Use of expectant management more than doubles since 2004

According to a presentation at the Genitourinary Cancers Symposium, there has been a significant, recent increase in the numbers of men with low-risk prostate cancer who get care in the USA through some form of expectant management (active surveillance, watchful waiting, etc.).

Maurice et al. used data collected in the National Cancer Database to look at trends in the use of expectant management for men with biopsy-proven, low-risk prostate cancer (Gleason score ≤ 6, no Gleason pattern 4 or 5, clinical stage T1/T2a). They focused on patients initially diagnosed between 2004 and 2011.

Here is a summary of the findings presented in their poster:

  • The database contained 287,562 men diagnosed with low-risk prostate cancer.
  • 34,132/287,562 patients (11.9 percent) received some form of expectant management.
  • There was a steady rise in the use of expectant management, starting in 2008.
  • Compared to 2004, patients diagnosed in 2011 had 2.5 times the odds of receiving expectant management (odds ratio [OR] = 2.52)
  • Year of diagnosis, age, and Charlson score were all strong predictors of the use of expectant management (p < 0.0001).
  • Other strong predictors of the use of expectant management included hospital type and insurance provider.
  • Patients were significantly less likely to receive expectant management if treated at comprehensive cancer centers (OR = 0.63) and/or if they had private health insurance (OR = 0.90).
  • Race, income, area of residence, and hospital location were only weakly associated with the use of expectant management.

Based on these data, it would be easy to come to the conclusion that expectant management was being used increasingly as a cost-saving method in the management of men covered through Medicare, receiving treatment at Veterans Administration medical centers, or at other charitable care centers. However, that is not necessarily the case. Other non-clinical factors may well be at play here too. The available data do not help us to know exactly why expectant management is less common under certain circumstances.

Maurice et al. conclude only that:

In recent years, low-risk prostate cancer has been increasingly managed with [expectant management], especially in older patients or patients with multiple comorbidities, who are least likely to benefit from active treatment.

4 Responses

  1. “Year of diagnosis, age, and Charlson score were all strong predictors of the use of expectant management (p < 0.0001).”

    I would hope so.

    “Other strong predictors of the use of expectant management included hospital type and insurance provider. … Patients were significantly less likely to receive expectant management if treated at comprehensive cancer centers (OR = 0.63) and/or if they had private health insurance (OR = 0.90).”

    And the ACA has not even begun. Still will be fodder for both sides debating “government control of your health.” I hope it will prove to be based on the first quote.

  2. It is worth noting that the two most prominent reasons why a patient ends up getting treatment at a comprehensive cancer center are: (a) they have been referred to such a tertiary care center by another physician because they appear to have a complex case and (b) they have referred themselves there because they are seeking the highest possible quality of treatment (whether that treatment is actually necessary or not). Both of these factors would tend to make any form of expectant management less likely for most men at most comprehensive cancer centers, so I don’t find it at all surprising that expectant management is a less common form of management for low-risk prostate cancer at such centers.

  3. Hooray! Huzzah! Bravo! Three cheers! (No, too few — Five cheers!)


    Thank you! Thank you! Thank you! This is the news many of us have been waiting for: a clear sign that the good news about active surveillance is penetrating into medical practice and patient/family decision making for low-risk prostate cancer! This is so welcome after years during which so many newly diagnosed patients with low-risk cases seemed to be heading, lemming-like, over the cliffs of over-treatment without very seriously considering active surveillance as an option.

    Putting these numbers into historical context encourages me even further. There are three key time points reported in this abstract:

    2004 — the starting point for counting the percentage choosing expectant management

    2008 — the year during which the percentage of expectant management began climbing steadily

    2011 — the final year for counting the percentage choosing expectant management

    The starting point, 2004, is less than a year after the first reported results of an active surveillance series in September 2003 — the series in Toronto by Dr. Klotz and his colleagues. Until that point, AS was in the early investigational stage; no practicing doctor had published results on which to base recommendations to newly diagnosed patients, even if they were low in risk. Moreover, some leading surgeons, including Drs. Patrick Walsh of Johns Hopkins and Dr. William Catalona of Washington University (in St. Louis) and later Northwestern University (in Chicago), vigorously expressed their concern that just a few cells could escape the prostate capsule and cause serious harm to these apparently low-risk patients. At that time, there was really no solid data on which to evaluate that concern.

    The report by Dr. Klotz in September 2003 was based on results from just 231 patients, about the size of a typical Phase II clinical trial, and follow-up was not even covered in the abstract. Later information in this very well documented series established the earliest enrollment in 1995, with the series formally initiated in 1996, so the average follow-up was fairly short at that time. This report no doubt caught the interest of many physicians, but it was clearly in the “hypothesis generating” class of evidence. Moreover, the abstract made no mention of how well the patients fared who proved to need treatment. It’s not at all surprising that few patients would have “jumped on the active surveillance bandwagon” that year; indeed, no bandwagon even existed.

    Over the coming few years other major active surveillance programs published their results. We heard next from Dr. Schröeder at the Erasmus Medical Center in the Netherlands, who is better known for his leadership of the European Randomized Screening for Prostate Cancer trial. After that Johns Hopkins (Dr. Carter) and Memorial Sloan-Kettering (Dr. Scardino) published, and we were also hearing from M. D. Anderson (Dr. Babaian) and UCSF (Dr. Carroll). Moreover, important aspects of active surveillance were reported, such as the vital point that men who had to discontinue AS based on surveillance results apparently were achieving great success with their deferred therapies.

    The year 2008 was within just a year of the first Conference on Active Surveillance in 2007, chaired by the eminent surgeon Dr. Peter Carroll at UCSF (and moonlighting coach of the USC Trojans and now tomorrow’s Super Bowl contender Seattle Seahawks — just kidding) for what one book describes as “200 of the leading prostate cancer experts in the world.” While standards of eligibility for AS were still in flux, that group reached a consensus statement on eligibility criteria that included ALL of a Gleason less than 7, less than 34% of biopsy cores positive, a PSA < 10, PSA velocity less than 2, PSA density less than 0.15, and no nodule felt during the DRE. Significantly, age was not included as a criterion.

    More research on AS continued to accumulate in an ever-swelling pattern, with more centers and researchers contributing. I just did a review of AS publications by year using PubMed, the US National Library of Medicine’s online repository of published research from all over the world. I used this search string (without the quotation marks), and the filter for papers related to clinical trials: “active surveillance AND prostate cancer AND 2003 [dp]”. Here are the results by year:

    2003 — 5; 2004 — 7; 2005 — 4; 2006 — 8; 2007 — 12; 2008 — 11; 2009 — 16; 2010 — 16; 2011 — 12; 2012 — 28; 2013 — 17

    By 2011, the endpoint year for this study, it appeared to many of us that the word was getting out fairly effectively. That's one of the reasons we were so disappointed and frustrated that the US Preventive Services Task Force blundered so badly in its draft statement in late 2011 that was fundamentally opposed to screening for prostate cancer, a position finalized in a basically unaltered statement in 2012. The USPSTF clearly knew of AS but showed scant appreciation of its role and significance for solving the over-treatment issue.

    Perhaps now, with the percentage of low-risk patients choosing expectant management rising from 9.8% in 2008 to 18.6% in 2011, groups like the USPSTF will take another look at the role of screening for prostate cancer, and perhaps more patients and their physicians will be likely to jump on what looks like a “bandwagon.”


    Over the weekend I read an interview with Dr. Klotz in which he discussed his active surveillance series. He mentioned that his team had first published about the series in 2002, a year earlier than the 2003 information I mentioned in the preceding post. I double-checked and found three publications for 2002 (none earlier with any results), with these early results based on 3 to 4 years of median follow-up and about 200 to 250 patients, depending on which patients were included in the analyses. The reports were aimed at different medical communities but covered similar ground.

    I learned some other interesting points from the interview. To support this planned active surveillance clinical trial, Dr. Klotz and two radiation oncologists applied for and received a grant from the Prostate Cancer Research Foundation, the first one they had given out. Their publication of results in 2002 “… created a firestorm, because many people felt this approach was wrong. People felt we were under-treating patients and that patients would die unnecessarily. It was a very intense period. But our results were superb. No one was dying of prostate cancer. Eventually, the pendulum swung dramatically. … Today, about 5,000 patients are enrolled in about 8 prospective series. The mortality rate is in the 1% range from prostate cancer. Median follow-up is 8 to 10 years….” (Prostate Forum, January 2014)

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