2014 Eeric Truumees, MD

AAOS Now

Published 7/1/2018
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Eeric Truumees, MD

Blurred Lines

The challenge of conflicting or limited evidence in orthopaedic care

I recently returned from the AAOS National Orthopaedic Leadership Conference (NOLC) in Washington, D.C. As always, I heard several great symposia, floor discussions, and debates. It started with an excellent presentation by AAOS Senior Manager of Biomedical Research and Regulation Katherine Sale, MPH, who discussed the current state of stem cell utilization in the United States. In AAOS Now, we have covered stem cells in the past and will certainly do so in the future. As I listened to the ensuing days’ discussions, I found that many of the controversies involve areas where the evidence base remains limited or contentious.

Recognizing that the evidence base for much of what might be called orthodox orthopaedic surgery remains suboptimal, it seems that there is a spectrum of evidentiary support. Of course, that’s not new; there has always been tension between the cutting edge and the tried and true. Each practice may lie on a different point along that spectrum (and their tendencies, like mine, may change over time). In discussion, some tend to take a harsh view of those practicing more aggressive or more conservative orthopaedics. It’s like driving: Anyone going faster than I am is a reckless idiot, and anyone going too slow is a bumbling moron.

As much as the NOLC attendees can argue about hyaluronate (HA) injections, cell-containing allografts, vertebroplasty, or knee arthroscopy for degenerative meniscal tears, we know that, for patients, it’s a jungle out there. It’s very difficult for the average layperson to understand the difference between the HA debate and amniotic tissue stem cell injections.

Medical organizations

Among individual practitioners, these differences are often left unsaid or, at most, amount to petty squabbles. However, when medical organizations get involved, the stakes change. Even if you are inclined to “toe the line” for evidence-based medicine, your institution may not. Many prominent medical institutions tout evidence-based medicine yet also offer “institutes of alternative and complementary care.”

In 2017, PBS NewsHour covered the “aggressive promotion” of alternative therapies by healthcare systems with strong reputations for scientific research, such as Yale, Duke, and Johns Hopkins. The story described “luxurious, spa-like wellness centers” meant to attract patients. In some cases, the model requires yearly membership—$1,800 a year at Duke.

The Duke Integrative Medicine store, for instance, sells “Po Chai pills” that are touted on the website as a cure for everything from belching to hangovers to headaches. The site explains that the pill “harmonizes the stomach, stems counterflow ascent of stomach qi, dispels damp, dispels pathogenic factors, subdues yang, [and] relieves pain.”

PBS NewsHour noted that even when integrative medicine clinics operate at a loss, hospital administrators maintain the programs because they are “essential to woo patients in a highly competitive marketplace. If they failed to offer ‘natural’ therapies … hospital executives fear they would lose a chance to attract patients who need more lucrative care, such as orthopaedic surgeries or cancer treatments.”

Last year, we covered the kerfuffle surrounding the Cleveland Clinic Wellness Institute’s director supporting the discredited theories linking vaccines to autism. In response, Cleveland Clinic made some staff and operational changes. Yet, on its website, Chief Executive Officer Toby Cosgrove wrote, “The old way of combating chronic disease hasn’t worked. … We have heard from our patients that they want more than conventional medicine can offer.”

Yes, that is true, but should we, as allopathic physicians, be the ones providing “more than conventional medicine can offer?” We’ve never claimed to be all things to all people. In fact, one of the most frequent but frustrating conversations I have with patients is that the skills I learned in training will not help them. Nothing in that statement implies they don’t need help, and I try to arrange it for them. But offering surgery plus some unproven, add-on element is not the answer either. Should we, instead, concentrate on how we can improve what allopathic medicine has to offer?

One telling quote in the PBS story came from Jill Schneiderhan, MD, codirector of the University of Michigan’s integrative family medicine program. She said, “We do use things that aren’t necessarily 100 percent evidence-based, but I would argue that’s also true within all of medicine.” She is correct: The level of evidence for what we do varies considerably. So where do we draw the line?

At NOLC, some of the debate was around the appropriate use criteria (AUCs) published by AAOS. The AAOS mission is “serving our profession to provide the highest quality musculoskeletal care,” which artfully balances our desire to improve musculoskeletal care for our patients versus some members’ expectation that AAOS acts like a trade organization.

When a member’s preferred treatment is “not recommended,” the response can be swift and negative. Many such members simply are not aware of the countless hours their peers spend rigorously and impartially evaluating the evidence. Others have what may be legitimate concerns about the evidence synthesis, GRADE (Grading of Recommendations Assessment, Development, and Evaluation) methodology, exclusion criteria, statistically significant effect versus clinically significant effect, and more.

But the fact that third-party payers use the AUCs, which are not meant to be proscriptive, as an excuse to deny coverage raises the greatest ire of our members. Moving forward, the AAOS Council on Research and Quality will place greater emphasis on communication with the membership and include a wider range of stakeholders in their discussions.

Although these coverage issues may have a significant impact on daily practice, AAOS fellows should be aware that state medical boards and other authorities are taking an increased interest in the evidence base around care offerings, with a particular focus on stem cells. At NOLC, Board of Councilors representatives from North Dakota explained how a stem cell clinic in their state was closed. They worked with the state attorney general’s division of consumer protection. They found that when injured patients contacted the attorney general, there was a greater response.

Additionally, the Federation of State Medical Boards working group reported on stem cells and regenerative medicine, including the problem of direct-to-consumer clinics. In February, 17 of 51 medical boards had responded to complaints against regenerative medicine or stem cell therapy clinics, with eight taking disciplinary action against physicians running such clinics. Their draft recommendations focus on MDs practicing outside their usual area of expertise, those using gag orders to squelch online complaints, and those charging excessive fees.

Problem areas

Although our efforts to educate patients are crucial, many have preconceived notions based on advertising and their own online “research.” Sometimes patients will offer self-diagnosis and preferred treatment, expecting you to execute it. Sometimes they are right, but unfortunately, more often they are wrong. The procedure or drug makes sense to them, even when there is little supporting data.

This is particularly true when our surgical solutions are morbid or associated with lifelong changes, such as spine surgery and joint replacement. In some cases, treatment options do not reflect the condition’s impact on the patient’s life. For example, with osteoarthritis, we may have several “little hammers” like nonsteroidal anti-inflammatory drugs and physical therapy and some “big hammers” like joint replacement and spinal fusion—but little in the middle. Frustration with treatment choices pushes some patients into alternative care pathways like “laser spine surgery” and stem cell injections. Some might see widespread utilization of arthroscopy for degenerative meniscal problems in this vein.

An article by David Epstein and ProPublica, published in The Atlantic in February 2017, detailed a variety of studies suggesting that arthroscopic meniscectomy held little utility for older patients with knee arthritis. The article quoted Pittsburgh orthopaedic surgeon John Christoforetti, MD, who noted that some subsets of patients may benefit from arthroscopy but many others do not. The article reported that, even when surgeons are not eager to offer the procedure, “many patients … want, or even demand, to be operated upon and will simply shop around until they find a willing doctor.”

The knee-jerk response is that we must take the time to properly engage our patients. We must provide them with evidence-based treatment options, including the risks, benefits, and alternatives of the approach recommended. But we know this does not always work and, in some settings, may be impractical.

Dr. Christoforetti detailed one couple’s response: A woman and her husband had traveled to see him, but he explained to them that surgery would not help. As they left the office, the husband posted a one-star review, declaring that Dr. Christoforetti is an “insensitive guy” who he “wouldn’t let operate on his dog.” Given the possible impact on satisfaction scores and the financial incentive to operate, Dr. Christoforetti noted, “Most of my colleagues will say: ‘Look, save yourself the headache, just do the surgery. None of us are going to be upset with you for doing the surgery. Your bank account is not going to be upset with you. Just do the surgery.’”

As a spinal surgeon, I know that sometimes the diagnosis itself is in question. When presented with nonspecific back pain, telling a patient that “I don’t know” is not satisfying, and there is significant pressure to offer some “real” diagnosis. Chronic Lyme disease is one of those diagnoses often attached to patients with ambiguous symptoms, typically including fatigue, generalized pain, and neurological symptoms. After performing their “online research,” patients often seek out “experts” in chronic Lyme disease and are therefore able to get the treatment they want. Online, these doctors are often described as “Lyme literate.”

Often, we shrug off these clinics and other alternative approaches, noting, “as long as it doesn’t hurt anyone.” Unfortunately, the risks may be more serious than previously understood. For chronic Lyme disease, the frequently recommended, months-long courses of intravenous antibiotics can result in serious harm. Although data describing the numbers of patients undergoing such treatments are not available, there are increasing reports of misdiagnosis. For example, a woman later diagnosed with amyotrophic lateral sclerosis (ALS) received several months of antimicrobial agents, including antifungals. Unfortunately, before she died of ALS, she experienced two years of Clostridium difficile infection with severe cramps and diarrhea.

Right to try

Reasonable orthopaedic surgeons debate how far they should go in giving patients what they want. Interestingly, I have found that even ethicists struggle over the question: When a physician refuses to comply with a patient’s request, is he restricting his or her autonomy?

The American Medical Association’s Virtual Mentor blog explains that, as physicians, we have far more training in dealing with a patient’s “negative rights” (e.g., the right to refuse a test or procedure). We have an incomplete understanding of positive rights (e.g., when a patient demands a given treatment).

In a New York Times article published in 2007, Sandeep Jauhar, MD, explained, “Ethically, given the relative dependency of the patient’s position, it has been argued that their preferences should be respected if their choice, even if not optimal, is reasonable. Doctors should decline to offer clearly suboptimal care.” But what is suboptimal care? He noted the 1991 Minnesota case of Helga Wanglie, who was 86 and in a coma. Her intensive care unit physicians planned to withdraw what they saw as futile care, but her family sued and won the right to maintain life support.

Our politics seem to be headed in the same direction. As of May 30, President Donald Trump signed a federal right-to-try bill into law, which allows terminally ill patients to bypass the U.S. Food and Drug Administration and request an investigational drug directly from the manufacturer. For proponents, this makes sense: If your loved one has a deadly disease with no good treatment options, why wouldn’t you want to try a potentially curative agent? Critics cite the difficulties in monitoring the risks when physicians are removed from the treatment plan. They note that, although the law protects manufacturers from liability, most major drug and implant manufacturers are not interested in such programs.

Even though a manufacturer can charge what it wants for an experimental treatment, it is never covered by insurance, and the administrative costs and the impact on limited supplies of the drug or implant adversely impact the manufacturer’s main focus: the clinical trial. Instead, fly-by-night purveyors are more likely to prey on vulnerable patients and their families.

Critics also report that the risks and unintended consequences of experimental agents are rarely understood by patients. For example, treatment with a potentially curative agent may limit access to hospice and other valuable services.

For orthopaedic surgeons, the argument is usually a little different: “This treatment may not work, but it’s not likely to hurt anything.” Key concepts in the patient-centered care model include shared decision-making and treating the “whole patient.” This process has led to the wholesale incorporation of complementary and alternative care into previously traditional, allopathic practices. But how far should we allow patient choice to be the ultimate arbiter? In his blog, David W. Goodman, MD, wrote that there are too many areas in which “patients are generally not adequately informed, knowledgeable, or, worse, misinformed to make the best informed decision. This assertion of patient choice leaves the patient vulnerable to the sales pitch of unproven treatments. This is not to say patients shouldn’t make their own choice of treatment, but it should be born out of a reasonable understanding of pros and cons of each treatment option from reliable sources of information. Let science be the designated driver on the highway of opinions.”

Although the federal law focuses on terminal illness, 40 states have their own right-to-try laws. In 2017, Texas legislature passed and the governor signed a new right-to-try bill that extended well beyond fatal diseases to other chronic conditions. Texas already had a law that supported the right to try for terminally ill patients. The new law focuses on stem cells for arthritis and other debilitating conditions. Texas already has 71 stem cell clinics.

When STAT News questioned Sean Morrison, PhD, a stem cell biologist at the University of Texas Southwestern Medical Center in Dallas about the bill, he said, “When patients get desperate, they have a capacity to suspend disbelief. When offered the opportunity of a therapy they believe in, even without data and if the chances of benefit are low, they’ll fight for access to that therapy. The problem is there are fraudulent stem cell clinics that have sprung up to exploit that.”

The article quoted Sally Temple, PhD, president of the International Society for Stem Cell Research: “It may sound like an appealing idea to allow seriously ill patients accelerated access to experimental therapies…but in the absence of full clinical testing, these bills will allow snake oil salesmen to sell unproven and scientifically dubious therapies to desperate patients.”

Conclusions

I came to this editorial asking myself, “How do we establish that orthopaedic surgeons are different from other musculoskeletal care providers, if not through evidence-based care?” Yet, with a practice widely removed from nonoperative care of knee arthritis, I listened to debates over HA with interest. It led to the thought: If there is this much debate over what should be a straightforward issue, how can I get on a high horse about other practices with limited or conflicting evidence? I suspect the major issue is that we have to draw the line somewhere. Of course, with science, there is no final answer, merely a constant reassessment of conclusions as new data become available.

In the coming issues, we will re-explore some previous controversies, including the impact of the AUCs on physician practice, payer denials, and patient acceptance. We will reassess the efficacy of vertebroplasty for osteoporotic vertebral compression fractures. As always, we are interested in your thoughts on these subjects and in recommendations for other controversies to explore.

Eeric Truumees, MD, is the chair of the AAOS Now Editorial Board, editor-in-chief of AAOS Now, and an orthopaedic spine surgeon in Austin, Texas, where he is also professor of orthopaedics at the Dell Medical School, University of Texas.

References

  1. https://www.pbs.org/newshour/health/top-u-s-hospitals-promote-unproven-medicine-side-mysticism
  2. https://www.theatlantic.com/health/archive/2017/02/when-evidence-says-no-but-doctors-say-yes/517368/
  3. https://www.washingtonpost.com/news/to-your-health/wp/2017/06/15/dangerous-unproven-treatments-for-chronic-lyme-disease-cause-are-on-the-rise/?noredirect=on&utm_term=.cefc54cb13a7
  4. http://virtualmentor.ama-assn.org/2011/01/ccas3-1101.html
  5. http://www.philly.com/philly/health/health-cents/right-to-try-means-right-to-be-harmed-by-unproven-treatments-20180530.html
  6. https://www.webmd.com/drug-medication/news/20170406/right-to-try-laws-a-patients-best-last-chance-or-false-hope#3
  7. https://www.optum.com/resources/library/right-to-try-laws.html
  8. http://www.addadult.com/bogus-arguments-for-unproven-treatments/