Giving voice to the child perspective: psychometrics and relative precision findings for the Child Health Questionnaire self-report short form (CHQ-CF45).

PURPOSE: To derive and evaluate a shorter self-report Child Health Questionnaire (CHQ) legacy measure for use in research and clinical trials/care. METHODS: Stepwise regression, factor analysis, and item scaling principles were used to derive and guide item selection, using data from a large general sample in the Netherlands (n = 933). Feasibility was assessed in a school sample (n = 114) and item internal consistency, discriminant validity, floor, and ceiling effects were evaluated using an external larger validation sample in the US (n = 1468). Reliabilities were estimated using Cronbach's alpha. Relative precision (RP), the ability to distinguish between clinical subgroups, was computed by comparing the proportion of variance explained by the short-form scales vs. respective full-length scales. RESULTS: The CHQ-CF was reduced from 87 to 45 items. The median alpha coefficient was 0.89. Ninety-seven to 100% scaling successes for item discriminant validity were observed. Floor effects were not observed; some ceiling effects were detected. RP estimates ranged from 0.73 to 1.37. CONCLUSION: The CHQ-SF45 is reliable and valid and exceeds item level scaling criteria.

Feasibility and Psychometric Properties of the Infant Toddler Quality of Life (ITQOL) Questionnaire in a Community-Based Sample of Healthy Infants in China.

Objective Evaluate the feasibility and psychometric properties of the Infant Toddler Quality of Life (ITQOL) questionnaire as a measure of health-related quality of life (HRQOL) in a sample of Chinese infants. Methods The linguistically validated Simplified Chinese version of the ITQOL was used in a multicenter, observational study of healthy, term infants (N = 427), age 6 weeks at enrollment, in China. At Days 1 and 48, parents/guardians completed the ITQOL, the Short Form Health Survey (SF-12v2) and the Infant Gastrointestinal Symptom Questionnaire (IGSQ). ITQOL feasibility, reliability, ceiling/floor effects, concurrent validity and discriminatory validity were evaluated. Results Feasibility of administering the ITQOL was supported by strong response rates (> 97%) with < 1% missing items for all scales except physical abilities. Reliability was acceptable (Cronbach's alpha > 0.70) for all scales except Day 1 General Health (0.67). Floor effects were minimal (< 2%), except Day 1 physical abilities (7%). Ceiling effects increased from Days 1 to 48 across all scales. Concurrent validity was demonstrated by correlations between ITQOL infant-focused scales and IGSQ score (r = -0.20 to - 0.34, p < 0.001) and between parent-focused scales and SF-12v2 mental health composite (MCS) scores (r = 0.29-0.46, p < 0.001). ITQOL scales discriminated between infant subgroups based on illness-related outcomes (sick visits, adverse events) and between parent subgroups based on SF-12v2 MCS scores. Conclusion The Simplified Chinese version of the ITQOL performed well in a community-based sample of Chinese infants, with evidence supporting the instrument's feasibility, reliability, and validity. These data support the ITQOL as a valuable tool to assess HRQOL in Chinese infants.

Children's family experiences: development of the PROMIS® pediatric family relationships measures.

PURPOSE: To describe the development of pediatric family relationships measures, with versions for child self-report (8-17 years) and parent-report for children 5-17 years old. Measures were created for integration into the Patient Reported Outcomes Measurement Information System (PROMIS®). METHODS: Semi-structured interviews with 10 experts, 24 children, and 8 parents were conducted to elicit and clarify essential elements of family relationships. A systematic literature review was conducted to identify item concepts representative of each element. The concepts were transformed into items that were iteratively revised based on cognitive interviews (n = 43 children) and item translatability review. Psychometric studies involving 2846 children and 2262 parents were conducted to further refine and validate the instruments. RESULTS: Qualitative procedures supported the development of content valid Family Relationships item banks. Final child- and parent-report item banks each contain 47 items. Unidimensional item banks were calibrated using IRT-modeling to estimate item parameters representative of the US population and to enable computerized adaptive test administration. Four- and eight-item short forms were constructed for standard fixed format administration. All instruments have strong internal consistency, retest-reliability, and provide precise estimates of various levels of family relationship quality. Preliminary evidence of the instruments' validity was provided by known-group comparisons and convergence with legacy measures. CONCLUSION: The PROMIS pediatric Family Relationships measures can be applied in research focused on determinants, outcomes, and the protective effects of children's subjective family relationship experiences.

Childhood chronic conditions and health-related quality of life: Findings from a large population-based study.

The objective of this study was to assess the impact of health-related quality of life (HRQOL) across prevalent chronic conditions, individually and comorbid, in school-aged children in the Netherlands. 5301 children aged 4-11 years from the Dutch Health Interview Survey were included. Parents completed questionnaires regarding child and parental characteristics. HRQOL of children was measured using the Child Health Questionnaire Parent Form 28 (CHQ-PF28). Independent-t tests were used to assess differences in the mean scores of the CHQ-PF28 summary scales and profile scales between children with a prevalent chronic condition (excluding or including children with multiple chronic conditions) and children without a chronic condition. Cohen's effect sizes (d) were calculated to assess the clinical significance of difference. The mean age of children was 7.55 (SD 2.30) years; 50.0% were boys. In children without any chronic condition, the mean score of physical summary scale (PhS) was 58.53 (SD 4.28) and mean score of the psychosocial summary scale (PsS) was 53.86 (SD 5.87). Generally, PhS and/or PsS scores in children with only one condition were lower (p<0.05) than for children without chronic conditions. When children with multiple conditions were included, mean scores of CHQ-PF28 summary and profile scales were generally lower than when they were excluded. The present study shows important information regarding the impact of prevalent chronic conditions on HRQOL in a representative population-based sample of school-aged children in the Netherlands. The information could be used for developing a more holistic approach to patient care and a surveillance framework for health promotion.

Filum Section for Urinary Incontinence in Children with Occult Tethered Cord Syndrome: A Randomized, Controlled Pilot Study.

PURPOSE: Occult tethered cord syndrome, in which there is normal neuroanatomic imaging despite clinical and urodynamic evidence of neuropathic bladder behavior, is controversial. Several uncontrolled series describe improvement in bladder function following section of the filum terminale. We performed a pilot randomized, controlled study comparing medical treatment to surgical section of the filum plus medical treatment in children with occult tethered cord syndrome. MATERIALS AND METHODS: Children refractory to standard medical management for 1 year or more with normal conus position on magnetic resonance imaging and abnormal urodynamics were randomized. Exclusion criteria included any neurological conditions, spinal dysraphism, bladder outlet obstruction and an atonic bladder. Patients were assessed at randomization and 1 year later with a standardized urodynamic score, the validated PEMQOL (Pediatric Enuresis Module on Quality of Life™) scale, and a validated bowel and bladder dysfunction score. RESULTS: After 8 years we accrued 21 patients. The bowel and bladder dysfunction score improved in the surgical and medical arms (20% and 24%) and the urodynamic score improved slightly (6% and 4%, respectively). The PEMQOL Child and Family Impact Scales improved modestly in both groups. All differences were nonsignificant. Interim analysis indicated that more than 700 patients in each arm would be required to demonstrate a statistical difference with respect to urodynamic score based on our preliminary data. CONCLUSIONS: There appears to be no objective difference in urological outcome between medical management plus or minus filum section for patients with occult tethered cord syndrome. These data challenge the existence of the concept of occult tethered cord syndrome, in which bowel and bladder dysfunction score is attributed to tethering by the filum despite a normally located conus.

Correction: Determinants of Health-Related Quality of Life in School-Aged Children: A General Population Study in the Netherlands.

[This corrects the article DOI: 10.1371/journal.pone.0125083.].

Determinants of health-related quality of life in school-aged children: a general population study in the Netherlands.

BACKGROUND: Health related quality of life is the functional effect of a medical condition and/or its therapy upon a patient, and as such is particularly suitable for describing the general health of children. The objective of this study was to identify and confirm potential determinants of health-related quality of life in children aged 4-11 years in the general population in the Netherlands. Understanding such determinants may provide insights into more targeted public health policy. METHODS: As part of a population based cross sectional study, the Child Health Questionnaire (CHQ) Parental Form 28 was used to measure health-related quality of life in school-aged children in a general population sample. Parents of 10,651 children aged 4-11 years were interviewed from January 2001 to December 2009. RESULTS: Multivariate and regression analyses demonstrated a declined CHQ Physical Summary score for children who had >1 conditions, disorders or acute health complaints and who were greater consumers of healthcare; children with a non-western immigrant background; and children whose parents did not work. Lower CHQ Psychosocial Summary score was reported for children who had >1 conditions, disorders or acute health complaints, boys, children of single parents and obese children. CONCLUSION: The best predictors of health-related quality of life are variables that describe use of health care and the number of disorders and health complaints. Nonetheless, a number of demographic, socio-economic and family/environmental determinants contribute to a child's health-related quality of life as well.

Factor structure of the Child Health Questionnaire Parent Form-50 and predictors of health-related quality of life in children with epilepsy.

PURPOSE: The present study investigated the higher-order summary factor structure of the Child Health Questionnaire Parent Form-50 (CHQ) in a sample of children with new-onset epilepsy. The secondary aim was to identify risk factors predicting health-related quality of life (HRQL) 24 months post-diagnosis. METHODS: Data came from the Health-related Quality of Life in Children with Epilepsy Study (HERQULES, N = 374), a multi-site study documenting HRQL among children with epilepsy from diagnosis through 24 months. Confirmatory factor analysis was used to determine goodness of fit between the original structure of the CHQ and HERQULES data. Multiple regression was used to identify risk factors at diagnosis for HRQL at 24 months. RESULTS: The models demonstrated good fit: baseline: CFI = 0.945; TLI = 0.941; WRMR = 1.461; RMSEA = 0.058; 24 months: CFI = 0.957; TLI = 0.954; WRMR = 1.393; RMSEA = 0.055. Factor loadings were high and no cross-loadings observed (first order: λ = 0.27-0.99, 0.24-0.98; second order: λ = 0.69-0.86, 0.54-0.92; p < 0.001 for all). Controlling for HRQL at diagnosis, predictors for better 24-month HRQL were as follows: physical health: fewer cognitive problems (p = 0.023) and parents with fewer depressive symptoms (p = 0.049); psychosocial health: older parent age (p = 0.043), fewer behavior problems (p = 0.004), and families with better functioning (p = 0.008) and fewer demands (p = 0.009). CONCLUSIONS: The CHQ higher-order summary factor structure was replicated in a sample of children with new-onset epilepsy, and child and family risk factors at diagnosis were found to predict HRQL 24 months post-diagnosis. These findings suggest it is possible to identify at-risk children early in the illness process and provide impetus for adopting family-centered care practices.

Impaired parent-reported health-related quality of life of underweight and obese children at elementary school entry.

PURPOSE: Examine the health-related quality of life of 5-6-year-old underweight, overweight and obese children. METHODS: Our cross-sectional study included 3,227 parent-child dyads from the "Be active, eat right" study. Parents completed questionnaires regarding child and parental characteristics. Health-related quality of life of the child was measured using the Child Health Questionnaire Parent Form 28. Children were classified normal weight, overweight, obese, severely obese, and underweight according to the international age and gender BMI cutoff points. Bootstrap analyses were performed for general linear models corrected for potential confounding variables. RESULTS: Severely obese children (ß, -2.60; 95% CI, -4.80 to -0.57, p < 0.01) and underweight children (ß, -1.11; 95% CI, -1.85 to -0.39, p < 0.01) had lower parent-reported scores on the physical summary scale. On the physical functioning profile scale parents of overweight and severely obese children also reported statistically significant lower scores (p < 0.05 and p < 0.01, respectively).There were no significant differences regarding the psychosocial summary scale scores between the different weight categories. CONCLUSION: Underweight and overweight children experience impaired health-related quality of life on the physical functioning domain. Physicians, teachers and parents should be aware of the possible negative impact on health-related quality of life in underweight and overweight 5-6-year-old children.

Health-related quality of life of infants from ethnic minority groups: the Generation R Study.

PURPOSE: To assess whether the health-related quality of life of infants from ethnic minority groups differs from the health-related quality of life of native Dutch infants and to evaluate whether infant health and family characteristics explain the potential differences. METHODS: We included 4,506 infants participating in the Generation R Study, a longitudinal birth cohort. When the child was 12 months, parents completed the Infant Toddler Quality of Life Questionnaire (ITQOL); ITQOL scale scores in each ethnic subgroup were compared with scores in the Dutch reference population. Influence of infant health and family characteristics on ITQOL scale scores were evaluated using multivariate regression models. RESULTS: Infants from ethnic minority groups presented significantly lower ITQOL scale scores compared to the Dutch subgroup (e.g., Temperament and Moods scale: median score of Turkish subgroup, 70.8 (IQR, 15.3); median score of Dutch subgroup, 80.6 (IQR, 13.9; P < 0.001)). Infant health and family characteristics mediated an important part of the association between the ethnic minority status and infant health-related quality of life. However, these factors could not fully explain all the differences in the ITQOL scale scores. CONCLUSIONS: Parent-reported health-related quality of life is lower in infants from ethnic minority groups compared to native Dutch infants, which could partly be explained by infant health and by family characteristics.

The impact of preschool wheezing patterns on health-related quality of life at age 4 years.

We assessed whether dynamic preschool wheezing patterns affect health-related quality of life (HRQOL) at age 4 years. The study included 3878 children participating a prospective cohort study. Information on preschool wheezing was obtained by questionnaire and children were categorised into: never, early, late and persistent wheezing. At age 4 years HRQOL was measured using the Child Health Questionnaire (CHQ). Persistent wheezing was associated with reduced scores for nine out of 13 CHQ scales. No differences in psychosocial CHQ scores (p>0.05), but lower physical CHQ scores were found in children with late and persistent wheezing, compared to children who never wheezed (p<0.001). Mean scores on general health perceptions were, respectively, eight and 12 points lower (on a 0-100 scale) in children with late and persistent wheezing (p<0.001), and children with one to three episodes and four or more episodes of wheezing in the fourth year respectively scored seven and 24 points lower (p<0.001), compared to children who never wheezed. Persistent wheezing during preschool age independently affects a child's HRQOL, particularly general health perceptions and physical domains at age 4 years. HRQOL was more affected by frequent wheezing episodes in the fourth year of life, rather than by the duration of wheezing at age 0-4 years.

Parent-reported health outcomes in infants/toddlers: measurement properties and clinical validity of the ITQOL-SF47.

PURPOSE: To derive and evaluate a shorter infant/toddler quality of life questionnaire (ITQOL) for use in clinical care. METHODS: Stepwise regression, factor analysis, and item-scaling principles were used to derive and guide item selection using data from a large general sample in the Netherlands (n = 5,211) and a "wheezing illness" sample (n = 138). Item internal consistency, discriminant validity, and floor and ceiling effects were evaluated using a general Dutch sample (Salland Region n = 410) and two clinical samples: Functional abdominal pain (FAP, n = 81) and Burn (n = 194). Reliabilities were estimated using Cronbach's alpha. Relative precision (RP), the ability to distinguish between clinical subgroups, was computed by comparing the proportion of variance explained by the short-form scales versus respective full-length scales. RESULTS: The ITQOL was reduced from 97 to 47 items. Median alpha coefficients were 0.77 Salland sample, 0.76 (FAP), and 0.84 (Burn). Ninety-one to 100 % scaling successes for item discriminant validity were observed for 21 of 24 tests (8 scales, 3 samples). Floor effects were not observed; some ceiling effects were detected. RP estimates in the Salland sample ranged from 2.40 (physical abilities) to 0.58 (temperament and moods). RP estimates in the FAP sample were 0.85 (bodily pain), 1.36 (temperament and moods) and 1.62 (parental-impact emotional) and for Burn, 1.51 (temperament and moods) and 0.59 (general health perceptions). CONCLUSION: The ITQOL-SF47 is reliable and valid and exceeds item-level scaling criteria.

Asthma-like symptoms in the first year of life and health-related quality of life at age 12 months: the Generation R study.

PURPOSE: This study compares HRQOL among subgroups of infants with asthma-like symptoms to a subgroup without such symptoms and examines independent associations between asthma-like symptoms during the first year of life and HRQOL at age 12 months. METHODS: Our study sample included 5,000 infants participating in the Generation R study. Their parents completed structured questionnaires to obtain information on asthma-like symptoms, HRQOL, infants', and maternal characteristics. Asthma-like symptoms were defined according to the number of positive answers to 12 items on lower respiratory symptoms. HRQOL was measured using the ITQOL. Higher scores indicated better HRQOL. RESULTS: Infants with asthma-like symptoms had significantly lower HRQOL scores for all ITQOL scales. Among the subgroup with severe symptoms (4% of the infants), relevant deficits in HRQOL were observed for most ITQOL scales, particularly for General Health, Bodily Pain, and Family Activities (effect sizes ≥ 0.8). In multivariate linear models, asthma-like symptoms were independently associated with 6 ITQOL scales. The population attributable risks were especially high for Family Activities, General Health, Parental Emotional, and Parental Time. CONCLUSIONS: Asthma-like symptoms during the first year of life are associated with impaired quality of life at age 12 months. At population level, asthma-like symptoms were associated with lower HRQOL, regardless of symptom severity.

Time course and predictors of health-related quality of life improvement and medication satisfaction in children diagnosed with attention-deficit/hyperactivity disorder treated with the methylphenidate transdermal system.

OBJECTIVE: The aim of this study was to evaluate the time course and predictors of improvement in health-related quality of life (HRQL) and medication satisfaction in children diagnosed with attention-deficit/hyperactivity disorder (ADHD) and treated with the methylphenidate transdermal system (MTS). METHODS: Temporal relationships between ADHD symptoms, medication satisfaction, and HRQL measures were examined via latent growth curve, structural path, and growth mixture models. RESULTS: Higher levels of medication satisfaction at the end of titration predicted greater increases in family HRQL (p=0.004) and, to a lesser extent, child HRQL (p=0.068) throughout the study. At 4 of 6 (p<0.05) and 5 of 6 (p<0.10) contemporaneous time points, ADHD symptoms predicted child HRQL. At 2 of 6 (p<0.05) and 3 of 6 (p<0.10) contemporaneous time points, ADHD symptoms predicted family HRQL. ADHD did not predict child or family HRQL improvements at subsequent time points. A uniform pattern of change for child HRQL was noted, with most HRQL change following the pattern of symptom change during titration. Three distinct patterns of change were noted for family HRQL. CONCLUSIONS: In most cases, medication satisfaction, ADHD symptoms, and HRQL improved simultaneously, suggesting that HRQL was not a delayed response to improvement in symptoms. Children showed a uniform pattern of improvement in HRQL that followed symptom change; three distinct patterns of change were found for improvement in family HRQL.

The Generation R study: a candidate gene study and genome-wide association study (GWAS) on health-related quality of life (HRQOL) of mothers and young children.

AIM: The aim of this paper is to describe the Generation R study as a template that enables candidate gene study and genome-wide association study regarding health-related quality of life (HRQOL) of mothers and their young children. METHODS: Generation R is a population-based prospective cohort study from fetal life onwards in Rotterdam, The Netherlands. Children were born in 2002-2006. Blood from mothers and placenta cord blood were sampled. Mothers' HRQOL was measured 5 times during pregnancy and after birth using SF-12 and EQ-5D. Children's HRQOL was measured 5 times between age 1 and 5/6 years using Infant-Toddler Quality Of Life questionnaire (ITQOL), Health Status Classification System PreSchool (HSCS-PS) and Child Health Questionnaire Parent Form 28 items (CHQ-PF28), respectively. RESULTS: DNA is available for 8,055 mothers and 5,908 children. Genotyping of various candidate genes and a genome-wide association (GWA) scan (Illumina 610K) of child DNA were done. A template for gene-HRQOL analyses is provided. We start with candidate gene study on HRQOL of mothers and children. Gene-environment interaction and interaction with medical indicators of health status will be explored. Next, GWA study on HRQOL will be performed. CONCLUSIONS: Gaining insight into the determinants of HRQOL is essential to assisting efforts in health policy and clinical application to improve well-being and health. In the future, it might be possible to complement HRQOL assessments by examinations of genetic markers. Strengths and weaknesses of the Generation R study are discussed.

Improvements in executive function correlate with enhanced performance and functioning and health-related quality of life: evidence from 2 large, double-blind, randomized, placebo-controlled trials in ADHD.

OBJECTIVE: To assess whether improvement in executive function correlates with perceived improvement in health-related quality of life (HRQOL) in adults with attention-deficit/hyperactivity disorder (ADHD) who are treated with stimulant medication. METHODS: We collected data on executive function and HRQOL using the Brown Attention-Deficit Disorder Scale (BADDS) and the ADHD Impact Module-Adult (AIM-A) during 2 large, randomized, double-blind, placebo-controlled trials evaluating the use of the triple-bead mixed amphetamine salt (MAS) SPD465 as stimulant pharmacotherapy for ADHD. BADDS and AIM-A data were collected at baseline and at study endpoint, and changes from baseline were assessed. Correlation coefficients for BADDS and AIM-A were calculated at baseline and endpoint for each study. RESULTS: The change from baseline in BADDS total score was -14.8 ± 24.49 in Study 1 (dose optimization strategy) and -26.1 ± 28.54 in Study 2 (forced-dose titration strategy), demonstrating substantial reported improvement in executive function. In both studies, improvement was reported for all 5 BADDS clusters at study endpoint versus baseline. While improvement was reported for all 6 AIM-A scales at study endpoint versus baseline, the most substantial change from baseline in AIM-A scores over the course of the studies was in Performance and Function (Study 1, +15.2 ± 25.68; Study 2, +26.3 ± 28.17). There was significant correlation of the BADDS total score with the AIM-A Performance and Function scale (Study 1, -0.6723; Study 2, -0.6982), and strong correlations were observed between each of the 5 individual clusters of BADDS and AIM-A performance and function in both studies. CONCLUSIONS: Reported improvement in executive function correlates with reported improvement in HRQOL as assessed in 2 independent clinical trials in which participants received either placebo or triple-bead MAS for the treatment of ADHD. In particular, there were strong correlations between all 5 individual clusters of BADDS and the AIM-A Performance and Function scale.

Moderators and mediators of symptoms and quality of life outcomes in an open-label study of adults treated for attention-deficit/hyperactivity disorder.

OBJECTIVE: The Quality of Life, Effectiveness, Safety, and Tolerability (QU.E.S.T.) study was designed to evaluate effectiveness of long-acting amphetamines in adults with attention-deficit/hyperactivity disorder (ADHD) in community practice settings. This article reports moderators and mediators of symptoms and quality of life outcomes. METHOD: This was an open-label study of 725 adults with DSM-IV-diagnosed ADHD, treated with mixed amphetamine salts extended release and followed for up to 8 months. Multiple regressions were used to determine if patient moderators impact response in ADHD symptoms and how ADHD symptoms and medication satisfaction mediate quality of life. The study was conducted from December 2003 to December 2004. RESULTS: Amphetamine treatment of ADHD resulted in a robust and enduring symptom response. Patient characteristics such as age, female gender, severity of illness, and treatment-naive status moderate improved symptom outcome. Symptom change and satisfaction with medication independently mediate change in mental but not physical quality of life outcomes. There is no time lag between changes in symptoms and improved quality of life. Attention is a stronger mediator of ADHD-specific quality of life outcomes than disruptive behavior. CONCLUSIONS: If symptoms and quality of life improve simultaneously, improvement in quality of life can be understood as more than just a downstream, secondary effect of symptom remission. Satisfaction with medication is a direct measure of the complex interplay of symptom change, tolerability, and patient perception of treatment that predicts self-report of quality of life benefits. Although the disruptive symptoms of ADHD are more obvious, adults self-report that attention has greater impact.

ADHD-hyperactive/impulsive subtype in adults.

This is the first study to evaluate ADHD-hyperactive/impulsive subtype in a large clinical sample of adults with ADHD. The Quality of Life, Effectiveness, Safety and Tolerability (QuEST) study included 725 adults who received clinician diagnoses of any ADHD subtype. Cross-sectional baseline data from 691 patients diagnosed with the hyperactive/impulsive (HI), inattentive (IA) and combined subtypes were used to compare the groups on the clinician administered ADHD-RS, clinical features and health-related quality of life. A consistent pattern of differences was found between the ADHD-I and combined subtypes, with the combined subtype being more likely to be diagnosed in childhood, more severe symptom severity and lower HRQL. Twenty-three patients out of the total sample of 691 patients (3%) received a clinician diagnosis of ADHD-hyperactive/impulsive subtype. Review of the ratings on the ADHD-RS-IV demonstrated, however, that this group had ratings of inattention comparable to the inattentive group. There were no significant differences found between the ADHD-HI and the other subtypes in symptom severity, functioning or quality of life. The hyperactive/impulsive subtype group identified by clinicians in this study was not significantly different from the rest of the sample. By contrast, significant differences were found between the inattentive and combined types. This suggests that in adults, hyperactivity declines and inattention remains significant, making the hyperactive/impulsive subtype as defined by childhood criteria a very rare condition and raising questions as to the validity of the HI subtype in adults.

Does switching from oral extended-release methylphenidate to the methylphenidate transdermal system affect health-related quality-of-life and medication satisfaction for children with attention-deficit/hyperactivity disorder?

BACKGROUND: To evaluate health-related quality of life (HRQL) and medication satisfaction after switching from a stable dose of oral extended-release methylphenidate (ER-MPH) to methylphenidate transdermal system (MTS) via a dose-transition schedule in children with attention-deficit/hyperactivity disorder (ADHD). METHODS: In a 4-week, multisite, open-label study, 171 children (164 in the intent-to-treat [ITT] population) aged 6-12 years diagnosed with ADHD abruptly switched from a stable dose of oral ER-MPH to MTS nominal dosages of 10, 15, 20, and 30 mg using a predefined dose-transition schedule. Subjects remained on the scheduled dose for the first week, after which the dose was then titrated to an optimal effect. The ADHD Impact Module-Children (AIM-C), a disease-specific validated HRQL survey instrument measuring child and family impact, was used to assess the impact of ADHD symptoms on the lives of children and their families at baseline and study endpoint. Satisfaction with MTS use was assessed via a Medication Satisfaction Survey (MSS) at study endpoint. Both the AIM-C and MSS were completed by a caregiver (parent/legally authorized representative). Tolerability was monitored by spontaneous adverse event (AE) reporting. RESULTS: AIM-C child and family HRQL mean scores were above the median possible score at baseline and were further improved at endpoint across all MTS doses. Similar improvements were noted for behavior, missed doses, worry, and economic impact AIM-C item scores. Overall, 93.8% of caregivers indicated a high level of satisfaction with their child's use of the study medication. The majority of treatment-emergent AEs (> 98%) were mild to moderate in intensity, and the most commonly reported AEs included headache, decreased appetite, insomnia, and abdominal pain. Seven subjects discontinued the study due to intolerable AEs (n = 3) and application site reactions (n = 4). CONCLUSION: This study demonstrates that MTS, when carefully titrated to optimal dose, may further improve child and family HRQL, as well as behavioral, medication worry, and economic impact item scores, as measured by the AIM-C in subjects switching to MTS from a stable dose of routinely prescribed oral ER-MPH after a short treatment period. Furthermore, following the abrupt conversion from oral ER-MPH to MTS, the majority of caregivers reported being highly satisfied with MTS as a treatment option for their children with ADHD. TRIAL REGISTRATION: NCT00151983.

HRQL and medication satisfaction in children with ADHD treated with the methylphenidate transdermal system.

OBJECTIVE: To evaluate the impact of methylphenidate transdermal system (MTS) on health-related quality of life (HRQL) and medication satisfaction in children with attention-deficit/hyperactivity disorder (ADHD) as well as to identify potential moderators of HRQL and medication satisfaction. RESEARCH DESIGN AND METHODS: Children aged 6-12 years diagnosed with ADHD were enrolled (N = 128) and 115 children completed the study. MTS dose was optimized over 5 weeks using 10-, 15-, 20-, or 30-mg patches worn for 9 hours. The efficacy of 4- and 6-hour wear times was then assessed in an analog classroom setting in a randomized, placebo-controlled, double-blind, three-way crossover design study. MAIN OUTCOME MEASURES: The ADHD Impact Module-Children (AIM-C), a validated HRQL instrument, was used to assess the impact of ADHD symptoms on children and their families. Satisfaction with MTS use was assessed via a Medication Satisfaction Survey (MSS). A parent or legally appointed representative (LAR) completed the measures. Tolerability was monitored by spontaneous adverse event reporting. RESULTS: Mean scores on AIM-C child and family HRQL scales improved from baseline to endpoint across all MTS doses and the magnitude of improvement increased with time from baseline. Improvement was noted for behavior, missed doses, worry, and economic impact AIM-C scores. Overall, parents/LARs indicated a high level of satisfaction with their child's use of MTS (Visit 7 [92.1%]; Visit 10 [89.1%]). Most treatment-emergent adverse events (TEAEs) were mild to moderate. The most frequent TEAEs included decreased appetite (28%), headache (21%), insomnia (20%), and abdominal pain (12%). CONCLUSIONS: At study endpoint, MTS treatment of ADHD was associated with robust improvement in child and family HRQL, key economic impact items, and overall medication satisfaction with the effectiveness and ease of use of MTS as an ADHD treatment. Also, the majority of MTS TEAEs were mild to moderate in severity. Limitations of this study included the potential for a significant halo effect when measuring HRQL and medication satisfaction as well as the uncertainty regarding whether the improvements seen over this relatively short study duration would be sustainable long term. CLINICAL TRIAL REGISTRATION: #NCT00151970.