Sudden infant death syndrome, or SIDS, has been recognized for hundreds, if not thousands of years. It is the leading cause of death for children between the ages of 28 days and 1 year. These tragedies happen when an apparently normal infant, generally two to three months of age, is put to bed, only to be found sometime later limp, not breathing, and to have no beating of his heart. Many times these infants are rushed to the hospital, where the doctors try to revive them. Most of the time we are unsuccessful. Or, if we do manage to get some return of heart rate, these unfortunate babies typically die several hours or a day or so later. I have been involved in dozens of cases like this during my career.
No one knows what causes the tragedy of SIDS. We do know that some children are at a higher risk than others. Known risk factors include these, among others:
- Being born prematurely or at a low birth weight
- Sleeping position
- Poor prenatal care for the mother
- Exposure to tobacco smoke
- Having a sibling or cousin die from SIDS
A good way to think of how these risk factors interact, called the “triple risk factor theory,” is shown in this diagram:
The idea is as follows. There is a critical time in early brain development when an infant’s brain learns how to regulate his breathing reflexes. When, during that critical time, an infant predisposed to get SIDS (such as from a family history of it), finds himself in an environment that affects breathing (such as tobacco smoke), the chances of getting it go up.
A large campaign begun in 1994, called “back to sleep,” has focused on the second of these SIDS risk factors in the list, sleeping position. There is an association between a baby sleeping prone — on his stomach — and SIDS. So we’ve stressed putting a baby to sleep on his back. And, probably because of this program, SIDS deaths have gone down, as shown in this graph. Although it’s an old statistical principle that correlation doesn’t prove causation, you can see that overall changes in infant sleep position and a reduction in SIDS cases correlate quite well.
So you can see we’ve made real progress in reducing SIDS. In 1990, before the “back to sleep” program, SIDS deaths were 126 infants per 100,000 births, or between 1 and 2 infants per thousand babies; only six years later the rate was half that. The graph below, however, shows that this positive trend has leveled off since then.
(Look at the middle line, the diamonds : the squares are unknown or otherwise mysterious deaths, and the top line, the triangles, are the two added together.)
One reason for this leveling off may be that, since 2001, the number of parents who put their infant to bed on his stomach has not changed — a recent survey indicated that about a quarter still do this, a number that has been unchanged over the past decade.
Looking at the data, the American Academy of Pediatrics recognized that, although a supine (on your back) sleeping position is important for reducing the risk of SIDS, there are other aspects of sleep position that also likely play a role in SIDS. This has led to a new set of recommendations for infant sleeping position. These include:
- Infants should be placed for sleep in a nonprone position. Supine (entirely on the back) confers the lowest risk for SIDS and is preferred. However, while side sleeping is not as safe as supine, it also has a significantly lower risk for SIDS than prone. If the side position is used, caretakers should be advised to bring the infant’s lower arm forward to lessen the likelihood of the infant rolling to the prone position.
- A crib that conforms to the safety standards of the Consumer Product Safety Commission is a desirable sleeping environment for infants. Although many cradles and bassinets also may provide safe sleeping enclosures, safety standards have not been established for these items. Sleep surfaces designed for adults may have the risk of entrapment between the mattress and the structure of the bed (for example, the headboard, foot board, side rails, and frame), the wall, or adjacent furniture, as well as between railings in the headboard or foot board.
- Infants should not be put to sleep on waterbeds, sofas, soft mattresses, or other soft surfaces.
- Avoid soft materials in the infant’s sleeping environment. These include such things as pillows, quilts, comforters, or sheepskins under the infant. Soft objects, such as pillows, quilts, comforters, sheepskins, stuffed toys, and other gas-trapping objects should be kept out of an infant’s sleeping environment. Also, loose bedding, such as blankets and sheets, may be hazardous.
- Overheating should be avoided. The infant should be lightly clothed for sleep, and the bedroom temperature should be kept comfortable for a lightly clothed adult. Over-bundling should be avoided, and the infant should not feel hot to the touch.
You can read more about the details, as well as about the rationale, the background science, for these new recommendations here.
There are some potential problems with back sleeping. One of them is getting a flat spot on the back of the infant’s head. This happens because an infant’s skull is so soft; if the back of the skull is always on a flat surface, it can make a dent there. There are various ways to prevent this, one of which is supervised “tummy time” for your infant.
When is it safe to let your child sleep on his tummy? A good, and quite sensible, rule of thumb is that when your child is able to roll back to front it is fine to let him end up in whatever position he likes best.
My last post was about asthma. This one is about another very common breathing problem in children — bronchiolitis. In some ways it is similar to asthma, but in other important ways it is very different. With winter nearly upon us it’s time to reacquaint ourselves with this common entity.
I’ve written before (here and here) about the reliable seasonal arrival of the virus we call RSV, the chief cause of bronchiolitis. To scientists, RSV is a fascinating virus with several unique properties.
One of these is its behavior in the population. When it is present, RSV is everywhere. Then it suddenly vanishes. There are exceptions to everything in medicine — I have seen sporadic cases during the off-months — but generally RSV arrives with a bang in mid-winter and then leaves suddenly in the spring. It is the only virus that consistently and reliably causes an epidemic every year, although it often alternates more severe with milder visitations. However, RSV epidemics may still have some regional variability. For example, often one city will have a much more severe epidemic than do others in other regions of the country.
Another aspect of RSV that interests medical scientists is how poor a job our immune systems do in fighting it off. Virtually all children are infected with RSV during the first year of life. Not only that, all of us are reinfected multiple times during our lives. Attempts at devising a vaccine for RSV have all been unsuccessful. In fact, early versions of an experimental vaccine seemed to make the disease worse in some infants, raising the possibility that some aspect of our immune response to the virus actually contributes to the symptoms.
RSV has a high attack rate — the term scientists use for the chances that a susceptible person will get the infection if exposed to it. That, plus our generally poor defenses against it, explain the frequent epidemics.
So what is bronchiolitis? What does it look like? In medical terminology, adding the ending “itis” to a word means that whatever comes before is inflamed. Thus tonsillitis is an inflammation of the tonsils and appendicitis means an inflamed appendix. So bronchiolitis is an inflammation of the bronchioles, which are the final part of the system of air-conducting tubes that connect the lungs with the outside world. Beyond the bronchioles are the aveoli, the grape-like clusters of air sacs where the business of the lungs — getting oxygen into our bodies and carbon dioxide out — takes place.
Bronchiolitis, like asthma, is a disorder of blocked small airways. This prevents air from getting in and out normally, primarily out. But the principal source of that blockage differs between the two lung problems. In bronchiolitis, the main problem is that the bronchiole tubes are blocked from swelling of the walls and from debris caused by the RSV infection — bits of broken airway cells and mucous plugs. Here’s what it looks like.
Infants are the ones who have the most trouble breathing with bronchiolitis. There are several reasons for this, but a key one is the construction of an infant’s chest. When small airways get blocked, we use our chest muscles — tightening them — to force air in and out of our lungs. We are helped in doing this by the fact that our lungs are encased in a fairly rigid rib cage; when we use our muscles to squeeze or expand our chest the system works like a bellows. Infants can’t do this well because the ribs across the entire front half of their chest are not yet solid bone — they are still soft cartilage. So when a small infant tries to suck air in against anything that is restricting airflow, like clogged bronchioles, his chest tends to sink inwards, causing what we call retractions. They also have trouble forcing air out, so their chests become hyperexpanded with air. The other reason infants have so much trouble handling debris in their bronchioles is that they are already narrow to start with, so they get more easily clogged up than do larger, adult-sized airways.
How does a child with bronchiolitis look? Typically they are breathing faster than the normal respiratory rate of 25-35; often they are puffing along at 60-70 breaths per minute. They also will show those chest retractions and have a cough. Fever is uncommon. They may look a bit dusky from not having enough oxygen in the blood. They often have trouble feeding because they are breathing so fast. The fast breathing, although with the poor feeding, often makes them become dehydrated. Our breath is completely humidified, so when we breathe fast we lose more water.
Can we do anything to treat this illness, make the symptoms better, make it go away faster? Sadly, the answer is no. I’ve been taking care of children with RSV for 30 years, and I’ve seen a long list of things tried — breathing treatments, anti-viral medicines, steroids, medicines intended to open up the small airways. None of them work very well, if at all. Even though the symptoms resemble asthma in some ways, none of the asthma medicines work very well, although often we try them just to see because the occasional child will get just a little better with them. The research of the past few years is conclusive — the best we can do is to use what we call supportive care and wait for the infection to pass, meanwhile helping breathing as needed with oxygen, clearing the lungs of mucous, and sometimes a mechanical breathing machine in severe cases.
RSV is generally not a serious illness, but for some children it can be life-threatening. These children are very small infants, especially those born prematurely, and those with underlying problems with their lungs or their hearts. For those infants we have a monthly shot (called Synagis) that helps reduce the severity of RSV when they get it, and may even prevent a few cases, but this is not ideal.
Since RSV cannot be prevented, the best thing a parent can do is try to postpone it. That is, if you have a newborn infant in the height of RSV season, try to minimize exposure of your child to people with cold symptoms, especially toddlers. And for those who do handle your infant, have them wash their hands first.
Asthma is a common problem in children — nearly 10% now have it — and the number is increasing. Researchers are not sure of the reasons for this steady increase (more here), but decreased air quality, lower activity levels among children, and an increase childhood obesity have all been implicated. Whatever the cause, it means that millions of American children take medicine for asthma. A significant number of these children end up in the PICU for a severe asthma attack. As I speak to their parents, it is clear that more than a few parents have only vague ideas of how the different types of asthma medicines we use work in their child’s body. This is an important subject, since using the medicines correctly is the best way to keep your child out of breathing trouble, and to use them correctly it very much helps to understand how they work.
The first thing to understand is what is taking place inside the lung during an asthma attack. Once you know that, you can see how the different asthma medicines relieve the symptoms. Here is a schematic drawing of what a normal lung looks like:
You can think of the lungs as being composed of two parts. The first is a system of conducting tubes that begin at the nose and mouth, move through the trachea (windpipe), split into ever smaller tubes, called bronchi, and end with tiny tubes called bronchioles. The job of this system is to get the air to the business portion of the lungs, which are the alveolar sacs. This second part of the lung brings the air right next to tiny blood vessels, or lung capillaries. Entering capillary blood is depleted in oxygen and loaded with carbon dioxide, one of the waste products of the body’s metabolism. What happens next is gas exchange: as the blood moves through the capillaries, oxygen from the air we breathe in goes into the blood, and carbon dioxide leaves the blood and goes into the air we breathe out. The newly recharged blood then leaves the lungs in an ever enlarging system of pulmonary veins and then goes out to the body.
The main problem in asthma is that the conducting airway system gets blocked in several ways, so the oxygen can’t get in and the carbon dioxide can’t leave. Although both are a problem in a severe asthma attack, getting the air out is usually a bigger issue than getting it in because it is easier for us to generate more force sucking in air than blowing it out. So the hallmark of asthma is not getting the air out — called air trapping. Why does this happen? There are two principal reasons: for one, the small airways, the bronchioles, constrict, get smaller; for another, the walls of the airways swell and the airways themselves fill with excess mucous, blocking air flow. Here’s another schematic drawing of what that looks like.
Thus during an asthma attack these things happen, all of which act together to narrow the airways and reduce air flow:
- The smooth muscle bands around the tiny airways tighten
- The linings of the airways get inflamed and swell
- The mucous glands in the airways release too much mucous, filling the airways
The medicines that we use to treat asthma work by reducing (or even preventing) one or more of these things. But before we get to them, an obvious question is why are our lungs are constructed in this way, especially if it can cause trouble? Why are those smooth muscle bands there? Why does there need to be mucous in our airways?
The smooth muscle bands are there for a good reason. The lungs need a way to direct the air we breathe in to the best spots, which are those regions of the lung with the best blood flow, and that changes from minute to minute from such things as changes in our position — lying down to standing up, for example. Those muscle bands function like the head gates of an irrigation system, opening and closing to direct air to the best places. The mucous is important because it is one of the chief defenses our lungs have against harmful or irritating things we breathe in. The mucous traps debris and steadily moves it up and out of our lungs. In asthma, both of these natural systems become deranged. The so-called triggers for this derangement vary from person to person, but the results are similar. The medicines we use are similar, too, no matter what started the asthma attack.
One of the mainstays of asthma treatment is a member of a class of medicines we call selective beta agonists. The generic name for the one we use most commonly is albuterol. Common brand names for albuterol are Ventolin and Proventil. Albuterol comes as a liquid, which we blow into a mist either with a device called a nebulizer or with what’s called a metered dose inhaler (“puffer”). The second of these is more convenient to carry around, but it can be more difficult to use with small children, although adding a special chamber to the device can help. The patient inhales the mist of albuterol. It works by soaking into the smooth muscle bands, making them relax, and in that way making the airway tubes bigger to allow more air flow. (There is also an oral form of albuterol, but for a variety of reasons it is not a good choice for children with asthma.) For many patients with asthma, inhaled albuterol alone is adequate treatment for their symptoms. A key thing to know about albuterol is that it goes to work right away, generally within minutes, so it is a good medicine for an acute asthma attack.
Another class of medicines long used in the treatment of asthma is corticosteroids, or steroids for short. These medicines work by being powerful blockers of inflammation. If you have ever had a poison ivy rash, for example, you are familiar with inflammation: redness, swelling, and seepage of fluid from the tissue (we can use steroids to treat poison ivy, too). A similar inflammation around the small airways is characteristic of asthma. It makes the linings of the airways swell, weep fluid, and increase mucous production. For a severe attack, we give steroids by mouth or intravenously (IV, directly into the bloodstream). They are very effective when given that way. But they do not go to work right away — several hours are needed at least. So although we may start them during an acute attack, we don’t expect them to help for a while.
Steroids are powerful drugs. When you take them by mouth they affect your entire body, not just your asthma, and that can cause problems. This is why we only use systemic steroids — those by vein or by mouth — for as short a time as possible, typically five days or so. We have other forms of steroids that are inhaled. This allows them to work directly on the airways without affecting the entire body. Common brand names of inhaled steroids are Pulmicort and Flovent. The inhaled steroids, like the systemic ones, don’t go to work right away. So they are intended primarily as a medicine to maintain control of the asthma. It is a common mistake for parents to give their child multiple doses of inhaled steroids when they have worsening breathing troubles — steroids are not intended to be used that way. The proper so-called “rescue medication” for worsening symptoms is albuterol or drugs like it.
These days we have hybrid medications that combine a long-acting albuterol type drug with an inhaled steroid. This combination is intended as something to be taken for chronic control of patients with moderate or worse asthma, and these agents are quite effective at doing that. Common brand names are Advair and Symbicort.
So albuterol (and beta-agonists like it) and steroids are mainstay medicines for treating asthma. In combination they make a good team because they attack the asthma via two different modes of action. We have some other medications that work by still other mechanisms. Montelukast (brand name Singulair) blocks airway inflammation by another mechanism than do steroids. Unlike systemic steroids, the action of montelukast is more selective and this medication is safe to take for prolonged periods. For some patients, montelukast and an occasional puff of albuterol is sufficient to keep them out of trouble. Finally, an inhaled drug called ipratropium (brand name Atrovent) blocks excessive mucous production by another method than blocking inflammation; it is often helpful as an adjunct to the other medicines. A couple of medications (brand names Combivent and DuoNeb) combine ipratropium and albuterol together so they can be inhaled at the same time.
So how do doctors decide what asthma medicines to use? One obvious principle is that it makes little sense to use more than one medication of the same category: combinations ought to work in different ways so they can work together. But beyond that obvious principle, how do we decide? The usual approach is to classify patients with asthma according to their severity and then add medicines in a logical, step-wise way until we get control of the symptoms. There are guidelines to help us do this. A good, recent summary is here, published by the National Institutes of Health. If you or your child has asthma it is a good place to find information. It is also useful to look at the actual decision tree doctors use to decide what medicines to use and in what order. You can find it here.
Asthma is common and is getting more common every year. Certainly speak with your child’s doctor about doing some good detective work to figure out what your child’s asthma triggers are. Then take steps to modify exposure to them or avoid them. Common sense tells us that if we can reduce symptoms by reducing exposure to common triggers, such as tobacco smoke, we should do everything we can to reduce the need for asthma medications. But for many children, this will not be enough; their parents should understand how these medicines work in order to make the best use of them.
A recent editorial in the New England Journal of Medicine makes an interesting contrast between the approaches public and private health plans have taken in controlling costs. It points out how governmental health programs — Medicare and Medicaid — have long focused on controlling costs by focusing on the unit cost of things. So they have paid less attention to how many of something, say a surgical procedure, gets done and more attention to the cost of each one. What can happen in this approach is that doctors do more and more of whatever it is in order to make up the lost revenue. This can be quite bad for patients. Overutilization of health services is already a huge problem; some estimates are that a quarter to a third of medical care provided in America is unnecessary. More care is not better care, although that notion in some way seems to go against our national ethic.
Private insurers have generally tended to take another approach: instead of largely focusing on the unit cost of providing a service, they have tried to control the number of times the service is provided, using such things as preauthorization requirements. The result has been predictable — the gap between the unit cost paid by private insurers, always more than public programs paid, has been getting steadily larger. In 2000, providers on average billed private payers about 15% more than public ones for the same procedure; by 2009, the difference was 30-40% more for the private ones. In the same period, public payers on average cut by 10-15% what they would pay for the procedure. So if you add up the math, you can see what has happened: in order to maintain revenues, providers are shifting the costs to the private payers. This cost shifting has been known in the healthcare world for years, but it is getting worse and worse. It represents a sort of hidden tax on all of us, and it is one of the things driving healthcare costs even higher.
I recommend the editorial. It’s short and easy to understand. It’s also eye-opening as to the magnitude of the cost-shifting phenomenon.
When most people go into the hospital it does not occur to them that, here in America, an acute scarcity of a standard medication will affect their health. But they would be wrong. Sudden, random, and dangerous shortages of key, life-saving medicines are happening increasingly frequently. For example, there is this recent shortage of chemotherapeutic drugs to treat cancer. We have also had recent shortages of such commonly used PICU drugs as phenobarbital and propofol. The latest shortage is with fentanyl, a synthetic narcotic pain-killer. I really need fentanyl in my practice to relieve pain and sedate children. There are other narcotics we can use, but none have the special attributes of fentanyl that make it, in experienced hands of course, a safe and key PICU medication.
Why do we have these drug shortages? Where do they come from? All of these drugs share the attribute of being generic medications. This means the patent has expired on them and any pharmaceutical company can choose to make and sell them. They can, but they won’t make much money doing so, because generic drugs are much, much cheaper than are ones still under patent. Injectable drugs, the drugs I and my ICU colleagues use every day, are especially expensive to make because they must be sterile. The result is that, for the majority of injectable generic drugs, there may only be a single company with a single factory even making it. If there is a problem at the factory, everything stops. And these are not esoteric drugs; they are ones hospital physicians rely on every day. People die when we don’t have them. The upshot is that our care of critically ill patients depends upon whether or not the roof of a factory somewhere leaks or not.
I don’t know what the answer to this problem is, but it is a serious danger. There are so-called “orphan drug” incentives to encourage drug companies to make rarely needed drugs that are essential for people with rare disorders. Since there is only a tiny market for such drugs, companies otherwise wouldn’t make them. To safeguard our nation’s injectable drug supply, I think we should figure out some sort of similar system for injectable generic drugs. The problem is getting worse, not better.
Update: President Obama, through the FDA, has taken steps to help the situation, offering incentives for companies to make injectable generic medications. You can read details here.
Another update: This editorial in the New England Journal of Medicine makes it clear: shortages of all manner of generic injectable drugs are popping up. These these shortages will cause people to die, if they haven’t already. For those lovers of the free market as the best way to practice medicine in America, this example shows how mistaken that viewpoint is.
It’s well known that an increasing number of Americans are overweight, and many more of those are frankly obese. The reasons are several. We have a more sedentary lifestyle and fewer and fewer of us get regular exercise. Our caloric intake, on average, has been steadily rising for decades, and many of us now eat the same number of calories as a person who a century ago did hard manual labor for 12 hours each day. Evolutionary biologists tell us that, in effect, our metabolism is programmed for how we lived 10,000 years ago, when our ancestors had infrequent, large meals and needed to store energy between them because the interval might be days. Now we have frequent large meals every day. As a result, our caloric balance is slanted toward accumulation, and we get fat.
The statistics for adolescent obesity are discouraging. The Centers for Disease Control says that 17% of our children are now obese, defined as a body mass index (BMI) of greater than 30. Because of the dismal results of traditional behavioral methods of weight contol in adolescents, more than a few experts are urging us to consider the role of weight-control surgery for so-called morbid obesity in children. It seems a bit extreme at first blush, but think about it: what we have to offer medically to these children virtually always fails, and we know that continued morbid obesity is a true threat to their lifes — not just in the long-term, but even in the medium-term. Why not do bariatric surgery on them?
The issue, of course, is what data do we have that this sort of surgery has any better long-term outcome than medical therapy. That is, does it work? Thinking of the risk-benefit ratio, is the short-term risk of surgery (which is not trivial) worth the chances of long-term success? A recent editorial in the New England Journal of Medicine asks that question. The answer is that we don’t know, but we should find out. We should offer the procedure to carefully selected cases, and keep track of how well it works out for them. From the editorial:
Will bariatric surgery in the young prove more effective over time than other less-invasive approaches? Given the health effects of massive obesity, many practitioners believe that weight-loss surgery is a better alternative than watching a morbidly obese youngster develop myriad complications. All adolescents must navigate the journey into adult life, and those who have undergone bariatric surgery will need to adhere to a stringent diet and medication and exercise regimens for the rest of their lives. There may be as-yet-unknown adverse effects of the surgery — for example, effects on bone density over decades. We also have much to learn about why bariatric surgery leads to a recalibration of the weight set point. It appears that bariatric surgery for adolescents has caught on, whether “right” or “wrong.” But the current strict requirements for having a bariatric procedure should not be relaxed until we know more.
Parents, and physicians, too, are increasingly concerned about the potential long-term effects on children of radiation from diagnostic procedures. We shouldn’t irrationally fear radiation from ordinary x-rays or CT scans, but we should use these tests judiciously — when the benefit of getting the information from them outweighs the still very small risk of doing them. Of course we should always have been doing things that way; the latest evidence reenforces what we’ve always known, but have not always acted upon. Some new recommendations about managing urinary tract infections (UTIs) in young children will allow us to reduce radiation exposure.
UTIs are not uncommon in children. They come in two forms. The less serious, and more common form, is an infection of only the bladder, called cystitis; the more serious, and less common form, is infection that involves the kidneys, called pyelonephritis. Both are generally caused by several bacteria that are normal inhabitants of the intestinal tract, particularly one called E. coli.
It has been known for many years that children with structural abnormalities of the urinary tract are especially susceptible to getting a UTI. These abnormalities can be misconnections of various parts of the urine drainage system, but the most common one is backward movement (reflux) of urine from the bladder upstream back to the kidneys. In spite of the association of these abnormalities with UTIs, the majority of children who get a UTI have a completely normal urinary tract.
For many years, certainly the 35 years I have practiced, children who had a UTI were checked to see if they had an abnormal urinary tract. The usual practice was to check boys after their first UTI, girls if they had a second one. One of the standard tests to do this was called a voiding cystourethrogram, or VCUG. This test is a radiological one, and it exposes children to radiation. How much radiation? Unlike many radiological procedures, the VCUG employs fluoroscopy, which means that the amount of radiation varies a bit from patient to patient because the amount of fluoroscopy time can vary with the operator. On average, though, the amount of radiation from a VCUG is anywhere from 10 to 20 times that of a simple chest x-ray. You can think of a VCUG as giving about the same amount of radiation as we get from background radiation from living about 6 months at sea level.
Radiologists have made a lot of progress in limiting the amount of radiation they need to do x-rays in children. But better than reducing radiation is not needing it at all, and that is what the American Academy of Pediatrics now recommends for evaluation of most children with a UTI. Some children will still need a VCUG, mainly those who have repeated UTIs, but for most children the most useful information can be obtained by ultrasound, which does not carry any radiation. You can read their recommendations here.
Why does the AAP no longer recommend a VCUG for most children? The answer is a good example of an important principle in medicine — balancing risk vs. benefit. The VCUG does give information, but it turns out that the information, although interesting, is not crucial in actually deciding to do for the child after their first infection. So the risk of the VCUG, although very, very tiny, brings no real benefit. So even that small risk is not worth taking.
There is a long tradition in folklore, one shared by shamans and occultists, that knowing the true name of something gives you power over it.
Many years ago I had a very sick patient in the PICU who one morning, totally out of the blue, broke out in a bright, red rash all over his body. The boy had many critical problems already and, although the rash didn’t seem to be causing him any additional difficulties, it was dramatic. I worked my way down the list of usual things that cause such rashes and nothing seemed likely, so I asked a skin expert, a dermatologist, to come and take a look at it.
The dermatologist was a distinguished professor with an international reputation. He arrived in his customary three-piece suit with a large entourage of residents and students. He gravely looked at the rash, removed his half-glasses, turned toward his accompanying crowd, and pronounced that the child had ” erythroderma universalis.” I was then a bit rash myself at times, so I demanded something like: “I can see his skin is red all over (which is what erythroderma universalis means translated into English), but what is the rash from and what should I do about it?” The professor was not amused. He had named the thing — that alone was useful and important.
What I call “Rumpelstiltskin syndrome” is the long tradition in medicine that merely putting a name to a disorder, for example a set of symptoms, goes a long way toward controlling the problem, because it gives our minds power over it. It is a little like the fairy tale in which Rumpelstiltskin conceals his name from the miller’s daughter. She is in his power until she happens to learn it, after which she is on top of the situation. No one wants to be a diagnostic enigma — we feel better when we have a name to call our malady, even if we can’t do anything about it.
Classifying diseases and relating them to one another, and in the process assigning them names, is an ancient tradition in medicine. Called nosology, physicians tried, like botanists studying plants, to derive a sort of family tree of diseases based upon observed characteristics. Even if there was no effective treatment, at least naming the disease would, they thought, allow prognosis, the art of predicting what will happen to the patient. That is a useful thing. For a very long time in medical history it was nearly all physicians had to offer.
We still use the Rumpelstiltskin approach when we confront an entirely new thing, because it can be a useful first step in figuring out the cause of a disease. For example, before we knew anything about the human immunodeficiency virus (HIV), clinicians had identified patients with an unusual cluster of symptoms and signs and called the problem acquired immunodeficiency syndrome, or AIDS. Now we know what causes AIDS, but at first all we could do was describe it and name it.
So, like the miller’s daughter and Rumpelstiltskin, knowing the ‘true name’ of a medical condition is helpful in understanding it. But it also is true that that the simple naming act makes us feel better about the situation, if only because now we know what to call it. In that respect we are little different from those diligent nosologists who labored hundreds of years ago to identify the intricate differences between various fevers, thinking they were, in the process, describing different diseases.
Medicine is particularly prone to be trapped by the well-known fallacy with the fancy Latin label of post hoc, ergo propter hoc — or, after this, therefore because of this. This is because, when a patient comes to us with a particular problem, physicians generally do things, like prescribing medicines and do procedures. We intend for what we do to help the situation, but often there is no way to tell if it has. The patient may improve; or the patient may not. What follows may be related to our treatment, or it may not be.
Even if there is good scientific evidence that a treatment works, that evidence generally relates to populations of people, not an individual. And individuals are, well, individual. A person is not a statistic. So if a treatment is a really, really good one, for example one that helps 90% of the time, 10% of the time it won’t help. It might even make the condition worse for some individuals.
Anyone who has watched late-night cable television has seen countless examples of this logical trap, in the form of personal testimonials from people who had this or that problem, took the pill or bought the product, and the problem went away. The fallacy, of course, is that the two events may be entirely unrelated, just as the fact I may drink coffee every morning before the sun comes up does not cause the heavens to move in that way. I’ve written about this in a little more detail here.
This uncertainty can drive a precise mind a little crazy. But for me it’s one of the things that makes practicing medicine fascinating. Medicine isn’t a science: it’s a mishmash of science, near-science, educated guessing, and blind luck. Sort of like life.
(Thanks to MacAllister Stone for the link to the pithy illustration of this dilemma.)
Personal experience is how we learn things, so individual anecdotes have enduring power in how we humans understand and explain things. I suppose we should not be surprised by this. After all, an anecdote is a story, and humans are story-tellers by nature.
These days physicians are exhorted to use only the hardest of hard evidence to make decisions, to use only what is called evidence-based medicine. Some think we should be compelled to do this by means of things such as practice guidelines. I have no objection to this in principle (who would?), but there is a problem with it: for much of what we do, even in the high-tech environment of the PICU, there are little (or even no) evidence-based guidelines to use. We do what we think is best based upon what we have been taught and what has worked in the past. And we use anecdotes — stories we have heard or things we have seen.
Rafael Campo, the award-winning physician and poet, has some interesting things to say here about the power of the anecdote, the human story. At the end of one of Dr. Campo’s lectures, a distinguished physician posed this question — or challenge, really:
“Do you really expect physicians to accept the notion that what any ignorant patient tells us about his disease should carry a weight equal to what our years of training and expertise reveals to us about complex pathophysiology?” Then came what was clearly meant to be his coup de grace, delivered in an almost derisive tone. “Really, sir, do you have anything more than the anecdotal evidence you shared to support your thesis?”
For myself, I continue to see medicine as a complicated mish-mask of science, near-science, intuition, guesswork, and blind luck. Although we should always use the best science we can, somewhere in the mix there is a place for the anecdote, the story. I wouldn’t want it to be any other way.
