When she was diagnosed with breast cancer, Winifred Chain just wanted to treat her disease, get healthy, and return to normal life. But there was an unexpected problem—as a side effect of a drug she was prescribed, her right thumb repeatedly popped out of its socket.
“It was so painful, I couldn’t hold a fork or sleep,” she recalls.
She spent months using a brace for her thumb and going to physical therapy, but with no relief. Chain was considering surgery when she finally tried a treatment that she says cured her thumb problem—acupuncture—through a research study at the University of Pennsylvania.
For many cancer patients, like Chain, quality of life suffers after the diagnosis. Many patients experience severe symptoms related to their disease as well as side effects from their treatments, including pain, fatigue, nausea, depression, and anxiety. Some patients continue to experience such symptoms and side effects for years after completion of their treatment.
Decades of research have improved cancer treatments and increased survival rates. Around 14 million people in the United States have been diagnosed with cancer at some time in their lives. As more people survive cancer and live longer after diagnosis, research has begun to shift to determining how they can best manage cancer symptoms and treatment side effects to improve their long-term quality of life. Cancer and its treatment influence much more than the patient’s physical well-being. Practical, psychological, social, spiritual, and financial problems commonly result from a cancer diagnosis.
“Study after study shows that patients during treatment, but also posttreatment cancer survivors, have a lot of unmet needs when it comes to symptoms and side effect management,” says Neeraj Arora, MS, PhD, a senior program officer at PCORI and a cancer survivor. “Many continue to suffer, showing that the healthcare system has not done a good job.”
PCORI is working to change that. Nine of PCORI’s 50 funded cancer studies focus primarily on approaches to managing symptoms and side effects, and seven studies have that as a secondary focus. Christopher Gayer, PhD, a program officer at PCORI, says, “It is critically important that we study and understand the treatments and interventions that work best for helping patients and their families cope with the symptoms and side effects associated with their disease.”
PCORI’s studies on symptom and side effect management cover a range of cancers, including breast, lung, brain, and gastrointestinal, some of the most prevalent types. In these studies, research teams examine symptoms and side effects in relation to cancer treatment options, patient self-management, communication with healthcare providers, posttreatment issues, and other aspects of care.
For example, a study in Pennsylvania is asking whether, after children receive intensive chemotherapy for acute myeloid leukemia, they do better staying in the hospital or being monitored at home. The team will measure patient and family quality of life as well as rates of infection. And a study in Colorado is testing whether teaching the caregivers coping skills will allow them to better attend to cancer patients and improve patients’ quality of life.
Study after study shows that patients during treatment, but also posttreatment cancer survivors, have a lot of unmet needs when it comes to symptoms and side effect management.
We are looking for opportunities to fund additional research that fills gaps in the evidence needed to allow patients and those who care for them to make better-informed decisions about treating cancer symptoms and side effects. As in the case of other high-burden conditions, we will work with patients, researchers, and others across the healthcare community to refine potential research questions in this area and see that those stakeholders serve as partners throughout the research process. As one step in that process of listening to stakeholders’ voices, we held a Twitter chat in December 2015 about cancer symptoms and side effects (see a summary of the answers we received to questions we posed during the chat).
The following are highlights of two studies that showcase the range of issues being addressed by PCORI-funded investigators in the area of cancer symptoms and side effects: one focuses on integrative medicine by comparing acupuncture with a type of psychotherapy for treating insomnia in cancer survivors, and another asks whether technology—specifically a mobile phone app—can improve experiences among patients taking oral chemotherapy.
Sleep troubles can wreak havoc on a patient’s life. Research shows that 30 to 50 percent of cancer patients experience insomnia, often caused by medications or by anxiety related to their diagnosis.
Jodi MacLeod frequently experienced insomnia while undergoing treatment for an aggressive form of breast cancer. “It makes every other symptom worse,” she says. Insomnia can aggravate pain and fatigue, as well as cloud a person’s thinking.
Even after treatments have ended, many cancer survivors experience insomnia. “You’re anxious about a possible recurrence,” MacLeod says. “With every new twinge, you go to the Internet, so then you can’t sleep. And because you can’t sleep, the worries get more entrenched in your mind. It’s a vicious cycle.”
MacLeod and Winifred Chain are patient co-investigators on a PCORI-funded study at the Memorial Sloan Kettering Cancer Center and the University of Pennsylvania on how best to treat the sleep disturbance. In the study, led by Jun Mao, MD, MSCE, of Memorial Sloan Kettering, cancer patients who are finished with treatment and have insomnia are randomly assigned to receive either acupuncture or sessions of cognitive behavioral therapy that target unhelpful beliefs, attitudes, and habits related to sleep. The research team then checks on the participants for a few months to see whether effects of the therapies last.
Both therapies had previously shown promise in treating insomnia; in fact, doctors regularly prescribe cognitive behavioral therapy for this problem. Mao and his team chose to focus on nonmedical treatments for insomnia because many cancer survivors—who tend to be older than the general population and have multiple health concerns—already take many drugs and so may prefer not to add to their medicine cabinet.
Before starting the study, Mao met with Chain, MacLeod, and other patient and community partners to find out what outcomes matter most to patients. They advised Mao that he should not only measure severity of insomnia, but also look at participants’ cognitive issues, fatigue, anxiety, and depression. “Patients really feel like the biggest issue with insomnia is daytime impairment,” he says.
Also, the suggestions of this stakeholder panel led Mao’s team to broaden the original design of the study to include not just patients who had finished treatment, but also those still being treated for stable late-stage disease. “A lot of studies on symptoms and side effects do not include those people,” Mao says.
Because minority populations are also underrepresented in cancer research, Chain is helping the team recruit African Americans. She described the study to the support group for African Americans with cancer that she had been attending. A retired school counselor, Chain has also spoken about the research at a hospital, at a conference for African-American women with breast cancer, and in a course she teaches through the National Alliance on Mental Illness. She also makes herself available to patients to discuss any concerns they might have about joining the trial.
“Everywhere I go, when I see people who might be able to join the study, I tell them,” she says. “Anybody who has insomnia should jump right on it. It’s an excellent opportunity to get treatment.”
If for the entire group, one treatment is better, but for a person like me, the other treatment works better, then that’s what matters.
MacLeod is also reaching out to potential research participants—and others who might want to learn about treating insomnia after cancer—by writing from her patient perspective for the study’s website.
Mao plans to enroll 160 survivors and patients. Once the data are in, he hopes not only to find out which therapy works better overall, but to get hints at whether one or the other might work better for specific groups of patients. Additional research can follow up on those clues.
For example, one of the therapies may do better than the other at reducing pain, and patients experiencing strong pain may sleep better in response to that therapy. Or patients who come to the clinic with a strong preference for acupuncture or cognitive behavioral therapy might have better outcomes when their therapy matches their preference.
“If for the entire group, one treatment is better, but for a person like me, the other treatment works better, then that’s what matters,” Mao says.
Keeping in Touch
Historically, chemotherapy has been given to patients intravenously in the hospital or clinic. Increasingly, however, new chemotherapy drugs can now be taken by patients in pill form at home. Not only are oral chemotherapies more convenient to patients, they have been shown to improve quality of life and survival. Joseph A. Greer, PhD, of Massachusetts General Hospital estimates that more than one-fourth of cancer patients receiving chemotherapy now get oral rather than intravenous treatment. Oral treatments are available for lung, breast, brain, hematologic, and some other cancers.
There are problems, however. Some of the regimens are complex, requiring multiple pills taken on varying schedules. Greer estimates that one-quarter to one-third of patients prescribed oral chemotherapy struggle to take their pills correctly.
Also, because oral chemotherapy doesn’t require frequent hospital visits, patients have fewer check-ins with their clinical team. And that can lead to anxiety. “Patients are trying to figure out how to cope with symptoms on their own, and they’re not sure when to follow up with a doctor,” Greer says.
So with a team of researchers and patient and community partners, Greer designed a smartphone app to help patients keep track—and inform their doctors—of their symptoms and side effects. “We wanted to allow for more direct communication and an extra layer of support,” he says.
In their PCORI-funded study, Greer’s team has recruited 190 patients, with cancers of various types, who are receiving chemotherapy in pill form. Over three months, everyone in the study receives standard care as well as an electronically monitored pill bottle to track when they take their medications, but half the participants also use the app.
The patients can use the app to report their symptoms to the study team as often as they like, and at least once a week. The team sends a report to patients’ oncologists every week. “This is in no way to replace the patients’ direct communication with the oncology team,” Greer says. It should instead prompt conversations at checkups.
If patients report one or more “red flag” symptoms with the app, it tells them to call their oncologist right away. With other symptoms, the app refers the patient to information about ways to cope with the specific problems. Following recommendations from the patient and community partners, the team programmed the app to provide recipes, information about nutrition, and links to social networks that may help patients with certain types of cancers.
The app is “very forward-looking” in helping patients connect with their doctors and access vetted information about cancer, says Judy Cadorette, a stakeholder partner in the study, whose daughter died three years ago after a 14-year battle with cancer. “I certainly could have used it to get answers about my daughter’s symptoms as well as to help her,” Cadorette says.
The research team will analyze whether patients who receive the app stick more closely to their prescribed regimen of medications. But for patients, there are other important outcomes, says Clare Karten, another stakeholder partner on the study. Karten, a healthcare consultant, says that cancer patients may be reluctant to start conversations about side effects with their oncology teams for fear of reducing their chances of remission or cure.
Greer notes other outcomes that often interest patients: improving symptom management and reducing distress due to symptoms. The study will determine whether the app leads to advances in all these realms.
Posted: June 13, 2016