NIH State-of-the-Science Conference Statement: Role of active surveillance in the management of men with localized prostate cancer.

OBJECTIVE: To provide healthcare providers, patients, and the general public with a responsible assessment of currently available data on the use of active surveillance and other observational management strategies for low-grade, localized prostate cancer. PARTICIPANTS: A non-U.S. Department of Health and Human Services, nonadvocate 14-member panel representing the fields of cancer prevention and control, urology, pathology, epidemiology, genetics, transplantation, bioethics, economics, health services research, shared decisionmaking, health communication, and community engagement. In addition, 22 experts from pertinent fields presented data to the panel and conference audience. EVIDENCE: Presentations by experts and a systematic review of the literature prepared by the Tufts Evidence-based Practice Center, through the Agency for Healthcare Research and Quality (AHRQ). Scientific evidence was given precedence over anecdotal experience. CONFERENCE PROCESS: The panel drafted its statement based on scientific evidence presented in open forum and on published scientific literature. The draft statement was presented on the final day of the conference and circulated to the audience for comment. The panel released a revised statement later that day at http://consensus.nih.gov. This statement is an independent report of the panel and is not a policy statement of the NIH or the Federal Government. CONCLUSIONS: Prostate cancer screening with prostate-specific antigen (PSA) testing has identified many men with low-risk disease. Because of the very favorable prognosis of low-risk prostate cancer, strong consideration should be given to modifying the anxiety-provoking term "cancer" for this condition. Treatment of low-risk prostate cancer patients with radical prostatectomy or radiation therapy leads to side effects such as impotence and incontinence in a substantial number. Active surveillance has emerged as a viable option that should be offered to patients with low-risk prostate cancer. More than 100,000 men a year diagnosed with prostate cancer in the United States are candidates for this approach. However, there are many unanswered questions about active surveillance strategies and prostate cancer that require further research and clarification. These include: • Improvements in the accuracy and consistency of pathologic diagnosis of prostate cancer • Consensus on which men are the most appropriate candidates for active surveillance • The optimal protocol for active surveillance and the potential for individualizing the approach based on clinical and patient factors • Optimal ways to communicate the option of active surveillance to patients • Methods to assist patient decisionmaking • Reasons for acceptance or rejection of active surveillance as a treatment strategy • Short- and long-term outcomes of active surveillance. Well-designed studies to address these questions and others raised in this statement represent an important health research priority. Qualitative, observational, and interventional research designs are needed. Due to the paucity of evidence about this important public health problem, all patients being considered for active surveillance should be offered participation in multicenter research studies that incorporate community settings and partners.

Extended follow-up of autologous bone marrow transplantation with 4-hydroperoxycyclophosphamide (4-HC) purging for indolent or transformed non-Hodgkin lymphomas.

Autologous blood or marrow transplantation (ABMT) for low-grade lymphomas can prolong event-free survival (EFS) but requires long-term follow-up. We report one of the longest follow-ups to a prospective transplantation study in such diseases. On a phase II study, 80 patients with low-grade, transformed, or mantle cell lymphoma received ABMT with 4-hydroperoxycyclophosphamide (4-HC) purging as part of initial or salvage therapy. Diagnoses included nontransformed follicular lymphoma in 63% and transformed lymphoma in 15%. With 16.6-year median follow-up for survival, actuarial 10-year EFS and overall survival (OS) were 34% (95% confidence interval [CI], 25%-46%) and 45% (35%-57%). Median EFS and OS were 3.0 and 8.0 years. Early nonrelapse mortality incidence was 8%; myelodysplastic syndrome or leukemia incidence was 4%. Most relapses occurred within 3 years, with a median time to diagnosis of relapse of 1.8 years (range: 0.1-15.6 years). On multivariate analysis, age >50 years, ≥3 prior chemotherapy regimens, and ABMT after relapse were associated with significantly inferior survival. Fifteen patients (19%) were event-free >15 years after transplantation, raising the possibility of a plateau in the progression-free survival curve. Thus, 4-HC-purged ABMT can produce extended remissions in a subgroup of patients with indolent lymphomas.