Retrospective Cohort Studies in Craniofacial Outcomes Research: An Epidemiologist's Approach to Mitigating Bias.

Retrospective cohort studies, defined as a follow-up study in which outcome occurred prior to study onset, are common in craniofacial outcomes research and will continue to be prevalent given the increasing availability of secondary datasets and inherent prospective study limitations. However, if available data are not adequately measured, or necessary variables are absent, retrospective cohort studies can be particularly prone to bias. This brief communication aims to highlight the primary sources of bias, including measurement error, selection bias, and confounding. Each source is clearly defined, examples pertinent to craniofacial outcomes are provided, and mitigation strategies are discussed.

Does Notching Along the Nasal Velar Surface During Nasopharyngoscopy Predict Discontinuity of the Underlying Levator Veli Palatini Muscle?

To determine the sensitivity and specificity of velar notching seen on nasopharyngoscopy for levator veli palatini (LVP) muscle discontinuity and anterior positioning.Nasopharyngoscopy and MRI of the velopharynx were performed on patients with VPI as part of their routine clinical care. Two speech-language pathologists independently evaluated nasopharyngoscopy studies for the presence or absence of velar notching. MRI was used to evaluate LVP muscle cohesiveness and position relative to the posterior hard palate. To determine the accuracy of velar notching for detecting LVP muscle discontinuity, sensitivity, specificity, and positive predictive value (PPV) were calculated.A craniofacial clinic at a large metropolitan hospital. PARTICIPANTS: Thirty-seven patients who presented with hypernasality and/or audible nasal emission on speech evaluation and completed nasopharyngoscopy and velopharyngeal MRI study as part of their preoperative clinical evaluation.Among patients with partial or total LVP dehiscence on MRI, presence of a notch accurately identified discontinuity in the LVP 43% (95% CI 22-66%) of the time. In contrast, the absence of a notch accurately indicated LVP continuity 81% (95% CI 54-96%) of the time. The PPV for the presence of notching to identify a discontinuous LVP was 78% (95% CI 49-91%). The distance from the posterior edge of the hard palate to the LVP, known as effective velar length, was similar in patients with and without notching (median 9.8 mm vs 10.5 mm, P = 1.00).The observation of a velar notch on nasopharyngoscopy is not an accurate predictor of LVP muscle dehiscence or anterior positioning.

Does Distraction Lower Risk of VPI Compared to Conventional Maxillary Advancement? A Retrospective Cohort Study of Adolescents with Cleft Palate.

OBJECTIVE: To determine whether method of maxillary advancement in adolescents with cleft palate with or without cleft lip (CP ± L) influences post-operative velopharyngeal function. DESIGN: Retrospective cohort. SETTING: Pediatric Tertiary Care Hospital. PARTICIPANTS: One hundred and ninety-nine patients with CP ± L after LeFort I osteotomy for maxillary advancement at our institution between January 2007 and June 2019. INTERVENTIONS: LeFort I osteotomy via distraction osteogenesis (DO) or conventional osteotomy (CO). MAIN OUTCOME MEASURES: Patients who underwent DO or CO were compared for the presence of new velopharyngeal insufficiency (VPI), as measured by perceptual rating by a craniofacial speech-language pathologist.Of the 199 patients who underwent maxillary advancement, 126 were available for analysis. The DO group was younger, male, and had more severe maxillary hypoplasia. Following surgery, 17/41 (41.5%) of the DO group had new VPI, compared to just 23/85 (27.1%) of the CO group. After adjusting for cleft type and predicted maxillary advancement, however, there was not sufficient evidence to reject the null hypothesis of no difference in risk of post-operative VPI between the two surgical groups (prevalence ratio [PR] 1.40, 95% CI 0.68-2.90). Increased prevalence of VPI after DO versus CO was primarily observed among patients with a pre-operative velopharyngeal need ratio < 0.8 (PR = 2.01, 95% CI 0.79-5.10) and patients with normal velopharyngeal function pre-operatively (PR = 2.86, 95% CI 0.96-8.50).Our results suggest an increased rather than decreased risk of VPI following DO relative to CO. This association is primarily seen among those with a smaller velopharyngeal ratio or perceptually normal velopharyngeal function pre-operatively.

Home Language Environment of Children With Orofacial Clefts as a Target for Intervention.

OBJECTIVE: To evaluate the home language environment (HLE) in children with orofacial clefts as a potential modifiable target for language and literacy intervention. DESIGN: Feasibility study examining longitudinal trends in HLE and responses to parent-focused literacy intervention. SETTING: Tertiary care children's hospital. PARTICIPANTS: HLE data were collected for 38 children with orofacial clefts between ages 7 and 23 months. Twenty-seven participants received parent-focused literacy intervention. INTERVENTIONS: Reach Out and Read, a literacy intervention, was introduced during a clinic visit. To assess response, participants were randomized to age at intervention (9, 18, or 24 months). MAIN OUTCOME MEASURES: Primary outcome measures included measurements from recordings in the home language environment of adult word count, child vocalizations, and conversational turns. RESULTS: Baseline (preintervention) results showed lower adult word count and conversational turns for caregivers and children with cleft lip and palate, as well as for those from lower socioeconomic groups. After the literacy intervention was introduced, this cohort showed increasing measures of child and caregiver vocalizations, particularly when introduced at 18 months. CONCLUSIONS: Although these results are preliminary, findings suggest that HLE characteristics vary as a function of children's cleft type as well as family socioeconomic status. Further, our caregiver-focused literacy intervention was feasible and resulted in short-term improvements in HLE. This is the first study to document HLE as a target for intervention in children with oral clefts. These findings support further research on HLE and caregiver-focused intervention to improve language/literacy outcomes for children with oral clefts.

Crowdsourcing to Assess Speech Quality Associated With Velopharyngeal Dysfunction.

OBJECTIVE: To assess crowdsourced responses in the evaluation of speech outcomes in children with velopharyngeal dysfunction (VPD). DESIGN: Fifty deidentified speech samples were compiled. Multiple pairwise comparisons obtained by crowdsourcing were used to produce a rank order of speech quality. Ratings of overall and specific speech characteristics were also collected. Twelve speech-language pathologists (SLPs) who specialize in VPD were asked to complete the same tasks. Crowds and experts completed each task on 2 separate occasions at least 1 week apart. SETTING: On-line crowdsourcing platform. PARTICIPANTS: Crowdsource raters were anonymous and at least 18 years of age, North American English speakers with self-reported normal hearing. Speech-language pathologists were recruited from multiple cleft/craniofacial teams. INTERVENTIONS: None. MAIN OUTCOME MEASURE(S): Correlation of repeated assessments and comparison of crowd and SLP assessments. RESULTS: We obtained 6331 lay person assessments that met inclusion criteria via crowdsourcing within 8 hours. The crowds provided reproducible Elo rankings of speech quality, ρ(48) = .89; P <.0001, and consistent ratings of intelligibility and acceptability (intraclass correlation coefficient [ICC] = .87 and .92) on repeated assessments. There was a significant correlation of those crowd rankings, ρ(10) = .86; P = .0003, and ratings (ICC = .75 and .79) with those of SLPs. The correlation of more specific speech characteristics by the crowds and SLPs was moderate to weak (ICC < 0.65). CONCLUSIONS: Crowdsourcing shows promise as a rapid way to obtain large numbers of speech assessments. Reliability of repeated assessments was acceptable. Large groups of naive raters yield comparable evaluations of overall speech acceptability, intelligibility, and quality, but are not consistent with expert raters for specific speech characteristics such as resonance and nasal air emission.

Cognitive, Motor, and Language Development of Preschool Children With Craniofacial Microsomia.

OBJECTIVE: To examine neurodevelopment in preschool-aged children with craniofacial microsomia (CFM) relative to unaffected peers. DESIGN: Multisite, longitudinal cohort study. SETTING: Tertiary care centers in the United States. PARTICIPANTS: We included 92 children with CFM ("cases") through craniofacial centers and clinics. Seventy-six children without CFM (controls) were included from pediatric practices and community advertisements. This study reports on outcomes assessed when participants were an average age of 38.4 months (SD = 1.9). MAIN OUTCOME MEASURES: We assessed cognitive and motor skills using the Bayley Scales of Infant and Toddler Development, third edition (Bayley-III), and language function using subtests from the Clinical Evaluation of Language Fundamentals-Preschool, second edition (CELF-P2). RESULTS: Case-control differences were negligible for Bayley-III cognitive (effect sizes [ES] = -0.06, P = .72) and motor outcomes (ES = -0.19, P = .25). Cases scored lower than controls on most scales of the CELF-P2 (ES = -0.58 to -0.20, P = .01 to .26). Frequency counts for "developmental delay" (ie, one or more scores > 1 SD below the normative mean) were higher for cases (39%) than controls (15%); however, the adjusted odds ratio = 1.73 (P = 0.21) was not significant. Case-control differences were most evident in children with microtia or other combinations of CFM-related facial features. CONCLUSIONS: Cognitive and motor scores were similar for preschool-aged children with and without CFM. However, children with CFM scored lower than controls on language measures. We recommend early monitoring of language to identify preschoolers with CFM who could benefit from intervention.

Behavioral Adjustment of Preschool Children With and Without Craniofacial Microsomia.

OBJECTIVE: The study aim was to assess behavioral adjustment in preschool children with and without craniofacial microsomia (CFM). DESIGN: Multisite cohort study of preschoolers with CFM ("cases") or without CFM ("controls"). PARTICIPANTS: Mothers (89%), fathers (9%), and other caregivers (2%) of 161 preschoolers. OUTCOME MEASURE: Child Behavior Check List (CBCL 1.5-5); linear regressions with standardized effect sizes (ES) adjusted for sociodemographic confounds. RESULTS: Child Behavior Check Lists for 89 cases and 72 controls (average age 38.3 ± 1.9 months). Children were male (54%), white (69%), and of Latino ethnicity (47%). Cases had microtia with mandibular hypoplasia (52%), microtia only (30%), or other CFM-associated features (18%). Nearly 20% of cases had extracranial anomalies. Composite CBCL scores were in the average range compared to test norms and similar for cases and controls. On the subscales, cases' parents reported higher Anxious/Depressed scores (ES = 0.35, P = .04), Stress Problems (ES = 0.40, P = .04), Anxiety Problems (ES = 0.34, P = .04), and Autism Spectrum Problems (ES = 0.41, P = .02); however, the autism subscale primarily reflected speech concerns. Among cases, more problems were reported for children with extracranial anomalies and certain phenotypic categories with small ES. CONCLUSIONS: Behavioral adjustment of preschoolers with CFM was comparable to peers. However, parental reports reflected greater concern for internalizing behaviors; thus, anxiety screening and interventions may benefit children with CFM. Among cases, more problems were reported for those with more complex presentations of CFM. Craniofacial microsomia-related speech problems should be distinguished from associated psychosocial symptoms during developmental evaluations.

Methods and Challenges in a Cohort Study of Infants and Toddlers With Craniofacial Microsomia: The Clock Study.

OBJECTIVE: The Craniofacial microsomia: Longitudinal Outcomes in Children pre-Kindergarten (CLOCK) study is a longitudinal cohort study of neurobehavioral outcomes in infants and toddlers with craniofacial microsomia (CFM). In this article, we review the data collection and methods used to characterize this complex condition and describe the demographic and clinical characteristics of the cohort. SETTING: Craniofacial and otolaryngology clinics at 5 study sites. PARTICIPANTS: Infants with CFM and unaffected infants (controls) ages 12 to 24 months were recruited from the same geographical regions and followed to age 36 to 48 months. METHODS: Phenotypic, neurodevelopmental, and facial expression assessments were completed during the first and third waves of data collection (time 1 and time 3, respectively). Medical history data were taken at both of these time points and during an intermediate parent phone interview (time 2). RESULTS: Our cohort includes 108 cases and 84 controls. Most cases and controls identified as white and 55% of cases and 37% of controls identified as Hispanic. Nearly all cases had microtia (95%) and 59% had mandibular hypoplasia. Cases received extensive clinical care in infancy, with 59% receiving care in a craniofacial clinic and 28% experiencing at least one surgery. Study visits were completed at a study site (92%) or at the participant's home (8%). CONCLUSIONS: The CLOCK study represents an effort to overcome the challenges of characterizing the phenotypic and neurodevelopmental outcomes of CFM in a large, demographically and geographically diverse cohort.

Speech and Surgical Outcomes in Children With Veau Types III and IV Cleft Palate: A Comparison of Internationally Adopted and Nonadopted Children.

OBJECTIVE: This study compares speech and surgical outcomes in internationally adopted and nonadopted patients undergoing cleft palate repair, and examines the influence of age at initial palatoplasty. DESIGN: Retrospective cohort study setting: Tertiary Care Children's Hospital. PATIENTS: 70 international adoptees and 211 nonadoptees with Veau type III and IV clefts (without associated syndrome) repaired at our institution. OUTCOME MEASURES: Outcomes included VPI, compensatory misarticulations, intelligibility, nasal air emission, oronasal fistula, and secondary speech surgery. Speech evaluations completed near 5 years of age were gathered from a prospectively collected database. RESULTS: Adoptees underwent palatoplasty 5.2 months after arrival, a mean of 10.4 months later than nonadoptees. Adoptees were significantly more likely to develop moderate/severe VPI and trended toward more frequent need for secondary speech surgery. Oronasal fistula occurred at similar rates. Increased age at initial palatoplasty was a significant predictor of moderate to severe VPI, and need for secondary speech surgery. CONCLUSIONS: International adoptees undergo palatoplasty 10.4 months later than nonadoptees and are significantly more likely to develop moderate/severe VPI, with a trend toward increased secondary speech surgery. An association between treatment delay and moderate/severe VPI and secondary speech surgery has been demonstrated. While a causal relationship between delayed repair and inferior outcomes in international adoptees has not been proven, this data suggests that surgical intervention upon unrepaired cleft palates soon after adoption may be beneficial. The opportunity for a change in practice exists, as half of the 10.4-month relative delay in palate repair occurs postadoption.

Characterizing Speech Phenotype in Individuals With Craniofacial Microsomia: A Scoping Review.

INTRODUCTION: Craniofacial microsomia (CFM) is a complex congenital condition primarily affecting the ear, mandible, facial nerve and muscles, and tongue. Individuals with CFM are at increased risk of hearing loss, obstructive sleep apnea, and feeding/swallowing difficulties. The purpose of this scoping review was to summarize evidence pertaining to speech production in CFM. METHOD: All articles reporting any characteristic of speech production in CFM were included and screened by two independent reviewers by title, abstract, and full text. Data charting captured details related to study population and design, CFM diagnostic criteria, speech outcome measurement, and key findings. The Preferred Reporting Items for Systematic Reviews and Meta-Analyses Extension for Scoping Reviews checklist guided reporting of results. Our protocol was registered on the Open Science Framework (https://osf.io/npr94/) and published elsewhere. RESULTS: Forty-five articles were included in the detailed review. Most articles originated from the United States, were published in the past decade, and utilized case report/series study design. A speech-language pathologist authored 29%. The prevalence of velopharyngeal insufficiency ranged from 19% to 55% among studies. Oral distortion of alveolar and palatal fricatives and affricates primarily characterized articulation errors. Studies identified increased disordered speech and lower intelligibility in adolescents with CFM compared to unaffected peers. Evidence pertaining to phonatory and respiratory speech findings is limited. CONCLUSIONS: Evidence supports that individuals with CFM are at increased risk of both velopharyngeal and articulatory speech differences. Additional information is needed to develop speech screening guidelines for children with CFM. Heterogeneity in study design and outcome measurement precludes comparisons across studies. SUPPLEMENTAL MATERIAL: https://doi.org/10.23641/asha.24424555.

Characterising the speech phenotype in individuals with craniofacial microsomia: a scoping review protocol.

INTRODUCTION: Asymmetric mandibular hypoplasia, microtia, tongue and laryngeal anomalies, and soft palate and facial nerve dysfunction are clinical features observed in children with craniofacial microsomia (CFM). Despite involvement of all these structures in hearing and speech, there is limited evidence reporting speech outcomes in this population. Systematic reviews of clinical and surgical interventions related to CFM have been published, but no methodological review of speech outcomes exists. This scoping review will summarise what is known about speech production in individuals with CFM as well as illustrate gaps in the existing body of literature that will guide future research. METHODS/ANALYSIS: This review will follow the methodological framework for scoping reviews first reported by Arksey & O'Malley and revised by Levac and others. Databases searched will include Ovid MEDLINE, EMBASE, CINAHL, PsycINFO and grey literature. Articles reporting any parameter of speech production in individuals with CFM will be considered for inclusion. Articles published in a language other than English will be excluded. Articles will be screened in three stages: (1) title review, (2) abstract review and (3) full text review. Ten per cent of articles will be rescreened by a second reviewer. Reference lists will be hand reviewed to identify additional relevant articles. Data charting will capture article metadata, study population and design, CFM diagnostic criteria, speech outcome measurement and key findings. The Preferred Reporting Systems for Systematic Reviews and Meta-Analyses Protocols-Extension for Scoping Reviews checklist will guide reporting of results. Descriptive analysis and data visualisation strategies will be used. ETHICS AND DISSEMINATION: Institutional review board approval is not required for a scoping review, as it does not directly involve human subjects. Results will be disseminated through peer-reviewed publication as well as conference presentation.

Sphincter Pharyngoplasty for Velopharyngeal Dysfunction: Impact of 22q11.2 Deletion Syndrome.

OBJECTIVE: Patients with 22q11.2 deletion syndrome (22q11DelS) often present with velopharyngeal dysfunction (VPD). VPD in patients with 22q11DelS is multifactorial beyond velopharyngeal insufficiency (VPI) alone, and differences in surgical outcomes are poorly understood. Our objective was to determine whether patients with 22q11DelS have an increased risk for persistent VPI after sphincter pharyngoplasty compared to patients without 22q11DelS. METHODS: We completed a retrospective cohort study of patients with 22q11DelS undergoing sphincter pharyngoplasty between 1995 and 2019 using a VPD clinic database. Patients with 22q11DelS were compared to a cohort of 2:1 frequency-matched (age, degree of velopharyngeal closure) patients without 22q11DelS. Variables included patient characteristics, surgical history, perceptual speech evaluation, and degree of closure on nasopharyngoscopic evaluations. Primary outcomes included postoperative VPI severity and hypernasality. Speech and nasopharyngoscopic characteristics were compared using Fisher's exact test. Postoperative VPI severity and hypernasality were compared between groups via relative risks (RR) from mixed effects Poisson regression models, with random effects of age and velopharyngeal closure. RESULTS: 134 patients (51 22q11DelS, 83 matched) were included, with mean age of 7.3 years (standard deviation 3.0) and 50% male. Cohorts had similar preoperative speech characteristics and nasopharyngoscopic findings. Patients with 22q11DelS had similar postoperative VP function as patients without 22q11DelS (RR 0.85, CI 0.46-1.57 for VPI severity, RR 0.83, CI 0.45-1.53 for hypernasality). Even after adjusting by preoperative variables, no differences were seen between both groups. CONCLUSION: Matched for age and pre-operative velopharyngeal closure, patients with and without 22q11DelS and VPI had similar benefits after sphincter pharyngoplasty. LEVEL OF EVIDENCE: Non-randomized controlled cohort study, 3 Laryngoscope, 133:2813-2820, 2023.

Speech Outcomes After Sphincter Pharyngoplasty for Velopharyngeal Insufficiency.

OBJECTIVES/HYPOTHESIS: To investigate perceptual speech outcomes following sphincter pharyngoplasty (SP) and to identify patient characteristics associated with velopharyngeal insufficiency (VPI) resolution or improvement. METHODS: Retrospective review of prospectively collected data was performed of consecutive patients that underwent SP for management of VPI between 1994 and 2016 at a single tertiary care pediatric hospital. Demographic data, nasendoscopic findings, and speech characteristics were recorded using a standardized protocol. Pre- and post-operative VPI was graded on a five-point Likert scale. Frequency of post-operative VPI resolution and improvement was assessed and associations with patient characteristics were analyzed. The association between odds of VPI resolution or improvement and five patient characteristics identified a priori was performed controlling for confounding factors. RESULTS: Two-hundred ninety-six subjects were included. All patients had at least minimal VPI pre-operatively; 72% were graded moderate or severe. Sixty-four percent experienced resolution and 83% improved at least one point on the VPI-severity scale. Of the five patient characteristics, only history of cleft palate repair was significantly associated with decreased odds of VPI improvement but not resolution when controlling for other variables. CONCLUSIONS: Sphincter pharyngoplasty resulted in resolution of VPI in 64% and improvement in 83% of subjects. Children with a history of cleft palate had significantly decreased odds of VPI improvement compared to those without a history of cleft palate. Neither syndrome diagnosis nor 22q11 deletion had a significant association with speech outcomes after sphincter pharyngoplasty. LEVEL OF EVIDENCE: 4 Laryngoscope, 131:E2046-E2052, 2021.

Speech, Language, and Communication Skills of Adolescents With Craniofacial Microsomia.

Purpose Youth with craniofacial microsomia (CFM) have anomalies and comorbidities that increase their risk for speech, language, and communication deficits. We examined these outcomes in youth with and without CFM and explored differences as a function of CFM phenotype and hearing status. Method Participants included youth ages 11-17 years with CFM (n = 107) and demographically similar controls (n = 306). We assessed speech intelligibility, articulation, receptive and expressive language, and parent and teacher report measures of communication. Hearing status was also screened at the study visit. Group differences were estimated using linear regression analyses with standardized effect sizes (ES) adjusted for demographic characteristics (adjusted ES) or negative binomial regression. Results Youth with CFM scored lower than unaffected peers on most measures of intelligibility, articulation, expressive language, and parent- and-teacher-rated communication. Differences were most pronounced among participants with CFM who had mandibular hypoplasia plus microtia (adjusted ES = -1.15 to -0.18). Group differences were larger in youth with CFM who failed the hearing screen (adjusted ES = -0.73 to 0.07) than in those who passed the hearing screen (adjusted ES = -0.34 to 0.27). Conclusions Youth with CFM, particularly those with mandibular hypoplasia plus microtia and/or hearing loss, should be closely monitored for speech and language concerns. Further research is needed to identify the specific needs of youth with CFM as well as to document the course of speech and language development in children with CFM.

Change in Quality of Life with Velopharyngeal Insufficiency Surgery.

OBJECTIVES: (1) To define the minimal clinically important difference (MCID) of the Velopharyngeal Insufficiency (VPI) Effects on Life Outcomes (VELO) instrument, and (2) to test for the change in quality of life (QOL) after VPI surgery. STUDY DESIGN: Prospective observational cohort. SETTING: VPI clinic at a tertiary pediatric medical center. SUBJECTS AND METHODS: Children with VPI and their parents completed the VELO instrument (higher score is better QOL) at enrollment and then underwent VPI surgery (Furlow palatoplasty or sphincter pharyngoplasty, n = 32), other treatments (obturator or oronasal fistula repair, n = 7), or no treatment (n = 18). They completed the VELO instrument again and an instrument of global rating of change in QOL at 1 year. The MCID was anchored to the global change instrument scores corresponding to "a little" or "somewhat" better. Within-group (paired t test) and between-group (Student t test) changes in VELO scores were tested for the VPI surgery and no treatment groups. The association between treatment group and change in VELO scores was tested with multivariate linear regression, adjusting for confounders. RESULTS: Follow-up was obtained for 37 of 57 (65%) patients. The mean (±standard deviation) change in VELO scores corresponding to the MCID anchor was 15 ± 13. The VELO score improved significantly more in the VPI surgery group (change, 22 ± 15; P < .001) than in the no treatment group (change, 9 ± 12; P = .04), after adjusting for confounders (P = .007 between groups). CONCLUSION: VPI surgery using the Furlow palatoplasty or sphincter pharyngoplasty improves VPI-specific QOL, and the improvement is clinically important.

Cleft palate repair and velopharyngeal dysfunction.

LEARNING OBJECTIVES: After studying this article, the participant should be able to: (1) Describe the technical details common to all cleft palate repairs that optimize outcomes and minimize complications. (2) Explain the subjective and objective evaluation of speech in children with cleft palate. (3) Practice with an increased awareness of the management of complications associated with cleft palate repair. (4) Design a treatment plan for velopharyngeal dysfunction. SUMMARY: Goals of a successful cleft palate repair include separation of the oral and nasal components without fistula, achieving sufficient velar length, and creating functional transverse orientation of the levator muscle sling. A number of techniques have been described to achieve these goals, but they all have the following technical details in common: elevation of oral mucosal flaps based on the greater palatine arteries, tension free nasal lining mobilization, and functional intervelar muscle dissection. After palate repair, speech evaluation needs to be performed by an objective interdisciplinary team following a standardized protocol. Identification of velopharyngeal insufficiency secondary to an incompetent nasopharyngeal port will necessitate secondary speech surgery. These secondary techniques include pharyngeal flaps, soft palate lengthening, or pharyngeal sphincters, which should be tailored to optimize speech, while minimizing the risk of obstructive sleep apnea.

Validity and responsiveness of VELO: a velopharyngeal insufficiency quality of life measure.

OBJECTIVE: Test the Velopharyngeal Insufficiency (VPI) Effects on Life Outcomes (VELO) instrument for validity, reliability, and responsiveness. STUDY DESIGN: Observational cohort. SETTING: Academic tertiary medical center. SUBJECTS: Children with VPI (n = 59) and their parents (n = 84) were prospectively enrolled from a pediatric VPI clinic. METHODS: Pediatric speech language pathologists diagnosed VPI using perceptual speech analysis and rated VPI severity and speech intelligibility deficit (each as minimal, mild, moderate, or severe). All parents and youth 8+ years old (n = 24) completed the VELO instrument and other quality-of-life questionnaires at baseline; the first 40 subjects completed the VELO instrument again 2 weeks later. Treatments included Furlow palatoplasty (n = 20), sphincter pharyngoplasty (n = 14), or an obturator (n = 2), and 29 of 36 (81%) subjects completed the questionnaires 3 months posttreatment. VELO was tested with correlations for criterion validity against VPI severity, construct validity against speech intelligibility and velopharyngeal gap size, and concurrent validity against other quality-of-life measures (r > .40 demonstrating validity); for test-retest reliability using intraclass correlation (>.6 demonstrating reliability); and for responsiveness with the 3-month posttreatment measure using the paired t test. RESULTS: Parental responses are reported; youth responses showed similar results. The VELO instrument did not meet criterion validity (r = -.18, P = .10), or functional construct validity (r = -.37, P = .001), but did meet anatomic construct and concurrent validity (each r > .50, P < .01). VELO scores demonstrated excellent test-retest reliability (r = .85, P < .001) and responsiveness (baseline 54 ± 14 to posttreatment 70 ± 18, P < .001). CONCLUSION: VELO provides a VPI-specific quality-of-life instrument that demonstrates concurrent validity, test-retest reliability, and responsiveness to change in quality of life with treatment.

Modification and evaluation of a Velopharyngeal Insufficiency Quality-of-Life instrument.

OBJECTIVES: To modify the existing 45-item Velopharyngeal Insufficiency (VPI) Quality-of-Life (QOL) instrument (VPIQL), to assess the modified instrument for reliability, and to provide further validation. DESIGN: Validation convenience sample from a previously conducted pilot study. SETTING: Two academic tertiary referral medical centers. PARTICIPANTS: Deidentified data were used from 29 patients with VPI and 29 control patients aged 5 to 17 years and their parents. MAIN OUTCOME MEASURES: Patients and parents completed the VPIQL and a generic pediatric QOL instrument (Pediatric Quality of Life Inventory, Version 4 [PedsQL4-0]). Twenty-two items were removed from the VPIQL for ceiling effects, floor effects, and redundancy to produce the modified instrument: the VPI Effects on Life Outcomes instrument (VELO). The VELO was tested for internal consistency (Cronbach α), discriminant validity (paired t test with control patients), and concurrent validity (Pearson correlation with the PedsQL4-0). These analyses were also completed for the parents. RESULTS: The 45-item VPIQL was reduced to the 23-item VELO, which had excellent internal consistency (Cronbach α, .96 for parents and .95 for patients with VPI). The VELO also discriminated well between the patients with VPI and the control patients, with a mean (SD) score that was significantly lower (worse) for patients with VPI (67.6 [23.9]) than for control patients (97.0 [5.2]) (P < .001). The VELO total score was significantly correlated with the PedsQL4.0 (r = 0.73) among the patients with VPI. Similar results were seen in parent responses. CONCLUSIONS: The VELO is a 23-item QOL instrument that was designed to measure and follow QOL in patients with VPI, with less burden than the original VPIQL. The VELO demonstrates internal consistency, discriminant validity, and concurrent validity with the PedsQL4-0.