Two Steps Forward...

Paula Span of the New York Times did the geriatric community a great service this past week by highlighting a change in coverage for physical therapy services. It's a change that hasn't gotten much press because it sounds pretty technical, but it has enormous ramifications for older or disabled patients undergoing rehab. For many years, Medicare insisted it would not pay for physical therapy unless a patient was getting better. Once a patient had “reached a plateau,” reimbursement would cease. In a class action suit settled in 2013, Jimmo v Sibelius, the Centers for Medicare and Medicaid Services was advised that the statutory requirement that Medicare pay for physical therapy services that are “reasonable and necessary to prevent or slow deterioration” did not support the “improvement standard" CMS has been using to implement the law. Bottom line: CMS needs to change its approach. It was instructed to embark on an educational campaign to clarify the correct policy and to modify its “Medicare Benefit Policy Manual” to reflect the court’s interpretation. According to a ruling by a US District Court in Vermont, CMS has modified its manual but fallen short in the education realm.

I’m a geriatrician and I confess I hadn’t heard about these changes. And they're changes that matter. The new approach means that the many patients getting PT at home who are no longer improving in their function but who are very likely to deteriorate if they stop getting PT are eligible for “maintenance” therapy. It means that patients in a post-acute facility after hospitalization who are using PT to get back on their feet don’t necessarily lose their SNF coverage as soon as their gains in physical function level off—provided the therapist has compelling reason to believe that further therapy is required to consolidate those gains.

The ruling to date appears to be narrowly confined to therapy services. But surely the same argument holds for nursing care: older people at home who get visiting nurse services, for example, are not currently eligible for ongoing nursing care, even though they may relapse when acute services are discontinued.

On balance, what is effectively an expansion of coverage is good for patients. Discontinuing beneficial services is often short-sighted, resulting in more acute illnesses, more hospitalizations, and more costs. But there is a problem. How do you know whether ongoing therapy (or nursing care) is necessary to maintain the gains that have already been made? And if you don’t know (without stopping the therapy and seeing what happens), how can you avoid over-use? It’s not so simple. And it all hinges on the "reasonable and necessary" standard that governs all Medicare coverage decisions.

The "reasonable and necessary" language was written into the original 1965 Medicare statute. It has bedeviled Medicare for years. This language has been interpreted to mean that Medicare may not make decisions about what to pay for based on either cost or cost-effectiveness--though many thoughtful people believe that paying a huge amount of money for a procedure or drug that doesn't do much good is completely unreasonable. This language has been interpreted to mean that the results of comparative effectiveness studies, evaluations that have the potential to disclose, for example, that approach A is identical to approach B in effectiveness but is twice as expensive, may not be used to limit Medicare coverage.

The phrase "reasonable and necessary" has been the source of no end of trouble. It is meant to be clear and precise, but it's neither. Past attempts to modify the law to define more clearly what Medicare is required to cover have met with stiff resistance from device manufacturers and other corporate interests in maintaining the very permissive status quo. Increasing their access to physical therapy is likely to be a net benefit to patients, but over the long run, we need to find a better way to determine just what Medicare should pay for. That will require legislative action, and it will require a consensus among the relevant stakeholders. If we don't undertake such a process, we risk jeopardizing the viability of the Medicare program itself.

My Mother Isn't Just a Fistula

Last week I posted the first half of a Q&A with Roz Chast, author of the wonderful book, Can't We Talk About Something More Pleasant? which was just released in paperback. Here is the rest of the interview:

MG: You also deal with death.  And you came to understand that dying can be a protracted process. You begin by saying you used to think that what would happen, as happened to “Mrs. McGillicudy,” was that she would feel unwell and take to her bed. She would stay there for 3 or 4 weeks, getting weaker. And one night she would develop a death rattle and after that, she would die. “The End.” But you learned that dying was “a lot more painful, humiliating, long-lasting, complicated, and hideously expensive.” I’m curious—before your parents’ deaths, had anyone you knew and cared about died? Do you think our lack of experience with dying contributes to our naivete?

RC: You bet!!!! It all happens off-camera.

MG: You do a splendid job of showing how preposterous we doctors can be when we propose “life-saving” treatment near the end of life. You describe how you brought your mother to a gastroenterologist who diagnosed a bowel fistula, an abnormal connection between the intestine and the outside world, and wanted to know if you wanted surgery (the only fix for this) because without the procedure, she might die. And you think: “wouldn't [surgery] be a little risky/painful/possibly insane for a 96-year old woman for whom sitting upright in a chair for half an hour…is a major effort?? And if she survives the operation, how does she manage the colostomy apparatus??? Have we lost all our marbles????” Did anyone other than the staff of “The Place,” the assisted living facility where your parents lived, and eventually hospice, evaluate what your parents could and could not do for themselves? Do you think this is as important as assessing the heart and lungs?

RC: Yes. Very much so. That we didn’t keep pretending that death is always completely avoidable. It made me think that the doctor wasn’t really seeing my mother as a person. She was just a fistula.

MG: At various points in the narrative, you talk about feeling guilty. You feel guilty about sometimes wishing your parents were dead, about spending so much money on their care, about spending time with your own family instead of them. And then near the end of your mother’s life, when she has a very devoted personal care attendant, you feel guilty that your mother “gets along better with Goodie [the aide] than with me.” It must have been hard to admit all these feelings. Was it cathartic for you to put all this down in images and dialogue?

RC: Not cathartic. I write and draw to remember. I’m not interested in catharsis. I’m afraid of forgetting.

MG: You tell the illuminating story of how your mother, after being enrolled in hospice care for some time while at the assisted living facility, suddenly improves. You acknowledge that you had “sort of adjusted to the idea that she was dying” and then—she was eating. Where in the adjustment process, you say, is “eating a tuna sandwich?” There are many aspects of the last stage of life that are hard on adult children—where would you rank the fact that it’s sometimes a real roller coaster?  

RC: I’d say that in general, the process that goes on in extreme old age was a total mystery. And yes, it was much more up and down than I had expected.

Go forth and read!

Can We Talk?

Roz Chast first published Can't We Talk About Something More Pleasant? in May of 2014. I thought it was one of the most honest, trenchant, and poignant descriptions of the last phase of life I'd seen. It's funny; it's sad; it's insightful. And it has much to teach caregivers. In honor of the imminent release of the paperback version, I had the opportunity to ask the author a few questions, which I'm posting here together with her responses. I'm including half the answers this week and the remainder will be posted next week.

MG: Your parents died in 2007 and 2009, but it looks as though you finished your book in 2014. Were you working on it all that time, creating the cartoons and the accompanying text, in which you capture your mother’s emotions, your father’s emotions, and your emotions, so beautifully—guilt, denial, affection, exasperation—or did you only start working on it years after their deaths? What made you decide to do this book, which is sometimes painfully honest and not the most flattering portrait of an aging couple?

RC: I wanted to remember my parents. I wanted to remember who they were—what they sounded like, the kinds of things they talked about. What they ate, what they argued about, how they looked… I have a kind of horror of forgetting things, and for me, writing and drawing about something is a way of remembering.

MG: In your book, there’s no mention of any doctor ever trying to engage your parents in a discussion of their wishes, not until years later when your father was 95 and hospitalized with a hip fracture and a DNR order was finally written. To the best of your knowledge, in the years between the beginning of the end (2001) and their respective ends (2007 and 2009), did any doctor ever try to bring this up? What do you make of this?

RC: As far as I know, no doctor brought this up. But I didn’t go with them to their doctor appointments until the very end, so I don’t know.

MG: One of my favorite cartoons in the book is the “Wheel of Doom.” The roulette ball could land on “death,” “deafness,” or “blindness then death,” to name a few. And then there are the “cautionary tales from [your] childhood,” truly implausible possibilities such as “killed by a baseball.”

RC: That is a completely true story. It was the son of my parents’ friends. His name, as I recall, was Ricky Laska. The  others were also stories I’d heard growing up: flower pot falling on guy, oboe, sitting on ground. ..Just thought I’d mention that.

MG: You present this as a way of showing that for your parents, all these outcomes are equally bad, a perspective that must have driven you crazy. But what it made me wonder was whether part of the problem with advance care planning as physicians conceive of it is that we tend to get too specific. We talk about “coma” and “persistent vegetative state” and “kidney failure,” which for normal people must sound much like your wheel of doom. Now your mother did say that she wouldn’t want to be a “pulsating piece of protoplasm.” Do you think your parents would have responded if their doctor had said, “So if you are ever a pulsating piece of protoplasm, we would not try to keep you going, but instead would try to keep you comfortable. But there’s a lot between the way you are now and a pulsating piece of protoplasm. If you couldn’t do anything for yourself—you couldn’t dress yourself, or get to the bathroom, or feed yourself—should we also focus mainly on comfort?”

RC: I think it’s so individual, when that border is crossed. And it’s possible that it changes. I.e., I remember when age 60 seemed impossibly old. Now, not so much. ;-)

MG: Speaking of states in between functioning—sort of—and being a pulsating piece of protoplasm, you do a terrific service by dwelling so much on what I call the in-between state, on frailty and decline, not just on death. For much of the time between your first visit to Brooklyn in 2001 and your mother’s death in 2009, she was frail. Your father spent over 5 years in this state of progressive dependence and disability. Do you have other thoughts about what would be useful to adult children when their parents are in that period of gradual decline?

RC: Get an elder lawyer to help your parents sort out stuff, to get a current will, to learn what “power of attorney” and “health care proxy” means, etc. And if you’re the person in charge, keep a notebook where you write down  all of the info you’ll have to keep track of: what drugs your parents are on, the dosages, their doctors, their doctors’ phone numbers, their pension plan information, the super and the super’s number, their neighbors and their numbers, bank info, your parents’ social security numbers… And if you’re in charge of hiring people to help take care of them, you’ll have to keep track of all of that too. Maybe to some people, this stuff is a big nothing. To me, it was very anxiety-producing.

The final part of this interview will be posted next week.

How is this Monoclonal Antibody Different from all Other Monoclonal Antibodies?

A new study published in Nature offers some potentially good news. The researchers report that a monoclonal antibody given intravenously to patients with mild cognitive impairment (referred to as “prodromal Alzheimer’s) or mild Alzheimer’s disease was generally well-tolerated and effective in reducing amyloid plaques in the brain. The drug also seemed to have beneficial clinical effects, although the study was not designed to address clinical outcomes. The results were impressive and the prospect of an effective treatment tantalizing. But the response to the paper was at best guardedly enthusiastic.  Why so tepid—given the hype that usually surrounds promising studies of this and other dread diseases?

The answer is that similar efforts in the past have repeatedly fizzled. The New England Journal of Medicine published back-to-back articles in January, 2014 of two drugs, both monoclonal antibodies, both tested in patients with mild to moderate Alzheimer’s disease. Both drugs flopped. Bapineuzumab failed to improve the cognitive subscale of the Alzheimer’s Disease Assessment Scale (ADAS-Cog11) or the Disability Assessment for Dementia (DAD) when given to patients with mild to moderate Alzheimer’s Disease who were positive for the strongly predictive apolipoprotein E4. And it failed to improve the scores of patients without that apolipoprotein E variant. Solanezumab did nothing for patients with mild to moderate Alzheimer’s disease whether the outcome was the ADAS-Cog11, the ADAS-Cog14, or the Alzheimer’s Disease Cooperative Study-Activities of Daily Living scale (ADCS-ADL). A different approach to immunotherapy, use of a “vaccine” to stimulate the production of antibodies against amyloid (rather than passive administration of those antibodies, as with Bapineuzumab and Solanezumab) only led to the desired antibodies in 25 percent of patients and it had an intolerably high likelihood of causing meningoencephalitis, a potentially lethal inflammation of the brain.

In light of these earlier disappointments, CNN asks if the new drug, Aducanumab is a game-changer (rather than simply asserting that it is). Fox News says it “shows promise.” The editorial accompanying the scientific report in Nature says that if the early findings are confirmed in larger studies, it “might help to accelerate the evaluation and regulatory approval of promising Alzheimer’s prevention therapies.”

Just what did the researchers, who were from Biogen (Cambridge, MA), Neurimmune (Zurich, Switzerland), Butler Hospital (Providence, RI), and the Institute for Regenerative Medicine (Zurich, Switzerland) do? They took as their starting point what scientists have known for over 100 years: in Alzheimer’s disease, there is an abnormal accumulation of amyloid plaques in the brain; next they acknowledged what scientists have known for 25 years: the abnormal amyloid peptide, Aβ a 42-amino acid fragment cleaved from amyloid precursor protein, is toxic to neurons (unlike, say, the 40-amino acid fragment more commonly formed from amyloid precursor protein). They then generated a human monoclonal antibody that selectively reacts with particular aggregates of Aβ. Previously, they demonstrated that this antibody, which they named Aducanumab, could cross the blood-brain barrier and clear amyloid plaque from transgenic mice. In the current work, they tested 165 patients with the very earliest stages of Alzheimer’s disease and evidence of amyloid plaques on PET scans. These patients received monthly infusions for one year, of either one of 4 different dosage levels of the drug or placebo.

Only 125 patients completed the trial, with 40 dropping out either because of adverse events they attributed to the study (20 patients), or because they no longer gave consent. Of note, “adverse events” were just as likely in the patients receiving placebo as in the patients receiving any of the three lower doses of the drug. Patients receiving the highest dose of the drug, however, were more likely to report an adverse event.

In terms of the primary outcomes of this small trial, the researchers found that active treatment reduced brain Aβ plaques as measured by PET scan in all the groups. The effects were similar in both the prodromal and mild Alzheimer’s patients.

As to the secondary outcomes—the clinical measures that the study was not designed to examine—the researchers found a dose-dependent slowing of the progress of disease on a variety of measures, including performance on the Mini Mental Status Exam, but no effect on a composite neuropsychological battery.

Finally, with respect to actual adverse events, the researchers did detect a dose-dependent risk of “amyloid-related vasogenic abnormalities,” in particular of edema surrounding blood vessels. This effect usually subsided within 4-6 months and it wasn’t associated with any symptoms, but it did occur in as many as 41 percent of those receiving the highest dose of the drug.

Why did the new drug seem to perform better, at least to the extent that even tentative conclusions can be drawn from this very small pilot study, than its predecessors? The scientists clearly learned something from the earlier failures. They opted not to use a vaccine: the vaccine had caused both meningo-encephalitis and accelerated brain atrophy. Instead, they used a monoclonal antibody. But they created an entirely human antibody, rather than the “humanized” mouse monoclonal antibodies that formed Bapineuzumab and Solaneuzumab. And finally, they made an antibody that targeted a different part of the abnormal Aβ peptide, hoping that this would be less toxic than earlier antibodies.

So will it work? An op ed in Scientific American gave the best response: “A new drug trial that some researchers are calling the most promising yet in the fight against Alzheimer’s suggests it may be possible to clear the brain of the amyloid protein that is characteristic of the disease. The study was small and researchers caution that it is far too soon to declare victory against a fatal disease that robs people of their memories and ability to function in daily life. But despite repeated failures of Alzheimer’s drugs in the past, there was room for enthusiasm about the trial…” (Italics added.) Like everyone else, I am cautiously optimistic. Given my usual level of skepticism, that’s actually pretty positive.