Alexander Zubkov, RajaNandini Muralidharan, George Petty, Jeffrey Winters.
Submitted July 05, 2011
The recent AAN guideline update on plasmapheresis in neurologic disorders [1] represents an expert review of controlled trials, revealing both a dearth of randomized controlled trials on the use of plasma exchange (PLEX) in the treatment of neurologic diseases and a focus on a limited number and more common neurologic diseases.
By limiting the current guidelines to controlled trials, there is a risk of overlooking rare neurologic diseases less amenable to the highest standard of evidence: randomized controlled trials with equivalent baseline groups. We observed 19 patients with Susac Syndrome over a 10-year period, 9 of whom had symptomatic benefit with PLEX. The heterogeneity of the disorder and the limited number of reported cases prevent robust assessment of the use of PLEX in this disorder. However, in our experience, patients with minimal or modest symptomatic response to IV oral steroids—sometimes including IV immunoglobulin— benefit from PLEX.
Given the devastating consequences of blindness, deafness, dementia, seizures, and other possible sequelae that may occur in this patient group of predominantly young women, PLEX was considered when other treatments failed. It was also administered to a pregnant woman in whom high dose steroids was deemed to be a less favorable treatment option.
We propose that the current recommendations for PLEX for the treatment of neurologic diseases also acknowledge that rare neurological diseases with a presumed autoimmune process be recognized as potentially treatable with PLEX. PLEX may also be beneficial in unique subpopulations of patients with otherwise common neurological diseases, such as pregnant women (current level of evidence: unproven or "U").
Because of the limited number of randomized controlled clinical trials of PLEX, the best available evidence should be considered in determining the recommendations for this treatment.
Disclosure: Dr. Zubkov received funding from
Sanofi-Aventis, Plavix Boehringer Ingelheim, Pradaxa Forest, Namenda Teva, Copaxon; serves on the Speakers' Bureaus for Sanofi-Aventis, Plavix Boehringer Ingelheim, Pradaxa Forest, Namenda Teva, Copaxon.
Dr Winters served as an investigator on research funded by CaridianBCT, Fenwal Inc, and Asahi Kasei Kuraray Medical Co. ; received funding from NIH grant #HL81027; serves as an Associate Editor for the Journal of Clinical Apheresis and is the president of the American Society for Apheresis. Dr. Mateen reports no disclosures.
The recent AAN guideline update on plasmapheresis in neurologic disorders [1] represents an expert review of controlled trials, revealing both a dearth of randomized controlled trials on the use of plasma exchange (PLEX) in the treatment of neurologic diseases and a focus on a limited number and more common neurologic diseases.
By limiting the current guidelines to controlled trials, there is a risk of overlooking rare neurologic diseases less amenable to the highest standard of evidence: randomized controlled trials with equivalent baseline groups. We observed 19 patients with Susac Syndrome over a 10-year period, 9 of whom had symptomatic benefit with PLEX. The heterogeneity of the disorder and the limited number of reported cases prevent robust assessment of the use of PLEX in this disorder. However, in our experience, patients with minimal or modest symptomatic response to IV oral steroids—sometimes including IV immunoglobulin— benefit from PLEX.
Given the devastating consequences of blindness, deafness, dementia, seizures, and other possible sequelae that may occur in this patient group of predominantly young women, PLEX was considered when other treatments failed. It was also administered to a pregnant woman in whom high dose steroids was deemed to be a less favorable treatment option.
We propose that the current recommendations for PLEX for the treatment of neurologic diseases also acknowledge that rare neurological diseases with a presumed autoimmune process be recognized as potentially treatable with PLEX. PLEX may also be beneficial in unique subpopulations of patients with otherwise common neurological diseases, such as pregnant women (current level of evidence: unproven or "U").
Because of the limited number of randomized controlled clinical trials of PLEX, the best available evidence should be considered in determining the recommendations for this treatment.
Disclosure: Dr. Zubkov received funding from Sanofi-Aventis, Plavix Boehringer Ingelheim, Pradaxa Forest, Namenda Teva, Copaxon; serves on the Speakers' Bureaus for Sanofi-Aventis, Plavix Boehringer Ingelheim, Pradaxa Forest, Namenda Teva, Copaxon. Dr Winters served as an investigator on research funded by CaridianBCT, Fenwal Inc, and Asahi Kasei Kuraray Medical Co. ; received funding from NIH grant #HL81027; serves as an Associate Editor for the Journal of Clinical Apheresis and is the president of the American Society for Apheresis. Dr. Mateen reports no disclosures.