Research Presented at Our Conference

Leading an Active Lifestyle with Hemophilia B: Estimating the Bleeding Risk with Different FIX Treatment Regimens

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Biomedical/Coagulation Research

RESEARCHERS:

Yan Xiong, Alexandra Khachatryan, Josh Epstein

A Half-Life Extended Fusion Peptide Inhibitor of TFPI Improves Hemostasis in Preclinical Models of Hemophilia

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Biomedical/Coagulation Research

RESEARCHERS:

Michael Dockal, Rudolf Hartmann, Thomas Polakowski, Erwin Panholzer, Willibald Kammlander, Frank Osterkamp, Ulrich Reineke, Alexandra Schiviz, Werner Hoellriegl, Friedrich Scheiflinger

Pharmacokinetics, safety, and efficacy of pdFX, a new high-purity factor X concentrate: a phase 3 study in patients with moderate or severe hereditary factor X deficiency

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

New Products

RESEARCHERS:

Steven Austin, Miranda Norton

Patient Satisfaction with US Hemophilia Treatment Centers 2015: National Results

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Quality of Life/Outcomes Research

RESEARCHERS:

Judith Baker, Karen Droze, Rick Shearer, Kathryn McLaughlin, Brenda Riske

My Life, Our Future: A “Genetics Day” to Facilitate Efficient Enrollment

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Collaboration/Team Models

RESEARCHERS:

Julie Smith, Nancy Hatcher, Frances Patterson, Ruthrolen Martinez, Vinod Balasa

Long-term safety and efficacy of recombinant factor VIII Fc (rFVIIIFc) for the treatment of severe hemophilia A: United States subgroup interim analysis of the ASPIRE study

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Clinical Research/Clinical Trials

RESEARCHERS:

Doris Quon, Guy Young, Margaret Ragni, Barbara Konkle, Roshni Kulkarni, Amy Shapiro, Elisa Tsao, Lynda Cristiano, Geoffrey Allen

SPACE (Study of Prophylaxis ACtivity, and Effectiveness): An interim descriptive analysis of patient activity levels and participation

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Quality of Life/Outcomes Research

RESEARCHERS:

Barbara Konkle, Michael Recht, Maggie Moore, Susan Lattimore, Elizabeth L Schwartz, Diane Ito, Josh Epstein, Iliana Leony-Lasso, Sharon A Richardson

Long-term safety and prophylactic efficacy of once-weekly subcutaneous administration of ACE910 in Japanese hemophilia A patients with and without FVIII inhibitors: Interim results of the extension study of a phase I study

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Clinical Research/Clinical Trials

RESEARCHERS:

Midori Shima

Update on a phase 1/2 open-label trial of BAX 335, an adeno-associated virus 8 (AAV8) vector-based gene therapy program for hemophilia B

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Clinical Research/Clinical Trials

RESEARCHERS:

Paul E. Monahan, Christopher E. Walsh, Barbara A. Konkle, Jerry S. Powell, Neil C. Josephson, Miguel A. Escobar, Scott W. McPhee, Boyan Litchev, Michael Cecerle, Bruce Ewenstein, Hanspeter Rottensteiner, Maurus De la Rosa, Birgit M. Reipert, R. Jude Samulski, Anne Prener, Friedrich Scheiflinger

Assessment of motor proficiency in people with bleeding disorders using the Bruininks-Oseretsky Test of Motor Proficiency, Second Edition (BOT-2™)

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Orthopedic and Physical Therapy

RESEARCHERS:

Bethany Sloane, PT, DPT, Brittany Gurgel, PT, DPT, Nancy Durben, MSPT, PCS, Paul Sochacki, MS, David Oleson, PT, PCS, Michael Recht, MD, PhD

A Novel Anti-Tissue Factor Pathway Inhibitor Antibody, BAY 1093884, Prevents Bleeding in Hemophilia A Mice

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Biomedical/Coagulation Research

RESEARCHERS:

Maria Koellnberger, Perry Liu, Derek Sim

Building ‘Zoris in the Sand’ – Best Practice for Bleeding Disorder Capacity Building in the Underserved US Commonwealth of the Northern Mariana Islands

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Peer Support/Outreach/Integration Models

RESEARCHERS:

Tiffany F. Lin, Pam Carhill, Judith Baker, James N. Huang

Kids B-LONG: Safety, Efficacy, and Pharmacokinetics of Long-Acting Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Previously Treated Children with Hemophilia B

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Clinical Research/Clinical Trials

RESEARCHERS:

Roshni Kulkarni, Kathelijn Fischer, Beatrice Nolan, Johnny Mahlangu, Savita Rangarajan, Giulia Gambino, Lei Diao, Alejandra Ramirez-Santiago, Glenn F Pierce, Geoffery Allen

Real-world dosing and patient characteristics of rFVIIIFc in hemophilia A patients

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

New Products

RESEARCHERS:

Brieana Buckley, Eric Hall, Ben Hagberg, Sangeeta Krishnan, Adi Eldar-Lissai

Swimming exercise ameliorates pain, swelling and bone quality in a blood-induced joint damage animal model

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Orthopedic and Physical Therapy

RESEARCHERS:

Fabio Souza, Liliam Takayama, Fernanda Roque, Gisele Picolo, Rosa Pereira, Edilamar Oliveira, Yara Cury, Clarice Tanaka, Suzana Mello

Efficacy of a Recombinant Factor IX Investigational Product, IB1001 (trenonacog alfa) in Previously Treated Patients with Hemophilia B

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Clinical Research/Clinical Trials

RESEARCHERS:

Bojan Drobic, Yi Hua, Tim Babinchak, Edward Gomperts

Pediatric Venous Thrombosis Associated With Staphylococcal Infections: A Single Institutional Experience

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Clinical Research/Clinical Trials

RESEARCHERS:

Divyaswathi Citla Sridhar, Ossama M. Maher, Fernando Corrales-Medina, Hatel Rana Moonat, Jorge Galvez Silva, Trinh Nguyen, Deborah Brown, Nidra I. Rodriguez

Description and Management of Pain and Functional Impairment in US Adults With Hemophilia: Initial Observations From the Pain, Functional Impairment, and Quality of Life (P-FiQ) Study

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Quality of Life/Outcomes Research

RESEARCHERS:

Michelle Witkop, Kim Baumann, Tyler Buckner, Stacie Akins, Sharon Funk, Grace Hernandez, Michael Recht, Michael Wang, Katharine Batt, Christine Kempton, David Cooper

Long-term safety and efficacy of recombinant factor IX Fc (rFIXFc) in adults and adolescents with severe hemophilia B: interim results of the B-YOND study

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Clinical Research/Clinical Trials

RESEARCHERS:

Amy D Shapiro, John Pasi, Margaret Ragni, Johnny Mahlangu, Margareth Ozelo, Johannes Oldenburg, Tadashi Matsushita, Ross I Baker, Huixing Yuan, Alejandra Ramirez-Santiago, Glenn F Pierce, Geoffrey Allen, Baisong Mei

Low Annualized Bleeding Rates in Phase 3 Studies of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Subjects with Target Joints at Baseline

AWARDED/PRESENTED: 2015

GRANT/PROGRAM:

Bleeding Disorders Conference

Clinical Research/Clinical Trials

RESEARCHERS:

Michael Wang, Beatrice Nolan, Guy Young, Savita Rangarajan, Bryce Kerlin, Roshni Kulkarni, Margaret V. Ragni, Ross I. Baker, Elisa Tsao, Desilu Glazebrook, Glenn Pierce, Geoffrey Allen