated factor VII: a new treatment for hemophilia]." 10/01/2014 - "Evaluating and monitoring the efficacy of recombinant activated factor VIIa in patients with haemophilia and inhibitors."
4.	AntibodiesIBA 07/01/2008 - "Currently, the main problem in patients with haemophilia is the onset of antibodies inactivating the infused clotting factor (inhibitors), even though immune tolerance regimens that are able to eradicate inhibitors and the availability of products that bypass the intrinsic coagulation defects have dramatically improved the management of these patients. " 03/01/2012 - "Reduced activity in vitro and improved retention of activity in the presence of antibodies suggested strong shielding of FVIII by the particle; thus, in vivo studies were conducted in hemophilia A mice. " 05/01/2015 - "There remains a substantial unmet medical need in hemophilia, especially in patients with inhibitory antibodies against replacement factor therapy, for novel and improved therapeutic agents that can be used prophylactically to provide effective hemostasis. " 01/16/2014 - "Our previous studies have demonstrated that platelet FVIII (2bF8) gene therapy can improve hemostasis in hemophilia A mice, even in the presence of inhibitory antibodies, but none of our studies has targeted human cells. " 01/06/2014 - "This study demonstrates that a combination regimen is highly promising as a treatment option for modulating anti-FVIII antibodies and facilitating induction of long-term tolerance to FVIII in hemophilia A mice. "
5.	Deamino Arginine Vasopressin (Desmopressin)FDA LinkGeneric 05/01/2002 - "We conclude that administration of 150 microg of high concentration intranasal desmopressin is safe and effective in patients weighing < or = 50 kg with mild hemophilia A or mild type 1 VWD." 09/01/2011 - "DDAVP is effective in the treatment of mild hemophilia A. " 09/01/2002 - "Desmopressin is not always effective in mild haemophilia A patients undergoing urological surgery: the need of standardized protocols." 09/01/2011 - "[Clinical efficacy of desmopressin in the treatment of mild hemophilia A in children]." 10/28/1987 - "Concentrated DDAVP: further improvement in the management of mild factor VIII deficiencies."
6.	rituximab (Mabthera)FDA Link 05/01/2006 - "Rituximab may be beneficial for patients with severe haemophilia and inhibitors in whom standard therapies have failed, but larger prospective studies are required to determine safety, efficacy and predictors of success." 06/01/2014 - "This case series demonstrates that rituximab is a useful first-line treatment to achieve sustained inhibitor eradication in adult patients with non-severe hemophilia A." 09/01/2011 - "Is a reduced intensity treatment with Rituximab effective in acquired haemophilia A?" 11/01/2010 - "Rituximab appears an effective and well-tolerated treatment for patients with acquired haemophilia." 01/01/2010 - "Recently, a therapy with rituximab, an anti-CD20 monoclonal antibody, has shown to be effective in acquired haemophilia."
7.	Blood Coagulation Factors (Coagulation Factor)IBA 11/01/2014 - "Studies conducted before the advent of safe and effective coagulation factor replacement therapy suggest that the haemophilia population was predominantly of low socioeconomic status with many social disadvantages, including high rates of disability and unemployment and low rates of marriage. " 08/01/2010 - "Canine models have been good predictors of efficacy of hemophilia treatments, including recombinant human coagulation factor (F)VIIa (hFVIIa). " 01/01/2006 - "Plasma-derived concentrates of coagulation factors VIII and IX allow for effective treatment of haemophilia A and B. " 12/28/2002 - "It has been shown that this treatment is effective in patients with haemophilia and an inhibiting antibody against a coagulation factor. " 06/01/2000 - "The availability of new safe coagulation factors has improved the prognosis of haemophilia."
8.	Factor IX (PTC)FDA LinkGeneric 07/01/1999 - "Since in most countries carrier detection is based on factor IX coagulant activity (FIX:C) assay, this study was designed to determine whether carriers' FIX:C values are dependent on the severity of haemophilia (mild, moderate or severe) or on the genetic anomaly in the family. " 08/10/1997 - "In the case of factor IX, we have produced a high-titer adenovirus vector capable of transducing gut epithelial cells resulting in synthesis of factor IX. The results of our in vitro studies indicate that gene transfer targeting gut epithelium as a new approach to hemophilia gene therapy is rational and merits in vivo studies in hemophilia animal models." 07/01/1995 - "Gene therapy is a potential treatment for hemophilia, wherein cells transduced with a normal factor IX gene could provide a continuous in vivo source of circulating factor IX. In this study, we examined the potential use of hematopoietic cells as a target for factor IX gene therapy. " 05/01/1953 - "[Studies on a new hemorrhagic diseases similar to hemophilia; plasma thromboplastin component deficiency]." 06/01/2015 - "In addition, recent advances in recombinant factor IX protein engineering have led some hemophilia treaters to reconsider the urgency of genetic cure. "
9.	FeibaIBA 04/01/2007 - "Excellent hemostasis was obtained in all cases, adding to accumulating data indicating that FEIBA is safe and effective in hemophilia patients with inhibitors who require surgery." 01/01/2014 - "Given the high risk for morbidity and mortality in haemophilia patients with inhibitors to FVIII or FIX, we conducted this Phase 3 prospective study to evaluate whether prophylaxis with FEIBA NF is a safe and effective treatment option. " 11/01/2009 - "FEIBA provides an effective and safe first-line peri- and postoperative haemostatic therapy for patients with haemophilia A and inhibitors, allowing both major and minor operations to be successfully performed." 05/01/2015 - "FEIBA prophylaxis was effective for preventing joint bleeds and subsequent joint damage, delaying arthropathy and improving outcomes in children with haemophilia A and inhibitors to FVIII, who failed or were ineligible for ITI. " 09/01/2014 - "This study investigates whether 12-month of FEIBA prophylaxis improved HRQoL in haemophilia patients with inhibitors. "
10.	Prothrombin (Factor II)IBA 04/01/2012 - "We report here the case of a boy with hemophilia A and high titers of inhibitors who responded well to prophylactic activated prothrombin complex concentrate (APCC) treatment. " 01/01/1950 - "Studies on serum prothrombin in hemophilia." 12/01/2012 - "Contact-inhibited plasmas from six healthy volunteers, one mild haemophilia A patient, and five patients with heterozygous prothrombin G20210 mutation were assayed. " 10/01/2012 - "Acquired hemophilia successfully treated with activated prothrombin complex concentrate and immunosuppressant combination: a case report." 04/01/2012 - "Cost effectiveness of prophylactic treatment with activated prothrombin complex concentrate in a patient with inhibitor-positive hemophilia A."

Therapies and Procedures

1.	Liver Transplantation 07/01/2008 - "Liver transplantation is the only potentially curative treatment available for these patients, providing a long-term phenotypic cure for hemophilia. " 01/01/2012 - "We focus on those aspects that are of specific interest in people with haemophilia: studies performed in haemophilia populations, the use of invasive diagnostic and therapeutic tools and the outcome of liver transplantation. " 10/01/2005 - "An ideal setting to study this problem is in individuals with hemophilia A following liver transplantation, as their FVIII is synthesized primarily in hepatic, but not extrahepatic endothelial cells, while VWF is synthesized primarily in extrahepatic vascular endothelium. " 01/01/2015 - "Long-term outcome of liver transplantation in HCV/HIV coinfected haemophilia patients. " 01/01/2015 - "Auxiliary partial orthotopic liver transplantation as a treatment for hemophilia a: a case report."
2.	Transplantation (Transplant Recipients) 06/15/2011 - "Hemophilia was clinically cured after successful transplantation. " 10/01/2008 - "Our further studies showed that transplantation with 1% to 5% 2bF8(tg+/-) BM cells still improved hemostasis in hemophilia A mice with inhibitors. " 02/01/2007 - "In this study, we assessed the efficacy of using a genetic therapy approach to correct haemophilia A in FVIII-deficient (FVIII(null)) mice by transplantation of bone marrow (BM) transduced with a lentivirus (LV)-based gene transfer cassette encoding 2bF8. " 12/05/2014 - "Based on the existing data and current rate of progress, clinical trials of HSC transplantation gene therapy for hemophilia are predicted to begin in the coming years. " 05/15/2006 - "In this study, we evaluated ex vivo retroviral gene transfer of a high-expression porcine fVIII transgene into bone marrow-derived stromal and hematopoietic stem/progenitor cells (MSCs and HSCs, respectively) and transplantation into genetically immunocompetent hemophilia A mice. "
3.	Transplants (Transplant) 01/01/2015 - "There are many documented cases of a person with haemophilia successfully receiving a solid organ transplant, including liver and kidney. " 09/01/2012 - "Is it justifiable to transplant infected livers into haemophilia recipients?" 04/01/2011 - "The main impediments to clinical application of haematopoietic stem cell (HSC) gene therapy for treatment of haemophilia A are the bone marrow transplant-related risks and the potential for insertional mutagenesis caused by retroviral vectors. " 05/01/2005 - "However, it is not known whether the graft of APOLT would be enough to cure hemophilia. " 06/27/2002 - "Furthermore, a graft from a living-related donor with hemophilia A carrier seems to be suitable provided such individuals receive adequate support for coagulopathies."
4.	Injections 08/01/2008 - "In conclusion, rAHF-PFM administered via continuous infusion or bolus injections is safe, non-immunogenic, and effective for perioperative hemostatic management in previously treated haemophilia A patients." 01/01/2010 - "A total of 250 patients will be enrolled with severe haemophilia A (<1% FVIII) on on-demand or secondary prophylaxis treatment and with documented bleeds or injections during the 6 months before study entry. " 09/01/2006 - "The lack of evidence-based information on the hemostatic levels to be maintained in specific clinical situations, and recent concerns regarding the development of inhibitors, particularly in patients with mild hemophilia treated with continuous infusion, need to be addressed by prospective, randomized studies that compare traditional intermittent injections and the continuous infusion mode of factor replacement." 11/01/1938 - "STUDIES ON THE REFRACTORY PHASE FOLLOWING REPEATED INJECTIONS OF GLOBULIN SUBSTANCE DERIVED FROM NORMAL HUMAN PLASMA IN HEMOPHILIA." 06/01/2015 - "Hemophilia remains a non-curative disease, and patients are constrained to undergo repeated injections of clotting factors. "
5.	Therapeutics 08/14/2014 - "Because mFVIIa-FMR models the TF-dependent and EPCR binding properties of rhFVIIa, our data unmask a novel contribution of EPCR on the action of rhFVIIa administration in hemophilia, prompting the rational design of improved and safer rhFVIIa therapeutics. " 07/01/2012 - "Models for assessing immunogenicity and efficacy of new therapeutics for the treatment of haemophilia." 03/01/2015 - "Research in the area of haemophilia therapeutics, however, is not restricted only to modifications in the recombinant products, but alternate therapeutic strategies are being developed which are in different phases of experimental and clinical trials. " 01/01/2014 - "The Committee of Latin America on the Therapeutics of Inhibitor Groups conducted a survey of its member country representatives on key aspects of haemophilia treatment in 10 countries. " 03/01/2012 - "We suggest that ranking haemophilia related interventions with the standard interventions of therapeutics and public health in CUA comparisons is inappropriate. "