Take a chance on me (Musings on PROMs and PCOs)

Updated: 30 Aug. 2018

August's blog derives from a scientific paper by authors in Ottawa, Canada (Further Reading) that I added to TraQ and tweeted about.

Staibano P, Perelman I, Lombardi J, Davis A, Tinmouth A, Carrier M, Stevenson C, Saidenberg E. Patient-centered outcomes in the management of anemia: a scoping review. Transfus Med Rev. 2018 Jul 12. pii: S0887-7963(18)30051-8. [Epub ahead of print]

First, out of curiosity I wondered about the meaning of 'scoping review', something I wasn't familiar with.

Second, patient-reported outcome measures (PROMs) used to evaluate the quality of patient-centered outcomes (PCO) struck home in my personal life. According, I obtained the full article to read. Then, when one of the authors on Twitter asked me for an opinion, I replied as follows.

What follows are the key points I took from the paper according to my own interests and career as a medical laboratory science educator who taught critical analysis of scientific literature for years.

This blog concentrates on only three features of the paper. I didn't include Methods and Results for the sake of brevity and to keep the blog more accessible to readers. Obviously, they are key to the paper.

• Scoping review (What's it all about?) 
• PROMs and PCOs (Research often omits all mention of them)
• Discussion (Great model for authors of research papers)

Don't expect an expert analysis because I'm unqualified to get into the review's nitty-gritty. What I present are generalizations within my personal and professional experience.

The blog's title derives from a 1978 ditty by Sweden's ABBA.

SCOPING REVIEW
After reading several resources (Further Reading) on 'scoping review' I'm not all the wiser but see it as follows:

• Relatively new approach; 
• Purpose is to map the literature on a topic vs a systematic review designed to summarize the best available research on a specific question;
• Scoping is broad, systematic is narrow.

If anyone can enlighten me further, please do so via Comments below.

PROMs and PCOs 
This is where it gets personal. Patient-reported outcome measures (PROMs) are used to evaluate the quality of patient-centered outcomes (PCOs - Further Reading). Both are usually absent in medical research on various treatments.

In this scoping review the most common PROM tools were Functional Assessment of Cancer Therapy (FACT) and Functional Assessment
of Chronic Illness Therapy (FACIT) scales (46.9% of studies).
See examples of PROMs in Further Reading. Headings typically include these types of well-being:

• Physical
• Social/Family
• Emotional
• Functional

The authors explain the concepts as follows:

'Patient-centered outcomes (PCOs) measure the impact of disease and treatment on a patient’s physical, social, and mental well-being.Tools used to measure such outcomes are known as patient-reported outcome measures (PROMs).'

'PROMs are necessary for understanding the holistic burden of various disorders from the patient perspective and for improving patient-physician communication, patient satisfaction, and treatment outcomes'

Five years ago my spouse was diagnosed with an incurable disease, idiopathic pulmonary fibrosis (IPF), with an average life span from diagnosis of  3-4 years, although some patients live much longer. At first the Alberta government did not cover the one drug that supposedly could help (Esbriet or pirfenidone). But later after more CADTH research gave it the okay, Alberta did fund the drug under certain disease conditions (at a cost of ~$43,000/yr). The studies that showed pirfenidone was useful were based on extenuating life, the so-called survival benefit.

In my opinion, pirfenidone's many side effects (See blogs in Further Reading) were downplayed in research articles and manufacturer's literature, saying they could be managed by lessening the dose. Perhaps for many but not in spouse's case, and who knows how many others.

Patient-reported outcome measures used to evaluate the quality of patient-centered outcomes related to the patient's physical, social, emotional, and functional well being were slim and mainly physical side effects. In reporting side effects outcomes were chosen that were mostly clear cut such as rash, sun-sensitivity, nausea, and of course, whether patients survived longer on the drug, the key.

As a result of my spouse's experience, if ever requiring treatment for a life-threatening condition, I hope health professionals will ask me for PROMs. I'm best qualified to assess my overall quality of life and well-being and would want those factors to be considered alongside how much my life and functionality were extended by the treatment.

DISCUSSION
I appreciated the paper's discussion for several reasons, especially how the authors extensively discuss limitations, some of which are presented below. This section could be used as a model for students learning how to evaluate literature. How the authors analysed the review's results follows.

Generalizability
Because the review analysed PCOs and anemia in mainly adult oncology patients (less than one-tenth evaluated pediatric or older adult
populations), authors note the limited generalizability of findings to those patients.

Bias
Although more than half of the included studies were RCTs,approximately 45% of these were open-label* and susceptible to patient allocation biases and biases related to the inherent subjectivity of PROMs (self-reported patient replies to survey questions).

*Open label clinical trials do not attempt to disguise the drug/treatment, meaning that no standard treatment or placebo is utilized. This can lead to bias, as both patients and physicians are aware of which groups are receiving which treatment. 

Transfusion
More than 75% of included studies investigated PCOs in anemic patients treated with erythropoiesis-stimulating agents (ESAs), whereas only 3.8% evaluated PCOs in studies of transfusions to treat anemia. The authors write,

 'As blood transfusions are one of the most common medical procedures in hospitals, are known to carry risk, and also use a limited resource, the lack of studies assessing how transfusion affects patient quality of life remains a troubling discrepancy.' 

I'm tempted to say, "It's the transfusions, stupid" as in the 1992 USA presidential race, where strategist James Carville ('the ragin' cajun') used  'It’s the economy, stupid' to focus the minds of Clinton's campaign workers.

Study Outcomes
Published studies evaluating ESAs to treat anemia, regardless of etiology, have nearly all had change in hemoglobin levels as the primary study outcome. Those related to the appropriate threshold at which to administer transfusion nearly always have mortality as the primary outcome.

To assess treatments for anemia or any condition, having a standardized set of core outcomes would help.

Bottom Line
My view: Using patient centered outcomes in research of any treatment for a specific condition faces many challenges. But they are essential if patient-centered care is to go beyond the cliché it often is today. Developing validated PROMs and requiring consistent and full reporting are key. Lots of work is needed to make this a reality.

FOR FUN
Could not resist choosing this ABBA song. I see it as asking medical researchers to take a chance on me and all patients. We patients should have a say in what the full outcomes of our treatment are. Plus ABBA songs are fun.

• Take chance on me (ABBA, 1978)

As always comments are most welcome.

FURTHER READING
Staibano P, Perelman I, Lombardi J, Davis A, Tinmouth A, Carrier M, Stevenson C, Saidenberg E. Patient-centered outcomes in the management of anemia: a scoping review. Transfus Med Rev. 2018 Jul 12. pii: S0887-7963(18)30051-8. [Epub ahead of print]

What is a scoping review?

Pham MT,et al. A scoping review of scoping reviews: advancing the approach and enhancing the consistency. Res Synth Methods. 2014 Dec; 5(4): 371-85. EPub 2014 Jul 24.

Patient-centered outcomes research from PCORI

PROMs (from Canada's CIHI)

PROM Examples
   All FACIT questionnaires
   FACT-L: For patients with lung cancer [Download pdf]

Personal Blogs
To be or not to be (Musings on IPF and Esbriet) Mar. 2016

IPF and Esbriet (Musings on extended life vs quality of life) Sept. 2016