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Wednesday, October 19, 2011

Oncology Patients in the Emergency Department

Dusting off the Blogger account and checking in......

JCO published a population-based snapshot that looks at the characteristics of patients with cancer who visited emergency departments in North Carolina during 2008. Lung cancer was the most common cancer identified in visits by a significant margin while breast, prostate, and colorectal cancer were each identifed in a smaller number of visits. Patients with lung cancer were more likely to be admitted to the hospital with a total of 63% of all ED visits for patients with cancer resulting in an admission. The top three complaints included pain, dyspnea, and gastrointestinal complaints. More ED visits occurred during night and weekend hours than regular office hours.

Few brief thoughts/questions to ponder:
  • What percentage of the ED visits were preventable? What systems could be introduced to prevent the visits? (Urgent clinic availability, telemedicine, enhanced home health, etc)
  • Would these system changes result in a more timely manner and would that result in reduced need for hospital admissions?
  • As a palliative care clinician, it's not a surprise to see lung cancer disproportionately represented. One of the results of the Temel et al study regarding palliative care involvement from the time of metastatic NSCLC diagnosis was reduced hospital admissions/ED visits. It's not clear what the "mechanism of action" is, but improved access to a team that can help with the top symptoms certainly is one plausible hypothesis.
  • What is the "right number" of appropriate ED visits/hospital admissions? Hard to say, but palliative care as a field should actively work to help our oncology colleagues to push the numbers as low as possible. As much as I like my ED colleagues, the ED is not a fun place to be.

Wednesday, October 19, 2011 by Lyle Fettig ·

The New Language of Medicine

NEJM published a perspective piece where the author briefly describes recent evolution of certain terms within medicine which may resonate with you.

"Patients are no longer patients, but rather “customers” or “consumers.” Doctors and nurses have been transmuted into “providers.” These descriptors have been widely adopted in the media, medical journals, and even on clinical rounds. Yet the terms are not synonymous. The word “patient” comes from patiens, meaning suffering or bearing an affliction. Doctor is derived from docere, meaning to teach, and nurse from nutrire, to nurture. These terms have been used for more than three centuries."

"The words “consumer” and “provider” are reductionist; they ignore the essential psychological, spiritual, and humanistic dimensions of the relationship — the aspects that traditionally made medicine a “calling,” in which altruism overshadowed personal gain."
The terms "providers" and "consumers" do strike me as "health care industry"-centered terms which have served to frame the discussion in a way that is favorable to said industry.  Hopefully, the terms (especially "consumers") don't advance any further in the lexicon and maybe more feedback needs to be given to media sources regarding better terms to use.

by Lyle Fettig ·

Oligometastatic Lung Cancer

NEJM presents a case of a man found to have a primary lung cancer and a solitary brain metastasis.  The discussion regarding management of the patient is noteworthy, especially the possible role of surgery and a brief discussion regarding the use of tyrosine kinase inhibitors.

The palliative care consultant should be aware of the available case series which suggest that a minority of patients might have a longer survival than what is usually expected in patients with brain mets.  (See Table 1 in the article.)  In the case series, patients received aggressive surgical intervention for the brain met and aggressive attempt at locoregional control of the primary cancer.

The article provides some guidance for selection of appropriate patients for aggressive disease-based management.  No particular quality of life outcomes are mentioned related to aggressive surgical management.

The bottom line dilemma: This is a scenario where multidisciplinary/multimodality management may give a small number of patients a chance at longer term survival.  The approach may be worthwhile to some, but talking about prognosis on multiple levels (survival, anticipated quality of life/burden of procedures) is essential.  I cannot remember encountering this scenario recently, but a palliative care consult would be appropriate in any patient with Stage IV lung cancer.  It's possible that the complexities of therapy and communication surrounding prognosis may only increase the usefulness of palliative care in this scenario.

by Lyle Fettig ·

NPR Series on Aging and End of Life: Hospice and Palliative Care in Prison Population

The NPR show Tell Me More has a week long series on issues surrounding aging and end of life.

Today, the show highlighted the documentary Serving Life about care provided to inmates at Angola Penitentiary in Louisiana.  The documentary aired this summer on the Oprah Winfrey Network. 

Also, see this recent perspective piece from Annals of Internal Medicine which addresses the issue of compassionate release of prisoners near the end of life.

Pallimed has featured stories on palliative care in prisons previously (see search results for "prison" here).

by Lyle Fettig ·

Friday, September 30, 2011

Quality Outcomes in Palliative Care, The Swedes, and The Dawn of ACO Patient Outcomes

by Lyle Fettig

The Journal of Clinical Oncology published a Swedish study which examines correlation between patients being informed of imminent death status and other palliative care quality outcome measures. In the study, 91% of patients were informed of their imminent death status.  How was this measured?  Sweden has a national palliative care registry which is described in the study text (click the little Union Jack at the top right of the page to view the front page in English) :

This online register was completed by the physician and/or nurse responsible for the patient's care during the last week of life, although HCPs were not aware of the research question of this study. The design of the SRPC Web questionnaire does not allow any missing data at submission. The Web questionnaire is based on the 11 principles constituting good death as defined by the British Geriatrics Society and was designed to be used in any care setting, independent of diagnosis, with the intent to look retrospectively at important aspects of care delivered during the last week of life. The 25 items on the SRPC include 14 questions that cover background data of patient and reporting unit and three questions that cover items not considered relevant to the aim of this study (ie, use of visual analog or numeric rating scales during last week of life, frequency of pressure ulcers, and staff reflection). The remaining eight questions covering, for example, degree of autonomy, information about imminent death, symptom control, parenteral as needed (ie, pro re nata or PRN) prescriptions, and preferred location at time of death, were included in the analysis in this study.
The ACP Hospitalist Blog commented on this study in more detail right after the study was released.  I'm going to keep my comments on the results brief.  The bottom line was no surprise.  Being informed of imminent death status correlated positively with more prn symptomatic medication orders, family being informed of imminent death status as well as being offered bereavement services, and patients recorded as dying in their preferred location.  Being informed did not correlate with increased anxiety or pain.  Minor methodological issues aside, this is yet more data (need we more?) to suggest that we should get over the worry about the effect of prognostic information on patients, even at the very end. When we don't sensitively explore patients' wishes for prognostic information and then give them info when they want it, it's usually because of our own discomfort rather than a protective effect for the patient.

What really strikes me about this study though is the existence of the database which made the study possible.  Considering the fact that I would struggle to access many of these data at my own institution, it's amazing that Sweden has a national database and that over 13,000 subjects could be included in the study.  Sweden is not unique in their attempt to measure various outcomes on a population level near the end of life.  See this Canadian study looking at the trajectory of performance status and symptom scores in patients with cancer in the last six months of life. 

Would something like this be possible in the United States? Could we develop a common repository for palliative care outcome measurement for individual patients? What are the true patient-centered outcomes which matter most to our patients and families when confronted with serious illness? 

These questions don't exist in a vacuum and should be viewed within the context of the Accountable Care Act and evolving healthcare system.  The Department of Health and Human Services recently released final rules for Accountable Care Organizations.  The rules document describes 33 quality outcomes which ACO's will need to report in order to qualify for "shared savings" above and beyond Medicare Part A and B reimbursements (see page 324). In the first year of the program, merely reporting all 33 outcomes will be sufficient to qualify for the "shared savings" payments.  In subsequent years, the ACO will need to meet a certain standard to qualify.

Take note of the first ten quality outcomes (all but the last three will be measured by patient survey data):
  • Getting Timely Care, Appointments, and Information
  • How Well Your Doctors Communicate
  • Patients' Rating of Doctor
  • Access to Specialists
  • Health Promotion and Education
  • Shared Decision Making
  • Health Status/Functional Status
  • Risk-Standardized, All Condition Readmission
  • Ambulatory Sensitive Conditions Admissions: Chronic Obstructive Pulmonary Disease
  • Ambulatory Sensitive Conditions Admissions: Congestive Heart Failure
The majority of these outcomes are near and dear to palliative care- don't we aim for these day-in, day-out? Palliative care teams can directly influence all of these outcomes for individual patients but also indirectly through local palliative care educational and quality improvement projects.  What other measurable palliative care outcomes correlate with each of these outcomes.  Could some of those other outcomes become standard in future ACO rules?  If you want to help the field (and the American healthcare system) take one small step in that direction, make sure your program applies for TJC Advanced Certification.  In the process, your program will be required to collect/analyze at least four palliative care performance measures on a continuous basis.  High penetrance of TJC Palliative Care Advanced Certification may lead TJC to build palliative care standards into the usual accreditation process for hospitals, etc.   

While I'm rambling on about everything under the sun, the Swedish Palliative Care Registry has a second questionairre that is filled out yearly at a program level (rather than specific to individual patients).  We do already have a registry similar to this: The CAPC National Palliative Care Registry.  Your program should sign up for that, too, if it hasn't already.

Friday, September 30, 2011 by Lyle Fettig ·

Tuesday, September 13, 2011

On your marks, set, go! Advanced Certification for Hospital-Based Palliative Care

by Holly Yang

On September 1st 2011, the Joint Commission started certifying hospital-based Palliative Care Programs!  It is open to all Joint Commission accredited hospitals including long-term acute care care and children's hospitals. The standards for certification were based on the National Consensus Project's Clinical Practice Guidelines for Palliative Care and A National Framework and Preferred Practices for Palliative and Hospice Care Quality from the National Quality Forum. For previous comments on the importance of this see Christian's post from spring when it was announced.

 It is "designed to recognize hospital inpatient programs that demonstrate exceptional patient and family-centered care in order to optimize the quality of life for patients with serious illness. Palliative care involves addressing physical, emotional, social and spiritual needs and facilitating patient autonomy, access to information and choice."

The full text is here, but some of the highlights of the requirements are (I'm paraphrasing, so please see the complete text for all the little words that make a big difference):

  • 24/7 coverage - by phone on nights and weekends, with availability of someone to come in to see the patient if necessary, and "must be able to provide the same level of palliative care services during nights/weekends as during normal weekday hours."
  • See patients - At least 10 in the past and at least one during the on-site review
  • Use evidenced-based approach to guide care (and/or practice guidelines to deliver care using a standardized method)
  • Have the ability to direct clinical management and coordinate care
  • Have an IDT (interdisciplinary team) and follow an organized approach to deliver care
  • QI  - Improve performance by collecting performance measurement data over time (at least 4 months' worth). No specific tools, but should be "evidenced-based, relevant, valid and reliable"
While some of these may be a challenge, it will help to standardize what "palliative care" delivery is in hospitals and help patients get the quality care they need. It will help hospitals know what is important in providing palliative care, since they don't live and breathe this every day. For example, making IDT a part of the requirements is so important, because it helps hospital administration types understand the necessity of the team approach to whole-patient care. While I don't think the hospital will be so excited to get this certification that they will immediately offer to hire a full-time social worker and chaplain/spiritual counselor for your team, I do think it is a step in the right direction and can only help you advocate for the inclusion of more psychosocial and spiritual support.

The 24/7 requirement has generated some discussion on the Center to Advance Palliative Care (CAPC) forum, as this may be one of the most difficult requirements for many programs, but does make sense for our patients' care. If I put myself in the patient's shoes, if I needed help from a palliative care team at 9 pm on a Saturday night, I really hope I could get it. Just like if I needed a cardiologist, one better be available. To expect the people who consult us to partner with us fully, we need to be available to help.  It can't rely on one person on call 24/7/365. We may need to get creative and partner with other providers (like your local hospices). It will be interesting to see what teams do, and it will be a great thing for us to share our experiences with each other, a kind of national brain-storming for good solutions. 

CAPC has done a great job getting you the resources you need including how-to guides and an audio conference. Check them out and best of luck!  

Thanks to all those who worked so hard to get these in place, and thanks to all of you out there taking care of the patients every day. 


Tuesday, September 13, 2011 by Holly Yang, MD ·

Monday, August 1, 2011

The Fellowship Quest With No Match, or O Brother, Where Are We?

Eric Widera over at GeriPal posted in April about changes in the National Resident Matching Program (NRMP) timeline for medical subspecialty fellowships, and the implications for Hospice and Palliative Medicine (HPM) and Geriatrics.

I'd like to talk about our Match-less HPM fellowship application process, from one applicant's perspective.
Photography by Ken Goldberg. From “Tele-Twister” CC 2004. Some rights reserved.

So, there I am minding my own business, I've interviewed at five impressive programs, with two more interviews scheduled. Then unexpectedly, I get an offer for a position from a stellar institution (I rather awkwardly ask for time to interview more fully before making a decision, which was granted). This is followed a week later by another offer from a great program. One is closer to my family, but culturally less vibrant, with an academic practice pattern and team culture to my liking; the other is farther from my family, but in a more culturally robust area, with a strong clinical program offering high volume and challenging cases. My wife is a professional musician and music journalist, so the cultural milieu where I train, and may wind up practicing in, is an important factor. Then an interview opportunity comes in from another great program in a great town an hour away from my wife's family.


Photography by Ken Goldberg. From “Tele-Twister” CC 2004. Some rights reserved.

Perhaps one might say, "I should be so lucky." My reaction is actually more like panic. The opportunity costs is high assuredly, but determining which I should choose and which I should forego is excruciatingly difficult. I still have 10 applications out (OK maybe too many applications. But, who knows at the outset how one is going to fare?). All of those programs are very interesting to me, for location, institution and program faculty, etc.

So, I ask for and get extensions of the offers. I interview at the program close to my wife's family. When I meet with the program director, I'm told their decision about my cohort of applicants will likely be made after my other offer extensions are up. I also get what is probably a generic invitation to contact the program if I needed an early decision to clarify matters. So, back to the offering programs to update them and ask for more time, which they graciously grant. I then ask for, but did not get, an early decision, which means I was still in the running. So, I am being pulled now in at least three ways, with irons still in the fire.

Interviewing is expensive, especially on a resident's salary, and doing this all during residency, with long hours while taking care of patients and collateral responsibilities, and having to use vacation time is all very stressful. As this is all going on interview offers continue to trickle in. I begin to turn down interview offers, even canceling interviews I already have scheduled, thinking I am likely to take one of these positions. At the same time, I am anxious about eliminating interviews out here in the Wild, Wild West because something could go wrong and my birds in the bush could fly the coop, and the whole thing could come down like a house of cards leaving me in the cold, and empty-handed.

The last few days start ticking down. Out of the blue, I get a contingent offer from a program that is very strong clinically and highly academic, in a culturally vibrant town, not too terribly far away from my family. They are working on getting approval for another Accreditation Council for Graduate Medical Education (ACGME) slot, versus perhaps another candidate may turn down an offer they have already made. At this point I make criteria for being "drawn and quartered," now being pulled in four different directions. The opportunity costs mount!


Illustrated by Jillian Gilliland © 2009. From “Tell Me A Story” © 2009 Universal Uclick. All rights reserved.

The way it felt, both as it was happening and in retrospect, was something like an uncertain, perilous and epic quest. It reminded me of my favorite epic, The Odyssey. Odysseus was struggling to get home after many long years away, but one problem after another cropped up to thwart him: the whim and malice of gods and goddesses, the threats of monsters, the thrall of enchantments, the lure of comfortable captivities, the sirens' call, tempestuous storms and even more monsters. It is at this point that imagery from the Odyssey wells up: Odysseus caught between the gorging and belching vortex of Charybdis on the one hand and the rapacious heads and voracious mouths of Scylla on the other, with no way out but through.


By luck and craftiness Odysseus barely averts disaster again and again, and by holding to the course, he finally achieves his quest and arrives home, travel-weary, older, perhaps wiser… but also with an unnecessary case of PTSD. To reason that since the hero survives and achieves the quest, the arduous and circuitous course he was made to take is justified is, in my opinion, a rationalization.


In the end, I secured a position and I am very happy with my outcome. I am however very unhappy with the process. Thank goodness for the Electronic Resident Application System (ERAS) where one's application, CV, personal statement, letters of recommendation, are uploaded into the electronic ERAS cloud once, and then are electronically submitted to one's desired programs (except for the programs that don't participate in ERAS and require old-fashioned paper applications, thus duplicating work for interested applicants and their recommenders). Having ERAS in place prevented the process from being even more labor-intensive and chaotic.

The diversity among programs in their respective timelines for application review, interviewing, and how and when to do what about making offers is frankly stunning. My process was cut short by a spoils-to-the-swift ethic. Because of the asynchronous timelines, I had to decline interview offers from programs I was very interested in. Given the inherent power disparity in the roles and the dynamics involved, I did not believe I had the latitude to walk away from offers from great programs in order to "explore my options" further. Perhaps my issue, but I doubt mine alone.

If there wasn't already an existing, simple, validated, large-scale, low-cost system to regulate and standardize behavior and decision-making among applicants and programs then one would be pragmatically stuck with the status quo. Luckily for us the kinks and bugs have already been worked out and a process is readily available for us to plug into. I'm speaking of course of the NRMP, or more simply "the Match."

Having been through an NRMP Match, and now this Matchless Meet Market, I find the latter is wholly unsavory and unsatisfactory. Rather than feeling like I've come through a fair process conducted on an even playing field to arrive at an optimized placement to advance my learning and professional development and to staff the houses of programs with the best they can attract, instead I feel like I just bought a house in today's market; I've closed the best deal I could, but I really would not want to do that over again.

Furthermore, I believe I was extremely lucky; my timelines were generous and fluid. I just think about those souls out there who are waiting for an interview in their preferred program, near their family, but get an acceptance from another program with a one week deadline to accept the offer (not an uncommon demand). Yes, I feel lucky for how it turned out in the end, but I also feel like I've been through the wringer unnecessarily, and I resent that.

My conclusion is that for the sake of learners (and I actually believe in the interests of most programs) HPM needs to align with the leaders in GME and participate in The Match on the same timeline as the other medical specialties. Incidentally, moving the process into applicants' final year of specialty training would bring it in line with the fellowship application process in other specialties, at least with Physical Medicine & Rehabilitation and Emergency Medicine.

If you have a war story, please feel free to add it. I would suggest de-identifying the data for the sake of human subjects protection, if you get my drift.

[Ed. Note: In the vast palliative care social media echo chamber, Eric Widera over at the GeriPal has provided the 2 to a 1-2 post combination on the problems of applicant/learners in the current state-of-the-fellowships. Please be sure to pop on over and check it out. - McMichael

And after you read that, you can see Christian Sinclair's alternate take on the Geripal post. - Sinclair]

Monday, August 1, 2011 by Brian McMichael, M.D. ·

Monday, July 25, 2011

Call for Submissions to the 3rd Annual AAHPM Interactive Educational Exchange!

If you are a med ed geek like me, you L-O-V-E the Interactive Educational Exchange which has been a fun and exciting part of the Americal Academy of Hospice and Palliative Medicine (AAHPM) Annual Assembly for the past two years. It's not your ordinary concurrent session. No! Instead, you listen to a quick overview of the different presentations and then you zip around to learn more about the ones that you find most intriguing. I have always come away with at least one interesting idea to try or tweek to fit my teaching style and audience. So... if you're students are yawning, you can A) buy them coffee or B) go to the Interactive Educational Exchange on March 9th, 2012 from 3:30 to 5:00 pm. 


If you've got an exciting way to teach palliative medicine to any discipline, please do share!  Submit it for the 2012 Interactive Educational Exchange at the AAHPM Annual Assembly in Denver! Hurry! Submissions are due by 5 pm PST August 2, 2011! Heck! They make it easy by even giving you previous year's submission examples.


"The Exchange provides a structured, interactive, and hands-on forum for palliative care educators from all disciplines to present, share and exchange materials and methods from model educational initiatives, including curricula, program development, educational process, assessment tools, online materials, and research in teaching and evaluation." (via the Educational Exchange Session Faculty: Laura Morrison, MD; Elise Carey, MD; Barbara Jones, MSW, PhD; Pamela Fordham, RN, DSN, CNRP)

Hope to see you there!!!


Photo by Elizabeth Thomsen via Flickr


Monday, July 25, 2011 by Holly Yang, MD ·

LIFE Before Death Short Films - Week 10 of 50!


Each week for 50 weeks, there will be a short documentary film about the international crisis in untreated pain. We've already made it through the first 10 weeks so far! This is a part of a larger project entitled LIFE Before Death which includes a full-length documentary film as well (see Christian’s earlier post Movie Trailer Premiere: LIFE Before Death).
Whether you are interested in watching about Opiophobia or debate the question of Torture in Health Care, you can catch up at TreatThePain.com. You can also download the videos for advocacy and training work at the Life Before Death website (the topic of two previous Pallimed posts here and here).

Read more »

by Holly Yang, MD ·

Tuesday, July 19, 2011

Denosumab, palifermin, and the costs of supportive cancer care


I've been following the emergence of denosumab as a preventive therapy for skeletal related events in patients with bone metastases, and thought I'd write about it.  Denosumab is a monoclonal antibody therapy which binds RANK-ligand, leading to osteoclast inhibition, decreased bone turnover, and presumably its salutary effects in preventing fractures and other skeletal related events (this glowing editorial on denosumab gives a nice/brief overview of its mechanism of action - the image is from this editorial.)

There have been several publications the last half-year in Journal of Clinical Oncology (breast cancer study here; myeloma & non-breast, non-prostate cancer study here) all showing that denosumab is non-inferior to zoledronic acid in preventing skeletal related events.  (SREs are a composite research outcome which combines pathologic fracture, hypercalcemia, and need for surgery/radiotherapy due to bone mets.)    I'd noted the abstracts, and already seen a few patients receiving it, but finally gave the research a closer look the last couple weeks.

What struck me, as I read the paper for the myeloma/non-breast/prostate one, was their power calculation:  they calculated needing 1700 patients to demonstrate noninferiority of denosumab to zoledronic acid given monthly.  That's a pretty high number for a supportive cancer trial, I thought to myself, Why?  Well the answer is obvious, and borne out by the study results.  The benefits of denosumab, compared to zoledronic acid, are marginal, and you'd need a lot of patients to minimally prove statistical significance:  denosumab met their non-inferiority definition; the median time to first on-study SRE was 20.6 months for denosumab vs 16.3 months for zoledronic acid (this difference did not achieve statistical significance).  There was some expected differences in side effects:  more flu-like symptoms and renal problems with zoledronic acid, some more hypocalcemia with denosumab.   That's about it. The breast cancer study had similar findings, a little more positive (by 27 months 50% of patients in the zoledronic acid group had had an SRE whereas only 40% of of the denosumab group had - this met their superiority criteria).  The paper doesn't present its data in a way that allows one to calculate this exactly, but by eye-balling a figure (2a) my conclusion is that the number needed to treat to prevent 1 SRE over 27 months is 10 (you'd have to give denosumab to 10 patients to further prevent a single SRE over 27 months compared to what you would have prevented with zoledronic acid).

This is a modest enough improvement that the investigators needed 2000 patients in the breast cancer study to demonstrate it actually is, and not noise.  I want to be clear:  I think the research supports that denosumab is as good as zoledronic acid, and for breast cancer patients it is better.  I don't doubt that.  That's what the N of 2000 showed.  The issue is, as a clinician, I have become more and more inclined over the years to think that the bigger N you need to show something, chances are that something is of a magnitude that is small enough, or of a frequency that is rare enough, that it's an intervention that is likely to help any given patient in theory only.  The 'better' of denosumab is probably barely worth it, at least for most patients (it may be worth it for patients with significant chronic kidney disease, but that's it).

Part of this is then re-learning what was beaten into me when I was younger, which is that bigger trials are better.  They probably are better if the outcome you're interested in is a small improvement, or rare event (and there are some outcomes for which that's appropriate - stroke prevention, for instance), but if the outcome you're interested in is anything else, including symptom control, you want something better than a small, barely statistically demonstrable, improvement.

Another way of looking at this are the costs involved.  I tried finding out more about this, and found a couple cost-effectiveness analyses (here, here - both from 2011 ASCO), dishearteningly brought to us by Novartis, the maker of zoledronic acid.  Yep. You can see Amgen (the maker of denosumab, and palifermin) & Novartis glowering at each other across the aisle at the poster session of ASCO.   Both  suggest that denosumab costs an additional $640,000 dollars per quality-adjust life year gained in breast cancer.  More for prostate cancer.  Denosumab's acquisition cost is $1650/dose vs $887/dose for zoledronic acid.  Ignoring arguments about QALYs (I'm not the biggest fan either), the magnitude of the dollar figure here gives you some sense of how marginal the improvement denosumab is bringing.  One wonders about all the ways one could use that money to help our patients in ways that will be actually helpful to them.

Which brings me to palifermin to prevent oral mucositis.  A recent JCO published the results of two (related, and Amgen funded, and randomized, double-blinded, placebo controlled) trials of weekly palifermin times 7 doses during definitive radiochemotherapy for head and neck cancer:  here & here.  Palifermin is a recombinant human keratinocyte growth factor, and is approved for the prevention of oral mucositis for patients undergoing stem cell transplants.  Revisiting my prior discussions, both studies estimated they needed ~200 patients to be adequately powered to demonstrate superiority of palifermin over placebo in preventing the incidence of severe oral mucositis (200 patients - now that's a study which is probably measuring something!)..  Rates of severe OM were reduced by ~15% (absolute risk reduction - NNT of 6.7).  Secondary outcomes suggested a (dramatic but apparently non-statistically significant) trend in one study and a statistically significant reduction in the other of duration of severe OM by two weeks.  Acknowledging that that's a sort of 'wow' outcome that gets one excited, I have to remind myself it was a secondary outcome.  Going through definitive radiochemotherapy for head and neck cancer has got to be one of the harshest medical interventions around, and reducing the most severe OM by a couple weeks seems huge.  Notably, however, they didn't look at health related quality of life, and one wonders if this was a missed opportunity.   I've tried to identify cost effectiveness analyses for palifermin, but haven't found any for radiochemotherapy.  The stem-cell transplant literature suggests it's cost effective in that setting.

Tuesday, July 19, 2011 by Drew Rosielle MD ·

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