Jonathan R. Skirko

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.

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.

Association of Velopharyngeal Insufficiency With Quality of Life and Patient-Reported Outcomes After Speech Surgery

Importance Patients with cleft palate and other causes of velopharyngeal insufficiency (VPI) suffer adverse effects on social interactions and communication. Measurement of these patient-reported outcomes is needed to help guide surgical and nonsurgical care. Objectives To further validate the VPI Effects on Life Outcomes (VELO) instrument, measure the change in quality of life (QOL) after speech surgery, and test the association of change in speech with change in QOL. Design, Setting, and Participants Prospective descriptive cohort including children and young adults undergoing speech surgery for VPI in a tertiary academic center. Participants completed the validated VELO instrument before and after surgical treatment. Main Outcomes and Measures The main outcome measures were preoperative and postoperative VELO scores and the perceptual speech assessment of speech intelligibility. The VELO scores are divided into subscale domains. Changes in VELO after surgery were analyzed using linear regression models. VELO scores were analyzed as a function of speech intelligibility adjusting for age and cleft type. The correlation between speech intelligibility rating and VELO scores was estimated using the polyserial correlation. Results Twenty-nine patients (13 males and 16 females) were included. Mean (SD) age was 7.9 (4.1) years (range, 4-20 years). Pharyngeal flap was used in 14 (48%) cases, Furlow palatoplasty in 12 (41%), and sphincter pharyngoplasty in 1 (3%). The mean (SD) preoperative speech intelligibility rating was 1.71 (1.08), which decreased postoperatively to 0.79 (0.93) in 24 patients who completed protocol (P < .01). The VELO scores improved after surgery (P<.001) as did most subscale scores. Caregiver impact did not change after surgery (P = .36). Speech Intelligibility was correlated with preoperative and postoperative total VELO score (P < .01) and to preoperative subscale domains (situational difficulty [VELO-SiD, P = .005] and perception by others [VELO-PO, P = .05]) and postoperative subscale domains (VELO-SiD [P = .03], VELO-PO [P = .003]). Neither the VELO total nor subscale score change after surgery was correlated with change in speech intelligibility. Conclusions and Relevance Speech surgery improves VPI-specific quality of life. We confirmed validation in a population of untreated patients with VPI and included pharyngeal flap surgery, which had not previously been included in validation studies. The VELO instrument provides patient-specific outcomes, which allows a broader understanding of the social, emotional, and physical effects of VPI. Level of Evidence 2.

Management of midnasal stenosis with infant surgically assisted rapid palatal expansion (iSARPE): iSARPE

INTRODUCTION Neonates are obligate nasal breathers; therefore, stenosis at any level can be associated with significant respiratory distress. Drill-out techniques are described for the management of pyriform aperture stenosis and choanal atresia; however, midnasal stenosis is a common associated feature not addressed by these procedures. Failed surgical management of pyriform stenosis and choanal atresia may be due to scarring or unrecognized concomitant midnasal stenosis. Additionally, craniofacial disorders such as Apert and Crouzon syndromes can be associated with nasal stenosis without pyriform aperture or choanal stenosis. Surgically assisted rapid palatal advancement (SARPE) is a technique of palatal distraction that has been used to provide space for crowded maxillary dentition and in maxillary hypoplasia. More recent usage includes adult obstructive sleep apnea (OSA) patients. Rapid maxillary expansion without osteotomies has been described in pediatric patients with OSA. SARPE has not been described in early childhood. We describe the use of infant SARPE (iSARPE) to expand the nasal cavities in two patients with different pathologies causing midnasal stenosis. This technique has not previously been described for nasal stenosis, and our early results have been promising for their efficacy.

Validity and Responsiveness of VELO: A VPI QoL Instrument

Objective: In a study of patients with velopharyngeal insufficiency (VPI) diagnosed by pediatric speech pathologists, test the VPI Effects on Life Outcomes (VELO) instrument for criterion validity, construct validity, reliability, and responsiveness (ability to detect change in quality of life). Method: VPI subjects/parents completed the VELO at baseline, 2 weeks later, and 3 months posttreatment. Criterion-validity tested correlation between VELO score and VPI severity; construct-validity tested correlation with speech intelligibility. Test-retest reliability tested intraclass-correlation between baseline VELO score and 2-week score. Responsiveness tested mean baseline and posttreatment scores with the paired t test. Results: Parental responses are reported (n = 83); youth responses (obtained in 8+ year-olds only; n = 24) showed similar results. VPI severity (minimal n = 11, mild n = 31, moderate n = 27, severe n = 14) and speech intelligibility were rated by pediatric speech pathologists. The VELO instrument did not meet criterion validity (Spearman r = −0.21, P = .07) but did meet construct validity (Spearman r = −0.36, P = .001). VELO scores demonstrated excellent test-retest reliability (r = 0.85, P < .001, n = 40). Treatments included Furlow palatoplasty (n = 20), sphincter pharyngoplasty (n = 14), or an obturator (n = 2) with n = 28 (78%) completing the VELO posttreatment. The VELO showed responsiveness to treatment improving from baseline 54 ± 15 to posttreatment 70 ± 18 (P < .001). Conclusion: VELO provides a VPI specific quality of life instrument that demonstrates construct validity, test-retest reliability, and responsiveness to change in quality of life with treatment.