Results Summary
What was the research about?
Ductal carcinoma in situ, or DCIS, is a condition where abnormal cells are present in the breast. DCIS may or may not turn into invasive cancer that spreads and causes illness.
Doctors often treat DCIS like invasive cancer; standard treatment is surgery or radiation given right away. Another care approach is active monitoring with mammograms and physical exams. With active monitoring, during the year after diagnosis, women may have no treatment or their treatment may be delayed.
The research team did two studies that compared how well standard treatment versus active monitoring worked for women with DCIS. In the first study, the research team looked at the risk of death. In the second, the team looked at health and quality of life.
What were the results?
Study 1. Overall, women who had active monitoring were more likely to die from any cause, including breast cancer, than women who had standard treatment.
When the research team looked more closely at the active monitoring approach, compared with women who had standard treatment,
- Women who received no treatment had a higher risk of dying from any cause, including breast cancer.
- Women who received delayed treatment had no difference in risk of dying.
Study 2. Fewer women with active monitoring had lasting breast or chest wall pain compared with women who received standard treatment (21 percent versus 37 percent). General pain, quality of life, anxiety, depression, and concerns about cancer coming back didn’t differ between the two approaches.
Who was in the study?
Study 1 used data from a national Medicare database that included 22,576 women diagnosed with DCIS between 1992 and 2011. Of these women, 9 percent were Black; 45 percent were over age 74 when diagnosed.
Study 2 included 912 women diagnosed with DCIS or similar conditions. These women had treatment at one of four cancer centers between 2012 and 2017 and were at least one year from their diagnosis. Of these women, 87 percent were White, 6 percent were Black, 5 percent were Asian, and 3 percent were other races. Also, 5 percent were Hispanic. The average age was 60.
What did the research team do?
The research team compared
- Data from women with standard treatment versus active monitoring to see how likely they were to die up to eight years after diagnosis in study 1
- Survey responses from women with standard treatment versus active monitoring in study 2
Members of breast cancer advocacy groups gave input to the research team.
What were the limits of the study?
In study 1, women were older than 64; many had high-risk DCIS. In study 2, most women were White, and most women who had active monitoring had precancerous conditions other than DCIS. Results may differ for other patients.
Future research could compare treatment approaches among a more diverse group of women and those with lower risk DCIS.
How can people use the results?
Women and their doctors can use the results when considering treatment for DCIS.
Professional Abstract
Objective
To compare the effect of guideline-concordant care (GCC) versus active monitoring (AM) on health outcomes in women with DCIS
Study Design
| Design Element | Description |
|---|---|
| Design | Study 1: Observational: cohort study Study 2: Observational: cross-sectional study |
| Population | Study 1: 22,576 women ages 65 and older and diagnosed with DCIS Study 2: 912 women ages17 and older and diagnosed with DCIS or other atypical lesions including atypical ductal hyperplasia, atypical lobular hyperplasia, or lobular carcinoma in situ |
| Interventions/ Comparators |
|
| Outcomes | Study 1: all-cause and breast cancer mortality Study 2: persistent breast and chest wall pain (primary); general pain, quality of life, concerns about recurrence, anxiety, depression |
| Timeframe | Study 1: up to 8-year follow-up for study outcomes Study 2: immediate follow-up for study outcomes |
Study 1. This retrospective cohort study compared the impact of two treatment approaches on mortality in women with DCIS. In the GCC approach, patients received treatment with surgery or radiation immediately after diagnosis. In the AM approach, doctors monitored patients; patients received treatment if their cancer progressed.
The study included data from the Surveillance, Epidemiology, and End Results (SEER) Medicare database for 22,576 women. Researchers identified women who received a low-, intermediate-, or high-risk DCIS diagnosis between 1992 and 2011. Of these women, 9% were Black; 45% were over age 74.
Researchers compared risk of mortality between the GCC versus AM approach, which included no treatment and delayed treatment within one year of diagnosis. Researchers also compared each AM approach to GCC. Among women receiving AM, 431 delayed treatment and 205 had no treatment.
Study 2. This cross-sectional study compared pain and other outcomes among women with DCIS treated with GCC versus women with DCIS or other atypical lesions treated with AM. All women had been diagnosed for a year or longer. The study included 912 women who received treatment at one of four Comprehensive Cancer Centers between 2012 and 2017. Of these women, 87% were White, 6% were Black, 5% were Asian, and 3% were other races. Also, 5% were Hispanic. The average age was 60. Women completed a survey.
Representatives from breast cancer advocacy groups gave input on both studies.
Results
Study 1. Overall, women receiving AM had a significantly higher risk of all-cause mortality (hazard ratio [HR]=1.41; 95% confidence interval [CI]: 1.33, 1.49) and breast cancer mortality (HR=2.34; 95% CI: 1.91, 2.86) compared with women receiving GCC.
Within the AM approach, compared with women receiving GCC,
- Women receiving AM without treatment had a significantly higher risk of all-cause mortality (HR=3.25; 95% CI: 3.03, 3.48) and breast cancer mortality (HR=7.37; 95% CI: 5.90, 9.19).
- Women receiving AM with delayed treatment did not differ significantly in all-cause or breast cancer mortality.
Study 2. Fewer women receiving AM reported clinically relevant persistent breast or chest wall pain compared with women receiving GCC (21% vs. 37%; p<0.001). The women did not differ significantly in other outcomes.
Limitations
In study 1, women were over age 64; the study included patients with high-risk DCIS. In study 2, most women were White and most of those who received AM had atypical lesions. Results may differ for patients from other backgrounds, or if the data included more women with DCIS who had the AM approach.
Conclusions and Relevance
In these studies, AM was associated with increased mortality in women over age 65, but fewer women reported persistent pain compared with GCC. When comparing only those women with AM treated within one year of diagnosis, mortality did not differ.
Future Research Needs
Future research could compare the effect of GCC and AM among a more diverse group of women with a lower risk DCIS.
Final Research Report
View this project's final research report.
Journal Citations
Related Journal Citations
Peer-Review Summary
Peer review of PCORI-funded research helps make sure the report presents complete, balanced, and useful information about the research. It also assesses how the project addressed PCORI’s Methodology Standards. During peer review, experts read a draft report of the research and provide comments about the report. These experts may include a scientist focused on the research topic, a specialist in research methods, a patient or caregiver, and a healthcare professional. These reviewers cannot have conflicts of interest with the study.
The peer reviewers point out where the draft report may need revision. For example, they may suggest ways to improve descriptions of the conduct of the study or to clarify the connection between results and conclusions. Sometimes, awardees revise their draft reports twice or more to address all of the reviewers’ comments.
Peer reviewers commented and the researchers made changes or provided responses. Those comments and responses included the following:
- The reviewers noted that although the goals of the study all involved comparisons of guideline-concordant care (GCC) versus active surveillance (AS) for ductal carcinoma in situ (DCIS), some patients were included in the AS group who had other types of high-risk lesions. Reviewers expressed concern that these patients were not comparable to those with DCIS because clinicians and patients tend to consider DCIS to be cancer, but that is not true for the other types of lesions included, and patient-reported outcomes would likely be different. The researchers acknowledged this, saying that very few women choose an AS approach for DCIS so they added women with other high-risk lesions as proxies since these patients would receive similar treatment under AS conditions. They also stated that if AS was accepted as a standard option for DCIS, it would likely also become a viable approach for women with these other conditions. Moreover, they did not observe differences in patient-reported outcomes between women with DCIS and women with other types of lesions in the AS group.
- The reviewers questioned the treatment category definitions in this study, since AS1, which is treatment after active surveillance, would seem to be the same as GCC, assuming both groups received treatment within the observation window. The researchers explained that women in the GCC group received treatment within one year of DCIS diagnosis, whereas women in the AS1 group initially refused treatment but received treatment after surveillance demonstrated cancer progression.
- The reviewers noted that the report was missing a model of treatment choice which could explain patient choices of GCC versus AS. The researchers acknowledged that a more complete treatment choice model would have improved their conclusions, but they were limited to the data available for them to measure patients’ care choices. They could not, for instance, consider patient motivations in their analyses because this information was not available in the databases the researchers used.