Developing Evidence to Help Breast Cancer Patients Make Informed Decisions

October is Breast Cancer Awareness Month. During this annual campaign, organizations strive to increase awareness of the disease, encourage early detection, and increase support for research into its cause, prevention, diagnosis, and treatment. Breast cancer is the second most common cancer in women; only skin cancer is more common. Each year there are about 232,000 new patients and about 40,000 deaths.

In the year 2000, I was one of those new patients. Although I received excellent care, I can’t say that it was patient-centered. Although my doctor, who had been through the same experience a year earlier, was reassuring—even offering to show me her scar—I was often in tears. In the flurry of visits to the surgeon, oncologist, radiation oncologist, and reconstructive surgeon (not needed, as it turned out), nobody offered me any useful psychological support.

Later, when I had daily radiation sessions, there was a counselor who pounced on patients in the waiting room. While I was distraught in facing my first potentially life-threatening illness, she offered platitudes and sample cosmetics. Eventually, I was assigned to a support group where the women traded advice, certainly not evidence based, about odd diets that they had heard could prevent cancer recurrence.

Looking for Answers

Throughout my treatment, I certainly had many questions. ­­The biggest one, of course, was, what will happen to me? Not only, will my life be shortened, but also, will I have scars that will disturb my young niece and her friends in the swimming pool locker room?

In consultation with the various physicians, I had to make several decisions. Regarding treatment options, we considered whether sentinel node biopsy—which was then replacing more extensive lymph node surgery—would be sufficient to determine whether the cancer had spread. Also, lumpectomy or mastectomy? My surgeon went back and forth on her recommendation as she consulted others on the hospital team. Later, after the lumpectomy, I had to decide whether to have chemotherapy in addition to radiation. Finally, several years later, I had to decide whether to follow my tamoxifen treatment with a second drug.

In making each of these decisions, I would have certainly liked to have information about how patients similar to me had fared. At each juncture, I was simply offered that if I was the type of patient who needed to be sure I had done everything possible—no matter what the cost, discomfort, inconvenience, and side effects—then I should choose the more aggressive treatment. In the future, I hope that patient-centered outcomes research offers more evidence-based guidance for such decisions.  PCORI is already funding projects to help patients, caregivers, clinicians, and other healthcare stakeholders make well-informed health choices.

Supporting Pragmatic Clinical Studies

PCORI recently began a program to fund large-scale Pragmatic Clinical Studies. Two of the first awards have gone to projects related to breast cancer. One of these projects will test a targeted approach to breast cancer screening, in which those at higher risk are screened more often than those at lower risk. The research team will compare participants following a personalized schedule with others who receive annual screening, the commonly accepted approach. The project aims to determine whether the targeted individualized approach maximizes early detection of cancers and reduces falsely positive cancer detection that could lead to unnecessary treatments.

The other pragmatic clinical studies award went to a project that will compare two types of radiation therapy used after surgery for stage II and III breast cancers. Patients will be followed to determine differences in cancer recurrence and heart problems that may be related to the radiation treatment. The team will also measure patients’ health-related quality of life, which includes body image and function, fatigue, anxiety, social roles, and satisfaction with outcomes.

Challenges Arising During Treatment…

After a woman is diagnosed with breast cancer, she and her physicians must make many decisions regarding treatment. One of these may be whether the surgeon should remove the breast without cancer, in hopes of avoiding cancer there later. Although the frequency of such surgery has increased dramatically, there is little evidence on whether it actually improves patients’ outcomes. A project in Texas will compare psychosocial outcomes for women who had noncancerous breast removal to those who did not . This project will also use modeling to estimate differences in life expectancies between these two groups. 

Patients may endure a variety of symptoms throughout their cancer experience. These symptoms interfere with daily activities and lead to depression, anxiety, and diminished quality of life. A project in Florida is testing a brief intervention that teaches patients symptom-management skills. About a quarter of the participants have breast cancer. This project will evaluate whether this intervention helps reduce symptom intensity, frequency, and related distress; it will also assess quality of life, anxiety, depression, and patients’ beliefs about their ability to manage symptoms.

…And Afterwards

Women who have been treated for breast cancer require extra surveillance in the years that follow treatment. A project is using data on more than 22,000 patients from previous clinical trials. It will calculate how risk of recurrence varies based on patient and cancer characteristics. The research team will also analyze data from a national cancer registry called the National Cancer Data Base to evaluate whether newer imaging technology, such as MRI, CT, or PET scanning, compared with mammography,  detects cancer recurrence earlier and improves survival.

Another project is comparing magnetic resonance imaging (MRI) to mammography using data from  more than 36,000 women collected in the Breast Cancer Surveillance Consortium—the largest US collection of breast cancer surveillance data. The research team will measure the tests’ effectiveness at finding second cancers, estimate the likelihood of different outcomes, and determine whether one test appears to work better than the other to prevent death from second breast cancers among different groups of women.

Finally, a project in Washington, DC, is conducting a randomized controlled trial of an intervention developed to improve quality of life for Latina breast cancer survivors  and their caregivers. Participants will either receive usual care or supplement it with eight group sessions, covering a different topic such as communication, stress management, treatment side effects, or impact of cancer on family. Measurement will include satisfaction with care and adherence to recommended follow-up. (You can see a feature on this project here.)

Supporting Involvement of Patients in Research

Patient-Powered Research Networks (PPRNs) are an important part of PCORnet, the National Patient-Centered Clinical Research Network that PCORI is developing. These networks are operated and governed by groups of patients and their partners interested in sharing health information and participating in research.  The ABOUT (American BRCA Outcomes and Utilization of Testing) PPRN focuses on hereditary breast and ovarian cancers. It is expanding its registry and making plans to conduct research to improve health outcomes for people with those cancers.

PCORI’s Engagement program focuses on facilitating the meaningful involvement of patients and other healthcare stakeholders in the research process. It is supporting several projects related to breast cancer. One New York project recently funded under the Eugene Washington PCORI Engagement Awards program is examining obstetrical, gynecological, and breast care for women with disabilities. These women have higher-than-average rates of death from breast and cervical cancer because they do not obtain regular checkups.

We also have funded two relevant projects in our Pipeline to Proposal Award program, which is helping build a national community of patients, researchers, and other stakeholders ready to participate in patient-centered CER and encourages partnerships that lead to high-quality research proposals. One project in California is looking into different types of survivorship care plans, intended to guide women on appropriate, coordinated follow-up care and surveillance after breast cancer treatment.

The other project, in New Mexico, seeks to understand issues faced by breast cancer survivors regarding prediabetes and diabetes. Studies have linked type 2 diabetes to increased risk of breast cancer and, in women with breast cancer, higher overall mortality.

We look forward to keeping you informed as these and other projects that we fund begin to provide valuable evidence to support informed decision making among women as they face breast cancer screening, treatment, and follow-up. Please let us know your ideas for other projects in this area.