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OBJECTIVE Children with autism spectrum disorder (ASD) face challenges across many functional domains. A tool that gathers relevant clinical information before visits, emphasizing symptoms that are likely to change over development and inform clinical interventions, could improve health care quality, allowing for more patient-centered and efficient care. This study evaluated the clinical utility and preliminary psychometrics of the ASD Parent Report for Outcome Monitoring (ASD-PROM), a web-based measure assessing competence in core features of ASD, along with the breadth of concerns and comorbidities that frequently co-occur with ASD. METHODS An interdisciplinary team drafted the ASD-PROM and made iterative revisions based on parent feedback. Parents of 62 children completed the ASD-PROM before their autism-specialty clinical visit, 53 completed the ASD-PROM twice, and 48 completed the Vineland Adaptive Behavior Scales, Second Edition (Vineland-II) concurrently. Parents (n = 25) and clinicians (n = 13) completed postvisit surveys to assess utility (phase 1). The ASD-PROM was then released for general clinical use (phase 2). RESULTS On a Likert scale (1 = very poorly, 10 = very well), parents found that ASD-PROM items described their child's abilities well (median = 8.0; interquartile range [IQR]: 7.0-9.5) and had a positive effect on care (median = 8.0; IQR: 7.0-10.0). Clinicians found the ASD-PROM effective in assessing parent-reported patient abilities (median = 9.0, IQR: 7.0-9.0) and felt the ASD-PROM helped make their care more patient-centered and efficient (both median = 8.0, IQR: 6.0-9.0). Two-week test-retest reliability was acceptable (0.95). ASD-PROM scores correlated positively with scores from similar domains on the Vineland-II (Pearson r 0.30-0.50, medium to large effects). CONCLUSION The ASD-PROM is a freely available tool to gather information on developmental and behavioral functioning in children with ASD before autism-specialty clinical visits. Clinical utility and preliminary psychometrics are promising, although limitations (including a low response rate during clinical use and a need for additional in-depth assessments and potential resulting modifications to the tool) remain to be addressed. Ultimately, the ASD-PROM may help promote patient-centered and efficient care for children across a wide range of ages and developmental levels.

Objectives To describe telephone interview completion rates among 12-month cardiac arrest survivors enrolled in the Therapeutic Hypothermia after Pediatric Cardiac Arrest In-Hospital and Out-of-Hospital trials, identify key characteristics of the completed follow-up interviews at both 3- and 12-month postcardiac arrest, and describe strategies implemented to promote follow-up. Setting Centralized telephone follow-up interviews. Design Retrospective report of data collected for Therapeutic Hypothermia after Pediatric Cardiac Arrest trials, and summary of strategies used to maximize follow-up completion. Patients Twelve-month survivors (n = 251) from 39 Therapeutic Hypothermia after Pediatric Cardiac Arrest PICU sites in the United States, Canada, and United Kingdom. Interventions Not applicable. Measurements and main results The 3- and 12-month telephone interviews included completion of the Vineland Adaptive Behavior Scales, Second Edition. Vineland Adaptive Behavior Scales, Second Edition data were available on 96% of 3-month survivors (242/251) and 95% of 12-month survivors (239/251) with no differences in demographics between those with and without completed Vineland Adaptive Behavior Scales, Second Edition. At 12 months, a substantial minority of interviews were completed with caregivers other than parents (10%), after calls attempts were made on 6 or more days (18%), and during evenings/weekends (17%). Strategies included emphasizing the relationship between study teams and participants, ongoing communication between study team members across sites, promoting site engagement during the study's final year, and withholding payment for work associated with the primary outcome until work had been completed. Conclusions It is feasible to use telephone follow-up interviews to successfully collect detailed neurobehavioral outcome about children following pediatric cardiac arrest. Future studies should consider availability of the telephone interviewer to conduct calls at times convenient for families, using a range of respondents, ongoing engagement with site teams, and site payment related to primary outcome completion.

Objective:To determine safety and perform a preliminary assessment of dose-dependent efficacy of dextromethorphan in normalizing electrographic spikes, clinical seizures, and behavioral and cognitive functions in girls with Rett syndrome.Methods:We used a prospective randomized, open-label trial in fast metabolizers of dextromethorphan to examine the effect of dextromethorphan on core clinical features of Rett syndrome. Interictal spike activity and clinical seizures were determined using EEG and parent reporting. Cognitive data were obtained using the Mullen Scales of Early Learning and Vineland Adaptive Behavior Scales, while behavioral data were obtained from parent-completed checklists, the Aberrant Behavior Checklist–Community Version, and the Screen for Social Interaction. Anthropometric data were obtained according to the National Health and Nutrition Examination Survey. The Rett Syndrome Severity Scale provided a clinical global impression of the effect of dextromethorphan on clinical severity.Results:Dextromethorphan is safe for use in 3- to 15-year-old girls with Rett syndrome. Thirty-five girls were treated with 1 of 3 doses of dextromethorphan over a period of 6 months. Statistically significant dose-dependent improvements were seen in clinical seizures, receptive language, and behavioral hyperactivity. There was no significant improvement in global clinical severity as measured by the Rett Syndrome Severity Scale.Conclusions:Dextromethorphan is a potent noncompetitive antagonist of the NMDA receptor channel that is safe for use in young girls with Rett syndrome. Preliminary evidence suggests that dextromethorphan may improve some core features of Rett syndrome.Classification of evidence:This study provides Class IV evidence that dextromethorphan at various doses does not change EEG spike counts over 6 months, though precision was limited to exclude an important effect.

OBJECTIVES To describe family burden among caregivers of children who survived out-of-hospital cardiac arrest and who were at high risk for neurologic disability and examine relationships between family burden, child functioning, and other factors during the first year post arrest. DESIGN Secondary analysis of data from the Therapeutic Hypothermia after Pediatric Cardiac Arrest Out-of-Hospital trial. SETTING Thirty-six PICUs in the United States and Canada. PATIENTS Seventy-seven children recruited to the Therapeutic Hypothermia after Pediatric Cardiac Arrest Out-of-Hospital trial who had normal prearrest neurologic functioning and were alive 1 year post arrest. INTERVENTIONS Family burden was assessed using the Infant Toddler Quality of Life Questionnaire for children less than 5 years old and the Child Health Questionnaire for children 5 years old or older at baseline (reflecting prearrest status), 3 months, and 12 months post arrest. Child functioning was assessed using the Vineland Adaptive Behavior Scale II, the Pediatric Overall Performance Category, and Pediatric Cerebral Performance Category scales and caregiver perception of global functioning. MEASUREMENTS AND MAIN RESULTS Fifty-six children (72.7%) were boys, 48 (62.3%) were whites, and 50 (64.9%) were less than 5 years old prior to out-of-hospital cardiac arrest. Family burden at baseline was not significantly different from reference values. Family burden was increased at 3 and 12 months post arrest compared with reference values (p < 0.001). Worse Pediatric Overall Performance Category and Pediatric Cerebral Performance Category, lower adaptive behavior, lower global functioning, and higher family burden all measured 3 months post arrest were associated with higher family burden 12 months post arrest (p < 0.05). Sociodemographics and prearrest child functioning were not associated with family burden 12 months post arrest. CONCLUSIONS Families of children who survive out-of-hospital cardiac arrest and have high risk for neurologic disability often experience substantial burden during the first year post arrest. The extent of child dysfunction 3 months post arrest is associated with family burden at 12 months.

Although functional impairment is very common among patients with major depressive disorder (MDD) and is a necessary diagnostic criterion for MDD, clinicians have not directed their attention beyond the mood symptoms of MDD. We aimed to multi-dimensionally assess the functional abilities of patients with MDD using a standardized scale of adaptive function reported by caregivers or parents to identify the nature and degree of functional impairment in patients with MDD.This study was conducted in 40 depressive patients aged 19 to 60 years. Patients were screened according to the DSM-IV-TR and a Hamilton Depression Rating Scale score ≥8. We administered the parent/caregiver rating form of the Korean Vineland Adaptive Behavior Scale to assess functional outcomes in the patients.Patients with MDD showed significant differences in both global and domain-specific functional abilities compared to those of the normal group (all t > -6.35, P 4.30, P < .001). The number of episodes among clinical factors was negatively correlated with overall adaptive functioning (r = -0.32, P < .05) and expressive communication (r = -0.42, P < .01).Our present study replicates existing evidence showing impairment in both broad and various functional areas in patients with MDD, suggesting the importance of quantitatively assessing functional outcomes and acquiring information about functioning from informants other than patients.

Advances in medical treatment have prolonged the lives of children with Hurler syndrome or mucopolysaccharidosis I requiring increased attention to the assessment of their long-term outcomes and functional abilities. Adaptive functions are critical for understanding functional outcomes after treatment and developing focused interventions. We investigated the development of various adaptive functions in children who have had hematopoietic stem cell transplant (HSCT) for Hurler syndrome and risk factors that are associated with the development of these functions. We examined the development of 41 children who had 3 or more Vineland Adaptive Behavior Scales records assessed before and after transplant. Communication, daily living skills, socialization, and motor functions were measured. While standard scores decline over time, development of skills continue with a slower than average rate compared with peers. A cross-sectional nontransplanted comparison group showed more deficits after age 2 years than the transplanted group. In contrast to cognitive ability, age at transplant was not significantly associated with ultimate adaptive level. Baseline cognitive level before HSCT and growth of cognition after HSCT were associated with adaptive functions especially for communication and daily living skills. Socialization was predicted by cumulative medical risk factors, likely due to restricted social exposure in children with complicated transplant courses. Overall, measurement of adaptive behaviors demonstrated that HSCT allows long-term slow improvement of functional outcomes for children with Hurler syndrome. Children with Hurler syndrome with good cognitive levels before HSCT and continued growth of cognition after HSCT show good adaptive functions. Although cognitive and orthopedic problems as well as medical complications limit adaptive ability, identifying these problems early allow beneficial targeted interventions.

Children undergoing surgery with infant-onset epilepsy were classified into those with medically refractory infantile spasms (IS), successfully treated IS, and no IS history, and the groups were compared for pre- and postsurgery clinical and Vineland Adaptive Behavior Scale (VABS) developmental quotients (DQ). Children without an IS history were older at surgery and had longer epilepsy durations than those with IS despite similar substrates, surgeries, and seizure frequencies. In all groups, better postsurgery VABS-DQ scores were associated with early surgical intervention indicating that infant-onset epilepsy patients with or without IS are at risk for seizure-induced encephalopathy.

Objective: To evaluate language and cognitive outcomes in elementary school children with a prior preschool diagnosis of developmental language impairment (DLI). Design/methods: A cohort of preschool children, consecutively diagnosed with isolated language impairment, was reassessed in elementary school. Measures used were the communication domains of the Battelle Developmental Inventory (BDI) and the Vineland Adaptive Behavior Scale, Peabody Picture Vocabulary and Expressive One Word Picture Vocabulary. Cognition was assessed using the BDI cognitive domain. Language impairment was defined as performance more than 1.25 SD below normative means on a language measure. Specific language impairment (SLI) was defined as language impairment concurrent with a cognitive score not more than one SD below the normative mean.Results: A total of 43/70 (61%) children were reassessed. Mean age in preschool was 3.6 ± 0.7 years and 7.4 ± 0.7 years at follow up. A total of 36/43 (84%) showed persistent language impairment. The mean BDI cognitive domain score was 80.0 ± 14.2 (15/42 below −2 SDs). Only 11/42 (26%) children met current research criteria for SLI, 24/42 (57%) had language impairment but had cognitive scores more than one SD below normative means, and 4/42 (10%) had normal language and cognitive skills. No factors could be identified at intake that predicted language outcome using univariate or multivariate analysis.Conclusions: While a preschool diagnosis of developmental language impairment predicted persisting language impairment, the specificity of this impairment did not persist. This suggests either undiagnosed cognitive impairment in preschool children with apparently isolated language impairment or an evolving profile of more global developmental impairment.

Parenting young children with pervasive developmental disorder engenders unique sustained stresses, which have been termed "burden of care." One specific source of stress for parents is the children's uneven developmental progress, in which delays and accelerations of adaptive functioning may exist side by side. This paper proposes a clinical method that may be incorporated into periodic child psychiatric consultations with parents of young children who have high-functioning autism or Asperger's disorder. Using a semistructured technique, the clinician reviews with the parents the details of the child's recent developmental course and attempts to identify emergent skills that may serve to preview upcoming developmental issues and gains. This method is aimed at enhancing parental abilities to track and anticipate developmental progress and the resultant shifts in the parent-child relationship, in order to reduce one source of sustained parental stress. Case illustrations of children ages 2-8 years old are discussed in light of recent literature on adaptive issues in families of individuals with a chronic medical or psychiatric condition and, specifically, families of children with pervasive developmental disorder.

Emotional reactions were examined cross-sectionally over the 1-to 6-year period in mothers of children with Down syndrome (N = 25) and with predominantly motor impairments (N = 20). Mothers rated their degree of concern about 16 early milestones and four events, and were also interviewed with the Vineland Adaptive Behavior Scales. Although milestones occurring during the first year caused higher levels of maternal concern than did later-occurring ones, concern was also evident for some later milestones. Mothers of Down syndrome and of motor-impaired children were equally concerned about motor milestones, but mothers in the Down syndrome group were more concerned about developments in communication. The two groups modulated their maternal concern based on different aspects of the child's communication skills. The timing, specificity, and sensitivity of maternal reactions identified in this study are discussed in relation to the unidirectional, time-bound model of “maternal mourning.” J Dev Behav Pediatr 13:118–123, 1992.