Researchers at The University of Texas MD Anderson Cancer will share in a funding award of $13.4 million with hopes of answering one of the biggest questions in the current management of breast cancer: do women with the earliest form of the disease, ductal carcinoma in situ (DCIS), need invasive surgery?
Alastair M. Thompson, M.D., professor, Breast Surgical Oncology, along with colleagues at Duke Cancer Institute and Dana-Farber Cancer Institute have been awarded the funding from the Patient-Centered Outcomes Research Institute (PCORI) to launch a national prospective, randomized trial comparing guideline-concordant care of invasive surgery with or without radiation to active surveillance.
The study will also research the quality of life and psychosocial outcomes of women with DCIS.
DCIS is the presence of abnormal cells inside a milk duct or of the breast. Often referred to as "stage 0" disease, more than 60,000 women each year in the U.S. alone are diagnosed with the condition, about 1 in 5 breast cancers. Most guidelines suggest that women with DCIS undergo a lumpectomy, with or without radiation. Many women, however, opt for the more invasive mastectomy or a prophylactic mastectomy as preventive measures.
The challenge, explains Thompson, is that the vast majority of the women will not develop invasive disease. This uncertainty has led many physicians to begin to question what should be removed, and a woman with DCIS is often left with a difficult personal decision of which surgery, if any, is best for her.
"Globally, as a medical community and a society, we have woken up to the fact that with forward strides in breast screening, we are identifying thousands and thousands of women with changes of the lining of breast tubes or ducts. These changes have the potential to turn into invasive breast cancer, but likely will never cause ill health. With what some consider to be over-detection, and over-diagnosis, is the potential to over-treat," explains Thompson, the study's co-principal investigator.
"Our study is designed to answer: does every woman need an operation for a condition that's non-fatal; what are the patient concerns, outcomes, and issues that arise from having DCIS, and finally how do women make their decisions for treatment," continues Thompson.
For the prospective, randomized trial, Comparing Operative to Medical Endocrine Therapy for low risk DCIS (COMET), E. Shelley Hwang, M.D., of Duke is the study's principal investigator and Ann H. Partridge, M.D., of Dana-Farber will serve with Thompson as co-principal investigators.
The institutions will partner with The Alliance for Clinical Trials in Oncology, which will provide the network and infrastructure to support the study. The researchers estimate that in the four-year study recruitment period, approximately 40,000 women will be diagnosed with low-risk DCIS at the 100 Alliance sites. Of those women, approximately five percent will participate in the trial.
Participants will be randomized to receive either: guideline-concordant care of surgical intervention, with or without radiation; or active surveillance of a mammogram every six months for five years. Both cohorts may opt for endocrine therapy.
The researchers will also survey for health-related quality of life measures, including anxiety, depression and other psychosocial outcomes in all participants in the first and second year post-diagnosis.
Invasive cancer rates and patient reported quality of life outcomes in both groups will be compared, with the hypothesis that active surveillance doesn't yield inferior cancer or quality of life outcomes compared to those receiving guideline concordant care.
"This project was selected for PCORI funding not only for its scientific merit and commitment to engaging patients and other healthcare stakeholders in a major study conducted in real-world settings, but also for its potential to answer an important question about DCIS and fill a crucial evidence gap," said PCORI Executive Director Joe Selby, M.D., M.P.H. "We look forward to following the study's progress results."
The MD Anderson-Duke-Dana-Farber study was selected for funding through PCORI's Pragmatic Clinical Studies Initiative, an effort to produce results that are broadly applicable to a diverse range of patients and care situations and can be more quickly taken up in routine clinical practice.
Many clinical studies test whether a treatment works under ideal conditions in specialized research centers, but health care is rarely delivered in such idealized situations and settings. Pragmatic clinical studies test a treatment's effectiveness in "real-life" practice situations, such as typical hospitals and outpatient clinics, and also can include a wider range of study participants, making their findings more generally applicable.
The study was selected through a highly competitive review process in which patients, caregivers, and other stakeholders joined scientists to evaluate the proposals.
The award has been approved pending completion of a business and programmatic review by PCORI staff and issuance of a formal award contract.
PCORI is an independent, nonprofit organization authorized by Congress in 2010. Its mission is to fund research that will provide patients, their caregivers, and clinicians with the evidence-based information needed to make better-informed healthcare decisions. For more information about PCORI's funding, visit http://www.pcori.org or http://www.pcori.org/funding/opportunities.