The Results Are In


“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 Siderosis

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. 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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Hemosiderin in the Cerebellum

Multiple Angioma Cerebellum Sagittal Siderosis

Putting A Face On The Enemy We’ve all read the descriptions of how hemosiderin deposits (Iron Salts) form. Do you sit looking at the MRI scan results wondering just where we are supposed to be looking? Nothing explains it better than a picture. When you look closely at this photo of hemosiderin in the cerebellum, you can see how thick the deposit is. It makes sense why researchers think it will take years for your iron buildup to begin to lessen. Deferiprone has to journey from your stomach to the upper G.I tract and into your bloodstream. Next, what doesn’t bind to the iron from your last meal will travel through the blood-brain barrier and into your spinal fluid. The molecules target free iron from the hemosiderin and help carry it out of your body via your urine. The process is indeed a wonder of modern medicine.  

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Following The Low Road:Low Iron Diet


Giving Ferriprox A Chance   When we decided to give Ferriprox a chance, we weren’t aware how hard getting your health insurance company to approve a tier 5 drug for off-label use is. Luckily, we have a hematologist experienced in gaining approval for high-level medications for his patients. Five months passed from when Gary was first diagnosed until the first bottle of Ferriprox arrived at our doorstep. Excitement, hope, anxiety, and worry all packed into one little box.   “Because Ferriprox will be used for years in most patients, we came up with a schedule to avoid iron deficiency. During the week when you’re taking the medication, you should avoid iron-rich foods and vitamin C 2 hours before and after taking the medication. Vitamin C-rich foods include tomatoes, grapefruit, and oranges. On the weekends, to replenish some of the lost iron reserves, eat a steak or something rich in iron. But not iron tablets. Iron tablets take a long time to digest (days) and will interfere with absorption of Ferriprox.” – Dr. Michael Levy   We decided to follow Dr. Levy’s recommendations because we felt an obligation to make the most of every dose of Ferriprox.   Who Knew   Trying…

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