Sunday, April 13, 2014

An Epidemiology Study of BFS (Part II)


Why Publish a Survey Data Paper?

Most medical professionals have been trained to believe the only acceptable information for publication are studies consisting of a control group, clinical trial, or observation of patients. Here are the reasons this survey is acceptable for publication:

First, this paper is not statistically flawed like most medical research papers or clinical trials. One research paper estimates that 2 in 5 medical research papers are flawed due solely to an inadequate sample size. [9] This paper meets survey guidelines to meet a 95% confidence level for its data results because it has surpassed a sample size of 384. To dismiss this survey as insignificant is the same as saying a population of 25 million people are lying about their symptoms.

Doctors will question whether survey participants are being truthful and accurate with survey responses. This paper eliminates outliers – responses that are questionable and may not belong with the overall population in two ways. First, the survey questionnaire asks control questions to eliminate any participants that have not been officially diagnosed with BFS and or had an EMG or MRI. Secondly, a statistical analysis is performed on the survey data of each test to eliminate participants whose responses to the questionnaire are consistently outside +/- 3 standard deviations of the normal distribution.

Besides, most medical studies using “acceptable methods” are statistically flawed, and what’s worse most doctors that read these medical research papers do know they are flawed: “The increasing volume of research by the medical community often leads to increasing numbers of contradictory findings and conclusions. Although the differences observed may represent true differences, the results also may differ because of sampling variability as all studies are performed on a limited number of specimens or patients. When planning a study reporting differences among groups of patients or describing some variable in a single group, sample size should be considered because it allows the researcher to control for the risk of reporting a false-negative finding (Type II error) or to estimate the precision his or her experiment will yield. Equally important, readers of medical journals should understand sample size because such understanding is essential to interpret the relevance of a finding with regard to their own patients. At the time of planning, the investigator must establish (1) a justifiable level of statistical significance, (2) the chances of detecting a difference of given magnitude between the groups compared, i.e., the power, (3) this targeted difference (i.e., effect size), and (4) the variability of the data (for quantitative data). We believe correct planning of experiments is an ethical issue of concern to the entire community.” [9] The survey analysis for this paper follows these guidelines.

Secondly, we know very little about BFS so all the information in this writing is not only statistically and practically significant, but it is also novel regardless of no, weak, moderate, or strong correlation between parameters. Regardless of correlation status, this study contributes to the body of evidence related to this disorder. For example, there is no correlation between spine and neck injuries and symptoms in the legs, however this is good to know when evaluating future patients.

Thirdly, we must combat the fallacies about survey usefulness and accuracy and highlight inadequacies of controlled studies, clinical trials, and patient observation. Surveys are cost effective while controlled studies and clinical trials are in most cases cost prohibitive. Doesn’t it make sense to conduct a survey to find statistical and practical significance and correlation before moving on to an expensive controlled experiment or clinical trial? One would hope so.

Also, proximity is not an issue with surveys, they are global meanwhile controlled studies and clinical trials can limit the sample size to a more localized region. For instance, this study learned that stress related BFS is more statistically significant outside North America whereas people with BFS who have a family history of neurological issues are more likely to reside within North America. In fact, nearly one third of survey participants are located outside of North America. As a result, proximity sample size restrictions can hide relevant results. Observation and examination would miss this relevant connection and therefore can be overrated.

Observation and examination can be overrated for other reasons as well. In fact, observation of BFS patients has no practical benefit since most patients look normal and exhibit few symptoms that can be viewed – most are all internal. Doctors can only rate a BFS patient based on their response to questions – similar to a survey or questionnaire. Since doctors cannot see symptoms, for this reason, neurologists and doctors alike may falsely label BFS as benign. Also patient observation can also be influenced by stress. For example, my BFS symptoms and even my blood pressure are much higher every time I see my doctor due to anxiety.

In an anonymous survey the reviewer can only rely on the data and the statistical analysis – there is no human bias. However, in clinical research trials, bias and opinion are routinely injected in these studies by medical personal. One study on 21 clinical trials found “On average, non-blinded assessors of subjective binary outcomes generated substantially biased effect estimates in randomized clinical trials, exaggerating odds ratios by 36%.” [10] This can be eliminated if the clinical trial is double blinded and controlled.

There is also evidence that money used to fund clinical trials by industry and the government can bias outcomes. "Thus, although there is little direct evidence that industry sponsorship has led to deliberate skewing of the results or reporting, there are multiple cases in which industry and government sponsors have withheld important study results and in which the conclusions presented in the reports appear to overstate the study findings. The risk of undue influence in research exists." [11]

Fourth, some may argue my lack of a medical degree disqualifies me from publishing a medical paper in a medical journal. However, there is nothing medical about this paper. This paper is merely a thorough statistical or epidemiology analysis about a medical issue that we know very little about - BFS. In the discussion section there is an attempt to tie the correlation results of this paper to a few medical conditions tied to BFS in previous research.

Does the Data Make Sense?

Every person analyzing data for their study must ask themselves the question: Does the data make sense? This is a difficult question to answer especially regarding neurological disorders because no one really knows what the expected outcome will be. However, there are some control questions within this survey where the expected outcome of the question is more predictable. So it comes as no surprise there is strong correlation between symptoms such as muscle pain and soreness with cramps and stiffness; pins and needles are more likely to occur in the feet; stress exasperates stress induced BFS symptoms; exercise is more likely to exasperate exercise induced BFS; an illness is more likely to exasperate illness induced BFS; and headaches are likely to affect the head to name a few. These trends are what one would suspect, so we can conclude that people are answering the survey correctly. Many more of these logical trends can be found in Table 1 through Table 7.

Misconceptions of BFS

This paper also refutes a few myths about BFS. The first myth is that BFS is the same for everyone – easy to live with. For most individuals it is true, BFS is no big deal and easy to live with, but there are many chronic sufferers from this disorder that are ignored. Consider this, the average BFS patient in this survey suffers from muscle twitching (76% of the time), pins and needles (37.9%), cramps (35%), numbness (30%), muscle fatigue and weakness (42%), headaches (25%), itching (24%), muscle soreness and pain (47%), muscle buzzing and vibration (46%), and sensitivity to temperatures (32%). They feel these symptoms in their feet (56% of the time), lower leg (74%), upper leg (52%), back (34%), buttock (39%), abdomen (31%), chest (24%), head (38%), hands (46%), and arms (49%). The data suggests people in the survey feel on average 7.7 of 11 symptoms over approximately 80% of their bodies. From this data we can conclude that the people in this survey suffer from chronic BFS because they live with symptoms 24/7 over their entire body. What’s worse, the best remedies for relieving symptoms inebriate suffers – sleeping pills (2.85 out of 10) and benzodiazepine drugs (3.4 out of 10). None of this should be surprising since the people taking this survey belong to a BFS social networking site. People would only go to these forums if they have a chronic condition for which they cannot find any relief or answers. Besides, nearly 50% of the participants in the survey have had both an MRI and EMG and over 80% have had either an EMG or MRI. This is significant because only people with chronic fasciculation issues would be subject to such expensive testing to rule out other more serious neurological conditions.

This paper also refutes the claim that BFS is always “benign”. In medicine, benign is defined as “of no danger to health; not recurrent or progressive; not malignant.” However, the survey results show that only stress induced BFS patients (this is the most common form of BFS) can control their symptoms over time. Patients with BFS induced by anything but stress will see their symptoms get progressively worse over time. Since these patients symptoms are progressive, therefore by definition BFS is not “benign” in all cases. A better name would be Progressive Fasciculation Disorder (PFD) for chronic sufferers of BFS that is not stress induced.

Multiple Triggers or Causes?

There are nine suggested causes or triggers for participants to choose from in this survey. In many cases, people believe they have had more than one potential trigger over the course of their lives. For instance, with the author’s own experience with BFS, he believes there may have been a multitude of triggers for his symptoms including exercise (high altitude climbing and mountaineering), history (grandmother with Parkinson’s disease), sickness (had a gamma globulin deficiency that caused infectious boils as a youth), prescription drugs (regular use of antibiotics for folliculitis, and allergy medications), and like others surveyed, had experienced a great deal of stress.

It is possible that once afflicted with BFS that other triggers can make symptoms worse and introduce new symptoms. This should be explored further. The multitude of triggers is what can make BFS unique in each patient. Considering this uniqueness in this umbrella diagnosis, it is no surprise that it is difficult to cure or find solutions to alleviate symptoms. While BFS is benign, it is still an illness for which its sufferers would benefit from an effective treatment or cure.

Many potential triggers were purposely omitted from the survey, such as alcohol or substance abuse. [2] The survey did not want to scare participants away because they felt it was becoming too personal (checking into illegal activity). Conversely, many people have found the use of medical marijuana as good remedy and it was purposely omitted because marijuana is controlled substance that is illegal in the U.S. [2]

How to Solve Neurological Mysteries?

Doctors and researchers strive to be one to find the cure to ALS and Parkinson’s disease. If they do, what they uncover will surely help find a cure for BFS. The reverse is also true, if researchers find a cure for BFS it will undoubtedly go a long way in helping to find a cure for ALS and Parkinson’s disease. As an engineer I witnessed too many projects go awry when projects attempted to make a product that included too many features. The project would result in a costly failure and years later the company would develop three or four products to perform the job of the one initially attempted to achieve all features. Over time, the products eventually could be integrated into one product as technology and knowhow were obtained. I see the same mistake in medicine. Researchers are working endlessly and racking up billions in costs to try to find that magical cure that will end the suffering of millions of people with neurological disorders. Instead, it would make more sense to try to take small steps and gains in order to achieve that ultimate goal. This approach would be much more cost and time efficient. Finding a cure for BFS or peripheral nerve hyperexcitation disorders is that first baby step.

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