First of all, we know most superficial siderosis patients are full of questions. If you’re one of the few who is prescribed the iron chelator 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 in 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.
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,every participant’s physcian submitted a baseline MRI of their brain. Blood test results were recorded weekly and a final MRI was submitted when the data collection period ended. The research was a two-year longitudinal study which means observational only with data collection.
Deciphering The Data
The team was able to make use of the Osirix software available at Johns Hopkins to develop a new procedure which would be able to determine the exact amount of hemosiderin deposits.
The baseline MRIs and the final MRIs were put through a software program. Three-dimensional whole brain images of gradient echo images were rendered. The OsiriX medical image viewer was then used to compare before and after scans by dividing the mean T2 hyperintensity to the maximal cerebrospinal fluid signal.
Good Job Ferriprox
In any event, it doesn’t matter if we fully understand the hows. Ultimately what matters is they now have a way to tell if your Ferriprox is doing the job conclusively. This information comes to us from an early report published in the Neurology Journal, April 2017¹.
This segment of the research showed 25% of the participants responded favorably to chelation with Ferriprox. This group had an average increase in mean T2 hyperintensity of 1.4% (increase good) indicating the removal of hemosiderin. There was an average decrease in T2 hyperintensity of 10.4% (decrease bad) over the two year study period signifying hemosiderin was still building up. 26 of the 38 subjects reported improved neurological function, 12 indicated either no change, good or bad or they experienced reduced neurological function.
Our first peek into some positive results from the first long-term study. In the final analysis, it’s important to realize more time will be needed to continue the research. The hope is the remaining 75% will eventually see not only a decrease in hemosiderin but also enjoy a clinical benefit.