The past and future of haemophilia: diagnosis, treatments, and its complications.

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The past and future of haemophilia: diagnosis, treatments, and its complications. is …
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scholarly articleQ13442814

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P356DOI10.1016/S0140-6736(15)01123-X
P698PubMed publication ID26897598

P50authorFlora PeyvandiQ39183632
P2093author name stringGuy Young
Isabella Garagiola
P2860cites workF8 gene mutation type and inhibitor development in patients with severe hemophilia A: systematic review and meta-analysisQ26827191
Origins and Mechanisms of miRNAs and siRNAsQ27860822
Inversions disrupting the factor VIII gene are a common cause of severe haemophilia AQ28117400
Sites of initial bleeding episodes, mode of delivery and age of diagnosis in babies with haemophilia diagnosed before the age of 2 years: a report from The Centers for Disease Control and Prevention's (CDC) Universal Data Collection (UDC) projectQ33488039
Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatmentQ33535682
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Efficient production of dual recombinant adeno-associated viral vectors for factor VIII deliveryQ34082003
Perinatal management of newborns with haemophiliaQ34135323
The hemophilias--from royal genes to gene therapyQ34276237
A CpG mutational hotspot in a ONECUT binding site accounts for the prevalent variant of hemophilia B LeydenQ34331863
Hemophilia B Leyden and once mysterious cis-regulatory mutationsQ34378589
Randomized comparison of prophylaxis and on-demand regimens with FEIBA NF in the treatment of haemophilia A and B with inhibitors.Q34436796
Recovery from Hemophilia B Leyden: An Androgen-Responsive Element in the Factor IX PromoterQ34471218
Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe hemophilia A, 2000-2011Q34580100
Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophiliaQ34661163
Genetic sequence analysis of inherited bleeding diseasesQ34663589
Haemophilia B mutations in Sweden: a population-based study of mutational heterogeneityQ45876762
Mutation analysis of haemophilia B in the Irish population: increased prevalence caused by founder effectQ45877553
Changes in treatment strategies for severe haemophilia over the last 3 decades: effects on clotting factor consumption and arthropathyQ45879749
Guidelines for the management of hemophiliaQ45880345
Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B.Q45880382
Analysis of mRNA in hemophilia A patients with undetectable mutations reveals normal splicing in the factor VIII gene.Q45880854
The North American Immune Tolerance Registry: contributions to the thirty-year experience with immune tolerance therapyQ45881127
Population pharmacokinetic modeling for dose setting of nonacog beta pegol (N9-GP), a glycoPEGylated recombinant factor IX.Q45881987
Recurrent inversion breaking intron 1 of the factor VIII gene is a frequent cause of severe hemophilia A.Q45882168
A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A modelQ45882197
Identification of deletion carriers in hemophilia B: quantitative real-time polymerase chain reaction or multiple ligation probe amplificationQ45883198
Short-term low-dose secondary prophylaxis for severe/moderate haemophilia A children is beneficial to reduce bleed and improve daily activity, but there are obstacle in its execution: a multi-centre pilot study in ChinaQ45883686
Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A.Q45884927
Identification of de novo deletion in the factor VIII gene by MLPA technique in two girls with isolated factor VIII deficiencyQ45885124
Concomitant infusion of low doses of rFVIIa and FEIBA in haemophilia patients with inhibitorsQ45885613
Variability in clinical phenotype of severe haemophilia: the role of the first joint bleed.Q45886344
Identification of factor VIII gene mutations in 101 patients with haemophilia A: mutation analysis by inversion screening and multiplex PCR and CSGE and molecular modelling of 10 novel missense substitutionsQ45886376
Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A.Q45886921
Polymorphisms in the IL10 but not in the IL1beta and IL4 genes are associated with inhibitor development in patients with hemophilia A.Q45889455
Rituximab for treatment of inhibitors in haemophilia A. A Phase II study.Q49866575
Aptamers as therapeutics.Q51813594
Haemophilia A mutations in patients with non-severe phenotype associated with a discrepancy between one-stage and chromogenic factor VIII activity assays.Q53071200
Inhibitor development in non-severe haemophilia across Europe.Q53378171
Presence of fetal DNA in maternal plasma and serumQ57075132
Inhibitor development in haemophilia according to concentrateQ59468965
Preimplantation genetic diagnosisQ80093090
Characterisation of 96 mutations in 128 unrelated severe haemophilia A patients from France. Description of 62 novel mutationsQ83120292
Fetal loss rate after chorionic villus sampling and amniocentesis: an 11-year national registry studyQ84018877
Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trialQ34737483
Long-term safety and efficacy of factor IX gene therapy in hemophilia B.Q34783922
Consensus perspectives on prophylactic therapy for haemophilia: summary statementQ35112424
Standardization of FVIII & FIX assaysQ35163622
Intensive exposure to factor VIII is a risk factor for inhibitor development in mild hemophilia A.Q35181684
Adenovirus-associated virus vector-mediated gene transfer in hemophilia BQ35691659
A cross-sectional study of bleeding phenotype in haemophilia A carriersQ35812405
Hemophilia B: molecular pathogenesis and mutation analysis.Q35832802
Hemophilic arthropathyQ35912735
Monitoring haemophilia severity and treatment: new or old laboratory tests?Q35915695
Inhibitors in congenital coagulation disordersQ35937076
Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients.Q36250920
Results of a phase I/II open-label, safety and efficacy trial of coagulation factor IX (recombinant), albumin fusion protein in haemophilia B patientsQ36380181
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variantQ36797248
Sequential therapy with activated prothrombin complex concentrates and recombinant FVIIa in patients with severe haemophilia and inhibitors: update of our previous experienceQ36819965
How to interpret and pursue an abnormal prothrombin time, activated partial thromboplastin time, and bleeding time in adultsQ36867591
Improved treatment feasibility in children with hemophilia using arteriovenous fistulae: the results after seven years of follow-upQ37175598
Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A.Q37489013
Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptideQ37634856
Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: a systematic reviewQ37719683
Blood-induced joint disease: the pathophysiology of hemophilic arthropathyQ37768520
Discrepancy between one-stage and chromogenic factor VIII activity assay results can lead to misdiagnosis of haemophilia A phenotype.Q37807879
Guideline on the management of haemophilia in the fetus and neonateQ37873540
Parallel use of by-passing agents in haemophilia with inhibitors: a critical reviewQ37927122
The diagnosis and management of congenital hemophiliaQ38046535
More than a decade of international experience with a pdFVIII/VWF concentrate in immune toleranceQ38070812
Factor VIII inhibitors in hemophilia A: rationale and latest evidenceQ38101041
FEIBA versus NovoSeven in hemophilia patients with inhibitorsQ38135321
Hemophilia a and hemophilia B: different types of diseases?Q38135323
Arthropathy in patients with moderate hemophilia a: a systematic review of the literatureQ38136140
Inhibitor eradication with rituximab in haemophilia: where do we stand?Q38195908
Current status of haemophilia gene therapyQ38206852
Innovative approach for improved rFVIII concentrateQ38207134
Prophylaxis with FEIBA in paediatric patients with haemophilia A and inhibitorsQ38325328
1-Deamino-8-d-arginine vasopressin: a new pharmacological approach to the management of haemophilia and von Willebrands' diseasesQ39433082
Personalized prophylaxisQ39596251
Inhibitor development and mortality in non-severe hemophilia A.Q41014569
Impact of inhibitors on hemophilia A mortality in the United States.Q41524981
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.Q41975229
The Fc gamma receptor IIa R131H polymorphism is associated with inhibitor development in severe hemophilia A.Q42211106
A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) StudyQ42598903
A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of Hemophilia A.Q42709633
Factor VIII products and inhibitor development in severe hemophilia A.Q43770114
"Founder" effect in different families with haemophilia B mutationQ43815426
Changing patterns of bleeding in patients with severe haemophilia A.Q44584056
F8 and F9 mutations in US haemophilia patients: correlation with history of inhibitor and race/ethnicityQ44646272
Physical functioning in boys with hemophilia in the U.S.Q44715249
Inhibitors occur more frequently in African-American and Latino haemophiliacsQ45855283
Factor VIII gene (F8) mutation and risk of inhibitor development in nonsevere hemophilia A.Q45855813
Prophylactic treatment for severe haemophilia: comparison of an intermediate-dose to a high-dose regimen.Q45855994
Severe hemophilia with mild bleeding phenotype: molecular characterization and global coagulation profileQ45856559
Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B.Q45858690
Clinical use of a new glycine-precipitated antihemophilic fractionQ45859062
Production of High-Potency Concentrates of Antihemophilic Globulin in a Closed-Bag SystemQ45859073
Primary and rescue immune tolerance induction in children and adults: a multicentre international study with a VWF-containing plasma-derived FVIII concentrate.Q45859168
Polymorphisms in the TNFA gene and the risk of inhibitor development in patients with hemophilia A.Q45861960
Spectrum of molecular defects and mutation detection rate in patients with mild and moderate hemophilia A.Q45862416
The effect of activated prothrombin-complex concentrate (FEIBA) on joint and muscle bleeding in patients with hemophilia A and antibodies to factor VIII. A double-blind clinical trialQ45864000
Polymorphisms in the CTLA-4 gene and inhibitor development in patients with severe hemophilia A.Q45865637
Similar bleeding phenotype in young children with haemophilia A or B: a cohort studyQ45865672
Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study.Q45865894
A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study).Q45866554
Trends in bleeding patterns during prophylaxis for severe haemophilia: observations from a series of prospective clinical trialsQ45866876
Comparing bleed frequency and factor concentrate use between haemophilia A and B patientsQ45867613
Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A.Q45868849
AIDS, hepatitis and hemophilia in the 1980s: memoirs from an insiderQ45869176
Non-invasive tool for foetal sex determination in early gestational age.Q45869375
Phenotypic heterogeneity in severe hemophiliaQ45869550
Randomized, prospective clinical trial of recombinant factor VIIa for secondary prophylaxis in hemophilia patients with inhibitorsQ45870481
A longitudinal study of orthopaedic outcomes for severe factor-VIII-deficient haemophiliacs. The Orthopaedic Outcome Study GroupQ45870861
Prenatal diagnosis and preimplantation genetic diagnosis: novel technologies and state of the art of PGD in different regions of the worldQ45871116
European study on orthopaedic status of haemophilia patients with inhibitorsQ45871281
Safety and pharmacokinetics of anti-TFPI antibody (concizumab) in healthy volunteers and patients with hemophilia: a randomized first human dose trialQ45872638
Single 270 microg kg(-1)-dose rFVIIa vs. standard 90 microg kg(-1)-dose rFVIIa and APCC for home treatment of joint bleeds in haemophilia patients with inhibitors: a randomized comparisonQ45873703
Evaluation of DHPLC in the analysis of hemophilia A.Q45874440
Identification of 217 unreported mutations in the F8 gene in a group of 1,410 unselected Italian patients with hemophilia A.Q45874679
Anti-inhibitor coagulant complex prophylaxis in hemophilia with inhibitorsQ45874687
The principal results of the International Immune Tolerance Study: a randomized dose comparisonQ45875180
Neonatal bleeding in haemophilia: a European cohort studyQ45875593
The incidence of factor VIII and factor IX inhibitors in the hemophilia population of the UK and their effect on subsequent mortality, 1977-99.Q45876258
P433issue10040
P407language of work or nameEnglishQ1860
P921main subjecthemophiliaQ134003
P304page(s)187-197
P577publication date2016-02-18
P1433published inThe LancetQ939416
P1476titleThe past and future of haemophilia: diagnosis, treatments, and its complications
P478volume388

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