Coagulation Disorders (Hemophilia) Research

• Universal Data and Serum Specimen Collection System for Hemophilia (UDC)
  The hemophilia treatment center (HTC) at Children’s National Medical Center is working with the Centers for Disease Control and Prevention (CDC) to monitor the health status of people with hemophilia and other bleeding disorders. This project involves collecting some information about health status from the medical record during the annual comprehensive exam, measuring how well your joints work and asking some questions about how the complications of hemophilia affect activities of living, such as working or going to school. The information from this project will be used to help HTC plan medical care and help the CDC develop and evaluate programs to reduce or prevent the complications of hemophilia.
  
  
• Epidemiology and Treatment of Circulating Anticoagulants in Patients with Hemophilia and von Wilebrand’s Disease (HTRS Registry)
  The Hemophilia & Thrombosis Research Society (HTRS) has established a national patient registry through which a common set of data are collected from Hemophilia Treatment Centers (HTCs) to gain a better understanding of the pathophysiology of hemophilia and other coagulation disorders; the current clinical management of patients with these disorders; and the safety and efficacy of alternative treatments.
  
  
• An Open-Label Study to Evaluate the Efficacy and Safety of XYNTHA in Children Younger Than 6 Years-of-Age in Usual Care Settings (Wyeth 3082B2-3315)
  The study's purpose is to collect additional information for regulatory authorities, clinicians, and researchers about the safety of Xyntha, and to collect information on how well Xyntha works in treating bleeding episodes in children under 6 years-of-age. The design of the study allows your study doctor to treat your child’s hemophilia with Xyntha similar to how he or she would usually treat it if your child was not in a study.
  
  
• A Postauthorization Safety Surveillance Study of XYNTHA in Usual Care Setting (Older Than 12 Years-of-Age) (Wyeth 3082B2-4418)
  The purpose of this study is to collect information that may provide regulatory authorities, clinicians, and researchers additional safety information about the use of Xyntha. The study's design allows your study doctor to treat your child’s hemophilia with Xyntha similar to how he would usually treat it if your child were not in the study.
  
  
• An International, Randomized, Controlled Trial of Immune-Tolerance Induction
  The International Immune Tolerance Study is a study that will compare 50 units per kilogram of factor VIII infused three times a week with 200 units per kilogram of factor VIII infused daily for up to 33 months in order to induce immune tolerance. After immune tolerance is achieved, children will be closely followed for an additional 12 months. Physicians at Children’s National hope to learn whether the two regimens of immune tolerance induction are equally effective or whether one regimen is superior to the other.

• Severe Chronic Neutropenia (SCN) International Registry (SCNIR)
  The SCN Disease Registry is a method to collect information about the health of persons with severe chronic neutropenia over time. The purpose of the study is to learn more about severe chronic neutropenia and assess the long-term safety of primary treatments; provide a research base for establishing better treatments; and minimize side effects of existing therapies for severe chronic neutropenia.

• Idiopathic Thrombocytopenic Purpura (ITP) Splenectomy Registry
  The Splenectomy study is an international registry of children and adults with ITP. The goal is to study patients with chronic ITP, lasting more than 6 months, who are having their spleen removed for treatment. In the splenectomy study, clinical information about patients having their spleen removed for ITP will be anonymously collected and evaluated in order to learn more about: reasons for splenectomy; timing of splenectomy; safety of splenectomy, including infections that occur afterwards; techniques used for performing splenectomy; and response rate of splenectomy.
  
• Pediatric and Adult Intercontinental Registry of Chronic ITP (PARC-ITP)
  The PARC ITP study is an international registry, or database, of children and adults with ITP. The goal is to study participants with chronic ITP, lasting more than 6 months. However, since we do not know initially which participants will develop chronic ITP, we are asking all patients with ITP to enroll in the registry. In the PARC-ITP Study clinical information of participants with chronic ITP will be anonymously collected and evaluated in order to learn more about the natural history and management of this disorder and to collect data that is necessary for the planning of future studies.

• Prospective Multi-Center Evaluation of the Duration of Therapy for Thrombosis in Children (Kids-DOTT)
  The aims for this study are the following: Evaluate the safety and efficacy of short-duration (6 weeks) versus standard-duration (three months) anticoagulation in patients whose clot has cleared after six weeks of medication; and to determine whether there is a difference in recurrent clots or post-thrombotic syndrome between patients whose clot resolves and those that do not resolve within 6 weeks.

• Michael F. Guerrera, MD- Hematologist
• Christine Guelcher, PNP- Nurse Practitioner
• Heather Wisnieski, BSN, RN- Hematology Nurse Coordinator
• Melissa Davis, PT, MS- Physical Therapist
• Liz Hardesty, LGSW- Social Worker
• Amanda Kasper, MPH- Clinical Research Coordinator
• Naomi Luban MD, Laboratory Medicine/Hematology
• Lori Luchtman-Jones MD, Hematology