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Highlights from the HAEA 2008 National Patient Conference

Over 275 members of the HAE community attended the 2008 HAE Association National Patient Conference that was held in Chicago on October 17-19, 2008. Below are the highlights of the conference:
  • HAEA President Anthony Castaldo kicked off the meeting and discussed: "Exciting Times for the HAE Patient Community."  Key points made during Tony's talk included:

    1. The approval of C1 inhibitor in the United States is the first step in preventing future HAE-related deaths (there was a solemn moment of silence during rthe conference opening in honor of three HAE friends that died of laryngeal swelling in 2008) and significantly improving the lives of those in our community who are disabled by HAE.
    2. HAE friends interested in getting access to C1 inhibitor or finding out more about the medicine should contact the HAEA's Patient Services group by sending an email to Donna Davis donna-davis@haea.org or Michelle Williamson michellewilliamson@haea.org.
    3. The HAE Association advocates that every HAE patient has a fundamental right to choose a treatment program that best fits their individual needs. The approval of C1 inhibitor opens up a new world for treating HAE as evidenced by the presentations from European HAE friends (see below), and we will help each and every HAE friend by providing the information necessary to support whatever therapy option is chosen
    4. The HAE Association will work closely with ViroPhama (the company that received the approval for C1 inhibitor) to support and represent each HAE friend in the insurance reimbursement process. ViroPharma also has a financial assistance program available for patients who might not be able to afford the medicine. Our Patient Services staff (Donna and Michelle) will be able to provide information on that program as soon as more is known.
    5. The HAEA's Burden of Illness study conclusions were presented. As HAE friends may recall, almost 500 patients participated in this landmark study designed to provide insurance companies with scientific data that proves new medicines are needed to adequately treat HAE. A press release that provides an overview of the study will appear on the HAEA web page shortly, and the HAE Association will ensure that the data gets into the hands of those who are responsible for insurance reimbursement. The bottom line conclusion of the study is: HAE poses significant financial, social, and emotional burdens that the current medicines do not address.
    6. The HAE Association is conducting the largest scale study of HAE patients ever attempted.  The study will be accessible over the internet and totally anonymous. The goal is to collect scientifically valid data that will once and for all provide accurate information regarding the safety, effectiveness, and side effects of HAE medicines.  Tony emphasized that patient participation in this important effort will help to improve the lives of all HAE patients as well as provide a brighter future for our children.  The androgen study will be rolled out soon - watch for more information on how you can participate.
  • The Conference Keynote Presentation "Decades of safe and effective use of C1 inhibitor for treating and preventing HAE attacks in European patients; and the effectiveness and safety of home infusion," was delivered by Dr. Marcel Levi, Professor of Medicine at the University of Amsterdam.

    Dr. Levi discussed his long-term observations (and published studies) of HAE patients in the Netherlands who have been using C1 inhibitor to control their HAE attacks.  His research has unequivocally shown that:

    1. C1 inhibitor has a decades-long track record as a remarkably safe and effective therapy; and
    2. HAE patients who have home access to C1 Inhibitor generally have no problems self-infusing the medicine or relying on a family member to give the injections.
    3. Dr. Levi and his staff require that patients complete a training course before they are allowed to begin home infusion.
  • Representatives from each of the five drug companies provided updates on the status of their US clinical programs.
    1. ViroPharma (formerly known as Lev Pharma)  ViroPharma's C1 inhibitor (the brand name is Cinrzye) was approved by the FDA as an attack prevention therapy. The company anticipates that the product will be available in early to mid December. ViroPharma is putting together an infrastructure to make Cinryze available to every HAE friend that could benefit from the medicine. The program is called Cinryze Solutions, and 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.
    2. CSL Behring (Berinert brand of C1 inhibitor) has completed its phase 3 trial and expects Berinert to be approved for treating acute HAE attacks (attacks that have begun) sometime in December 2008.  CSL Behring officials noted that the company is a global leader in the plasma protein biotherapeutics industry, and are experts in producing products to treat rare diseases, and that Berinert has been safely and effectively used throughout the entire world for decades.
    3. Dyax (Ecallantide) completed its second phase 3 clinical trial for its subcutaneous HAE attack treatment and reported that the data generated during the trial indicates the medicine is an effective and safe therapy.  Ecallantide 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 company filed for FDA licensure in September 2008, and the product could be approved in early 2009.
      oPharming NV (recombinant C1 inhibitor) announced 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 plans to apply for a US license in the near future.
    4. Jerini AG (Firazyr) conducted a US clinical trial that did not meet the statistical significance requirements for effectiveness required by the FDA. Firazyr is delivered through a subcutaneous injection and works by blocking the peptide that causes HAE-related swelling. The product has been licensed in Europe and Jerini is now working on plans to conduct another clinical trial in the US.
    5. Pharming NV (recombinant C1 inhibitor) announced 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 plans to apply for a US license in the near future.
  • HAE friends from Europe Discussed How Access to C1 Inhibitor Changed Their Lives-Ursula Huffer from Germany and Henrik Boysen from Denmark--chronicled their HAE treatment experiences. Danazol's adverse side effects and general lack of effectiveness led both Ursula and Henrik to stop steroid therapy and take advantage of a treatment regimen (offered by compassionate physicians in Germany and Denmark, but not necessarily elsewhere in Europe) that features regular injections of C1 inhibitor. Ursula and Henrik described how switching from steroids to using C1 inhibitor to prevent attacks was life changing. They are now both leading normal lives and are especially happy that they can travel without having to worry about life threatening throat swelling or the awful side effects they experienced while on androgens.
  • HAE Association Study Results: Quality Of Life Aspects Of C1 Inhibitor Home Access Compared Receiving Therapy At A Medical Facility--HAEA Medical Advisor Professor Bruce Zuraw summarized the results of a HAE Association sponsored study that compared the safety, effectiveness and quality of life of patients who had home access to C1 inhibitor concentrate to those that traveled to a medical facility for their treatments. Professor Zuraw provided the study's top line results: "Physician Supervised self-managed C1 Inhibitor infusions are a safe and cost effective alternative to treatment in a medical facility and also enhance the patients' quality of life."
  • HAE Association's Scientific Registry--Dr. Zuraw later presented information on the HAE Association's Scientific Registry. Dr. Zuraw and the HAEA, working together, have designed a state-of-the-art registry that will produce high quality "scientific grade" data. Dr. Zuraw stressed the importance of obtaining maximum patient participation once the registry is up and running- it is the collected data that will entice researchers to continue searching for better HAE treatments and, ultimately, a cure!
  • Physician Questions and Answer Period--HAE specialists-HAEA Medical Advisor Professor Bruce Zuraw, Dr. Henry Li from Wheaton, MD, and Dr. Marc Riedl from UCLA,and Dr. Andrew Grant from Galveston, TX fielded a wide variety of questions from the patient community. There is not enough space to post the detailed questions and answers in this summary. However, the HAE Association is happy to field any and all questions from each and every member of our community, and get definitive answers from world class HAE scientists. HAE friends can contact our patient services group (see email addresses above) or submit a question through our website at this link: http://www.haea.org/contactus.php
  • The HAE Association 2008-2009 Program--officers presented ongoing programs and plans that will benefit all HAE patients: The programs discussed included:
    1. Expanding the HAEA's Patient Services-we are growing to ensure we are well positioned to serve as the primary problem solver for HAE friends who are interested in exploring new therapies, or have any HAE-related issues or needs.
    2. Implementing a robust research infrastructure that includes offering medical research grants aimed at bettering the lives of HAE patients.

      o   Rolling out and recruiting patients for our HAE Scientific Registry;
    3. Conducting regional meetings, workshops, and lectures for patients and physicians interested in learning more about HAE.
    4. Establishing a 24/7 toll free number to help patients deal with HAEissues that arise.
    5. Continuously upgrading our web page to ensure it remains the most authoritative source on the web for HAE-related information.
Over the next several months, the HAE Association staff will be sending out newsletters with updates on the status of medicines that are awaiting FDA approval. We will also keep you apprised on progress in building the HAE Patient Registry and expanding our patient services capabilities.

The best way to help the cause and make sure we can keep you informed about forthcoming new treatments is to ensure that you and all of your affected family members have signed up as HAE Association members. Remember its free!

So, please, if you have not already done so, fill out the membership form. Keep in mind, we go to great lengths to ensure your personal information is secure and protected. Access to information is strictly limited and tightly controlled. We have never and will never disclose any personally identifiable information to an outside party.


Tony Castaldo, President US HAEA
Janet Long, Vice President
Donna K. Davis, Director of Patient Services and Clinical Programs





Support for the HAE Association's 2009 Activities Provided by:
















The US Hereditary Angioedema Assoc., Seven Waterfront Plaza, 500 Ala Moana Blvd., Suite 400, Honolulu, HI 96813, (866) 798-5598
The US HAEA's 2010 corporate sponsors
ViroPharma CSL Behring Dyax Shire Pharming