TREAT Versus Treatment: A Patient's View of a Scientific Interpretation
Article Outline
In recent months articles and editorials have presented varied physician views on an issue with broad ramifications, ever more relevant, in our transitional medical age: treat the patient as an individual, or treat in line with the latest empirical study.
Some have questioned the value of newer strategies like darbepoetin for the treatment of anemia in patients with late-stage chronic kidney disease (CKD). As one of those patients twice over (my first bout was treated with a kidney transplant which is now failing), I feel that it is relevant to consider studies like TREAT1 from the patient's view, particularly in conditions like anemia which can impact patients' well-being independent of “hard” outcomes such as incident-free longevity.
Beyond CKD, I have severe cardiovascular disease: coronary artery disease with 2 separate 3-vessel coronary artery bypass grafting procedures, advanced heart failure with a profoundly enlarged heart, an ejection fraction of 15%, and an ocular stroke that took half my vision, all of which is related to a lifelong history of CKD. Indeed, when I was born my father was told I would not live 24 hours due to significant bilateral ureteral obstruction. Yet somehow I was among the first to survive bilateral nephrostomies and to be saved by an experimental miracle drug—aureomycin, an early tetracycline antibiotic. At age 51, I was fortunate to receive a kidney transplant at Stanford University. I then survived intracranial posttransplant lymphoproliferative disorder through radiation—refusing to end immunosuppression. (Please understand that some otherwise rational and educated patients would rather save their transplant than their lives.) Currently, my glomerular filtration rate (GFR) is 15 mL/min in my transplanted kidney, and my life depends on maintaining a fine balance between perfusing the kidney while protecting the heart from a relentless tendency towards fluid overload.
Despite that complex history, thanks to all of you, I am now 65 years old. I have outlived 2 of my nephrologists and exceeded my father's lifespan.
I am an active, type A personality, and none of these medical impediments has truly slowed me down—except anemia, which in recent years had forced me toward retirement and had prevented me from virtually all exercise, even walking 30 minutes per day to protect my heart. It was the anemia that seemed to promote a rapid decline in cardiac function leading to severe shortness of breath, expectoration of bloody fluid, and increased angina. For more than a year, I have followed a strict protocol of darbepoetin, 100 μg subcutaneously every other week, along with my dozen other medications.
The results have been electrifying. As someone who has had to fight, scrap, and lie seemingly his whole life to get any health insurance, my new-found energy allowed me to resume my work as a nationally published journalist and political campaigner, deeply involved in a national priority—health care reform. In September 2009, my hemoglobin was over 12 g/dL for the first time in more than 5 years. While my wife suffers endless, nonlethal back pain, I am symptom free and feel great, despite a persistently low ejection fraction and a GFR that hovers in a range where many patients are considered for dialysis. I can again exercise 3 times weekly and after 8 years, my angina is virtually gone. My cardiologist cleared me for an expedition to South America in November—the last inhabited continent my wife and I had not visited—a trip which completed one of my life goals.
“So what?” you might say. “Your anecdotal n-of-1 story means nothing compared to the impressively unimpressive results of TREAT.” I would argue the following:
Dr Marsden, in editorial comment2 on the TREAT study, refers to merely “modest improvement in patient-reported fatigue”. I would respectfully submit that clinical trials report averages. Subjective findings are more challenging. As for the value of “patient-reported” events, I consider these to be critical. Journalism taught me to be a trained observer, and I have been reporting my symptoms for 7 nephrologists, 4 cardiologists, and various neurologists and oncologists in 3 languages for almost 40 years.
I have learned that medical professionals, most of whom themselves have never experienced the symptoms their patients report, are at a critical disadvantage when it comes to selecting the best treatment options for their patients and our perception of morbidity/mortality as illness progresses.
Therefore, TREAT should not stand alone in seeking to “influence practice guidelines and inform physicians”. It is overly paternalistic to base medical care on one-size-fits-all guidelines, and to assume that all individual patients would weigh the risks and benefits and arrive at the same risk-benefit calculation. I would make the same reasoned, albeit personal evidence–based choice again and, in fact, vote with my syringe at home every other week, even after TREAT.
An entire lifetime of uncertain mortality may have distorted my thinking, but I am simply remaining true to my father's decision before nephrostomy. He was given 3 choices and he opted for the one with by far the longest odds but which offered the best possible quality of outcome. Choosing long odds for high payoff is not an extreme worldview. Having learned one's own mind and body, the empirical evidence of my own case and given priority to longevity for 95% of my life, I'm actively engaged with my physician in now balancing survival and quality of life—a mainstream approach.
In this field, the conventional wisdom is that medicine is an art. But does medical science allow you to act on those words? If only because there are other patients out there like me, I ask you to reflect.
We live in a transitional medical age in which many patients cannot be cured, but more patients than ever can be helped to continue leading productive and fulfilling lives, if for a time. Because I live and you practice in this transitional age, I heartily endorse sophisticated quality-of-life studies and a tailored approach to each individual. Quality-of-life should be considered along with more classic “hard clinical end-points” such as those studied in TREAT, to inform physician and policy-making guidelines and facilitate informed patient consent.
The author would like to acknowledge the integral assistance of Dr Myles Wolf, University of Miami Miller School of Medicine, Director of Clinical Research in preparing the manuscript.
Mr Prisant is a journalist and former advisor to British and other governments. He has worked on healthcare policy in the Middle East and helped bring high tech industry to Nelson Mandela’s South Africa.
PII: S0272-6386(10)00033-8
doi:10.1053/j.ajkd.2010.01.005
© 2010 National Kidney Foundation, Inc. Published by Elsevier Inc All rights reserved.
