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Discussion: Quality of life in patients with overactive bladder
DISCUSSION:
ASSESSMENT OF SYMPTOMS IN PATIENTS WITH AN OVERACTIVE BLADDER OSAMU
n patients with overactive bladder symptoms, urinary incontinence (UI) may be a manifestation of involuntary bladder contractions. Some patients with overactive bladder symptoms can be diagnosed solely by clinical evaluation, although others require objective diagnostic measures, such as pad tests and cystometry. Urodynamic assessment, including cystometry, remains the cornerstone when a precise diagnosis of the etiology underlying UI is required. Many incontinent patients with overactive bladder are treated with medical or behavioral therapy initially, with surgical treatment, such as bladder augmentation and chronic electrical sacral nerve stimulation using an implantable neural prosthesis is considered if initial treatments fail. Each of the therapeutic interventions is costly, and most are recognized as having bothersome side effects. Therefore, if treatment efficacy for a patient with overactive bladder symptoms is evaluated only by clinical judgment and objective diagnostic measures, the result is inaccurate. The complete evaluation of patients with overactive bladder symptoms requires the inclusion of quality-of-life (QOL) measures. We have used five grades of the subjective degree of bother for five symptoms (daytime frequency, nocturia, urgency, urge incontinence, and sensation of incomplete emptying) to evaluate the effect of anticholinergic drugs on the overactive bladder. The five grades of subjective degree of bother are as
I
From the Department of Urology, Shin& University School of Medicine, Matsumoto City, Japan Reprint requests: Dr. Osamu Nishizawa, Professor of Urology, Shinshu University School of Medicine, 3-I-1 Asahi Matsumoto City 390, Japan
NISHIZAWA
follows: not a problem, a rare problem, a bit of a problem, quite a problem, and a serious problem. Our data showed that these five grades of the subjective degree of bother did not detect dose dependency of the drug. However, in our doubleblind study of overactive bladder patients (who met eligibility criteria including age >20 years, having any of 4 symptoms [daytime frequency >8 times, nocturia >2 times, urgency, or urge incontinence], and involuntary bladder contractions demonstrated on cystometry), five grades of subjective degrees of bother clearly showed that the anticholinergic drug was effective for the overactive bladder. The bladder filling symptoms, such as urgency, frequency, and urge incontinence, are more troublesome than the bladder emptying symptoms. In particular, urge incontinence occurring secondary to overactive bladder has a greater impact than that due to sphincter incontinence. There is little consensus regarding the correlation between symptomatic and urodynamic response of the overactive bladder to drug treatment. The involuntary bladder contractions remit more often on drug treatment than on placebo, but they persist in many patients, even those who subjectively improve. We feel that inclusion of the bother factor in the judgment criteria consisting of symptomatic evaluation and urodynamic assessment may provide a better understanding of the efficacy of drug treatment for overactive bladder. In conclusion, the QOL measures based on patients’ reports are no substitute for diagnostic tests and clinical judgment, but they can be a valuable aid to physicians and are crucial to credible outcome studies. In addition, in order to measure the profound impact of overactive bladder symptoms on patients’ lives, it may be important to develop gender-specific scores for lower urinary tract symptoms. 0090-4295/97/$17.00 PI1 SOO90-4295(97)00581-5
0 1997, ELSEVIER SCIENCE INC. ALLRIGHTSRESERVED
DISCUSSION:
QUALITY OF LIFE IN PATIENTS OVERACTIVE BLADDER CHRISTINE
ealth-related quality of life (QOL) has been widely accepted as a valid outcome measure for health care interventions in the past decade. This has brought with it a whole new group of health scientists, with their own branch of medical statistics, related jargon, and journals. Some medical researchers are unconvinced, believing that QOL measures are inherently “soft” and that this is not true science. Indeed, this is true for much of what is reported in the literature as “QOL measurement.”
H
From St. Mark’s Hospital, London, United Kingdom Reprint requests: Christine Norton, Nurse Specialist (Continence), St. Mark’s Hospital, Northwick Park, Watford Road, Harrow, Middlesex HA1 3UJ, United Kingdom 0 1997, ELSEVIER SCIENCE INC. ALL RIGHTS RESERVED
WITH
NORTON
As outlined in the article by Jackson in this supplement,’ there is a complex process involved in developing and then validating robust instruments, and it is essential that we be aware of that process and read reports or use instruments with this in mind. Have the authors used an instrument that has been developed appropriately and validated with a relevant patient group (in terms of medical condition, age, gender, and culture)? It is not acceptable, for instance, to use a psychometrically unvalidated instrument or to lift a tool from one context to another without a further validation process. Elements of QOL are likely to be very different for elderly Japanese men than for young Dutch women, even though both groups may have detrusor instability. Even seemingly similar situations will need revalidation work and back-translation to check un0090-4295/97/$17.00 PII SOO90-4295(97)00582-7
23
derstanding of concepts and phrases (eg, translation from American to English, or vice versa!). Furthermore, even validated questionnaires can have ambiguous phrases (eg, one questionnaire asks, “How much are you affected by urgency” which could mean “How much do you have urgency?” or “How much are you adversely affected by urgency?“). Focus groups are often recommended as the starting point for generating items. Different items will be needed if an instrument is intended to discriminate between individuals at a given point in time rather than evaluate changes over time. Psychometric validation involves complex statistical processes for examining the interrelationship between items (for instance, using Cronbach’s alpha coefficient, where experts suggest that a minimum of 250 responses are needed-a number achieved in few incontinence studies to date). Incontinence seldom provides an absolute barrier to any activity; sufferers do not want to do certain things, rather than being absolutely unable to do these things. For this reason, generic instruments seldom provide sufficient sensitivity to measure change. How specific does an instrument need to be? Must it be specific to a symptom (such as urge incontinence) or to a condition (eg, detrusor overactivity)? If so, the available instruments have used very small numbers of relevant subjects. The objective level of symptoms may have to improve a great deal, and reliably over a long period of time, before an individual will feel confident enough to resume social activities that have been abandoned because of incontinence. For the patients most restricted by symptoms, it is the uncertainty, rather than the frequency of symptoms, that is most restricting, and for most patients with an overactive bladder it is difficult to find a treatment that will restore absolute certainty of continence. Quality of life is very individual and has many possible components. Nobody’s life is perfect. The perception of QOL
depends on many factors, including the individuals self-image, aspirations, and expectations, and some measurement of the individuals psychological status should also be used if not part of the QOL instrument. Some of the more complex QOL research now recognizes this individuality in QOL by attempting to devise an individualized scale for each patient-that is, to find an individual list of restrictions or aspirations pretreatment and then measure progress in these areas for each individual. This necessitates a very complex methodology and is too time consuming for routine use, but perhaps ultimately it does measure something more meaningful. Motivation for human activities is hugely complex. Some people undoubtedly find symptoms useful as an excuse for avoiding unwanted activities. We are all familiar with patients who experience secondary gain from their symptoms and who develop substitute symptoms if their primary complaint is cured. Patients presenting for help may initially feel a need to emphasize the restriction on their life in order to feel justified in using health care resources. Those who have had a course of treatment may feel better and more positive about their lives just because action has been taken, rather than because their symptoms are better. Thus, all studies should report control group data. In conclusion, it may be premature to assume that we know what we are measuring, or that we have tools that can simply be taken and used in any setting. I remain to be convinced that we yet have anything more useful than asking the patient, “Do you feel better?” and “How much better do you feel?” REFERENCES 1. Jackson S: The patient with an overactive bladdersymptoms and quality-of-life issues. Urology 5O(suppl 6A): 18-22,1997.
QUESTION AND ANSWER SECTION Dr. Paul Abrams: Dr. Nishizawa introduced the concept of bothersomeness in addition to looking at quality of life (QOL). Ms. Norton brought in other important concepts, including the introduction of qualitative research, which is a skill that is foreign to most of us who were brought up on the god of quantitative research. Audience member: The question I think we all have is, What is the test, the QOL instrument, that can be used to measure response to treatment? Second, do we need different instruments for patients with genuine stress incontinence as opposed to patients with instability? Dr. Simon Jackson: The only instrument that I am aware of that has been validated for change following therapeutic intervention is the Schumacher instrument, which is the Urological Distress Inventory. The one instrument can be used and you can analyze the individual items separately. You do not have to do a global score with this instrument, and you can look separately at unstable symptoms or stress symptoms as you wish. Ms. Christine Norton: My feeling would be that you need to develop a condition-specific instrument not only for incontinence, but also for the different types of incontinence. If you look at the validation that has been done, there have been very small numbers in each subgroup with stress incontinence or urge incontinence. Audience member: I would like to introduce the way that concepts of impairment, disability, and handicap are used in rehabilitation medicine. This could, in principle, be introduced in incontinence. To give you an example, detrusor hyperreflexia in a stroke patient would be the impairment; the disability would be the loss of urine 3 times a day. The handicap would be very different in somebody who has a very active life from somebody who sits at home and watches TV. 24
Audience member: I would like to know whether generic instruments are advocated as opposed to disease-specific instruments. I got the impression that the disease-specific instruments are preferred, but I believe that the generic instruments are also very useful provided you have, as Ms. Norton said, a sufficient number of patients and a good control population because then you can take it down to a multifactorial level if there is another disease present at the same time. If you had a sufficient number of patients, you would remove the problems of other diseases at the same time. Audience member: I would like to comment on diseasespecific instruments. I think that our goal for measuring treatment should be to have a general instrument about incontinence. If we treat stress incontinence and find that it goes away but produces urge incontinence and we do not measure that, we are going to be misled on the QOL. Similarly, if we treat urge incontinence but the patient then has a larger bladder capacity and has stress leakage, we need to measure that in addition. We must look at the total outcome of urinary tract function with any treatment. Audience member: I think that one of the things that QOL research has taught us is that the various outcome measures do not correlate with each other. Therefore, it is necessary to accept different methods of measurement. There is no way that you can use a single method of measurement. I do not think that you should ask for either condition-specific or generic instruments, but use both, depending on what the objectives are. It is characteristic that objective measurementurodynamics, history, symptom, physical findings, QOL-do not correlate with each other. UROLOGY
50 (Supplement 6A), December 1997
ASSESSMENT OF SYMPTOMS IN PATIENTS WITH AN OVERACTIVE BLADDER OSAMU
n patients with overactive bladder symptoms, urinary incontinence (UI) may be a manifestation of involuntary bladder contractions. Some patients with overactive bladder symptoms can be diagnosed solely by clinical evaluation, although others require objective diagnostic measures, such as pad tests and cystometry. Urodynamic assessment, including cystometry, remains the cornerstone when a precise diagnosis of the etiology underlying UI is required. Many incontinent patients with overactive bladder are treated with medical or behavioral therapy initially, with surgical treatment, such as bladder augmentation and chronic electrical sacral nerve stimulation using an implantable neural prosthesis is considered if initial treatments fail. Each of the therapeutic interventions is costly, and most are recognized as having bothersome side effects. Therefore, if treatment efficacy for a patient with overactive bladder symptoms is evaluated only by clinical judgment and objective diagnostic measures, the result is inaccurate. The complete evaluation of patients with overactive bladder symptoms requires the inclusion of quality-of-life (QOL) measures. We have used five grades of the subjective degree of bother for five symptoms (daytime frequency, nocturia, urgency, urge incontinence, and sensation of incomplete emptying) to evaluate the effect of anticholinergic drugs on the overactive bladder. The five grades of subjective degree of bother are as
I
From the Department of Urology, Shin& University School of Medicine, Matsumoto City, Japan Reprint requests: Dr. Osamu Nishizawa, Professor of Urology, Shinshu University School of Medicine, 3-I-1 Asahi Matsumoto City 390, Japan
NISHIZAWA
follows: not a problem, a rare problem, a bit of a problem, quite a problem, and a serious problem. Our data showed that these five grades of the subjective degree of bother did not detect dose dependency of the drug. However, in our doubleblind study of overactive bladder patients (who met eligibility criteria including age >20 years, having any of 4 symptoms [daytime frequency >8 times, nocturia >2 times, urgency, or urge incontinence], and involuntary bladder contractions demonstrated on cystometry), five grades of subjective degrees of bother clearly showed that the anticholinergic drug was effective for the overactive bladder. The bladder filling symptoms, such as urgency, frequency, and urge incontinence, are more troublesome than the bladder emptying symptoms. In particular, urge incontinence occurring secondary to overactive bladder has a greater impact than that due to sphincter incontinence. There is little consensus regarding the correlation between symptomatic and urodynamic response of the overactive bladder to drug treatment. The involuntary bladder contractions remit more often on drug treatment than on placebo, but they persist in many patients, even those who subjectively improve. We feel that inclusion of the bother factor in the judgment criteria consisting of symptomatic evaluation and urodynamic assessment may provide a better understanding of the efficacy of drug treatment for overactive bladder. In conclusion, the QOL measures based on patients’ reports are no substitute for diagnostic tests and clinical judgment, but they can be a valuable aid to physicians and are crucial to credible outcome studies. In addition, in order to measure the profound impact of overactive bladder symptoms on patients’ lives, it may be important to develop gender-specific scores for lower urinary tract symptoms. 0090-4295/97/$17.00 PI1 SOO90-4295(97)00581-5
0 1997, ELSEVIER SCIENCE INC. ALLRIGHTSRESERVED
DISCUSSION:
QUALITY OF LIFE IN PATIENTS OVERACTIVE BLADDER CHRISTINE
ealth-related quality of life (QOL) has been widely accepted as a valid outcome measure for health care interventions in the past decade. This has brought with it a whole new group of health scientists, with their own branch of medical statistics, related jargon, and journals. Some medical researchers are unconvinced, believing that QOL measures are inherently “soft” and that this is not true science. Indeed, this is true for much of what is reported in the literature as “QOL measurement.”
H
From St. Mark’s Hospital, London, United Kingdom Reprint requests: Christine Norton, Nurse Specialist (Continence), St. Mark’s Hospital, Northwick Park, Watford Road, Harrow, Middlesex HA1 3UJ, United Kingdom 0 1997, ELSEVIER SCIENCE INC. ALL RIGHTS RESERVED
WITH
NORTON
As outlined in the article by Jackson in this supplement,’ there is a complex process involved in developing and then validating robust instruments, and it is essential that we be aware of that process and read reports or use instruments with this in mind. Have the authors used an instrument that has been developed appropriately and validated with a relevant patient group (in terms of medical condition, age, gender, and culture)? It is not acceptable, for instance, to use a psychometrically unvalidated instrument or to lift a tool from one context to another without a further validation process. Elements of QOL are likely to be very different for elderly Japanese men than for young Dutch women, even though both groups may have detrusor instability. Even seemingly similar situations will need revalidation work and back-translation to check un0090-4295/97/$17.00 PII SOO90-4295(97)00582-7
23
derstanding of concepts and phrases (eg, translation from American to English, or vice versa!). Furthermore, even validated questionnaires can have ambiguous phrases (eg, one questionnaire asks, “How much are you affected by urgency” which could mean “How much do you have urgency?” or “How much are you adversely affected by urgency?“). Focus groups are often recommended as the starting point for generating items. Different items will be needed if an instrument is intended to discriminate between individuals at a given point in time rather than evaluate changes over time. Psychometric validation involves complex statistical processes for examining the interrelationship between items (for instance, using Cronbach’s alpha coefficient, where experts suggest that a minimum of 250 responses are needed-a number achieved in few incontinence studies to date). Incontinence seldom provides an absolute barrier to any activity; sufferers do not want to do certain things, rather than being absolutely unable to do these things. For this reason, generic instruments seldom provide sufficient sensitivity to measure change. How specific does an instrument need to be? Must it be specific to a symptom (such as urge incontinence) or to a condition (eg, detrusor overactivity)? If so, the available instruments have used very small numbers of relevant subjects. The objective level of symptoms may have to improve a great deal, and reliably over a long period of time, before an individual will feel confident enough to resume social activities that have been abandoned because of incontinence. For the patients most restricted by symptoms, it is the uncertainty, rather than the frequency of symptoms, that is most restricting, and for most patients with an overactive bladder it is difficult to find a treatment that will restore absolute certainty of continence. Quality of life is very individual and has many possible components. Nobody’s life is perfect. The perception of QOL
depends on many factors, including the individuals self-image, aspirations, and expectations, and some measurement of the individuals psychological status should also be used if not part of the QOL instrument. Some of the more complex QOL research now recognizes this individuality in QOL by attempting to devise an individualized scale for each patient-that is, to find an individual list of restrictions or aspirations pretreatment and then measure progress in these areas for each individual. This necessitates a very complex methodology and is too time consuming for routine use, but perhaps ultimately it does measure something more meaningful. Motivation for human activities is hugely complex. Some people undoubtedly find symptoms useful as an excuse for avoiding unwanted activities. We are all familiar with patients who experience secondary gain from their symptoms and who develop substitute symptoms if their primary complaint is cured. Patients presenting for help may initially feel a need to emphasize the restriction on their life in order to feel justified in using health care resources. Those who have had a course of treatment may feel better and more positive about their lives just because action has been taken, rather than because their symptoms are better. Thus, all studies should report control group data. In conclusion, it may be premature to assume that we know what we are measuring, or that we have tools that can simply be taken and used in any setting. I remain to be convinced that we yet have anything more useful than asking the patient, “Do you feel better?” and “How much better do you feel?” REFERENCES 1. Jackson S: The patient with an overactive bladdersymptoms and quality-of-life issues. Urology 5O(suppl 6A): 18-22,1997.
QUESTION AND ANSWER SECTION Dr. Paul Abrams: Dr. Nishizawa introduced the concept of bothersomeness in addition to looking at quality of life (QOL). Ms. Norton brought in other important concepts, including the introduction of qualitative research, which is a skill that is foreign to most of us who were brought up on the god of quantitative research. Audience member: The question I think we all have is, What is the test, the QOL instrument, that can be used to measure response to treatment? Second, do we need different instruments for patients with genuine stress incontinence as opposed to patients with instability? Dr. Simon Jackson: The only instrument that I am aware of that has been validated for change following therapeutic intervention is the Schumacher instrument, which is the Urological Distress Inventory. The one instrument can be used and you can analyze the individual items separately. You do not have to do a global score with this instrument, and you can look separately at unstable symptoms or stress symptoms as you wish. Ms. Christine Norton: My feeling would be that you need to develop a condition-specific instrument not only for incontinence, but also for the different types of incontinence. If you look at the validation that has been done, there have been very small numbers in each subgroup with stress incontinence or urge incontinence. Audience member: I would like to introduce the way that concepts of impairment, disability, and handicap are used in rehabilitation medicine. This could, in principle, be introduced in incontinence. To give you an example, detrusor hyperreflexia in a stroke patient would be the impairment; the disability would be the loss of urine 3 times a day. The handicap would be very different in somebody who has a very active life from somebody who sits at home and watches TV. 24
Audience member: I would like to know whether generic instruments are advocated as opposed to disease-specific instruments. I got the impression that the disease-specific instruments are preferred, but I believe that the generic instruments are also very useful provided you have, as Ms. Norton said, a sufficient number of patients and a good control population because then you can take it down to a multifactorial level if there is another disease present at the same time. If you had a sufficient number of patients, you would remove the problems of other diseases at the same time. Audience member: I would like to comment on diseasespecific instruments. I think that our goal for measuring treatment should be to have a general instrument about incontinence. If we treat stress incontinence and find that it goes away but produces urge incontinence and we do not measure that, we are going to be misled on the QOL. Similarly, if we treat urge incontinence but the patient then has a larger bladder capacity and has stress leakage, we need to measure that in addition. We must look at the total outcome of urinary tract function with any treatment. Audience member: I think that one of the things that QOL research has taught us is that the various outcome measures do not correlate with each other. Therefore, it is necessary to accept different methods of measurement. There is no way that you can use a single method of measurement. I do not think that you should ask for either condition-specific or generic instruments, but use both, depending on what the objectives are. It is characteristic that objective measurementurodynamics, history, symptom, physical findings, QOL-do not correlate with each other. UROLOGY
50 (Supplement 6A), December 1997