Developmental Risk for Infants with Cleft Lip with or Without Cleft Palate Based on Caregiver-Proxy Reports.

OBJECTIVES: (1) Assess caregiver-reported development in infants born with cleft lip ± alveolus (CL ± A) and cleft lip and palate (CLP); (2) determine factors associated with increased developmental risk; and (3) determine consistency of developmental risk before and after surgery for cleft lip. DESIGN: Prospective, longitudinal assessment of development. Time (T) 1, prior to lip closure; T2, 2 months post lip closure. SETTING: Three US craniofacial teams and online parent support groups. PARTICIPANTS: 123 total caregivers (96% mothers); 100 at T1, 92 at T2, and 69 at both T1 and T2. MEASURE: Ages and Stages Questionnaire-3 (ASQ-3): Communication, Gross Motor, Fine Motor, Problem Solving, Personal Social Domains. RESULTS: At T1 47%; at T2 42% passed all 5 Domains; 36% of infants pass all 5 Domains at both T1 and T2. Infants with CLP were at greatest risk on Communication [B = 1.449 (CI = .149-20.079), p = .038; Odds Ratio (OR) = 4.3 (CI = .923-19.650)] and Gross Motor Domains [B = 1.753 (CI = .316-20.605), p = .034; OR = 5.8 (CI = 1.162-28.671)]. Male infants were at greatest risk on Fine Motor [B = 1.542 (CI = .495-20.005), p = .009; OR = 4.7 (CI = 1.278-17.101)] and Problem Solving Domains [B = 1.200 (CI = .118-19.708), p = .044; OR = 3.3 (CI = .896-12.285)]. CONCLUSIONS: Based on caregiver report, infants with CL ± A and CLP meet referral criteria at a high rate. Infants with CLP and male infants were at greatest risk. Regular developmental screening is recommended.

A Pilot Randomized Clinical Trial of Pediatric Cystic Fibrosis Pulmonary Exacerbations Treatment Strategies.

Rationale: Despite the high prevalence and clear morbidity of cystic fibrosis (CF) pulmonary exacerbations (PEx), there have been no published clinical trials of outpatient exacerbation management. Objectives: To assess the feasibility of a pediatric clinical trial in which treatment of mild PEx is assigned randomly to immediate oral antibiotics or tailored therapy (increased airway clearance alone with oral antibiotics added only for prespecified criteria). The outcome on which sample size was based was the proportion of tailored therapy participants who avoided oral antibiotics during the 28 days after randomization. Methods: In this randomized, open-label, pilot feasibility study at 10 U.S. sites, children 6-18 years of age with CF were enrolled at their well baseline visits and followed through their first randomized PEx. Results: One hundred twenty-one participants were enrolled, of whom 94 (78%) reported symptoms of PEx at least once; of these, 81 (86%) had at least one exacerbation that met randomization criteria, of whom 63 (78%) were randomized. Feasibility goals were met, including enrollment, early detection of symptoms of PEx, and ability to randomize. Among the 33 participants assigned to tailored therapy, 10 (30%) received oral antibiotics, while 29 of 30 (97%) assigned to immediate antibiotics received oral antibiotics. The avoidance of oral antibiotics in 70% (95% confidence interval, 54-85%) was statistically significantly different from our null hypothesis that <10% of participants assigned to the tailored therapy arm would avoid antibiotics. Conclusions: Our pilot study demonstrates that conducting a randomized trial of oral antibiotic treatment strategies for mild PEx in children with CF is feasible and that assignment to a tailored therapy arm may reduce antibiotic exposure. Clinical trial registered with www.clinicaltrials.gov (NCT04608019).

Exploration of Caregiver Interrater Agreement and Test-Retest Reliability on the Infant Cleft Observer Outcomes (iCOO).

Caregiver and observer-reported measures are frequently used as outcomes for research on infants and young children who are unable to report on their own health. Our team developed the Infant with Clefts Observation Outcomes Instrument (iCOO) for infants with cleft lip with or without cleft palate. This exploratory study compared test-retest and interrater reliabilities to inform whether differences in caregiver perspective might affect the iCOO.This study is a secondary analysis comparing caregiver interrater agreement to test-retest reliability. Twenty-five pairs of caregivers completed the iCOO before surgery, 1 week later for test-retest reliability, 2 days after surgery, and 2 months after surgery. Reliability was assessed using intraclass correlations (ICCs) and t-tests were used to compare ratings between caregivers.Infants had cleft lip (28%) or cleft lip and palate (72%). Primary caregivers were predominantly mothers (92%) and secondary caregivers were predominantly fathers (80%). Test-retest reliability met psychometric standards for most items on the iCOO (81%-86% of items). Caregiver agreement on the iCOO items was lower than test-retest reliability (33%-46% of items met psychometric standards). Caregivers did not systematically differ in whether they rated infants as healthier or less healthy than the other caregiver (5%-16% of items had statistically significant differences).Caregivers used the measure consistently, but had different experiences and perceptions of their infant's health and functioning. Future studies are needed to explore mechanisms for the differences in test-retest and interrater reliability. Whenever possible, the same caregiver should provide ratings of the infant, including on the iCOO.

Caregiver Observations of Infant Well-Being Before and After Cleft Lip Surgery.

OBJECTIVE: To evaluate the sensitivity to change of daily ratings of the comfort (COMF) and behavioral/emotional health (BEH) domains of the Infants with Clefts Observation Outcomes Instrument (iCOO) at 3 time points, and to assess the association of post-surgical interventions on iCOO ratings. DESIGN: The COMF and BEH domains were completed by caregivers before (T0), immediately after (T1), and 2-months after (T2) cleft lip (CL) surgery. Analyses included descriptive statistics, correlations, t-tests, and generalized estimating equations. PARTICIPANTS: Caregivers (N = 140) of infants with CL with/without cleft palate. MAIN OUTCOME MEASURES: The COMF and BEH domain scores of the iCOO: Scale (SCALE), a summary of observable signs; and Global Impression (IMPR), a single item measuring caregivers' overall impression. RESULTS: Daily COMF and BEH SCALE and IMPR scores changed significantly during T1 (P's < 0.001) but not T0 or T2. Day 1 and 7 T0 scores were significantly higher than Day 1 and 7 T1 scores (P's <0.001 to <0.012) but similar at T2 (P's > 0.05). After CL surgery, the combined use of immobilizers and nasal stents and the use of multiple feeding methods with treatment for gastroesophageal reflux were associated with lower daily scores in COMF and BEH SCALE and IMPR (P's: 0.040 to <0.001). CONCLUSIONS: COMF and BEH iCOO scores were sensitive to daily changes in infant well-being following CL surgery. Future studies should further investigate impact of post-surgical treatments on infant well-being.

Infant with Clefts Observation Outcomes Instrument (iCOO): A New Outcome for Infants and Young Children with Orofacial Clefts.

OBJECTIVE: We evaluated the measurement properties for item and domain scores of the Infant with Clefts Observation Outcomes Instrument (iCOO). DESIGN: Cross-sectional (before lip surgery) and longitudinal study (preoperative baseline and 2 days and 2 months after lip surgery). SETTING: Three academic craniofacial centers and national online advertisements. PARTICIPANTS: Primary caregivers with an infant with cleft lip with or without cleft palate (CL ± P) scheduled to undergo primary lip repair. There were 133 primary caregivers at baseline, 115 at 2 days postsurgery, and 112 at 2 months postsurgery. MAIN OUTCOME MEASURE(S): Caregiver observation items (n = 61) and global impression of health and function items (n = 8) across eight health domains. RESULTS: Mean age at surgery was 6.0 months (range 2.7-11.8 months). Five of eight iCOO domains have scale scores, with Cronbach's alphas ranging from 0.67 to 0.87. Except for the Facial Skin and Mouth domain, iCOO scales had acceptable intraclass correlation coefficients (ICCs) ranging from 0.76 to 0.84. The internal consistency of the Global Impression items across all domains was 0.90 and had acceptable ICCs (range 0.76-0.91). Sixteen out of 20 (nonscale) items had acceptable ICCs (range 0.66-0.96). As anticipated, iCOO scores 2 days postoperatively were generally lower than baseline and scores 2 months postsurgery were consistent with baseline or higher. The iCOO took approximately 10 min to complete. CONCLUSIONS: The iCOO meets measurement standards and may be used for assessing the impact of cleft-related treatments in clinical research and care. More research is needed on its use in various treatment contexts.

Unilateral Hearing Loss in Youth: Development of Candidate Items for a Condition-Specific Validated Instrument.

OBJECTIVE: This study interviewed youth with unilateral hearing, utilizing their responses to generate candidate items for a condition-specific patient-reported instrument. STUDY DESIGN: Mixed methods, cross-sectional. SETTING: Tertiary care children's hospital. PARTICIPANTS AND METHODS: Youth with unilateral hearing loss and normal hearing in the contralateral ear were identified and recruited for participation through query of an audiometric database and through hearing loss clinics. Interviews with the youth were qualitatively analyzed to identify common themes and generate items related to functional impact. A multi-institutional expert panel reviewed items with prespecified item selection criteria. Participants rated items for impact on daily life. For preliminary criterion validity assessment, statistical analyses explored correlations between functional scores and type and severity of hearing loss. RESULTS: Thirty-nine youth aged 9 to 18 years with unilateral hearing loss participated; 31% used a hearing device. Fifteen youth participated in interviews; thematic analysis, item crafting, and expert panel item review resulted in 41 items. Twenty-six youth responded to the items, reporting low functional scores in the domains of sound localization, ear positioning, and noise environment. They reported better levels of function in carrying out group conversations, focusing on schoolwork, and feeling safe during activities. Multivariate linear regression found that youth scored 0.4 points (or approximately 8%) lower on the functional impact scale with every 20-dB HL increase in pure tone average in the abnormal ear. CONCLUSION: Youth with unilateral hearing loss report functional impact, particularly related to sound localization, ear positioning, and noise environment; therefore, they may benefit from a condition-specific functional assessment instrument.

Patient-Reported Outcome Measures in Urethral Reconstruction.

PURPOSE OF REVIEW: Treatment of anterior urethral stricture disease (USD) has shifted from endoscopic approaches to urethroplasty with significantly higher success rates among reconstructive urologists. This academic stance has led to a critical evaluation of "success" and developing disease-specific instruments to assess surgical outcomes focusing on patients' satisfaction rather than the historical goal of avoiding secondary procedures. RECENT FINDINGS: Many disease non-specific and/or non-validated patient-reported outcome measures (PROMs) have been utilized to evaluate the voiding symptoms and sexual of function of patients after urethroplasty in the literature. Urethral Stricture Surgery PROM (USS PROM) is the first validated, disease-specific PROM for anterior USD which has been designed in 2001. Urethral Stricture Symptoms and Impact Measure (USSIM) is a comprehensive PROM and is currently being validated at multiple institutions. This article reviews the tools used to assess success after urethroplasty and elaborates the need to develop a comprehensive USD-specific PROM.

Establishing Equipoise: National Survey of the Treatment of Pediatric Para-Pneumonic Effusion and Empyema.

BACKGROUND: Despite six randomized trials of various treatments for pediatric para-pneumonic effusion (PPE), management approaches differ. The purpose of this study was to gain insight into opinions on PPE treatment with the goal of designing a definitive trial to generate consensus intervention guidelines. METHODS: To evaluate physician opinions regarding PPE management, we developed a survey based on input from a nationwide, multi-disciplinary advisory group that established content validity. The survey was disseminated broadly to six pediatric medicine and interventional radiology groups. Descriptive and χ2 statistics were calculated. RESULTS: There were 741 respondents (response rate 13.1%), of whom 52.2% were surgeons, 15.2% hospitalists, 14.2% pulmonologists, 12.4% intensivists, and 6.0% interventional radiologists. Nearly all respondents (97.3%) reported caring primarily for pediatric patients. Eighty percent reported no written institutional treatment guidelines. Nearly all (90.3%) agreed that patients require antibiotics, but there was disagreement regarding their duration. Respondents also were split as to how often PPE required drainage. There were multiple absolute indications for drainage, including mediastinal shift on chest radiograph (67.2%) and loculations on imaging (47.7%). There were substantial differences in the preferred first-line methods of drainage based on the treating physician's specialty, with surgeons preferring tube thoracostomy and a fibrinolytic agent (42.0%) or video-assisted thoracoscopic surgery (41.6%), whereas interventional radiologists preferred either a tube thoracostomy (46.4%) or a tube thoracostomy with a fibrinolytic agent (39.3%) (p < 0.001). A large majority (75.3%) believed that the published evidence does not identify the optimal intervention. CONCLUSIONS: There is a lack of consensus regarding the optimal treatment of PPE. Respondents believed the published evidence is inconclusive and were willing to participate in a prospective trial. These findings will help inform the design of a randomized, pragmatic clinical trial to optimize PPE management.

Effectiveness of a Medical vs Revascularization Intervention for Intermittent Leg Claudication Based on Patient-Reported Outcomes.

Importance: Intermittent claudication (IC) is the most common presentation of infrainguinal peripheral artery disease. Both medical and revascularization interventions for IC aim to increase walking comfort and distance, but there is inconclusive evidence of the comparative benefit of revascularization given the possible risk of limb loss. Objective: To compare the effectiveness of a medical (walking program, smoking cessation counseling, and medications) vs revascularization (endovascular or surgical) intervention for IC in the community, focusing on outcomes of greatest importance to patients. Design, Setting, and Participants: Longitudinal (12-month follow-up) prospective observational cohort study conducted between July 3, 2011, and November 5, 2014, at 15 clinics associated with 11 hospitals in Washington State. Participants were 21 years or older with newly diagnosed or established IC. Interventions: Medical or revascularization interventions. Main Outcomes and Measures: Primary end points were 12-month change scores on the distance, speed, and stair-climb domains of the Walking Impairment Questionnaire (score range, 0-100). Secondary outcomes were change scores on the Walking Impairment Questionnaire pain domain (score range, 0-100), Vascular Quality of Life Questionnaire (VascuQol) (score range, 1-7), European Quality of Life-5 Dimension Questionnaire (EQ-5D) (score range, 0-1), and Claudication Symptom Instrument (CSI) (score range, 0-4). Results: A total of 323 adults were enrolled, with 282 (87.3%) in the medical cohort. At baseline, the mean duration of disease was longer for participants in the medical cohort, while those in the revascularization cohort reported more severe disease. Other characteristics were well balanced. At 12 months, change scores in the medical cohort reached significance for the following 3 outcomes: speed (5.9; 95% CI, 0.5-11.3; P = .03), VascuQol (0.28; 95% CI, 0.08-0.49; P = .008), and EQ-5D (0.038; 95% CI, 0.011-0.066; P = .006). In the revascularization cohort, there were significant improvements in the following 7 outcomes: distance (19.5; 95% CI, 7.9-31.0; P = .001), speed (12.1; 95% CI, 1.4-22.8; P = .03), stair climb (11.4; 95% CI, 1.3-21.5; P = .03), pain (20.7; 95% CI, 11.0-30.4; P < .001), VascuQol (1.10; 95% CI, 0.80-1.41; P < .001), EQ-5D (0.113; 95% CI, 0.067-0.159; P < .001), and CSI (-0.63; 95% CI, -0.96 to -0.31; P < .001). Relative improvements (percentage changes) at 12 months in the revascularization cohort over the medical cohort were observed as follows: distance (39.1%), speed (15.6%), stair climb (9.7%), pain (116.9%), VascuQol (41%), EQ-5D (18%), and CSI (13.5%). Conclusions and Relevance: Among patients with IC, those in the revascularization cohort had significantly improved function (Walking Impairment Questionnaire), better health-related quality of life (VascuQol and EQ-5D), and fewer symptoms (CSI) at 12 months compared with those in the medical cohort, providing important information to inform treatment strategies in the community.

Suitability of the PROMIS alcohol use short form for screening in a HIV clinical care setting.

BACKGROUND: At-risk alcohol use is important to identify in clinical settings to facilitate interventions. The Patient-Reported Outcomes Measurement Information System (PROMIS) Alcohol Use Short Form was developed through an item response theory process, but its utility as a screening instrument in clinical care has not been reported. OBJECTIVE: To determine the ability of the PROMIS Alcohol Use Short Form to identify people with current or future at-risk alcohol use defined by the Alcohol Use Disorders Identification Test consumption (AUDIT-C) instrument. METHODS: Observational study of people living with HIV (PLWH) in clinical care at four sites across the US. Patients completed a tablet-based clinical assessment prior to seeing their providers at clinic appointments. We used 3 definitions of clinically-relevant at-risk alcohol use and determined the proportion of PLWH with current or future at-risk drinking identified by the PROMIS instrument. RESULTS: Of 2497 PLWH who endorsed ≥1 drink in the prior 12 months, 1500 PLWH (60%) endorsed "never" for all PROMIS items. In that group, 26% had clinically-relevant at-risk alcohol use defined by one or more AUDIT-C definitions. At follow-up (N=1608), high baseline PROMIS scores had 55% sensitivity for at-risk drinking among those with at-risk drinking at baseline, and 22% sensitivity among those without baseline risk. CONCLUSIONS: The PROMIS Alcohol Use Short Form cannot be used alone to identify PLWH with clinically-relevant at-risk alcohol use. Optimal assessment of problem drinking behavior is not clear, but there does not seem to be an important role for the PROMIS instrument in this clinical setting.

Optimizing Surgical Treatment of Internationally Adopted Children With Cleft Lip and/or Palate: Understanding the Family Experience.

OBJECTIVE: To understand the experience of families with children undergoing cleft surgery following adoption from a country outside the United States. To identify factors, including the timing of surgery, that influence family function throughout the surgical experience. DESIGN: Semistructured qualitative interviews were conducted with parents of internationally adopted children postrepair of cleft lip and/or cleft palate and coded by a multidisciplinary study team. Mixed methods were used to contextualize themes derived from the parent interviews. RESULTS: Twenty parent interviews were conducted, and four core themes were identified: (1) parental anxieties prior to surgery, (2) considerations for the timing of surgery, (3) impact of the surgical experience on the child and family, and (4) modifiable sociocontextual factors. Parents considered a strong child bond with at least one parent and the ability of the child to communicate basic needs to be important before undergoing surgery. In retrospect, parents generally felt that the surgical experience did not have a negative impact on their child or their families and that the surgical experience may have even facilitated bonding and attachment with their child. Acceleration of family bonding was expressed more often by parents of children who were adopted at older than 2 years. CONCLUSIONS: In our study, parents reported that cleft surgery soon after international adoption did not appear to impair child bonding or adjustment. Specific family and provider factors that could optimize the experience for families were identified.

Pain and Urinary Symptoms Should Not be Combined into a Single Score: Psychometric Findings from the MAPP Research Network.

PURPOSE: The purpose of this study was to create symptom indexes, that is scores derived from questionnaires to accurately and efficiently measure symptoms of interstitial cystitis/bladder pain syndrome and chronic prostatitis/chronic pelvic pain syndrome, collectively referred to as urological chronic pelvic pain syndromes. We created these indexes empirically by investigating the structure of symptoms using exploratory factor analysis. MATERIALS AND METHODS: As part of the MAPP (Multi-Disciplinary Approach to the Study of Chronic Pelvic Pain) Research Network 424 participants completed questionnaires, including GUPI (Genitourinary Pain Index), ICSI (Interstitial Cystitis Symptom Index) and ICPI (Interstitial Cystitis Problem Index). Individual items from questionnaires about bladder and pain symptoms were evaluated by principal component and exploratory factor analyses to identify indexes with fewer questions to comprehensively quantify symptom severity. Additional analyses included correlating symptom indexes with symptoms of depression, which is a known comorbidity of patients with pelvic pain. RESULTS AND CONCLUSIONS: Exploratory factor analyses suggested that the 2 factors pain severity and urinary severity provided the best psychometric description of items in GUPI, ICSI and ICPI. These factors were used to create 2 symptom indexes for pain and urinary symptoms. Pain, but not urinary symptoms, was associated with symptoms of depression on multiple regression analysis, suggesting that these symptoms may impact patients with urological chronic pelvic pain syndromes differently (B ± SE for pain severity = 0.24 ± 0.04, 95% CI 0.16-0.32, ß = 0.32, p <0.001). Our results suggest that pain and urinary symptoms should be assessed separately rather than combined into 1 total score. Total scores that combine the separate factors of pain and urinary symptoms into 1 score may be limited for clinical and research purposes.

The Lymphatic Malformation Function (LMF) Instrument.

OBJECTIVE: The Lymphatic Malformation Function (LMF) instrument is a preliminary parent-report assessment designed to measure outcomes in children with cervicofacial lymphatic malformation (LM). This study aimed to assess the measurement properties of the LMF, refine it, test criterion validity, and evaluate the test-retest reliability. STUDY DESIGN: Cross-sectional. SETTING: Two pediatric tertiary referral centers. SUBJECTS: Parents of 60 children from 6 months to 15 years old with cervicofacial LM. METHODS: Parents were recruited via mail and online. The LMF was administered on paper or online initially and again within 21 days. Response distributions and interitem correlations were examined for item reduction. Exploratory factor analysis was conducted on retained items. Cronbach's α, Spearman correlation, and intraclass correlation (ICC) coefficients were calculated to test internal consistency, criterion validity (compared to stage), and test-retest reliability, respectively. RESULTS: One item was removed due to a floor effect. The response scale was collapsed from a 5-point scale to a 3-point scale due to skewness. Six items were discarded due to redundancy (interitem correlations >0.7); 2 items were discarded due to factor loadings <0.4. Exploratory factor analysis revealed a 2-factor structure explaining 84% of variance, and the domains Signs and Impacts had good internal consistency (all Cronbach's α >0.80 and <0.90), significant association with stage (P < .05), and good overall test-retest reliability (ICC, 0.82). CONCLUSION: The LMF has been refined into a 12-item, 2-domain instrument measuring LM-specific signs and impacts with internal consistency, criterion validity, and test-retest reliability.

Quality of life in youth with severe to profound sensorineural hearing loss.

IMPORTANCE: Adolescence is a life stage with rapid and major developmental changes, yet little is known about how these changes influence the quality of life of young people who are deaf or hard of hearing (DHH). OBJECTIVE: To determine differences in the 3 domains of a hearing-specific quality-of-life instrument between youth who had severe to profound sensorineural hearing loss based on whether they used no technology, hearing aids, or cochlear implants. DESIGN AND SETTING: A multi-institutional prospective cohort study. PARTICIPANTS: A convenience sample of 11- to 18-year-old youths with severe to profound sensorineural hearing loss recruited between January 1 and December 31, 2008. MAIN OUTCOME MEASURES: Youth Quality of Life-Research Instrument and Youth Quality of Life Instrument-Deaf and Hard of Hearing (YQoL-DHH) scores. The YQoL-DHH was composed of 3 domains: participation, self-acceptance/advocacy, and stigma-related quality of life. RESULTS: A total of 157 individuals participated. Overall mean (SD) age was 14.1 (2.3) years, and the female-male ratio was 82:75. Forty-nine individuals (31.2%) were not using any technology, 45 (28.7%) were using hearing aids, and 63 (40.1%) were using cochlear implants. Mean age of unilateral or first cochlear implant was 62.9 months. Thirty-eight individuals (24.2%) attended schools with DHH programs, 55 (35.0%) attended schools without DHH programs, and 58 (36.9%) attended schools for the deaf. Statistically significant differences were noted in YQoL-DHH participation and perceived stigma scores between the groups when stratified by technology used and school setting. CONCLUSIONS: These data suggest that the domains of quality of life as measured by our instrument differ significantly among youth based on technology used and school setting. Youth using no technology or cochlear implants tended to score higher than those using hearing aids in mainstream schools with or without DHH programs and in schools for the deaf. The YQoL-DHH instrument is able to detect differences in quality of life within a group of youth with severe to profound hearing loss.

Weight status, quality of life, and cigarette smoking among adolescents in Washington State.

PURPOSE: We examine age- and sex-specific associations between weight status and intensity of cigarette smoking in a large sample of adolescents. Additionally, we test whether quality of life (QOL) and weight control behaviors (i.e., trying to lose, gain, or stay the same weight) mediate the association. METHODS: We used cross-sectional data from the 2010 Washington State Healthy Youth Survey collected in grades 8, 10, and 12 (n = 11,222). Multinomial logistic regression was used to model cigarette smoking (none, light, frequent) as a function of weight status, weight control behaviors, and QOL by sex and age. Indirect effects of presumed mediators were assessed using the product of coefficients approach. RESULTS: Weight status was not associated with smoking. Trying to stay the same weight was associated with lower odds of light smoking for younger girls (OR = 0.25; 95% CI = 0.08, 0.84), whereas trying to lose weight was associated with higher odds of light smoking for older girls (OR = 1.73; 95% CI = 1.11, 2.70). Low QOL was associated with higher odds of light and frequent smoking for both girls and boys (P < 0.001). The mediation effects of weight control behavior and QOL combined were significant in the associations between body mass index percentile and smoking among older girls. CONCLUSION: Targeted interventions designed to promote QOL and healthy weight control behaviors among youth may help to decrease the prevalence of smoking.

Functional and symptom impacts of pediatric head and neck lymphatic malformations: developing a patient-derived instrument.

OBJECTIVE: Lymphatic malformations cause significant symptoms and functional deficits. Patients seek care for functional and symptomatic effects of their disease, but current disease burden and treatment outcome measures focus primarily on anatomy and histopathology. The authors describe disease impacts reported by patients and parents as a step toward more comprehensive disease burden assessments. STUDY DESIGN: Cross-sectional. SETTING: Children's hospital vascular anomaly clinic. SUBJECTS AND METHODS: Participants were recruited through a pediatric vascular anomaly clinic. A panel of senior pediatric otolaryngologists and an outcomes scientist developed interview questions based on clinical and research experience and available literature. The outcomes scientist conducted parent and adolescent interviews. The panel reviewed responses to define relevant items within functional domains. Participants rated impact on daily life for each domain. RESULTS: Thirty-one participants represented all 5 de Serres stages (mean [SD] age, 9 [6] years; n = 11 adolescents and 20 parents). Adolescents reported frequent sickness as the domain with greatest impact. Sleep was more affected in adolescents with higher stage lesions. Parents of younger children reported greatest impact on breastfeeding. For adolescents, lesion stage predicted perceived social stigma (controlling for age), whereas increasing age was associated with greater impact from swelling (controlling for stage). For parents, stage predicted breastfeeding impact (controlling for stage). CONCLUSION: This is the first detailed assessment of patient- and parent-reported functional and symptomatic impacts of head and neck lymphatic malformations. Both adolescent patients and parents of younger children reported significant symptom and functional effects of this disease.

Feasibility of using pedometers to measure daily step counts in cystic fibrosis and an assessment of its responsiveness to changes in health state.

BACKGROUND: Evaluation of physical activity is integral to the assessment of daily physical function and a potential objective outcome measure for clinical trials. We evaluated the feasibility of using pedometers to measure physical activity in adolescents and adults with cystic fibrosis (CF) and assessed the responsiveness of its measurement to changes in health state. METHODS: Participants were recruited through two CF clinics in Seattle, WA. Subjects were instructed to use their pedometer for at least one ill and two well periods (each lasting 7 days). Step rate was calculated as steps per hour of use. Daily symptoms were also recorded using the CF Respiratory Symptom Diary (CFRSD). Generalized estimating equation linear regression was used to compare mean step rate between health states and by self-reported symptom category. RESULTS: We enrolled 30 CF patients with a mean (±SD) age of 22 (±7) years and a mean forced expiratory volume in 1s (FEV(1)) of 57% (±25%) predicted. The mean period step rate increased from 397 (95% CI 324-497) steps/hour when ill to 534 (95% CI 413-654) steps/hour when well (p=0.015). Pedometer-recorded step rate also correlated with self-reported physical activity items on the CFRSD. CONCLUSION: Step rate measured with a pedometer correlates significantly with changes in health status and self-reported activity, and could be used as an outcome measure in CF.

Validation of a quality-of-life measure for deaf or hard of hearing youth.

OBJECTIVE: Quality-of-life (QOL) measures targeting youth with hearing loss are useful in population needs assessment, educational placement, and program design and evaluation. This study assesses the cross-sectional validity of the Youth Quality of Life Instrument-Deaf and Hard of Hearing Module (YQOL-DHH). STUDY DESIGN. Instrument development and cross-sectional survey. SETTING: Recruitment through schools, professional organizations, clinics, and programs for youth who are deaf or hard of hearing. SUBJECTS AND METHODS. Thirty-five candidate items were administered to 230 adolescents aged 11 to 18 years: 49% female, 61% white, 11% mild hearing loss, 20% moderate/moderate-severe, 41% severe/profound, and 28% with cochlear implants. Participants completed individual or group-administered questionnaires by paper and pencil (58%), Web-based English (29%), American Sign Language (ASL) or Pidgin Signed English (PSE) (9%) on DVD, or interviewer-supervised ASL or PSE DVD (4%). The Children's Depression Inventory (CDI-S) was also completed. Factor structure, reliability, construct validity, and respondent burden were assessed. RESULTS: Thirty-two items were retained in the final instrument covering 3 domains: self-acceptance/advocacy (14 items, Cronbach α = 0.84), perceived stigma (8 items, Cronbach α = 0.85), and participation (10 items, Cronbach α = 0.86). QOL was not significantly associated with hearing level. One-week test-retest coefficients were acceptable: self-acceptance/ advocacy (0.70), perceived stigma (0.78), and participation 0.92). As predicted, the total CDI-S score was associated in the appropriate direction (P < .0001) with all YQOL-DHH domains. Time to complete the paper-and-pencil version was 12 minutes. CONCLUSION: The YQOL-DHH shows good reliability and validity for assessing hearing-specific QOL in adolescents.

Comparison of 7-day and repeated 24-hour recall of symptoms of cystic fibrosis.

BACKGROUND: Patient reporting of symptoms in a questionnaire with a 7-day recall period is expected to differ from reporting in daily symptom diaries. METHODS: 38 patients with cystic fibrosis (CF) completed 77 week-long symptom diaries. Each diary day comprised 13 symptom items with 5-point response scales. Days 1-6 of the diary had a 24-hour recall period. Day 7 had a 7-day recall period. Concordance of 7-day recall with summary descriptors of daily reports (e.g. mean, maximum) was examined and ability of 7-day recall and mean of daily reports to discriminate between well and ill periods of health compared. RESULTS: The average difference in scores was less than 0.25 response scale points. 7-day recall was most concordant with the mean of daily reports. Discriminant ability was comparable. CONCLUSIONS: In this study sample, a questionnaire with 7-day recall provided information similar to a daily diary about the week-long experience of CF symptoms.

Approaches to craniofacial-specific quality of life assessment in adolescents.

OBJECTIVE: To ascertain the domains that adolescents aged 11 to 18 years with congenital and acquired craniofacial differences (CFDs) consider important to their quality of life (QoL) to create a craniofacial-specific module. DESIGN: Interviews and inductive qualitative methods were used to guide the development of a conceptual and measurement model of QoL among adolescents with CFDs. SETTING: The Craniofacial Center at Children's Hospital and Regional Medical Center in Seattle, Washington. PATIENTS, PARTICIPANTS: Thirty-three in-depth interviews with adolescents (aged 11 to 18 years), one young adult interview (age 19 years), 14 in-depth interviews with parents, one young adult focus group, one parent focus group, and one panel of researchers and clinical professionals working in the field. RESULTS: Using the qualitative methodology, grounded theory, seven domains that adolescents with CFDs perceive are important to having a good QoL were found. Six of the domains (coping, stigma and isolation, intimacy and trust, positive consequences, self-image, and negative emotions) comprised the Youth Quality of Life Instrument-Facial Differences module. One other domain, surgery, was a salient issue for many of the youth, but not all, so it was made into a separate module, the Youth Quality of Life Instrument- Craniofacial Surgery module. This module relates to the experience of surgery, outcomes of surgery, and preferences for future surgery. CONCLUSIONS: Using an established qualitative methodology, two QoL modules specific to adolescents with CFDs were developed and are ready for psychometric validation. Potential uses of the instruments are discussed.