We know most Superficial Siderosis patients are full of questions. If you’re one of the few prescribed the iron chelator, deferiprone, you want to know if chelation is working? The original Pilot Saftey Trial of Deferiprone, M.Levy, and R. Linas, offered the first early evidence chelation using deferiprone 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. With data collection ending, March 2017, the estimated completion date is December 2017.
The Expanded 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 and follow the standard treatment dosing protocol: 1000 mg of Ferriprox twice daily × 5 days per week. First, every participant’s physician submitted a baseline MRI of their brain. Then, researchers collected their recorded blood test results 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 use the Osirix software available at Johns Hopkins to develop a new procedure that would determine the exact amount of hemosiderin deposits.
Every participant’s baseline MRI and final MRI were run through a software program to render a three-dimensional whole-brain image from their gradient-echo series. The OsiriX medical image viewer was then used to compare the baseline vs. ending scans by dividing the mean T2 hyperintensity to the maximal cerebrospinal fluid signal.
An Early peek Into The Findings
One segment of the research showed 25% of the participants responded favorably to chelation with deferiprone. In addition, this group had an average increase in mean T2 hyperintensity of 1.4% (increase good), indicating the removal of hemosiderin. However, an average decrease in T2 hyperintensity of 10.4% (decrease bad) over the two-year study period signifying hemosiderin was still building up. In addition, 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 that the remaining 75% will eventually see a decrease in hemosiderin and enjoy a clinical benefit. This information comes to us from an early report published in the journal, Neurology April 2017¹.
¹Two-Year Observational Study of Deferiprone in Superficial Siderosis (P2.299) Remi Kessler, Kateryna Schwartz, Xu Li, Maureen Mealy, Michael Levy Neurology Apr 2017, 88 (16 Supplement) P2.299;