68: Use of Patient-Reported Outcomes
This chapter will take approximately 28 minutes to read.
In medical practice, patient-self reports of their experience related to a specific disease or a treatment for a given condition may significantly differ from those of caregivers or practitioners. Patient-reported outcomes are a formal mechanism by which practitioners are able to collect data about a patient’s experience and gain valuable insight into ways to improve patient experience and the approach to care. These data allow patients and providers to come together in a shared decision-making process to improve the quality of care, treatment approaches, and interventions.
However, what constitutes a meaningful patient-reported may be a subject of debate. Patient-Reported Outcomes (PROs), as defined by the US Food and Drug Administration are “any report of the status of a patient’s health condition that comes directly from the patient, without interpretation of the patient’s response by a clinician or anyone else.”1 PROs may be used to measure the effect of a given intervention (medical or behavioral) on a specific concept, such as:
- A specific symptom or group of symptoms (e.g., fatigue, pain, depression), or
- A type of bodily or neuropsychological function or group of such functions.1
One specific type of patient-reported outcome is Quality of Life (QoL). According to the WHO, QoL is a multi-faceted construct that measures the composite of all positive and negative aspects of life.2 Health, in turn, was first described by the WHO Constitution in 1948 as the “state of complete physical, mental, and social well-being, and not merely the absence of disease or infirmity.”3 Health-related QoL (HRQoL) focuses on QoL and health in the context of any existing disease or ongoing treatment.4,5 Generic measures focus on QoL or HRQoL as a whole, while disease-specific measures contextualize HRQoL concerns within the confines of a specific disease (Table 1). When a measure focuses on one component of QoL, e.g. social functioning, this is called a dimensional assessment.5 Functioning involves assessment of task performance in the context of social, physical, or behavioral/psychological activities.6 Measures used in pediatric urology have encompassed one or all of these concepts (Figure 1).7
Table 1 Examples of HRQoL and Disease-Specific HRQoL for Social Health
* The SF-36, or 36-item Short Form Survey, is a set of generic and easily administered QoL measures. ** The QUALAS, or QUality of Life Assessment in Spina Bifida, is a disease-specific measure of QoL in the setting of Spina Bifida.
|Generic (SF-36) *||Disease-Specific (Spina Bifida, QUALAS) **|
|To what extent have your health or emotional problems interfered with normal social activities?8||To what extent do urine problems stop you from doing fun things?9|
Figure 1 Types of Measures and Relationships. Adapted from Raveendran, et al. 2021.7
Measure validation is a rigorous process by which a patient-reported outcome can be ascertained to be clinically meaningful, relevant, and assured to accurately represent the patient’s experiences.10 The validation criteria established by the Scientific Advisory Committee (SAC) are considered the gold-standard approach to measure validation, and include several detailed steps. These steps are confirmation of the conceptual and measurement model, reliability, validity, interpretability, respondent burden, alternative forms for administration, and multi-cultural or multi-lingual adaptations (Table 2).10 Studies using non-valid, or informal, measures to obtain PROs are still valuable, as the amalgamation of responses from such studies can assist with designing a conceptual model and then a measure for HRQoL in specific disease states.11 Pediatric urologists should therefore know how to evaluate a measure’s validity prior to use. In fact, some published measures have not passed rigorous validation techniques, and have been recommended for further revision prior to clinical administration.12
Table 2 SAC criteria.
|Conceptual Model||A justification and detailed description of what is being measured and why. This often involves a systematic literature review to create the conceptual model. Qualitative interviews are then used to obtain patient input to revise/refine the model. The model is then used to guide the design of the measure.|
|Reliability||Tests that ensure that the measure performs consistently and is free from random error. Some examples include test-retest reliability and inter-rater reliability.|
|Validity||The tests that confirm a measure accurately measures what it is intended to measure. Using the example above, validation of the QUALAS-T for Spina Bifida may require the comparison of patient scores for continence questions to those of the validated Pediatric Incontinence Questionnaire and the social functioning questions to the validated Strengths and Difficulties Questionnaire, etc. (Convergent validity)|
|Interpretability||Establishes whether the response range and scale are clinically meaningful. For example, if patients consistently respond Always (ceiling value) or Never (floor value) to a specific question, the question may not yield a meaningful scaled result, and needs to be altered.|
|Respondent Burden||The amount of time and number of questions required for the patient to complete the measure and for a provider to administer the measure.|
|Alternative Forms||The availability of several versions of the form for ease of administration.|
|Cultural/Multilingual Adaptations||The availability of culturally modified and translated forms to increase the generalizability of the measure.|
Selecting the Appropriate PRO in Pediatric Urology
Factors to Consider When Creating or Using a PRO in Pediatric Urology
For Which Population is the Measure Intended
The key question when examining a measure is whether the measure is designed for the pediatric population at large (e.g., a generic measure) or for patients with a specific disease process (e.g., spina bifida)? If a generic measure is used in a rare disease process, it must be with the understanding that the measure may not be sensitive enough to detect a difference in PROs or HRQoL. One such example is the Incontinence Symptom Index Pediatric (ISIP) form, which was first validated in bladder dysfunction, then in the more specific neurogenic bladder population, prior to its use in a spina bifida study.13,14
Who is the Intended Respondent
The age, developmental stage, and cognitive ability of the respondents are key factors when selecting an appropriate measure. Validated measures must usually be adapted for these considerations. For instance, the Incontinence Symptom Index, a measure that had been validated in adult women to assess symptom bother in either stress or urge incontinence, has been adapted and validated in the pediatric population for 11–17 year olds.13 In the process of developing the ISI-pediatric form, the authors added two domains more relevant to the pediatric population, nocturnal enuresis and insensate wetting symptoms and dropped an item that was irrelevant. Specific types of reports include:
- Youth Self Reports, which are the youth responding to a direct question about their experience. For example, a youth questionnaire for spina bifida asks, “To what extent do urine problems stop you from doing fun things?”9
- Caregiver Proxy Reports are forms designed to assess PROs in young children or the cognitively impaired, and are designed to be completed by the caregiver of the subject in question. In these reports, the caregiver reports on the subject’s outcomes based upon feedback from or observations of the subject. For instance, a proxy report in spina bifida might ask, “How, if at all, has your child’s social life been affected by continence-related concerns?”
- Parent or Caregiver Reports are designed to assess the caregiver’s personal experiences and condition or day-to-day life. For example, a parent/caregiver report in spina bifida might ask, “How much, if at all, have the patient’s continence-related concerns affected your [the parent/caregiver’s] social life?”
What are the Domains Being Measured
When developing a conceptual model, the model often contains multiple themes or facets. These themes can also be termed “domains.” For instance, the QUALAS-Teen (QUALAS-T) measure’s conceptual model includes Family and Independence and Bladder and Bowel function as the two major thematic domains.9 These domains were rigorously identified using expert consensus, detailed literature review, and patient interviews. Knowing the conceptual model and domains within can help investigators target specific areas/domains of HRQoL or create a more comprehensive measure of HRQoL.
Key Points: Selecting a Patient-Reported Outcome
- When choosing a measure for a study, pediatric urologists should take note of the following for each measure they select to ensure that findings are valid, reliable, and clinically meaningful:
- the target population
- intended respondents
- conceptual model/domains
Potential Applications of PROs in Pediatric Urology
PROs are a powerful tool in the arsenal of any pediatric urologist, and their potential applications in clinical practice are vast and depend on their purpose, i.e., QoL, treatment outcome, indications for surgical intervention, or any other disease-related outcome.
The application of QoL instruments in urology in particular has been highly variable. A recent integrative literature review of QoL instrument use in pediatric urology identified a total of 43 studies examining QoL in pediatric urology.7 Nine of these studies were focused on functioning, while the remainder examined some combination of QoL, HRQoL, disease-specific HRQoL, and functioning. Some specific examples of applications in pediatric urology include the following:
As a Primary Treatment Outcome in Clinical Trials
In a non-inferiority study in the voiding dysfunction population, the validated Vancouver Non-Neurogenic Lower Urinary Tract Dysfunction/Dysfunctional Elimination Syndrome Questionnaire was used to measure QoL outcomes in a randomized clinical trial comparing outcomes after a bladder training video versus standard urotherapy.15 The bladder training video was not inferior to standard urotherapy in QoL score improvements.
To Determine Indications for Operation or Re-Operation
In a cohort of 25 women with bladder exstrophy and pelvic organ prolapse, the authors demonstrated improvements in both continence and sexual function PROs after correction of pelvic organ prolapse.16
To Obtain Information About the Natural History of an Illness
One group validated a parent, parent-proxy, and child measure to characterize the experiences of children and parents of children with differences of sexual differentiation (DSD), and characterized parental stress related to diagnosis, surgery, and decisional regret.17
To Identify Patient Priorities
One study tried to identify how patient preferences could dictate the surgical approach to kidney or pelvic surgery, using an informal crowdsourcing survey, and found that patients preferred a Pfannenstiel incision for pelvic surgery and a dorsal lumbotomy incision for kidney surgery to well-hidden abdominal port sites for robotic surgery.18
To Assess Long-Term Psychosocial Outcomes Related to Genitourinary
Anomalies or Reconstruction
Using generic, valid measures for anxiety and depression, one group found that male caregivers of children with life-threatening conditions and ambiguous genitalia were more likely to exhibit symptoms of anxiety and require additional support when compared to female caregivers.19 The authors concluded that male caregivers may require active support earlier after diagnosis.
Current Measures in Pediatric Urology
Informal measures have been used in pediatric urology to assess outcomes after urologic procedures. Two such examples include an informal survey was administered to the families of patients whose children had undergone meatotomy.20 The survey focused on age, pain control, and urinary tract symptoms, and identified that 79% of families reported major improvements after the procedure. Another informal survey found that adult patients who had undergone pediatric procedures preferred Pfannenstiel incisions for pelvic surgery and dorsal lumbotomy for kidney surgery to minimally invasive abdominal port sites.18 Many valid measures have been produced to assess disease-specific patient experiences in the areas of bowel and bladder dysfunction, hypospadias, spina bifida, vesicoureteral reflux, transplant, and DSD. Summaries of existing measures are available in Table 3.
Table 3 Valid, Disease-Specific Measures in Pediatric Urology. * Meets SAC criteria for rigorous psychometric validation, see Table 2.
|Condition||Measure||Items||Details||Age of Intended Respondents||Validation Data|
|Bowel and Bladder Dysfunction||DVSS||10||Domains not delineated, includes bladder and bowel questions||3-10 y/o, 1 question for parent||Farhat, et al. 200021|
|Bowel and Bladder Dysfunction||DVISS||13||Domains not delineated, includes bladder and bowel questions; Concise form Concise form domains: daytime incontinence, enuresis, hesitancy, urgency||Parent-proxy report of 4-10 y/o||Akbal, et al. 200522|
|Bowel and Bladder Dysfunction||PIN-Q||20||Domains: social interaction, self-esteem, family and home, body image, independence, mental health, treatment effects||6-15 y/o||Bower, et al. 200623,24,25|
|Bowel and Bladder Dysfunction||VSS||14||Domains not delineated, includes bladder and bowel questions||4-16 y/o||Afshar, et al. 200926|
|Bowel and Bladder Dysfunction||QDES||15||Domains not delineated, but questions cover bladder/bowel symptoms, family history, psychological stress, infection risk||Children (age not specified), and parents||Tokgoz, et al. 200727|
|Bowel and Bladder Dysfunction||ISIP||11||Domains: stress incontinence, urge incontinence, insensate incontinence, nocturnal urinary symptoms, pad use, impairment scale; Also validated for neurogenic incontinence||11-17 y/o||Nelson, et al. 200713 Hubert-Chan, et al. 201514|
|Bowel and Bladder Dysfunction||Iowa BBD Measure||18||Domains: urinary incontinence, constipation, bowel symptoms, nocturnal enuresis, lower urinary tract symptoms||Parent proxy for 3-8 y/o; Self report 9-19 y/o||Anwar, et al. 201928|
|Hypospadias||GPS||9 (child) / 20 (Adult)||Compared patient to surgeon estimations of penile cosmesis; One component focused on Genital Perception (GPS), other component on Body Perception (BPS)||9-18 y/o, Adults||Mureau, et al 199529|
|Hypospadias||PPPS/PPS||4||Compared patient estimation of penile cosmesis to those of care providers (nurses, surgeons); Focuses on penile appearance only||6-17 y/o, Adults||Weber, et al. 200830 Weber, et al. 201331|
|Hypospadias||HOSE||5||Compared parent estimations to care provider (nurses, surgeons) estimations of penile cosmesis; Includes 1 item about urinary stream||Parent-proxy report for children (median age 23 months)||Holland, et al. 200132|
|Hypospadias||PGWBI||22||Evaluates overall emotional state, including domains for anxiety, depression, health, self-control, positive well-being, and vitality||14-25 y/o males||Andersson, et al. 201833|
|Hypospadias||BESAA||30||Evaluates feelings about his own appearance and perceptions of how others see his appearance||14-25 y/o males||Andersson, et al. 201833|
|Hypospadias||SIGHT||11||Asks patients to reflect on masculinity, general genitalia appearance, intercourse frequency, and erections||14 -18 y/o males, Adults (> 18 y/o)||Ardelt, et al. 201734|
|Vesicoureteral Reflux||G-VUR||18||Assesses physical function, psychological well-being, school activities, disease-specific symptoms, and satisfaction with care||Parent-proxy for child||Minnillo, et al. 201235|
|Vesicoureteral Reflux||PO-VUR||27||Assesses Disease-specific symptoms, objective measures of function, and complications after surgery||Parent-proxy for child||Minnillo, et al. 201235|
|Renal Transplant||Peds QL ESRD Module||34||Assesses 7 domains: General Fatigue, About My Kidney Disease, Treatment Problems, Family and Peer Interactions, Worry, Perceived Physical Appearance, Communication||5-18 y/o, Parent-proxy for child||Goldstein, et al. 200736|
|Renal Transplant||Pat 2.0_Gen||57||Seven Domains: Family structure and resources, Family social support, Family problems, Parent Stress Reactions, Family beliefs, Child Problems and sibling Problems||Parent-proxy report for 7-19 y/o||Pai, et al. 201137|
|Spina Bifida||HR-QoL SB||44–47||Evaluates functional domains including social, emotional, financial, medical, intellectual, environmental, independence, recreation, physical, vocational; No questions on continence (bowel/bladder)||5-12 y/o, 13-20 y/o||Parkin, et al. 1997|
|Spina Bifida||QUALAS*||10 items (C)/10 items (T)/15-items (A)||C: 2 domains include Esteem and Independence, Bowel and Bladder / T: 2 domains include Family and Independence, Bladder and Bowel / A: 3 domains include Health and Relationships, Esteem and Sexuality, Bladder and Bowel||8-12 y/o (C) 13-17 y/o (T) 17 and older (A) BBD items validated for 8 y/o-adult||Szymanski, et al. 201538 Szymanski, et al. 201639 Szymanski, et al. 20179|
|DSD||QoL-DSD-Proxy||24||Domains include: physical functioning, gender expression, socio-emotional functioning, medical concerns||Proxy report for children from birth-6 y/o||Alpern, et al. 201617|
|DSD||QoL-DSD-Parent||55||Domains include: role functioning, decision-making, gender expression, social functioning, emotional functioning, future concerns, healthcare communication, disclosure, medications, surgery, doctor’s visits, and earliest experiences||Parents||Alpern, et al. 201617|
|Cloacal Anomalies||HAQL||44||Domains include: laxative diet, constipating diet, diarrhea, constipation, fecal continence, urinary continence, emotional functioning, social functioning, body image, physical symptoms, and sexual functioning||8-11 y/o form 12-16 y/o form 17 and older form||Hanneman, et al. 200140 Wigander, et al. 201441 Baayen, et al. 201742|
Bowel and Bladder Dysfunction
PROs in bowel and bladder dysfunction are widely used in pediatric urology to assess treatment efficacy. Widely used and valid measures include the following (Table 3).
Dysfunctional Voiding Scoring System
This measure was the first measure of its kind, and was designed to quantitatively measure bowel and bladder symptoms from a validated patient report.43 Of note, one of the authors of this chapter developed this measure. Questions were initially adapted from the International Reflux Study in children, and underwent modifications in subsequent studies.21,22 The instrument underwent a name change in an effort to use standardized International Children’s Continence Society (ICCS) terminology.44 This measure contains 10 items focused on effects of incontinence, bowel movements, urgency, straining, dysuria, and a parent-proxy report of child’s stress level related to the voiding symptoms.21 As a result of its rigorous validation and minimal response burden, this measure has been used in numerous studies, and has even been validated and culturally adapted for Serbian, Thai, Japanese, Brazilian, Korean, and Iranian populations.45,46,47,48,49,50
Pediatric Incontinence Questionnaire
This is a robust, 20-question measure with questions focused on the effects of incontinence on QoL.23,24 It is also the only pediatric BBD measure that directly assesses effects of incontinence on QoL. The authors identified several relevant domains in their conceptual model, including social interactions, self-esteem, family and home, body image, independence, mental health and treatment effects. The measure has also been culturally adapted and translated for Chinese-speaking, Swedish, and Dutch-speaking populations.24,25
Vancouver Symptom Score
This is a 14-item measure that includes assessments of bother related to voiding and storage symptoms, as well as constipation. The measure was validated in a population of 4–16-year-old children (males and females) and has since been translated and adapted to a Dutch population.26,51
This measure was adapted from an adult measure to assess symptom bother in either stress or urge incontinence in women. The authors focused on adaptation and validation in the pediatric population for 11-17 year olds, using rigorous psychometric validation techniques.13 As previously mentioned, the authors also revised the conceptual model for incontinence in this population, and identified that nocturnal enuresis and insensate wetting symptoms were of unique importance to the pediatric population. Another group successfully confirmed the reliability and validity of the ISI-P for patients with spina bifida and neurogenic incontinence.14
Iowa Bowel and Bladder Dysfunction Questionnaire
This measure underwent a rigorous development and validation process that closely assimilated the SAC criteria.28 Further, the authors compared the discriminant validity, or similarity of overall scaled responses of this measure to the VSS described above. The authors developed a subclassification within their instrument that would more reliability characterize the type of BBD experienced by the patients based upon their responses. Questions assessed the presence and absence of symptoms, as well as symptom bother. The result was an 18-item questionnaire that was validated in 3–19-year-olds. Parent-proxy reports were used for 3–8-year-olds, and self-reports were used for patients aged 9 and above.
Questionnaire for Dysfunctional Elimination Syndrome
Other existing measures requiring further validation include the Questionnaire for Dysfunctional Elimination Syndrome (QDES) 15-item measure that includes both a patient self-report and a parent-proxy report.27 Measure items focus on bowel and bladder symptoms, psychological stress assessments, infectious symptoms, and family history assessments. In addition, two adaptations of the DVSS, the Dysfunctional voiding and incontinence scoring system (DVISS), and its shortened version, the Concise Lower Urinary Tract Dysfunction Symptom Scale were developed but failed tests of reliability.22,52
Key Points: PROs in Bowel and Bladder Dysfunction
- 8 validated measures exist.
- Each measure has different strengths.
- Some measures, like the ISIP, focus exclusively on urinary symptoms, while others incorporate bowel symptoms and psychological symptoms.
- The DVSS, PIN-Q, ISIP, and Iowa form have the most robust validation data.
- The PIN-Q form incorporates QoL assessments.
PROs in hypospadias have been extensively explored for a myriad of purposes, including comparison of surgeon and patient priorities in cosmesis, effects of surgical technique, studying the varying effects of disease complexity on psychosocial health, and assessment of urinary symptoms.31,30,53,54,29,55 The authors of this chapter proposed a conceptual model for hypospadias-specific HRQoL was proposed based upon thematic coding of existing literature, and includes domains of penile appearance, voiding function, social function, psychological health, and sexual health.11 When looking at existing PROs in hypospadias, many measures have been used to explore penile appearance, voiding function and sexual health, but no single measure comprehensively explores all proposed domains.56 Existing measures in hypospadias have made important strides in determining patient priorities related to hypospadias surgery. Based upon our previous rigorous assessment of validation, the highest-quality measures in hypospadias include the following:
Genital Perception Scale
The Genital Perception Scale (GPS) was the first PRO to be developed in the hypospadias population.54,29 The measure reviewed glans anatomy, penile girth, flaccid penile length, and appearance of the scrotum and testes. It was validated in two forms, a pediatric form and an adult form, and demonstrated that proximal hypospadias patients tended to have a lower GPS score. A Body Perception Score (BPS) to understand patients’ body image was also developed with the GPS but did not meet validity criteria. To date, this measure has not been used for additional clinical studies.
Pediatric Penile Perception Scale/Penile Perception Scale
Pediatric Penile Perception Scale/Penile Perception Scale (PPPS/PPS) was dedicated to obtaining the patient’s opinion on post-operative appearance.31,30 This measure focuses solely on aspects of penile appearance, including general appearance, meatal position, glans anatomy, and shaft skin. It has been used in several other studies as a measure of technical outcomes after hypospadias repair.10,57 The pediatric form was written for 6–17-year-old males, while the adult form was adapted for older patients.
Hypospadias Objective Score System
Hypospadias Objective Score System (HOSE) was a 5-point measure designed to compare parent-proxy PROs focused on penile appearance to estimations of surgeons and RNs.32 In addition to an assessment of penile appearance, the authors included a question on voiding function to increase utility. Its minimal response burden and wide applicability have led to its use in multiple studies of surgical technique.58,59
Body-Esteem Scale for Adolescents and Adults
The Body-Esteem Scale for Adolescents and Adults (BESAA) measures how patients feel about their own appearance and how patients believe others perceive their appearance.60 This scale was shown to be both reliable and valid in adolescents and adults with hypospadias.33
Mini-International Prostate Symptom Score
Measures requiring further validation include the mini-International Prostate Symptom Score (mini-IPSS), which was designed to assess penile appearance, voiding function, and parental worry.55 The measure was translated into English and Spanish and has been used in multiple studies reviewing postoperative preferences, but validation studies are pending.61,62,63
Psychological Well-Being Index
Psychological Well-Being Index (PGWBI) is a generic valid scale that measures domains pertinent to anxiety, depression, health, well-being, vitality, and self-control.33 One group demonstrated that this measure was a reliable measure of psychological well-being in hypospadias patients but did not meet other criteria for validity and interpretability.
Satisfaction in Genital Hypospadias Treatment
The Satisfaction in Genital Hypospadias Treatment (SIGHT) is a German questionnaire designed to assess psychosexual development in adolescents who had previously undergone hypospadias surgery, and was shown to be valid.34 However, its use in research has been limited due to the absence of additional cultural and linguistic adaptations. In addition, generic measures of QoL and function have been used to evaluate the associations between penile appearance and overall HRQoL, social functioning, and psychological or sexual functioning.64,65,66 However, a narrative review performed by the authors of this chapter has demonstrated that generic measures often yield conflicting results, further supporting the need for a comprehensive, disease-specific assessment of HRQoL in hypospadias.11
Key Points: PROs in Hypospadias
- 7 valid measures exist.
- Most measures focus on aspects of penile appearance, while two include assessments of voiding function, and 2 others include psychological function.
- One measure reviews sexual function.
Valid measures in Vesicoureteral reflux have focused on pre and post-operative experiences, and include assessments of physical function, psychological well-being, school activity function, disease-related symptoms and satisfaction with care.35 Valid measures in transplant focus on general fatigue, facets of kidney disease, treatment problems, family and peer interactions, worry, physical appearance, communication, and psychosocial risk factors.36,67,37
Valid measures of QoL in Spina Bifida have investigated by pediatricians, rehabilitation medicine specialists, and pediatric urologists.9 The measures produced by urologists have a specific focus on understanding the effects of urinary and bowel function,9,38,39 while measures produced by non-urologists, such as the HRQoL-SB, often must be used in conjunction with continence measures to understand the effects of continence on QoL.68 Valid measures in BBD have also been used as treatment outcome measures in neurogenic incontinence.14,69
Quality of Life Assessment in Spina Bifida
The QUAlity of Life Assessment in Spina Bifida (QUALAS) was designed to be a concise, comprehensive measure of disease-specific HRQoL that incorporated an assessment of bowel and bladder function.9,38,39 Its conceptual model was rigorously developed with extensive stakeholder input, and tests of reliability and convergent and discriminant validity yielded excellent results. An additional benefit is that is has been adapted for childhood, teenaged, and adult populations.9,38 This adaptation will allow for longitudinal studies of HRQoL in spina bifida patients. The battery of QUALAS measures (Child, Teenager, and Adult) has been adapted for a Brazilian70 and Japanese-speaking population.71,72
Key Points: PROs in Spina Bifida
- 2 valid measures exist.
- The QUALAS measures have been adapted for pediatric, adolescent, and adult populations and include a measure of bowel and bladder function.
- Measures of continence in BBD have also been validated in the spina bifida population.
Measures in Complex Urologic Conditions
Differences in Sexual Development
One valid, disease-specific measure of HRQoL exists in the DSD population.17 This included a battery of measures, include a proxy measure for child experiences (QOL-DSD-Proxy), and the parent self-report form (QoL-DSD-Parent). The authors found that, due to the heterogeneity of the conditions represented, validity was limited for specific sub-domains of HRQoL such as physical functioning, based upon the variety of subtypes of DSD syndromes. Other assessments using informal or generic measures have explored psychosocial and psychosexual health, gender expression, concerns about the future, parental stress, and the effects of communication approaches by the healthcare team.73,74,75,76,77,78,79,80,81,82,83,84,85,86,87,88,89,90
The Hirschsprung’s Disease/Anorectal Malformation Quality of Life Questionnaire (HAQL) is the only disease-specific, valid measure developed for this population. Domains assessed included a type of diet, diarrhea, constipation, fecal continence, urinary continence, emotional functioning, social functioning, body image, physical symptoms, and sexual functioning.42,40,91 Generic and informal measures of QoL also suggest that continence is closely tied to psychosocial and emotional outcomes.67,91
Only one standardized measure was developed and never validated in this population.92 Social and emotional were found to be related worry about peers discovering their primary condition. Generic measures have found that pelvic organ prolapse correction is critical to overall QoL,93 and that the condition of exstrophy negative affects psychosocial and sexual interactions.94,95,96,97
Key Points: PROs in Complex Urologic Conditions
- One valid measure exists for DSD and one for Anorectal Malformations.
- Several informal surveys have been used in conjunction with generic measures to assess psychosexual development and function in children and adults with DSD, cloacal anomalies, and exstrophy/epispadias.
PROs in Pediatric Urology have evolved to encompass common conditions, such as bowel and bladder dysfunction, to very specific conditions, such as DSD. Existing measures provide important information to providers about how better to support our patients throughout the course of their care for a specific disease. The most psychometrically robust measures are found in bowel and bladder dysfunction and spina bifida, while ongoing work is being done to provide similarly rigorous measures in hypospadias, DSD, and exstrophy. Often, our existing work is limited by the lack of an overarching conceptual model, which should help with the creation of comprehensive, clinically meaningful measures that allow us to closely monitor patient outcomes. Another challenge is finding an efficient way to administer and interpret measures in day-to day practice for the clinical care and research. While electronic administration outside of the hospital setting would permit more people to complete the PROs,98 ensuring access to such resources for all patients could be challenging. Additional work has suggested that patients who are economically disadvantaged or from a minority population may have more difficulty completing PROs, likely due to limited resources.99 Understanding how to equitably, efficiently, and meaningfully capture the patient experience remains a focus of ongoing work.
To date, much of the work focusing on PRO in pediatric urology has focused on QoL and functional scores. Emerging research is focused on identifying the lived experience of patients and patient priorities during their care for a variety of diseases.100 This process involves obtaining extensive input from patients and families on their experiences, also known as the identification of “stakeholders.” Patient advocacy, with the identification of such stakeholders on the patient and provider level, will help tailor the creation of measures for PROs, and will also help providers take action to support patient priorities. These efforts will likely change the shared-decision making model of care to a patient-prioritized model of care, with a focus on patient-prioritized outcomes, rather than simply patient-reported outcomes.
Summary and Conclusions
PROs have provided valuable information to pediatric urologists about a variety of urologic conditions and have the power to inform both the medical and surgical treatment of urologic disease. Future studies focusing on patient-prioritized medicine will yield more rigorous and clinically valuable measures that enhance the provider’s ability to provide individualized care.
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- Raveendran L, Koyle M, Bagli D, Twardowski K, Cicci N, Ronen GM, et al.. Integrative review and evaluation of quality of life related instruments in pediatric urology. J Pediatr Urol 2021; 17 (4): 443.e1–443.e14. DOI: 10.1016/j.jpurol.2021.03.011.
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Last updated: 2023-02-22 09:32