HAEA in ACtion February 2019 Newsletter Driving HAE Research

The HAEA Continues to be a Driving Force for HAE Research

The HAEA will never be satisfied until every HAE patient is attack free, including members of our community who have HAE with normal C1 inhibitor. That is why we continue to aggressively pursue all types of HAE research. Our own US HAEA Angioedema Center at UCSD is actively involved in (1) clinical research aimed at upgrading HAE care in the United States, and (2) deep scientific inquiries into the molecular, genetic, and biochemical causes of HAE in a quest to find new therapeutic targets.
We are happy to report that Physician/Scientists at the US HAEA Angioedema Center recently discovered a potential breakthrough assay (blood test) that can inform the choice of optimal medicines by distinguishing the biological cause of a patient’s swelling. Moreover, the HAEA maintains close contact with key leaders in pharmaceutical companies with an interest in HAE. Our constant advocacy for the development of new and better HAE therapies is supported by our longstanding success in recruiting for clinical trials. Because continued access to and insurance coverage for HAE medicines is among our highest priorities, the HAEA is conducting ground breaking research that demonstrates and quantifies the value of HAE medicines from a health economics perspective.
Below are additional details on HAEA supported research.
US HAEA Angioedema Center at the University of California at San Diego
The HAEA founded the US HAEA Angioedema Center at the University of California at San Diego (UCSD). The Center is the only medical facility in the United States, solely dedicated to (1) expert HAE clinical care, (2) professional medical education, and (3) basic (molecular and genetic) and translational HAE research. Researchers at the US HAEA Angioedema Center recently announced the results of a potentially ground-breaking assay (blood test) that will allow physicians to definitively determine the cause of swelling in cases where the diagnosis is not clear. This test will be of particular help for HAE patients with normal C1 inhibitor to get access to the medicines they need to improve their quality of life. The HAEA is working with the University of California at San Diego to make the assay widely available in the US.
Conducting Innovative Health Economics Research that Demonstrates the Value of HAE Therapies
The first item in the publication list below describes how the HAEA’s creative, self-initiated research serves as the foundation for publications that quantify, and argue for, the substantial quality of life and economic value of HAE medicines. We are honored that a publication we have just prepared has been accepted for presentation at the annual meeting of the American Academy of Asthma, Allergy, and Immunology. These are the specialists that treat the vast majority of HAE patients in the US.
Working with Pharma to Bring New Medicines to HAE Patients
The HAEA was founded 20 years ago when there were no HAE medicines available other than anabolic steroids. In the early 2000s, none of the pharmaceutical companies were looking at HAE, so a small group of pioneering patients figured out a way to spark pharma industry interest and investment. Even though HAE patients now have access to 7 FDA approved medicines, the strategy developed so many years ago continues to be incredibly effective. Currently, two medicines are in active clinical trials with the prospect of two new companies conducting clinical trials on additional innovative HAE treatments.
US HAEA Advance HAE Scientific Registry
The Registry collects and stores biological samples as well as clinical data from over 1,200 HAE patients and family members. Scientist/Researchers are currently analyzing the Registry’s data to better understand the: Full medical history of HAE patients; symptom differences within patients in the same family; effectiveness of various HAE therapies; and subtle, but real patterns of health challenges yet to be associated with HAE.
Publications Resulting from Research Supported by the US HAEA
Below is a partial list of publications featuring US HAEA-sponsored research.
A Comprehensive Approach to Assessing the Value of Prophylactic Therapy for the Rare Disease Hereditary Angioedema Using Real World Patient Data. A. J. Castaldo, C. Jervelund, A. R. Kirk, D. Corcoran, H. B. Boysen, J. F. Long, S. C. Christiansen, B. L. Zuraw. 2019. Abstract and Poster Accepted for Presentation at Annual Meeting of American Academy of Asthma, Allergy and Immunology. Manuscript in Preparation.
Threshold-stimulated kallikrein activity distinguishes bradykinin- from histamine-mediated angioedema. Lara-Marquez ML, Christiansen SC, Riedl MA, Herschbach J, Zuraw BL. Clin Exp Allergy
US Hereditary Angioedema Association Medical Advisory Board 2013 recommendations for the management of hereditary angioedema due to C1 inhibitor deficiency. Zuraw BL, Banerji A, Bernstein JA, Busse PJ, Christiansen SC, Davis-Lorton M, Frank MM, Li HH, Lumry WR, Riedl M. J Allergy Clin Immunol Pract.
Hereditary angioedema with normal C1 inhibitor function: consensus of an international expert panel. Zuraw BL, Bork K, Binkley KE, Banerji A, Christiansen SC, Castaldo A, Kaplan A, Riedl M, Kirkpatrick C, Magerl M, Drouet C, Cicardi M. Allergy Asthma Proc.
Management of Children with Hereditary Angioedema Due to C1 Inhibitor Deficiency. Frank MM, Zuraw B, Banerji A, Bernstein JA, Craig T, Busse P, Christiansen S, Davis-Lorton M, Li HH, Lumry WR, Riedl M; US Hereditary Angioedema Association Medical Advisory Board. Journal of Pediatrics.
Hereditary angioedema from the patient's perspective: A follow-up patient survey. Banerji A, Li Y, Busse P, Riedl MA, Holtzman NS, Li HH, Davis-Lorton M, Bernstein JA, Frank M, Castaldo AJ, Long J, Zuraw B, Lumry W, Christiansen S. Allergy Asthma Proc.
Tolerability and Effectiveness of 17-α-Alkylated Androgen Therapy for Hereditary Angioedema: A Re-examination. Zuraw BL, Davis DK, Castaldo AJ, Christiansen SC. J Allergy Clin Immunol Pract.
Pediatric Hereditary Angioedema: Onset, Diagnostic Delay, and Disease Severity. Christiansen SC, Davis DK, Castaldo AJ, Zuraw BL. Clin Pediatr (Phila).
Current State of Hereditary Angioedema Management: A Patient Survey. Banerji A, Busse P, Christiansen SC, Li H, Lumry W, Davis-Lorton M, Bernstein JA, Frank M, Castaldo A, Long JF, Zuraw BL, Riedl M. Allergy Asthma Proc.
Before and After, the Impact of Available On-demand Treatment for HAE. Christiansen SC, Bygum A, Banerji A, Busse P, Li H, Lumry W, Davis-Lorton M, Bernstein JA, Frank MM, Castaldo A, Long JF, Riedl M, Zuraw BL. Allergy Asthma Proc.
Anabolic Androgen Use in the Management of Hereditary Angioedema—Not so Cheap After All. Kevin Y. Tse, Bruce L. Zuraw, Qiaoling Chen, and Sandra C. Christiansen
HAE Pathophysiology and Underlying Mechanisms. Zuraw BL, Christiansen SC. Clin Rev Allergy Immunol.  


Apply for a GRANT NOW 1

2019 US HAEA National Patient Summit Conference Grants Available

If you wish to attend the 2019 HAEA National Patient Summit – Imagine the Possibilities, but need assistance with travel and/or hotel expenses, you can apply for an HAEA Summit Conference Grant. Grants will be awarded through a lottery process. HAEA Summit Conference Grants are available for hotel accommodation, air travel and/or ground transportation. The deadline for grant applications is Thursday, February 28th. The HAEA will notify grant recipients by Friday, March 15th. If there are grants still available after this date, we will announce a second drawing. 

Register today and apply for a Summit Conference Grant here! 


YOUTH SUMMIT 2019 February

Announcing the 1st Global Summer Youth Advocacy Program: July 25-27

The HAEA is proud to announce this three-day program designed to help the HAEA’s young leaders and young adults ages 12-25 learn how to create positive and lasting change as an individual within their community. The program, which will take place a few days prior to the 2019 US HAEA National Patient Summit in Atlanta, will host kids and young adults from around the world. The Global Summer Youth Advocacy Program will include tips, tools, and real-life examples of how HAEA youth can use their voices to advocate on behalf of issues they care about, and how they can play a powerful role in creating lasting and meaningful change. Participants will earn an HAEA/HAEi Advocacy certification after the completion of the program. Make sure to register for the Summit and select the Global Summer Youth Advocacy Program (Wed-Fri ages 12-25) as a track you will attend. Conference grants are available for participants, and will be awarded through a lottery process. 

Register for the 1st Global Summer Youth Advocacy Program here! 


Pam King Scholarship Fall 2019 ART 2

Pam King HAEA Fall 2019 Scholarship Applications are Now Open

Fall 2019 Pam King HAEA Scholarship Applications open February 1st. The scholarship program provides financial support for HAE patients seeking to improve their lives through academic achievement. College scholarships are available to US citizens who are diagnosed with HAE, and are awarded to students who demonstrate the characteristics that Pam brought to the HAE community: relentless hard work, resilience, courage, and a passion for giving back to the HAE community. For more information about the Pam King HAEA Scholarship Program visit: www.haea.org

 Apply for a Scholarship here!

You can support the Pam King HAEA Scholarship Program by making a donation.
We invite you to celebrate Pam's legacy by contributing to this worthy educational cause. Your financial support will fulfill Pam's goal of empowering young patients to break free of the impediments caused by a rare, chronic disease and embrace a better future by pursuing a college degree. Scholarships are especially important for young people in our community, because families with HAE often face financial challenges caused by the many burdens that result from dealing with the condition.
Your generous scholarship contribution will help inspire, educate, and empower young HAE patients who can achieve a brighter future through academic success. Together we can help make their dreams come true.

To support the Pam King HAEA Scholarship Program click here!



Action Alert HAEA 2019 1

Reach out to your Members of the New (2019) US Congress, Share Your Personal Story, and Educate Them about Hereditary Angioedema
On January 3rd, the 435 House Representatives and 100 Senators comprising the 116th US Congress were sworn in. The new Congress has well over 100 freshman House members (taking office for the first time) and 10 new Senators. A new Congressional seating chart means the membership of key healthcare committees in Congress has also changed. There are many members of Congress who will be making crucial decisions this year about medical research, public health, drug development, and healthcare coverage and they are likely unaware of Hereditary Angioedema (HAE) and the patient experience. As the new Congress gets started, please take a few minutes to reach out to the offices of your House Representative and/or two US Senators. Now is the time to educate them about HAE, share your personal story, and ask for their assistance on key issues moving forward. The positive relationships that you establish now have the potential to benefit the entire HAE community in meaningful ways. Moreover, members of Congress (particularly new legislators) welcome constituent outreach and your efforts to personally share the issues that impact you and the HAE community.
Take Action
Here are a few easy steps for you to take action:
1) E-mail John Williamson at john@haea.org, and let him know of your interest in reaching out as an HAE advocate. John can provide you with the names of the appropriate staffers to contact and their e-mail address.
2) Compose an email to staffers that includes a brief note like the one below.
3) Attach the “Welcome to Congress” letter found in the link below with a personal note, sharing your own HAE story.
4) If you receive any feedback, please let John Williamson know. With your help, we believe these Congressional offices will become HAE legislative champions.
Dear [Health Legislative Assistant’s Name],
My name is [your name] and I am an advocate for the Hereditary Angioedema patient community from [your town]. I just wanted to introduce myself and share the very important letter attached. I hope your office will support the key issues that impact HAE patients, such as advancing medical research and improving coverage and access to HAE therapies (particularly through the charitable assistance safety net).
[you can include your HAE story here – please be brief]
Sincerely, Name & Address [they need to verify that you are a constituent]

  Download the LETTER that you need to attach to your email HERE!



Brady Club Send your Story 2 2019

Sign up for the Brady Club!
It's never too late to sign your kids up for the Brady Club and receive the Winter Activity Book. The Brady Club is a fun interactive online space for kids to learn about HAE! When you log in, visit the Submit Your Story section where your kids can submit their superhero HAE story and receive a FREE Brady Club t-shirt. 

 If you'd like your child to become a Brady Club member, to receive the Brady Club Activity Book, and more, register here!



Join the Youth Leadership Council

It doesn’t matter how old you are, you can help make a difference! It only takes one person to make a positive change in the world. Take a moment to think about what you can do to educate, advocate, and inspire the HAE community! Sign up for the HAEA Youth Leadership Council (YLC) to be connected to new opportunities that will make a difference.
- Meet other young people with HAE
- Join the HAEA at events around the country raising awareness and advocating for HAE
- Receive invites to virtual HAEA Youth Events and video chats

Register for the HAEA Youth Leadership Council here!


HAEA Insurance

The HAEA can help you navigate insurance issues
Insurance challenges can occur any time throughout the year, don’t go at it alone. The HAEA is here to help with your appeals/denials. As patients, we all go through these at times when we need a new prescription or prior authorization. Don’t let the stress get to you. Reach out to the HAEA health team and we can discuss some strategies to help lessen the stress, and help you through the process.
• Keep calm
• Open and respond to all notifications from your insurance company in a timely manner
• Communicate with your physician and keep all your appointments
• Let the pharmaceutical company know you have received a notification and that you might need to appeal
• Contact the US HAEA health team to assist with the process here (866) 798-5598.


HAEA Advocacy Webinar

Learn how you can take action by joining the Advocacy Webinar

Join us for the HAEA Advocacy Webinar with the Health and Medicine Council of Washington (HMCW) on Tuesday, February 12th. The webinar will cover the new Congress’ priorities, issues that will impact the HAE community in 2019, what can you do to defend HAE patient rights and access to healthcare, how can you protect charitable assistance programs, a template Welcome to Congress Letter for your legislators, and more.
Webinar Link: https://haea.zoom.us/j/362126962 
Phone: +1 646 558 8656 (Webinar ID: 362 126 962)
"Small deeds done are better than great deeds planned." - Peter Marshall

Click here to say you're going!



HAEA Virtual 5K 1

Announcing the 2019 hae day :-) virtual walk/run!

Get ready to get some exercise, spread awareness and fundraise for hae day :-) 2019! The HAEA is launching its 1st virtual walk/run to commemorate hae day :-) between February 28th (Rare Disease Day) and May 16th (HAE Day), 2019! Registration is free, and we will be tracking the progress of all our HAEA community participants to see how much distance was covered together to spread awareness of HAE. Funds raised from the virtual walk/run will support the HAEA’s three main programs: Pam King HAEA Scholarship Program, HAEA Compassion Fund, and HAE Research! All individuals that raise $50 or more will receive an official 2019 hae day :-) virtual walk/run Finisher Medal! Register today here, set-up your team, and start logging in your distance as of February 28th! 

REGISTER for the 2019 hae day :-) Virtual Walk/Run HERE!



HAEA Swell Fotos

Send us your swell photos and help educate others

Do you have photos of yourself during an HAE attack? The HAE maintains a library of images that depict what an attack looks like. We use these photos to educate physicians and other medical professionals about how to recognize HAE symptoms.
Please send your high-resolution photos to ianiceviel@haea.org. Then click the link below to complete the consent form, allowing us to use your photos for HAE education. 

Fill out the CONSENT FORM here!

Upcoming Events

HAEA Meet & Greet!
The HAEA is planning a Meet and Greet event in your area. This Meet and Greet is designed to offer you an overview of the great programs available through the HAEA, as well as give you an opportunity to connect with other patients in your area.
Through this program, you and your family member or caregiver can: Learn about HAEA programs and services available to you, hear from HAEA staff, ask questions, meet and gain valuable information and support from others in the HAE community.
For more information, please contact, Mike Mallory mikemallory@haea.org or (513) 858-9370. 

HAEA Meet & Greet Dallas

HAEA Meet & Greet San Francisco

The HAEA is a patient advocacy and research organization committed to actively engaging our community in a wide variety of grass roots activities that promote disease education and awareness. We provide personalized services to address the unique needs of HAE patients and their families, which include helping them secure access to and reimbursement for modern HAE medicines. Our great success in supporting clinical research has resulted in a variety of FDA-approved therapeutic options. We work closely with expert physicians to continuously upgrade patient quality of life through improving diagnosis and knowledge of the disease, and encouraging a tailored, patient-focused use of available therapeutic options. The HAEA is product neutral and continues to enthusiastically support drug discovery research aimed at the next generation of HAE therapies.

The US Hereditary Angioedema Association
10560 Main Street , Suite PS40
Fairfax City, VA 22030


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