Spring 2010

A Message From Your US HAEA President

Dear HAEA friends 

Your HAE Association continues to find ways to live up to one of our primary and longstanding principles–patients helping patients lead a normal life. This important work has gotten a bit easier because there are now 3 new FDA approved products available for treating HAE, and two more new medicines are likely to be available in the very near future. We should all be very proud that HAE patients throughout the country selflessly took the time to participate in the clinical trials that convinced FDA to approve these important new medicines. 

The HAEA continues to be totally committed to providing personalized service and ensuring that every HAE friend has enough information to work with their doctor on a treatment plan that relegates HAE to a mere nuisance instead of the painful, debilitating condition that many of us have faced for far too long. Keep in mind that the HAEA’s Patient Services team works around the clock to provide caring and individualized assistance to every HAEA friend who has a need. We approach our day to day work with empathy because each Patient Services staff member either suffers from HAE or serves as the primary care giver for a severely affected family member. Your HAE Patient Services Representative is ready to help you (1) with an HAE-related emergency, (2) answer questions about insurance reimbursement for current HAE medications, and (3) by providing information on current clinical trials for promising HAE medicines that will be available in the future. Please check the Patient Services list below to find the phone number for your Patient Services Representative and give him or her a call today. 

So far, 2010 has been a very busy year! A major initiative has involved planning Regional HAE Patient meetings around the country. Regional events provide more time for HAE friends to connect with fellow patients, ask questions about available medicines, and interact with world class HAE physician/researchers. We will be conducting six Regional meetings in 2010, and two have already been held–Washington, DC and Cincinnati (a synopsis of these two meetings is provided below). 

Please visit the HAEA website, www.haea.org often because it is your ONLY source for authoritative, unbiased information on all things HAE. The website will soon have a fresh look, additional features, and be easier to navigate. By the way, if you have not signed up to be a member of the HAEA, please do so and make sure all of your family members also join! There is strength in numbers and a growing HAEA will ensure that stakeholders such as the government and pharmaceutical industry listen and respond. 

I want to stress the US HAEA’s commitment to research. Our HAE Patient Scientific Registry and the data it generates are the keys that researchers will use to unlock HAE’s remaining secrets and move us towards better therapies and a cure. So far, over 250 people have signed up for the Scientific Registry at our national and regional meetings. You can sign up for the Registry and donate blood and DNA samples conveniently and at no cost right at a Regional meeting site (yet another great reason to attend one of the four remaining meetings). Get more information by going to www.haea.org and clicking on the Registry icon in the middle of the page. You will make a difference for yourself and future generations by participating in the US HAEA Scientific Registry. 

Finally, we are always moving forward to better serve each and every HAE friend. In addition to the Regional meetings and website upgrade mentioned above, our 2010 project list includes physician educational outreach programs, and new patient brochures specific to the needs expressed by our patient community. Your US HAEA is here for you so do not hesitate to call a Patient Services Representative. I hope to see each of you soon at one of our regional meetings. 

Warmest Regards to All! 

Tony Castaldo
President, US HAEA
President, HAE International
2010 Regional Patient Meetings – Coming to a Location near YOU!

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Following up on the great success of our October 2009 National Patient Conference, the US HAEA is conducting 6 Regional Patient Meetings in 2010. 

These meetings will provide an opportunity for our HAEA friends to gather in a smaller and more intimate setting. All six will provide the chance to make new HAE friends, develop lasting connections with other patients in your geographical area, as well as be provided with access to the top HAE physician experts in the US. 

US HAEA Regional Patient Meetings 2010
Jan 23 Metro DC area
Apr 17 Cincinnati, OH
June 12 Portland, OR
Sept 11 Dallas, TX
Oct 2 New York City
Nov 6 CA (city TBD) 

Registration is now open for the June 12 meeting- Sign up online at www.haea.org today ! 

Please contact an HAE Association Patient Services Representative (see list below) for more information about any of these meetings. 

We hope to see each of you, our HAEA friends, ato one of our Regional Patient Meetings in 2010! 

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US HAEA 2010 Regional Patient Meetings – Bethesda, MD and Cincinnati, OH – Highlights 

Over 100 HAEA friends arrived in Bethesda, MD on January 23, 2010 to attend the first of our US HAEA Regional Patient Meetings scheduled for 2010. And over 80 HAEA friends arrived in Cincinnati, OH on April 17 for our second Regional Patient Meeting. 
Below are highlights from both meetings: 

Opening Remarks by HAEA President Anthony Castaldo
Tony opened up both conferences. 

Tony said, “The HAE Association is steadfast in demanding that physicians and insurance companies recognize that HAE patients have a fundamental right to choose a treatment program that best fits their individual needs. We now have a variety of products from which to choose, and we are optimistic that additional HAE treatments are going to be approved. The HAEA’s Patient Services group is dedicated to continuing and expanding its efforts to provide each and every HAE friend and their physicians with all of the information necessary to establish a treatment program to control and hopefully eliminate HAE attacks.” 

Tony reiterated that the HAEA is a patient services and research organization run by patients. He went on to say, “Our day-to-day interactions with HAE friends provides a chance for us to demonstrate the HAEA’s “culture” of caring and kindness, and absolute devotion to doing whatever humanly possible to help every HAE friend lead a normal life. 

Bethesda Keynote Presentation – Dr. Henry Li and Dr. Marc Riedl 

The meeting’s keynote presentation was shared by two of our country’s most renown HAE experts – Dr. Henry Li, Professor of Medicine in Chevy Chase, MD and Dr. Marc Riedl, Assistant Professor of Medicine at UCLA. 

Dr. Li presented a fascinating history of HAE as well as a discussion of the disease itself in all of its variability. One other point of note was the great increase in scientific papers on HAE, increasing by more than 400% over the past 60 years and a 200% jump in just the last decade. Dr. Li concluded with comments on optimal mangement of HAE – including early diagnosis and stopping attacks from interfering with one’s social and personal life. 

Dr. Marc Riedl’s presentation covered HAE therapies -approaches to treatment, conventional therapies, and new options. Dr. Riedl noted the historical use of androgens (steroids), discussing the questions of their efficacy and their accompanying side effects. All 5 new HAE mediations (3 FDA-approved and 2 seeking FDA approval) were also discussed and compared. The presentation closed with thoughts on a new treament paradigm for HAE – - individualized treatment plans – -and on using new therapies to one day treat other forms of andioedema. 

Cincinnati Keynote Presentation – Dr. Jonathan Bernstein and Dr. Paula Busse 

The meeting’s keynote address was shared by two of our nation’s most respected HAE experts, Dr. Jonathan Bernstein, University of Cincinnati Medical Center and Dr. Paula Busse, Mount Sinai School of Medicine, NYC. 

Dr. Busse presented a look at HAE diagnosis and management. Her talk reviewed the clinical symptoms and causes of HAE as well as covered issues of diagnosis, and past and current treatments. She concluded that while older therapies may work, they are associated with often severe side-effects. She encouraged all patients to be sure to put an emergency plan in place, whether currently on preventive or on-demand therapy for their HAE attacks. 

Dr. Bernstein presented a look at current and future treatments for HAE. His talk touched on the social and economic burden of the disease. He discussed thoroughtly each and every currently available HAE treatment option as well as those excpected to be used in the future. Dr. Bernstein encouraged patients to continue to support clinical trials for new HAE medications and the US HAEA Scientific Registry – each effort continues to make the future even brighter for all HAE patients. 

Representatives from each of the five drug companies provided updates on the status of their US clinical programs in Bethesda and Cincinnnati 

ViroPharma’s (Cinryze brand of C1 inhibitor) was approved last year by the FDA as an attack prevention therapy. ViroPharma has put together an infrastructure, Cinryze Solutions, to make their product available to every HAE friend that could benefit from the medicine. The HAE Association is ready to (1) provide you with a direct referral into the Cinryze Solutions program, and (2) act as your advocate and confidant and help you work with your doctor, the specialty pharmacy who will ship your medicine, drug company representatives, and insurance providers. Please contact the HAEA’s Patient Services group for more information– Donna Davis donna-davis@haea.org or Michelle Williamson michellewilliamson@haea.org. 

CSL Behring (Berinert brand of C1 inhibitor) has received FDA approval for their C1 inhibitor infusion for treating acute HAE attacks (attacks that have already begun) on October 12, 2009. CSL has put together an infrastructure, B.E.N., to make their product available to every HAE friend that could benefit from the medicine. The HAE Association is ready to (1) provide you with a direct referral into the B.E.N. program, and (2) act as your advocate and confidant and help you work with your doctor, the specialty pharmacy who will ship your medicine, drug company representatives, and insurance providers. Please contact the HAEA’s Patient Services group for more information– Donna Davis donna-davis@haea.org or Michelle Williamson michellewilliamson@haea.org. 

Dyax (Kalbitor) Kalbitor is a recombinant product that inhibits kallikrein – an enzyme that plays a key role in the biochemical processes that lead to HAE-related swelling. The product received FDA approval in early December 2009. The HAE Association is ready (1) provide you with a direct referral into the Kalbitor Access program, and (2) act as your advocate and confidant and help you work with your doctor, the specialty pharmacy who will ship your medicine, drug company representatives, and insurance providers. Please contact the HAEA’s Patient Services group for more information– Donna Davis donna-davis@haea.org or Michelle Williamson michellewilliamson@haea.org. 

Shire (Firazyr) Firazyr is an acute attack therapy that is administered by a subcutaneous injection (under the skin injection similar to how diabetics take insulin). The product has already been approved in Europe, and Shire is currently conducting a clinical trial in the US. If you would like more information about this product or about participating in this clinical trial, please contact the HAEA’s Patient Services group for more information– Donna Davis donna-davis@haea.org
or Michelle Williamson michellewilliamson@haea.org. 

Pharming NV (Rhucin brand of recombinant C1 inhibitor) cited successful clinical trial results for their product, Rhucin – a recombinant human C1 inhibitor protein that is derived from the milk of genetically altered rabbits. Clinical trials have shown that Rhucin is a safe and effective treatment for acute HAE attacks. The company is still in the process of applying for a US license for their product. If you would like more information about this product, please contact the HAEA’s Patient Services group for more information– Donna Davis donna-davis@haea.org
or Michelle Williamson michellewilliamson@haea.org. 

US HAE Association Scientific Registry
Vice President Janet Long presented a brief overview of the US HAEA’s Scientific Registry. Dr. Bruce Zuraw and the HAEA, working together, have designed a state-of-the-art patient registry that will produce high quality “scientific grade” data. The Registry was launched on August 12, 2009 – to promote new research in HAE, to study HAE genetics, and to increase understanding of the disease – ultimately finding a cure! Meeting attendees were given the opportunity to join the Scientific Registry right at the Hotel – by the end of the day, 76 new members had signed up in Bethesda and 27 more signed up in Cincinnati. All had their blood drawn and submitted DNA samples right on the spot!
If YOU are interested in participating in this great project, you can simply go to
www.haea.org and click on the Scientific Registry icon. 

Physician Question and Answer Panel
Dr. Henry Li, his colleague Dr. Mark Scarupa, and Dr. Marc Riedl fielded a wide variety of questions from the patient community at our Bethesda meeting. In Cincinnati, Dr. Paula Busse and Dr. Jonathan Bernstein took questions from attendees. There is not enough space to post the detailed questions and answers in this summary. However, the HAE Association is happy to field all questions from each and every member of our community, and respond with definitive answers from world class HAE scientists/physicians. HAE friends can contact their Patient Services Representative or submit a question through our website at this link:
http://www.haea.org/contactus.php 

The US HAE Association 2009-2010
On-going programs and plans that will benefit all HAE patients were discussed at the end of both regional patient meetings and included:
* Expanding the HAEA’s Patient Services- we are growing to ensure we are well-positioned to serve as the primary problem solver for HAEA friends who are interested in exploring new therapies, or have any HAE-related issues or needs.
* Implementing a robust research infrastructure via our Scientific Registry that includes offering medical research grants aimed at bettering the lives of HAE patients.
* Conducting regional meetings, workshops, and lectures for patients and physicians interested in learning more about HAE.
* Continuously upgrading our web page to ensure it remains the most authoritative, unbiased Internet source for HAE-related information. 

We were glad to see all of our HAE friends at our first two US HAEA Regional Patient Meetings. We look forward to seeing everyone at our next Meeting! Please check www.haea.org for more information. 

Tony Castaldo, President US HAEA
Janet Long, Executive Vice President
Donna K. Davis, Assistant Vice President for Patient Services and Clinical Programs
US HAEA Partners with Rare Disease Day – Feb. 28, 2010 

The US HAEA partnered with NORD to promote awareness of rare diseases in support of the global observance of Rare Disease Day on February 28, 2010. 

We displayed the Rare Disease Day logo on our webpage and some of our HAEA friends took the initiative to send a letter to their government representative or to reach out to their local newspaper to tell their own HAE story. 

Rare Disease Day is, of course, not a single event. It is an educational and awareness campaign that began three years ago and now is rippling out around the world. 

We are very happy to become part of this important effort, and we look forward to working every year to raise awareness of all rare diseases today, but especially awareness of HAE in the US and abroad.
Your US HAEA – Representing Our Members at Two Major US Physician Conventions 

Your US HAEA represented all of its members at two recent national meetings for allergists and immunologists. 

The first, theACAAI was held in Miami, FL in November, 2009. The second, the AAAAI, was held in New Orleans, LA this past Feb. 26 – Mar. 2. 

The US HAEA hosted a booth where phy
sicians could learn more about HAE – disease, diagnosis and treatment -as well as the numerous patient resources offered by our Association. 

More than 4000 physicians attend both the ACAAI and the AAAAI meetings and your US HAEA
is proud to have attended on your behalf.

Introducing our newest Patient Services Representative 

Your HAEA
Patient Advocates
By Region
US HAEA Scientific Registry – Join Today! 

If you check our website counter, you will see that we
now have more than 515 registrants total! 

We are looking for every person who has been
diagnosed with HAE and all those who are a blood
relative of someone with HAE to join the Registry. 

So…
if you have not already, please go to www.haea.org and click on the Scientific Registry icon! Please encourage your family members to join too! 

What does it mean to be a member of the Scientific Registry? It means you will donate a small blood sample and a cheek swab containing your DNA. These samples are stored in a biorepository in a completely secure manner – there will be no identifying information that could connect you to these samples. Researchers will ask the HAEA for permission to use some of the collected samples to better understand HAE and, eventually, find a cure! 

You will also be required to answer some online questionnaires over a five year period. This data will also help us to discover answers to all the questions we have about how HAE affects our lives. 

So please sign up now and be part of this great undertaking to improve the life of every HAE patient! You can make a difference!
The US HAEA is now on Facebook! 

As you may already know, your HAEA is now on Facebook! Please join your HAEA friends and family on Facebook by going to http://www.facebook.com/group.php?gid=340733657767 . 

This Facebook page is provided for our HAEA friends in response to your requests for more social networking opportunities. We value the struggles and triumphs of all of our HAEA friends and hope you will make use of this page to share your HAE stories. 

It is important for you to remember this is a public forum – it is not HIPAA compliant and although we will try our best, we cannot guarantee your privacy. Please help us keep this Facebook page as it is intended – a place to meet other HAE patients and families and talk about your own experiences. 

Any questions regarding medical difficulties, physician referrals, medicines, etc. should be posted on our privacy-protected HAEA Listserv (http://mailman.listserve.com/listmanager/listinfo/discusshae ). 

Members of the US HAEA may be confirmed as Facebook friends for this site – if you are not yet a US HAEA member, please go to www.haea.org and sign up today! (Note: For the safety of our members, the page administrator reserves the right to monitor posts and to deny access to anyone who violates commonly accepted Facebook etiquette.) 

We are excited about providing this Facebook page for our HAEA community and look forward to all the great HAE connections that will be made here!
Introducing our newest Patient Services Representative 

Candice (Candi) Fuqua – Patient Services Representative 

Candi and two of her children have HAE. Candi
inherited HAE from her father whose tragic death in 1996 at
age 44 was attributed to a lack of information regarding his
condition. Having watched her father suffer along with
managing her own condition and providing care for her
severely affected children makes Candi well-versed in managing chronic medical conditions. 

Candi says, “In the last 2 years since my children were diagnosed, our lives have been like a rollercoaster ride. The US HAE Association, through its Patient Services staff, compassionately cared for us, providing knowledge and guidance.” Recognizing how dramatically patients’ lives can be changed by information about and access to HAE therapies is what led Candi to join the Patient Services team. 

Candi is a Patient Service Representative for Region 6 (see below). She is dedicated to doing everything possible to make a difference in the lives of HAE friends- providing information on new HAE treatment options, answering questions, and dealing with HAE’s inevitable challenges. We are pleased to introduce Candi as our newest US HAEA Patient Services Representative.
Your Patient Services Representatives by Region 

Donna Davis, Assistant Vice President for Patient
Services and Clinical Programs
(808) 216-1029
donna-davis@haea.org
(Supervises All Regions) 

Region -1 Michelle Williamson, Director for Patient Services and Clinical Programs
(972) 814-5205
michellewilliamson@haea.org
(Region 1 includes – Alaska, Colorado, Hawaii, Illinois, Kentucky, Missouri, Nevada, New Mexico, South Dakota, Utah and Washington DC) 

Region – 2 Lois Perry, Assitant Director for Patient Services and Clinical Programs
(559) 259-0572
loisperry@haea.org
(Region 2 includes – California, Iowa, Maine, Mississippi, Montana, Oregon, Vermont, Virginia and West Virginia) 

Region – 3 John Williamson, Patient Services Representative
(972) 984-0621
john@haea.org
(Region 3 Includes – Arizona, Idaho, North Dakota, Pennsylvania, South Carolina, Texas and Wyoming) 

Region – 4 Sally Urbaniak, Patient Services Representative
(904) 826-6700
surbaniak@haea.org
(Region 4 includes – Connecticut, Delaware, Florida, Massachusetts, New Hampshire, New York, and Rhode Island) 

Region – 5 Jenny Barnes, Patient Services Representative
(252) 585-0763
jennybarnes@haea.org
(Region 5 includes – Alabama, Arkansas, Georgia, Louisiana, Michigan, Minnesota, North Carolina and Oklahoma) 

Region – 6 Candi Fuqua, Patient Services Representative
(308) 627-4586
candifuqua@haea.org
(Region 6 includes – Indiana, Kansas, Maryland, Nebraska, New Jersey, Ohio, Tennessee, Washington and Wisconsin) 

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For Clinical Trial Referrals, Contact Patient Services/Clinical Programs Representative, John Williamson
(972) 984-0621
john@haea.org