COLUMBUS, OH—Three years ago, a fourth grader named Aria Gibson developed severe abdominal pains after attending a Christmas event near her home. Her mother, Aubrey, a third-grade teacher, drove her to a nearby emergency department. Aria had appendicitis, and the doctors told Aubrey that the family had a choice: the standard treatment—surgery to remove the appendix—or a newer, noninvasive option, a course of antibiotics without surgery.
The decision was a tough one, and Aubrey and her husband, Jason, had to make it quickly. After consulting with doctors, the family chose antibiotics. A factor in that decision: the Gibsons had planned a surprise trip to Disney World in two weeks. Surgery has a one- to two-week recovery time; with antibiotics, Aria would be ready to travel in just a few days.
"It was pretty wonderful," Aubrey says. "We went to Disney World, and there were no problems, and Aria’s been really healthy ever since."
Aubrey, Jason, and Aria have since worked with researchers at Nationwide Children’s Hospital in Columbus and other families, clinicians, and insurance company representatives on a PCORI-funded research project. The team developed and is now testing a tablet computer–based app to help families of children with uncomplicated appendicitis quickly decide between surgery and antibiotics. The team is comparing families that did or didn’t use that tool, looking at satisfaction with their decision making and the child’s care, as well as medical outcomes.
The app aims to help patients become more involved in their own care and better prepared to make care decisions. Increasingly common, such patient activation tools have been shown effective in adult medicine but have rarely been tested in pediatrics, emergency medicine, or surgery, says Katherine Deans, MD, principal investigator of the study and a pediatric surgeon at Nationwide Children’s Hospital.
Appendicitis, which affects more than 70,000 US children each year, is truly an emergency. To stave off a burst appendix, decisions must be made, and treatment started, within hours of symptom onset.
Studies primarily in Europe have concluded that using antibiotics without surgery can treat appendicitis in many adult patients, and PCORI has funded two studies to compare outcomes of antibiotic and surgical treatments for adults and children in US hospitals (see box below).
Although surgeons in Europe are beginning to offer patients antibiotics instead of surgery as the first-line treatment for some cases of appendicitis, US surgeons are reluctant to change their approach without evidence from rigorous, large-scale trials in the United States showing that antibiotics are effective. PCORI recently awarded two such studies, one focused on adults and the other on children. The results will help inform the hundreds of thousands of patients who face appendicitis each year—and their clinicians.
The first, named Comparing Outcomes of Drugs and Appendectomy (CODA), will measure both clinical outcomes and the patient-reported outcomes that matter most to patients. Led by David Reed Flum, MD, MPH, from the University of Washington, the project plans to enroll 1,552 adult patients with uncomplicated appendicitis. The largest professional societies of US surgeons and emergency medicine clinicians are partners in this five-year project and plan to incorporate the results into practice guidelines and educational materials.
The second study, called Multi-Institutional Trial of Non-Operative Management of Uncomplicated Pediatric Appendicitis, will test a nonsurgical approach in children. Families of 908 children will choose one of two treatment options for their child. This research plan was recommended by the project’s stakeholder partners, including patients, caregivers, pediatricians, emergency medicine physicians, surgeons, nurses, patient educators, hospital and insurance company administrators, and pediatric surgery leaders from professional societies. The researchers, led by Peter Minneci, MD, of Nationwide Children’s Hospital, and their stakeholder partners selected as outcomes the amount of time a child misses from everyday activities and the clinical success of antibiotics alone, evaluated after a year.
In 2012, Deans and co-investigator Peter Minneci, another pediatric surgeon and also Deans’ husband, began studying antibiotics to manage uncomplicated appendicitis in children. Like other recent studies in children, theirs showed that the nonsurgical option can be as effective as surgery in uncomplicated appendicitis. In cases where both approaches are deemed medically effective, which treatment is best for any single child may depend on the family’s values, situation, and preferences.
For example, Deans explains, families in which someone has had a bad experience with anesthesia may want to avoid surgery if there’s an equally effective alternative. Parents who have difficulty taking time off from work to care for a sick child may also favor the nonsurgical option, with its typically speedier recovery.
On the other hand, families may choose surgery if they are concerned about ending their child’s appendicitis once and for all. About one in every four patients on antibiotics later needs an appendectomy, either because the antibiotics didn’t completely wipe out the first bout of appendicitis or the condition reoccurs.
In their PCORI-funded project, Deans and Minneci worked closely with patients and their families to develop an interactive tablet-based app that cut through the stress and bustle of a hospital emergency department and took patients’ specific situations into account.
“It was important to understand patient and family perspectives, for example, across race and ethnicity lines,” Deans says. The researchers invited the Gibsons and other families to join an advisory panel that also included pediatricians, emergency-department doctors, nurses, and representatives of insurance companies.
The panel helped the researchers decide on the final content and form of the tool—it includes videos describing appendicitis and its treatments, including their risks and benefits. Going into the study, Deans and Minneci weren’t sure what form the intervention would take, but the patient partners expressed a strong preference for a tablet-based app over paper and other delivery methods.
The panel’s families suggested that vignettes about children’s experiences be included in the app, and they also asked for a drag-and-drop interactive portion in which families can rank their concerns and values. The children also wanted to see and hear from other kids and requested an avatar—a patient selects one of four—to walk them through the presentation. All these suggestions were implemented.
Improved Script for Enrolling Patients
After the app was finalized, the researchers started enrolling appendicitis patients and their families to test it. Families in the emergency department with children ages 7 to 17 who have uncomplicated appendicitis were invited to participate. The patients and family members on the panel helped refine the wording of the script used to recruit participants.
"The families wanted us to stress that the intervention was designed by patients and families, so we did that,” says Deans. The change boosted recruitment success from 55 percent to over 90 percent. Deans estimates that the improved recruitment script saved 13 months and more than $500,000 in the recruitment costs. The team has now enrolled all of its 200 patients.
Those who enroll are randomly assigned to one of two procedures. In the first, a surgeon from the research team verbally provides a standard surgical consultation. In the second, a surgeon from the research team also gives the patient and family the tablet with the app. The consultation and app cover the same health information, and both stress the importance of communicating with clinicians to resolve any lingering indecision. In each case, patients and families have the opportunity to ask the surgeon questions.
Outcomes That Matter
The surgeons check in with patients’ families 30 days after treatment begins and again after a year. When asked how to get more patients and families to respond to these follow-ups, the family partners told the researchers that they’d be more likely to respond to the surgeon than to other staff members. The stakeholder panel also helped the researchers decide what outcomes to ask about.
“As researchers, we know what we think is important, such as medical complications,” Minneci says. However, the panel recommended additional outcomes, such as the level of confidence parents felt in their treatment decisions and the number of days before parents and children resumed normal activities.
“We don’t necessarily believe that the device will improve medical outcomes,” Minneci says. “But the parents who get the tablet computer–based app may feel much more confident that they made the correct choice for their child and their family.”
The researchers expect results in the summer of 2017. The app may become a blueprint for other activation tools.
“Appendicitis is really just the test case,” says Minneci. “If we are successful, we can implement the app in other emergency situations in pediatrics, like premature births or crises in critically ill children in intensive care units.”
I can’t emphasize enough how much the stakeholders affected each aspect of this project.
Upsides of Patient Involvement
Aubrey Gibson describes her experience in the project as highly positive. “Before this, I would not have chosen to have my child involved in any kind of a research project or unproven treatment,” she says. But because of the project, her 10-year-old son, Liam, is participating in a study of contact lenses to slow the development of nearsightedness, and Aria, now 13, wants to go into medicine.
As for the app, Gibson thinks it fills a need. “You can move at your own pace,” she explains. “You can go back and watch the videos again. And because it is cartoony, it’s less scary for the kids.”
Deans and Minneci are proud to helm a project that has engaged patients and other healthcare stakeholders so thoroughly throughout. The researchers expect the stakeholder involvement to increase the chance that if the app succeeds, it will become part of medical practice.
“There are so many studies where something is statistically significant, but it isn’t put into practice because patients or doctors don’t like it. The most rewarding thing about this research is that you have taken care of that from the start,” Deans says. “I can’t emphasize enough how much the stakeholders affected each aspect of this project.”
Randomized Controlled Trial of a Patient Activation Tool in Pediatric Appendicitis
Principal Investigator: Katherine Deans, MD
Goal: To test whether adding an interactive computer app to routine surgical consultation improves decision making, satisfaction with care, and clinical outcomes when parents choose between surgery and antibiotic therapy for their child’s uncomplicated appendicitis.
Posted: June 29, 2016