Recursive partitioning vs computerized adaptive testing to reduce the burden of health assessments in cleft lip and/or palate : comparative simulation study

Background: Computerized adaptive testing (CAT) has been shown to deliver short, accurate, and personalized versions of the CLEFT-Q patient-reported outcome measure for children and young adults born with a cleft lip and/or palate. Decision trees may integrate clinician-reported data (eg, age, gender, cleft type, and planned treatments) to make these assessments even shorter and more accurate. Objective: We aimed to create decision tree models incorporating clinician-reported data into adaptive CLEFT-Q assessments and compare their accuracy to traditional CAT models. Methods: We used relevant clinician-reported data and patient-reported item responses from the CLEFT-Q field test to train and test decision tree models using recursive partitioning. We compared the prediction accuracy of decision trees to CAT assessments of similar length. Participant scores from the full-length questionnaire were used as ground truth. Accuracy was assessed through Pearson’s correlation coefficient of predicted and ground truth scores, mean absolute error, root mean squared error, and a two-tailed Wilcoxon signed-rank test comparing squared error. Results: Decision trees demonstrated poorer accuracy than CAT comparators and generally made data splits based on item responses rather than clinician-reported data. Conclusions: When predicting CLEFT-Q scores, individual item responses are generally more informative than clinician-reported data. Decision trees that make binary splits are at risk of underfitting polytomous patient-reported outcome measure data and demonstrated poorer performance than CATs in this study

Cleft lip and/or palate mortality trends in the USA: a retrospective population-based study

Background Cleft lip and/or palate (CL/P) is one of the most common congenital anomalies worldwide. Although CL/P management may require a series of interventions, mortality resulting from CL/P alone is rare. This study aims to examine recent trends of CL/P mortality rates in the USA.Methods A retrospective population-based study was conducted using official US birth and death certificate data from the Centers for Disease Control and Prevention from 2000 to 2019. Annual mortality rates per 1000 births with CL/P were calculated across sex and racial groups. Multivariable logistic regression models estimated the effects of sex and race on the risk of mortality with CL/P, and linear regression models were used to examine temporal changes in mortality rate across sex and race.Results From 2000 to 2019, 1119 deaths occurred in patients with documented CL/P, for an overall incidence of 20.3 deaths per 1000 births with CL/P (95% CI 18.9 to 22.8). Of these, Patau syndrome was the listed cause of death in 167 cases (14.9%). Black individuals (OR 1.93, 95% CI 1.85 to 2.01), Hispanic (1.54, 1.49 to 1.58) and American Indian individuals (1.28, 1.20 to 1.35) were at a greater risk of CL/P mortality compared with white individuals. Additionally, females were also at a greater risk (1.35, 1.21 to 1.49). A significant upward trend in CL/P mortality was observed in Hispanic (r2=0.70, p<0.01) and American Indian individuals (r2=0.81, p<0.01) from 2000 to 2019.Conclusions Cleft birth and mortality surveillance is essential in healthcare and prevention planning. Future studies are required to understand the differences in CL/P mortality rates across various sociodemographic groups

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 (n=40, 26%) and Arabic (n=17, 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 ratabl

CLEFT-Q: detecting differences in outcomes among 2434 patients with varying cleft types

Background: Measuring the patient perspective is important in evaluating outcomes of cleft care. Understanding how treatment outcomes vary depending on cleft type may allow for better planning of treatments, setting of expectations, and more accurate benchmarking efforts. The CLEFT-Q is a patientreported outcome measure for patients with cleft lip and/or palate. Methods: The 12 CLEFT-Q scales measuring appearance (i.e., face, nose, nostrils, lips, cleft lip scar, teeth, and jaws), function (i.e., speech), and health-related quality of life (i.e., psychological, school, social, and speech-related distress) were field tested in a cross-sectional study in 30 centers in 12 countries. Patients with cleft lip and/or cleft palate aged 8 to 29 years were recruited from clinical settings. Differences in CLEFT-Q scores by cleft subtypes were evaluated usingone-way analysis of variance or Kruskal-Wallis H tests, with Tukey or Dunn procedure with Bonferroni corrections post hoc analyses, respectively. Scores are presented using radar charts to visualize all outcomes simultaneously. Results: The field test included 2434 patients. Scores on all CLEFT-Q scales varied significantly with cleft subtype. Patients with unilateral or bilateral cleft lip and/or palate scored lower on all appearance scales compared with patients with cleft palate or unilateral incomplete cleft lip. Scores on the speech function and speech-related distress scales decreased with each progressive group in the Veau classification. Patients with complete bilateral cleft lip and palate scored lowest on the social, school, and psychological scales. Conclusions: Patient-reported outcomes measured with the CLEFT-Q vary significantly with cleft type. Visualizing multiple outcomes simultaneously with radar charts allows for an understanding of a patient’s overall status in a single graph

Psychometric Validation of the FACE-Q Craniofacial Module for Facial Nerve Paralysis

Background: Systematic reviews have identified the need for a patient-reported outcome measure for facial nerve paralysis (FNP). The aim of this study was to determine the psychometric properties of FACE-Q Craniofacial module scales when used in a combined sample of children and older adults with FNP. Methods: Data were collected between December 2016 and December 2019. We conducted qualitative interviews with children and adults with FNP. FACE-Q data were collected from patients aged 8 years and older with FNP. Rasch measurement theory analysis was used to examine the reliability and validity of the relevant scales in the FNP sample. Results: Twenty-five patients provided 2052 qualitative codes related to appearance, physical, psychological, and social function. Many patient concerns were common across age. The field-test sample included 235 patients aged 8-81 years. Of the 13 scales examined, all 122 items had ordered thresholds and good item fit to the Rasch model. For 12 scales, person separation index values were ≥0.79 and Cronbach's alpha values were ≥0.82. The 13th scale's reliability values were ≥0.71. Conclusion: The FACE-Q Craniofacial module scales described in this study can be used to collect and compare evidence-based outcome data from children and adults with FNP

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 Patient-reported Outcome Measure for Ear Conditions: The EAR-Q

Background:. Patient-reported outcome measures are widely used to improve health services and patient outcomes. The aim of our study was to describe the development of 2 ear-specific scales designed to measure outcomes important to children and young adults with ear conditions, such as microtia and prominent ears. Methods:. We used an interpretive description qualitative approach. Semi-structured qualitative and cognitive interviews were performed with participants with any type of ear condition recruited from plastic surgery clinics in Canada, Australia, United States, and United Kingdom. Participants were interviewed to elicit new concepts. Interviews were audio-recorded, transcribed, and coded using the constant comparison approach. Experts in ear reconstruction were invited to provide input via an online Research Electronic Data Capture survey. Results:. Participants included 25 patients aged 8–21 years with prominent ears (n = 9), microtia (n = 14), or another condition that affected ear appearance (n = 2). Analysis of participant qualitative data, followed by cognitive interviews and expert input, led to the development and refinement of an 18-item ear appearance scale (eg, size, shape, look up close, look in photographs) and a 12-item adverse effects scale (eg, itchy, painful, numb). Conclusions:. The EAR-Q in currently being field-tested internationally. Once finalized, we anticipate the EAR-Q will be used in clinical practice and research to understand the patient’s perspective of outcomes following ear surgery

FACE-Q Craniofacial Module: Part 1 validation of CLEFT-Q scales for use in children and young adults with facial conditions

Background: The CLEFT-Q includes 12 independently functioning scales that measure appearance (face, nose, nostrils, teeth, lips, jaws), health-related quality of life (psychological, social, school, speech distress), and speech function, and an eating/drinking checklist. Previous qualitative research revealed that the CLEFT-Q has content validity in noncleft craniofacial conditions. This study aimed to examine the psychometric performance of the CLEFT-Q in an international sample of patients with a broad range of facial conditions. Methods: Data were collected between October 2016 and December 2019 from 2132 patients aged 8 to 29 years with noncleft facial conditions. Rasch measurement theory (RMT) analysis was used to examine Differential Item Function (DIF) by comparing the original CLEFT-Q sample and the new FACE-Q craniofacial sample. Reliability and validity of the scales in a combined cleft and craniofacial sample (n=4743) were examined. Results: DIF was found for 23 CLEFT-Q items when the datasets for the two samples were compared. When items with DIF were split by sample, correlations between the original and split person locations showed that DIF had negligible impact on scale scoring (correlations ≥0.995). In the combined sample, RMT analysis led to the retention of original content for ten CLEFT-Q scales, modification of the Teeth scale, and the addition of an Eating/Drinking scale. Data obtained fit with the Rasch model for 11 scales (exception School, p=0.04). Person Separation Index and Cronbach alpha values met the criteria. Conclusion: The scales described in this study can be used to measure outcomes in children and young adults with cleft and noncleft craniofacial conditions