Lisette Hilton is president of Words Come Alive, based in Boca Raton, Florida.
Quality-of-life data can help guide treatment.
Recent studies suggest that judging stone treatment success on patients’ stone-free status is not aligned with how many patients define success.
The urologists’ stone-free metric doesn’t necessarily take into consideration patient quality of life, which is important to patients and for personalized medicine, according to Kristina Penniston, PhD, RDN, CD, FAND, a senior scientist in urology at the University of Wisconsin School of Medicine and Public Health in Madison.
“Most of us have dismissed patient-reported outcomes as not being hard evidence or data, like when we measure somebody’s stone-free rate by looking at a CT scan or measure stone risk by doing a 24- hour urine collection,” said Penniston, who has published studies on quality of life in patients with kidney stones.
Considering patients’ quality of life when measuring health outcomes is becoming more important not only for reimbursement but for research used in the drug approval process.
“Increasingly, we have to get more comfortable with these ‘murkier’ measures as outcomes, which are probably just as important as the hard data from a CT scan. There are a lot of studies in the health literature suggesting that patients’ own assessment of their health-related quality of life is a very good indicator of their overall health,” Penniston said.
According to Penniston, a decade of research in this area has revealed that urologists usually focus on kidney stone procedure outcomes based on results from surgery. A scan showing the patient is stone free means the outcome is optimal, whereas evidence of residual stones or fragments after surgery suggests the patient may need more surgery or treatment.
The patient, on the other hand, might view another intervention as less desirable than having an asymptomatic stone.
Another example: Patients battling chronic or recurrent stone disease often have anxiety even between stone events. They worry about when the next stone might come. Thus, stone clearance may be the goal for those patients.
Quality-of-life information helps guide not only surgical options, but also medical and preventive stone therapy.
“I work in the world of prevention; specifically, nutrition therapy. There are some patients whose quality of life actually decreases with nutritional therapy. That’s important for us to know and guides us toward alternative approaches and shared decision-making,” Penniston said.
Stone clearance or stone-free rate as an outcome is undeniably important but doesn’t capture all the experiences that can be important to patients. Identifying what is important to patients helps them make better informed decisions, according to Gregory E. Tasian MD, MSc, MSCE, an associate professor of surgery and epidemiology at Perelman School of Medicine at University of Pennsylvania in Philadelphia and a pediatric urologist at Children’s Hospital of Philadelphia.
“Quality-of-life assessment of our patients is probably underutilized,” said Justin B. Ziemba, MD, MSEd, an assistant professor of urology and surgery at the Hospital of the University of Pennsylvania, Philadelphia, who has had studies published on quality of life and stone disease. “We find that not only in kidney stones but a lot of diseases that we study and treat in urology. We tend to focus a fair amount of time on trying to figure out what is the best clinical outcome, which is usually from the perspective of the doctor.”
Patients generally want to know that they are going to feel better and are concerned about pain, according to Ziemba.
“What we [have] found so far is that people tend to think of pain of intensity on the classic 1 to 10 scale. Another important concept is pain interference, which is how likely the pain is to disrupt daily activities,” Ziemba said.
Quality-of-life assessment not only important in adults
Stone disease is rising dramatically among children, according to Tasian.
“Up until about 20 years ago, stones were a condition that occurred predominately in adults. Over the last 20 years, we’ve seen the incidence rise dramatically in children, such that the age group in whom stones are increasing at the fastest rate is adolescents—particularly adolescent girls,” Tasian said.
Unfortunately, little is known about how to best treat pediatric patients with stones, and there is scant evidence on quality of life and stone disease in children, he said.
“Ureteroscopy in an 18-month-old is very different than ureteroscopy in a 60-year-old. With that we have some evidence on how those surgical interventions differ with respect to stone clearance, but no information about other outcomes that are important to patients, which includes the patient experience before and after surgery,” said Tasian, whose research on stone disease in all ages has been published extensively.
To learn more about optimal treatment for pediatric patients with stones, Tasian started and directs the Pediatric KIDney Stone (PKIDS) Care Improvement Network. The group of 25 US health centers is conducting a Patient-Centered Outcomes Research Institute (PCORI)-funded comparative effectiveness trial of ureteroscopy, shockwave lithotripsy, and percutaneous nephrolithotomy (NCT04285658).
“One of the key components of the network is called the PKIDS Patient and Family Partners. This is a group of engaged adolescents, all of whom have stone disease, as well as caregivers and family members,” he said. “We started by asking the question, ‘What are the outcomes that are important to you?’”
So far, Tasian and colleagues have learned that pediatric patients with stones strongly identify with how the stone disease experience affects them psychologically, as well as their peer relationships and sleep. Patients also ranked anxiety high on the list.
Measuring quality of life in patients with stones
Quality-of-life aspects include patients’ emotions and social functioning, how they feel, and how that impacts their daily lives. There are various instruments that broadly measure quality of life, but only 1 is stone-specific: the Wisconsin Stone Quality of Life Questionnaire (WISQOL, bit.ly/wisqol), developed by Penniston and Stephen Y. Nakada, MD, FACS, FRCS (Glasg), chairman of the Department of Urology and The David T. Uehling Chair in Urology at the University of Wisconsin School of Medicine and Public Health. Penniston was awarded an American Urological Association (AUA) Research Scholar Award to help fund the development of WISQOL, which has since been translated into 7 different languages.
“To study the WISQOL further, we formed a consortium in 2014, and today 16 academic centers are involved in [it]. Our consortium has published 2 dozen papers and there are more in manuscript form. We’re learning a ton,” Penniston said.
WISQOL helps identify treatments that are aligned with patients’ goals. Although it can be used in the clinic, the tool is mostly used in research. Penniston and colleagues continue to study how to make the tool more user friendly for urology clinics, including determining how which findings are and are not clinically relevant.
There are other instruments that urologists can use to gauge patients’ quality of life. The widely used Short Form 36 (SF-36) is a generic health-related quality-of-life survey. The Patient-Reported Outcomes Measurement Information System (PROMIS, bit.ly/promisinstrument) is another helpful tool. The National Institutes of Health-funded PROMIS ascertains outcomes in different health domains, such as physical, mental, and social health. PROMIS instruments can be applied across populations, including those with stone disease, according to Tasian.
Ziemba said the downside of the current WISQOL is its length. The SF-36, although not designed specifically for urology or kidney stones, lets urologists know if patients are doing better in terms of their mental and physical outcomes. The customizable PROMIS measures, according to Ziemba, are short forms that can take as little as 2 minutes to complete and can inform urologists about patients’ pain and how it might interfere with daily activities.
Although instruments are helpful, they are not always necessary.
“The goals of urologists and stone patients should be aligned, but it comes down to how well the patients are informed and how realistic their expectations are. Hence, they should not just have a verbal chat but should also be given written procedural literature and access to national urology association websites, such as AUA, which also has patient-centered information and information about different conditions and procedures,” said Bhaskar K. Somani, MRCS, FEBU, DM, FFSTEd, FRCS (Urol), a professor and consultant urological surgeon at University Hospital Southampton NHS Trust in Southampton, United Kingdom.
Setting realistic expectations includes addressing quality-of-life issues that patients often ask about, such as what treatments are going to be like, how long they will be out of work or school, and how stone disease and treatment might impact their lives in the long term, according to Tasian.
Patients who are worried about pain, for example, should know that there is no such thing as pain-free surgery.
“But you can provide reasonable expectations and safely try to reduce the amount [of pain] or at least the pain interference that one experiences,” he said.
Getting a sense for how a treatment might impact quality of life starts with asking the question, according to Tasian.
“It necessitates getting out of the framework that we’ve had in surgery for so long of clinicians’ defining outcomes. It takes…humility to ask the question [of] patients: ‘What is important to you?’” he explained.