I am pleased to announce that as of this past Friday, there are now two patients who have started one-year, individual trials of Therapeutic Plasma Exchange (TPE) using the protocol that my research group developed as a starting point for clinicians planning individual trials of TPE. Note that this is not a formal study but is designed so that we may be able to pool the patient data as part of a case series after the initial one-year trial period. You can view these Guidelines here in both US and A4 formats:
For HIPAA privacy reasons, I can’t tell you much about the patients. What I can tell you is that both patients are relatively early stage, one is in Europe and the other is in the US, and both have rare scleroderma-specific antibodies. One has U3-RNP antibodies, the rare, third antibody associated with diffuse systemic scleroderma. Most patients with diffuse systemic scleroderma have either Scl-70 or RNA Polymerase III antibodies. There is one published case report where TPE was used to treat a patient with U3-RNP antibodies. The other patient has Th/To antibodies, the rare, second antibody associated with limited diffuse scleroderma. Most patients with limited systemic scleroderma have centromere antibodies. There are no published reports of anyone trying TPE to treat a scleroderma patient with this antibody.
Since our comprehensive review of the use of TPE as a treatment for systemic scleroderma has not yet been published (in press), you may wonder how these two patients were able to arrange to start these clearly experimental trials. It starts with very educated and assertive patients. Both patients read everything on this website about TPE and the research behind this treatment approach. They then contacted me with questions about the procedure. Once they reached the decision that this was something they were interested in considering, the next “challenge” was getting their clinicians to also become educated on TPE and ultimately convinced that this made sense to consider for them. In the case of the European patient, that was relatively easy since TPE is much more commonly used there than in the US. As a result, the clinician didn’t have the typical (mostly incorrect) concerns about issues such as safety. In the case of the US patient, the clinician was very open to learning about TPE and the research behind it and was ultimately willing to move forward to see if this could be done. In each of these two cases, a member of the Scleroderma Education Project Medical Advisory Board and I participated in video conference calls with both the patient and clinician to answer any remaining questions/concerns.
The final challenge was cost. Here we lucked out a bit. The patient in Europe is going to self-pay for the first year (TPE is much cheaper in Europe than it is here) and if successful, work towards getting insurance coverage to continue long-term TPE. In the case of the US patient, his/her company is self-insured and willing to support this one-year trial.
I am talking with both patients about their giving periodic, anonymous updates over the next year about their experiences with TPE (good or bad) through this website. I will let you know if I am able to work this out.