Beware Of "Off-Label" Drugs

Hello All,

 
I posted this on our PPN Support, Education and Advocacy Group today. I wanted to share this information once again with those who have PP or anyone who has a family member with PP. It is an important message, which I have written about several times before.

We are a patient advocacy group as well as a support group and educational group. It is our duty to provide information to keep each of you informed, safe and to be the "best you can be." For that reason I am passing along this information once again.

It has come to our attention that someone we know with PP, who is not genetically diagnosed, was placed on the known medications typically prescribed for PP sometime back. This person has had no end of serious issues and complications connected to this. So, I wanted to pass this information along once again.

Some people do well on those medications and we are not telling anyone to stop taking them if that is the case. However, others do not do well and/or they may develop side effects from these drugs which can be harmful and can become life-threatening. If that is the case, there is more information here for you to consider. If you have been given a prescription for one of them, you may want to read the information here before you start it, so you will know what to expect or what to look for, in order to be safe.

Periodic Paralysis is a mineral metabolic disorder (ion channelopathy). There are NO drugs recommended/approved by the FDA for Periodic Paralysis. The ones typically used, that we know about are strictly "off-label" (made and used for other conditions).

From: https://www.rarediseasesnetwork.org/cinch/learnmore/faqs.htm

"Q: Are there drug treatments for periodic paralysis or the myotonias (PMC)?"

"There are no FDA approved treatments for periodic paralysis or the myotonias. Any medications currently prescribed for these disorders are used "off-label". "Off-label" means the drug is approved to treat another condition/disease, but was not scrutinized under the quality control protection of the FDA's clinical trials process for treatment of myotonias or periodic paralysis".....

This is the complete blog:

http://livingwithperiodicparalysis.blogspot.com/2014/06/beware-of-off-label-drugs.htmlhttp://rarediseasesnetwork.epi.usf.edu/.../learn.../faqs.htm :

"Q: Are there drug treatments for periodic paralysis or the myotonias (PMC)?"

"There are no FDA approved treatments for periodic paralysis or the myotonias. Any medications currently prescribed for these disorders are used "off-label". "Off-label" means the drug is approved to treat another condition/disease, but was not scrutinized under the quality control protection of the FDA's clinical trials process for treatment of myotonias or periodic paralysis....."

While researching this I found out some interesting information about some forms of Hypokalemic Periodic Paralysis and the use of those drugs. Some forms do not respond to them and some cause paralysis and/or worse symptoms:

 A study from 2001 relates two different types in particular but I am sure more have been discovered by now: CACNL1A3=Hypokalemic Periodic Paralysis-1 and SCN4A=Hypokalemic Periodic Paralysis-2, each with several mutations...and of course there are the ones that have not been found yet.

When someone is diagnosed with Hypokalemic Periodic Paralysis, clinically, (based on their symptoms), one must be very careful. One form either does not respond to diamox (acetazolamide), causes more paralysis or causes serious symptoms. So, diamox should not automatically be given to people clinically diagnosed with Hypokalemic Periodic Paralysis or if it is, it should be used with extreme caution.

From the article:

 “In conclusion the present study demonstrates that genetic characterization of HypoPP patients is important to decipher the clinical and histopathological features of the disease, and to predict the response to therapy. We suggest that mutations in the SCN4A gene should be systematically sought in HypoPP patients suffering from paralytic attacks followed by myalgias or worsened by acetazolamide, and when muscle biopsies reveal the presence of tubular aggregates.”

 http://brain.oxfordjournals.org/content/124/6/1091.full.pdf

About half of us do not have a genetic diagnosis and do do know what form of PP we actually have. If we are going to take these medications, we must be very careful and closely monitor our symptoms and the side effects.

For more information about this subject you may want to read in our files:

"Why we discourage discussion of diamox and acetazolamide.dot · version 1"

"Cautionary Information about Acetazolamide and Diamox for Individuals With PP.dot · version 1"

"Information on diamox.dot · version 1"

Or on the blog:

http://livingwithperiodicparalysis.blogspot.com/2014/02/some-forms-of-pp-worsened-by-diamox.html

http://livingwithperiodicparalysis.blogspot.com/2013/12/periodic-paralysis-and-metabolic.html

Again, we are not trying to tell anyone to stop taking any medications. If they are working and there are no side effects, then there are no problems. If, however, these meds may be causing side effects or if you are trying to decide whether to take them or not, hopefully this information can help you to make an informed decision.

Hugs to you all...