PART THREE OF DIAGNOSIS
Focus on Symptomatic Relief
Introduction to Clinical Trials
Complementary And Alternative Medicines Overview
Antidotes For Anecdotes
Prognosis And The Future
Taking Your Time, Avoid Timelines
Autonomy; Everything Begins And Ends There
A Final Few Words
Doctors must pay attention to cultural differences. People from different backgrounds and parts of the world have very different ways of relating to the physician. This may range from deification, rarely some level of disdain and even total acquiescence and deference. Despite the variability, it always remains wise for doctors to engage their patients as the folks in charge and the doctor as the expert consultant who will not allow avoidable errors in decision-making. Patient autonomy is pivotal to a successful treatment road. Providers must stress who is and is not God and make it clear that it is not the doctor. This is at times unnerving but usually surprisingly very well received.
Focus On Symptomatic Relief
A difficult scenario arises when in rendering the diagnosis it is also clear that only symptomatic or so-called palliative care is available. Sometimes diseases are so advanced or resistant to treatment that the discussion may need a predominant focus on relief of symptoms. Sometimes, the patients’ general medical condition makes it reasonable to have such a focus. On occasion, a well-informed, competent patient may choose only symptomatic relief for any number of reasons. Thus, discussing the option of symptomatic care is always germane.
`The patient and physician must be mutually clear as to what the odds are for a response, its duration, the odds and nature of relapse or progression and the costs- psychologically, physically and financially. The first step is making it clear that such care refers to improvements in symptoms, irrespective of temporary changes in blood tests or other diagnostic studies. Otherwise, expenses and anxiety can grow exponentially and perhaps unnecessarily while temporarily inflated hopes die on the rocks of reality.
Introducing Clinical Trials
Although I cover clinical trials separately later, the issues initially arise when explaining either the diagnosis or the rationale for proposed treatment. Trials are a perfect match for some personalities, but the whole idea of “experiment” or randomization (explained later), no matter how carefully couched, can be frightening. This is a major dilemma for medicine in general as clearly this is a major way in which treatments are developed yet less than 5% of all eligible patients enroll in clinical trials. This is particularly sad when considering that these trials are the only valid means of determining a treatment’s effectiveness.
When there is an opportunity to offer a patient a trial, it is wise to follow a common sense rule; be sure the patient knows exactly what is happening at all times and always provide a copy to the patient, their primary care provider as well as place one in their chart. One cannot predict who will volunteer for trials. The ethical rule is straightforward as all ethical rules should be. Physicians should present the trial in terms of a balance of what other therapy is available with possible results, the goods that the trial may offer and the harms that the patient may experience from either the trial or other therapy. Secondly, the patient must give a fully informed consent after significant time to reflect and have their questions answered.
The trials have to go through all manner of review boards and must be among the next best questions to ask. Your credentialed physician or anywhere near you may not have a trial for which you are qualified. No worries, the issue is to receive at least the standard of care and happily essentially all U.S. trained U.S. board certified oncologists deliver it. Sadly, fewer than 15% of those who could be on a trial enroll but that is getting better. You get all manners of scrutiny from being on one for obvious reasons and if they are level three or higher you are guaranteed receiving a treatment as good as the standard of care. The trial is asking if there is something we can do better.
Introduction to Complementary and Alternative Medicine
It is imperative that physicians explain, using every form of metaphor, simile, parable, analogy or allegory, how medicine has learned what it has about a disease over the years and that this occurs usually because of carefully planned research. There are notable unplanned observations leading to breakthroughs, but in cancer care, they are the exception. Patients need to understand when there is certainty based on vast data versus when there is only suggestive information. Successful clinical trials involved many patients and showed how groups similar to an individual patient fared. However, individual patients, although likely to behave like the ones in a similar group, are still individuals
Sadly, largely owing to desperation, non-approved (non-FDA, non-National Cancer Institute) treatment regimens can find an easy mark in the cancer patient owing to unscrupulous practitioners offering savory hints of wondrous results. Patients are often easy prey for quackery. That is why I have included a special section on Complementary and Alternative Medicine (CAM). Patients and families spend billions on unproven therapies every year. Significant portions of cancer patients try CAM while most at least listen to if not solicit information regarding it. There are rarely corroborated results of success that can withstand scientific scrutiny. The reason is simple. There is no financial or political-legal pressure to do so. The “claims” that do exist regarding wondrous results from CAM raise more questions than answers. The questions they raise are of massive importance. Each must be addressed at the time of rendering the diagnosis to a patient or shortly thereafter as the issue of trying CAM will undoubtedly arise.
Quackery always has a pitch which many cancer patients will struggle with if left on their own such as “ “medicine is hiding the real cure because of profit and bad pharmaceutical companies”; “we have the secret”; “you can do this only by fresh air, diet and exercise; there is no need for ‘bad’, nasty, poisonous medicine or radiation. That is sheer nonsense if one calmly walks through this both in this section and the one that follows. I also refer the reader to the NCI site, Mayo clinic’s cancer site and www.QuackWatch.org .
The major issues CAM raises are these. How do we know if responses that are real are due to the alleged CAM therapy? How do we know if these alleged responses are durable and are reproducible in most patients and can be applied to a given patients’ situation? What monitoring is necessary? What are the toxicities of these therapies and are they compatible with conventional therapy? How do these CAM therapies truly compare to conventional therapy? Head to head well-done comparisons are rare.
At the very least, I recommend knowledge of everything that enters a patients’ body. Although I stand firm as regards the right of a patient to autonomy, I believe the right of these purveyors of unproven potions to hawk their wares must remain under constant scrutiny. I sometimes simply refuse therapy and state it must be one or the other and patients must chose. Although the patient may abandon conventional therapy, the physician should give assurances that they will not abandon the patient. The best way to fight quackery is with facts and calm, critical thinking. It is sometimes an uphill battle in appearing as physicians protecting their own turf. So be it.
There is little complimentary (with an “I”) to say about complementary and alternative medicine in patients with diagnosed malignancy. Level heads with tens of years of experience cannot say, I know this cannot harm you. Proponents also cannot say I know this is safe and effective to take against your cancer. They cannot say they know the exact ingredients in their concoction. Supporters of CAM may not even know the purity, other ingredients, and effects from processing (since none of this is required) and they do not know how it interacts with all other chemotherapy drugs as tested under the rigors of clinical trials. Wake up, hear the call of your anxiety and stop the craziness. CAM adherents cannot tell you any of those things because they do not know them.
Sellers of CAM routinely do not do any testing except making sure your credit cards and checks are good. Harsh? Not harsh enough. With rare exception, you will not see them go through complete scrutiny of the FDA. At best, they will get FDA statements of purity of ingredients but that is all. One hundred percent of nothing that works is not necessarily nothing. Fact is, it may be something that hurts you and unless the FDA has thoroughly investigated them and stated they are safe and effective, beware.
Be aware that once a drug is proven safe and effective by the FDA for one indication it is not illegal to use it as doctors see fit to use it for other off label uses based on well-scrutinized peer reviewed literature. This has lead to well reasoned application of drugs off label. This is not wrong and you will find these are all building blocks for the most part of intelligent studies showing their benefit.
When some substance looks promising from the plant and animal kingdom it is tested, not held back by some evil conspiracy. Think about how self-defeating that is. If it is sufficiently non toxic and has some mechanisms of action that make sense and some minimal response rate, it will find its way into early level trials described later. A number of our front line drugs exists today from the plant and animal kingdom. However this is after extensive years of testing. Ignore the shaman and the gurus hawking claims they may really believe as they have no high quality data nor relevant training. If what they had worked, it would not be complementary medicine, it would be front line.
There is also no rational reason to support any of the nonsense that “the man” or “Big Pharma” or whatever is holding CAM back. Ridiculous! Capitalism is doing just what one wants it to do; reward the real deal that will go the distance and not kill people or usurp their hope and resources. The system is not perfect but it is, without any equivocation, the best in the world for declaring a new drug as safe and effective. Moreover, our system does the right thing by limiting patents, expediting highly promising drugs and creating special tracks for enormously promising ones. The “Man” or “Big Pharma” would be idiots and bankrupt to proceed in any other way.
Furthermore, those of you who know how to read an annual report or balance sheet should ponder this. It takes almost 7 years for a new drug to go to marketing as safe and effective and over $600 million dollars. Consider how much is spent on non-winners. Profit margins, not profits but the real numbers such as how much is finally left after everything is paid for range from negative 7-10% in bad years and 5-20% in good and overall about average 6-9%. Nobody is routinely getting ripped off.
The cost in lives, pain and suffering, manpower, health care cost dollars wasted and clogging up the system because of CAM barkers and naive folks exercising their admittedly free choice is outrageous. Many put it as a ratio of one out of every 6 dollars is wasted in Oncology by the pursuit of, application of, and costs from using complementary or alternative medicine.
Beware mass media, they are often hit and run news du jour artists without the time, temperament or talent to clearly put any news or breakthroughs in perspective let alone in individualized terms . Sometimes they get a lot right, especially when they are fact based stories and not predominantly emotional content. Some incredible network as well as cable television shows highlight the exception of a bad physician and run the story without comments of the overwhelmingly good data out there when it comes to the constancy and quality of Oncology care.
Yes, the media have a crucial and welcome role to help watch, encourage and support and in a sense, police the field for the betterment of all. But remember their job is not meet you in the E.R., take your calls at three a.m., work with other consultants, calculate your chemotherapy, talk with the family and be the responsible individual from rumor to tumor to treat and beyond.
Antidotes For Anecdotes
One of the biggest poisons in our field is the anecdote. It is the irresponsible presentation without hard verified, in-context data of some patient response or treatment with an against all odds outcome or surprise ending ( such things happen rarely) that the storyteller or listeners then wrong headedly thinks present new rules and lessons to all broadly or especially to them.
Anecdotal medicine frequently causes suffering and in oncology where margins are slimmer, it can cause death.
For example- all seem to think if you flip a penny ten times and get 3 heads and then 7 tails in a row that tails is favored. No, not even if you got the same ratio doing it again.
Prognosis and the Future
It may or may not be premature to discuss prognosis early on. This is a case-by-case situation. It can be very confusing at first to present to individuals how groups have handled highly similar situations. Using comparative hypothetical or historical groups to predict an individual’s prognosis can be relevant and helpful but it is not a crystal ball into the individual patients’ future. No one is to be hopeless. I have seen enough surprise turns to the positive to jewel a crown with joy rather than be jaded by some anchor around my heart. Odds are things patients’ beat from time to time.
Oncologists and patients would be wise to outline and explain the potentially long list of experts needed in future care early in the journey. The list may be long, confusing and frightening. It may include ostomy nurses and physicians needed to perform further biopsies, diagnostic tests or radiation. Oncologists must prepare their patients for the entourage in their service; rather than position patients for a frightening flood of new doctors and procedures.
Take the Time and Avoid Timelines
Most importantly and least easily done, rendering the diagnosis takes time. The vagaries of managed care and reimbursement issues have private oncologists seeing enormous numbers of patients per day. This can be insane and inhumane. It is easy and understandable to state that there simply is not the time. Somehow, some way, there must be. In the end, spending the time will eventually save time. After all, it may be all the doctor has to give at some point.
Oncologists must not give Hollywood time lines. The best approach is to refer to the literature and the ranges that a patient most likely fits while stressing once again that patients are individuals, not statistics. Patients and providers should avoid attempting to quote dates or make dramatic pronouncements. That is pandering to fear and fatalism.
Odds should be explained, both as relative and absolute odds. As an example of this explanation, I will relate what I do to illuminate these crucial concepts to the patient. I take out a dime, a penny, and a one and ten dollar bill in front of the patient. The difference between the penny and the dime in “relative” value is that the penny is one tenth of the dime. The same is true for one and ten dollars. The one dollar is one tenth of the ten dollars. However, the difference between the “absolute” amount of ten cents and one cent is nine cents. The difference in absolute terms between the ten dollars and one dollar is nine dollars. Nine dollars is absolutely much larger than nine cents yet nine cents is 90%, relatively speaking, of ten cents as nine dollars in 90%, relatively speaking, is of ten dollars. However, the absolute difference between nine cents and nine dollars is absolutely quite large.
Why care? A ten percent improvement in survival or response or duration of response can vary greatly depending on what odds you started with. This concept is crucial when discussing adjuvant therapy. This is treatment given when statistics say there are high odds of recurrence of unseen lurking disease after apparent removal of the primary cancer and no visible evidence of cancer cells remaining anywhere. If adjuvant therapy is given, it is because it has a certain relative and absolute risk of preventing recurrence at some future point in time.
Oncologists offer many breast cancer patients adjuvant therapy, treatment after the primary surgery to kill hidden distant disease. The concepts of absolute and relative risk play a large role in this decision. It is crucial to know what the real difference, the absolute difference is between those treated adjuvantly and those not. A ten percent improvement in the odds of recurrence, when the odds of recurrence are only ten percent, is just an improvement from ten to eleven percent. However, a ten percent improvement of odds of recurrence when the odds of recurrence were eighty percent is the difference between eight and eighty-eight. That is eight percent. That is eight more people potentially alive out of one hundred.
Understanding basic statistical language does not end there and the education to build understanding begins at the time of giving the diagnosis. Patients must be able to understand what the percent improvement stated above really refer. Is it an improvement in the odds of response to therapy or the risk of recurrence? Does it translate into a ten percent improvement in the time to recurrence? Is it referring to a ten percent improvement in the odds of survival? Does it translate into how long one lives before eventually dying from the disease? On the other hand, and as is commonly the case, does the ten percent apply to some, but not all, of these very different notions? These are all very large, quality of life, practical issues and they are very different concepts.
Remember Statistics Can Be Your Friends
Statistics tell us number needed to treat to tell you if a study had any ability or power to tell you anything.
Statistics also give us wondrous definitions like the level of power ( predicting the truth) of published studies. They tell you if you can definitely say yes or no is true with some degree of certainty and if the type of study is rock solid and the evidence for it.
They tell you if you can know the odds of an outcome by others are likely or not for you.
They are so important they are discussed in multiple areas of the book.
I address second opinions in the legal section in detail but a few points are germane when initially discussing the diagnosis. They should be encouraged for all patients and families who broach the subject. A word to the wise; such second opinions may be for therapy that is either far more or less aggressive than what was originally offered. Even more rarely, some who render second opinions may libel or slander the primary physician to the family and not condone what the primary doctor has done so far. Therefore, prudence, candor and pre planning is wise when assisting the patient obtain second opinions or commenting on them. The only significant problems I have ever encountered in this regard were when complementary or alternative medicine practitioners get into the mix. This dilemma is addressed more fully in a later chapter.
Although stated later in the legal section, it bears mentioning here that patients are expecting a contract and the list of what is required is long: They expect doctors to listen, to teach, to be available, and to address pain (almost all pain can be effectively treated), get help with sleep and bowel habits and prevent nausea, and vomiting. Patients need to hear that we now have medications that may increase appetite and stamina. Providers must tell the truth and be clear on the schedule of progress reports. An often-overlooked area is the golden rule that doctors must never “keep secrets” from the patient no matter what the significant other or family says. Patients must always be clear on what their policy is regarding messages and answering machines and receivers of physician calls who are other than the patient.
Remember Autonomy – It Begins And Ends There
Will the real patient stand up? It is you but you are not your disease. YOU must have a relationship with your disease built on knowledge to lower your burden. There is immense power in the gift of autonomy as described earlier in the book. Go back and read the first few chapters again and remember this is your journey and you call the steps.
A Final Few Words
One of the deadliest weapons both against the cancer and unwittingly against the patient and family is not chemotherapy; it is the physicians’ mouth. The clinician must not only think about what they are about to say, but say only what they are thinking-no more and no less. For example, more than once, I have heard stories of the devastating blow caused by an answering machine message thoughtlessly left for all to hear and often misunderstand.
In sum, this chapter is simply saying that the tongue, as well as the cancer, can be the “enemy of the neck” or the liberator of the patient’s autonomous license to engage fully and vigorously in their treatments.