Understanding Ferriprox

Ferriprox-deferiprone

There is some confusion when it comes to understanding Ferriprox and superficial siderosis. Ferripriox (Deferiprone) is a prescription drug whose primary purpose is treating people who have transfusional iron overload due to Thalassemia syndromes. Deferiprone was designed to bind to this iron and remove it in a process called Chelation therapy. It’s my understanding if a person suffers from iron overload they feel better when chelation lowers iron levels. Superficial siderosis patients do not have too much iron in their bloodstream. They have iron deposits (hemosiderin) stuck to areas of their brain (most often in the cerebellum or spinal cord) that are the result of blood infiltrating into their central nervous system from a trauma. An accident, surgery, stroke, etc., but somehow blood was introduced into their spinal fluid. The average person will naturally absorb and remove this blood. A rare few people will not be able to remove it. This iron is toxic to nerve function and ultimately can end in nerve death. There are many other prescription iron chelation medications on the market. Studies have shown Ferriprox as the only prescription drug, at this time, able to cross the blood-brain barrier so it might have a chance at removing hemosiderin…

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The Results Are In

Ferriprox

“Does Deferiprone provide a clinical benefit to the superficial siderosis patient?”   If you remember, the April 2017 edition of The Neurology Journal gave us an early peek at what the study findings might be in the final revision of Two-year Observational Study Of Deferiprone In Superficial Siderosis¹. The study was released this past December 28th, and the results have been published. This blog post is our cliff note version of the study, keeping in mind, we’re not medical researchers or doctors.   Initial recruitment included 48 participants.   Nine withdrew from the study because their insurance wouldn’t cover the off-label use of Ferriprox and one for other reasons. 38 people began the investigation, and over the course, two dropped out because of neutropenia concerns, four dropped for cost issues and one left for unrelated health reasons. 31 people completed the study. Each participant submitted a current neurological exam, a baseline MRI and prescribed a beginning dosage of 1,000 mg of Deferiprone taken twice daily on a five day on two days off dosage cycle. Adjustments were made to the dosage schedule for those with fatigue complaints to 500mg in the morning and 1,500mg in the evening.   All participants…

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Is My Ferriprox Working?

MRI Superficial Siderosis5

  First of all, we know the mind of Superficial Siderosis patients is full of questions. But if you’re one of the few who uses Deferiprone you want to know is my Ferriprox working? The original Pilot Saftey Trial of Deferiprone by Dr. Levy and Dr. Linas offered the first evidence chelation using Ferriprox could be successful without severe side effects. A longer term observational study began March 2012. Clinicaltrials.gov: Phase IV Observational Study of Deferiprone (Ferriprox®) in the Treatment of Superficial Siderosis. The estimated completion date is this month (Dec. 2017) with data collection ended in March 2017. Study Study This study expanded the number of participants to 38. The average age of the subjects was 64 years old, the youngest 37 and the eldest 86. Just under half of the participants were female (47%) and 94% were Caucasian. All participants had to show evidence of Superficial Siderosis in addition to following the standard treatment dosing protocol: 1000 mg of  Ferriprox twice daily ×5 days per week. First, each participant submitted a baseline brain MRI. Blood test results recorded weekly. A final MRI in when the data collection period ended. The research was a two-year longitudinal study which means observational only.…

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Testing, Testing, Hope

Test Hope

The Whirlwind Test Tour We’ve been doing the whirlwind testing tour with our many specialists these past few weeks. Not only was it time for our quarterly neurology follow-up but we found ourselves eagerly waiting for the first MRI and hearing tests since starting on Ferriprox this past year. Our health insurance network is being changed from a PPO next year to an HMO, so we suffered few weeks of panic after we received notice from BCBS of Texas. HMO networks are not popular with doctors in our rural area. Seven specialists and one PCP currently provide care for Gary, and no one was in an HMO network. UPDATE: Every doctor but the neurologist joined an HMO network before January.   First Stop   Our first stop was the audiologist. Dr. Lee had mailed us a copy of a  magazine article this summer from one of her professional publications. It outlined audiologists now being asked to screen patients who present with a sensorineural hearing loss. Identify case history, determine if an MRI and neurological referrals were necessary. The article then describes a diagnosis of a rarely seen neurodegenerative condition called Superficial Siderosis, its effect on hearing loss and the importance of early diagnosis.…

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Superficial Siderosis : Education and Standard of Care

treatment protocol

Superficial Siderosis physician education and a Standard of Care policy are two areas that need work. Doctors touch on superficial siderosis during their medical training. But it presents so rarely during their everyday practice it’s no wonder a diagnosis is either considered inconsequential or untreatable. Even when presented with new research some physicians feel reviewing fresh data is not a priority. Need to know. If you’ll never have to treat it why bother? In the United States, with any other serious condition, there is a national standard of care for your particular diagnosis. A documented clinical guideline for your physician to follow. No matter where you live geographically, your physician should have access to a standard treatment protocol. In their defense, we now have a treatment option that was not available five years ago. The progression of Superficial Siderosis is better understood now. The knowledgeable physician can now offer hope of a prolonged and comfortable quality of life for their patient. The first problem is Ferriprox. Many doctors seem to consider it nothing more than experimental. When you do find a provider willing to prescribe it dosage protocols vary between doctors so much you can’t possibly track its effectiveness. Secondly, whether you…

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Understanding Ferriprox Part Two

Ferriprox-deferiprone

Understanding Ferriprox Part Two   Trying to remove superficial siderosis associated hemosiderin deposits with Ferriprox (deferiprone) will be an unfamiliar treatment option to your doctors. You need to gather as much reliable information as you can. Having information packets available when you meet with a new physician can save valuable time.  Step one: track down the open research online, download and print out multiple copies.   We passed out this research to every doctor along the way. We were able to present a credible, study-backed argument for trying Ferriprox. Our first meeting was the neurosurgeon who initially ordered the tests that discovered the siderosis. He emailed Dr. Levy and after review of the protocol felt neurology better suited to oversee care. We began the search. Step Two: Find a neurologist   While we were trying to find a neurologist, we returned to our PCP, presented our information packet and made our case for beginning chelation as soon as possible.  He studied our research before we returned for our follow-up meeting. He agreed with our conclusion; if we wanted to slow decline progression, this was our only choice. Our PCP was honest in telling us he felt he was uneasy overseeing…

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Chipping Away: Ferriprox

depression

Chipping Away: Ferriprox   Starting Down The Chelation Road   It was a big day when the first bottle of Ferriprox arrived at our door. Our hematologist had laid out what would be happening during chelation in some pretty easy to understand terms: “Picture the iron that is covering the areas inside your head as a rust that has been building up on the inside of  your head coating parts of your brain and nerves. For chelation to work the drug has to be able to cross the blood-brain barrier. It’s hard and will take awhile to go away.” The protocol, recommended by the manufacturing company All SS patients who are on chelation therapy with Ferriprox have regular blood tests run to track their neutrophil levels and liver function. There have been rare instances of other patients developing agranulocytosis. Agranulocytosis is a deficiency of a type of white blood cell whose purpose is to fight off infection. If your neutrophil level drops too low, you need to cycle off the medication until it stabilizes. One suggested medication dose schedule is taking your twice daily dosage for five days and cycle off for two days. Ferriprox removes iron from everywhere in…

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