Guidelines on the use of prophylactic factor replacement for children and adults with Haemophilia A and B

Safety analysis of rFVII a with emicizumab dosing in congenital hemophilia A with inhibitors: Experience from the HAVEN clinical program

(2019) Gallia G. Levy et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Emicizumab and thrombosis: The story so far

(2019) Michael Makris et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Emicizumab prophylaxis among infants and toddlers with severe hemophilia A and inhibitors—a single‐center cohort

(2019) Assaf A. Barg et al.   PEDIATRIC BLOOD & CANCER

Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non-randomised phase 3 study

(2019) Steven W Pipe et al.   Lancet Haematology

ST‐Segment Elevation Myocardial Infarction (STEMI) and Pulmonary Embolism in a Hemophilia A Patient Receiving Emicizumab and recombinant Activated Factor VII

(2019) Krishna Gundabolu et al.   HAEMOPHILIA

A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors

(2019) Guy Young et al.   BLOOD

Laboratory coagulation tests and emicizumab treatment A United Kingdom Haemophilia Centre Doctors' Organisation guideline

(2019) P. Vincent Jenkins et al.   HAEMOPHILIA

Physiotherapist inter-rater reliability of the Haemophilia Early Arthropathy Detection with Ultrasound protocol

(2018) D. Stephensen et al.   HAEMOPHILIA

Defining extended half-life rFVIII-A critical review of the evidence

(2018) J. Mahlangu et al.   HAEMOPHILIA

Treatment of bleeding episodes in haemophilia A complicated by a factor VIII inhibitor in patients receiving Emicizumab. Interim guidance from UKHCDO Inhibitor Working Party and Executive Committee

(2018) P. W. Collins et al.   HAEMOPHILIA

Tailored frequency-escalated primary prophylaxis for severe haemophilia A: results of the 16-year Canadian Hemophilia Prophylaxis Study longitudinal cohort

(2018) Brian M Feldman et al.   Lancet Haematology

Emicizumab Prophylaxis in Patients Who Have Hemophilia A without Inhibitors

(2018) Johnny Mahlangu et al.   NEW ENGLAND JOURNAL OF MEDICINE

New Therapies for hemophilia

(2018) Angela C. Weyand et al.   BLOOD

Treatment regimens and outcomes in severe and moderate haemophilia A in the UK: The THUNDER study

(2018) Martin J. Scott et al.   HAEMOPHILIA

Effects of vitamin D supplementation on musculoskeletal health: a systematic review, meta-analysis, and trial sequential analysis

(2018) Mark J Bolland et al.   Lancet Diabetes & Endocrinology

Patient powered prophylaxis: A 12-month study of individualized prophylaxis in adults with severe haemophilia A

(2017) Haowei Linda Sun et al.   HAEMOPHILIA

Pattern of bleeding in a large prospective cohort of haemophilia A patients: A three-year follow-up of the AHEAD (Advate in HaEmophilia A outcome Database) study

(2017) K. Khair et al.   HAEMOPHILIA

Estimating and interpreting the pharmacokinetic profiles of individual patients with hemophilia A or B using a population pharmacokinetic approach: communication from the SSC of the ISTH

(2017) A. Iorio et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Emicizumab Prophylaxis in Hemophilia A with Inhibitors

(2017) Johannes Oldenburg et al.   NEW ENGLAND JOURNAL OF MEDICINE

AAV5–Factor VIII Gene Transfer in Severe Hemophilia A

(2017) Savita Rangarajan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Tailoring treatment of haemophilia B: accounting for the distribution and clearance of standard and extended half-life FIX concentrates

(2017) Kathelijn Fischer et al.   THROMBOSIS AND HAEMOSTASIS

Clinical evaluation of glycoPEGylated recombinant FVIII: Efficacy and safety in severe haemophilia A

(2017) Tatiana Andreeva et al.   THROMBOSIS AND HAEMOSTASIS

PK-driven prophylaxis versus standard prophylaxis: When a tailored treatment may be a real and achievable cost-saving approach in children with severe hemophilia A

(2017) Samantha Pasca et al.   THROMBOSIS RESEARCH

Recombinant factor IX Fc fusion protein in children with haemophilia B (Kids B-LONG): results from a multicentre, non-randomised phase 3 study

(2017) Kathelijn Fischer et al.   Lancet Haematology

Ultrasound for Early Detection of Joint Disease in Patients with Hemophilic Arthropathy

(2017) Matteo Di Minno et al.   Journal of Clinical Medicine

New concepts for anticoagulant therapy in persons with hemophilia

(2016) R. E. G. Schutgens et al.   BLOOD

The use of enhanced half-life coagulation factor concentrates in routine clinical practice: guidance from UKHCDO

(2016) P. Collins et al.   HAEMOPHILIA

Long-term effects of joint bleeding before starting prophylaxis in severe haemophilia

(2016) A. Nijdam et al.   HAEMOPHILIA

Association of peak factor VIII levels and area under the curve with bleeding in patients with haemophilia A on every third day pharmacokinetic-guided prophylaxis

(2016) L. A. Valentino et al.   HAEMOPHILIA

Physician trust and depression influence adherence to factor replacement: a single-centre cross-sectional study

(2016) D. Q. Tran et al.   HAEMOPHILIA

Validation of Accelerometer Prediction Equations in Children with Chronic Disease

(2016) Samantha Stephens et al.   PEDIATRIC EXERCISE SCIENCE

Discontinuing early prophylaxis in severe haemophilia leads to deterioration of joint status despite low bleeding rates

(2016) A. Nijdam et al.   THROMBOSIS AND HAEMOSTASIS

Nonacog beta pegol (N9-GP) in haemophilia B: A multinational phase III safety and efficacy extension trial (paradigm™4)

(2016) Guy Young et al.   THROMBOSIS RESEARCH

Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A

(2015) B. A. Konkle et al.   BLOOD

Clinical, instrumental, serological and histological findings suggest that hemophilia B may be less severe than hemophilia A

(2015) D. Melchiorre et al.   HAEMATOLOGICA

Central venous access device (CVAD) complications in Haemophilia with inhibitors undergoing immune tolerance induction: Lessons from the international immune tolerance study

(2015) V. Rodriguez et al.   HAEMOPHILIA

How to achieve full prophylaxis in young boys with severe haemophilia A: different regimens and their effect on early bleeding and venous access

(2015) A. Nijdam et al.   HAEMOPHILIA

Early prophylaxis in children with severe haemophilia A: clinical and ultrasound imaging outcomes

(2015) C. Altisent et al.   HAEMOPHILIA

Recombinant factor VIII Fc fusion protein for the prevention and treatment of bleeding in children with severe hemophilia A

(2015) G. Young et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Inhibitor development in haemophilia according to concentrate

(2015) Riita Lassila et al.   THROMBOSIS AND HAEMOSTASIS

Benefits of prophylaxis versus on-demand treatment in adolescents and adults with severe haemophilia A: the POTTER study

(2015) Giulio Feola et al.   THROMBOSIS AND HAEMOSTASIS

Clinical, instrumental, serological and histological findings suggest that hemophilia B may be less severe than hemophilia A

(2015) D. Melchiorre et al.   HAEMATOLOGICA

Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial

(2014) P. W. Collins et al.   BLOOD

Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A

(2014) T. Calvez et al.   BLOOD

Factor VIII brand and the incidence of factor VIII inhibitors in previously untreated UK children with severe hemophilia A, 2000-2011

(2014) P. W. Collins et al.   BLOOD

Adherence to prophylaxis is associated with better outcomes in moderate and severe haemophilia: results of a patient survey

(2014) S. Krishnan et al.   HAEMOPHILIA

A haemophilia disease management programme targeting cost and utilization of specialty pharmaceuticals

(2014) N. Duncan et al.   HAEMOPHILIA

The incidence of factor VIII inhibitors in severe haemophilia A following a major switch from full-length to B-domain-deleted factor VIII: a prospective cohort comparison

(2014) C. R. M. Hay et al.   HAEMOPHILIA

Atrial fibrillation in patients with haemophilia: a cross-sectional evaluation in Europe

(2014) Roger E. G. Schutgens et al.   HAEMOPHILIA

Controlled, cross-sectional MRI evaluation of joint status in severe haemophilia A patients treated with prophylaxis vs. on demand

(2014) J. Oldenburg et al.   HAEMOPHILIA

Definitions in hemophilia: communication from the SSC of the ISTH

(2014) V. S. Blanchette et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

(2014) Amit C. Nathwani et al.   NEW ENGLAND JOURNAL OF MEDICINE

Intermediate-dose versus high-dose prophylaxis for severe hemophilia: comparing outcome and costs since the 1970s

(2013) K. Fischer et al.   BLOOD

A systematic review of MR imaging as a tool for evaluating haemophilic arthropathy in children

(2013) M. W. Chan et al.   HAEMOPHILIA

Population pharmacokinetics of recombinant factor IX: implications for dose tailoring

(2013) S. Björkman   HAEMOPHILIA

Pharmacokinetics of plasma-derived and recombinant factor IX - implications for prophylaxis and on-demand therapy

(2013) S. Björkman   HAEMOPHILIA

Joint health scores in a haemophilia A cohort from Pakistan with minimal or no access to factor VIII concentrate: correlation with thrombin generation and underlying mutation

(2013) F. Khanum et al.   HAEMOPHILIA

Rapid musculoskeletal ultrasound for painful episodes in adult haemophilia patients

(2013) A. Ceponis et al.   HAEMOPHILIA

Haemophilia Joint Health Score in healthy adults playing sports

(2013) D. Sluiter et al.   HAEMOPHILIA

Prevalence and Risk Factors for Hypertension in Hemophilia

(2013) Annette von Drygalski et al.   HYPERTENSION

Randomized, controlled, parallel-group trial of routine prophylaxis vs. on-demand treatment with sucrose-formulated recombinant factor VIII in adults with severe hemophilia A (SPINART)

(2013) M. J. Manco-Johnson et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Factor VIII Products and Inhibitor Development in Severe Hemophilia A

(2013) Samantha C. Gouw et al.   NEW ENGLAND JOURNAL OF MEDICINE

Updating the Canadian Hemophilia Outcomes–Kids Life Assessment Tool (CHO-KLAT Version2.0)

(2013) Nancy L. Young et al.   VALUE IN HEALTH

Diagnosis and treatment of factor VIII and IX inhibitors in congenital haemophilia: (4th edition)

(2012) Peter W. Collins et al.   BRITISH JOURNAL OF HAEMATOLOGY

Population pharmacokinetics of plasma-derived factor IX in adult patients with haemophilia B: implications for dosing in prophylaxis

(2012) Sven Björkman et al.   EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY

An MRI scale for assessment of haemophilic arthropathy from the International Prophylaxis Study Group

(2012) B. Lundin et al.   HAEMOPHILIA

Guidelines for the management of hemophilia

(2012) A. Srivastava et al.   HAEMOPHILIA

Magnetic resonance imaging and joint outcomes in boys with severe hemophilia A treated with tailored primary prophylaxis in Canada

(2012) J. KRAFT et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Overweight and Obesity in Hemophilia

(2011) Trisha E. Wong et al.   AMERICAN JOURNAL OF PREVENTIVE MEDICINE

Population pharmacokinetics of recombinant factor VIII: the relationships of pharmacokinetics to age and body weight

(2011) S. Bjorkman et al.   BLOOD

Prevalence and risk factors of cardiovascular disease (CVD) events among patients with haemophilia: experience of a single haemophilia treatment centre in the United States (US)

(2011) A. A. SHARATHKUMAR et al.   HAEMOPHILIA

A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study)

(2011) A. GRINGERI et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Associations between intracranial haemorrhage and prescribed prophylaxis in a large cohort of haemophilia patients in the United States

(2010) Char Witmer et al.   BRITISH JOURNAL OF HAEMATOLOGY

A United Kingdom Haemophilia Centre Doctors’ Organization guideline approved by the British Committee for Standards in Haematology: guideline on the use of prophylactic factor VIII concentrate in children and adults with severe haemophilia A

(2010) Michael Richards et al.   BRITISH JOURNAL OF HAEMATOLOGY

Treatment related factors and inhibitor development in children with severe haemophilia A

(2010) P. S. MACLEAN et al.   HAEMOPHILIA

Implications of coagulation factor VIII and IX pharmacokinetics in the prophylactic treatment of haemophilia

(2010) P. W. COLLINS et al.   HAEMOPHILIA

Comparative pharmacokinetics of plasma- and albumin-free recombinant factor VIII in children and adults: the influence of blood sampling schedule on observed age-related differences and implications for dose tailoring

(2010) S. BJÖRKMAN et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Validation of a new pediatric joint scoring system from the International Hemophilia Prophylaxis Study Group: Validity of the hemophilia joint health score

(2010) Brian M. Feldman et al.   ARTHRITIS CARE & RESEARCH

Population pharmacokinetics of recombinant factor VIII:C (ReFacto®) in adult HIV-negative and HIV-positive haemophilia patients

(2009) Anastasia Karafoulidou et al.   EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY

Pharmacokinetics and dose requirements of factor VIII over the age range 3–74 years

(2009) Sven Björkman et al.   EUROPEAN JOURNAL OF CLINICAL PHARMACOLOGY

Development and preliminary testing of a Paediatric Version of the Haemophilia Activities List (pedhal)

(2009) W. G. GROEN et al.   HAEMOPHILIA

Efficacy and safety of secondary prophylactic vs. on-demand sucrose-formulated recombinant factor VIII treatment in adults with severe hemophilia A: results from a 13-month crossover study

(2009) P. COLLINS et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Models for Prediction of Factor VIII Half-Life in Severe Haemophiliacs: Distinct Approaches for Blood Group O and Non-O Patients

(2009) Kathelijn Fischer et al.   PLoS One

Reliability and construct validity of the compatible MRI scoring system for evaluation of elbows in haemophilic children

(2008) A. S. DORIA et al.   HAEMOPHILIA

Break-through bleeding in relation to predicted factor VIII levels in patients receiving prophylactic treatment for severe hemophilia A

(2008) P. W. COLLINS et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS