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Abstract
Introduction Existing patient-reported outcome measures (PROMs) do not meet accepted international criteria for measuring health outcomes of hypospadias treatment. This protocol describes the qualitative development (phase I) of a novel PROM to evaluate outcomes of hypospadias treatments.
Methods and analysis Participants aged 7 years and older with hypospadias and caregivers of children under 8 years seeking treatment at Boston Children’s Hospital, Children’s Hospital of Eastern Ontario (CHEO), Children’s Hospital of Philadelphia (CHOP) and McMaster Children’s Hospital), will be invited to participate in concept elicitation and cognitive interviews. Concept elicitation interviews will be in-depth and semi-structured to understand concepts important to patients seeking treatment for hypospadias. Cognitive interviews will be performed concurrently to ensure that the scale items, instructions and response options are relevant, understandable and comprehensive. Cognitive interviews will be complemented by expert input. Concept elicitation and cognitive interview transcripts will be coded line-by-line. Participant quotes will be categorised into top-level domains, themes and subthemes. The primary outcome of this research will be to develop a conceptual model representing the patient experience of hypospadias and a novel PROM.
Ethics and dissemination Ethics approval was obtained from Boston Children’s Hospital’s Institutional Review Board (HHS Registration: IRB00000352; Protocol number IRB-P00042425). CHOP, McMaster and CHEO have reliance agreements with Boston Children’s Hospital. Findings from this research will be disseminated at national and international conferences and published in relevant peer-reviewed journals for the target audience.
- UROLOGY
- Patient Reported Outcome Measures
- Paediatric urology
- Adolescents
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Strengths and limitations of the study
Recruitment of a heterogeneous sample of patients will ensure that concepts capture the patient’s authentic experience of hypospadias and will enable the development of a novel patient-reported outcome measure (PROM).
We adhere to published guidelines for establishing content validity and developing new PROMs.
This new PROM will offer a clinically meaningful PROM, developed using a modern psychometric approach called Rasch measurement theory analysis.
This protocol is limited to patients that are English speakers seeking care in a North American healthcare setting, therefore, lacking geographic and cultural diversity.
This study lacks the perspective of the middle-aged/senior adult hypospadias population and lacks patients with moderate to severe uncorrected hypospadias, as most are fixed in infancy.
Introduction
Patient-reported outcome measures (PROMs) are valid and reliable tools developed to enable direct reports of a patient’s health status from their perspective.1 PROMs are increasingly used to inform care delivery, comparative effectiveness research and health policy decisions.2 Little is known about the most critical outcomes for young patients; therefore, including the patient (or proxy) perspective in paediatric surgical decision-making is critically important.3
Hypospadias, a condition where the urethra opens on the underside or below the penis, which is associated with varying degrees of penile curvature and foreskin anomalies, is the second most common congenital disability in boys, occurring in approximately one in every 200 male births in the USA4 and a global prevalence of 20.9 per 10 000 births.5 Since boys cannot outgrow this defect, surgical repair is often undertaken during infancy to improve urinary and sexual function and overall quality of life (QOL). Surgical success has been measured primarily by surgeon assessments of urine flow rates or complications.6 However, there is a need to incorporate the patient’s perspective of health outcomes following hypospadias treatment.
Surgical outcomes for correcting hypospadias vary widely and may impose physical and psychosocial consequences on patients and their families. Recent advances in surgical technique have shown improved outcomes in hypospadias; however, eight percent of boys still experience complications;7 this risk doubles to quadruples in boys with severe defects (ie, proximal) or those with prior complications. Poor cosmetic or functional outcomes can lead to considerable long-term cognitive, behavioural and self-esteem consequences, including poor school performance,8 sexual avoidance9 10 and negative genital perception.11 12
Presently, no PROM exists that meets the accepted international criteria for measuring health outcomes of hypospadias treatment.13 14 A systematic literature review revealed a need for validated PROMs for patients with hypospadias;15 PROMs were primarily ad hoc, with limited evidence of their development and validation. Existing PROMs do not address self-perception, embarrassment, satisfaction with voiding/sexual function or QOL; therefore, they lack content validity (ie, they do not capture the full range of concerns experienced by patients and their families).
The lack of disease-specific and well-validated patient-reported endpoints has hampered the evaluation of surgical techniques or non-operative management of hypospadias. There is wide variation in the surgical technique, including non-intervention, with an unknown impact on the patient’s QOL. Furthermore, commonly reported outcomes, such as surgical complications or urine flow rates, have not demonstrated benefits in patients’ health status after treatment.
This research programme aims to develop and validate a novel PROM for patients with hypospadias. This protocol describes the qualitative development (phase I) of a new PROM to evaluate outcomes of hypospadias treatments.
Methods and analysis
Phase 1: qualitative development
Best practice guidelines of the USA Food and Drug Administration12 and the Consensus-based standards for the selection of health status measurement instruments13 are being employed to inform the development of this new PROM. Interpretive description, an applied health services research approach, will be used. A systematic literature review was conducted to inform the development of a preliminary conceptual model.15 Subsequent qualitative interviews with patients aged over 7 years, with a focus on children, teens, young adults with prior paediatric hypospadias care and their proxies were undertaken to inform the conceptual model further and develop initial items for scale development.
Additional in-depth and semi-structured concept elicitation interviews will be conducted (May 2024–March 2025, including a more heterogeneous sample of patients aged 7 years or older to better understand concepts important to patients seeking treatment for hypospadias. Qualitative data will be used to develop the conceptual model and content further. Cognitive interviews will be performed concurrently to ensure that the scale items, instructions and response options are relevant, understandable and comprehensive. Cognitive interviews will inform the final scales which will be circulated to an international group of experts in hypospadias for feedback (estimated July 2025). Concepts elicited and changes proposed by expert informants will be reviewed with patient participants for final scale creation.
Setting
Participants were recruited from the urology clinics at the Children’s Hospital of Eastern Ontario (CHEO, Canada) and Boston Children’s Hospital (BCH, USA) for the completed concept elicitation interviews. All participants participating in concept elicitation and cognitive interviews provided informed consent or assent (for participants <16: guardian consent, participant assent) as approved by local institutional review boards or research ethics boards. For the subsequent concept elicitation and cognitive interviews, interested participants will be recruited from the urology clinics at BCH, USA and Children’s Hospital of Philadelphia (CHOP, USA).
Sample
Participants from the completed concept elicitation interviews were identified through a retrospective search of surgical billing codes within the electronic health record system and new patient referrals to the urology clinic for hypospadias. New patient referrals and existing patients of the urology clinics for hypospadias will be approached to participate in the subsequent concept elicitation and cognitive interviews.
A maximum variation sample will be purposively selected to vary by disease severity (mild to severe disease), stage of treatment (preoperative and postoperative), age of treatment, clinical outcomes, urethral location and the presence or absence of complications. Definitions of complications are based on a consensus statement from the International Paediatric Urology Task Force on Hypospadias.16
Recruitment
A care team member recruited participants from the completed concept elicitation interviews, and those who consented to participate were invited to complete an in-person or virtual interview. Subsequent concept elicitation and cognitive interview recruitment will involve care team members asking patients if they would be interested in participating in the study. Patients will provide written or verbal consent (patients under 16 will assent and their guardians will consent). Patients or parents who consented to participate were invited to an interview on an online platform (eg, Microsoft Teams or Zoom) telephone interview with an experienced qualitative researcher.
Experts in the field of hypospadias known to our network through the Hypospadias International Society (n=20) will be invited via email invitation to provide feedback on the scales, and feedback will be captured through a Research electronic data capture (REDCap) survey. This step will ensure that the content of the proposed scales comprehensively measured outcomes that matter to patients and that instructions, response options and items are clear, meaningful and unambiguous.
Data collection
Subsequent concept elicitation and cognitive interviews will follow the same data collection procedures used previously for the completed concept elicitation interviews. All interviews will be audio recorded, transcribed and coded. Participants will be interviewed using a semi-structured qualitative interview guide, including surgical experience, appearance, function and psychological well-being probes (online supplemental appendix 1). Questions regarding perceptions of ideal hypospadia outcomes will be probed. Topics included in the interview guide will be informed by a systematic literature review of PROMs used in hypospadias. The interview guide will be adapted accordingly to each participant to avoid asking irrelevant questions (eg, asking young participants about sexual well-being). The interview guide will be adjusted as interviews are conducted to include any new concepts identified after each interview. Recruitment will continue until saturation (ie, no new concepts are elicited from the interviews).17
Supplemental material
Multiple rounds of cognitive interviews will be performed with as many participants as necessary to establish the content validity of the scales. Participants will review the scales via an online REDCap survey17 and will be encouraged to use the ‘think aloud’ approach with probing to obtain feedback on the relevance, comprehensibility and comprehensiveness of the scale items, instructions, and response options.18–21
Experts in hypospadias will review the scale content before the final round of patient cognitive interviews. A REDCap survey will be designed with targeted questions about the items’ relevance, instructions, response options and any missing content. Relevant changes suggested in each round of patient interviews and from expert input will be applied and reviewed in a final round of participant interviews.
Data analysis
Subsequent concept elicitation and cognitive interviews will follow the same data analysis procedures used previously for the completed concept elicitation interviews. Data collection and analysis will be conducted concurrently. Concept elicitation and cognitive interview transcripts will be coded line-by-line by a research team member and confirmed by a second member for the first five interviews or until an agreed-upon coding framework is achieved. Coded data will be transferred onto an Excel spreadsheet for analysis.
Participant quotes from the concept elicitation interviews will be categorised into top-level domains and themes/subthemes. Qualitative analysis will refine a conceptual framework covering the concepts concerning people seeking treatment for hypospadias. The coding framework will guide scale development and codes will inform the development of a comprehensive item pool. Items will be devised such that they retain participant language to ensure that they resonate with patients and are easy to understand. A comprehensive set of independently functioning scales will be formed from the item pool, alongside instructions, a time frame for responding to the scales, and a set of response options. Feedback obtained from the cognitive interviews will be categorised according to the following top-level domains: interpretation, relevance, clarity, missing content and suggested changes.
Subsequent phases
The phase II field-test study will involve the recruitment of a large international sample of participants. We will use the data collected to refine and validate the Hypospadias PRO scales using RMT (Rasch Measurement Theory analysis) analysis. This will allow us to identify the items in each scale that can serve as more accurate indicators of patient outcomes, thereby reducing the length of the scales for patient ease of completion and creating clinically meaningful and valid scales. Once developed and validated, we will conduct further psychometric research to determine each scale’s ability to measure clinical change following hypospadia repair.
Patient and public involvement
Our patient-oriented approach engages patients with hypospadias parents of children and teenagers with hypospadias and healthcare providers in all stages of our research as experts and research team members whose input is crucial to the design of the study and development of content for our hypospadias scales. Participants involved in qualitative and cognitive interviews will be invited to continue to collaborate in our study by taking part in scale refinement interviews where they can provide feedback on our findings and help refine the final set of scales. We are collaborating with patients and families to host a patient-centred hypospadias conference in the Summer of 2026, where study results will be presented for discussion and further dissemination in patient-centred research.
Ethics and dissemination
Ethics approval was obtained from Boston Children’s Hospital’s Institutional Review Board (HHS Registration: IRB00000352; Protocol number IRB-P00042425). CHOP, McMaster and CHEO have reliance agreements with BCH. Participants will provide written and audio-recorded verbal consent (for participants <16: guardian consent, participant assent). Participants will be de-identified from their transcripts, and institution rules for data storage will be followed.
Once developed, this new PROM will be made available to licence free for all non-profit users. We will seek to advertise and promote its use through presentations at national and international meetings. Findings about the development will also be published in relevant peer-reviewed journals for the target audience.
Ethics statements
Patient consent for publication
References
Footnotes
X @anneklassen
Contributors MK initiated the study at BCH and CHOP, designed the qualitative interview guide, conducted qualitative interviews, coded interview transcripts, drafted and revised the paper. ET conducted qualitative and cognitive interviews, coded interview transcripts and drafted and revised the manuscript. SG designed interview guide, conducted qualitative and cognitive interviews and coded interview transcripts. CL implemented the study at CHOP, participated in research planning meetings and revised the manuscript. EM and LHB participated in research planning meetings and revised the manuscript. AK provided mentorship in designing the interview guide, provided mentorship in coding interview transcripts, scale design and revised the manuscript. MK is the guarantor.
Funding This work was supported by the Canadian Urological Association Research Foundation Award.
Competing interests None declared.
Patient and public involvement Patients and/or the public were involved in the design, or conduct, or reporting, or dissemination plans of this research. Refer to the Methods section for further details.
Provenance and peer review Not commissioned; externally peer reviewed.
Supplemental material This content has been supplied by the author(s). It has not been vetted by BMJ Publishing Group Limited (BMJ) and may not have been peer-reviewed. Any opinions or recommendations discussed are solely those of the author(s) and are not endorsed by BMJ. BMJ disclaims all liability and responsibility arising from any reliance placed on the content. Where the content includes any translated material, BMJ does not warrant the accuracy and reliability of the translations (including but not limited to local regulations, clinical guidelines, terminology, drug names and drug dosages), and is not responsible for any error and/or omissions arising from translation and adaptation or otherwise.