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"Patient Power!"

aporzeca Message
2 Jan 2016, 03:23 PM

I am pleased to report that the U.S.National Institutes of Health has acknowledged, via an article in the Winter 2016 issue of their patient-centered magazine, _MedlinePlus_, the vital collaboration of the members of this Community in furthering scientific research on SCLS. The article is reproduced below and appears at http://nw08.american.edu/~aporzeca/Patient%20Power.pdf *Patient Power!* _Thanks to the Internet, the "crowdsourcing" of disease information among patients and doctors is becoming a valuable lifeline around the globe. For patients with serious rare diseases, crowdsourcing can mean the difference between life and death._ "Indispensible" is the way NIH researcher Dr. Kirk Druey describes the actions taken by one of his research patients in spreading awareness of a deadly but very rare disease. Ten years ago, systemic capillary leak syndrome (SCLS) almost took the life of then-56-year-old economist and American University professor Arturo Porzecanski. Parts of his vascular system suddenly leaked out plasma into other parts of his body. In trying to save him before his organs could fail, doctors flooded his system with too much replacement fluid that damaged his leg and arm muscles. When the attack occurred, Porzecanski and most doctors knew nothing about SCLS. Realizing that another bout of the disease would probably kill him, Porzecanski set out to discover more about SCLS and any other patients who had experienced it. Through his tenacity and smart use of the Internet, Porzecanski was not only able to find information on new treatments but also identify other patients seeking help in the United States and across the globe. He set up an SCLS community group on a social website called RareShare (www.rareshare.org). Patients, their loved ones, and health care professionals were sharing contacts, treatment information, and the latest research. _The NIH Connection_ In 2008, having built up a network of patients and front-line physicians who could provide medical histories and blood and tissue samples needed to carry out research on SCLS, Porzecanski sought out investigators at NIH's National Institute of Allergy and Infectious Diseases (NIAID). With research already under way on other, related vascular diseases, NIAID's Druey made use of Porzecanski's virtual community to enable a research protocol on SCLS. For a rare disease that has fewer than 1,000 known cases, to recruit patients and their doctors to collaborate with researchers is very difficult yet crucial to eventual success. "Arturo's efforts were indispensable," says Druey. "And we now have the largest registry of patients for that disease in the world." Through Porzecanski's efforts on RareShare, he quickly became the go-to patient resource for those with SCLS. He also helped get blood samples from patients to assist Druey and his colleagues in their research at NIH. "Arturo came to NIH as an inpatient and stayed several days in the Clinical Center hospital here in Bethesda," Maryland, says Druey. Porzecanski also came to NIH to show Druey and his colleagues all of the research he had put together on SCLS—to pass along his knowledge in the hope that it could help speed along life-saving information to others. Some 40 SCLS patients and their physicians have provided Druey with blood and tissue samples, or at least medical histories, to further knowledge of the rare disease. He has already published 10 research articles that shed more light on SCLS, with more papers in the pipeline. Porzecanski has also helped spread the word to SCLS patients that French researchers have developed a treatment of highdose intravenous immunoglobulins (IVIg) that can be effective in stopping SCLS and lengthening the lives of patients—including Porzecanski. Porzecanski and Druey have made quite a team in their efforts to understand and defeat SCLS. "I'm fully invested in this research," says Druey. "I feel this is something I owe to these patients." In fact, Druey and his colleagues have already applied for a grant to help fund a gathering of scientists from across the globe who are now studying SCLS. Porzecanski couldn't be happier that such "patient power" efforts will help SCLS patients who otherwise would be without hope.
rnuara Message
2 Jan 2016, 03:46 PM

Arturo, First- Happy New Year!!! This is an outstanding article on SCLS and the progress that has been made. A special thank you to you and Dr. Druey. Your combined efforts and tenacity have been a life saving resource for the SCLS community. We all owe you great gratitude for your efforts and your support.
WazzaACT Message
2 Jan 2016, 10:14 PM

Arturo. A terrific article recognising your fantastic work that has helped all of us in so many ways. It is great to see your efforts acknowledged. From me I again thank you for your magnificent effort. Best wishes for the New Year. Warren
Josephite Message
3 Jan 2016, 06:37 AM

Thanks for posting Arturo. That was a good read.

3 Jan 2016, 02:48 PM

Arturo, I too thank you for all your work and research on SCLS. Thanks to you and Dr. Druey, I celebrate another happy, healthy New Year with my family. I am grateful beyond measure to you both. Cristina
Barney Message
4 Jan 2016, 03:21 PM

Arturo, Thanks for sharing this and thanks to you and Dr. Druey and Dr. Greipp for your work on this cause. All of us and our families have benefitted greatly from your willingness to support those of us with SCLS. Words cannot express what we owe to you. I remember a couple of the things we discussed when you visited me at the NIH: "I am one of the lucky ones". When you said it, I had a hard time getting my head around it. But you were right - I was diagnosed early, had no lasting physical effects, and now IVIG is working very well for me. And the other thing you said was; that when going in for IVIG treatments, I might be fortunate to have SCLS vs. what some others are battling. I had a hard time with that as well, but again, you were right. While IVIG causes two full day monthly trips, my health has been very good. I witness every month, the battles others are having as they have chemotherapy and other very challenging treatments. I have known a few now that have lost their battles and have watched that play out a few times. So again, thanks to you, Dr. Druey and Dr. Greipp for the awareness you have brought to SCLS.
Ritz Message
4 Jan 2016, 03:38 PM

Thank you! We need to keep the research going. As one of the people who is off of IVIG for now, due to the fact that it started causing attacks, I am hoping for a new treatment.
tiggrrr027 Message
6 Jan 2016, 09:52 AM

Arturo, Thank you for sharing this! Thank you to you,Dr.Druey and Dr Greipp for everything you have done in regard to the identification, research and treatment of SCLS. More than that you have done this with great compassion and kindness for those stricken with this syndrome. There is more to be done as a cure has not been found although IVIG is a blessing. With new patients being diagnosed more quickly and accuratly and the use of IVIG we have grown in numbers. That means more voices to be heard!Happy New Year to all! Linda
catsanmice Message
23 Jan 2016, 02:50 PM

I was diagnosed and put on IViG relatively quickly and have had such an easy time with SCLS compared to others. I can't imagine what life would have been like for me if not for your efforts. Thank you, Arturo, and God bless you.
genecridge Message
24 Jan 2016, 11:20 AM

Happy New Yea Arturo. Thank you and Congratulations on the recognition that you greatly deserve.