When the Food and Drug Administration (FDA) approved the drug hydroxyurea to help reduce the excruciating pain suffered by people with sickle cell disease, the health care community was hopeful. The year was 1998, and the prospect of a drug that could offer significant relief from the devastating effects of a disease that had no cure seemed promising.
Only thing, patients would have to take the drug as prescribed to fully reap its benefits. And in the decades since its approval, that has proven to be more of a challenge than expected, particularly among busy, active young adults and adolescents who are just beginning to manage their care themselves.
Now researchers are developing a smartphone app—complete with visual prompts, text messages, and videos—that shows big promise in helping solve the problem. The so-called InCharge Health app is still being tested in a clinical trial, but early findings, published in April, suggest that it could be a critical aid for young people with sickle cell disease who report that they simply cannot remember to take their medication.
While a few digital health-based programs already focus on medication adherence among people with sickle cell disease, Nicole M. Alberts, Ph.D., said this one is different because of the inclusion—from the app’s conception through its testing—of both adolescents and adults between 15 and 45 years old.
“Optimizing hydroxyurea adherence at an early age is critical to improving the health of people with sickle cell disease,” said Alberts, co-lead study author and assistant member in the psychology department at St. Jude Children’s Research Hospital in Memphis. “This is particularly important for adolescents and adults who have a higher risk for disease-related complications and are less likely to take hydroxyurea as recommended by their doctors.”
In sickle cell disease, red blood cells form sickled shapes that can get stuck in the body’s tiny blood vessels. Hydroxyurea helps those cells stay round and flexible. It also helps reduce the number of necessary blood transfusions. Years of clinical evidence have validated this, proving the drug reduces the frequency of pain crises, decreases the amount of time a patient spends in the hospital, and lowers health care costs. The National Heart, Lung, and Blood Institute guidelines for management of sickle cell disease strongly endorse the drug as an effective standard of care.
Still, adherence has been low among young people. And because studies of other chronic health conditions like asthma and diabetes have shown that technology can improve patient engagement, self-care and management skills, Alberts and her colleagues had the idea that it could be helpful for sickle cell disease, too.
To understand what kind of technology might be most effective, the team recruited 118 patients who were 22 years old on average. They came from two Memphis institutes that are members of the NHLBI-supported Sickle Cell Disease Implementation Consortium (SCDIC), which was established in 2016 to help improve the health of adolescents and young adults with sickle cell disease. One key goal of the group was to increase patient adoption of evidence-based treatment tools and interventions, including hydroxyurea, that could relieve the suffering that has been the hallmark of the genetic condition.
“With access to mobile phones and the Internet on the rise, using technology backed by evidence-based approaches could empower young people with sickle cell disease to consistently take hydroxyurea,” said Sharon Smith, Ph.D., program officer of the consortium at NHLBI.
The researchers wanted to get insights into the barriers to taking the medication, so they included patients who had never used it, even though their doctors had prescribed it, and those who had taken the drug but stopped.
Study participants took part in all four phases of the design process, which included surveys, interviews and focus groups that helped researchers better understand their needs and, later, their experiences with the new technology. The participants also helped decide which platform for the technology would be most suitable—the mobile phone app or, say, a wearable sensor or web-based application.
Nearly three quarters of study participants said that forgetting to take medication was the main barrier to taking hydroxyurea. These kinds of lapses, the researchers noted, can be exaggerated by cognitive deficits that people with the disease can face. As a result, Alberts said, participants believed a customizable medication reminder/tracker could help them best and that they would also be more likely to pay attention to a personalized reminder versus a generic one.
“The app addresses this with text messaging reminders that can be personalized by the user,” she said. “The user can enter contacts who are then notified if the user does not take their medication within the specified time period.”
Alberts said participants also reported that the side effects of hydroxyurea—nausea and vomiting, hair loss, and darkening of the skin or nail beds, for example—posed another barrier to its use. In response the researchers designed the app with education content, all in easy-to-read text, to increase the patients’ ability to understand what to expect from the drug and how to manage it. The app also includes visual prompts, such as graphs and images, as well as videos, downloadable materials, and links to other websites and resources.
Finally, study participants strongly indicated that they needed help with motivation and communication, as well as support from others during a pain episode. As a result, the app provides a means for social support, allowing patients to connect with their doctor and other patients. It also lets them log into their medical charts.
Before the prototype could even be designed, however, Jane S. Hankins, M.D., M.S., the study’s corresponding author, said researchers made sure the participants could digest basic health information, which for younger patients is often challenging, as well as information about how hydroxyurea actually works.
Hankins, who also is an associate member in the department of hematology at St. Jude Children’s Research Hospital, said this was critical because she and her colleagues noticed that participants had views about hydroxyurea that could affect their perceptions about the need even to take it. Others, she said, had questions about the complications that can come with sickle cell disease and also about how best to maintain good health. Only 21% of the participants, in fact, reported having an adequate level of health literacy in general, backing up prior research on other chronic diseases that tie poor medication adherence to low health literacy. This revelation, Hankins said, informed the researcher’s efforts to make the information highly accessible.
“We worked very hard to ensure that these multi-component features of the app are simple and intuitive, and that they provide easy-to-understand information to patients about their condition and effective treatments,” she said.
If proven to be effective, Hankins said the InCharge Health app could become a part of normal patient care routine, ensuring improved medication adherence. The app also serves as a model to optimize medication adherence, quality of life, and health outcomes for other chronic health conditions.
George Mensah, M.D., director of NHLBI’s Center for Translation Research and Implementation Science at NHLBI, said this would be a good thing. “Such evidence-based applications that are reliable will go a long way in helping doctors and their patients improve adherence to medications,” he said. “It will also enable people with sickle cell disease to prevent life-threatening complications.”