Barr L, Thibeault SL, Muntz H, de Serres L. Quality of Life in Children With Velopharyngeal Insufficiency. Arch Otolaryngol Head Neck Surg. 2007;133(3):224-229. doi:10.1001/archotol.133.3.224
To determine if quality of life (QOL) is affected in children with velopharyngeal insufficiency (VPI).
Interview and survey.
Two university pediatric ambulatory centers.
This population-based sample included 58 children, aged 5 to 17 years, and their parents; 29 of the children were diagnosed as having VPI and 29 were age-matched normal controls.
Each participant (child and parent) completed 2 questionnaires: the Velopharyngeal Insufficiency Quality of Life (VPIQL), an instrument specifically designed for children with VPI, and the Pediatric Quality of Life Inventory, version 4.0 (PedsQL4.0), a standardized generic assessment instrument that systematically assesses the perception of health-related QOL in pediatric patients with chronic health conditions.
Main Outcome Measures
The VPIQL questionnaire assessed patients' and parents' perceptions of speech, swallowing, situational and emotional difficulty, activity limitations, and perception of the patient by others. Caregiver impact was also assessed in the parent version of the VPIQL.
The patients with VPI and their parents perceived a more statistically significant negative QOL compared with the normal controls and their parents in all domains of the VPIQL and the PedsQL4.0. The parents of the children with VPI perceived a more negative emotional impact (P = .02), greater speech limitations (P = .05), and fewer swallowing problems (P<.001) compared with their children.
Children with VPI and their parents perceive negative QOL greater than that of normal controls and their parents. Parental proxy may be an adequate substitute in this population.
Health-related quality of life (QOL) is an important measure of patient-based health outcome.1 In the past few decades, there has been an increased awareness of the impact of health and health care on the quality of human life. Therapeutic efforts across all disciplines are frequently directed toward quality, not quantity, of life. Quality of life questionnaires aim to measure and assess subjective states in a reproducible and valid fashion and have been a successful method of evaluating subjective states in a sound and scientific manner. Health-related QOL questionnaires have been generated for the pediatric population to assist in tracking health status and measuring patient-based treatment outcomes. Novel strategies are being sought to assess disorder-specific QOL questionnaires in children, which will assist in measuring treatment outcomes.
Velopharyngeal insufficiency (VPI) is composed of a subset of childhood speech disorders in which there are few standardized methods available for assessing psychosocial consequences.2 Velopharyngeal insufficiency can affect both speech and swallowing, which may lead to situational difficulties. Disruptions of speech in children, regardless of the cause, are associated with reductions in scholastic performance and increased risk of retention in grade school.3 Speech impediments have been reported to disrupt social, emotional and educational development, effective communication, self-esteem, and participation in activities.3 Peers and caregivers often perceive these children negatively,4 and the resulting emotional impact may ultimately affect their activity, participation, and scholastic performance.
Currently, reliable diagnosis and measures of clinical outcome in children with VPI are dependent on history, examination (including nasometry and instrumental measures of nasal air emission), and imaging (including videofluoroscopy and endoscopy). These patients have hypernasal speech (nasal emissions and nasal turbulence), which results from air escaping from the oral to the nasal cavity during nonnasal consonant production. Children may also occasionally experience reflux of liquid and solid oral contents into the nasal airway. The hypernasality inherent in children with VPI creates several speech patterns that are also often recognized in these children. They may substitute oral phonemes with nasals (/m/, /n/, and /ng/), which may result in abnormal airflow with plosives, fricatives, and affricates. These children may also omit consonants because of an inability to generate air, and there may be short utterance length due to loss of air pressure through the nose. Compensatory or maladaptive articulation using alternate techniques to valve the airway may lead to chronic sequelae such as hoarseness. Because of these difficulties, it would seem apparent that children with VPI would have an altered QOL; however, to our knowledge, this has not yet been investigated.
The objective of this study was to determine if there are alterations of QOL in children aged 5 to 17 years with VPI. Furthermore, we were interested in determining if there was agreement between children with VPI and their parents concerning perceived QOL.
All children aged 5 to 17 years who had been seen within the past 5 years at Primary Children's Medical Center (PCMC) in Salt Lake City, Utah, with signs or symptoms of VPI were contacted for recruitment either by telephone or in clinic. Inclusion into the study required the patient to be a native speaker of English with documented VPI at the time of evaluation or within the past year. The diagnosis of VPI was made by an otolaryngologist and/or speech pathologist. Patients were recruited from the clinical practices at PCMC and Columbia University, New York, NY. Thirteen patients with VPI were identified during clinic visits over a 3-month period and were asked to participate (11 at PCMC and 2 at Columbia University). All of these patients were enrolled and received questionnaires at the time of enrollment. In addition, the medical charts of 78 patients with VPI were reviewed. The International Classification of Diseases, Ninth Revision code 750.29, which is used for billing purposes for patients with VPI, was used to assist in identifying these patients. Of these patients, 40 were found to meet criteria after medical chart review and were contacted by mail and 16 (40%) returned completed questionnaires. A total of 29 children with VPI (15 male and 14 female, with a mean age of 8.7 years) participated.
Twenty-nine age-matched children without VPI (16 male and 13 female, with a mean age of 8.1 years) were randomly selected through the clinical practices to serve as controls. These children were native speakers of English with normal speech and language skills. Controls were excluded if they had a prior speech/language disorder or pharyngeal or laryngeal surgery. Prior to enrolling and obtaining consent from any subjects, approval was obtained from The University of Utah institutional review board.
Two questionnaires were provided to both patient and parent: the Pediatric Quality of Life Inventory, version 4.0 (PedsQL4.0)5 and the Velopharyngeal Insufficiency Quality of Life Inventory (VPIQL). Parents were asked to assist the child with the questionnaire as necessary. The VPIQL was developed to evaluate QOL in children with VPI. This was formulated to systematically assess patients' and parents' perceptions of functional, social, and emotional quality of life in children with VPI. Assessment measures included 6 subscales: speech limitations, swallowing problems, situational difficulty, emotional impact, perception by others, and activity limitations. Parents were provided with the same questionnaire that had 5 additional items addressing caregiver impact. The 5-point rating scale used for our questionnaire was modeled to match that used in the PedsQL4.0, which was also provided to children and parents. The PedsQL4.0 is a standardized generic assessment instrument that systematically assesses patients' and parents' perceptions of health-related QOL in pediatric patients with chronic health conditions. Assessment measures in the PedsQL4.0 include problems with physical functioning, emotional functioning, social functioning, and school functioning. This instrument has been shown to be a reliable and valid generic measure of health-related QOL.5
Means (SDs) were determined for each response within each domain and also for each domain. Analysis of covariance was used to determine if there were differences on the domains of the PedsQL4.0 and VPIQL between (1) parents and children with VPI, (2) parents of children with VPI and parents of control children, and (3) children with VPI from control children, adjusting for each child's age and sex. Scale values were calculated using the mean of scale items. Parent to child differences were calculated by subtracting each child's scores from his or her parent's scores. P<.05 was considered statistically significance.
Mean (SD) child and parent responses for each question of each domain of the PedsQL4.0 and VPIQL are given in Table 1 and Table 2, respectively. Responses ranged from 0.00 (not at all) to 4.00 (a lot). The highest-ranked mean child responses on the PedsQL4.0 were reported for “lifting something heavy,” “feeling angry,” “getting teased by others of same age,” and “forgetting things,” and the smallest mean ratings were for “walking more than a block,” “taking a shower by yourself,” and “doing chores.” Parents of children with VPI had high-ranked mean responses similar to those of their children with the exception of “lifting something heavy,” which ranked low. Additional high-ranked responses included “feeling afraid or scared,” “trouble sleeping,” and “worrying about what will happen to him or her.” The lowest-ranked mean responses for parents of children with VPI were mainly in the physical functioning domain, ie, “walking more than a block,” “lifting something heavy,” “taking a bath or shower by yourself,” and “doing chores.” Control children and their parents had relatively low mean responses for all questions with the exception of “forgetting things.”
For the VPIQL, the highly ranked responses of children with VPI included the following: “speech is nasal,” “difficulty being understood,” and “speech sounds abnormal,” with “speech is difficult for strangers to understand” being the highest affected. Parents of these children also ranked these high and in addition included “difficulty being understood at school,” “difficulty being understood over the phone,” “gets frustrated and/or gives up when he/she is not understood,” and “I am concerned about my child's nasal speech.” Control children and parents ranked all questions low.
Table 3 demonstrates the mean (SD) differences in responses between children with VPI and their parents for each domain of the PedsQL4.0 and VPIQL. The child's response was subtracted from the parent's response so that a negative difference indicates that the child perceived a more significant impact. Significant differences were found for emotional functioning (P = .02) on the PedsQL4.0 and swallowing problems (P<.001) on the VPIQL. Compared with their children, parents perceived a more negative QOL for emotional problems and a less affected QOL for swallowing problems. No significant difference was found for speech limitations (P = .05), yet the positive means represent that parents perceived a more affected QOL secondary to speech limitations.
Table 4 demonstrates the means (SDs) of the parents' and children's responses for each domain of the PedsQL4.0 and VPIQL. Significance was achieved for all categories in the 2 QOL scales. This indicates that children with VPI and their parents perceive a negative QOL significantly more than normal controls and their parents. Parents of children with VPI had the largest mean score for speech limitations (1.77), situational difficulty (1.79), and emotional functioning (1.63) and the lowest for swallowing problems (0.35) and activity limitations (0.35). Children with VPI had the highest mean score for speech limitations (1.51) and swallowing function (1.49) and the lowest for activity limitations (0.38).
We present data regarding perceived QOL in children with VPI and their parents. This is the first health-related QOL measure to specifically assess subjective states in children with VPI. Bosely and Hartnick6 used a 4-item Pediatric Voice Outcome Survey to assess functional outcome of VPI surgery, with the 4 questions in the survey being specific to voice disorders but not to VPI. The modules for the VPIQL used in our study have been designed to be disease specific while reflecting the broader impact of the condition. The VPIQL is a measurement tool that is designed to be both practical and flexible to assist in a diversity of applications such as clinical trials and performance improvement studies. Affected QOL was divided into 6 categories including speech limitations, swallowing problems, situational difficulty, emotional impact, activity limitations, and perception by others. The VPIQL written for parents was written in the third person and designed to include questions regarding caregiver impact. All items were equally weighted, which provides consistency by allowing clinicians to evaluate differences in concerns regardless of age, as well as to track concerns longitudinally.
Children with VPI and their parents reported negatively affected QOL for all domains in both instruments compared with the control group. These results corroborate the findings for similar domains that were present on both questionnaires including school, social, emotional, and physical functioning. On the VPIQL, speech limitations appeared to be the most affected domain perceived by both child and parent. These findings are not surprising given previous reports on the impact that speech disorders have on QOL.3,4 Because children with VPI often have secondary compensatory articulation errors as well as voice problems, it is impractical to differentiate these difficulties from isolated hypernasality. In addition, there may be some age-appropriate articulation errors that are commonly seen among children with or without VPI, but segregating these errors may be unrealistic. Attempts were made to make the questionnaire VPI specific with questions such as “my speech is nasal,” “I have liquids/solids come from my nose,” and “others make fun of me when I eat because food or liquids leak from the nose,” all of which were ranked as more affected in the VPI groups. However, it is also important to ask questions nonspecifically related to voice because secondary pathologic conditions may also affect the QOL of these patients (ie, “speech is too soft” and “difficulty in being understood when in a hurry”). Interestingly, other qualities such as physical functioning were significantly negatively perceived even though behaviors such as “lifting something heavy,” “running,” and “taking a bath or shower by yourself” would not typically be expected to be affected by VPI. Further refinement of the VPIQL in the process of validation will require reflection on specific questions in each domain to demonstrate the most affected outcomes.
Although proxy reports of the child's health-related QOL may differ from the child's perception of the impact of his or her condition, a parent's perception of the impact of the VPI is instrumental in the process of seeking treatment for the child. In addition, parent ratings may be necessary when the patient is unable or unwilling to rate his or her own QOL. Children with VPI and parent perceptions varied slightly in that parents noted a lesser impact on swallowing problems with a greater impact on emotional function compared with their children, when controlled for age. Considering the number of domains assessed, these findings are consistent with what has been reported in the literature, that children's and parents' health-related QOL are highly consistent when concerning children without serious illness.7 However, it has been noted that parents, more than anyone else, may be better able to judge the impact of particular context. Weissman et al8 suggest that parental reports are better predictors of emotional functioning than child reports. Therefore, children with VPI may have more negatively effected emotional functioning than they report in the present study. Interestingly, the P value for speech limitations was close to significance (P = .05), so we may have achieved significance if more children had enrolled. If significance had been reached, our findings would indicate that children perceived a lesser effect of their speech limitations on their QOL than their parents did. Eiser9 noted that parents might be less likely to make judgments regarding symptom experience. Eiser and Morse10 conducted a review to determine the relationship between ratings made by children and their parent in pediatric health-related QOL. Fourteen studies were reviewed, which showed a greater agreement for observable functioning (physical) than for nonobservable functioning (emotional and social). The heterogeneity in the latter measures may contribute to inconsistent results.10 On the whole (with careful interpretation of described swallowing problems, emotional impact, and speech limitations), our findings lend confidence to situations in the VPI population in which only 1 reporting resource (parent) is available. This may be applicable in the younger or developmentally disabled population in which patient response may be greatly influenced by parental input owing to an increased amount of guidance required for the questionnaire to be completed.
Unique to this study is the assessment of the affect of caregiver impact on parents of children with VPI. These parents perceive a negatively affected QOL. This is similar to caregiver burden that has been reported in the literature for parents of children with other chronic health conditions such as tracheotomy, rheumatoid arthritis, asthma, and epilepsy.11- 14
A limitation of this study was that we did not report or correlate severity of VPI with affected QOL. The severity of VPI was documented when available, but due to limited subject number, it was not controlled for. We may have found more significant differences depending on the severity of the deficit. We also chose not to control for age and sex given the small number of participants. A larger study is crucial to determine if male vs female and younger vs older children are affected differently as has been reported in the literature for different pediatric diseases.
This preliminary questionnaire was written using terminology thought to be appropriate for children. If however, the child was having difficulty in understanding or maintaining attention throughout the questionnaire, it was advised to allow the parent to assist him or her. This could create a potential for parental impact on child response resulting in a lesser difference seen in their mean scores. One potential solution to this problem would be to exclude younger patients who are not able to complete the questionnaire. Questionnaire simplification for this population may assist in their independent responses and should be considered in validation of the VPIQL.
This study establishes that VPI affects QOL in children with VPI aged 5 to 17 years. Quality of life questionnaires are a successful method of evaluating subjective states in a sound and scientific manner. This VPIQL hopes to provide a useful clinical index in assessing the effectiveness of speech therapy and surgical intervention in the treatment of VPI. Future studies using longitudinal data will be necessary to better assess whether the VPIQL is able to detect a reproducible and valid change in quality of life over time—an important factor that needs to be considered when validating a new instrument. Nevertheless, this study is the first step in developing the VPIQL, which will serve as an important measure of patient-based health outcome.
Correspondence: Susan L. Thibeault, PhD, CCC-SLP, Division of Otolaryngology–Head and Neck Surgery, University of Wisconsin–Madison, K4/709 600 Highland Ave, Madison, WI 53792-7375 (firstname.lastname@example.org).
Submitted for Publication: August 16, 2006; accepted September 28, 2006.
Author Contributions: Drs Barr and Thibeault had full access to all the data in the study and take responsibility for the integrity of the data and the accuracy of the data analysis. Study concept and design: Barr, Thibeault, Muntz, and de Serres. Acquisition of data: Barr, Thibeault, and Muntz. Analysis and interpretation of data: Barr and Thibeault. Drafting of the manuscript: Barr. Critical revision of the manuscript for important intellectual content: Barr, Thibeault, Muntz, and de Serres. Obtained funding: Thibeault. Administrative, technical, and material support: Barr and Thibeault. Study supervision: Thibeault, Muntz, and de Serres.
Funding/Support: This work was funded by a University of Utah Research Committee Creative Grant (Dr Thibeault).
Previous Presentation: This study was presented at the American Society of Pediatric Otolaryngology; May 22, 2006; Chicago, Ill.
Acknowledgment: This inception of this project was a result of the collaboration of pediatric otolaryngologists at the initial Cleft Research Meeting held in Salt Lake City, Utah, in February 2003. We acknowledge Kathleen Sie, MD, Department of Otolaryngology–Head and Neck Surgery, Seattle Children's Hospital, for her contributions to this study's inception and development.
Financial Disclosure: None reported.