Superficial Siderosis and Chelation Therapy
Researching a rare disease on your own is tricky. Superficial siderosis is a serious diagnosis and most of the early research papers we found scared us to death. At first glance, the prognosis seemed horrendous. I discovered a paper online titled Pilot Safety Trial of Deferiprone in 10 Subjects With Superficial Siderosis-Michael Levy and Rafael Llinas ¹.
This small drug trial was the first positive piece of information. Gary wanted to begin looking into chelation as a therapeutic option so I printed out every reference I found about the iron chelator deferiprone.
Research studies are written in a language for fellow academics and physicians. Read enough of them, you begin to form a simple understanding of the data. I began to compile a hard copy folder of all the information I had collected covering the superficial siderosis prognosis and this new drug trial. We carried this folder to Gary's appointment follow-up with his primary care physician. Dr. Hozdic spent several days reviewing everything and agreed with us.
This brand new drug therapy was looking like Gary's only option.
In 2012 Deferiprone received approval in the U.S for use by superficial siderosis patients. Since Deferiprone was intended for management of iron overload in Thalassemia patients, using it for in superficial siderosis was designated off-label. Gary's PCP reviewed the possible side effects and felt it was critical to follow the manufacturer recommended schedule to monitor blood levels. Dr. Hozdic was honest, telling us he felt it would be better if he referred us to a hematologist to oversee this drug therapy.
The Hematologist
Two months later we met Gary's new hematologist and turned over our research folder. He was more than happy to accept Gary as a patient after reviewing his case and the proposed treatment protocol. Dr. Engstrom told us it was the type of unique case every good physician should love.
A small ray of hope was here at last.
He contacted Dr. Levy by email to confirm the protocol and prepared a request for our health insurance company. Deferiprone is a Tier-5 specialty prescription drug so being this was an off-label use request, we were told to expect delays and denials of approval. One month and one insurance appeal later we had received permission to start chelation therapy for a one-year trial period after which it would come up for review again.
Update November 2019
We had to say goodbye to Dr. Engstrom due to an insurance provider change but we landed with an equally caring hematologist, Dr. Philip Haddad. Gary began chelation in July of 2014 and continues today. Dr. Haddad has been candid about his early concern but Gary has tolerated Deferiprone for five+ years now.
This was the first piece I wrote when livingwithss.com was launched in May 2015. Time has brought me to a better understanding of the chelation process. I feel our expectations are more pragmatic with the realization this continues to be a long slow process. Five years may have uncovered new data and clearer understanding but, it also brings a new sense of urgency.
How can this chelation process be improved? Right now a research project is ready for funding. We just need to bring this home over the finish line.
