Overdiagnosis: how our compulsion for diagnosis may be harming children

October 17, 2015  |  General  |  No Comments

A recent article in the journal Pediatrics is both intriguing and sobering. It is intriguing because it lays bare something we don’t talk much about or teach our students about; it is sobering because it describes the potential harm that can come from it, harm I have personally witnessed. The issue is overdiagnosis, and it’s related to our relentless quest to explain everything.

Overdiagnosis is the term the authors use to describe a situation in which a true abnormality is discovered, but detection of that abnormality does not benefit the patient. It is not the same as misdiagnosis, meaning the diagnosis is inaccurate. It is also distinct from overtreatment or overuse, in which excessive treatment is given to patients for both correct and incorrect diagnoses. Overdiagnosis means finding something which, although abnormal, doesn’t help the patient in any way.

Some of the most controversial, and compelling examples of overdiagnosis come from cancer research. Two of the most common cancers, prostate cancer for men and breast cancer for women, run smack into the issue. It is certainly generally true early diagnosis and treatment of cancer is better than late diagnosis and treatment . . . usually, not always. A problem can arise when we use screening tests for early cancer as a mandate to treat them aggressively when we find them. The PSA (prostate-specific antigen) blood test was developed when researchers noticed its value went up in men with prostate cancer. From that observation is was a short, but significant leap, to use the test in men who were not known to have cancer to screen for its presence. The problem is at least two-fold. There is overlap between cancer and normal, and many small prostate cancers do not progress quickly. Since the treatment for prostate cancer is seriously invasive and has several bad side effects, the therapy may be worse than the disease, especially in older men who will likely die of something else first. You can read more about the PSA controversy here. There are similar questions about screening for breast cancer; you can read a nice summary here. The controversy has caused fierce debates.

Children don’t get cancer very often, but there are plenty of examples of overdiagnosis causing mischief with them, too. The linked article above describes several common ones. A usual scenario is getting a test that, even if abnormal, will not lead to any meaningful effect on the child’s health. Additionally, an abnormal test then typically leads to getting other tests, which can lead to other tests, and so on down the rabbit hole. I have seen that many times. As the authors state:

Medical tests are more accessible, rapid, and frequently consumed than ever before. Discussions between patients [or their parents] and providers tend to focus on the potential benefits of testing, with less regard for the potential harms. Yet a single test can give rise to a cascade of events, many of which have the potential to harm.

This is kind of a new frontier in medicine, and the issue grows larger as the huge number of diagnostic tests we have mushrooms every year. For a parent, a good rule of thumb is to ask the doctor not just what the benefits of a proposed test are, but also the risks. Importantly, ask what the doctor will actually do with the result. We are prone to think more information is always a good thing, but that clearly is not the case. And never, ever get a test just because you (or your doctor) are merely curious.


California ends personal belief and religious exemptions for childhood vaccinations

October 7, 2015  |  General  |  No Comments

California has recently ended most exemptions from childhood vaccinations. Only exemptions for medical conditions remain, and such exemptions must be certified by a physician. The requirement applies to children attending elementary or secondary school, as well as day-care centers; home schooled children are not included. A recent editorial in the New England Journal of Medicine reviews the politics behind passage of the new law.

Clearly the recent outbreak of measles in the state played a large role in convincing the legislature to pass the law. That, plus the progressive fall in the percentage of children vaccinated. Epidemiological research has shown that when the percentage of the population that is vaccinated falls below a certain number, what is termed herd immunity no longer functions. That concept is that, if the great majority of the population is immune to a disease, the few who are not are protected by the overall rarity of the infection. The particular threshold for herd immunity varies with the disease, but it is usually in the neighborhood of 80-95%. The more infectious the disease, the higher the percentage of immune people needs to be to prevent spread. If sufficient herd immunity can be maintained for long enough, the disease can actually be eradicated. Thus far only smallpox and rinderpest (a disease of cattle) have been eliminated in this way. Perhaps the purest example of the importance of herd immunity is whooping cough, or pertussis. The people most prone to contract severe, even lethal infection are small infants. Yet they cannot begin to get the vaccine (it takes several doses) until they are several months old because it doesn’t work before that age. They are entirely dependent upon not encountering older persons who have the disease.

In my view, vaccine requirements are lawful applications of the state’s interest in public health. Adults have a right to do whatever they like to their bodies (although not their children’s) as long as their actions don’t affect others. In the case of vaccines, not participating in maintenance of herd immunity has significant and potentially serious effects on the health of others.

More here.

Cerebral palsy is generally not related to events occurring around the time of birth

September 15, 2015  |  General  |  No Comments

Cerebral palsy is an old term, and an imprecise one. It refers to a heterogenous group of lifelong movement disorders that derive from problems in the brain. Although it is generally related to the motor system, it is often accompanied by intellectual deficits, seizures, or sensory issues. It affects about 2 out of every 1,000 children.

Cerebral palsy has many causes, but for many years it was assumed that many, even most cases originate from things that happen around the time of birth, some sort of birth injury. I was taught that 40 years ago in medical school and pediatric residency. It was assumed that the most common injury was the result of interruption of oxygen delivery to the infant’s brain during labor or delivery, termed birth asphyxia. Several minutes of insufficient oxygen is well known to cause brain injury, both in this setting and in many other ones. This assumption is probably the principal reason for the invention and then the huge increase in invasive fetal monitoring over the past decades. The notion was that by closely watching the infant’s heart rate and other things during labor, potential problems could be identified. Once a baby in trouble was identified, a rapid delivery by emergency surgery, cesarian section, would rescue the baby before any damage could occur.

Not surprisingly, the increase in monitoring also led to a large increase in the proportion of babies delivered by cesarian. Better safe than sorry, ran the reasoning. There certainly are multiple conditions and situations when cesarian is life-saving, both to the infant and the mother. But most experts agree that we have overused cesarians and there have been recent initiatives to limit them to situations in which they are actually needed.

There is another thing to consider. If cerebral palsy is mostly related to injuries occurring during labor and delivery, then the dramatic increase in the proportion of babies delivered by cesarian should have reduced the number of children who develop the condition. A very useful recent study examines this question, and goes on to review what we know about the causes of cerebral palsy. It begins with this very instructive graph:

The author’s interpretation is succinct:

The increasing proportions of both elective and emergency Cesarian deliveries since 1980 have not been accompanied by any change in the proportion of live births with cerebral palsy.

So what is going on here? Why have we made no progress in reducing the number of children with cerebral palsy? In the remainder of the article the authors review what we know about causes of the disorder. They conclude that only a small minority of affected children suffer birth injury as a cause. For most, it is much more complicated.

The article reviews the many conditions we know are associated with cerebral palsy. A key observation is one we’ve known for many years: other birth defects, ones not affecting the brain, occur more frequently in children with cerebral palsy. Poor fetal growth is also correlated with it. There is also clearly a genetic component; children who have a sibling with cerebral palsy have a 10-fold higher risk of developing it themselves. Premature birth is a well-known risk factor, although this particular article is about babies born at term, on time.

The bottom line is that things are complicated, and the more we learn the more complex the entity we term cerebral palsy becomes. Although clearly children who experience severe birth asphyxia are at high risk, the majority of cases are not the result of birth injury. This conclusion has significant medical-legal implications, since obstetricians are frequently sued by families of affected children, even when the events around the time of birth were routine or nearly so. Fetal monitoring data are often pointed to, even though such data have never been shown to be predictive of cerebral palsy. The author’s final conclusion is apt:

In the past, assumptions about an asphyxial cause of cerebral palsy have led to an increase in surgical deliveries, harmed maternal services, and blinkered research. It is now evident that in advantaged countries, most cases of cerebral palsy in term or near-term neonates must have other explanations.

From my perspective it appears that the thing we call cerebral palsy is probably the final common pathway to how the fetal brain responds to a wide variety of stresses, most of which occur long before birth.


Reading to your children has demonstrable positive effects on their brain development

August 13, 2015  |  General  |  No Comments

The beneficial effects of stimulating a child’s brain have been known for decades, at least in general terms. That is to say, children who have been regularly played with, read to, and generally interacted with by adults have a great advantage over those children who did not receive these things. The key period for this appears to be up to the age of three years. For an example of this sort of research see here, whose authors conclude:

Child development was strongly associated with socio-economic position, maternal schooling and stimulation.

General observations like this demonstrate how mental growth is entangled with the effects of socioeconomic status. Children who are economically disadvantaged encounter many problems that affect cognitive development, such as poorer nutrition, more chaotic home life, and emotional stress. Any solid information on the effect of stimulation, and of what kind, would help us sort out the relative importance of these various things. Now we some fascinating recent data about that issue.

A recent study used functional magnetic resonance imaging (fMRI) to examine just what reading to a child does to the child’s brain. The reason to examine reading in particular is that literacy and language skills correlate  with later achievement. As the investigators state:

Disparities in home cognitive environment during childhood can have dramatic impact on achievement and health. Parent-child reading has been shown to improve certain emergent literacy skills, though its effect on the brain has not yet been shown.

So a big question here is precisely what does mental stimulation, particularly reading, do to a child’s brain? Can we document what is happening between the ears? Now we have some information about that. The investigators did fMRI scans on children to identify what regions of the brain reading activated. What they found was this (from the American Academy of Pediatrics summary):

Results showed that greater home reading exposure was strongly associated with activation of specific brain areas supporting semantic processing (the extraction of meaning from language). These areas are critical for oral language and later for reading. Brain areas supporting mental imagery showed particularly strong activation, suggesting that visualization plays a key role in narrative comprehension and reading readiness, allowing children to “see” the story. This becomes increasingly important as children advance from books with pictures to books without them, where they must imagine what is going on in the text.

It is important that these observations held up even after controlling for socioeconomic status. I should note that this research is reported in what we term abstract form — the complete details are yet to be published. It also has not been confirmed (as far as I know) by other investigators yet. Even with these caveats, finding a physical locus in the brain for complicated mental events is exciting stuff.

There is a footnote to this research that goes back to the Baby Einstein controversy in 2007. If you didn’t know, the Baby Einstein products were videos whose authors claimed were educational in the sense of improving learning and brain development in infants and toddlers. The company was sued for false advertising claims and the Disney Corporation (the owner) paid out refunds to those who had bought them. More about that controversy here. Research published in 2007 actually showed regression of language in children who watched a lot of these videos. So how can we square that with the experience of reading to your child being good for the brain?

I have no data to offer about this, but I suspect the difference between putting your child in front of a TV and reading to him or her is the personal interaction that accompanies reading.


How placebo and nocebo effects work

July 18, 2015  |  General  |  No Comments

All physicians are familiar with what is called the placebo effect: the improvement in a patient’s symptoms after receiving a treatment that has no known effect on the particular disease. I was taught that statement could be further refined to state: improvement in a patient’s symptoms after receiving a treatment that has no known biological effect on the particular disease. The standard example is a “sugar pill.” That’s an important distinction, but it’s also clearly wrong; improvement in symptoms is obviously a biological effect, no matter the mechanism. The placebo effect is evidence of the complex interactions between mind and body because symptoms, by definition, are things perceived by the brain and cannot be specifically measured. Placebo effects are perception. They do not, for example, shrink tumors, cure asthma, or control diabetes.

The placebo effect is a key reason why, as much as possible (sometimes it isn’t), we study patient response to new treatments using a placebo arm in randomized, controlled trials. These are studies in which a patient is randomly chosen to receive either the new drug or something presumed biologically to have no effect on the particular disease. Neither the patient nor the investigator knows which one the patient is getting until after the trial is over. (Some trials compare a new treatment with an existing one — that’s a different topic.) The placebo effect is when a patient receiving the inert substance, the sugar pill, experiences an improvement. How big is the effect? Sometimes it can be as high as 30% of patients. This is why a placebo group is so important for studying disorders in which subjective symptoms are the essence of the disease, such as migraine headaches. There is no objective test one can do to assess improvement; it is all patient-reported.

How does the placebo effect work? There is a wonderful and highly understandable discussion of this in a recent edition of The New England Journal of Medicine. The article is only a couple of pages long and well worth a read. There is also a good podcast accompanying the piece. The bottom line is that clearly the placebo is not really an inert substance in the sense of doing nothing. It is doing something to the patient’s perception of symptoms, often by using known pathways of neurotransmitters in the brain. From the article:

Moreover, recent clinical research into placebo effects has provided compelling evidence that these effects are genuine biopsychosocial phenomena that represent more than simply spontaneous remission, normal symptom fluctuations, and regression to the mean.

The authors describe a fascinating example of how the placebo effect can play a role in a complex disorder like asthma. In asthma we can measure air flow as the patient breathes; that is, we can get an objective measure of how severe a patient’s problem is. The patient typically feels short of breath, and the degree of reduction of airflow correlates with that symptom. But feeling short of breath is a subjective thing. It comes from the brain. I have seen both patients in severe subjective distress with only modest reduction in airflow and patients surprisingly comfortable with very decreased objective numbers. There is a significant subjective component to asthma. This has been demonstrated by giving an placebo breathing treatment to an asthmatic and then showing that, as expected, there is no improvement in airflow. Yet the patient may experience an impressive improvement in perceived breathing symptoms.

There is also another side to the coin, what is called the nocebo effect.

 . . . the psychosocial factors that promote therapeutic placebo effects also have the potential to cause adverse consequences, known as nocebo effects. Not infrequently, patients perceive side effects of medications that are actually caused by anticipation of negative effects or heightened attentiveness to normal background discomforts of daily life in the context of a new therapeutic regimen.

Here is an example. Patients in randomized controlled trials do not know if they are receiving the placebo or not. But just in case they are receiving the real drug they are informed of possible side effects. Interestingly, 4 – 26% of patients in the placebo groups in such trials stop their participation because of these perceived adverse effects. This is the nocebo effect.

One of the most fascinating aspects of this is that there appear to be genetic predispositions among people for experiencing a placebo effect. This is an area of active research. The authors’ conclusion is a good one, I think:

Of course, placebo effects are modest as compared with the impressive results achieved by lifesaving surgery and powerful, well-targeted medications. Yet we believe such effects are at the core of what makes medicine a healing profession.

The placebo effect has always been a part of medicine. Patient’s perceptions of their physician’s compassion have long been known to be important. Really, until quite recently in medicine the placebo effect was all physicians had to offer. And it’s not a bad thing. I think it explains the modest improvements reported by some patients receiving a wide variety of what we call these days alternative therapies, such as homeopathy.

Anyway, the essay is a good review of this fascinating subject, and I recommend it to you.

Half of children may not be drinking enough water

June 30, 2015  |  General  |  No Comments

Adequate hydration, getting enough water, is vital to good health. After all, we’re mostly made of water — about 60% of our bodies is water. Our bodies are quite good at hanging on to most of this water. Our skin holds nearly all of it inside, for example. But we do lose water through what we call obligatory or insensible losses. Some is lost through our skin, such as through sweating. A fair amount is lost through our breath, since the air we breathe out is fully humidified. Our kidneys also lose water through urine. Even though these organs are very, very good at conserving water, they still need to manufacture a minimal amount of urine to keep good function. So we need to take in water every day. There is some water in food, of course, more in some foods than in others. But the bulk of our daily water needs come from drinking.

How much water do we need every day? A good general rule is that an average adult male needs around 3 liters (about 3 quarts) per day and an average adult woman requires 2.2 liters. Of course that is just the baseline. If you are doing something that increases water loss, such as moderate or severe exercise, you need additional water on top of the basal amount to make up for what you are losing. What about children? On a per weight basis children generally need more. We have some simple calculations we use to determine what we call their maintenance fluid need: 100 mL/kg for the first 10 kg of body weight, 50 mL/kg for the next 10 kg body weight, and 20 mL/kg for every kg after that until the child is adult sized. That works out to about 1 liter/day for a 10 kg (22 pound) child, 1.5 liters/day for a 20 kg (44 pound) child, and 1.9 liters for a 40 kg (88 pound) child. Again, that’s just the baseline; you need to add more for activity.

Our thirst mechanism is a key way we regulate our water, but both adults and children can take in less than their requirement but not get too thirsty if the deficit is not much. Yet that water deficit can still be significant. Chronic, mild dehydration makes you feel generally unwell, something probably all of us have experienced. For example, if you’ve been out and about most of the day and feel a bit lethargic — maybe you have a mild headache as well. Then you drink a tall glass of water and within a few minutes feel much better.

An interesting recent research study asked the question if children are, on average, as hydrated as we would recommend. They used data from the National Health and Nutrition Examination Survey, which spanned the years 2009-2012, and involved over 4,000 children between the ages of 6 – 19 years. The study was a powerful one because it didn’t just ask parents how much fluid their children drank; it measured the osmolality of the urine, which indicates how concentrated the urine is. The body normally makes urine more concentrated if it is trying to conserve water because the person isn’t drinking much.

The results were interesting. The authors found that just over half of all children were not optimally hydrated. They weren’t dehydrated, that is to say sick, but their urine osmolality was higher than what we would recommend. The authors also calculated that around 8 ounces of additional water (about 350 mL) would be sufficient to bring the average school age child up to the recommended amount. That’s the size of a standard kitchen glass of water.

The take home message for me is to offer your children plenty of water at meals and make sure they take water with them when they go to various activities, especially sports.

CBO finds that 19 million would lose their health insurance if the ACA is repealed

June 19, 2015  |  General  |  No Comments

[This is important. It was written by Phil Galewitz and republished (by permission) from Kaiser Health News (KHN), a nonprofit national health policy news service.]

Repealing the federal health law would add an additional 19 million to the ranks of the uninsured in 2016 and increase the federal deficit over the next decade, the Congressional Budget Office said Friday.

The report is the first time CBO has analyzed the costs of the health law using a format favored by congressional Republicans that factors in the effects on the overall economy. It is also the agency’s first analysis on the law under Keith Hall, the new CBO director appointed by Republicans earlier this year.

CBO projected that a repeal would increase the federal deficit by $353 billion over 10 years because of higher direct federal spending on health programs such as Medicare and lower revenues. But when including the broader effects of a repeal on the economy, including slightly higher employment, it estimated that the federal deficit would increase by $137 billion instead.

Both estimates are higher than in 2012, the last time that the CBO scored the cost of a repeal.

The latest report from the nonpartisan congressional watchdog and the Congressional Joint Committee on Taxation comes just days before the Supreme Court is expected to rule on the health law’s premium subsidies in the nearly three dozen states that rely on the federal marketplace. Such a ruling would cut off subsides to more than 6 million people and be a major blow to the Affordable Care Act. It could also boost Republican efforts to repeal the entire 2010 law, which would likely face a presidential veto.

Last week, President Barack Obama said nearly one in three uninsured Americans have been covered by the law—more than 16 million people.

The CBO said repealing the health law would first reduce the federal deficits in the next five years, but increase them steadily from 2021 through 2025. The initial savings would come from a reduction in government spending on the federal subsidies and on an expanded Medicaid program. But repealing the law would also eliminate cuts in Medicare payment rates to hospitals and other providers and new taxes on device makers and pharmaceutical companies.

The CBO projected that repeal would leave 14 million fewer people enrolled in Medicaid over the next decade. Medicaid enrollment has grown by more than 11 million since 2013, with more than half the states agreeing to expand their programs under the law.

By 2024, the number of uninsured would grow by an additional 24 million people if the law is repealed.

In 2012, the CBO projected repealing the health law would increase the federal deficit by $109 billion over 10 years.  It said the higher amount in Friday’s report reflected looking at later years when federal spending would be greater.

cbo repeal 600




Despite recent media spotlight there really is little controversy over the existence of shaken baby syndrome

June 1, 2015  |  General  |  No Comments

A recent series of articles in the Washington Post and a segment on NPR have caused quite a stir. The articles are about what we have called for decades “shaken baby syndrome.” It can be fatal. We now use the term non-accidental head trauma. This term replaced the older one because it is more specific; children can be deliberately harmed in other ways besides shaking. In addition, inflicted trauma can happen in other places besides the head. The Post article was about the shaking variety, and it focused on several things. It highlighted several individuals who had been apparently wrongly convicted of injuring a child through shaking. It also interviewed physicians who do not believe in the diagnostic entity; they say shaken baby syndrome does not exist. Not surprisingly the article generated a lot of comment and debate, debate that has actually been going on for some time. As a pediatric intensivist for over 30 years I have dealt with many unfortunate examples of this entity, and I have no doubt that it exists. But, like all disorders that do not have a specific, definitive test for them, deciding whether or not a child has suffered shaken baby syndrome depends upon more that some x-rays and an eye examination; you need to consider the entire context of the story.

Shaken baby syndrome was first described in the 1960s to describe the combination of several injuries: subdural hematoma (bleeding around the brain), retinal hemorrhages (bleeding at the back of the eye), and brain swelling. Rib fractures are also common because the person doing the shaking typically squeezes the child’s chest hard enough to crack ribs. How do these injuries happen with shaking? The fundamental cause is that a small baby has a relatively large head compared to the rest of his body and is unable to hold his head firmly in place because the muscles aren’t strong enough yet to do that. So shaking snaps the head back and forth, generating very large forces inside the skull as the brain bangs back and forth. This can lead to rupture of some of the small veins that surround the brain, as well as tiny vessels in the back of the eye. The brain then often swells afterwards, as any tissue does when injured. If death or severe injury follows it is generally because of the brain swelling. If ribs are broken from squeezing the chest, the fractures happen at the back of the bones where the ribs come off the spinal column. It is often illustrated in this way.

There have always been some issues about diagnosing the syndrome. The main one is that all of the components of shaken baby syndrome can occur individually in other settings. Another issue is that, as in most cases of potential child abuse, the alleged assault is unwitnessed and the victim cannot give any evidence. So all evidence is circumstantial. And, of course, the stakes are very high not just for the injured child; adult caregivers can be convicted for murder. The Post article focuses on several cases like that. Some physicians have gone as far to claim that the syndrome doesn’t even exist. The American Academy of Pediatrics vigorously disagrees:

Journalists can be commended for addressing child abuse. Unfortunately, the Post’s report is seriously unbalanced, sowing doubt on scientific issues that actually are well-established. It is very clear that shaking a baby is dangerous.

It is important to acknowledge that mistakes probably have been made in both over-diagnosing and under-diagnosing abuse. The Post focused on over-diagnosis, but under-diagnosis also is a problem, leaving babies vulnerable to further abuse and even death. It’s critical we get this right.

Well okay, you might say. Of course the Pediatric Establishment would say something like that. But I think it is clear the syndrome exists. I have seen it many times and have been involved in legal proceedings charging the perpetrator, the majority of whom ultimately confessed to the act. The thing is, in all the cases I have been involved in there were other things that pointed toward child abuse. For example, a baby’s tissues are delicate and the squeezing and shaking often causes obvious bruising. I have seen several cases where the bruises even matched adult finger marks. If rib fractures are present, there is essentially no way a baby could break ribs in the typical places without shaking. Finally, and very important, is the history. Injuries like bleeding in the brain need to be explained. If there was no child abuse, then there has to be another coherent, logical explanation. I have never been involved in a case in which the potential perpetrators (or their lawyers) could give such an explanation. I have been involved, however, in cases in which no perpetrator was identified because several were possible and none came forward with the real story. More from the AAP:

What are the facts? [about denying the existence of the syndrome] . . . it involves a tiny cadre of physicians. These few physicians testify regularly for the defense in criminal trials — even when the medical evidence indicating abuse is overwhelming. They deny what science in this field has well-established. They are well beyond the bounds where professionals may disagree reasonably. Instead, they concoct different and changing theories, ones not based on medical evidence and scientific principles. All they need to do in the courtroom is to obfuscate the science and sow doubt.

Miscarriages of justice are tragic. But so is child abuse, and it is unfortunately not uncommon. Jury trials are imprecise and blunt tools of justice. If I were sitting in the jury box, I would like some other evidence besides just the triad of subdural hematoma, retinal hemorrhages, and brain swelling. In my experience there is generally additional evidence. I think the Post article is more than a little like the way the media portrays other scientific issues — controversy sells (or these days attracts page views). The media presents several scientific issues, for example childhood vaccinations, as he-said-she-said stories even when the scientific consensus is overwhelmingly one way and not the other.


“Cowboy” doctors mostly increase costs and risks without benefiting patients

May 12, 2015  |  General  |  2 Comments

Some months back I read an interesting interview with Jonathan Skinner, a researcher who works with the group at the renowned Dartmouth Atlas of Health Care. More than anyone else I can think of, the people at the Dartmouth Atlas have studied and tried both to understand and to explain the amazing variations we see in how medicine is practiced in various parts of the country. It turns out that specific conditions are treated in quite different ways depending upon where you live. Atul Gawande documented a detailed example of the phenomenon in an excellent New Yorker article here. A major determinant appears to be local physician culture, how we doctors “do things here.” The disturbing observation is that patient outcomes aren’t much different, just cost. Of course it’s more than cost. Doing more things to patients also increases risk, and adding risk without benefit is not what we want to be doing.

Skinner is interested in something else, a phenomenon he calls “cowboy doctors.” By this he means physicians who are individual outliers, who go against the grain by substituting their own individual judgements for those of the majority of their peers. In theory such lone wolf practitioners could go both ways. They could do less than the norm, but almost invariably they do more — more tests, more treatments, more procedures. Such physicians not only may put their patients at higher risk, they also add to medical costs. I have met physicians like that and have usually found them to be defiant in their nonconformity. A few revel in it. They maintain they are doing it for the good of their patients, but there is more than a little of that old physician ego involved. There is also the subtext of what many physicians feel these days, especially old codgers like me who have been practicing for 35 years: it is the tension between older notions of medicine as an art, a craft, and newer evidence-based, team driven practice. Skinner describes it this way:

It’s the individual craftsman versus the member of a team. And you could say, ‘Well, but these are the pioneers.’ But they’re less likely to be board-certified; there’s no evidence that what they’re doing is leading to better outcomes. So we conclude that this is a characteristic of a profession that’s torn between the artisan, the single Marcus Welby who knows everything, versus the idea of doctors who adapt to clinical evidence and who may drop procedures that have been shown not to be effective.

Leaving aside outcomes and moving on to costs, Skinner and his colleagues were quite surprised to discover how much these self-styled cowboys and cowgirls were adding to the nation’s medical bills. They found that such physicians accounted for around 17% of the variability in regional healthcare costs. To put that in dollars, it amounts to a half-trillion dollars. That is an astounding number.

So what we are looking at here is a dichotomous explanation for the huge regional variations in medical costs. On the one hand we have physicians who conform to the local culture, stay members of the herd and go along with the group, even if the group does things in a much more expensive way that confers no additional benefit to patients. On the other hand we have self-styled mavericks who scorn the herd and believe they have special insight into what is best, even if all the research shows they’re wrong.

I think what is coming from all this cost and outcome research is that best practice, evidence-based medicine (when we have that — often we don’t for many diseases) will be enforced by the people who pay the bills and professional organizations. Yes, some will bemoan this as the loss of physician autonomy and the reduction of medical practice to cookbooks and protocols. I sympathize with that viewpoint a little, especially since I am the son and grandson of physicians whose practice experience goes back to 1903. But really, there are many things we used to do that we know now are useless or even harmful. An old professor of mine had a favorite saying for overeager residents: “Don’t just do something — stand there!”

For those who would like to dive into the data and see the actual research paper from the National Bureau of Economic Research describing all this, you can read it here.


Recalling a public health triumph: the eradication of cretinism

April 30, 2015  |  General  |  No Comments

One of the words we don’t use any more is cretin; it’s long been a derogatory slur rather than a precise description of something. But a century ago cretinism actually meant a specific thing: a person, generally a child, who was severely damaged by a lack of thyroid hormone during early development, particularly fetal development. Now we call the condition congenital hypothyroidism. A few cases still exist, which is why we screen all newborns for thyroid function. But the overwhelmingly most common cause a century ago was hypothyroidism — low thyroid hormone — in pregnant women. The overwhelmingly most common cause of that was deficiency of iodine in the diet.

The thyroid gland sits in the front of your neck, just below your voice box (larynx). It has two lobes on either side connected by a little bridge. Its job is to make, store, and release thyroxine, or thyroid hormone. This hormone has several important functions, acting upon nearly every cell in the body in one way or another. It affects the metabolism of cells, how they use energy, and is key to cellular growth and development. The thyroid gland needs iodine to make thyroxine properly. A thyroid that is not making thyroxine properly may swell into a goiter, another thing that once was common and now is rare. There are various reasons adults may develop low thyroxine levels, become hypothyroid. These days this condition is easily treated by taking oral thyroid hormone every day. The problem for a baby developing in the womb is that a deficiency of thyroxine in the mother causes irreversible damage before the baby is born, and thus before we can give the infant thyroid hormone.

Congenital hypothyroidism is now rare in the developed world. Why? You can read the history lesson of why in a nice review here, but the reason is iodine supplementation of food, particularly salt. This is a fascinating example of several companies, particularly the giant Morton Salt Company, listening to the advice of medical experts and then just adding iodine to their product. This turned out to be an easy thing to do.

The result was an astounding public health triumph. Congenital hypothyroidism on the basis of iodine deficiency is still a problem in the developing world, but it has been eliminated from the developed world. To me it brings to mind the addition of fluoride to water and the subsequent dramatic reduction in dental caries in children. Interestingly, although I have a graduate degree in history of medicine, I am unaware of any organized efforts by people to resist iodized salt as there has occasionally been for fluoridated water. You can buy salt without iodine, although I don’t know why you would want to, but salt is found in nearly every food product that has been processed in any way, such as bread. So you can’t really avoid it.

Again, this is an example of a simple, well targeted population intervention, like vaccination, that conquered a disease that had plagued people for millennia.