Webinar – Chronic Nosebleeds & Future Clinical Trials


nosebleed5March 12, 2015                                      
8:00 - 9:00 pm  EDT  (7pm CT / 6pm MT / 5pm PT)
Treating Chronic Nosebleeds in HHT: Today's Care and Future Directions

Join Stanford School of Medicine Otolaryngologists, Dr. Amelia Clark and Dr. Peter Hwang, as they discuss current treatments and new research in treating HHT-related nosebleeds. Learn about a new clinical trial going on now that uses Avastin to treat bleeding- A Randomized Controlled Trial of Bevacizumab for HHT-Related Epistaxis, at Stanford Sinus Center that is co-funded by Cure HHT.




LISTEN to all previously recorded webinars, including the February webinars on (1) Brain AVMs in HHT and (2) Cardiac and Liver Manifestations in HHT.

Cardiac and Liver Manifestations in HHT

Dr. Kevin Whitehead covers various ways that the heart can be affected in HHT, including heart failure due to liver AVMs and pulmonary hypertension.
Presented By: Dr. Kevin Whitehead, Co-Director University of Utah HHT Center of Excellence

NEW Clinical Trial for HHT


Evaluation of Pazopanib on Bleeding in Subject with HHT

pazopanib pillsSince our organization was founded in 1991, Cure HHT has been at the center of the HHT community, proving many times over that we are the most trusted resource for information and advice regarding HHT. Now, we are pleased to announce our partnership with one of the most respected names in medicine - GlaxoSmithKline (GSK). We believe that collaborations like this will advance therapeutic options . To follow our Year of Action in 2014, we have made 2015 our Year of Transformation, and our association to GSK will be instrumental as we seek to alter the face of this disease.

Cure HHT is excited to reveal the initiation of a new GSK sponsored study designed to test whether bleeding episodes can be reduced in patients with chronic HHT related bleeding, with a particular focus on individuals with persistent anemia or who require iron infusions.  This study will examine the effects of a drug called Pazopanib, which partially blocks the response to various vascular growth factors, including VEGF. In addition to a baseline screening period, participants in this study will also partake in up to three months of drug therapy, as well as follow-up visits.  Details of this study are available in a public listing on clinicaltrials.gov.


  • 18 - 75 years of age
  • Details of Inclusion and Exclusion Criteria are listed on the clinicaltrials.gov page, below the section titled “Purpose”


Choosing to participate in any clinical trial is an important personal decision that should be treated with care. Talk with your doctor and family or friends before deciding to be a part of this study. To learn more, you or your doctor may contact the study research staff with the contact information provided below. To answer general questions, visit the Learn About Clinical Studies page.

U.S. GSK Clinical Trials Call Center
[email protected]

When requesting information, please refer to this study by its Clinical Trial identifier: NCT02204371

Brain AVM in HHT: Knowledge is Key to Care

Presented by Dr. Marie Faughnan, Cure HHT Scientific Research Director

Webinar covers management and treatment of brain AVMs in HHT patients as well as initial findings of NIH Brain Vascular Malformation Consortium research study. Presentation by Dr. Faughnan - 35 minutes, Q&A session - 25 minutes

2015: Year of Transformation


2015Cure HHT is the only organization in the world with the perseverance and the experience to integrate and drive vital progress across patient, medical and scientific communities. Despite our small staff and modest budget, we have demonstrated a remarkable track record of success in all three of these arenas.

Cure HHT is at the epicenter of the national and global effort to advocate for patients and families, raise awareness of HHT, catalyze and sponsor critical research, create lasting collaborations, and advance treatments.

As we approach our 25th anniversary, Cure HHT is poised to seize opportunities that can change the course of this disease in our lifetime. Our goal is to increase awareness of HHT among the public and medical community, which will lead to earlier diagnosis of those with the disease; to ensure wider availability and consistency of quality care; and to fund research that will enable scientific breakthroughs, increase our understanding of the disease, improve existing therapies, deliver new therapies, and ultimately lead to a cure.

Cure HHT Research Activities

  • Focus Groups - Cure HHT has partnered with a pharmaceutical company that believes their drug will impact bleeding in HHT patients. A third party is conducting focus group interviews in Southfield, MI, Raleigh, NC, and Boston, MA in early 2015 to learn more about nosebleeds related to HHT and fatigue.
  • Brain AVM Study - Cure HHT is now a recruiting center for qualified patients who are not affiliated with a participating HHT Center of Excellence. We will begin to actively recruit patients once we receive our IRB (Internal Review Board) approval. If you have a clinical or genetic diagnosis of HHT, are at least 3 years of age, and have a brain AVM (whether its' been treated or not) then you should contact [email protected] to let us know that you are interested in learning more about participating in this study.
  • Young Scholar Awards - Cure HHT will be awarding five (5) $30,000 research grants to Ph.D.'s who are not more than 5 years beyond completion of their post-doctoral training. This is a new program designed to attract young researchers into the field of HHT. Applications are due by January 15, 2015 and winners will be notified on March 16, 2015.
  • HHT Scientific Conference - Cure HHT will host the 11th International HHT Scientific Conference in Captiva, FL on June 11-14, 2015. This is the first time that this international event will be held in North America. We anticipate over 200 scientists and clinicians from throughout the world who specialize in HHT to attend this event. Abstract submissions are due by February 10, 2015 and early bird registration expires on April 1, 2015.
  • Peer Reviewed Medical Research Program (PRMRP) - Cure HHT was successful in obtaining vascular malformations into the Defense Appropriations bill which includes HHT as a type of vascular malformation.  This means that the HHT research community will now be able to compete for research grants from the Department of Defense’s Peer Reviewed Medical Research Program (PRMRP). We anticipate that the PRMRP will issue a program announcement on the availability of funding and grant guidelines sometime in February or March 2015.   The total amount of funding is approximately $240MM and 23 diseases, including HHT, are part of this program.
  • Outcomes Registry for HHT - Cure HHT awarded a research grant to Dartmouth's Geisel School of Medicine to develop, in collaboration with Cure HHT's Scientific Research Director, this critical tool for gathering natural history, treatment outcomes and quality of life issues from HHT patients. The Outcomes Registry for HHT will undergo Beta Testing in early 2015.
  • Scientific/Medical Publications - Since 2004, over 500 HHT related articles have been published by members of the Cure HHT Global Research and Medical Advisory Board (GRMAB). A complete publication listing is located in the Resource section of the Cure HHT website along with a list of pertinent articles by organ/HHT impact topics.


Cure HHT Increase Access to Quality Care Activities

  • Establish new Centers of Excellence in underserved areas
  • Expand the number of community physicians participating in the online HHT Physician Directory to connect patients and physicians throughout North America
  • Advance HHT Care Guidelines to reflect new information from latest clinical and translational research to ensure best practices and quality care standards


Cure HHT Awareness Activities

  • Create innovative programs and tool kits for specialists who encounter HHT
  • Provide resources to HHT Physicians to educate medical students, interns and residents
  • Collaborate with professional medical and scientific organizations to sponsor HHT speakers
  • Educate and empower patients, family members and friends to become HHT advocates through webinars and resource materials

HHT Outcomes Registry

Outcomes Image


Fig. 1  (provided by Nathan Shedroff, Geisel School of Medicine at Dartmouth / UNC Chapel Hill) illustrates how Data collection is necessary for research creation and discovery of the disease which leads to Information that is a critical stimulus to the education of consumers (patients, doctors and scientists). This Knowledge provides a better understanding of the disease which ultimately leads to the Wisdom of scientists, doctors and pharmaceutical companies that impact future research and therapeutic breakthoughs.


Over the course of the last eighteen months, Dr. Marie Faughnan, Cure HHT Scientific Research Director, has been working with collaborators at Geisel School of Medicine at Dartmouth to develop the HHT Outcomes Registry. The registry’s purpose is to collect patient data that will allow scientists to conduct natural history studies as a critical step in furthering research on HHT.  In particular, pharmaceutical companies have indicated that access to this patient data resource is a determining factor in their decision to invest financial resources in drug development for HHT.

The HHT Outcomes Registry will include de-identified (privacy protected) clinical, radiographic (X-ray, CT scan, MRI scan), genetic, and treatment information on all HHT patients in North America who are evaluated in HHT Treatment Centers of Excellence and voluntarily consent to participate. The data collected by the registry will relate to every aspect of the disease (from nasal bleeding to stroke), with emphasis on determining the outcomes (severity of symptoms, quality of life, heart failure, stroke, severe bleeding, etc. and death), the personal factors that affect these outcomes (genetics, lifestyle, age, sex, etc.) as well as therapies. This information can then be used to plan and appropriately target clinical research studies of new therapies. By linking all North American HHT Centers, the number of patients is increased twenty times over what any single HHT Center could gather and analyze which makes the Registry a powerful research resource.

We anticipate that a pilot with two HHT Centers will be conducted in Spring 2015.  The HHT Outcomes Registry’s short-term goal is to recruit 1000 HHT patients in the first year with 500 data fields per participant while the projected annual recruitment rate is 1,000 per year with an expected maximum of 80,000 patients. Funding of this registry is another example of Cure HHT’s goal to transform this disease to advance therapies that are more effective, accelerate discovery, and cure HHT.


Excerpts from July 2014 National Patient & Family Conference Presentation by Kristen Anton, Principal Investigator at BioInformatics Service Center at Geisel School of Medicine at Dartmouth

What is a research registry?

A research registry is an organized system that uses observational study methods to collect uniform data to evaluate specified outcomes for a population defined by a particular disease or condition.

Kind of data typically collected includes:

- Patient demographics
- Family history
- Risk factors, exposures
- Disease characteristics
- Disease testing
- Treatment protocols
- Sometimes: biological samples to address genetic characteristics

Why are research registries important?

Research registries provide the ability to:

- Capture standardized, quantifiable information about patients and disease
- Collect population-based data, with enough subjects to scientifically draw conclusions
- Ensure high data quality - so outcomes are reliable
- Collect longitudinal data (sometimes decades)
- Measure or monitor safety and/or effectiveness of treatments
- Springboard new scientific questions

Examples of a successful research registries:

1. Colon Cancer Family Registry (C-CFR)

- Unique resource for investigators to use in conducting studies on the genetics and molecular epidemiology of colon cancer
- The C-CFR continues to collect data from Registry participants
- Investigators collect information on the patient, the patient’s family, and incidence of cancer. Information from family members both with and         without cancer is important
- C-CFR sites span the globe
-Blood and tissue are collected to support ongoing molecular characterization of each family
- 62,353 individuals from 10,662 families
- C-CFR has generated more than 375 peer reviewed publications

 2. Infertility Family Research Registry (IFRR)

- Helping to improve the health of people and families that have faced a diagnosis of infertility or dealt with infertility treatments
- With 943 participants, the IFRR population has provided enough information to launch three research studies