erdheim-chester disease october 2010 ecd global alliance

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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