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...

"This Book Lead To My Genetic Diagnosis Of Anderson-Tawil Syndrome II": Book Review by Guest Blogger Tammy

Hello All,

Calvin and I wrote and published the book living with Periodic Paralysis: The Mystery Unraveled, last year. I have written much about, but recently a member of our Periodic Paralysis Network Support, Education and Advocacy Group, Tammy Collins-Levi, wrote a wonderful review on Amazon.com after purchasing it and reading it. She discussed how it changed her life. We have decided, with her permission, to share it with you today.

Thank you so much Tammy for writing this and allowing us to share it with everyone!!

This Book Lead To My Genetic Diagnosis Of Anderson-Tawil Syndrome II

If you or someone you love is fighting the uphill battle of getting a diagnosis, this book is for you. I had been sick for years. I became seriously ill 4 years ago. So many opinions with out any educated knowledge followed. I cannot begin to tell how much was lost during the past years of having a rare illness. But Susan speaks my story perfectly. I had already been to Orlando to meet with many others that suffer from Periodic Paralysis and had my genetic DNA sent to Germany. It seemed like all the doctors kept putting me off with waiting on my results from Germany. As some of you might know this can take years and with my heart problems it could of cost me my life. I am 38, happily married, and a mom of four. This rare condition almost took my life many times. I was left drained and with brain fog that was hard to see through. I was searching for answers and others who had been told this is not real!

I bought Susan and Calvin’s book and was shocked that other people have suffered so badly. I was overjoyed to find someone that had taken time to tell the story with hopes of helping others heal! I realized it was time I pull it together and go through my records. I pulled all the facts. That is what matters to any other doctor and makes them stop in their tracks. I pulled all the labs, test, EKGs, EMGs, and left the doctors opinions in the dust where they belong!! “Hit them with the facts” Susan you hit that one right on the head. I had heard this from others, but with no true advice about how to do this. You guys walked me through it step by step. The organized thought process to getting through all the red tape and nailing down what is being missed is the best I have come across. And I am a fighter for what was lost. I have done countless hours of research and spent a ton of money traveling around the country to figure out what stole my health. This book is the best I have found to address it all.

I meet with my geneticist on June 15^th and she took all my organized medical facts and gave me a 100% clinical diagnosis of ATS. She informed me my labs showed electrical heart dysfunction and I am scheduled to have a pacemaker and defibrillator in a few weeks. If I had given up she said I would of probably passed from sudden arrhythmic death syndrome. How sad that would of been for my big family that has fought right beside me to find answers.

To sum it up this book helped organize my foggy mind, lifted my spirits, and give me the drive to fight like Susan did to get a real diagnosis before it was too late. This book saved my life and opened up new doors to my future. My life is different, but at least I know what I am fighting now. That was a priceless gift that this book gave my family and I.

Read this book and follow the advice offered and it will lead you in the right direction. Because you have all been on a never ending roller coaster ride and it is time to get off. It is time to walk up to your fears and say it’s time to get this right this time. No more games. Your life is important! Remember it is your body that is failing and only you know how that feels. Don’t settle for a diagnosis that doesn’t fit or make sense.

Thanks Again Susan for standing up for us all!!!!!




Until later...

Changing Lives Naturally

Hello All,

It has been awhile since I have posted a new blog. Actually, it is because I have been doing so well and have been able to do so much more than I have in several years, that I have been catching up on lost time doing things I never thought I would do again.

I had been on a medication (mirapex) which I thought I could never get off of. I tried many times and my symptoms got worse each time I tried over the years and I thought it was really helping my restless leg syndrome. For my 2014 New Year's resolution, I decided I really needed to try to get off of it again. I really needed to know if it was causing the symptoms that continued to keep me weak, etc and unable to function. I tapered off of it for months and just lived with the awful effects of withdrawal. Once I stopped it completely, I still had some issues, but over time and with working on my diet and other little changes, I was suddenly better and had no more restless leg also!! The fog lifted, I no longer had the awful weakness and I have had no paralysis for three months!!!

I am ready to get back to work with my writing, but I still need to take it easy because I continue have exercise intolerance, blood pressure issues and heart arrhythmia issues. After all, I still do have Periodic Paralysis, but it is definitely under better control, all by natural means.

Today's blog is something I wrote awhile back on our Periodic Paralysis Support, Education and Advocacy Group. It is time again to post it on our board and on our blog. We make no apologies for our philosophy, which has changed and saved many lives. It is posted on our logo: "Changing Lives naturally":

Hello All,

Since we have had so many new members join in the past few weeks, we wanted to post this information again. Our group is a little different and unique compared to the other groups. Not everyone is aware of this when they join. We are a support group and an educational group and also an "advocacy" group. We are concerned with your safety and how you are being treated, by the medical professionals in your life.

Please read the following.....for your information.

"Since we have so many new members I wanted to review with everyone about what the Periodic Paralysis Network is all about. We have a forum with several different options for information and support. We have four discussion groups (support, genealogy/genetic, book discussion and medical professional) which are all private for obvious reasons. We have two pages....mostly for just reaching others who need to find our help and education. We have our original website with a great deal of information. And we have a blog which is open in order to reach everyone around the world (has a translator), including medical professionals. We also have written a book, "Living With Periodic Paralysis: The Mystery Unraveled." It has 444 pages of information, is fully indexed and and we use over 250 references. It took us three years to write.

This group is our support, education and advocacy group. We welcome everyone and anyone who has any form of Periodic Paralysis or who suspects they have it (also family members). We provide support to those who are diagnosed or attempting to get a diagnosis.

We are an educational group because we provide information based on research. We attempt to answer any and all questions with information we find/found through research. Many of the answers are also in our book and on our website and our blog. Some things are also found in the "Files" section of this page.

We are an advocacy group. We work toward the improvement of quality and safety in healthcare for everyone with Periodic Paralysis. We are a member of the Patient Advocate Directory!!! They are a world-wide network of individuals and independent organizations working toward the improvement of quality and safety in healthcare.

We are here to help you "be the best you can be". Many of us cannot tolerate the pharmaceuticals/drugs/medications which are typically prescribed and through research and experimentation, we have found natural ways to manage and ease the symptoms of Periodic Paralysis. We share them with you.

As advocates for you, we also pass along the information about the pharmaceuticals/drugs/medications which are typically prescribed for every form of Periodic Paralysis. We caution anyone on them or who is considering using them to be aware of the side effects and possible problems from them. We also caution everyone who has Periodic Paralysis to be very careful if you are taking any pharmaceuticals/drugs/medications, anesthetics for surgery and IVs.

Periodic Paralysis is a mineral metabolic disorder and must be treated as such.

Our focus is on educational resources and self-reliance. Our approach to treatment focuses on the self-monitoring of vitals and the management of symptoms through natural methods such as the elimination of triggers and awareness of proper nutrition and supplementation. This approach evolved from the inability of the medical community to provide appropriate medical diagnosis and treatment. We continue to do research and provide the latest information to our members."

Thanks,
Susan and Calvin

 Until later...