關於我們
我們的使命
ECD聯盟致力於與厄Erdheim-Chester病相關的意識、支援、教育和研究。該組織與全球社區合作完成以下工作：
提高對 ECD 的認識。 我們努力促進疾病的早期診斷，並激發關注和努力增加對疾病和治療的瞭解。
為受疾病影響的人提供支援。 我們將繼續尋求一切途徑，盡可能減輕受 ECD 影響的人的負擔。提供資訊、轉診和與受疾病影響的其他人進行一對一聯繫只是通過 ECDGA提供的支援中的一小部分。
宣導和支援研究計劃。 此類研究計劃的目的是更多地瞭解 （a） 疾病，（b） 疾病的有效治療方法，（c） 可能的治療方法，和/或 （d） 完全預防疾病的途徑。這將包括但不限於與醫務人員、研究人員和其他衛生組織合作，以幫助確定需要研究的領域，資助研究，並説明向其他衛生機構宣傳 ECD 研究的好處。該聯盟還將作為管道，根據書面指南將所需資訊從其成員傳輸到研究界。
Kathy Brewer，創始人兼總裁
共用教育材料並促進相關方之間有關 ECD 的信息共用。 這包括向患者、親人、醫務人員、研究人員、媒體和其他組織提供與 ECD 相關的最新、可靠和事實資訊。它還將包括促進、贊助和舉辦研討會、專題討論會和其他會議，以促進聯盟的目標。
十年的影響力
對於大多數人來說，Erdheim-Chester 病的經歷始於令人沮喪、恐懼的答案尋找。
對於 ECD 全球聯盟創始人 Kathy Brewer 和她已故的丈夫 Gary 來說，這無疑是正確的。在他去世時，加里已經病了 20 年，病重了 3 年。他有腎病的癥狀，事實上，他接受了腎移植手術。但直到屍檢，Kathy 才知道 Erdheim-Chester 是真正的原因。
這是一種非常罕見的疾病所面臨的挑戰。當一種疾病在全球範圍內估計只折磨著 1,000 人時，很少有醫生具備診斷它所需的熟悉度，尤其是當它表現為從骨痛和體重減輕到視力和平衡問題等癥狀時。（在許多身體系統中， ECD 會影響垂體和腎上腺、肺、心臟周圍的膜、腎臟和大腦。
然而，今天的 ECD 情況與 2008 年截然不同，原因是 ECD 全球聯盟。該聯盟由通過互聯網找到彼此的患者護理人員發起，現已從6個家庭發展到近800個家庭，它極大地改變了 ECD 患者的前景。
今天，大多數 ECD 患者可以通過現在使用的幾種治療方法中的一種來控制疾病的進展。許多人已經從面臨 3-5 年的典型預期壽命轉變為在繼續生活時控制癥狀。
我們對未來的希望
閱讀您的倡導團體目前正在實施的5年戰略計劃。
該聯盟在國際領先的醫療機構建立了一個護理中心網路，每個中心都有一個準備診斷和治療 ECD 的專家團隊。
“在 ECDGA 之前，沒有治療疾病或獲取資訊的中心場所，也沒有組織。你會遇到一位主動為你治療的醫生，然後發現他們從未治療過一名 ECD 患者。– ECD 患者的照護者
“從科學和人類的角度來看，過去十年取得了 如此多的成就 ，其中很少有沒有聯盟的印記。” – ECD ） 研究員
它建立了一個家庭支持網路，並將國際 ECD 社區的成員聚集在一起，參加一系列年度會議 – 這是患者相互認識並與 ECD 專業醫生交談的機會。
它啟動了患者登記處以建立 ECD 數據檔案，並與 NIH 合作招募患者進行自然歷史研究——這是瞭解疾病進展的關鍵。
“全球聯盟堪稱典範——真的、真的宣導、聯繫醫生，讓人們走出孤島並建立聯繫。”- ECD 護理中心醫生
這個組織感覺就像一份禮物。致力於推進對 ECD患者的護理的人。我從未體驗過如此發自內心的奉獻。 – ECD 患者
而且，最引人注目的是，它説明使基因治療工作成為可能，導致 FDA 批准了一種治療 ECD 有效的藥物。
更多成就
| Year | Accomplishment | Details |
|---|---|---|
| 2021 | Webinars, Chats, and Strategy continue | The organization continues to support patients, families, and physicians with meaningful connections as much as possible during the COVID pandemic. All while implementing our 5-Year Strategy of growth and improvement. |
| Virtual Medical Symposium | With a pandemic that no one expected or hoped for, the annual events were canceled for 2020. This year we pulled together our top physicians and researchers to create an online conference, continuing the collaboration and growth in the medical field without delay. | |
| 2020 | Community Webinars | Educational webinars hosted by our expert medical members replaced the in-person events normally held annually for the ECD community. This involved partnerships with other histiocytosis advocacy groups. These partnerships have strengthened and streamlined this project, benefiting all involved. |
| High-Level Strategy Planning | The ECD Global Alliance is a small but extremely effective organization leading the fight against the disease. Over the past decade, it has helped achieve major advances in treatment and offered invaluable support to patients and caregivers. As a result, the Alliance has helped change the meaning of an ECD diagnosis. Going forward, with expanded donor investment, the Alliance has plans to accomplish even more. | |
| 2019 | Endocrinology Conference ECD Exhibit | The purpose of the ECD Global Alliance exhibition is to generate awareness of Erdheim-Chester Disease, communicate the recent research landscape and new treatment opportunities for patients, and facilitate the growth of the ECD Global Alliance medical professional network among interested physicians and scientists. In addition, connect with other exhibitors offering services that can help the ECDGA further awareness and education of ECD. |
| Second Consensus Article Published | As a result of the ECD Medical Symposia in previous years, the second-ever comprehensive guidelines were published by leading ECD experts. This important document was a collaborative collection of data gleaned from extensive research of the disease and the treatment of patients worldwide. | |
| Chat Program | The infamous COVID pandemic pushed the ECDGA to find creative ways to engage the community. With online meetings becoming normal, the Alliance began hosting chats for various audiences. This includes patients, friends, family, caregivers, physicians, and regional and international language meetings. | |
| 2018 | Hematology Exhibit | Hematologists from around the world eagerly engage with ECDGA exhibitors to learn more about this rare disease. |
| Grand Round Medical Presentations | ECD-knowledgeable physicians provided education on ECD at the University of Miami and Houston Methodist. | |
| 6th Annual Event held in Orlando, FL at Orlando Health | A total of 109 members from eight countries attended to learn about the drug trial process, how to manage multiple medications, how to care for yourself with nutrition and exercise, and about care management. | |
| 7th Annual Events held in Milan, Italy | The purpose of the medical symposium is to unite international medical professionals in discussion and education of studies, experiences, and knowledge regarding Erdheim-Chester Disease. In this space, medical professionals are encouraged to present findings and knowledge to audience members. During the patient meeting, patients and family members are encouraged to ask questions, reach out and learn about the ultra-rare disease that affects their lives. This meeting was co-hosted with Dr. Lorenzo Dagna. | |
| $50,000 Grant Awarded to Dr. Nicole Coufal at UCSD (Announced in 2020) | In 2019, a $50,000 young investigator award was granted to Nicole Coufal with the University of California, San Diego. “Pathophysiology of Neurodegenerative Erdheim-Chester Disease and Langerhans Cell Histiocytosis” | |
| 2017 | 1st FDA-Approved Drug: Vemurafenib (Zelboraf) | The Memorial Sloan Kettering Basket Trial led to favorable results for the Erdheim-Chester patients enrolled, eventually resulting in approval. The ECDGA assisted in the recruitment process to make this possible. |
| Care Centers Added to Network | Locations in Israel, China, Nashville, Canada, and Australia were welcomed to the network. | |
| Grand Round Medical Presentations | ECD-knowledgeable physicians were sponsored by the ECDGA to give educational talks at universities in Houston, TX, New Orleans, and Shreveport, LA. | |
| Pathology Conference Exhibit | The ECDGA medical community volunteered to create materials to educate attendees at an annual pathology conference. The ECDGA team hosted an exhibit booth, where they introduced and discussed ECD at length with pathologists around the world. | |
| 5th Annual Events in New York | Patients, family members, nurses, research teams, and numerous physicians attended the 2017 Patient & Family Gathering and Medical Symposium held in New York. Attendees were brought up to date on the latest research, treatments, and care management techniques, including the psychological effects of living with ECD. This meeting was co-hosted with Dr. Eli L. Diamond. | |
| $50,000 Grant Awarded to Dr. Benjamin Durham at MSK (Announced in 2018) | The ECD Global Alliance awards Erdheim-Chester disease research grant to MSK to define the disease-initiating cell population in ECD and compare genetic alterations. | |
| 2016 | 4th Annual ECD Patient & Family Gathering | The first European event, held in Paris, France, included 63 attendees from 17 different countries who heard from 16 physicians about ECD organ involvement, the use of scans to monitor the disease and its treatments, techniques to manage pain, and the latest research findings including treatment options. This meeting was co-hosted with Prof. Julien Haroche. |
| 4th ECD Medical Symposium | The first European ECD Medical Symposium was held in Paris, France at Hôpital Pitié-Salpêtrière, where over 50 physicians gathered to collaborate and share data on ECD. This meeting was co-hosted with Prof. Julien Haroche. | |
| 10 Languages Added to Website | Volunteers from around the globe dedicated their time and talents to translating important content in ten languages for the ECDGA website. | |
| 2015 | Development of ECD Care Center Network | Care centers with ECD-knowledgeable medical teams began to form a network to support patients with care and consultation around the world. To date, the ECDGA collaborates with 35 ECD Care Centers serving patients. |
| New Website | An updated website was developed to engage members on all devices. | |
| $143,000 for Three Research Grants Awarded | ||
| 3rd Annual Patient & Family Gathering | Patients, families, and physicians united in Houston, TX for a memorable two-day gathering. | |
| 3rd Annual ECD Medical Symposium | The symposium was hosted at MD Anderson Cancer Center, with Dr. Filip Janku. | |
| 2014 | 2nd ECD Patient & Family Gathering | Patients and family members gather in Bethesda, MD for another two-day meeting including ECD presentations delivered by the experts. |
| 2nd International Medical Symposium | Physicians and researchers presented their work and discussed the state of Erdheim-Chester disease care for patients around the world. | |
| $316,000 Grant to Dr. Eli L. Diamond and Pr. Matthew Collin (Announced in 2015) | Drs. Eli L. Diamond and Matthew Collin were awarded for the development of the ECD Patient Registry. This allowed patients and physicians the opportunity to collaborate and lead research more effectively so that treatment and care can continue to improve and evolve. | |
| 2013 | 1st ECD Medical Symposium | In addition to the patient meeting, the first international meeting was held this year, inviting doctors to present ECD research, case studies, and treatments. This meeting was also the catalyst for collaboration in developing the significant 2014 Blood Journal ECD Consensus document. Collaborative annual meetings have continued to take place around the world each year since. |
| ECD Documentary Film | A patient documentary film was developed and produced to support outreach and education of Erdheim-Chester disease. | |
| 3rd ECD Research Grant Awarded | $50,000 was awarded to Drs. Abdel-Wahab, Diamond, and Hyman at Memorial Sloan-Kettering Cancer Center (MSK). “Somatic Genetic Alterations in the Pathogenesis and Therapy of Histiocytic Disorders” | |
| 2012 | 2nd ECD Research Grant Awarded | $50,000 was awarded to Drs. Haroche and Arnaud ECD research. “Understanding dendritic cell lineages in Erdheim-Chester disease: towards a non-invasive diagnosis” |
| 1st ECD Medical Meeting | A meeting was held in Houston, TX with investigators from various institutions in the US. | |
| 1st ECD Patient & Family Gathering and Medical Symposium | ECD patients and their families came together for the first time this year and the annual event has been a successful way to bring families together ever since. | |
| 2011 | First Grant Awarded to ECD Research | For the first time, $50,000 was awarded to an ECD researcher, and each year following. This progressive mission has set out to enhance research discoveries in Erdheim-Chester disease with the leading researchers and institutions in the field. A total of $750,000 has been granted toward this initiative to date. |
| ECD Natural History Study | The National Institute of Health (NIH) was integral in ECD research and pursuing new treatments that help educate the medical community about ECD. NIH clinical director, William Gahl, M.D., Ph.D., and principal investigator, Juvianee Estrada-Veras, M.D. conducted a natural history study on the disease. The study began in 2011 and this led to a treatment trial in 2014. Multiple medical journals have been published with the data collected from this thorough study of ~60 patients. | |
| 2010 | 501(c)3 Tax Exemption Granted | ECDGA was granted federal tax exemption following the formation of the Board of Directors and Medical Advisory Board. |
| 2008 | ECD Caregivers Meet | In April 2008, a few ECD caregivers, Carol, Kathy, and RuthAnn found each other via the internet. |
| First Chat Session Held | With a spirit of “If you build it, they will come,” an online chat session was scheduled for all those affected by ECD. We started with a group representing only six patients. | |
| ECD Website Published | A website was created with the hope that it would provide much-needed information to families in search of answers for this rare illness. |