Press Release: Launch of Multi-State Pilot Program

 

 

Cure HHT to Present Congressman David E. Price with its

"Champion Award" at launch of Multi-State Pilot Program

Headquartered at UNC Chapel Hill

 

Cure HHT has been partnering with the Center for Disease Control and Prevention (CDC) for over a decade.  Through this relationship and ongoing meetings, the HTC / HHT concept was born.  Cure HHT's advocacy efforts, along with the support of Congressman David Price, resulted in federal funding for a two year, multi-state pilot program involving three Hemophilia Treatment Centers (HTC) in underserved areas of the country. 

 

Chapel Hill, NC - On Thursday, August 24 at 5:00pm at the Carolina Club at UNC, Chapel Hill, Cure HHT, the national advocacy organization for people with the rare hereditary disease HHT, will present its' “Champion Award” to Congressman David E. Price (D-NC, 4th District) for his leadership in securing funding of a new HHT multi-state pilot initiative.  Prior to the event, the Congressman along with Cure HHT Executive Director Marianne Clancy and UNC Chapel Hill HHT Center Director Dr. Raj Kasthuri will tour the UNC HHT Clinic and meet some of the patients.

Cure HHT recently celebrated 25 years of building awareness of HHT, educating the public and medical professionals, leveraging funding for research, and advocating for patients and families affected by the disease.  In order to provide expert, multidisciplinary care for people with HHT, Cure HHT has helped establish 25 Centers of Excellence in North America over the last 2 decades.

While Cure HHT will continue to build the number of its Centers of Excellence, working with Dr. Kasthuri it proposed a pilot program where three Hemophilia Treatment Centers would receive guidance and training to allow them to serve HHT patients as well. If successful, this could potentially expand access to care significantly for HHT patients, especially in areas where no HHT Center of Excellence exists.

All that was needed was the funding to make the pilot program a reality.  Enter Congressman David Price who made securing federal funding for the pilot program one of his three top legislative priorities.   “I was inspired by the leadership of the team at UNC and Cure HHT in their efforts to leverage existing infrastructure to bring comprehensive care to an underserved community of patients,” said Rep. Price. “I’m optimistic that a successful pilot program will serve as a model for communities across the country to bring awareness and treatment to those affected by this disease.”

In many ways the treatment and management of HHT can be compared to treatment for hemophilia, the hereditary condition where the blood fails to coagulate.  Due to the efforts of The National Hemophilia Foundation, created in 1948, there are currently more than 130 federally funded Hemophilia Treatment Centers in the U.S.  Many of these have teams of doctors and specialists such as hematologists, genetics counselors and nurse coordinators.

“Unfortunately our current Centers of Excellence can’t reach everyone affected by the disease,” comments Cure HHT’s Executive Director Marianne Clancy. “There is so much more work to be done on this disease.  Due to lack of awareness by both the public and medical providers, it can take as long as 30 years for someone to be properly diagnosed.  Only ten percent of the people who have HHT are aware of it.”

 

Cure HHT is excited to begin this new endeavor. By partnering with the CDC, Congressman Price, three Hemophilia Treatment Centers and UNC Chapel Hill HHT Center, the possibility of providing knowledgeable care to HHT patients in all fifty states becomes attainable. Thank you to all constituents who have supported Cure HHT's efforts over the last twenty years. Without you, this project and future projects would not be possible.

 

NOSE Study Results Are In!

 

PUBLICATION: Journal of American Medical Association; JAMA. 2016;316(9):943-951. doi:10.1001/jama.2016.11724

 

We are very pleased to report the first multi-center clinical study on nosebleed treatment has been published and released in the Journal of American Medical Association: “Effect of Topical Intranasal Therapy on Epistaxis Frequency in Patients with Hereditary Hemorrhagic Telangiectasia a Randomized Clinical Trial” on September 6, 2016.

This is the first randomized double blind placebo controlled Phase II Clinical trial ever documented! This study has propelled other studies forward, such as the Avastin Epistaxis study at Stanford University and the Paznopanib trial with Glaxo Smith Kline Pharmaceutical Company.

Results

120 patients received topical therapy of either Avastin 1% (4mg/d), Estriol 0.1% (0.4mg/d), Tranexamic acid 10% (40mg/d) or placebo (0.9% saline) for 12 weeks to assess the risks and benefits for treatment of moderate to severe nose bleeding. As you know all too well, interventional techniques such as laser and cautery have provided temporary help, but the need for better therapeutic approaches in managing nosebleeds is desperately needed.

The study showed treatment with topical therapy is safe, well tolerated and improves nosebleed severity in most patients. Though the study didn’t show any impact on nosebleed frequency, patients on Avastin showed a borderline reduction in nosebleed duration in comparison with the tranexamic acid and placebo over the 24 weeks.

Variables

As HHT patients do not routinely irrigate the nose prior to the administration of drug therapies (which might improve absorption of medication), we do not know if this procedure impacted the results. Additionally, it may be that a higher concentration of the Avastin (bevacizamab) could have produced greater improvement.

Conclusion

This study concluded simple treatment with nasal saline spray twice daily is beneficial to many patients. The addition of tranexamic acid, estriol or bevacizumab does not seem to offer significant advantage over plain saline. However, if you are currently using one of these medicated sprays, do not discontinue before speaking with your doctor.

GOSSAGE SIGNATURE FOR LETTER

 

 

James Gossage

Medical Director

Marianne Clancy signature

 

 

Marianne Clancy

Executive Director

 

Cure HHT Launches New York Center

New Center Hospital Photo

24th HHT Center of Excellence in North America

NewYork Presbyterian/Columbia University Medical Center

New York, NY

“Two years ago, we made a commitment to New York and New Jersey families that we would work very hard to encourage a team of experienced physicians, with our guidance and a HHT mentor center, to come together as an HHT Center of Excellence,” said Executive Director Marianne Clancy. “I am so happy to see this work become a reality. I am confident this superb team, which I met during a site visit, will provide outstanding care. We look forward to working closely together in the years to come."

nyc

NewYork Presbyterian/Columbia University Medical Center is the only facility in New York that specializes in the diagnosis, evaluation and treatment of HHT. The newest HHT Center offers compassionate, family-centered multidisciplinary care, led by Dr. Sophie Chheang, a dynamic and action-oriented physician. At the NewYork Presbyterian/Columbia University HHT Center, each patient is treated by HHT-knowledgeable physicians who are compassionate and devoted to maintaining the highest standards of care. Dr. Chheang’s team includes a full team of pediatric specialists lead by Dr. Emilio Arteaga-Solis.

Chheang, Sophie MDDr.Chheang is a board certified radiologist at NewYork Presbyterian and an assistant professor of radiology at Columbia University Medical Center. After graduating from the New York University School of Medicine, she completed her Diagnostic Radiology residency at the University of California, Los Angeles. Dr. Chheang then finished her Interventional Radiology fellowship at NewYork-Presbyterian/Weill Cornell Medical Center,which included additional training at the Memorial Sloan Kettering Cancer Center.In addition to English, Dr.Chheang speaks Khmer and Spanish. She is Board certified with the American Board of Radiology.

 

Specialties at NewYork Presbyterian/Columbia University HHT Center

Cardiology

Dermatology

Genetics/Genetic Counseling

Gastroenterology

Hematology

Interventional Radiology

Neuro-interventional Surgery

Neurosurgery

Otolaryngology (ENT)

Pediatrics

Pulmonology

 

To Make an Appointment

Adult Appointment: Contact Helen Pena-Chacon at 212-305-7094

Pediatric Appointment: Contact Emilio Arteaga-Solis, MD, PhD at 212-305-5122

Cure HHT Launches 23rd Center

mass gen23rd HHT Center of Excellence in North America

Massachusetts General Hospital

Boston, MA

 

 

mas gen notesCure HHT has brought Massachusetts General Hospital (MGH) together with Mass General Hospital for Children and Massachusetts Eye & Ear Infirmary to form the only facility in the state specializing in the diagnosis, evaluation and treatment of HHT.

This collaboration allows them to provide specialized care across a patient’s lifespan. The MGH HHT Center offers compassionate, family-centered multidisciplinary care, led by Dr. Josanna Rodriguez-Lopez, coordinating care for adult and pediatric patients. She is joined by an outstanding group of physicians, including Dr. Raymond Liu as the Associate Director, who share her dedication for the multidisciplinary treatment of this disease.

Rodriguez-Lopez, Josanna MGH

 

Dr. Rodriguez-Lopez has dedicated her career to treating patients with pulmonary vascular disease. As the Associate Director of the Pulmonary Hypertension and Thromboendarterectomy Program at MGH, she specializes in all aspects of pulmonary vascular disease, with a particular interest in pulmonary arteriovenous malformations. She has several years of experience managing HHT and is looking forward to growing the HHT program as well as developing a strong partnership with Cure HHT and the HHT community.

 

Specialties at MGH HHT Center  (*adult and pediatric services)

Cardiology*

Dermatology

Dentist/Oral Surgeon

Genetics/Counseling

Gastroenterology*

Hematology*

Interventional Radiology

Neuro-interventional Surgery

Neurology/Stroke

Neurosurgery*

OB/GYN

Otolaryngology (ENT)*

Primary Care (PCP)

Pulmonology*

Radiology

Surgery*

 

MGH_Mass General Childrens Hospital

MGH_Harvard Logo

MGH_Mass Eye and Ear

MGH_Hospital

 

 

 

Let’s Get Personal – HHT in Women

Presenter

Dr. Els Maria de Gussem is an HHT clinician in the Respirology Division at Grace Hospital, University of Manitoba in Canada. She has dedicated her career to developing an expertise in HHT, both in clinical care and research. Dr. de Gussem has performed a study on the influence of pregnancy in HHT along with studies on quality of life and life expectancy in HHT. She received the Robert I. White Young Clinician Award in 2009 for her clinical compassion and dedication to improving HHT care.

Webinar

Join HHT Physician Dr. de Gussem for an in-depth look at the effects of HHT on the changing female body. She will discuss the impact of birth control, pregnancy and menopause as well as recommended management or treatment options for women with HHT.

Webinar Highlights

  • Hormonal changes in women through puberty, pregnancy and menopause.
  • Pregnancy complications, prevention of complications, and recommended management during pregnancy in women with HHT
  • Birth control in women with HHT
  • Post-menopausal management of HHT-related symptoms and complications
  • HHT and Pregnancy handout and Website links provided in the webinar

ACT NOW

  • SHARE this information and webinar link with your family members affected by HHT

Cure HHT

Cure HHT is the cornerstone of the HHT community, advancing one common hope - to give those affected by HHT a chance for a normal life. This webinar series is just one of many ways we accomplish this mission. If you found value in this information, please consider making a donation NOW to keep this initiative moving forward. It is through the generosity of our donors that we can continue to provide this valuable education to the HHT community.

 Donate Now Button - Blue Rectangle

 (Please write "webinar" in comment section of donation form)

Lay Down the Law with Cure HHT

January 13, 2016
Presented by: Mark Vieth, Senior Vice President, CRD Associates and Marianne Clancy, Executive Director, Cure HHT

Presenter

Mark Vieth is a Senior Vice President for CRD Associates, a government relations, public policy and strategic development firm in Washington, DC.  Mark has served as the Legislative Advisor for Cure HHT for the last six years. He has helped clients secure federal funding, build new partnerships with federal agencies and mobilize grassroots to advocate for legislation pending in Congress.

Marianne Clancy became the first Executive Director of Cure HHT in 2001. Prior to that, Marianne served on the HHT Foundation board of directors, founded a health care consulting company specializing in trans-dermal drug delivery and oral health care, worked in national health care management with Oral-B Laboratories, a Gillette Company for 14 years, and held faculty positions at Forsyth Dental Center, Boston University and Tufts University School of Dental Medicine.

 

Webinar

Join Cure HHT Executive Director Marianne Clancy and Legislative Advisor Mark Vieth for a behind-the-scenes look at the HHT DATA Act. Learn what passage of the act would mean for you, what progress has been made since the act was introduced this past April, the most recent push for senate sponsorship and what you can do to lay down the Cure HHT law.

Webinar Highlights

  • Congress 101 - who are your Members of Congress, when are they in session in DC and their home offices, how are their Congressional offices staffed?
  • HHT Advocacy Initiatives
  • HHT Diagnosis and Treatment Act (H.R.1849) - why is it needed, what does it do, what will happen when it's enacted, what is the status of the bill, and how can I help?
  • HHT Legislative Fact Sheet and Website links provided in the webinar

ACT NOW

Cure HHT

Cure HHT is the cornerstone of the HHT community, advancing one common hope - to give those affected by HHT a chance for a normal life. This webinar series is just one of many ways we accomplish this mission. If you found value in this information, please consider making a donation NOW to keep this initiative moving forward. It is through the generosity of our donors that we can continue to provide this valuable education to the HHT community.

 Donate Now Button - Blue Rectangle

 (Please write "webinar" in comment section of donation form)

Post Traumatic Stress and Depression in Adults with HHT

surveyDr. Shruti Chaturved at Vanderbilt University Medical Center is conducting a research survey to investigate the symptoms of post traumatic stress disorder and depression in individuals with HHT.

Objective: To determine if individuals living with HHT experience symptoms of post-traumatic stress and depression because of their condition.

Why Participate: People who have experienced a stressful event such as serious injury, threatened death or serious illness often have problems with anxiety, depression and post-traumatic stress disorder. Your involvement in the study will help us understand the psychological impact of living with HHT and identify resources to assist in improving quality of life for HHT patients.

EligibilityIf you have an HHT diagnosis you are eligible to participate in this research study. No other qualifications are necessary. The survey will take 10-15 minutes to complete.

Get Started:

Open the survey in your web browser by clicking the link below:
Post traumatic stress and depression in adults with Hereditary Hemorrhagic Telangiectasia

If the link above does not work, copy the link below into your web browser:
https://redcap.vanderbilt.edu/surveys/?s=TYNE7YMYMF

Privacy: All responses will be anonymous and no identifying information will be collected. The data will be kept completely confidential and stored in a secure electronic database at Vanderbilt University. Only the investigators will have access to the database and information will not be shared with any external agencies.  The results of this study may be published, however, individual information will not be disclosed.

vumc08-4c-blk873

Recruiting Patients at Cleveland Clinic for Trial

 

Cleveland Clinic LogoPrincipal Investigator: Keith McCrae, MD

This study will be conducted at Cleveland Clinic Main Campus and Regional Hospital Facilities. This study will evaluate patients with GI bleeding due to HHT or with HHT-related nosebleeds that require ongoing transfusion or iron replacement therapy. Patients with GI bleeding must have a requirement for at least 4 units of blood or 1 gm of iron infusion per 4 month period, while patients with nosebleeds must require 2 units of blood or 500 mg of iron per 4 month period. The effect of Pomalidomide on bleeding will be assessed during an initial 4-5 month period in which the dose of Pomalidomide is increased monthly, as needed. Patients will then be observed over a 4 month period on a dose of Pomalidomide found to be effective, then over a dose tapering period and for 6 months after stopping the drug.

Study Question: Will Pomalidomide therapy reduce bleeding and the requirement for ongoing transfusion or iron therapy  in patients with HHT by 50% or greater?

Study Goals:

  1. To determine whether the use of Pomalidomide will reduce the requirement for transfusion and intravenous iron administration for patients with chronic gastrointestinal bleeding or nosebleeds due to HHT
  2. To monitor possible bleeding episodes in patients 6 months after discontinuation of Pomalidomide therapy.

You may qualify to participate if you:

  • Are 18 years of age or older with an HHT diagnosis
  • Have GI Bleeding OR Nosbleeds related to HHT
    • GI Bleeding MUST require at least 4 units of blood or 1gm of iron infusion per 4 month period
    • Nosebleeds MUST require 2 units of blood or 500mg of iron per 4 month period
  • Platelet count >125,000
  • Do not have any conditions listed in "Who Cannot Participate" section below

Who Cannot Participate:

  • Pregnant women
  • Prior treatment with Avastin within the past 6 weeks
  • Myelodysplasia
  • Requirement for chronic aspirin, NSAID therapy or anticoagulation therapy
  • Renal insufficiency
  • Currently enrolled in other interventional trials
  • Hemophilia, advanced liver disease, or known effect in the coagulation system (not including HHT itself)

Study Procedure: 

  • Screening period including labs and signing of informed consent.
  • Oral administration of 1 mg/day of Pomalidomide, increasing to a maximum of 5 mg/day at the physician’s discretion on a monthly basis.
  • Continuation of stable dose of Pomalidomide for 4 months.
  • Gradual reduction of Pomalidomide dose between 1-5 months until discontinuation.
  • Monitoring for 6 months after discontinuation of Pomalidomide.

What is required of me?

  • Travel to the Cleveland Clinic Main Campus approximately once each month
  • Blood tests at a doctor’s office or hospital near you two weeks after each Cleveland Clinic visit
  • There will be compensation for parking on the days when coming to the main campus is required

To participate in this trial, contact the Principal Investigator:  

Keith McCrae, MD
9500 Euclid Avenue, R35
Cleveland OH 44195
(216) 445-7809

2015 MIDWEST REGIONAL CONFERENCE

Register Now

sheraton milwaukee brookfield hotelSaturday, September 19, 2015
Brookfield, Wisconsin

This one-day conference is sponsored by Cure HHT and hosted by the medical team at the Froedtert & Medical College of Wisconsin (MCW) HHT Treatment Center of Excellence. You will benefit from learning how HHT impacts each organ over time and the most current treatment options. This regional conference will highlight several new "hot topics" - IV Bevacizumab and Pediatric treatments - as well as a Patient Panel that will discuss "Living with HHT and Utilizing Your HHT Center". No matter where you live or how long you've known that you've had HHT, you should join us in Wisconsin.

Sheraton Milwaukee Brookfield Hotel
375 S. Moorland Road
Brookfield, WI  53005

Room Reservations:  If you would like to spend the night in Brookfield, WI, we have reserved a few discounted rooms ($99/night plus tax) Just call the hotel at 262-364-1100 and mention the Group Code "HHT Conference".

Conference Program:    VIEW Program Schedule

Learn from the Experts . . . We are thrilled to acknowledge our distinguished speakers from Froedtert & MCW:

Dr. Robert Krippendorf
Ms. Yi-Lee Ting, MS, CGC
Dr. William Rilling
Dr. Patricia Burrows
Dr. Patrick Foy
Dr. David Poetker

Not only will you receive the most current information on HHT research, diagnosis, management and treatment but you'll be able to talk one-on-one with leaders in the field of HHT, network with individuals and families affected by HHT and participate in small group discussions.

Registration: $125 per person includes all education materials and meals served during the conference. Registration does not include hotel reservations. There is a discounted fee for Froedtert & MCW staff; contact Zachary Timm (Froedtert & MCW) or Nicole Schaefer (Cure HHT) for the registration discount code.

Froedtert & MCW HHT Center:  Visit their website to learn more about the Froedtert & MCW HHT team, hospital, diagnostics, treatments, and much more.

2015 HHT DATA Act Updates

The HHT Diagnoses and Treatment Act

Laying Down the HHT Law

The HHT Diagnosis and Treatment Act HR 1849 was introduced in the House of Representatives on April 16, 2015 as part of the 114th Congress.

The bill would be the first to create a federally led and financed initiative for early diagnosis and appropriate treatment of hereditary hemorrhagic telangiectasia.

The HHT DATA Act would require the Centers for Disease Control and Prevention to conduct surveillance initiatives, start an internal HHT resource center and conduct public awareness programs.

The act would also authorize the appropriation of $5 million per year for the next five years to be used to improve early detection, screening, diagnosis and treatment of HHT.

Our goal is to get as many legislators as possible to co-sponsor this bill! It's time to lay down the law for Cure HHT. 

Click to get the HHT DATA Act passed by the end of the year!

U.S. House of Representatives

H.R. 1849
The HHT Diagnosis and Treatment Act of 2015 was introduced by U.S. Representative Ed Royce of California and U.S. Representative Jim Himes of Connecticut on April 16, 2015.

Co-Sponsors: 2/100

House of Representatives Sponsors:

Ed Royce (R – CA)
Jim Himes (D – CT)

House of Representatives Co-Sponsors:

Rosa DeLauro (D-CT)
Zoe Lofgren (D-CA)
Eleanor Norton (D-DC)

Find your Representative

Senate

We are actively working on finding a senate champion for the HHT DATA cause!

Do you think your representative is right for the job? Let us know at 410-357-9932 or [email protected]

Click to Find your Senator