Guy A. Young, MD

Following medical school, I completed my residency in pediatrics at Schneider Children’s Hospital-Long Island Jewish Medical Center which is part of the Albert Einstein College of Medicine. I then completed a fellowship in pediatric hematology/oncology at Children’s National Medical Center in Washington, DC which is part of the George Washington University School of Medicine. I then began my research career at the Children’s Hospital of Orange County under the mentorship of Dr. Diane Nugent. There, I became interested in improving the treatment for patients with hemophilia and inhibitors and for children with blood clots. I received my first independent grants there inculding a grant from the FDA to study a new anticoagulant in children. In addition, I became interested in thromboelastography. In 2007, I moved the Children’s Hospital Los Angeles where I am continuing to perform research in the same areas as I had started in Orange County.

• National Hemophilia Foundation: Physician of the Year, 2013

• Corticosteroid use to mitigate transaminitis-associated decline in FVIII levels following valoctocogene roxaparvovec gene therapy: clinical practice guidance. J Thromb Haemost. 2025 Jul; 23(7):2086-2094.. View in PubMed
• Safety and efficacy of a fitusiran antithrombin-based dose regimen in people with hemophilia A or B: the ATLAS-OLE study. Blood. 2025 Jun 19; 145(25):2966-2977.. View in PubMed
• Invitation and welcome to the 2025 ISTH Congress. J Thromb Haemost. 2025 May; 23(5):1461-1463.. View in PubMed
• Global Comparative Antithrombin Field Study: Impact of Laboratory Assay Variability on the Assessment of Antithrombin Activity Measurement at Fitusiran Clinical Decision-Making Points. Haemophilia. 2025 May; 31(3):566-574.. View in PubMed
• Extended-phase anticoagulant treatment of acute venous thromboembolism in children: a cohort study from the EINSTEIN-Jr phase 3 trial. Lancet Haematol. 2025 May; 12(5):e357-e364.. View in PubMed
• Fondaparinux Approval in Pediatrics: The Story of My Third Child. Pediatr Blood Cancer. 2025 May; 72(5):e31638.. View in PubMed
• Reply to Letter to the Editor: “Deconstructing the ISTH Hemophilia Guidelines for the Clinician”: comment from Mancuso-ME et al. J Thromb Haemost. 2025 May; 23(5):1737-1738.. View in PubMed
• Accurate evaluation of factor VIII activity of efanesoctocog alfa in the presence of emicizumab. J Thromb Haemost. 2025 May; 23(5):1516-1521.. View in PubMed
• Deconstructing the ISTH hemophilia guidelines for the clinician. J Thromb Haemost. 2025 May; 23(5):1483-1495.. View in PubMed
• Concizumab prophylaxis in people with hemophilia A or B without inhibitors: patient-reported outcome results from the phase 3 explorer8 study. Res Pract Thromb Haemost. 2025 Feb; 9(2):102705.. View in PubMed
• Induction of factor VIII tolerance by hemophilia gene transfer to eradicate factor VIII inhibitors. Blood Adv. 2025 Jan 28; 9(2):265-269.. View in PubMed
• Bleed treatment with eptacog beta (rFVIIa) results in a low incidence of rebleeding in adult and adolescent patients with haemophilia A or B with inhibitors. Haemophilia. 2025 Jan; 31(1):78-86.. View in PubMed
• CHIEF: A retrospective self-control study of children with severe hemophilia A without inhibitors comparing emicizumab to FVIII prophylaxis. Pediatr Blood Cancer. 2024 Dec; 71(12):e31351.. View in PubMed
• Concizumab prophylaxis in people with haemophilia A or haemophilia B without inhibitors (explorer8): a prospective, multicentre, open-label, randomised, phase 3a trial. Lancet Haematol. 2024 Dec; 11(12):e891-e904.. View in PubMed
• Real-world effectiveness of eptacog beta in patients with haemophilia and inhibitors: A multi-institutional case series. Haemophilia. 2024 Nov; 30(6):1321-1331.. View in PubMed
• Fitusiran prophylaxis in people with hemophilia A or B who switched from prior BPA/CFC prophylaxis: the ATLAS-PPX trial. Blood. 2024 05 30; 143(22):2256-2269.. View in PubMed
• Emicizumab is well tolerated and effective in people with congenital hemophilia A regardless of age, severity of disease, or inhibitor status: a scoping review. Res Pract Thromb Haemost. 2024 May; 8(4):102415.. View in PubMed
• Emicizumab prophylaxis in infants with hemophilia A (HAVEN 7): primary analysis of a phase 3b open-label trial. Blood. 2024 04 04; 143(14):1355-1364.. View in PubMed
• How I treat pediatric venous thromboembolism in the DOAC era. Blood. 2024 02 01; 143(5):389-403.. View in PubMed
• Pharmacokinetics and coagulation biomarkers in children and adults with hemophilia A receiving emicizumab prophylaxis every 1, 2, or 4 weeks. Res Pract Thromb Haemost. 2024 Jan; 8(1):102306.. View in PubMed
• Simoctocog alfa (Nuwiq®) in children: early steps in life’s journey for people with severe hemophilia A. Ther Adv Hematol. 2024; 15:20406207241245511.. View in PubMed
• Corrigendum to ‘Evaluation of Venous Thromboembolism Risk Factors Reveals Subtype Heterogenicity in Children with Central Venous Catheters: A Multi-Center Study from the CHAT Consortium’ [Journal of Thrombosis and Haemostasis 21/9 (2023) 2441 – 2450]. J Thromb Haemost. 2023 Dec; 21(12):3658.. View in PubMed
• Delivery of gene therapy in haemophilia treatment centres in the United States: Practical aspects of preparedness and implementation. Haemophilia. 2023 Nov; 29(6):1430-1441.. View in PubMed
• The immunogenicity, safety, and efficacy of N8-GP in previously untreated patients with severe hemophilia A: pathfinder6 end-of-trial results. J Thromb Haemost. 2023 11; 21(11):3109-3116.. View in PubMed
• Severe muscle bleeds in children and young adults with hemophilia A on emicizumab prophylaxis: Real-world retrospective multi-institutional cohort. Am J Hematol. 2023 10; 98(10):E285-E287.. View in PubMed
• Effects of PK-guided prophylaxis on clinical outcomes and FVIII consumption for patients with moderate to severe Haemophilia A. Haemophilia. 2023 Sep; 29(5):1234-1242.. View in PubMed
• Evaluation of venous thromboembolism risk factors reveals subtype heterogenicity in children with central venous catheters: a multicenter study from the Children’s Hospital Acquired Thrombosis consortium. J Thromb Haemost. 2023 09; 21(9):2441-2450.. View in PubMed
• Neutralizing antidrug antibody to emicizumab in patients with severe hemophilia A: Case report of a first noninhibitor patient and review of the literature. Res Pract Thromb Haemost. 2023 Aug; 7(6):102194.. View in PubMed
• International Council for Standardization in Haematology recommendations for laboratory measurement of factor VIII and FIX type I inhibitors. Int J Lab Hematol. 2023 Aug; 45(4):413-424.. View in PubMed
• Prophylaxis with a recombinant factor VIII Fc in hemophilia A: long-term follow-up on joint health, efficacy, and safety from phase 3 studies in children and adults. Res Pract Thromb Haemost. 2023 Aug; 7(6):102180.. View in PubMed
• Efficacy and safety evaluation of eptacog beta (coagulation factor VIIa [recombinant]-jncw) for the treatment of hemophilia A and B with inhibitors. Expert Rev Hematol. 2023 Jul-Dec; 16(10):715-729.. View in PubMed
• Time to revisit the classification of hemophilia: if it ain’t broke, don’t fix it! J Thromb Haemost. 2023 07; 21(7):1755-1756.. View in PubMed
• Nonfactor Therapies for Hemophilia. Hemasphere. 2023 Jun; 7(6):e911.. View in PubMed
• Antithrombin lowering in hemophilia: a closer look at fitusiran. Res Pract Thromb Haemost. 2023 May; 7(4):100179.. View in PubMed
• Efficacy and safety of fitusiran prophylaxis in people with haemophilia A or haemophilia B with inhibitors (ATLAS-INH): a multicentre, open-label, randomised phase 3 trial. Lancet. 2023 04 29; 401(10386):1427-1437.. View in PubMed
• Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: facilitating research through infrastructure, workforce, resources and funding. Expert Rev Hematol. 2023 03; 16(sup1):107-127.. View in PubMed
• The safety and efficacy of N8-GP (turoctocog alfa pegol) in previously untreated pediatric patients with hemophilia A. Blood Adv. 2023 02 28; 7(4):620-629.. View in PubMed
• Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. N Engl J Med. 2023 02 23; 388(8):706-718.. View in PubMed
• The dosing conundrum of emicizumab: To waste product or not? Res Pract Thromb Haemost. 2023 Feb; 7(2):100087.. View in PubMed
• Safety of FEIBA and emicizumab (SAFE): Dose escalation study evaluating the safety of in vivo administration of activated prothrombin complex concentrate in haemophilia A patients on emicizumab. Haemophilia. 2023 Jan; 29(1):100-105.. View in PubMed
• Surgical outcomes in people with hemophilia A taking emicizumab prophylaxis: experience from the HAVEN 1-4 studies. Blood Adv. 2022 12 27; 6(24):6140-6150.. View in PubMed
• Risk factors for hospital acquired venous thromboembolism in congenital heart disease patients: A report from the children’s hospital acquired thrombosis (CHAT) consortium. Thromb Res. 2022 12; 220:116-120.. View in PubMed
• Risk of venous thromboembolism in pediatric hospitalized patients undergoing noncardiac surgery: A report from the Children’s Hospital-Acquired Thrombosis consortium. Res Pract Thromb Haemost. 2022 Oct; 6(7):e12810.. View in PubMed
• Current practices in pediatric hospital-acquired thromboembolism: Survey of the Children’s Hospital Acquired Thrombosis (CHAT) Consortium. Res Pract Thromb Haemost. 2022 Oct; 6(7):e12793.. View in PubMed
• Eptacog beta efficacy and safety in the treatment and control of bleeding in paediatric subjects (<12 years) with haemophilia A or B with inhibitors. Haemophilia. 2022 Jul; 28(4):548-556.. View in PubMed
• Long-term efficacy and safety of subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors. Blood Adv. 2022 06 14; 6(11):3422-3432.. View in PubMed
• Predictive Modeling Identifies Total Bleeds at 12-Weeks Postswitch to N8-GP Prophylaxis as a Predictor of Treatment Response. Thromb Haemost. 2022 Jun; 122(6):913-925.. View in PubMed
• Hemophilia A: Strategies for Improving Long-Term Holistic Management, Adherence, and Quality of Life. J Adv Pract Oncol. 2022 May; 13(Suppl 3):7-20.. View in PubMed
• Direct oral anticoagulants for use in paediatrics. Lancet Child Adolesc Health. 2022 03; 6(3):207-214.. View in PubMed
• Markers of hypercoagulability in children with newly diagnosed acute lymphoblastic leukemia. Pediatr Blood Cancer. 2022 03; 69(3):e29522.. View in PubMed
• A New Risk Assessment Model for Hospital-Acquired Venous Thromboembolism in Critically Ill Children: A Report From the Children’s Hospital-Acquired Thrombosis Consortium. Pediatr Crit Care Med. 2022 01 01; 23(1):e1-e9.. View in PubMed
• Post-hoc analysis on the long-term response to fixed-dose prophylaxis with N8-GP in patients with haemophilia A. Haemophilia. 2022 Jan; 28(1):27-35.. View in PubMed
• Direct Oral Anticoagulants: Overcoming the Challenges of Managing Venous Thromboembolism in Children. J Pediatr. 2022 01; 240:14-23.. View in PubMed
• Symptomatic pulmonary embolus after catheter removal in children with catheter related thrombosis: A report from the CHAT Consortium. J Thromb Haemost. 2022 01; 20(1):133-137.. View in PubMed
• Can we do something about ICH in hemophilia? Blood. 2021 12 30; 138(26):2750-2751.. View in PubMed
• Emerging drugs for hemophilia A: insights into phase II and III clinical trials. Expert Opin Emerg Drugs. 2021 12; 26(4):337-350.. View in PubMed
• The safety of activated eptacog beta in the management of bleeding episodes and perioperative haemostasis in adult and paediatric haemophilia patients with inhibitors. Haemophilia. 2021 Nov; 27(6):921-931.. View in PubMed
• Emicizumab prophylaxis in infants with severe haemophilia A without inhibitors: Illustrative real-world cases to support shared decision-making. Haemophilia. 2021 Sep; 27(5):724-729.. View in PubMed
• Determining the approximate factor VIII level of patients with severe haemophilia A on emicizumab using in vivo global haemostasis assays. Haemophilia. 2021 Sep; 27(5):730-735.. View in PubMed
• Management of children with hemophilia A: How emicizumab has changed the landscape. J Thromb Haemost. 2021 07; 19(7):1629-1637.. View in PubMed
• Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies. Blood. 2021 04 22; 137(16):2231-2242.. View in PubMed
• Safety evaluation of emicizumab prophylaxis in individuals with haemophilia A. Expert Opin Drug Saf. 2021 Apr; 20(4):387-396.. View in PubMed
• Pharmacokinetics and Pharmacodynamics of Emicizumab in Persons with Hemophilia A with Factor VIII Inhibitors: HAVEN 1 Study. Thromb Haemost. 2021 Mar; 121(3):351-360.. View in PubMed
• Outcomes for studies assessing the efficacy of hemostatic therapies in persons with congenital bleeding disorders. Haemophilia. 2021 Mar; 27(2):211-220.. View in PubMed
• BAX 335 hemophilia B gene therapy clinical trial results: potential impact of CpG sequences on gene expression. Blood. 2021 02 11; 137(6):763-774.. View in PubMed
• Key questions in the new hemophilia era: update on concomitant use of FVIII and emicizumab in hemophilia A patients with inhibitors. Expert Rev Hematol. 2021 02; 14(2):143-148.. View in PubMed
• Development of a Risk Model for Pediatric Hospital-Acquired Thrombosis: A Report from the Children’s Hospital-Acquired Thrombosis Consortium. J Pediatr. 2021 01; 228:252-259.e1.. View in PubMed
• Comparison of bypassing agents in patients on emicizumab using global hemostasis assays. Haemophilia. 2021 Jan; 27(1):164-172.. View in PubMed
• Implications of the first FDA-approved anticoagulant in pediatrics: One ship has sailed but the next ones are at the dock. Pediatr Blood Cancer. 2020 12; 67(12):e28731.. View in PubMed
• Emicizumab-Induced Seronegative Full-House Lupus Nephritis in a Child. Pediatrics. 2020 11; 146(5).. View in PubMed
• Health-related quality of life and caregiver burden of emicizumab in children with haemophilia A and factor VIII inhibitors-Results from the HAVEN 2 study. Haemophilia. 2020 Nov; 26(6):1009-1018.. View in PubMed
• Safety and efficacy of anticoagulant therapy in pediatric catheter-related venous thrombosis (EINSTEIN-Jr CVC-VTE). Blood Adv. 2020 10 13; 4(19):4632-4639.. View in PubMed
• Nonacog beta pegol (N9-GP) in hemophilia B: First report on safety and efficacy in previously untreated and minimally treated patients. Res Pract Thromb Haemost. 2020 Oct; 4(7):1101-1113.. View in PubMed
• Neutralizing antidrug antibody to emicizumab in a patient with severe hemophilia A with inhibitors: New case with detailed laboratory evaluation. J Thromb Haemost. 2020 09; 18(9):2205-2208.. View in PubMed
• FondaKIDS III: A long-term retrospective cohort study of fondaparinux for treatment of venous thromboembolism in children. Pediatr Blood Cancer. 2020 08; 67(8):e28295.. View in PubMed
• Rivaroxaban for treatment of pediatric venous thromboembolism. An Einstein-Jr phase 3 dose-exposure-response evaluation. J Thromb Haemost. 2020 07; 18(7):1672-1685.. View in PubMed
• The Edoxaban Hokusai VTE PEDIATRICS Study: An open-label, multicenter, randomized study of edoxaban for pediatric venous thromboembolic disease. Res Pract Thromb Haemost. 2020 Jul; 4(5):886-892.. View in PubMed
• Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: Final results from the ASPIRE extension study. Haemophilia. 2020 May; 26(3):494-502.. View in PubMed
• Patients with hemophilia A and inhibitors: prevention and evolving treatment paradigms. Expert Rev Hematol. 2020 04; 13(4):313-321.. View in PubMed
• Comparative validation study of risk assessment models for pediatric hospital-acquired venous thromboembolism. J Thromb Haemost. 2020 03; 18(3):633-641.. View in PubMed
• Peripherally inserted central catheters lead to a high risk of venous thromboembolism in children. Blood. 2020 01 16; 135(3):220-226.. View in PubMed
• Rivaroxaban compared with standard anticoagulants for the treatment of acute venous thromboembolism in children: a randomised, controlled, phase 3 trial. Lancet Haematol. 2020 Jan; 7(1):e18-e27.. View in PubMed
• A multicenter, open-label phase 3 study of emicizumab prophylaxis in children with hemophilia A with inhibitors. Blood. 2019 12 12; 134(24):2127-2138.. View in PubMed
• Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: phase 2 trial results. Blood. 2019 11 28; 134(22):1973-1982.. View in PubMed
• Bodyweight-adjusted rivaroxaban for children with venous thromboembolism (EINSTEIN-Jr): results from three multicentre, single-arm, phase 2 studies. Lancet Haematol. 2019 Oct; 6(10):e500-e509.. View in PubMed
• How I treat children with haemophilia and inhibitors. Br J Haematol. 2019 08; 186(3):400-408.. View in PubMed
• The changing face of immune tolerance induction in haemophilia A with the advent of emicizumab. Haemophilia. 2019 Jul; 25(4):676-684.. View in PubMed
• Diagnosis and treatment of hemophilia. Clin Adv Hematol Oncol. 2019 Jun; 17(6):344-351.. View in PubMed
• Why plasma-derived factor VIII? Haemophilia. 2019 May; 25(3):e183-e185.. View in PubMed
• Laboratory assay measurement of modified clotting factor concentrates: a review of the literature and recommendations for practice. J Thromb Haemost. 2019 Apr; 17(4):567-573.. View in PubMed
• Clinical utility of viscoelastic testing (TEG and ROTEM analyzers) in the management of old and new therapies for hemophilia. Am J Hematol. 2019 02; 94(2):249-256.. View in PubMed
• Efficacy of EHL N9-GP for on-demand treatment of bleeding episodes in hemophilia B: analysis of pivotal trial data. J Blood Med. 2019; 10:243-250.. View in PubMed
• The effect of emicizumab prophylaxis on health-related outcomes in persons with haemophilia A with inhibitors: HAVEN 1 Study. Haemophilia. 2019 Jan; 25(1):33-44.. View in PubMed
• Inhibitors: A Need for Eradication? Acta Haematol. 2019; 141(3):151-155.. View in PubMed
• Management of perioperative hemostasis in a severe hemophilia A patient with inhibitors on emicizumab using global hemostasis assays. Ther Adv Hematol. 2019; 10:2040620719860025.. View in PubMed
• Once-weekly prophylaxis with 40 IU/kg nonacog beta pegol (N9-GP) achieves trough levels of >15% in patients with haemophilia B: Pooled data from the paradigm™ trials. Haemophilia. 2018 Nov; 24(6):911-920.. View in PubMed
• Emicizumab for hemophilia A with factor VIII inhibitors. Expert Rev Hematol. 2018 11; 11(11):835-846.. View in PubMed
• The importance of inhibitor eradication in clinically complicated hemophilia A patients. Expert Rev Hematol. 2018 11; 11(11):857-862.. View in PubMed
• Implementing emicizumab in hemophilia inhibitor management: emicizumab should be prescribed after tolerance. Blood Adv. 2018 10 23; 2(20):2780-2782.. View in PubMed
• Obesity and risk for venous thromboembolism from contemporary therapy for pediatric acute lymphoblastic leukemia. Thromb Res. 2018 05; 165:44-50.. View in PubMed
• Deep vein thrombosis in pediatric patients. Pediatr Blood Cancer. 2018 03; 65(3).. View in PubMed
• A contemporary look at FVIII inhibitor development: still a great influence on the evolution of hemophilia therapies. Expert Rev Hematol. 2018 02; 11(2):87-97.. View in PubMed
• Novel therapies and current clinical progress in hemophilia A. Ther Adv Hematol. 2018 Feb; 9(2):49-61.. View in PubMed
• Rivaroxaban versus standard anticoagulation for acute venous thromboembolism in childhood. Design of the EINSTEIN-Jr phase III study. Thromb J. 2018; 16:34.. View in PubMed
• Exploratory evaluation of pharmacodynamics, pharmacokinetics and safety of rivaroxaban in children and adolescents: an EINSTEIN-Jr phase I study. Thromb J. 2018; 16:31.. View in PubMed
• A multi-institutional registry of pediatric hospital-acquired thrombosis cases: The Children’s Hospital-Acquired Thrombosis (CHAT) project. Thromb Res. 2018 01; 161:67-72.. View in PubMed
• Anticoagulation Therapies in Children. Pediatr Clin North Am. 2017 12; 64(6):1257-1269.. View in PubMed
• Pediatric Anticoagulation: Time for a New Paradigm? J Pediatr. 2017 10; 189:21-23.. View in PubMed
• How I treat pediatric venous thromboembolism. Blood. 2017 09 21; 130(12):1402-1408.. View in PubMed
• Safety and efficacy of recombinant activated coagulation factor VII in congenital hemophilia with inhibitors in the home treatment setting: A review of clinical studies and registries. Am J Hematol. 2017 Sep; 92(9):940-945.. View in PubMed
• Anticoagulation in children: Making the most of little patients and little evidence. Blood Cells Mol Dis. 2017 09; 67:48-53.. View in PubMed
• Emicizumab Prophylaxis in Hemophilia A with Inhibitors. N Engl J Med. 2017 08 31; 377(9):809-818.. View in PubMed
• Tailoring treatment of haemophilia B: accounting for the distribution and clearance of standard and extended half-life FIX concentrates. Thromb Haemost. 2017 06 02; 117(6):1023-1030.. View in PubMed
• Thromboelastographic characterization of the activated clotting system in children with sickle cell trait or sickle cell disease. Thromb Res. 2017 Mar; 151:44-50.. View in PubMed
• Treatment of Venous Thromboembolism in Pediatric Patients. Front Pediatr. 2017; 5:26.. View in PubMed
• The perils of rivaroxaban in younger patients. Thromb Res. 2016 12; 148:154-155.. View in PubMed
• Impact of high-risk thrombophilia status on recurrence among children with a first non-central-venous-catheter-associated VTE: an observational multicentre cohort study. Br J Haematol. 2016 Oct; 175(1):133-40.. View in PubMed
• The past and future of haemophilia: diagnosis, treatments, and its complications. Lancet. 2016 Jul 09; 388(10040):187-97.. View in PubMed
• Long-acting recombinant factor VIII Fc fusion protein (rFVIIIFc) for perioperative haemostatic management in severe haemophilia A. Thromb Haemost. 2016 07 04; 116(1):1-8.. View in PubMed
• A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A. N Engl J Med. 2016 May 26; 374(21):2054-64.. View in PubMed
• Nonacog beta pegol (N9-GP) in haemophilia B: A multinational phase III safety and efficacy extension trial (paradigm™4). Thromb Res. 2016 May; 141:69-76.. View in PubMed
• The bleeding newborn: A review of presentation, diagnosis, and management. Semin Fetal Neonatal Med. 2016 Feb; 21(1):44-9.. View in PubMed
• Global assays in hemophilia. Semin Hematol. 2016 Jan; 53(1):40-5.. View in PubMed
• Understanding patient preferences and willingness to pay for hemophilia therapies. Patient Prefer Adherence. 2015; 9:1623-30.. View in PubMed
• Anticoagulants in children and adolescents. Hematology Am Soc Hematol Educ Program. 2015; 2015:111-6.. View in PubMed
• Evaluation of algorithms for the treatment of problem bleeding episodes in patients with hemophilia having inhibitors. Clin Appl Thromb Hemost. 2015 Jan; 21(1):10-8.. View in PubMed
• Recombinant long-acting glycoPEGylated factor IX in hemophilia B: a multinational randomized phase 3 trial. Blood. 2014 Dec 18; 124(26):3880-6.. View in PubMed
• FondaKIDS II: long-term follow-up data of children receiving fondaparinux for treatment of venous thromboembolic events. Thromb Res. 2014 Sep; 134(3):643-7.. View in PubMed
• Developmental hemostasis: clinical implications from the fetus to the adolescent. Pediatr Clin North Am. 2013 Dec; 60(6):1407-17.. View in PubMed
• Pharmacokinetics and pharmacodynamics of anticoagulants in paediatric patients. Clin Pharmacokinet. 2013 Nov; 52(11):967-80.. View in PubMed
• Incidence of thrombosis in children with tunneled central venous access devices versus peripherally inserted central catheters (PICCs). Thromb Res. 2013 Nov; 132(5):527-30.. View in PubMed
• Antithrombin III administration in neonates with congenital diaphragmatic hernia during the first three days of extracorporeal membrane oxygenation. J Pediatr Surg. 2013 Sep; 48(9):1837-42.. View in PubMed
• A novel approach for detecting hypercoagulability utilizing thromboelastography. Thromb Res. 2013 Apr; 131(4):352-6.. View in PubMed
• Thrombin generation and whole blood viscoelastic assays in the management of hemophilia: current state of art and future perspectives. Blood. 2013 Mar 14; 121(11):1944-50.. View in PubMed
• Factor VIII inhibitors in hemophilia A: rationale and latest evidence. Ther Adv Hematol. 2013 Feb; 4(1):59-72.. View in PubMed
• PRO-PACT: retrospective observational study on the prophylactic use of recombinant factor VIIa in hemophilia patients with inhibitors. Thromb Res. 2012 Dec; 130(6):864-70.. View in PubMed
• Dosing and effectiveness of recombinant activated factor VII (rFVIIA) in congenital haemophilia with inhibitors by bleed type and location: the experience of the Haemophilia and Thrombosis Research Society (HTRS) Registry (2004-2008). Haemophilia. 2012 Nov; 18(6):990-6.. View in PubMed
• Eradication of factor VIII inhibitors in patients with mild and moderate hemophilia A. Am J Hematol. 2012 Sep; 87(9):933-6.. View in PubMed
• Pharmacokinetic- and pharmacodynamic-based antithrombotic dosing recommendations in children. Expert Rev Clin Pharmacol. 2012 Jul; 5(4):389-96.. View in PubMed
• Difficult clinical challenges in haemophilia: international experiential perspectives. Haemophilia. 2012 Jul; 18 Suppl 5:39-45.. View in PubMed
• From boy to man: recommendations for the transition process in haemophilia. Haemophilia. 2012 Jul; 18 Suppl 5:27-32.. View in PubMed
• Patient/caregiver-reported recombinant factor VIIa (rFVIIa) dosing: home treatment of acute bleeds in the Dosing Observational Study in Hemophilia (DOSE). Haemophilia. 2012 May; 18(3):392-9.. View in PubMed
• New challenges in hemophilia: long-term outcomes and complications. Hematology Am Soc Hematol Educ Program. 2012; 2012:362-8.. View in PubMed
• Capturing daily assessments and home treatment of congenital hemophilia with inhibitors: design, disposition, and implications of the Dosing Observational Study in Hemophilia (DOSE). J Blood Med. 2012; 3:131-8.. View in PubMed
• Old and new antithrombotic drugs in neonates and infants. Semin Fetal Neonatal Med. 2011 Dec; 16(6):349-54.. View in PubMed
• FondaKIDS: a prospective pharmacokinetic and safety study of fondaparinux in children between 1 and 18 years of age. Pediatr Blood Cancer. 2011 Dec 01; 57(6):1049-54.. View in PubMed
• Impact of persistent antiphospholipid antibodies on risk of incident symptomatic thromboembolism in children: a systematic review and meta-analysis. Semin Thromb Hemost. 2011 Oct; 37(7):802-9.. View in PubMed
• When should prophylaxis therapy in inhibitor patients be considered? Haemophilia. 2011 Sep; 17(5):e849-57.. View in PubMed
• Argatroban therapy in pediatric patients requiring nonheparin anticoagulation: an open-label, safety, efficacy, and pharmacokinetic study. Pediatr Blood Cancer. 2011 Jul 01; 56(7):1103-9.. View in PubMed
• Modeling costs and outcomes associated with a treatment algorithm for problem bleeding episodes in patients with severe hemophilia a and high-titer inhibitors. Am Health Drug Benefits. 2011 Jul; 4(4):219-31.. View in PubMed
• Pediatric catheterization laboratory anticoagulation with bivalirudin. Catheter Cardiovasc Interv. 2011 Apr 01; 77(5):671-9.. View in PubMed
• New anticoagulants in children: a review of recent studies and a look to the future. Thromb Res. 2011 Feb; 127(2):70-4.. View in PubMed
• Pharmacokinetic and pharmacodynamic basis for effective argatroban dosing in pediatrics. J Clin Pharmacol. 2011 Jan; 51(1):19-28.. View in PubMed
• Transitioning issues in adolescent to young adult hemophilia patients with inhibitors: an approach for a growing population. Blood Coagul Fibrinolysis. 2010 Sep; 21 Suppl 1:S7-10.. View in PubMed
• Comparison of kaolin and tissue factor activated thromboelastography in haemophilia. Haemophilia. 2010 May; 16(3):518-24.. View in PubMed
• Impact of thrombophilia on risk of arterial ischemic stroke or cerebral sinovenous thrombosis in neonates and children: a systematic review and meta-analysis of observational studies. Circulation. 2010 Apr 27; 121(16):1838-47.. View in PubMed
• Off-label use of rFVIIa in children with excessive bleeding: a consecutive study of 153 off-label uses in 139 children. Pediatr Blood Cancer. 2009 Aug; 53(2):179-83.. View in PubMed
• Bivalirudin for anticoagulation in children. Pediatr Blood Cancer. 2008 Dec; 51(6):798-801.. View in PubMed
• Impact of inherited thrombophilia on venous thromboembolism in children: a systematic review and meta-analysis of observational studies. Circulation. 2008 Sep 23; 118(13):1373-82.. View in PubMed
• Evaluation of thromboelastography for monitoring recombinant activated factor VII ex vivo in haemophilia A and B patients with inhibitors: a multicentre trial. Blood Coagul Fibrinolysis. 2008 Jun; 19(4):276-82.. View in PubMed
• 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 comparison. Haemophilia. 2008 Mar; 14(2):287-94.. View in PubMed
• New anticoagulants in children. Hematology Am Soc Hematol Educ Program. 2008; 245-50.. View in PubMed
• Recombinant activated factor VII effectively reverses the anticoagulant effects of heparin, enoxaparin, fondaparinux, argatroban, and bivalirudin ex vivo as measured using thromboelastography. Blood Coagul Fibrinolysis. 2007 Sep; 18(6):547-53.. View in PubMed
• Pilot dose-finding and safety study of bivalirudin in infants <6 months of age with thrombosis. J Thromb Haemost. 2007 Aug; 5(8):1654-9.. View in PubMed
• Differential effects of direct thrombin inhibitors and antithrombin-dependent anticoagulants on the dynamics of clot formation. Blood Coagul Fibrinolysis. 2007 Mar; 18(2):97-103.. View in PubMed
• Heparin use in children. Pediatr Res. 2007 Feb; 61(2):139-40.. View in PubMed
• Safety and pharmacokinetics of recombinant factor XIII-A2 administration in patients with congenital factor XIII deficiency. Blood. 2006 Jul 01; 108(1):57-62.. View in PubMed
• Diagnosis and treatment of thrombosis in children: general principles. Pediatr Blood Cancer. 2006 May 01; 46(5):540-6.. View in PubMed
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• Demetriades, Demetrios
• Hay, Joel
• Inaba, Kenji
• Martin, Matthew
• Liebman, Howard
• Lieberman, Jay
• Nichol, Michael
• Matsushima, Kazuhide