Erdheim-Chester Disease
ECD Global Alliance
(http://www.erdheim-chester.org/ECDStudies.html)
The following provides information related to ongoing studies on ECD. These studies are still active and your participation is encouraged.
|
Title (click on title for more
information) |
Study Objective |
Contact Information |
Requested ECD Patient Involvement |
Date Study Began |
|
Clinical and Basic Investigations into Erdheim-Chester Disease |
Natural history study designed to
understand the disease, identify its cause, and classify it
according to presentation and progression.
The study does not include any modifications to
patient treatment plans although follow on studies may
address this. |
Juvianee
Estrada-Veras, M.D. |
ECD Patients are being recruited for
one week of testing at the NIH in Bethesda, Maryland.
(The NIH will pay for all testing costs.
Travel costs will be paid on a case-by-case basis.) |
August 2011 |
|
Erdheim Chester Disease:
Clinical Phenotype and Outcome.
A Multicentre Survey |
Correlate the involvement of
different organs with the response to therapy and the
outcome. Expand
the knowledge of ECD, allowing earlier diagnosis, better
classification of the different disease forms, and improved
prediction of the patient prognosis. |
Augusto Vaglio,
M.D. |
Requesting complete medical records
of ECD patients. |
December 2010 |
|
Developing a Comprehensive Genomic Database -
Histiocytosis Research Laboratory |
Understand the basic causes of
histiocytosis which hopefully will lead to more effective
treatments. |
Carl E. Allen,
M.D., Ph.D. |
Requesting ECD patient surgical
biopsies, blood samples, cerebrospinal fluid, and/or medical
information. |
Unknown |
|
Investigate the class I and class II
HLA phenotypes of ECD patients to determine whether specific
haplotypes are associated with an increased susceptibility
to the disease or segregate with specific tissular
involvement of ECD. |
Julien Haroche,
M.D., Ph.D. |
Results of HLA I and II blood tests
from ECD patients. |
October 2008 |
|
|
Pilot Study of Sirolimus with Prednisone as
Treatment for ECD |
Assess whether (and if yes, to what
extent) the combination of sirolimus and prednisone may
prolong progression-free survival of ECD patients. |
Augusto Vaglio,
M.D. |
Newly diagnosed, untreated patients
and patients who experience progressive disease (both
treated with other regimens and not undergoing treatment)
can be enrolled. |
November 2007 |
|
Retrospective Chart Review of Patients With
Histiocytosis: Langerhans Cell Histiocytosis Or
Erdheim-Chester Disease (Non-Langerhans Cell Histiocytosis)
Treated With Interferon-alpha To Determine The Clinical
Response And Outcome (RCR04-0567) |
Understand clinical characteristics,
underlying biology, response to therapy and outcome of
patients with Erdheim-Chester disease. |
Razelle
Kurzrock, MD, FACP |
Patients being treated with imatinib
mesylate or interferon-alpha |
Unknown |
|
Better define the pro-inflammatory
microenvironment inside ECD lesions.
To evaluate the possibility to interfere with the
progression of ECD by means of specific inhibitors already
available for human usage, in order to find new therapeutic
options for the disease |
Lorenzo Dagna,
M.D. |
None known. |
January 2011 |
|
|
A transcriptome analysis of the
monocytes in Erdheim-Chester disease |
Perform a transcriptome analysis on a
large international series of ECD patients in order to
better understand the pathophysiology of the disease. |
Julien Haroche,
M.D., Ph.D. |
None known. |
2009 |
Last updated:September 25, 2011