Systemic sclerosis is the single deadliest autoimmune rheumatic disease; rare, progressive, and notoriously difficult to treat, this FDA-designated "orphan disease" has a devastatingly bleak treatment landscape as compared to other autoimmune diseases like Rheumatoid Arthritis and Lupus. At this time, standard operating procedure for physicians is to treat symptoms of the disease as they arise, but no action is taken to treat the underlying disease process outside of the off-label use of pharmaceutical immunosuppression. To date, there are no cohesive treatments that are FDA-approved to treat the underlying disease process in systemic sclerosis; everything is used off-label in the distant hope that it might work to slow disease progression and mitigate symptom presentation. Though some patients find that immunosppression controls their symptoms effectively, others continue to suffer as the disease eats away at their longevity and their quality of life.

In the 1990’s, software company CEO and Scleroderma sufferer Ed Harris pioneered the use of therapeutic apheresis–a common procedure utilized for many other diseases, both acute and chronic–in an effort to treat his own worsening Scleroderma. His working theory was and still is that the protocol he developed is effective in systemic sclerosis because it “de-clumps” red blood cells, which are believed to aggregate abnormally in systemic sclerosis patients; the red blood cells, aggregated as they are, struggle to travel through the veins, thereby causing repeated vascular insult and, ultimately, the systemic fibrosis characteristic of the disease. By undergoing an on-again, off-again protocol of therapeutic plasmapheresis dubbed “Pulsed Plasma Exchange,” or PPE, the patient receives treatment once a week for four weeks, rests for eight weeks, and repeats; in doing so, the red blood cells are systematically de-clumped, thereby arresting disease progression without the risks and side-effects that can come with immunosuppression. Some patients on the protocol have even gone on to report complete remission from active disease, provided they keep up with their treatment protocol. It’s by no means a perfect solution: advanced fibrosis characteristic of late or end-stage Scleroderma can’t be reversed, patients can experience anemia and low blood pressure from treatments, and–perhaps most importantly–patients must undergo PPE indefinitely in order to continue seeing benefits. But the treatment shows clear and considerable promise as evidenced by prior research, and fortified by a large-scale clinical trial could offer patients a safe, effective alternative to immunosuppression.

But there’s a snag. That’s where you come in.

Though some major insurance coverages like Medicare, Aetna, and Blue Cross Blue Shield have begun to list Therapeutic Plasma Exchange as a covered procedure for systemic sclerosis, many insurance coverages like Cigna, UHC, and commercial generic carriers consider the procedure experimental. Physicians, similarly, demonstrate a widespread lack of awareness of the procedure’s efficacy, often citing infection risk or lack of compelling evidence among their reasons for dissent. Patients report having to doctor-hop to find a physician aware of this procedure’s use and willing to prescribe it, and even report having to resort to using concierge and pay-to-play doctors to secure a TPE prescription, making TPE not just logistically challenging to arrange, but inordinately cost-prohibitive.

All of these barriers are due in large part to the absence of a large, well-executed one-year clinical trial by a major player in the rheumatological sphere. Though patient anecdotes and assorted smaller-scale trials have yielded positive results in arresting disease progression in early and mid-Scleroderma, widespread physician awareness and insurance coverage will not be realistically achievable without at least one large-scale study reflecting positive results. TPE has become a white whale for Scleroderma sufferers, who are too sick, too exhausted, and too inundated with medical debt to doctor-hop and arm-wrestle their insurance carriers on the razor-thin chance of getting TPE prescribed and approved. A large-scale clinical trial is the only way to give PPE staying power and a realistic shot at becoming a mainstream treatment modality.

This is the part where I need your help. I’m drawing up a letter, a petition, and some assorted research to submit to Johns Hopkins, calling for a large-scale clinical trial of TPE/PPE for systemic sclerosis, ideally wherein patients in early-to-mid-disease undergo treatment for one year. With a clinical trial proving the treatment’s efficacy, this widely-sought but rarely-won treatment will have the scientific backing that will bring TPE and Pulsed Plasma Exchange to the forefront of Scleroderma treatment by boosting physician awareness and securing better, more comprehensive insurance coverage.

Ultimately, my downstream goal in securing a large-scale clinical trial is that it can be used to petition the American Society for Apheresis to change their designation of TPE for SSc from “Level III – Optimum Role of Therapy Not Established” to “Level II – Second-line Therapy.” This change in designation from an organization at the epicenter of apheretic medicine would be transformative, morphing PPE from a quasi-experimental treatment for SSc to a mainstream, clinically-indicated treatment, to be pursued alongside or instead of immunosuppression depending on the patient’s clinical status. Without a sizeable clinical trial, ASFA will not consider re-designating TPE for SSc. And without your help, I may not be able to convince a Scleroderma Center to conduct a clinical trial.

If you believe that this treatment deserves its own clinical trial, please sign your name and share with friends, family, and other patients. Thank you so much for your time and for your help; I really believe that together we can raise awareness about this treatment and help make it a feasible, easily accessible option open to patients who are interested in trying it.

If you’re looking to learn more about TPE/PPE for systemic sclerosis, check out the Therapeutic Plasma Exchange and Research Articles sections of the Scleroderma Education Project’s website: https://sclerodermainfo.org/research/therapeutic-plasma-exchange/

https://sclerodermainfo.org/research/research-articles/

Note: I am not affiliated with the Scleroderma Education Project or with Ed Harris. I’m a UCTD-at-risk-SSc patient, writer, and healthcare worker looking to use whatever skills I have to make whatever difference I can. Please consider helping me in that mission.