KevinM
Senior member
- Joined
- Mar 30, 2019
- Messages
- 559
- Reason
- PALS
- Diagnosis
- 06/2019
- Country
- US
- State
- FL
- City
- Tallahassee
The benefits and side effects of Riluzole have been discussed at great length here and on other forums over the years. However, I continue to be surprised at the number of pALS and many neurologists still citing results from the original studies that showed only a very modest life extension of about three months, and that occurring in the later stages. Much more recent studies have shown that the effects are much more robust, which was first brought to my attention by my neurologist.
This from the 2017 NIH study: “The original analyses, and subsequent meta-analyses, of data obtained from randomized controlled trials (RCTs) suggest that riluzole typically extends survival by 2–3 months and increases the chance of an additional year of survival by ~9%. However, published real-world evidence (RWE) from 10 clinical ALS databases indicates that riluzole therapy may afford much greater extension of survival, and improvements in median survival times of more than 19 months have been reported in the overall ALS patient population.” If all neurologists were citing this analysis, I believe the reputation of the drug as being basically useless would change.
Given the old estimates many pALS still use in evaluating cost/benefit, they choose not to take it because they feel the side effects far outweigh those almost nonexistent benefits. And it is true that some simply cannot tolerate the drug. But the main side effect generally cited is the fear of liver damage, which certainly can occur. Many neurologists suggest blood draws every three months to check on liver enzymes, and that is certainly warranted, at least for the first year or two.
But I also think there pmay be an assumption by many pALS that this is a very common occurrence, which is not true. A 2012 study published by NIH indicated that a little more than one out of ten users (+/- 12%) experience elevated numbers. “These elevations are usually mild-to-moderate in severity and are rarely associated with symptoms. Most elevations resolve spontaneously, but persistent or marked elevations require drug discontinuation or dose modification.” Less than 3% experience the seriously high numbers—greater than 3x the normal range.
A 2020 study of Japanese patients indicated a much higher incidence of mild-moderate increase, about 50%. I think it is important to recognize that persons of Asian descent have a much higher incidence of liver cancer and other disorders which could potentially skew the data, and this study also included several risk factors such as smoking that increased the risk.
I totally get that any treatment, approved or otherwise, is a personal choice, it won’t work at all for some, and any ALS therapy analysis is rife with statistical land mines. But I do think neurologists in general and neuromuscular specialists in particular need to include this more recent data to help with the cost/benefit decisions we each face.
This from the 2017 NIH study: “The original analyses, and subsequent meta-analyses, of data obtained from randomized controlled trials (RCTs) suggest that riluzole typically extends survival by 2–3 months and increases the chance of an additional year of survival by ~9%. However, published real-world evidence (RWE) from 10 clinical ALS databases indicates that riluzole therapy may afford much greater extension of survival, and improvements in median survival times of more than 19 months have been reported in the overall ALS patient population.” If all neurologists were citing this analysis, I believe the reputation of the drug as being basically useless would change.
Given the old estimates many pALS still use in evaluating cost/benefit, they choose not to take it because they feel the side effects far outweigh those almost nonexistent benefits. And it is true that some simply cannot tolerate the drug. But the main side effect generally cited is the fear of liver damage, which certainly can occur. Many neurologists suggest blood draws every three months to check on liver enzymes, and that is certainly warranted, at least for the first year or two.
But I also think there pmay be an assumption by many pALS that this is a very common occurrence, which is not true. A 2012 study published by NIH indicated that a little more than one out of ten users (+/- 12%) experience elevated numbers. “These elevations are usually mild-to-moderate in severity and are rarely associated with symptoms. Most elevations resolve spontaneously, but persistent or marked elevations require drug discontinuation or dose modification.” Less than 3% experience the seriously high numbers—greater than 3x the normal range.
A 2020 study of Japanese patients indicated a much higher incidence of mild-moderate increase, about 50%. I think it is important to recognize that persons of Asian descent have a much higher incidence of liver cancer and other disorders which could potentially skew the data, and this study also included several risk factors such as smoking that increased the risk.
I totally get that any treatment, approved or otherwise, is a personal choice, it won’t work at all for some, and any ALS therapy analysis is rife with statistical land mines. But I do think neurologists in general and neuromuscular specialists in particular need to include this more recent data to help with the cost/benefit decisions we each face.
