510Hemophilia Natural History Study (ATHN 7): Baseline Characteristics, Adverse Events, andSelf-Reported Health Status of Individuals with Hemophilia A and B
AUTORES Tyler W. Buckner, MD, MSc1; Shannon L Carpenter, MD2; Stacy E. Croteau, MD3; Adam Cuker, MD, MS4; Nabil Daoud, MA5;Christine L Kempton, MD, MSc6; Lynn M Malec, MD7; Thomas W. McLean, MD8; Leslie J. Raffini, MD9; Janice M Staber, MD10; Michael Wang, MD11, Crystal Watson, BS5; Ayesha Zia12 and Michael Recht, MD, PhD5,13 . 1Divisions of Hematology and Pediatric Hematology/Oncology, Hemophilia and Thrombosis Center, University of Colorado School of Medicine, Aurora, CO; 2Children’s Mercy Hospital, Kansas City, MO; 3Children’s Hospital Boston, Boston, MA; 4Department of Medicine and Department of Pathology and Laboratory Medicine, University of Pennsylvania, Philadelphia, PA; 5American Thrombosis and Hemostasis Network, Rochester, NY; 6Emory University, Atlanta, GA; 7Blood Research Institute, Versiti, Milwaukee, WI; 8Wake Forest University School of Medicine, Winston Salem, NC; 9Division of Hematology, The Children’s Hospital of Philadelphia, Philadelphia, PA; 10University of Iowa Stead Family Children’s Hospital, Iowa City, IA; 11Anschutz Medical Campus, University of Colorado, School of Medicine, Hemophilia and Thrombosis Center, Aurora, CO; 12University of Southwestern Medical Center, Dallas, TX; 13The Hemophilia Center, Oregon Health and Sci. Univ., Portland, OR.
868Inhibit Clinical Trials Platform to Prevent and Eradicate Inhibitors: Feasibility Survey of Current Prophylaxis and Immune Tolerance Practices
AUTORES Margaret V. Ragni, MD, MPH1; Frederico Xavier, MD, MS2; Craig D. Seaman3; Suchitra Acharya, MD4; Catherine E. McGuinn, MD5; Eric J. Werner, MD6; Courtney Elizabeth Lawrence, MD, MS7; Allison P. Wheeler, MD8; Ulrike Reiss, MD9; Irmel Ayala, MD10; Erin Cockrell, DO11; Cristina Tarango, MD12; Amy Dunn13; Roshni Kulkarni, MD14; Sanjay P Ahuja, MD15; Meera B. Chitlur, MD16; Steven W. Pipe, MD17; Lynn M Malec, MD18; Vilmarie Rodriguez, MD19; Shelley Crary, MD, MS20; Deborah Brown, MD21; Rosa Diaz, MD22; Maria Velez, MD23; Cindy A. Leissinger, MD24; Shannon L Carpenter, MD25; Christine M. Knoll, MD26; Michael Wang, MD27; Guy Young, MD28; Courtney D ThornburgMD29 ; Joseph L Lasky III, MD30; Tiffany Lin Lucas, MD31; Nina Hwang, MD32; Deborah Vehec, MSN, RN33; Dana Ivanco34; Tamara L. Haller, BS35; Marnie Bertolet, PhD36; Maria M Brooks, PhD37
1University of Pittsburgh Medicine, Division Hematology/Oncology, Pittsburgh, PA; 2Children’s Hospital of Pittsburgh, University of Pittsburgh Medical Center and Hemophilia Center of Western PA,Pittsburgh, PA; 3University of Pittsburgh, Warrendale, PA; 4Pediatric Hematology, Oncology and Cellular Therapy, Cohen Children’s Medical Center of New York, New Hyde Park, NY; 5Weill Cornell Medical College, New York, NY; 6Children’s Hospital of The King’s Daughters, Norfolk, VA; 7Division of Pediatric Hematology / Transfusion Medicine, Johns Hopkins University School of Medicine, Baltimore, MD; 8Dept. of Pediatric Hem./Onc., Monroe Carell Jr. Children’s Hospital at Vanderbilt, Nashville, TN; 9Department of Surgery, St. Jude Children’s Rsch. Hosp., Memphis, TN; 10Pediatric Cancer and BloodDisorders Center, Johns Hopkins All Children’s Outpatient Care Center, St. Petersburg, FL; 11St. Joseph’s Children’s Hospital, Tampa, FL; 12Division of Hematology, Cancer and Blood Diseases Institute, Cincinnati Children ’s Hospital Medical Center, Cincinnati, OH; 13Nationwide Children’s Hospital, Columbus, OH; 14Michigan State University Center for Bleeding and Clotting Disorders, East Lansing, MI; 15University Hospitals Cleveland Medical Center, Rainbow Babies and Children’s Hospital, Cleveland, OH; 16Hemostasis and Thrombosis Center, Children’s Hospital of Michigan, Detroit, MI; 17Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI; 18Blood Research Institute, Versiti, Milwaukee, WI; 19Division of Pediatric Hematology, Mayo Clinic, Rochester, MN; 20University of Arkansas for Medical Sciences, Little Rock, AR; 21Department of Pediatrics- Hematology Division, University of Texas Health Science Center at Houston, Houston, TX; 22Texas Children’s Hospital, Houston, TX; 23Children’s Hospital New Orleans, Louisiana State University, New Orleans, LA; 24Tulane University, New Orleans, LA; 25Children’s Mercy Hospital, Kansas City, MO; 26Arizona Hemophilia and Thrombosis Center, Phoenix Children’s Hospital, Phoenix, AZ; 27Anschutz Medical Campus, University of Colorado, School of Medicine, Hemophilia and Thrombosis Center, Aurora, CO; 28University of Califormia Los Angeles, Children’s Hospital Los Angeles, Los Angeles, CA; 29Rady Children’s Hospital, San Diego, CA; 30University of Nevada, Cure 4 the Kids Foundation, Las Vegas, NV; 31University of California, San Francisco, San Francisco, CA; 32CHOC Children’s Hospital, Orange County, Center for Inherited Blood Disorders (CIBD), Orange, CA; 33Hemophilia Center of Western PA, Institute for Transfusion Medicine, Vitalant, Pittsburgh, PA; 34Institute for Transfusion Medicine, Hemophilia Center of Western PA, Pittsburgh, PA; 35Department of Epidemiology, Epidemiology Data Center, University of Pittsburgh, Pittsburgh, PA; 36Epidemiology Data Center, University of Pittsburgh, Pittsburgh, PA; 37Graduate School of Public Health, University of Pittsburgh, Pittsburgh, PA.
1Thrombosis and Hemostasis Research Center, Nara Medical University, Kashihara, Japan; 2Chugai Pharmaceutical Co., Ltd., Tokyo, Japan; 3Department of Hematology, Gunma University Graduate School of Medicine, Maebashi, Japan; 4Department of Laboratory Medicine, Tokyo Medical University, Tokyo, Japan; 5Department of Pediatrics, Nara Medical University, Kashihara, Japan.
1787 Real-World Clinical Outcomes in Previously Untreated and Minimally Treated Patients with Congenital Factor VIII Deficiency: The San Diego Experience
AUTORES Kelly A Bush, MD1,2; Hilda Ding, MD, MS1,2; Kate O’Flaherty-Keese, RN, MSN, CPNP1; Rosalie Brooks, RN1; Katharine Farrow, RN, BSN1; Jovanee Recendez, MPH1; Jacqueline Limjoco, RN1; Courtney D Thornburg, MD1,2.
1Hemophilia and Thrombosis Treatment Center, Rady Children’s Hospital San Diego, San Diego, CA; 2Department of Pediatrics, Division of Hematology/Oncology, UC San Diego School of Medicine, La Jolla, CA.
1Pediatric Hematology, Oncology and Cellular Therapy, Cohen Children’s Medical Center, New Hyde Park, NY; 2Pediatric Hematology, Oncology and Cellular Therapy, Cohen Children’s Medical Center of New York, New Hyde Park, NY.
1793 Analysis of Hemophilia a Outcomes and Treatment Patterns Using Real-World Data from the Canadian Hemophilia Bleeding Disorder Registry
AUTORES Adrienne Lee, MD1; Federico Germini, MD, MSc2; Man-Chiu Poon, MD3; Arun Keepanasseril2; Quazi Ibrahim, MSc2; Aijing Shang,MD, PhD4; Francis Nissen4; Oliver Meier4; Cami S. Sima, MD5; Davide Matino2; Alfonso Iorio, MD, PhD6,7.
1Department of Medicine, Cumming School of Medicine University of Calgary, Calgary, AB, Canada; 2Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, ON, Canada; 3University of Calgary Cumming School of Medicine, Alberta, Canada; 4F. Hoffmann-La Roche Ltd, Basel, Switzerland; 5Genentech, Inc., South San Francisco, CA; 6Department of Health Research Methods, Evidence, and Impact, McMaster University, Hamilton, Canada; 7McMaster-Bayer Endowed Research Chair in Clinical Epidemiology of Congenital bleeding Disorders, Department of Medicine, McMaster University, Hamilton, Canada.
1800Safety and Efficacy of Emicizumab in Persons with Hemophilia a with or without FVIII Inhibitors: Pooled Data from Four Phase III Studies (HAVEN 1–4)
AUTORES Michael U. Callaghan, MD1; Claude Négrier2; Ido Paz-Priel, MD3; Tiffany Chang3; Sammy Chebon4; Michaela Lehle4; Johnny Mahlangu5; Guy Young, MD6; Rebecca Kruse-Jarres, MD7; Maria Elisa Mancuso8, Markus Niggli4; Peter Kuebler3; Nives Selak Bienz4; Midori Shima, MD9; Victor Jimenez-Yuste, Medical Degree. PhD10; Christophe Schmitt4; Elina Asikanius4; Gallia Levy3; Steven W. Pipe, MD11; Johannes Oldenburg12.
1Children’s Hospital of Michigan, Detroit, MI; 2Edouard Herriot University Hospital, Lyon, France; 3Genentech, Inc., South San Francisco, CA; 4F. Hoffmann-La Roche Ltd, Basel, Switzerland; 5University of the Witwatersrand and NHLS, Johannesburg, South Africa; 6University of Southern California Keck School of Medicine, Children’s Hospital Los Angeles, Los Angeles, CA; 7BloodWorks NorthWest, Seattle, WA; 8Angelo Bianchi Bonomi Haemophilia and Thrombosis Center, Fondazione IRCCS Ca’ Granda Ospedale Maggiore Policlinico, Milano, Italy; 9Nara Medical University Hospital, Kashihara, Japan; 10Hospital Universitario La Paz, Autonoma University, Madrid, Spain; 11University of Michigan, Ann Arbor, MI; 12University of Bonn, Bonn, Germany.
2532Problem Joints and Their Clinical and Humanistic Burden in Children and Adults with Moderate and Severe Hemophilia a: CHESS Paediatrics and CHESS II
AUTORES Paul McLaughlin, MSc, MMACP1; Cedric Hermans, MD, MRCP, PhD2; Sohaib Asghar, MSc3; Tom Burke3; Francis Nissen4; Martynas Aizenas5; Oliver Meier4; Harpal Dhillon3; Jamie O’Hara, MSc3,6.
1Katharine Dormandy Haemophilia Centre and Thrombosis Unit, Royal Free London NHS Foundation Trust, London, United Kingdom; 2Division of Hematology, Cliniques Univ. Saint-Luc, Brussels, Belgium; 3HCD Economics, Daresbury, United Kingdom; 4F. Hoffmann-La Roche Ltd, Basel, Switzerland; 5F. Hoffmann-La Roche Ltd, Basel, CA, Switzerland; 6University of Chester, Chester, United Kingdom.
2697 In Vitro Evaluation of Thrombin Generation of Eptacog Beta (Factor VIIa, Recombinant) and Emicizumab in Congenital Hemophilia Α Plasma with and without Inhibitors
AUTORES Steven W. Pipe, MD1; Michael Recht, MD, PhD2; Michael U. Callaghan, MD3; Robert F. Sidonio Jr., MD, MSc.4; Jerry Grandoni, PhD5; Veronique Duretz6; Daniel Bonzo, PhD5; Jean Luc Plantier, PhD7; Sean Evans, PhD, RPh5; Ian S Mitchell, PhD8; Cedric Hermans, MD, MRCP, PhD9.
1Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI; 2American Thrombosis and Hemostasis Network, Rochester, NY; 3Children’s Hospital of Michigan, Detroit, MI; 4Aflac Cancer and Blood Disorders and Emory University, Atlanta, GA; 5LFB, Framingham, MA; 6LFB, Lille, France; 7LFB, Loos, France; 8GLOVAL, LLC, Lafayette, CO; 9Cliniques Univ. Saint-Luc, Brussels, Belgium.
3457Impact of Hemophilia a Inhibitor on Joint Health and Health-Related Quality of Life from the Hemophilia Utilization Group Studies Part VIII in the U.S
AUTORES Megan M. Ullman, MA, MPH1; Marilyn J Manco-Johnson, MD2; Jonathan C. Roberts, MD3; Nicole Crook, RN4; Rahul Khairnar5; Joanne Wu, MD, MS6; Steven Carrasco, MPH7; Randall Curtis, MBA8; Judith Baker, DrPH, MHSA4; Duc Quang Tran Jr., MD9; Michael B. Nichol, PhD7.
1Gulf States Hemophilia & Thrombophilia Center, University of Texas Health Science Center at Houston, Houston, TX; 2University of Colorado Anschutz Medical Campus, Aurora, CO; 3Bleeding & Clotting Disorders Institute, Peoria, IL; 4Center for Inherited Blood Disorders, Orange, CA; 5Genentech, Inc., South San Francisco, CA; 6University Of Southern California, Los Angeles, CA; 7University of Southern California, Los Angeles, CA; 8Factor VIII Computing, Berkeley, CA; 9Emory University, Hemophilia of Georgia Center for Bleeding & Clotting Disorders of Emory, Atlanta, GA.