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  1. American Family Children's Hospital, Madison, WI, USA
  2. Department of Urology, Division of Pediatric Urology, American Family Children's Hospital, Madison, WI, USA


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 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.

Criterion Definitions/Examples
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

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
Exstrophy/Epispadias [None]     N/A N/A

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

Incontinence-Symptom Index-Pediatric

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.

Vesicoureteral Reflux

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

Spina Bifida

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

Anorectal Malformations

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.

Future Directions

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.


  1. H. Services USD, D. Administration F, R. C C, editors. Guidance for Industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims. In: . 20993-0002: Office of Communications, Division of Drug Information Center for Drug Evaluation and Research Food and Drug Administration. Silver Spring, MD: 2009. DOI: 10.1186/1477-7525-4-79.
  2. Group TW. The World Health Organization quality of life assessment (WHOQOL): Development and general psychometric properties. Soc Sci Med 1998; 46 (12): 1569–1585. DOI: 10.1016/s0277-9536(98)00009-4.
  3. Sartorius N. Liberal democracy: its multiple meanings in United States democracy promotion. Democratic Futures 2006; 47: 137–161. DOI: 10.4324/9780203073131-16.
  4. Prevention CDC. Measuring healthy days: Population assessment of health-related quality of life. Atlanta, Georgia: Centers for Disease Control and Prevention; 2000, DOI: 10.1037/e372122004-001.
  5. Fayers P. M.: Quality of Life. 3rd ed., Southern Gate, Chichester: The Atrium; 2016.
  6. WHO: Internal Classification of Functioning, Disability, and health. Geneva: Icf; 2001, DOI: 10.1007/978-0-387-78665-0_5935.
  7. 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.
  8. Bech P. Social Functioning. CNS Drugs 2005; 19 (4): 313–324. DOI: 10.2165/00023210-200519040-00004.
  9. Szymański KM, Misseri R, Whittam B. Validation of QUALAS-T, a health-related quality of life instrument for teenagers with spina bifida. Cent European J Urol 2017; 70: 306, DOI: 10.5173/ceju.2016.1195.
  10. Lohr K, Aaronson N, Alonso J, Burnam A, Patrick D, Perrin E, et al.. C16. Evaluating quality-of-life and health status instruments: development of scientific review criteria. Clin Ther 2002; 18: 30. DOI: 10.1016/s0149-2918(96)80126-3.
  11. Bhatia VP, Mittal AG, Austin PF, Hilliard ME. The hypospadias-specific health-related quality of life conceptual framework: a scoping review of the literature. Qual Life Res 2021; 30 (6): 1537–1546. DOI: 10.1007/s11136-021-02773-w.
  12. Bachmann C, Ackmann C, Janhsen E, Steuber C, Bachmann H, Lehr D. Clinical evaluation of the short-form pediatric enuresis module to assess quality of life. Neurourol Urodyn 2010; 29 (8): 1397–1402. DOI: 10.1002/nau.20896.
  13. Nelson CP, Park JM, Bloom DA, Wan J, Dunn RL, Wei JT. Incontinence Symptom Index-Pediatric: Development and Initial Validation of a Urinary Incontinence Instrument for the Older Pediatric Population. J Urol 2007; 178 (4s): 1763–1767. DOI: 10.1016/j.juro.2007.03.180.
  14. Hubert KC, Sideridis G, Sherlock R, Rosoklija I, Kringle G, Johnson K, et al.. Urinary incontinence in spina bifida: Initial instrument validation. Res Dev Disabil 2015; 40: 42–50. DOI: 10.1016/j.ridd.2015.01.008.
  15. Brownrigg N, Braga LH, Rickard M, Farrokhyar F, Easterbrook B, Dekirmendjian A, et al.. The impact of a bladder training video versus standard urotherapy on quality of life of children with bladder and bowel dysfunction: A randomized controlled trial. J Pediatr Urol 2017; 13 (4): 374.e1–374.e8. DOI: 10.1016/j.jpurol.2017.06.005.
  16. Everett RG, Lue KM, Reddy SS, Friedlander DA, Alexander CE, Young EE, et al.. Patient-Reported Impact of Pelvic Organ Prolapse on Continence and Sexual Function in Women With Exstrophy-Epispadias Complex. Female Pelvic Med Reconstr Surg 2017; 23 (6): 377–381. DOI: 10.1097/spv.0000000000000414.
  17. Alpern AN, Gardner M, Kogan B, Sandberg DE, Quittner AL. Development of Health-Related Quality of Life Instruments for Young Children With Disorders of Sex Development (DSD) and Their Parents. J Pediatr Psychol 2017; 42: jsw022. DOI: 10.1093/jpepsy/jsw022.
  18. Garcia-Roig ML, Travers C, McCracken C, Cerwinka W, Kirsch JM, Kirsch AJ. Surgical Scar Location Preference for Pediatric Kidney and Pelvic Surgery: A Crowdsourced Survey. J Urol 2017; 197 (3 Part 2): 911–919. DOI: 10.1016/j.juro.2016.11.033.
  19. Wolfe-Christensen C, Fedele DA, Mullins LL, Lakshmanan Y, Wisniewski AB. Differences in anxiety and depression between male and female caregivers of children with a disorder of sex development. J Pediatr Endocrinol Metab 2014; 27 (7-8): 617, DOI: 10.1515/jpem-2014-0102.
  20. Varda BK, Logvinenko T, Bauer S, Cilento B, Yu RN, Nelson CP. Minor procedure, major impact: Patient-reported outcomes following urethral meatotomy. J Pediatr Urol 2018; 14 (2): 165.e1–165.e5. DOI: 10.1016/j.jpurol.2017.10.012.
  21. Jiang R, Kelly MS, Routh JC. Commentary to ‘Assessment of pediatric bowel and bladder dysfunction: a critical appraisal of the literature.’ J Pediatr Urol 2018; 14 (6): 502. DOI: 10.1016/j.jpurol.2018.09.010.
  22. FARHAT WALID, B??GLI DARIUSJ, CAPOLICCHIO GIANPAOLO, O???REILLY SHEILA, MERGUERIAN PAULA, KHOURY ANTOINE, et al.. The Dysfunctional Voiding Scoring System: Quantitative Standardization Of Dysfunctional Voiding Symptoms In Children. J Urol 2000; 164: 1011–1015. DOI: 10.1097/00005392-200009020-00023.
  23. AKBAL CEM, GENC YASEMIN, BURGU BERK, OZDEN ENDER, TEKGUL SERDAR. Dysfunctional Voiding And Incontinence Scoring System: Quantitative Evaluation Of Incontinence Symptoms In Pediatric Population. J Urol 2005; 173 (3): 969–973. DOI: 10.1097/01.ju.0000152183.91888.f6.
  24. Wallis MC, Khoury AE. Symptom score for lower urinary tract dysfunction in pediatric urology. Curr Urol Rep 2006; 7 (2): 136–142. DOI: 10.1007/s11934-006-0073-2.
  25. Cirovic D, Petronic I, Nikolic D, Knezevic T, Vukadinovic V, Pavicevic P. Validation of Serbian Version of Dysfunctional Voiding Symptom Score (DVSS) Questionnaire. J Clin Med 2018; 7 (8): 217. DOI: 10.3390/jcm7080217.
  26. Calado AA, Araujo EM, Barroso Jr. U, Netto JMB, Zerati Filho M, Macedo Jr. A, et al.. Cross-cultural adaptation of the dysfunctional voiding score symptom (DVSS) questionnaire for Brazilian children. Int Braz J Urol 2010; 36 (4): 458–463. DOI: 10.1590/s1677-55382010000400009.
  27. Imamura M, Usui T, Johnin K, Yoshimura K, Farhat W, Kanematsu A, et al.. Cross-cultural Validated Adaptation Of Dysfunctional Voiding Symptom Score (Dvss) To Japanese Language And Cognitive Linguistics In Questionnaire For Pediatric Patients. Nihon Hinyokika Gakkai Zasshi 2014; 105 (3): 112–121. DOI: 10.5980/jpnjurol.105.112.
  28. Lee H-E, Farhat W, Park K. Translation and Linguistic Validation of the Korean Version of the Dysfunctional Voiding Symptom Score. J Korean Med Sci 2014; 29 (3): 400. DOI: 10.3346/jkms.2014.29.3.400.
  29. Chang S-J, Chen TH-H, Su C-C, Yang SS-D. Exploratory factory analysis and predicted probabilities of a Chinese version of Dysfunctional Voiding Symptom Score (DVSS) questionnaire. Neurourol Urodyn 2016; 31 (8): 1247–1251. DOI: 10.1002/nau.22254.
  30. POURMOMENY AA, ZARGHAM M, FANI M. Reliability and Validity of the Quality of Life Questionnaire in Iranian Patients with Lower Urinary Tract Symptoms. Low Urin Tract Symptoms 2013; 10 (1): 93–100. DOI: 10.1111/luts.12147.
  31. Bower WF, Wong EMC, Yeung CK. Development of a validated quality of life tool specific to children with Bladder dysfunction. Neurourol Urodyn 2006; 25 (3): 221–227. DOI: 10.1002/nau.20171.
  32. Bower WF, Sit FKY, Bluyssen N, Wong EMC, Yeung CK. PinQ: A valid, reliable and reproducible quality-of-life measure in children with bladder dysfunction. J Pediatr Urol 2006; 2 (3): 185–189. DOI: 10.1016/j.jpurol.2005.07.004.
  33. Jönson Ring I, Nevéus T, Markström A, Arnrup K, Bazargani F. Nocturnal enuresis impaired children’s quality of life and friendships. Acta Paediatr 2017; 106 (5): 806–811. DOI: 10.1111/apa.13787.
  34. Afshar K, Mirbagheri A, Scott H, MacNeily AE. Development of a Symptom Score for Dysfunctional Elimination Syndrome. J Urol 1939; 182 (4s): 1939–1944. DOI: 10.1016/j.juro.2009.03.009.
  35. Hoen LA ’t, Korfage IJ, Verhallen JTCM, Ledden-Klok MJ van, Hoek J van den, Blok BFM, et al.. Vancouver Symptom Score for Dysfunctional Elimination Syndrome: Reliability and Validity of the Dutch Version. J Urol 2016; 196 (2): 536–541. DOI: 10.1016/j.juro.2016.02.2973.
  36. Anwar T, Cooper CS, Lockwood G, Ferguson KJ, Barlow PB, Storm DW. Assessment and validation of a screening questionnaire for the diagnosis of pediatric bladder and bowel dysfunction. J Pediatr Urol 2019; 15 (5): 528.e1–528.e8. DOI: 10.1016/j.jpurol.2019.07.016.
  37. Tokgöz H, Tan MÖ, Şen İ, İlhan MN, Biri H, Bozkırlı İ. Assessment of urinary symptoms in children with dysfunctional elimination syndrome. Int Urol Nephrol 2007; 39 (2): 425–436. DOI: 10.1007/s11255-006-9062-0.
  38. Ebiloglu T, Kaya E, Kopru B. Concised Form for Lower Urinary Tract Dysfunction Symptom Scale in Children. J Clin Diagn Res 2016; 10: 04, DOI: 10.7860/jcdr/2016/19056.8130.
  39. Weber DM, Landolt MA, Gobet R, Kalisch M, Greeff NK. The Penile Perception Score: An Instrument Enabling Evaluation by Surgeons and Patient Self-Assessment After Hypospadias Repair. J Urol 2013; 189 (1): 189–193. DOI: 10.1016/j.juro.2012.08.178.
  40. Weber DM, Schonbucher VB, Landolt MA. Faculty Opinions recommendation of The Pediatric Penile Perception Score: an instrument for patient self-assessment and surgeon evaluation after hypospadias repair. Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature 2008; 180: 1080, DOI: 10.3410/f.719560881.793500484.
  41. Helmy TE, Hashem A, Mursi K, AbdelHalim A, Hafez A, Dawaba MS. Does intraoperative penile tourniquet application during hypospadias repair affect the patients and surgeons reported outcomes? A randomized controlled trial. J Pediatr Urol 2020; 16 (5): 683.e1–683.e7. DOI: 10.1016/j.jpurol.2020.07.044.
  42. Mureau MAM, Slijper FME, Meulen JC van der, Verhulst FC, Slob KA. Psychosexual Adjustment of Men Who Underwent Hypospadias Repair. J Urol 1997; 141: 1351–1355. DOI: 10.1097/00005392-199510000-00023.
  43. Mureau MAM, Slijper FME, Slob AK, Verhulst FC. Psychosocial Functioning of Children, Adolescents, and Adults Following Hypospadias Surgery: A Comparative Study. J Pediatr Psychol 1997; 22 (3): 371–387. DOI: 10.1093/jpepsy/22.3.371.
  44. Keays MA, Starke N, Lee SC. Faculty Opinions recommendation of Patient Reported Outcomes in Preoperative and Postoperative Patients with Hypospadias. Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature 2016; 195: 1215, DOI: 10.3410/f.726183244.793522973.
  45. Bhatia VP, Hilliard ME, Austin PF, Mittal AG. Evaluating quality of patient-reported outcome measures in patients with hypospadias. J Pediatr Urol 2021; 17 (1): 50–58. DOI: 10.1016/j.jpurol.2020.11.043.
  46. Cui X, He Y, Huang W, Chen L, Wang Y, Zhou C. Clinical efficacy of transverse preputial island flap urethroplasty for single-stage correction of proximal hypospadias: a single-centre experience in Chinese patients. BMC Urol 2020; 20 (1): 118, DOI: 10.1186/s12894-020-00686-3.
  47. Han JH, Park MU, Lee SM, Park S, Song SH, Kim KS. Effect of additional Tunica Vaginalis flap coverage protecting urethrocutaneous fistulas in tubularized incised plate urethroplasty: A prospective, randomized controlled trial. Eur Urol Open Sci 2020; 19: e1134. DOI: 10.1016/s2666-1683(20)33340-1.
  48. Yang H, Xuan X-xiao, Hu D-lai, Zhang H, Shu Q, Guo X-dong, et al.. Comparison of effect between dartos fascia and tunica vaginalis fascia in TIP urethroplasty: a meta-analysis of comparative studies. BMC Urol 2020; 20 (1): 161, DOI: 10.1186/s12894-020-00737-9.
  49. Duan SX, Li J, Jiang X, Zhang X, Ou W, Fu M, et al.. Diagnosis and Treatment of Hypospadias With Megameatus Intact Prepuce. Front Pediatr 2020; 8: 135–138. DOI: 10.3389/fped.2020.00128.
  50. Holland AJA, Smith GHH, Ross FI, Cass DT. HOSE: an objective scoring system for evaluating the results of hypospadias surgery. BJU Int 2001; 88 (3): 255–258. DOI: 10.1046/j.1464-410x.2001.02280.x.
  51. Khirallah M, El-dossuky N. Hybrid Mathieu Urethroplasty: A Simple Modification Outcomes. Res Rep Urol 2021; Volume 13: 473–478. DOI: 10.2147/rru.s318900.
  52. Ali MM, El-Hawy MM, Galal EM. Results of two-stage transverse preputial island flap urethroplasty for proximal hypospadias with chordee that mandate division of the urethral plate. Cent European J Urol 2021; 74: 89, DOI: 10.5173/ceju.2021.0245.r2.
  53. Al-Adl AM, Abdel Aal AM, El-Karamany TM, Noureldin YA. Two-stage repair of proximal hypospadias with moderate to severe chordee using inner preputial skin graft: prospective evaluation of functional and cosmetic outcomes. World J Urol 2020; 38 (11): 2873–2879. DOI: 10.1007/s00345-020-03075-5.
  54. Khalil M, Gharib T, El-shaer W, Sebaey A, Elmohamady B, Elgamal K. Mathieu technique with incision of the urethral plate versus standard tubularised incised-plate urethroplasty in primary repair of distal hypospadias: A prospective randomised study. Arab J Urol 2017; 15 (3): 242–247. DOI: 10.1016/j.aju.2017.03.007.
  55. Hongyong J, Shuzhu C, Min W, Weijing Y, Yidong L. Comparison of lingual mucosa and buccal mucosa grafts used in inlay urethroplasty in failed hypospadias of pre-pubertal boys in a Chinese group. PLoS One 2017; 12 (8): e0182803. DOI: 10.1371/journal.pone.0182803.
  56. Shuzhu C, Min W, Yidong L, Weijing Y. Selecting the right method for hypospadias repair to achieve optimal results for the primary situation. Springerplus 2016; 5 (1): 1624, DOI: 10.1186/s40064-016-3314-y.
  57. Mendelson BK, Mendelson MJ, White DR. Body-Esteem Scale for Adolescents and Adults. PsycTESTS Dataset 2001; 76: 90, DOI: 10.1037/t00047-000.
  58. Andersson M, Sjöström S, Wängqvist M, Örtqvist L, Nordenskjöld A, Holmdahl G. Psychosocial and Sexual Outcomes in Adolescents following Surgery for Proximal Hypospadias in Childhood. J Urol 2018; 200 (6): 1362–1370. DOI: 10.1016/j.juro.2018.06.032.
  59. Chan KH, Panoch J, Carroll A, Downs S, Cain MP, Frankel R, et al.. Community engagement of adolescents in the development of a patient-centered outcome tool for adolescents with a history of hypospadias repair. J Pediatr Urol 2019; 15 (5): 448.e1–448.e8. DOI: 10.1016/j.jpurol.2019.04.011.
  60. Ardelt PU, Cederqvist M, Barth M, Frankenschmidt A. The SIGHT questionnaire: A novel assessment tool for Satisfaction In Genital Hypospadias Treatment. J Pediatr Urol 2017; 13 (1): 33.e1–33.e8. DOI: 10.1016/j.jpurol.2016.10.011.
  61. Jones BC, O’Brien M, Chase J, Southwell BR, Hutson JM. Early Hypospadias Surgery May Lead to a Better Long-Term Psychosexual Outcome. J Urol 2009; 182 (4s): 1744–1750. DOI: 10.1016/j.juro.2009.02.089.
  62. Kanematsu A, Tanaka S, Hashimoto T, Nojima M, Yamamoto S. Analysis of the association between paternity and reoperation for urethral obstruction in adult hypospadias patients who underwent two-stage repair in childhood. BMC Urol 2019; 19 (1): 88, DOI: 10.1186/s12894-019-0512-2.
  63. Vandendriessche S, Baeyens D, Van Hoecke E, Indekeu A, Hoebeke P. Body image and sexuality in adolescents after hypospadias surgery. J Pediatr Urol 2010; 6 (1): 54–59. DOI: 10.1016/j.jpurol.2009.04.009.
  64. Minnillo BJ, Barbosa JABA, Carvas FA, Penna FJ, Huang L, Ziniel S, et al.. Development and Initial Validation of a Disease Specific Quality of Life Survey for Children With Vesicoureteral Reflux. J Urol 2012; 187 (5): 1828–1833. DOI: 10.1016/j.juro.2011.12.108.
  65. Goldstein SL, Graham N, Warady BA, Seikaly M, McDonald R, Burwinkle TM, et al.. Measuring Health-Related Quality of Life in Children With ESRD: Performance of the Generic and ESRD-Specific Instrument of the Pediatric Quality of Life Inventory (PedsQL). Am J Kidney Dis 2008; 51 (2): 285–297. DOI: 10.1053/j.ajkd.2007.09.021.
  66. Anthony SJ, Hebert D, Todd L, Korus M, Langlois V, Pool R, et al.. Child and parental perspectives of multidimensional quality of life outcomes after kidney transplantation. Pediatr Transplant 2010; 14 (2): 249–256. DOI: 10.1111/j.1399-3046.2009.01214.x.
  67. Pai ALH, Tackett A, Ittenbach RF, Goebel J. Psychosocial Assessment Tool 2.0_General: Validity of a psychosocial risk screener in a pediatric kidney transplant sample. Pediatr Transplant 2012; 16 (1): 92–98. DOI: 10.1111/j.1399-3046.2011.01620.x.
  68. Szymanski KM, Misseri R, Whittam B, Raposo S-M, King SJ, Kaefer M, et al.. QUAlity of Life Assessment in Spina bifida for Adults (QUALAS-A): development and international validation of a novel health-related quality of life instrument. Qual Life Res 2015; 24 (10): 2355–2364. DOI: 10.1007/s11136-015-0988-5.
  69. Szymanski KM, Misseri R, Whittam B, Yang DY, Raposo S-M, King SJ, et al.. Quality of Life Assessment in Spina Bifida for Children (QUALAS-C): Development and Validation of a Novel Health-related Quality of Life Instrument. Urology 2016; 87: 178–184. DOI: 10.1016/j.urology.2015.09.027.
  70. Olesen JD, Kiddoo DA, Metcalfe P. The association between urinary continence and quality of life in paediatric patients with spina bifida and tethered cord. Paediatr Child Health 2013; 18: 32, DOI: 10.1093/pch/18.7.e32.
  71. Baka-Ostrowska M, Bolong DT, Persu C, Tøndel C, Steup A, Lademacher C, et al.. Efficacy and safety of mirabegron in children and adolescents with neurogenic detrusor overactivity: An open-label, phase 3, dose-titration study. Neurourol Urodyn 2021; 40 (6): 1490–1499. DOI: 10.1002/nau.24657.
  72. Pompermaier J, Andrade MCde, Cruz MLda, Macedo Júnior A. Translation And Validation Of The Brazilian Version Of The “Quality Of Life Assessment In Spina Bifida” Questionnaire For Children And Teenagers. Rev Paul Pediatr 2021; 39: 2019312, DOI: 10.1590/1984-0462/2021/39/2019312.
  73. Kawahara T, Sugita Y, Momose H, Szymanski KM, Hida E, Yamazaki A. Development and validation of the Japanese version of the Quality of Life Assessment of Spina Bifida in Teenagers ( QUALAS -T-J). Pediatr Int 2019; 61 (12): 1232–1238. DOI: 10.1111/ped.14003.
  74. Kawahara T, Sugita Y, Momose H, Szymanski KM, Hida E, Yamazaki A. Development and Validation of the Japanese Version of Quality of Life Assessment of Spina Bifida in Children (QUALAS-C-J). Pediatr Int 2021; 63 (4): 423–429. DOI: 10.1111/ped.14416.
  75. Kreukels BPC, Köhler B, Nordenström A, Roehle R, Thyen U, Bouvattier C, et al.. Gender Dysphoria and Gender Change in Disorders of Sex Development/Intersex Conditions: Results From the dsd-LIFE Study. J Sex Med 2018; 15 (5): 777–785. DOI: 10.1016/j.jsxm.2018.02.021.
  76. Rapp M, Duranteau L, Grift TC. Response to commentary re ‘Self- and proxy-reported outcomes after surgery in people with disorders/differences of sex development (DSD) in Europe (dsd-LIFE).’ J Pediatr Urol 2021; 17 (3): 368–369. DOI: 10.1016/j.jpurol.2021.02.025.
  77. Brinkmann L, Schuetzmann K, Richter-Appelt H. ORIGINAL RESEARCH–INTERSEX AND GENDER IDENTITY DISORDERS: Gender Assignment and Medical History of Individuals with Different Forms of Intersexuality: Evaluation of Medical Records and the Patients’ Perspective. J Sex Med 2007; 4 (4): 964–980. DOI: 10.1111/j.1743-6109.2007.00524.x.
  78. E B, S B, P L, TC van de G, A N, M R, et al.. Early Genital Surgery in Disorders/Differences of Sex Development: Patients’ Perspectives. Yearbook of Paediatric Endocrinology 2021; 50: 913, DOI: 10.1530/ey.18.6.9.
  79. Szymanski KM, Whittam B, Kaefer M, Frady H, Cain MP, Rink RC. What about my daughter’s future? Parental concerns when considering female genital restoration surgery in girls with congenital adrenal hyperplasia. J Pediatr Urol 2018; 14 (5): 417.e1–417.e5. DOI: 10.1016/j.jpurol.2018.07.010.
  80. WISNIEWSKI AMYB, MIGEON CLAUDEJ, MALOUF MATTHEWA, GEARHART JOHNP. Psychosexual Outcome In Women Affected By Congenital Adrenal Hyperplasia Due To 21-hydroxylase Deficiency. J Urol 2004; 171 (6 Part 1): 2497–2501. DOI: 10.1097/01.ju.0000125269.91938.f7.
  81. Krege S, Walz KH, Hauffa BP, Körner I, Rübben H. Long-term follow-up of female patients with congenital adrenal hyperplasia from 21-hydroxylase deficiency, with special emphasis on the results of vaginoplasty. BJU Int 2000; 86 (3): 253–258. DOI: 10.1046/j.1464-410x.2000.00789.x.
  82. Schönbucher V, Schweizer K, Rustige L, Schützmann K, Brunner F, Richter-Appelt H. Sexual Quality of Life of Individuals with 46,XY Disorders of Sex Development. J Sex Med 2012; 9 (12): 3154–3170. DOI: 10.1111/j.1743-6109.2009.01639.x.
  83. Köhler B, Kleinemeier E, Lux A, Hiort O, Grüters A, Thyen U. Satisfaction With Genital Surgery and Sexual Life of Adults With XY Disorders of Sex Development. Obstet Gynecol Surv 2012; 67 (6): 339–341. DOI: 10.1097/ogx.0b013e318259bef1.
  84. Schober JM. Sexual Behaviors, Sexual Orientation And Gender Identity In Adult Intersexuals: A Pilot Study. J Urol 2001; 165: 2350–2353. DOI: 10.1097/00005392-200106001-00033.
  85. Friseń L, Nordenström A, Falhammar H, Filipsson H, Holmdahl G, Janson PO, et al.. Gender Role Behavior, Sexuality, and Psychosocial Adaptation in Women with Congenital Adrenal Hyperplasia due toCYP21A2Deficiency. J Clin Endocrinol Metab 2009; 94 (9): 3432–3439. DOI: 10.1210/jc.2009-0636.
  86. Kanhere M, Fuqua J, Rink R, Houk C, Mauger D, Lee PA. Psychosexual development and quality of life outcomes in females with congenital adrenal hyperplasia. Int J Pediatr Endocrinol 2015; 2015 (1): 21, DOI: 10.1186/s13633-015-0017-z.
  87. Nordenstrom A, Frisen L, Falhammar H. Sexual Function and Surgical Outcome in Women with Congenital Adrenal Hyperplasia Due to CYP21A2 Deficiency: Clinical Perspective and the Patients’ Perception. Yearbook of Urology 2010; 2011: 227–228. DOI: 10.1016/j.yuro.2010.12.017.
  88. D’Alberton F, Assante MT, Foresti M, Balsamo A, Bertelloni S, Dati E, et al.. Quality of Life and Psychological Adjustment of Women Living with 46,XY Differences of Sex Development. J Sex Med 2015; 12 (6): 1440–1449. DOI: 10.1111/jsm.12884.
  89. Hou L, Zhao M, Fan L, Cao B, Chen J, Cui Y, et al.. Correction to: One hundred twelve cases of 46, XY DSD patients after initial gender assignment: a short-term survey of gender role and gender dysphoria. Orphanet J Rare Dis 2021; 17 (1): 416, DOI: 10.1186/s13023-022-02296-8.
  90. Johannsen TH, Ripa CPL, Mortensen EL, Main KM. Quality of life in 70 women with disorders of sex development. Eur J Endocrinol 2006; 155 (6): 877–885. DOI: 10.1530/eje.1.02294.
  91. Şentürk Pilan B, Özbaran B, Çelik D, Özcan T, Özen S, Gökşen D, et al.. Quality of Life and Psychological Well-being in Children and Adolescents with Disorders of Sex Development. J Clin Res Pediatr Endocrinol 2021; 13 (1): 23–33. DOI: 10.4274/jcrpe.galenos.2020.2020.0141.
  92. Cassia Amaral R, Inacio M, Brito VN, Bachega TASS, Oliveira AA, Domenice S, et al.. Quality of life in a large cohort of adult Brazilian patients with 46,XX and 46,XY disorders of sex development from a single tertiary centre. Clin Endocrinol (Oxf) 2015; 82 (2): 274–279. DOI: 10.1111/cen.12572.
  93. Baayen C, Feuillet F, Clermidi P, Crétolle C, Sarnacki S, Podevin G, et al.. Validation of the French versions of the Hirschsprung’s disease and Anorectal malformations Quality of Life (HAQL) questionnaires for adolescents and adults. Health Qual Life Outcomes 2017; 15 (1): 24, DOI: 10.1186/s12955-017-0599-7.
  94. Hanneman MJG, Sprangers MAG, De Mik EL, Heurn ELW van, De Langen ZJ, Looyaard N, et al.. Quality of life in patients with anorectal malformation or hirschsprung’s disease. Dis Colon Rectum 2001; 44 (11): 1650–1660. DOI: 10.1007/bf02234386.
  95. Wigander H, Nisell M, Frenckner B, Wester T, Brodin U, Öjmyr-Joelsson M. Quality of life and functional outcome in Swedish children with low anorectal malformations: a follow-up study. Pediatr Surg Int 2019; 35 (5): 583–590. DOI: 10.1007/s00383-018-04431-8.
  96. Hassink EAM, Rieu PNMA, Brugman ATM, Festen C. Quality of life after operatively corrected high anorectal malformation: A long-term follow-up study of patients aged 18 years and older. J Pediatr Surg 1994; 29 (6): 773–776. DOI: 10.1016/0022-3468(94)90367-0.
  97. Poley MJ, Stolk EA, Tibboel D. Short term and long term health related quality of life after congenital anorectal malformations and congenital diaphragmatic hernia. Arch Dis Child 2004; 89 (9): 836–841. DOI: 10.1136/adc.2002.016543.
  98. Amae S, Hayashi J, Funakosi S, Kamiyama T, Yoshida S, Ueno T, et al.. Postoperative psychological status of children with anorectal malformations. Pediatr Surg Int 2008; 24 (3): 293–298. DOI: 10.1007/s00383-007-2077-5.
  99. Goyal A, Williams JM, Kenny SE, Lwin R, Baillie CT, Lamont GL, et al.. Functional outcome and quality of life in anorectal malformations. J Pediatr Surg 2006; 41 (2): 318–322. DOI: 10.1016/j.jpedsurg.2005.11.006.
  100. Ludman L, Spitz L. Psychosocial adjustment of children treated for anorectal anomalies. J Pediatr Surg 1995; 30 (3): 495–499. DOI: 10.1016/0022-3468(95)90065-9.
  101. Ludman L, Spitz L, Kiely EM. Social and emotional impact of faecal incontinence after surgery for anorectal abnormalities. Arch Dis Child 1994; 71 (3): 194–200. DOI: 10.1136/adc.71.3.194.
  102. Versteegh HP, Hondel D van den, IJsselstijn H, Wijnen RMH, Sloots CEJ, Blaauw I de. Cloacal malformation patients report similar quality of life as female patients with less complex anorectal malformations. J Pediatr Surg 2016; 51 (3): 435–439. DOI: 10.1016/j.jpedsurg.2015.07.020.
  103. Funakosi S, Hayashi J, Kamiyama T, Ueno T, Ishii T, Wada M, et al.. Psychosocial liaison-consultation for the children who have undergone repair of imperforate anus and Hirschsprung disease. J Pediatr Surg 2005; 40 (7): 1156–1162. DOI: 10.1016/j.jpedsurg.2005.03.059.
  104. Hartman EE, Oort FJ, Aronson DC, Sprangers MA. Quality of life and disease-specific functioning of patients with anorectal malformations or Hirschsprung’s disease: a review. Arch Dis Child 2011; 96 (4): 398–406. DOI: 10.1136/adc.2007.118133.
  105. Aasland A, Diseth TH. Can the Harter Self-Perception Profile for Adolescents (SPPA) be used as an indicator of psychosocial outcome in adolescents with chronic physical disorders? Eur Child Adolesc Psychiatry 1999; 8 (2): 78–85. DOI: 10.1007/s007870050088.
  106. Elstad EA, Taubenberger SP, Botelho EM, Tennstedt SL. Beyond incontinence: the stigma of other urinary symptoms. J Adv Nurs 2010; 66 (11): 2460–2470. DOI: 10.1111/j.1365-2648.2010.05422.x.
  107. Catti M, Paccalin C, Rudigoz R-C, Mouriquand P. Quality of life for adult women born with bladder and cloacal exstrophy: A long-term follow up. J Pediatr Urol 2006; 2 (1): 16–22. DOI: 10.1016/j.jpurol.2005.07.002.
  108. LEE CELINE, REUTTER HEIKOM, GRAssER MELANIEF, FISCH MARGIT, NOEKER MEINOLF. Gender-associated differences in the psychosocial and developmental outcome in patients affected with the bladder exstrophy-epispadias complex. BJU Int 2006; 97 (2): 349–353. DOI: 10.1111/j.1464-410x.2005.05910.x.
  109. EBERT A, SCHEUERING S, SCHOTT G, ROESCH WH. Psychosocial And Psychosexual Development In Childhood And Adolescence Within The Exstrophy-epispadias Complex. J Urol 2005; 174 (3): 1094–1098. DOI: 10.1097/01.ju.0000169171.97538.ed.
  110. Taskinen S, Suominen JS, Mattila AK. Health-related Quality of Life and Mental Health in Adolescents and Adults Operated for Bladder Exstrophy and Epispadias. Urology 2015; 85 (6): 1515–1519. DOI: 10.1016/j.urology.2015.02.020.
  111. Dellenmark-Blom M, Sjöström S, Abrahamsson K, Holmdahl G. Health-related quality of life among children, adolescents, and adults with bladder exstrophy–epispadias complex: a systematic review of the literature and recommendations for future research. Qual Life Res 2019; 28 (6): 1389–1412. DOI: 10.1007/s11136-019-02119-7.
  112. Wigander H, Frenckner B, Wester T, Nisell M, Öjmyr-Joelsson M. Translation and cultural adaptation of the Hirschsprung’s Disease/Anorectal Malformation Quality of life Questionnaire (HAQL) into Swedish. Pediatr Surg Int 2014; 30 (4): 401–406. DOI: 10.1007/s00383-014-3478-x.
  113. Izard JP. The emerging role of patient reported outcomes in urology. Can Urol Assoc J 2017; 11 (12): 411. DOI: 10.5489/cuaj.5007.
  114. Sisodia RC, Rodriguez JA, Sequist TD. Digital disparities: lessons learned from a patient reported outcomes program during the COVID-19 pandemic. J Am Med Inform Assoc 2021; 28 (10): 2265–2268. DOI: 10.1093/jamia/ocab138.
  115. Tasian GE, Ellison JS. Measuring patient-centered outcomes: The need to move beyond quality of life. J Pediatr Urol 2021; 17 (4): 444. DOI: 10.1016/j.jpurol.2021.03.033.

Last updated: 2023-02-22 09:32