Assessing Therapies for Benign Prostatic Hypertrophy and Localized Prostate Cancer

Patient Outcomes Research Team

Principal Investigator: John E. Wennberg, M.D., M.P.H., Dartmouth Medical School.
Grant Number: HS 06336.
Project Period: August 1989 to February 1995.

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AHCPR funded a multidisciplinary Patient Outcomes Research Team (PORT) to assess surgical and non-surgical interventions for benign prostatic hyperplasia (BPH) and localized cancer of the prostate. For most cases of prostate cancer, the PORT found that the demonstrable benefits of radical prostate surgery and radiation therapy are relatively small, and that the choice of therapy should account for patient preferences. For many men with localized prostate cancer, watchful waiting is a reasonable treatment choice.

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Prostate PORT Contents (Spring 1995)

• Introduction
• Findings
• Dissemination
• Implications
• Bibliography

Introduction

As men age, they are at increased risk of both prostate cancer and benign prostatic hyperplasia. BPH, a noncancerous enlargement of the prostate gland that causes difficulty with urination, occurs as part of the normal aging process. By age 80, as many as 35 percent of men will have under-gone surgery to relieve BPH symptoms such as frequent urination, straining to urinate, a weak urine stream, and a sensation of incomplete emptying of the bladder.

Prostate cancer is the most common cancer and the second most common cause of cancer death in American men, causing about 38,000 deaths in 1994. However, the vast majority of men with microscopic evidence of prostate cancer will never develop clinically significant disease. In recent years, the use of radical prostate surgery has increased dramatically for treatment of newly diagnosed, clinically localized prostate cancer.

To assess surgical and nonsurgical interventions for BPH and localized prostate cancer, AHCPR funded a multidisciplinary PORT. The full range of patient outcomes, including survival, morbidity, symptom relief, and self-assessed functional status, are assessed. PORT investigators developed decision models based on probabilities of outcomes and their relevance to patients. These models were used to analyze the effectiveness and cost effectiveness of treatment alternatives for BPH to support informed clinical decisionmaking, practice guidelines, research, and health care policy.

For BPH, the PORT investigated watchful waiting (no active treatment, but monitoring of symptoms), balloon dilation (a balloon catheter is inserted into the urethra and inflated to stretch it so urine flows more easily), pharmacological intervention, and surgery. Prostate surgery includes prostatectomy (usually transurethral prostatectomy [TURP], in which part of the prostate is removed by an instrument inserted into the urethra) and transurethral incision of the prostate ([TUIP], in which an instrument inserted into the urethra makes cuts in the prostate, allowing easier urination).

For prostate cancer, the PORT assessed early detection, radical prostatectomy (removal of the prostate), external beam radiation, and watchful waiting (observation followed by hormonal therapy if symptomatic metastatic disease develops).

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Findings

The PORT has published the following findings:

Benign prostatic hyperplasia

• Watchful waiting should be the standard approach for BPH patients who have mild symptoms. Many men with more symptomatic BPH also choose watchful waiting.
• There is little information on the natural progression of BPH. Untreated men often do well for years.
• Differences in practice style have contributed to large variations—up to fourfold—in the rates of prostatectomy in adjacent geographic areas.
• The American Urological Association (AUA) Symptom Index is reliable and valid, and it is sensitive to clinically important changes in the status of men with BPH. In addition to testing the Index, the PORT participated in its development.

Localized prostate cancer

• Existing studies cannot be used to compare the effectiveness of radical prostate surgery, radiation therapy, and watchful waiting, according to the PORT's systematic literature review. Until scientific evidence improves, patients and physicians cannot make informed choices.
• The choice of watchful waiting is a reasonable alternative to invasive treatment for many men with localized prostate cancer.
• Despite the lack of evidence of clinical effectiveness, the rate of radical prostate surgery in general increased almost sixfold from 1984 to 1990, according to an analysis of Medicare data. Although there was a higher rate of complications in very old men, the increase in surgery was the same in men over age 75 as in those ages 65-74.
• A greater than twentyfold variation in the rates of radical prostate surgery among States was found.
• Complication rates after surgery were higher than previously reported. Interviews of a national sample of Medicare beneficiaries found that a third were wearing pads for incontinence 2-4 years after surgery. In addition, up to 89 percent of the men reported problems with impotence. The PORT interviews give the first estimates of the rate of complications of radical prostatectomy that apply to the typical patient facing the procedure.
• Decision models showed that the possible benefits of surgical or radiation treatment of cancer become very small with advancing age and that decisionmaking should reflect patient preferences. Men age 75 and older are not likely to benefit from either radical prostate surgery or radiation therapy when compared with watchful waiting. Yet, rates of radical surgery for men age 75 and older have markedly increased in recent years.
• Although the therapeutic benefit of radical prostatectomy and radiation therapy for early prostate cancer has not been shown, a decision model shows that they may benefit some patients in limited circumstances, such as younger patients with higher grade tumors.

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Dissemination

Findings of the prostate PORT have been published in many professional journals in the United States and Europe. They include The Journal of the National Cancer Institute, Urology, Journal of Urology, Lancet, Archives of Family Medicine, European Journal of Urology, Health Management Quarterly, and International Journal of Technology Assessment in Health Care.

Many newspaper articles have mentioned PORT findings, particularly after two articles were published in the May 26, 1993, issue of The Journal of the American Medical Association that found watchful waiting compared favorably with surgery for men with localized prostate cancer. Major nationwide daily newspapers covered the story, including The New York Times, The Wall Street Journal, and The Washington Post. In addition, several wire services and news syndicates carried the story (Associated Press, Gannett News Service, Medical Tribune News Service, New York Times News Service, and United Press International).

Dissemination Summary: Benign Prostatic Hypertrophy and Localized Prostate Cancer PORT

Presentations:	97
Professional Articles:	60
Health Industry Articles:	21
Professional Mentions:	57
Consumer Print:	445
Consumer Broadcast:	2
Total:	682

Finally, the PORT findings were mentioned in several magazine features on prostate cancer that were published in Atlantic Monthly, Forbes, Fortune, and Newsweek, among others.

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Implications

The prostate disease PORT has documented an epidemic of prostate cancer detection and treatment that occurred even though there is no evidence from controlled trials that morbidity and mortality will be reduced as a result. Despite this lack of evidence, radical prostate surgery increased by more than 500 percent between 1984 and 1990. Accompanying those disturbing trends are higher rates of persistent complications (such as impotence and incontinence) than previously reported. Furthermore, half of all Medicare-reimbursed prostatectomies are performed on men age 70 and older, though these men are much less likely to benefit.

Taken together, the PORT's findings challenge accepted clinical practice. They have led to a consensus in the United States that randomized trials are needed to compare the benefits and risks of treatments for prostate disease. PORT researchers have been part of the planning process of a definitive clinical trial, the Prostate cancer Intervention Versus Observation Trial (PIVOT) to settle the controversy.

Watchful waiting has emerged as a reasonable approach for many patients with localized prostate cancer or BPH. Many BPH patients prefer watchful waiting or conservative medical treatment rather than surgery, according to early testing of an educational tool for patients. Computer-based interactive video disks have been designed to inform patients of the risks and benefits of surgery and alternative treatments for BPH and localized prostate cancer. The video disks, which the PORT helped develop, are now being used throughout the United States.

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Bibliography

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