Karen W. Y. Wong Riff

International multiphase mixed methods study protocol to develop a cross-cultural patient-reported outcome instrument for children and young adults with cleft lip and/or palate (CLEFT-Q)

Introduction Patient-reported outcome (PRO) instruments should be developed according to rigorous guidelines in order to provide clinically meaningful, scientifically sound measurement. Understanding the methodology behind instrument development informs the selection of the most appropriate tool. This mixed methods protocol describes the development of an internationally applicable PRO instrument, the CLEFT-Q, for evaluating outcomes of treatment for cleft lip and/or palate (CL/P). Methods and analysis The study includes three main phases that occur iteratively and interactively. In phase I, we determine what concepts are important to patients regarding their outcome. A conceptual framework for the CLEFT-Q is formed through a systematic review and an extensive international qualitative study. The systematic review ascertains what concepts have previously been measured in patients with CL/P. The qualitative study employs interpretive description and involves in-depth interviews with patients in high-income and lower-middle income countries. Preliminary items are generated from the qualitative data. Preliminary scales are then created for each theme in the framework. Cognitive debriefing interviews and expert clinician input are used to refine the scales in an iterative process. In phase II, the preliminary scales are administered to a large international group of patients with CL/P. The modern psychometric method of Rasch Measurement Theory analysis is employed to define the measurement characteristics. The preliminary scales are shortened based on these results. In phase III, further tests assess reliability, validity and responsiveness of the instrument. Ethics and dissemination The study is approved by Research Ethics Boards for each participating site. Findings from this study will be published in open access peer-reviewed journals and presented at national and international conferences. Integrated knowledge translation is employed to engage stakeholders from the outset of the study. Successful execution of the CLEFT-Q will result in an internationally applicable PRO instrument for children and young adults with CL/P.

Infant Robotic Cleft Palate Surgery: A Feasibility Assessment Using a Realistic Cleft Palate Simulator

Background: A surgical robot offers enhanced precision, visualization, and access and the potential to improve outcomes in cleft palate surgery. The goal of this study was to investigate the feasibility of using the da Vinci robot for cleft palate repair in infants using a cleft palate simulator test bed. Methods: A high-fidelity cleft palate simulator was developed that allows performance of a robotic cleft palate repair procedure. A complete cleft palate repair was performed with the da Vinci Si with 5-mm instruments and the da Vinci Xi with 8-mm instruments. The advantages of the robotic approach were assessed in comparison with using standard instruments. For each system, arm repositioning, collisions, instrument and endoscope excursion, wrist orientation, and vision were compared for 12 steps of the repair. Results: The cleft palate simulator provided a reproducible platform for testing robotic cleft palate surgery. The advantages of the robotic approach were the ability to articulate a miniature wrist intraorally with superior visualization, increased ambidexterity, and improved ergonomics compared with using standard instruments. Cleft palate repair with the Xi was superior to the Si with respect to arm repositioning, instrument collisions and excursion, and wrist orientation. However, Xi performance remained suboptimal because of the larger instruments. Conclusions: Robotic cleft palate repair using the da Vinci system offers advantages compared with the traditional approach. Cleft palate repair is more feasible with the Xi and 8-mm instruments. However, performance is limited by the instrumentation, which requires modification to ensure safety and efficacy. CLINICAL QUESTION/LEVEL OF EVIDENCE: Therapeutic, V.

Establishing content validity of the CLEFT-Q: A new patient-reported outcome instrument for CLEFT lip/palate

Background: The CLEFT-Q is a new patient-reported outcome instrument designed to measure outcomes that matter to patients. The aim of this qualitative study was to establish content validity of the CLEFT-Q in patients who differ by age and culture. Methods: Patients aged between 6 and 29 years were recruited from plastic surgery clinics in Canada, India, Ireland, the Philippines, the Netherlands and the United States. Healthcare providers and other experts participated in a focus group or provided individual feedback. Input was sought on all aspects of the CLEFT-Q (item wording, instructions, and response options), and to identify missing content. Patient interviews and expert feedback took place between September 2013 and September 2014. Results: Sixty-nine patients and 44 experts participated. The first draft of the CLEFT-Q consisted of 163 items measuring 12 constructs. The first round of feedback identified 92 items that required revision. In total, 3 rounds of interviews, and the involvement of an artist to create pictures for 17 items, were needed to establish content validity. At the conclusion of cognitive interviews, the CLEFT-Q consisted of 13 scales (total 171 items) that measure appearance, health-related quality of life, and facial function. The mean Flesch-Kincaid readability statistic for items was 1.4 (0 to 5.2). Conclusion: Cognitive interviews and expert review allowed us to identify items that required re-wording, re-conceptualizing, or to be removed, as well as any missing items. This process was useful for refining the CLEFT-Q scales for further testing.

Translation and cultural adaptation of the CLEFT-Q for use in Colombia, Chile, and Spain

BackgroundCleft lip and/or palate (CL/P) is a common congenital craniofacial anomaly that may negatively affect an individual’s appearance, health-related quality of life, or speech. In Spain, Colombia, and Chile the overall prevalence of CL/P ranges from 0.53 to 1.59 cases per 1000 live births. Currently, there is no patient-reported outcome (PRO) instrument that is specific for patients with CL/P. The CLEFT-Q is a new PRO instrument developed to measure outcomes of treatment in patients 8 to 29xa0years of age with CL/P. The aim of this study was to translate and culturally adapt the CLEFT-Q for use in Colombia, Chile, and Spain.MethodsThe CLEFT-Q was translated from English to 3 Spanish language varieties (Colombian, Chilean, and Spanish (Spain)) and Catalan. Translation and cultural adaptation guidelines set forth by the International Society for Pharmacoeconomics and Outcomes Research were followed.ResultsThe field- test version of the CLEFT-Q consisted of 13 scales (total 154 items) measuring appearance, health-related quality of life, and facial function. Forward translations revealed 10 (7%) items that were difficult to translate into Chilean, and back translations identified 34 (22%) and 21 (13%) items whose meaning differed from the English version in at least 1 of the 3 Spanish varieties and Catalan respectively. Twenty-one participants took part in cognitive debriefing interviews. Participants were recruited from plastic surgery centres in Bogotá, Colombia (nu2009=u20094), Santiago, Chile (nu2009=u20097), and Barcelona, Spain (nu2009=u200910). Most participants were males (nu2009=u200914, 67%) and were diagnosed with CL/P (nu2009=u200917, 81%). Participants reported difficulty understanding 1 item in the Colombian, 1 item in the Spanish (Spain), and 11 items from the Catalan version. Comparison of the 3 Spanish varieties revealed 61 (40%) of the 154 items whose wording differed across the 3 Spanish versions.ConclusionTranslation and cultural adaptation processes provided evidence of transferability of the CLEFT-Q scales into 3 Spanish varieties and Catalan, as semantic, idiomatic, experiential, and conceptual equivalence of the items, instructions, and response options were achieved.

What Matters to Patients With Cleft Lip and/or Palate: An International Qualitative Study Informing the Development of the CLEFT-Q

Objective: The goal of treatment for individuals with cleft lip and/or palate (CL/P) is to improve physical, psychological, and social health. Outcomes of treatment are rarely measured from the patient’s perspective. The aim of the study was to develop a conceptual framework for a patient-reported outcome (PRO) instrument for individuals with clefts (CLEFT-Q) by developing an in-depth understanding of issues that individuals consider to be important. Design: The qualitative methodology of interpretive description was used. Setting, Participants, and Intervention: We performed 136 individual in-depth interviews with participants with clefts of any age, presenting for cleft care, across 6 countries. Parents were involved if the child was more comfortable. Interviews were audio-recorded, transcribed verbatim, and coded using constant comparison. The data were used to develop a refined conceptual framework. Results: Participants described concepts of interest in 3 top-level domains, each of which included subdomains: appearance (face, nose, nostrils, teeth, lips, jaw, cleft lip scar), health-related quality of life (psychological, social, school, speech-related distress), and facial function (speech, eating/drinking). Participants were able to describe changes over time with regard to the 3 domains. Conclusions: A conceptual framework of concepts of interest to individuals with CL/P formed the basis of the scales in the CLEFT-Q. Each subdomain represents an independently functioning scale. Understanding what matters to patients is essential in guiding PRO measurement.

Content Validity of Patient-Reported Outcome Instruments Used With Pediatric Patients With Facial Differences: A Systematic Review.

Objective: The aim of this systematic review was to identify patient-reported outcome (PRO) instruments used in research with children/youth with conditions associated with facial differences to identify the health concepts measured. Design: MEDLINE, EMBASE, CINAHL, and PsycINFO were searched from 2004 to 2016 to identify PRO instruments used in acne vulgaris, birthmarks, burns, ear anomalies, facial asymmetries, and facial paralysis patients. We performed a content analysis whereby the items were coded to identify concepts and categorized as positive or negative content or phrasing. Results: A total of 7,835 articles were screened; 6 generic and 11 condition-specific PRO instruments were used in 96 publications. Condition-specific instruments were for acne (four), oral health (two), dermatology (one), facial asymmetries (two), microtia (one), and burns (one). The PRO instruments provided 554 items (295 generic; 259 condition specific) that were sorted into 4 domains, 11 subdomains, and 91 health concepts. The most common domain was psychological (n = 224 items). Of the identified items, 76% had negative content or phrasing (e.g., “Because of the way my face looks I wish I had never been born”). Given the small number of items measuring facial appearance (n = 19) and function (n = 22), the PRO instruments reviewed lacked content validity for patients whose condition impacted facial function and/or appearance. Conclusions: Treatments can change facial appearance and function. This review draws attention to a problem with content validity in existing PRO instruments. Our team is now developing a new PRO instrument called FACE-Q Kids to address this problem.

Psychometric findings and normative values for the CLEFT-Q based on 2434 children and young adult patients with cleft lip and/or palate from 12 countries

BACKGROUND: Patients with cleft lip and/or palate can undergo numerous procedures to improve appearance, speech, dentition and hearing. We developed a cleft-specific patient-reported outcome instrument to facilitate rigorous international measurement and benchmarking. METHODS: Data were collected from patients aged 8–29 years with cleft lip and/or palate at 30 hospitals in 12 countries between October 2014 and November 2016. Rasch measurement theory analysis was used to refine the scales and to examine reliability and validity. Normative CLEFT-Q values were computed for age, sex and cleft type. RESULTS: Analysis led to the refinement of an eating and drinking checklist and 12 scales measuring appearance (of the face, nose, nostrils, teeth, lips, jaws and cleft lip scar), health-related quality of life (psychological, social, school, speech distress) and speech function. All scales met the requirements of the Rasch model. Analysis to explore differential item functioning by age, sex and country provided evidence to support the use of a common scoring algorithm for each scale for international use. Lower (worse) scores on CLEFT-Q scales were associated with having a speech problem, being unhappy with facial appearance, and needing future cleft-related treatments, providing evidence of construct validity. Normative values for age, sex and cleft type showed poorer outcomes associated with older age, female sex and having a visible cleft. INTERPRETATION: The CLEFT-Q represents a rigorously developed instrument that can be used internationally to collect and compare evidence-based outcomes data from patients aged 8–29 years of age with cleft lip and/or palate.

Development of a New Module of the FACE-Q for Children and Young Adults with Diverse Conditions Associated with Visible and/or Functional Facial Differences

Abstract Appearance and facial function are concepts not well addressed in current pediatric patient‐reported outcome measures (PROM) for facial conditions. We aimed to develop a new module of the FACE‐Q for children/young adults with facial conditions that include ear anomalies, facial paralysis, skeletal conditions, and soft tissue conditions. Semi‐structured and cognitive interviews were conducted with patients aged 8‐29 years recruited from craniofacial centers in Canada, USA, UK, and Australia. Interviews were used to elicit new concepts and to obtain feedback on CLEFT‐Q scales hypothesized to be relevant to other facial conditions. Interview data were recorded, transcribed, and coded. Experts were emailed and invited to provide feedback via Research Electronic Data Capture (REDCap). Eighty‐four participants and 43 experts contributed. Analysis led to the development of a conceptual framework and 14 new scales that measure appearance, facial function, health‐related quality of life, and adverse effects of treatment. In addition, 12 CLEFT‐Q scales were determined to have content validity for use with other facial conditions. Expert input led to minor changes to scales and items. This new FACE‐Q module for children/young adults is being field‐tested internationally. Once finalized, we anticipate this PROM will be used to inform clinical practice and research studies.

Economy of Hand Motion During Cleft Palate Surgery Using a High-Fidelity Cleft Palate Simulator.

Objective: The objectives of this study were to assess economy of hand motion of residents, fellows, and staff surgeons using a high-fidelity cleft palate simulator to (1) stratify performance for the purpose of simulator validation and (2) to estimate the learning curve. Design: Two residents, 2 fellows, and 2 staff surgeons performed cleft palate surgery on a high-fidelity cleft palate simulator while their hand motion was tracked using an electromagnetic hand sensor. The time, number of hand movements, and path length of their hands were determined for 10 steps of the procedure. The magnitude of these metrics was compared among the 3 groups of participants and utilized to estimate the learning curve using curve-fitting analysis. Results: The residents required the most time, number of hand movements, and path length to complete the procedure. Although the number of hand movements was closely matched between the fellows and staff, the overall total path length was shorter for the staff. Inverse curves were fit to the data to represent the learning curve and 25 and 113 simulation sessions are required to reach within 5% and 1% of the expert level, respectively. Conclusion: The simulator successfully stratified performance using economy of hand motion. Path length is better matched to previous level of experience compared to time or number of hand movements.

Translation and cultural adaptation of the CLEFT-Q into Arabic, Dutch, Hindi, Swedish, and Turkish

BackgroundTreatment for cleft lip and/or palate (CL/P) involves a multidisciplinary team of experts who aim to improve ones’ appearance, health-related quality of life, and speech function. To appropriately measure outcomes in CL/P from the patient perspective, a CL/P-specific patient-reported outcome (PRO) instrument is needed. The CLEFT-Q is a self-report PRO instrument developed to evaluate treatment outcomes in patients with CL/P. The aim of this study was to translate and culturally adapt the CLEFT-Q.MethodsThe CLEFT-Q was translated and culturally adapted from English into Arabic, Dutch, Hindi, Swedish, and Turkish using guidelines set forth by the International Society for Pharmacoeconomics and Outcomes Research. For each language, two forward translations, one back translation, and cognitive debriefing interviews with patients were conducted.ResultsThe field test version of the CLEFT-Q consisted of 154 items across 13 scales. Forward translations for each language revealed few items that were difficult to translate into the various languages. Comparison of each back translation to the English version identified that a change in the meaning of an item was more common in the Turkish (nu2009=u200940, 26%) and Arabic (nu2009=u200917, 11%) translations, and required re-translation. Cognitive debriefing interviews involved 41 participants from plastic surgery centers in India, Qatar, Sweden, the Netherlands, and Turkey. Participants reported few difficulties in understanding the items, instructions, and response options in each CLEFT-Q translation.ConclusionsSemantic, idiomatic, experiential, and conceptual equivalence of the CLEFT-Q was achieved for all language versions, thus providing evidence of the CLEFT-Q’s transferability to other languages and cultures.Level of Evidence: Not ratable