selected publications
- The Bleeding Disorders Research Collaborative. Blood vessels, thrombosis & hemostasis. 2025 Review GET IT
- Impaired tolerance in the International Immune Tolerance Induction study due to the presence of very low-titer inhibitors. Journal of thrombosis and haemostasis : JTH. 2025 Academic Article GET IT
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The roadmap to integrate diversity, equity, and inclusion in hematology clinical trials: an American Society of Hematology initiative.
Blood advances.
2025
Academic Article
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Times cited: 4 -
ISTH clinical practice guideline for treatment of congenital hemophilia A and B based on the Grading of Recommendations Assessment, Development, and Evaluation methodology: considerations for practice management and implementation.
Journal of thrombosis and haemostasis : JTH.
2024
Guideline
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Times cited: 2 -
Soliciting international perspectives on an American national research agenda for inherited bleeding disorders.
Expert review of hematology.
2023
Editorial Article
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Times cited: 4 -
The National Hemophilia Foundation State of the Science Research Summit initiative: executive summary.
Expert review of hematology.
2023
Academic Article
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Times cited: 12 -
The National Hemophilia Foundation's State of the Science Research Summit: the foundation of a national research blueprint for inherited bleeding disorders.
Expert review of hematology.
2023
Editorial Article
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Times cited: 9 -
Building the blueprint: Formulating a community-generated national plan for future research in inherited bleeding disorders.
Haemophilia : the official journal of the World Federation of Hemophilia.
2022
Academic Article
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Times cited: 12 -
Big picture initiatives in bleeding disorders.
Haemophilia : the official journal of the World Federation of Hemophilia.
2022
Review
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Times cited: 11 -
End points for sickle cell disease clinical trials: renal and cardiopulmonary, cure, and low-resource settings.
Blood advances.
2019
Academic Article
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Times cited: 28 -
Executive summary of the NHLBI State of the Science (SOS) Workshop: Overview and next steps in generating a national blueprint for future research on factor VIII inhibitors.
Haemophilia : the official journal of the World Federation of Hemophilia.
2019
Review
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Times cited: 7 -
Origins and organization of the NHLBI State of the Science Workshop: Generating a national blueprint for future research on factor VIII inhibitors.
Haemophilia : the official journal of the World Federation of Hemophilia.
2019
Academic Article
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Times cited: 6 - Navigating Speed Bumps on the Innovation Highway in Hemophilia Therapeutics. HemaSphere. 2018 Review GET IT
- Catalyzing innovation in clinical trial design and analysis at NHLBI: a funding opportunity for clinical trials in blood disorders. Blood advances. 2017 Academic Article GET IT
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Complications of haemophilia in babies (first two years of life): a report from the Centers for Disease Control and Prevention Universal Data Collection System.
Haemophilia : the official journal of the World Federation of Hemophilia.
2016
Academic Article
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Times cited: 72 -
Hemophilia Therapy--Navigating Speed Bumps on the Innovation Highway.
The New England journal of medicine.
2016
Editorial Article
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Times cited: 6 -
Thrombin generation and bleeding in haemophilia inhibitor patients during immune tolerance induction.
Haemophilia : the official journal of the World Federation of Hemophilia.
2015
Academic Article
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Times cited: 3 -
Central venous access device (CVAD) complications in Haemophilia with inhibitors undergoing immune tolerance induction: Lessons from the international immune tolerance study.
Haemophilia : the official journal of the World Federation of Hemophilia.
2015
Academic Article
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Times cited: 22 -
Design of clinical trials for new products in hemophilia: communication from the SSC of the ISTH.
Journal of thrombosis and haemostasis : JTH.
2015
Academic Article
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Times cited: 9 -
Planning for the future workforce in hematology research.
Blood.
2015
Academic Article
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Times cited: 18 -
Citation impact of NHLBI R01 grants funded through the American Recovery and Reinvestment Act as compared to R01 grants funded through a standard payline.
Circulation research.
2015
Academic Article
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Times cited: 15 -
Prior publication productivity, grant percentile ranking, and topic-normalized citation impact of NHLBI cardiovascular R01 grants.
Circulation research.
2014
Academic Article
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Times cited: 39 -
Use of global assays to understand clinical phenotype in congenital factor VII deficiency.
Haemophilia : the official journal of the World Federation of Hemophilia.
2013
Academic Article
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Times cited: 14 -
Prevalence of clinical hip abnormalities in haemophilia A and B: an analysis of the UDC database.
Haemophilia : the official journal of the World Federation of Hemophilia.
2012
Academic Article
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Times cited: 17 -
The polygenic nature of inhibitors in hemophilia A: results from the Hemophilia Inhibitor Genetics Study (HIGS) Combined Cohort.
Blood.
2012
Academic Article
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Times cited: 95 -
Are central institutional review boards the solution? The National Heart, Lung, and Blood Institute Working Group's report on optimizing the IRB process.
Academic medicine : journal of the Association of American Medical Colleges.
2012
Academic Article
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Times cited: 25 -
Inhibitors in childhood hemophilia A: genetic and treatment-related risk factors for development and eradication.
Pediatric blood & cancer.
2012
Review
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Times cited: 8 -
Immune tolerance in haemophilia: the long journey to the fork in the road.
British journal of haematology.
2012
Review
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Times cited: 62 -
Clinical trial design in haemophilia.
Haemophilia : the official journal of the World Federation of Hemophilia.
2012
Academic Article
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Times cited: 20 -
Paroxysmal nocturnal hemoglobinuria in pediatric patients.
Pediatric blood & cancer.
2011
Academic Article
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Times cited: 30 -
Knowledge and therapeutic gaps: a public health problem in the rare coagulation disorders population.
American journal of preventive medicine.
2011
Academic Article
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Times cited: 15 -
The principal results of the International Immune Tolerance Study: a randomized dose comparison.
Blood.
2011
Academic Article
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Times cited: 391 -
Mutation in the factor VII hepatocyte nuclear factor 4α-binding site contributes to factor VII deficiency.
Blood coagulation & fibrinolysis : an international journal in haemostasis and thrombosis.
2011
Academic Article
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Times cited: 8 -
Haemostasis prophylaxis using single dose desmopressin acetate and extended use epsilon aminocaproic acid for adenotonsillectomy in patients with type 1 von Willebrand disease.
Haemophilia : the official journal of the World Federation of Hemophilia.
2011
Academic Article
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Times cited: 12 -
Neutralizing antibodies against adeno-associated virus examined prospectively in pediatric patients with hemophilia.
Gene therapy.
2011
Academic Article
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Times cited: 188 -
Successful hip arthroplasty in an adult male with severe factor XI deficiency using Hemoleven®, a factor XI concentrate.
2011
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Times cited: 11 -
Neoangiogenesis contributes to the development of hemophilic synovitis.
Blood.
2010
Academic Article
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Times cited: 107 -
Successful cryoablation of atrioventricular nodal reentrant tachycardia in a child with hemophilia A.
2010
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Times cited: 3 -
Recombinant activated factor VII safety and efficacy in the treatment of cranial haemorrhage in patients with congenital haemophilia with inhibitors: an analysis of the Hemophilia and Thrombosis Research Society Registry (2004-2008).
Haemophilia : the official journal of the World Federation of Hemophilia.
2010
Academic Article
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Times cited: 12 - Treatment of congenital bleeding disorders: future trends in management. Thrombosis research. 2009 Editorial Article GET IT
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The "parallel-cohort RCT": Novel design aspects and application in the Kids-DOTT trial of pediatric venous thromboembolism.
Contemporary clinical trials.
2009
Academic Article
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Times cited: 24 -
Pharmacokinetics and safety of fibrinogen concentrate.
Journal of thrombosis and haemostasis : JTH.
2009
Academic Article
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Times cited: 106 -
Splenic infarction and subsequent splenic rupture in a patient with paroxysmal nocturnal hemoglobinuria and heparin-induced thrombocytopenia.
2009
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Times cited: 5 -
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) project.
Haemophilia : the official journal of the World Federation of Hemophilia.
2009
Academic Article
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Times cited: 133 -
Risk factors for inhibitor formation in haemophilia: a prevalent case-control study.
Haemophilia : the official journal of the World Federation of Hemophilia.
2009
Academic Article
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Times cited: 65 -
Bleeding symptoms and laboratory correlation in patients with severe von Willebrand disease.
Haemophilia : the official journal of the World Federation of Hemophilia.
2009
Academic Article
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Times cited: 29 -
Rare inherited disorders of fibrinogen.
Haemophilia : the official journal of the World Federation of Hemophilia.
2008
Review
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Times cited: 205 -
The North American Immune Tolerance Registry: contributions to the thirty-year experience with immune tolerance therapy.
Haemophilia : the official journal of the World Federation of Hemophilia.
2008
Academic Article
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Times cited: 85 -
Power Doppler sonography in the diagnosis of hemophilic synovitis--a promising tool.
Journal of thrombosis and haemostasis : JTH.
2008
Academic Article
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Times cited: 50 -
Ethical considerations in clinical investigation: exploring relevance in haemophilia research.
Haemophilia : the official journal of the World Federation of Hemophilia.
2008
Review
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Times cited: 7 -
Haemophilia in the first years of life.
Haemophilia : the official journal of the World Federation of Hemophilia.
2008
Academic Article
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Times cited: 31 -
A prospective longitudinal multicenter study of coagulation in pediatric patients undergoing allogeneic stem cell transplantation.
Pediatric blood & cancer.
2008
Academic Article
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Times cited: 19 -
The use of factor VIII/von Willebrand factor concentrate for immune tolerance induction in haemophilia A patients with high-titre inhibitors: association of clinical outcome with inhibitor epitope profile.
Haemophilia : the official journal of the World Federation of Hemophilia.
2007
Academic Article
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Times cited: 49 -
Immune tolerance therapy utilizing factor VIII/von Willebrand factor concentrate in haemophilia A patients with high titre factor VIII inhibitors.
Haemophilia : the official journal of the World Federation of Hemophilia.
2007
Academic Article
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Times cited: 49 -
Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia.
The New England journal of medicine.
2007
Academic Article
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Times cited: 1693 -
Inhibitor development in haemophilia B: an orphan disease in need of attention.
British journal of haematology.
2007
Review
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Times cited: 216 -
Immune tolerance therapy for factor VIII inhibitors: moving from empiricism to an evidence-based approach.
Journal of thrombosis and haemostasis : JTH.
2007
Review
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Times cited: 36 -
Immune tolerance: critical issues of factor dose, purity and treatment complications.
Haemophilia : the official journal of the World Federation of Hemophilia.
2006
Review
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Times cited: 27 -
Inhibitor treatment in haemophilias A and B: inhibitor diagnosis.
Haemophilia : the official journal of the World Federation of Hemophilia.
2006
Review
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Times cited: 24 -
Inhibitor treatment in haemophilias A and B: summary statement for the 2006 international consensus conference.
Haemophilia : the official journal of the World Federation of Hemophilia.
2006
Academic Article
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Times cited: 82 -
A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) Study.
Blood.
2006
Academic Article
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Times cited: 404 -
The international immune tolerance study: a multicenter prospective randomized trial in progress.
Journal of thrombosis and haemostasis : JTH.
2006
Letter
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Times cited: 59 -
A retrospective postlicensure survey of FEIBA efficacy and safety.
Haemophilia : the official journal of the World Federation of Hemophilia.
2006
Academic Article
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Times cited: 107 -
Ethical issues in haemophilia.
Haemophilia : the official journal of the World Federation of Hemophilia.
2006
Review
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Times cited: 14 -
Management of factor VIII inhibitors.
International journal of hematology.
2006
Review
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Times cited: 27 -
Management of factor VIII inhibitors.
Best practice & research. Clinical haematology.
2006
Review
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Times cited: 20 -
Haemophilia Inhibitor Genetics Study - evaluation of a model for studies of complex diseases using linkage and association methods.
Haemophilia : the official journal of the World Federation of Hemophilia.
2005
Letter
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Times cited: 13 -
Gene therapy for hemophilia? The debate reframed.
Journal of thrombosis and haemostasis : JTH.
2005
Letter
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Times cited: 4 -
High frequency of premature termination mutations in the factor V gene: three factor V deficiency case reports and a mutation review.
2005
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Times cited: 11 -
Inhibitors in haemophilia: clinical aspects.
Haemophilia : the official journal of the World Federation of Hemophilia.
2004
Review
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Times cited: 53 -
Clinical evaluation of an advanced category antihaemophilic factor prepared using a plasma/albumin-free method: pharmacokinetics, efficacy, and safety in previously treated patients with haemophilia A.
Haemophilia : the official journal of the World Federation of Hemophilia.
2004
Academic Article
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Times cited: 143 -
In praise of long-term prospective data collection: questions and perspectives.
Journal of thrombosis and haemostasis : JTH.
2004
Comment
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Times cited: 2 -
Rare Bleeding Disorder Registry: deficiencies of factors II, V, VII, X, XIII, fibrinogen and dysfibrinogenemias.
Journal of thrombosis and haemostasis : JTH.
2004
Academic Article
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Times cited: 265 -
Impact of inhibitor epitope profile on the neutralizing effect against plasma-derived and recombinant factor VIII concentrates in vitro.
Haemophilia : the official journal of the World Federation of Hemophilia.
2003
Academic Article
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Times cited: 48 -
Management of pregnancy in a patient with severe haemophilia A.
2003
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Times cited: 25 -
Transjugular liver biopsy is safe and diagnostic for patients with congenital bleeding disorders and hepatitis C infection.
Haemophilia : the official journal of the World Federation of Hemophilia.
2003
Academic Article
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Times cited: 31 -
Immune tolerance therapy dose as an outcome predictor.
Haemophilia : the official journal of the World Federation of Hemophilia.
2003
Review
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Times cited: 21 -
Clinical manifestations of the prothrombin G20210A mutation in children: a pediatric coagulation consortium study.
Journal of thrombosis and haemostasis : JTH.
2003
Academic Article
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Times cited: 53 -
Long-term FEIBA prophylaxis does not prevent progression of existing joint disease.
Haemophilia : the official journal of the World Federation of Hemophilia.
2003
Academic Article
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Times cited: 73 -
Gene therapy ethics and haemophilia: an inevitable therapeutic future?.
Haemophilia : the official journal of the World Federation of Hemophilia.
2003
Review
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Times cited: 23 -
The upward spiral of drug costs: a time series analysis of drugs used in the treatment of hemophilia.
Thrombosis and haemostasis.
2002
Comment
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Times cited: 21 -
End-stage liver disease in persons with hemophilia and transfusion-associated infections.
Blood.
2002
Academic Article
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Times cited: 168 -
Inhibitors: resolving diagnostic and therapeutic dilemmas.
Haemophilia : the official journal of the World Federation of Hemophilia.
2002
Review
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Times cited: 134 -
Continuous infusion of porcine factor VIII: stability, microbiological safety and clinical experience.
Haemophilia : the official journal of the World Federation of Hemophilia.
2002
Academic Article
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Times cited: 3 -
The North American Immune Tolerance Registry: practices, outcomes, outcome predictors.
Thrombosis and haemostasis.
2002
Academic Article
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Times cited: 261 -
Continuous infusion of porcine factor VIII in patients with haemophilia A and high-responding inhibitors: stability and clinical experience.
Haemophilia : the official journal of the World Federation of Hemophilia.
2001
Academic Article
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Times cited: 14 -
Heart transplant in a factor VIII-deficient patient with a high-titre inhibitor: perioperative management using high-dose continuous infusion factor VIII and recombinant factor VIIa.
2001
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Times cited: 34 -
The maintenance of tolerance after successful immune tolerance induction in hemophilia A and B: the North American Registry. Factor VIII/IX Subcommittee of the International Society for Thrombosis and Hemostasis.
Haematologica.
2000
Academic Article
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Times cited: 22 -
Inhibitors in haemophilia: a primer.
Haemophilia : the official journal of the World Federation of Hemophilia.
2000
Review
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Times cited: 39 -
Anaphylactic response to factor IX replacement therapy in haemophilia B patients: complete gene deletions confer the highest risk.
Haemophilia : the official journal of the World Federation of Hemophilia.
1999
Academic Article
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Times cited: 97 -
Utilization of previously treated patients (PTPs), noninfected patients (NIPs), and previously untreated patients (PUPs) in the evaluation of new factor VIII and factor IX concentrates. Recommendation of the Scientific Subcommittee on Factor VIII and Factor IX of the Scientific and Standardization Committee of the International Society on Thrombosis and Haemostasis.
Thrombosis and haemostasis.
1999
Academic Article
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Times cited: 61 -
Analysis of the North American Immune Tolerance Registry (NAITR) 1993-1997: current practice implications. ISTH Factor VIII/IX Subcommittee Members.
Vox sanguinis.
1999
Academic Article
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Times cited: 47 -
Hemophilia. A new approach to an old disease.
Hematology/oncology clinics of North America.
1998
Review
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Times cited: 54 -
Highly probable anaphylactic reaction to systemic thrombolytic therapy with high dose urokinase in a child with a prosthetic valve.
Journal of pediatric hematology/oncology.
1998
Letter
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Times cited: 3 -
Inhibitors occur more frequently in African-American and Latino haemophiliacs.
Haemophilia : the official journal of the World Federation of Hemophilia.
1998
Letter
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Times cited: 101 - Factor IX inhibitors and anaphylaxis in hemophilia B. Haemophilia : the official journal of the World Federation of Hemophilia. 1997 Academic Article GET IT
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Nephrotic syndrome as a complication of immune tolerance in hemophilia B.
Blood.
1997
Letter
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Times cited: 107 -
Factor IX inhibitors and anaphylaxis in hemophilia B.
Journal of pediatric hematology/oncology.
1997
Academic Article
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Times cited: 170 -
Factor IX inhibitors and anaphylaxis in haemophilia B.
Haemophilia : the official journal of the World Federation of Hemophilia.
1996
Letter
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Times cited: 5 -
Hemophilia 1996. New approach to an old disease.
Pediatric clinics of North America.
1996
Review
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Times cited: 32