The final diagnosis
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The final diagnosis from our rheumatologist was drug-induced lupus from the TPO receptor agonists (Promacta and Nplate).
Zero cases have been reported, which is why no one truly entertained this hypothesis when I first mentioned it. But just because something isn't reported doesn't mean it's impossible.
Our rheumatologist has no intention of publishing or reporting this. Therefore, any other clinician with a patient in a similar position will tell their patient their symptoms/labs have nothing to do with the medication.
Once again, this will get swept under the rug and go unnoticed. Eventually, this will affect someone who wants to be vocal (but this takes time).
Evidence-Based Medicine
But this experience isn't isolated to this medication. In fact, this article has nothing to do with TPO agonists. Instead, it highlights the biggest flaw in medicine, which is the one thing many of us hold in high regard.
Evidence-based medicine is a double-edged sword. It helps the majority but may unintentionally hurt the minority.
Medicine is an art, and the clinician is an artist. Pablo Picasso said, "Learn the rules like a pro so you can break them like an artist."
Medical education teaches clinicians the rules quite well. However, clinicians often forget that their education doesn't stop (ever). The beauty of practicing medicine for 50 years is the experience of seeing thousands of patients; this is something that can't be learned in a textbook or from guidelines.
Unfortunately, evidence-based medicine (has the potential) to strip away the very art as it forces every person to go down the same algorithm. There is an overreliance on practicing based strictly on the studies.
Clinical Decision Making
There are so many variables that must be considered to change the algorithm:
The patient needs to report an adverse event to their healthcare provider.
Patients don't always know what's important.
Specialists can go 3, 6, or 12 months before seeing their patients. It's very easy for something to be missed if the patient is trying to fit multiple complaints into one visit.
The patient may put the one thing that’s most important to their long-term care at the bottom of the list (wants and needs are not always easy to distinguish).
The healthcare provider must be interested in research, writing, and/or adding one more thing to their plate.
Multiple case studies must be seen by someone who cares enough to propose a study.
Most who see these studies will consider the reports to be outliers and, therefore, exclude them from the clinical decision-making process.
A study involves many people and requires funding.
Getting the right team for any reason is very hard.
Trials that require large amounts of funding may need help from companies with their own incentives. Otherwise, why would they fund a trial?
Depending on the funding, you might not be able to conduct the trial that answers your initial hypothesis. Instead, you might need to answer a different question before moving forward with your initial question of interest (lengthening the timeframe).
Randomized double-blind placebo control studies are considered the gold standard, and many won't believe any study was conducted in this manner.
You must have the right patient population (very hard to do).
The reality is that it's impossible to have the exact same patient population. For example, every primary mitochondrial disease is clumped into one category for clinical trials. Because each mutation is rare on its own, they include all mutations in the same way so that there are enough participants and funding. Therefore, if treatment fails, did it truly fail, or did it fail for a subpopulation and succeed for another? It's hard to know.
The right time frame must be tested (very hard to know).
The longer the trial, the more funding is required. Therefore, the right time frame could be incentivized by cost.
None of the above happens overnight, and many patients may be harmed as the data is gathered (there are numerous examples of this throughout history).
Even if all this happens, we must remember we live in a capitalistic society (nothing inherently wrong with this) run by humans (everyone is incentivized and motivated differently within each organization).
The politics behind change, which ultimately affects incentives, can be incredibly difficult. Many will not willingly give up money, power, and control.
The change must then happen inside multiple organizations (FDA, various society guidelines, hospitals/clinics, etc.).
Final Thoughts…
Evidence-based medicine has its place; there must be a base for this to be considered science. But it can't be the only thing we rely on.
After learning the rules, you must know when to apply them and when to break them. The only way to provide personalized care is to become an artist.