erdheim-chester disease october 2010 ecd global alliance
TRANSCRIPT
Erdheim-Chester Disease
October 2010
ECD Global Alliance
What is Erdheim-Chester Disease?
Non-Langerhans Cell Histiocytosis Too Many Histiocytes (cells which normally fight
infections) Infiltrate Organs
Causes Scar-Like Tissue to Surround the Organs & Arteries Bone, Brain, Kidney, Eyes, Lungs, Abdominal
Tissue/Organs, Cardiovascular, Skin and More Rarely Others
Symptoms Vary Between Patients Depending on Which Organs are Involved
Without Successful Treatment: Organ Failure
Why Have I Never Heard of ECD?
First Described in 1930 by an Austrian (Dr. Erdheim) and an American (Dr. Chester) Pathologist
Only About 350 Published Cases in the World
Never Heard of by Most Doctors
Diagnosis is Difficult and Time Consuming, Made Only by Chance in Some Cases
Not Known if Cancer, Auto-Immune, Genetic, etc.
What is the Status of ECD?
Only a Handful of Doctors Have Treated Multiple Cases
Anecdotal Treatments Available Immunotherapy, chemotherapy, radiation, surgery, hormone therapy,
corticosteroids, anti-inflammatory drugs, antimetobolite, new cancer drug which specifically inhibits a particular enzyme
Results are Variable Literature Says Most Succumb in 2 to 3 Years,
However We See Some Patients Live for Decades
International Disease Advocacy Group Formed –ECD Global Alliance – www.erdheim-chester.org
First a Diagnosis is Needed
“When you hear hoofbeats, think horses, not zebras”
Specialists Often Only Know What it is Not
Doctors have Not Seen or Studied Many of the Rare Diseases
Often Patients Find the Validity of Their Symptoms Being Questioned
Diagnosis Often Takes Years with Patients Shuffled from One Specialist to Another
What Are the Patient Issues?
Sickness Itself Getting a Diagnosis Finding Knowledgeable Doctors No Agreement on Best Treatments Health Care Costs / Insurance Coverage
Expert Doctors Most Likely Out of Network Treatments not Scientifically Verified
Transportation / Housing Costs Disability Difficult to Obtain Lack of Support Structure Lack of Information Relative to
Progression
Status of the ECD Global Alliance
Carol, Kathy, RuthAnn Met
by Phone
First Online Chat
Session Held
Published www.erdheim-chester.org
website
Teleconference with ECD
Patients and Dr. Kurzrock
First Newsletter Published
501(c)(3) Non-Profit Paperwork
Submitted & Board of Directors Formed
Jan. ‘08
Apr. ‘08
Sep. ‘08
Oct. ‘08
Oct. ‘08
Jan. ‘09
Apr. ‘09
Aug. ‘09
Dr. Vaglio’s Treatment Protocol
Communicated
Dr. Haroche’s HLA Study
Communicated
Organizational Activity Support Activity Bringing Information to Patients
Medical Advisory
Board Formed
Dec. ‘09
501(c)(3) Tax Exempt
Status Granted,
Effective as of Aug. 28,
2009
Mar. ‘10
Promoting Research
Apr. ‘10
RFP for Research
Project Communicated
ECD Global AllianceWhat is Needed?
Raise Additional Research Funds
Continue Dialogue with Research Physicians
Create and Maintain Patient Registry
Future ECD Symposium
Support for Patients with Regardto Disability and Other RelatedIssues
What Can We All Do?
Be Supportive
Be an Advocate
Be an Organ Donor
Be Generous
Questions?
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