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Ip S, Dahabreh IJ, Chung M, et al. An Evidence Review of Active Surveillance in Men With Localized Prostate Cancer. Rockville (MD): Agency for Healthcare Research and Quality (US); 2011 Dec. (Evidence Reports/Technology Assessments, No. 204.)

  • This publication is provided for historical reference only and the information may be out of date.

This publication is provided for historical reference only and the information may be out of date.

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An Evidence Review of Active Surveillance in Men With Localized Prostate Cancer.

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In 2011, more than 240,000 men are projected to be diagnosed with prostate cancer and 33,000 to die from the disease in the United States.1 In the United States, most cases of prostate cancer are detected via prostate-specific antigen (PSA) screening. The cancer is usually localized, and most tumors have low histological grades and low Gleason scores. Indeed, more than half of prostate cancers detected by PSA screening are expected to be early-stage, low-risk tumors.4 Such cancers are an infrequent cause of death, and those affected are more likely to die of unrelated causes.

A number of immediate active treatment options are available for localized prostate cancer. Most commonly, radical prostatectomy (RP) or radiation therapy (RT), with or without androgen deprivation therapy (ADT) are offered with curative intent. However, the clinical benefit of immediate therapy with curative intent has not yet been demonstrated for localized prostate cancer in a PSA-screened population. It is likely that a large number of men are receiving treatment with curative intent without clinical benefit due to the slow progression of many prostate tumors.4 Both surgical and radiation treatments result in significant short- and long-term adverse events, including impotence, urinary dysfunction, and other complications. Thus, determination of the appropriate management strategy for early-stage, low-risk prostate cancer is an important public health concern.

Active surveillance (AS) and watchful waiting (WW) are two observational followup strategies that forego immediate therapy in patients with prostate cancer. AS is curative in intent, while WW palliative. AS is appropriate in men with disease believed to be indolent and therefore may not require therapy. Because prediction tools are imperfect, these men are monitored closely and treated with curative intent at signs of progression or at patients’ discretion. In this way, the considerable adverse effects of treatment are at best avoided and at least deferred. This approach is to be distinguished from men for whom treatment is deemed inappropriate due to comorbidity; for these men, WW is generally considered, as it offers the option of palliative therapy upon symptomatic disease progression. AS often entails a multifactorial followup of patients—monitoring of PSA values, digital rectal examinations (DRE), prostate imaging, and periodic prostate biopsies—while WW is commonly a relatively passive strategy, with interventions triggered by symptoms. However, there is a continuum of aggressiveness of followup for both AS and WW, as currently practiced. Even though the two terms are used commonly in the scientific literature, the underlying intent (curative vs. palliative) is not always made clear. Furthermore, many analyses or databases combine AS, WW, and noncurative treatments like primary ADT in their analyses, making it impossible to ferret out issues specifically related to AS.

The choice of immediate active treatment requires the careful consideration of a number of tradeoffs, such as balancing the harms of short- and long-term complications from curative treatments against the benefits of potential reductions in long-term morbidity and mortality. AS and other observational management strategies may, therefore, be considered by men who are more interested in avoiding the risks of curative treatment. Thus, it is important to clarify appropriate eligibility criteria and followup protocols for observational strategies that could minimize both unnecessary early curative treatments and avoidable prostate cancer symptoms and deaths. Of course, this strategy depends on the supposition that AS is as effective as (or no worse than) immediate curative treatments in an appropriate subgroup of men diagnosed with prostate cancer; this, however, remains to be proven. Furthermore, some men may be uncomfortable with observational management and feel a strong need to “do something,” and thus AS may be rarely offered, chosen, or adhered to. Therefore, the factors affecting these actions also warrant further investigation.

The National Cancer Institute (NCI) and the Centers for Disease Control and Prevention (CDC) are sponsoring a National Institutes of Health (NIH) State-of-the-Science Conference in December 2011 to examine the role of AS (as opposed to immediate curative intent therapy) in the management of early-stage, low-risk prostate cancer. The NIH has tasked the Agency for Healthcare Research and Quality (AHRQ) Evidence-based Practice Center (EPC) Program to provide an evidence review for use in this conference. The objective of this report is to summarize the existing literature on the role of AS in the management of early-stage, low-risk prostate cancer. Both the report and the corresponding NIH State-of-the-Science conference are a part of the NIH Consensus Development Program (CDP), the purpose of which is to evaluate the scientific evidence on a particular topic and develop a statement that advances research in this area. This statement is developed by an independent panel that is assembled for the conference. The panel will hear the scientific data, including the findings of this evidence review, and will then use that information to compose their statement. Additional information about the NIH CDP can be found at the NIH CDP Web site (

The Conference planning committee crafted the Key Questions to be addressed at the conference, and the contracted EPC charged with systematically reviewing the literature to address them. Key Question 1 pertains to temporal trends in the natural history of prostate cancer in the United States. Key Question 2 relates to the definitions of observational (no active treatment) management strategies for prostate cancer used in the published literature. Key Question 3 relates to the factors that influence the offer or acceptance of or adherence to AS. Key Question 4 pertains to the comparative effectiveness of AS versus active treatments for localized prostate cancer. And Key Question 5 addresses recommendations for future research on observational management strategies for localized prostate cancer. It should be noted that this review primarily concerns active surveillance versus curative treatments. The widespread use of primary ADT in localized prostate cancer5 is outside the scope of this review. The exact wording of the Key Questions to be addressed is provided below.

Key Questions

  1. How have the patient population and the natural history of prostate cancer diagnosed in the United States changed in the last 30 years?
    1. Patient Characteristics
      1. Age
      2. Comorbidity
      3. Race/ethnicity
    2. Tumor Characteristics
      1. Stage
      2. Tumor volume
      3. Gleason score
      4. PSA
    3. Diagnostic Strategies
      1. Biopsy Frequency
      2. # of cores
      3. Histopathologic grading changes
    4. System Characteristics
      1. Differences in geographical access
  2. How are active surveillance and other observational management strategies defined?
    1. Common Metrics
      1. Age
      2. Gleason
      3. # cores
      4. % cores
      5. PSA (velocity, doubling time)
      6. Imaging
      7. Behavioral indicators
    2. Followup Protocols
      1. Gleason
      2. # cores
      3. % cores
      4. PSA
      5. Imaging
      6. Behavioral indicators
  3. What factors affect the offer of, acceptance of, and adherence to active surveillance?
    1. Physician Factors
      1. Primary care
      2. Diagnosing physician
      3. Consultant – second opinion
      4. Clinical factors
    2. Patient Factors
      1. Family involvement
      2. Personal preferences
      3. Risk perceptions
      4. Family history
      5. Social support
    3. Delivery System
      1. Economic incentives and disincentives
        1. Insurance Type (HMO, military, private)
        2. Availability of technology
      2. Geographic location
        1. Small area variation
        2. Regional variation
        3. Urban vs. rural
      3. Academic centers vs. private practice
    4. Communication Strategies
      1. Risk assessment, predictive models
      2. Decision-making tools and aids
  4. What are the comparative short- and long-term outcomes of active surveillance versus immediate treatment with curative intent for localized prostate cancer?
    1. Prostate specific and all cause mortality
    2. Morbidity of primary treatment decision
    3. Incidence of metastatic disease
    4. Quality of life
    5. Costs
  5. What are the research needs regarding active surveillance (or watchful waiting) in localized prostate cancer?


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