One of the great untold stories of this community is its role in finding and disseminating information about what has become the best preventive treatment for episodes of SCLS: intravenous immunoglobulins (IVIG, also available in smaller doses delivered subcutaneously, usually in children with SCLS).
In mid-2006, two years before RareShare and our SCLS community came into existence, I took the initiative to contact all the physicians who had published case reports or other articles on SCLS, to find out if they had learned anything new that might be valuable to us survivors -- something that they had not yet made known to the rest of the world. I dug up or guessed the email addresses of the lead authors of medical articles on SCLS published since the 1980s, and sent them a message introducing myself as an SCLS patient who had been encouraged by his physicians to contact them in search for any and all additional information on the condition that might not yet have been published -- for example, because of the time lags involved in running clinical trials.
Most of the physicians did not reply or did so merely to say that they had not done any further work on SCLS. Typically, they had just treated the one patient they had published about, and that had been it. But I was stunned when I received a substantive reply from a physician and professor of medicine in Paris, Dr. Zahir Amoura, who was then -- and still is -- on the staff of the largest hospital in France, the Hôpital Universitaire Pitié-Salpêtrière. It is a renowned public facility which serves as the teaching hospital for the Sorbonne’s medical school.
In 1997, Amoura and a dozen fellow doctors from around France had published an article reporting on the (until then) largest cohort of SCLS patients -- thirteen -- concluding that “no prophylactic therapy, including terbutaline associated with aminophylline [theophylline], has clearly proven its efficacy in treating SCLS.” The article reported that IVIG had been administered to two of the 13 patients, but that they had continued to have episodes “though they seemed milder.”
However, Amoura’s reply to me in June 2006 -- namely, almost a decade later after his article -- included the following tantalizing statement: “We have now enough patients to compare those who received IVIG to those who were not treated (the control group), and while I cannot firmly conclude, it seems that the results obtained with IVIG are interesting.” However, Amoura made it clear in his email that he would not write about or otherwise disclose the results of his experiments with IVIG until a scientific manuscript was eventually written and duly published.
The Mayo Clinic’s Dr. Greipp and I then followed up with Amoura in subsequent months, inviting him to meet with us to discuss further his findings, especially if he ever visited the East Coast of the United States. And so it was that on his way to a medical conference in Boston in November 2007 -- namely, a year-and-a-half after his intriguing reply to my original email -- Amoura did stop by in New York and met with me and my fellow SCLS patient Judy Davis. (Unfortunately, Dr. Greipp could not attend this meeting.) At that face-to-face encounter, Amoura revealed much greater confidence that IVIG treatments were key to stopping episodes of SCLS, but he again refused to put anything in writing pending the outcome of his experiments and the publication of his results, which he estimated would happen the following year (namely, in 2008).
During 2007-08, namely in the run-up to the founding and take-off of this virtual community, Judy and I continued to endure limb- and life-threatening episodes of SCLS, but we couldn’t act on the encouraging but unofficial news about the success of IVIG treatments that Amoura had given us. The reason was that our doctors, with whom we had shared the information, had told us that it would be impossible to convince our medical insurance companies to cover an expensive, experimental treatment with IVIG until there was at least one convincing scientific article from Amoura or someone else documenting the effectiveness of IVIG for SCLS. In the meantime, they said, we would have to endure more episodes, building a track record that would demonstrate the futility of the Mayo Clinic’s recommended treatment with theophylline and terbutaline.
The outlook for SCLS patients around the world began to change for the better in the second half of 2008, when one of the French physicians (Pierre-Yves Hatron) who had collaborated with Dr. Amoura, broke with him and decided to reveal to the world the good news about IVIG for SCLS. He did so by reporting on three SCLS patients treated successfully with IVIG in Lille, a city at the northern tip of France. The resulting article in the July 2008 edition of the journal Critical Care Medicine said it all in its title: “High-Dose Intravenous Immunoglobulins Dramatically Reverse Systemic Capillary Leak Syndrome.”
It was on the back of this newsworthy article that the physician in Quebec who had been treating Josée Lizotte unsuccessfully with theophylline and terbutaline decided, in August 2008, to start treating her with IVIG -- and the good results obtained made themselves evident immediately. To my knowledge, Josée was the first SCLS patient in the Western Hemisphere to be treated with IVIG for SCLS, and Judy and I found out about her encouraging experience thanks to the newly founded RareShare site which she joined early on. Her physician, Dr. Sepehr Javaheri, would go on to write a testimonial letter about the case which eventually would help me and other SCLS patients get insurance coverage for our first infusions of IVIG.
Cheering us on from the sidelines through the newly inaugurated RareShare site was Claude Pfefferé, the physician-SCLS patient from Switzerland. He was part of the group of French and other European patients being treated and monitored by Dr. Amoura, and he had been put on IVIG starting in April 2005 after failing various other therapies including theophylline and terbutaline. While we were anxiously waiting for Amoura’s article to be published, Claude provided us his own, first-person testimony about the effectiveness of IVIG. He urged us to insist with our doctors and insurance companies that they should give IVIG a try, because the results would quickly speak for themselves. Claude even wrote to Dr. Greipp at the Mayo Clinic to tell him of the smashing success of IVIG therapy in his case and among Amoura’s other patients, thus presenting him with a better alternative to the theophylline and terbutaline cocktail that Mayo had long promoted.
It was also thanks to that important first article about IVIG for SCLS that Nolan Peterson eventually (in July 2009) persuaded his doctor in Iowa to start treating him with IVIG, given that all other therapies had also failed for him, and he was having weekly episodes. Nolan found out about the June 2008 article upon joining our RareShare community, and to my knowledge, he was the first SCLS patient in the United States to be treated with IVIG -- likewise with immediate, positive results.
My turn finally came in November 2009, by which point it had become evident that my treatment with theophylline and terbutaline was also a failure: I was having episodes every other week, up from once a quarter at the start of the year. In addition to the July 2008 article, I had gathered for my insurance company several supportive, testimonial letters from different physicians, among them one I never expected to obtain: it was from Dr. Greipp himself, on Mayo Clinic stationery. Despite his long advocacy of theophylline and terbutaline as the best treatment for SCLS, Greipp was moved to change his mind by the 2008 article and the experiences of Claude, Josée and Nolan, as told in RareShare, to the point that he wrote a formal letter recommending that I be covered for IVIG. I’m sure it was very influential.
As concerns Dr. Amoura’s long-awaited article discussing his encouraging experience with IVIG for SCLS, it was finally published in April 2011 -- an interminable five years after he had written to me saying that “the results obtained with IVIG are interesting.” If it weren’t for the more limited but hugely revealing article that one of his colleagues had published three years earlier, chances are that Josée, Nolan, I and many others would have long been dead by April 2011.
In fact, it is a great shame that my best SCLS friend and RareShare partner, Judy Davis, died in November 2009 before becoming the beneficiary of IVIG treatments. She suffered a massive episode of SCLS and succumbed in the hospital the same week that I was in another hospital not too far away, going through what would turn out to be my last episode of SCLS -- because I was receiving my first infusion of IVIG in the nick of time.