Serene Forest

Showing posts with label advocacy. Show all posts
Showing posts with label advocacy. Show all posts

Saturday, June 21, 2014

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.”




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:









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...

Saturday, June 14, 2014

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...





Thursday, December 19, 2013

Finding a Doctor Who Cares



Hello All,

I had a great day yesterday. I was recovered enough to run errands and grocery shop with Calvin. We had a great time and when we got home and I did well except for some weakness and dizziness in the evening. I was very excited when a package was delivered in the afternoon. It was my first copy of my second book, which I actually wrote many years ago, but finally was able to publish it in paperback. It is about my daughter Sandy, who was born with Sotos Syndrome. She passed away at the age of 5. Her life and death touched me in many ways and prepared me for living with my own disabilities. It was a wonderful early Christmas gift.



Finding a Doctor Who Cares
“NOT AN EASY TASK BUT POSSIBLE”
So many people with all forms of Periodic Paralysis struggle with locating doctors who can and will help them with both a diagnosis and proper treatment. They go from doctor to doctor and disappointment after disappointment, misdiagnosis after misdiagnosis and mistreatment after mistreatment. This can go on for years with the misinformation following them from physician to specialist. Often, this will lead to a diagnosis of hypochondria, malingering or the archaic “conversion disorder” and a referral to a psychiatrist. Many of the patients become depressed and begin to doubt themselves after being prescribed medications for mental disorders that invariably make the symptoms worse or different, thus creating the need for yet another referral to yet another specialist.

This vicious cycle can continue for years as the symptoms worsen and the patient becomes more disabled and debilitated. Family and friends tire of dealing with the situation and many friends are lost, marriages end in divorce and family members withdraw their support. The patient is left in more pain and despair and the humiliation can be unbearable. Many are never diagnosed and never receive the treatment they need and deserve.

I myself saw 30 different physicians in 6 years before I was diagnosed at the age of 62 after a lifetime of illness, disability and loss of friends, family and a marriage. For the most part they were rude and did not understand what was happening to me. Most of them were frustrated and believed it to be all in my head.
 
At one time,
I was trying to find a new doctor because my previous doctor moved out of town suddenly. The new doctor I chose told me on my first and only visit, "You are too sick, I will not take you as a new patient". I started crying and cried all the way to the insurance office. I reported him and was told that he did not have the right to do that. They called it "cherry picking",  but they did refer me to another doctor.

After my diagnosis I have seen twelve more doctors, all of who do not know how to help me, including one who is a specialist in Andersen-Tawil Syndrome. I have been in contact with a physiologist who studies ion channelopathies who would like to help me and my family but is not sure how. I have spoken with one of the neurologists who misdiagnosed me, who has now apologizes for missing some important findings in my labs and the opportunity to diagnose me two years earlier.
 
I also was able to finally locate a few doctors who have been willing to work with me (two more left town after seeing them for a short while) and have read the information I provided for them about Periodic Paralysis and done some research on their own. They do not deal directly with my PP symptoms, however, but with the things they know about, such as, diabetes, oxygen therapy and referrals to specialists as I need them.

After all of my experiences with finding doctors who will work with me, I have devised a common sense plan that can assist anyone to locate a doctor (Primary Care Physician: PCP) who will be willing to work with them before they ever step into the physician’s office. There will be no more insults from a person who should be showing compassion and no more leaving the office in tears and despair.

1. The most obvious place to begin your search, if you have a good doctor who has decided to move on in his or her career, is to ask your present doctor for the name of a physician who knows about periodic paralysis or who would be willing to work with you. If he or she gives you a referral, be sure to have them confer with the new doctor about your disease and provide him with as much information as possible before your first visit. You may also want to provide information of your own ahead of time.

2. If you are not that lucky, the next thing you can do if you have insurance, is to call your insurance company and request a “patient advocate” or “case manager”.  Most insurance companies have employees whose job is to help their clients who have “more than the average” or “out of the ordinary” medical needs.

”A patient advocate is someone who works on behalf of patients and their families during stays in the hospital or other medical situations. Many different organizations employ on-site advocates to deal with the daily needs of patients… The patient advocate's primary function is to serve the patient's needs, along with the needs of their family.”


Once an advocate is assigned to you, you will need to explain your situation and Periodic Paralysis and explain your need to find a doctor who knows about the disease or who will be willing to work with you. It would be wise to seek out neurologists, internal medicine doctors and endocrinologists. You may need to see more than one doctor before you find the “right” one for you.

3. If your insurance company does not have patient advocates and has a restrictive list of particular doctors and specialists covered in their program, again, you will need to explain your situation and Periodic Paralysis and explain your need to find a doctor who knows about the disease or who will be willing to work with you. They will sometimes do the work for you.

If not, you can go through the lists of neurologists, internal medicine doctors and
endocrinologists and call each office and ask for the office manager. You will need to explain your situation and Periodic Paralysis and explain your need to find a doctor who knows about the disease or who will be willing to work with you. In most cases, the office managers will speak to the doctor or doctors about your case and get back with you if the doctor is willing to see you.

4. If your insurance is not restrictive, you will have to check your local phone book or the web for neurologists, internal medicine doctors and endocrinologists in your area and proceed with the phone calling until you find one who will work with you.

Another good option, if your insurance is not restrictive and requires no referrals, is to check out the clinics in your area. The one I attend has 85 doctors and are connected to a local hospital. There should be at least one doctor willing to work with you.

You can proceed as explained previously; call and speak with one of their representatives. Explain your situation. The representative will go through the list of their physicians and chose a few that may work for you. The doctors will be consulted and one or two may agree to see you. It may be wise to spend time with each to decide the “best fit” for you.

5. Without insurance, seeking out help from your local health department can be productive. Also your local services for disabled will have some possible options for finding a local doctor. You may be able to secure a social worker. He or she may do the work for or with you.

6. Thinking “outside of the box” can bring some surprises. I was so frustrated and about to give up on ever finding a local doctor who could diagnose and treat me, when I had a wild idea.

One of our local television stations offers a wonderful service every Tuesday evening during their two-hour news coverage. A local physician, and a guest specialist of her choosing, take calls from viewers and answer medical questions.

I called and asked if she knew about Periodic Paralysis. I was shocked when she told me she had a patient who had it and that the patient saw a local neurologist who treated her. She gave me the name of the neurologist. I made an appointment and after two visits was diagnosed! (I must explain that I had all of my medical records in hand with years of medical testing ruling everything else out and a referral from my nurse practitioner.)

7. The next option I offer must be used with caution. You may search the web for specialists. Seeking out these specialists in the field of Periodic Paralysis or Andersen-Tawil Syndrome, may lead you to some severe disappointment. There are several across the United States and a few around the world. Many do not see patients and are involved in research only.

The specialists will only diagnose based on genetics or a very “pure” form of the disease. (This may be based on the fact that most of them are researchers and their funding is based on working with only those who are genetically diagnosed. This leaves out a high percentage of us.) Their view can be extremely narrow and I have been surprised that their knowledge of the disease can be severely lacking in some areas. Their only option for treatment is limited to medications that do not work for many of us. If you have other conditions co-existing with your Periodic Paralysis or do not have a known genetic code, you will be sent packing in tears and humiliation. It is not worth the time or money you may spend to travel to another state or country.

Recently, I know of several individuals who had symptoms of Periodic Paralysis. They went to great expense to travel to the “specialists” only to be told that they absolutely did not have Periodic Paralysis. Within a few months, genetic testing proved that they did indeed have variants of Periodic Paralysis.
 
8. The Muscular Dystrophy Association (MDA) is an organization that treats patients with muscle diseases, and lists all the forms of periodic paralysis under that umbrella. In order to see their doctors you must already have a diagnosis or be referred for a diagnosis by a doctor. However, most of the doctors we have information about at the MDA Clinics, do not know about Periodic Paralysis or Andersen-Tawil Syndrome. Most offices around the country do not know about Periodic Paralysis. If you call for a referral or information, you will probably be told that they do not know what you are talking about. I have had to call many MDA offices around the country for patients to tell them that they do indeed treat Periodic Paralysis and Andersen-Tawil Syndrome patients. I refer them to this information at their own website: http://www.mda.org/disease

Also, although you may already have a diagnosis, you must see their doctors and be re-diagnosed before you will receive any treatment or benefits offered. If the MDA doctor does not agree with your previous doctors due to their lack of knowledge of the disease or their narrow view based on old facts and research, you may lose your diagnosis. It is not worth the chance. That being said, there are some very good MDA doctors and some of our friends with Periodic Paralysis are having some success with the ones they are seeing.


Conclusion:

Many of the people with genetic codes that have been located do have good doctors and receive good treatment and proper medication for their particular forms of Periodic Paralysis. They are very lucky. The medications help them and they can lead nearly normal lives as well.

I understand, however, it is difficult to find a doctor who will work with those of us who suffer from the effects of Periodic Paralysis, if we have not yet been diagnosed or those of us with variants for which no genetic code has been discovered yet, even if we have been diagnosed clinically (based on symptoms) or for those of us who have other diseases which co-exist with our form of PP. The truth is, very few of us will get any real help from a doctor even if we find one who knows about the disease and is kind, sympathetic and empathetic. This is due to the fact that most of us are unable to tolerate the known medications and the doctors do not know how to help us.

 So, although I have a Primary Care Physician and several good specialists, Calvin and I are still left to deal with my episodes of paralysis and my other symptoms with no real help from the medical field. They do not know how to help me, but treat me well and look after the things they can; like my heart problems, power wheelchair, oxygen, diabetes strips, labs, etc. I appreciate and understand their lack of knowledge of such a rare and baffling disease. They trust us with the plan we have created after much research and trial and error.
 
We have created a website and a forum with several support and discussion groups and this blog and we have written a book “Living With Periodic Paralysis: The Mystery Unraveled” (11/28/2017...and now three more books) to pass along this information and hope to help others in a way the doctors are unable to at this time.

I “walk the tightrope” daily to avoid episodes and the serious symptoms they create.  This plan is outlined on our website and in our book and is discussed on our support and education group.


Until later...