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Project Title: Impact of Geographical Distance from Hemophilia Treatment Centers on Outcomes in Patients with Bleeding Disorders
Mentor: Michael Recht, MD, Professor, Yale School of Medicine, and Chief Medical and Scientific7 Officer, National Bleeding Disorders Foundation
Abstract: This project aims to generate hypotheses by investigating how geographical distance from Hemophilia Treatment Centers (HTC) impacts patient outcomes. Timely access to HTCs notably reduces mortality and hospitalizations byat least 40% among hemophilia patients who received care at an HTC. Access to care is a known significant barrier and geographical barriers have been recurrent themes while evaluating barriers to access for people with bleeding disorders. Using patient-reported data and spatial analysis, we hope to identify disparities and inform targeted interventions. This research will lay groundwork for further exploration and care optimization for patients with bleeding disorders.
Funding Period: Two years

Project Title: Prevalence and Risk Factors for Thrombosis in People with Congenital Bleeding Disorders
Mentor: Vilmarie Rodriguez, MD, MS
Abstract: The overall objective of this study is to use the ATHNdataset to determine the prevalence and risk factors for thrombosis in congenital bleeding disorders. This multicenter collaborative project will help advance our understanding of thrombotic complications in bleeding disorders and close the current gaps in knowledge, with the aim of improving patient care and clinical outcomes.
Funding Period: Two years

Project Title: Factor-ONE-ing for Surgery: An Assessment and Survey of Perioperative Management for Hereditary Fibrinogenemias Across US Hemophilia Treatment Centers
Mentor: Margaret Ragni, MD, MPH
Co-Mentor: Peter Kouides, MD, University of Rochester
Abstract: This project for the DREAM Award aims to improve surgical care for patients with inherited fibrinogen disorders by investigating current surgical care across ATHN-affiliated hemophilia treatment centers (HTCs). The ATHNdataset will be utilized to describe the disease, treatment, procedure type, and capture surgical bleeding complications by treatment strategy.
Funding Period: Two years

Project Title: Pediatric Antiphospholipid Syndrome: A National Registry Study
Mentor: Steve Pipe MD, University of Michigan
Funding Period: Two years

Project Title: Elucidating the age-dependent variation of clinical and laboratory phenotype of hemophilia carriers along with prescription trends of hemostatic therapies - Analysis of the ATHNdataset
Mentor: Anjali Sharathkumar, MD, MS, University of Iowa
Co-Mentor: Peter Kouides, MD, University of Rochester
Funding Period: One year

Project Title: Platelet Function Disorders – Practice Patterns from U.S. Hemophilia Treatment Centers
Mentor: Sanjay P. Ahuja, MD, MSc, MBA, University Hospitals Cleveland Medical Center, Rainbow Babies and Children’s Hospital
Co-Mentor: Robert F. Sidonio Jr., MD, Aflac Cancer and Blood Disorders Center, Hemophilia of Georgia Center for Bleeding and Clotting Disorders
Funding Period: One year

Project Title: Characterizing the Real-World Use of Direct Oral Anticoagulants in Pediatric Thrombosis Patients Using ATHN: a VENUS Subcommittee Study
Mentor: Leslie Raffini, MD, MSCE, Children’s Hospital of Philadelphia
Co-Mentor: Courtney Thornburg, MD, Rady Children’s Hospital
Collaborator: Fernando F. Corrales-Medina, MD, FAAP, Assistant Professor of Clinical Pediatrics, University of Miami Miller School of Medicine
Funding Period: Two years

Project Title: Impact of Obesity on Laboratory Profiles, Treatment, and Outcomes of Individuals with Type 1 VWD and Low VWF
Mentor: Peter A. Kouides, MD, University of Rochester School of Medicine/Mary M. Gooley Hemophilia Center
Funding Period: One year

Project Title: ATHENA1: Characterizing the Impact and Treatment of Reproductive Tract Bleeding on Women with Bleeding Disorders
Mentor: Michael Recht, MD, PhD, Professor of Pediatrics and Medicine, Oregon Health & Science University
Co-Mentor: Roshni Kulkarni, MD, Professor Emerita, Michigan State University
Collaborators: Robert F. Sidonio, Jr., MD, MSc, Assistant Professor, Associate Director of Hemostasis and Thrombosis, Emory University and Shirley Abraham, MD, Director, Ted R. Montoya Hemophilia Treatment Center, Assistant Professory, Pediatrics, University of New Mexico
Funding Period: Two years

Project Title: Is Prophylaxis Putting Hemophilia Joints in the PINK: An ATHN-LINKED Observational Study
Abstract: The overall objective of this study is to discern use the impact of extended half-life (EHL) products in prophylaxis by determining the proportion of patients with severe hemophilia adopting its use and the impact on bleeding events, joint health and quality of life. 
Mentor: Margaret V. Ragni, MD, Professor of Medicine, University of Pittsburgh
Funding Period: Two years

Project Title: Evolving Real-World Use of Standard and EHL Products in Moderate and Severe Hemophilia Patients: A Practical Application of the ATHNdataset
Abstract: This longitudinal, observational study characterizes the evolving real-world use of factor replacement products for patients with moderate and severe hemophilia A and B without inhibitors. Serial queries of the ATHNdataset at eight-month intervals will compare the proportions of factor products prescribed, median weekly factor consumption and annual infusion rate by factor product and the impact of EHL products on prophylaxis rates. In order to test the quality (timeliness and accuracy) of ATHNdataset and the feasibility of using it as a PK data repository, a 10-site real-time data entry pilot will be conducted as a sub-study.
Mentor: Ellis J. Neufeld, MD, PhD, Professor of Pediatrics, Boston Children's Hospital
Funding Period: Two years

Project Title: Characteristics Associated with Prescribed Prophylaxis of Factor Replacement
Abstract: Prophylactic factor replacement therapy is recommended for patients with severe hemophilia A and B to reduce joint disease. The characteristics of those with prescribed prophylaxis are not well described. This cross-sectional design study proposes to describe the characteristics of patients with severe hemophilia that are prescribed prophylaxis using CORE data elements of the ATHNdataset. Additionally, an exploratory aim would research neighborhood variables to identify potential social/environmental determinants of prescribed prophylaxis through geographic information systems.
Mentor: Christine Kempton, MD, MSc, Associate Professor, Department of Pediatrics and Hemophilia/Medical Oncology, Hemophilia of Georgia
Funding Period: One year

Project Title: Regional and Center-Level Variation in Utilization of Orthopedic Surgical Interventions in Persons with Hemophilia
Abstract: This study evaluates predictors of and outcomes associated with orthopedic surgical procedures in individuals with hemophilia. The study's results will help to guide future efforts to improve perioperative and long-term outcomes associated with these procedures. The aims of this study are to determine whether regional differences or HTC size affect the utilization of invasive joint surgeries and to quantify the impact of orthopedic surgery on the frequency of opioid use in men with hemophilia. Evaluation of additional outcomes that may be affected by surgery will allow us to identify other potential measures of treatment effectiveness and care quality.
Mentor: Marilyn Manco-Johnson, MD, Professor of Pediatrics, Director of University of Colorado HTC
Funding Period: One year

Project Title: Inhibitors in Non-Severe Hemophilia A Patients
Abstract: Using the ATHNdataset as a source population consisting of 3,821 (44.7%) non-severe hemophilia A (NSHA) patients (per ATHN Research Brief Report, March 31, 2015), this retrospective cohort study seeks to determine the mortality rate, cumulative inhibitor incidence, clinical presentation, and therapeutic approaches for inhibitor eradication (if any), and their resultant outcome of NSHA patients with inhibitors throughout the United States over a 35 year period (January 1, 1981 to December 31, 2015).
Mentor: Nigel Key, MB, ChB, FRCP, Director, University of North Carolina Hemophilia and Thrombosis Center
Funding Period: One year