A two-step multidisciplinary approach to treat recurrent esophageal strictures in children with epidermolysis bullosa dystrophica
Male; Treatment Outcome; Follow-Up Studies; Child; Humans; Adolescent; Female; Child Preschool; Infant; Recurrence; Combined Modality Therapy; Gastrostomy; Dilatation/methods; Epidermolysis Bullosa Dystrophica/complications; Esophageal Stenosis/etiology/therapy; feeding difficulties; Epidermolysis Bullosa; surgical intervention; orthograde balloon dilation; retrograde dilation; growth improvement; nutrition
In children with severe generalized recessive dystrophic epidermolysis bullosa (RDEB), esophageal scarring leads to esophageal strictures with dysphagia, followed by malnutrition and delayed development. We describe a two-step multidisciplinary therapeutic approach to overcome malnutrition and growth retardation. In Step 1, under general anesthesia, orthograde balloon dilation of the esophagus is followed by gastrostomy creation using a direct puncture technique. In Step 2, further esophageal strictures are treated by retrograde dilation via the established gastrostomy; this step requires only a short sedation period. A total of 12 patients (median age 7.8 years, range 6 weeks to 17 years) underwent successful orthograde balloon dilation of esophageal strictures combined with direct puncture gastrostomy. After 12 and 24 months in 11 children, a substantial improvement of growth and nutrition was achieved (body mass index [BMI] standard deviation score [SDS] + 0.59 and + 0.61, respectively). In one child, gastrostomy was removed because of skin ulcerations after 10 days. Recurrent esophageal strictures were treated successfully in five children. The combined approach of balloon dilation and gastrostomy is technically safe in children with RDEB, and helps to promote catch-up growth and body weight. In addition, recurrent esophageal strictures can be treated successfully without general anesthesia in a retrograde manner via the established gastrostomy.
Vowinkel T; Laukoetter M; Mennigen R; Hahnenkamp K; Gottschalk A; Boschin M; Frosch M; Senninger N; Tubergen D
Endoscopy
2015
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1055/s-0034-1391308" target="_blank" rel="noreferrer noopener">10.1055/s-0034-1391308</a>
Technological aids to support choice strategies by three girls with Rett syndrome
tone and motor problems; Rett syndrome; physical intervention; assistive technology; photocells; interface; personal computer; stereotyped movements
This study was aimed at extending the use of assistive technology (i.e., photocells, interface and personal computer) to support choice strategies by three girls with Rett syndrome and severe to profound developmental disabilities. A second purpose of the study was to reduce stereotypic behaviors exhibited by the participants involved (i.e., body rocking, hand washing and hand mouthing). Finally, a third goal of the study was to monitor the effects of such program on the participants' indices of happiness. The study was carried out according to a multiple probe design across responses for each participant. Results showed that the three girls increased the adaptive responses and decreased the stereotyped behaviors during intervention phases compared to baseline. Moreover, during intervention phases, the indices of happiness augmented for each girl as well. Clinical, psychological and rehabilitative implications of the findings are discussed.Copyright © 2014 Elsevier Ltd. All rights reserved.
Stasolla F; Perilli V; Di Leone A; Damiani R; Albano V; Stella A; Damato C
Research in Developmental Disabilities
2015
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1016/j.ridd.2014.09.017" target="_blank" rel="noreferrer noopener">10.1016/j.ridd.2014.09.017</a>
Quantifying behaviors of children with Sanfilippo syndrome: The Sanfilippo Behavior Rating Scale
Medicine; amygdala volume; autism; Behavior phenotype; Behavior rating scale; Endocrinology & Metabolism; Genetics & Heredity; kluver; management; mucopolysaccharidosis type iiia; MPSIII; Research & Experimental; Sanfilippo Syndrome; behavioral problems; trajectory; characteristics
The Sanfilippo Behavior Rating Scale (SBRS), a 68 item questionnaire, has been developed to assess the behavioral phenotype of children with Sanfilippo syndrome and its progression overtime. Fifteen scales rate orality, movement/activity, attention/self-control, emotional function including anger and fear, and social interaction. Items within scales intercorrelate; measures of internal consistency are adequate. Twelve scales are grouped into 4 abnormality clusters: Movement, Lack of fear, Social/emotional and Executive Dysfunction. A Loess age-trajectory analysis showed that Lack of Fear, Social/Emotional and Executive Dysfunction increased steadily with age; Orality and Mood/Anger/Aggression leveled off. Movement peaked around 6 years, then declined as children's excessive/purposeless actions stopped. Compared with standard scales, SBRS Movement was appropriately associated with the Vineland Motor scale; SBRS Lack of Fear had significant associations with the Autism Diagnostic Observation Schedule (ADOS), indicating a symptom overlap between Sanfilippo syndrome and autism. This suggests that reduced fearfulness may be the most salient/sensitive SBRS marker of disease progression. Volumetric MRI showed that increased Lack of Fear was significantly associated with reduced amygdala volume, consistent with our hypothesis that the behavior seen in Sanfilippo syndrome is a variant of Kluver-Bucy syndrome. Hippocampal volume loss had twice the effect on Social-Emotional Dysfunction as amygdala loss, consistent with a hippocampal role in attachment and social emotions. In conclusion, the SBRS assesses the Sanfilippo behavioral phenotype; it can measure behavior change that accompanies disease progression and/or results from treatment (C) 2015 Elsevier Inc. All rights reserved.
Shapiro E G; Nestrasil I; Ahmed A; Wey A; Rudser K R; Delaney K A; Rumsey R K; Haslett P A J; Whitley C B; Potegal M
Molecular Genetics and Metabolism
2015
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1016/j.ymgme.2015.02.008" target="_blank" rel="noreferrer noopener">10.1016/j.ymgme.2015.02.008</a>
Actigraphic investigation of circadian rhythm functioning and activity levels in children with mucopolysaccharidosis type III (Sanfilippo syndrome)
Actigraphy; Sleep; rare disease; Neurosciences & Neurology; controlled-trial; intellectual disability; disturbance; melatonin; autistic spectrum disorders; Circadian rhythms; light; Mucopolysaccharidosis type III; multiple disabilities; Sanfilippo; sleep problems; smith-magenis-syndrome; therapy; sleep disturbance/disorders; MPSIIIA; MPSIIIB; trajectory; characteristics
Background: Sleep disturbance is part of the behavioural phenotype of the rare genetic condition mucopolysaccharidosis (MPS) type III. A growing body of evidence suggests that underlying disturbance in circadian rhythm functioning may explain sleep problems within the MPS III population. Methods: Actigraphic data were recorded in eight children with MPS III over 7-10 days and compared to age-matched typically developing controls. Parameters of circadian rhythmicity and activity levels across a 24-h period were analysed. Results: Statistically and clinically significant differences between the two groups were noted. Analysis indicated that children with MPS III showed significantly increased fragmentation of circadian rhythm and reduced stability with external cues (zeitgebers), compared to controls. Average times of activity onset and offset were indicative of a phase delayed sleep-wake cycle for some children in the MPS III group. Children with MPS III had significantly higher activity levels during the early morning hours (midnight-6 am) compared to controls. Conclusions: Results are consistent with previous research into MPS III and suggest that there is an impairment in circadian rhythm functioning in children with this condition. Implications for clinical practice and the management of sleep difficulties are discussed.
Mumford R A; Mahon L V; Jones S; Bigger B; Canal M; Hare D J
Journal of Neurodevelopmental Disorders
2015
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1186/s11689-015-9126-5" target="_blank" rel="noreferrer noopener">10.1186/s11689-015-9126-5</a>
Pilot Validation of the Tuberous Sclerosis-Associated Neuropsychiatric Disorders (TAND) Checklist
psychosocial; children; Pediatrics; cognition; behavior; Neurosciences & Neurology; mental health; autism; gene; recommendations; academic; attention deficits; complex consensus conference; diagnostic-criteria; identification; neuropsychology; population; sample; TAND; tuberous sclerosis complex; behavioral problems; tuberous sclerosis; tool development; scale development
BACKGROUND: Tuberous sclerosis complex is a multisystem disorder that includes a range of tuberous sclerosis associated neuropsychiatric disorders (TAND). The lifetime prevalence rates of TAND are very high; yet surveys suggest that the majority of individuals with tuberous sclerosis never receive appropriate assessment or treatment for TAND. To aid systematic enquiry, a TAND Checklist was developed. Here, we performed pilot validation of the TAND Checklist. METHOD: Mixed methods were used across two stages. In stage 1, we gathered feedback on the Checklist from tuberous sclerosis "expert professionals" and "expert parents and caregivers." The aim was to examine face and content validity. Stage 2 involved the administration of the refined TAND Checklist to 20 parents of individuals with tuberous sclerosis concurrently with four widely used validated rating scales, to examine external validity and obtain qualitative feedback on face-to-face administration of the TAND Checklist. RESULTS: Twenty professionals and 62 parents and caregivers from 28 countries participated in the pilot. The TAND Checklist demonstrated good face and content validity with high overall mean and median scores. Qualitative analysis highlighted concerns about the likely use of the TAND Checklist, suggesting that family members and individuals with tuberous sclerosis should drive usage. Stage 2 results showed moderate-to-very good external validity across TAND domain and key subdomains. Internal consistency of domains and subdomains was acceptable to very good. Ninety-three percent of all participants (93%) reported four or more lifetime TAND behavioral difficulties. CONCLUSION: The pilot validation suggested that the TAND Checklist could provide a useful screening tool in clinical settings.
Leclezio L; Jansen A; Whittemore V H; de Vries P J
Pediatric Neurology
2015
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1016/j.pediatrneurol.2014.10.006" target="_blank" rel="noreferrer noopener">10.1016/j.pediatrneurol.2014.10.006</a>
Robotic and clinical evaluation of upper limb motor performance in patients with Friedreich's Ataxia: an observational study
children; Rehabilitation; Neurosciences & Neurology; Engineering; coordination; therapy; rating-scale; Accuracy; and Rating of Ataxia; cerebellar-ataxia; Friedreich's ataxia; Kinematics; movement; multiple-sclerosis; quantitative-evaluation; Reaching task; rehabilitation; Robot-mediated evaluation; Scale for the Assessment; smoothness; Smoothness; stroke recovery; Submovements; Upper limb; tone and motor problems; tool development; scale development; InMotion Arm Robot
Background: Friedreich's ataxia (FRDA) is the most common hereditary autosomal recessive form of ataxia. In this disease there is early manifestation of gait ataxia, and dysmetria of the arms and legs which causes impairment in daily activities that require fine manual dexterity. To date there is no cure for this disease. Some novel therapeutic approaches are ongoing in different steps of clinical trial. Development of sensitive outcome measures is crucial to prove therapeutic effectiveness. The aim of the study was to assess the reliability and sensitivity of quantitative and objective assessment of upper limb performance computed by means of the robotic device and to evaluate the correlation with clinical and functional markers of the disease severity. Methods: Here we assess upper limb performances by means of the InMotion Arm Robot, a robot designed for clinical neurological applications, in a cohort of 14 children and young adults affected by FRDA, matched for age and gender with 18 healthy subjects. We focused on the analysis of kinematics, accuracy, smoothness, and submovements of the upper limb while reaching movements were performed. The robotic evaluation of upper limb performance consisted of planar reaching movements performed with the robotic system. The motors of the robot were turned off, so that the device worked as a measurement tool. The status of the disease was scored using the Scale for the Assessment and Rating of Ataxia (SARA). Relationships between robotic indices and a range of clinical and disease characteristics were examined. Results: All our robotic indices were significantly different between the two cohorts except for two, and were highly and reliably discriminative between healthy and subjects with FRDA. In particular, subjects with FRDA exhibited slower movements as well as loss of accuracy and smoothness, which are typical of the disease. Duration of Movement, Normalized Jerk, and Number of Submovements were the best discriminative indices, as they were directly and easily measurable and correlated with the status of the disease, as measured by SARA. Conclusions: Our results suggest that outcome measures obtained by means of robotic devices can improve the sensitivity of clinical evaluations of patients' dexterity and can accurately and efficiently quantify changes over time in clinical trials, particularly when functional scales appear to be no longer sensitive.
Germanotta M; Vasco G; Petrarca M; Rossi S; Carniel S; Bertini E; Cappa P; Castelli E
Journal of Neuroengineering and Rehabilitation
2015
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1186/s12984-015-0032-6" target="_blank" rel="noreferrer noopener">10.1186/s12984-015-0032-6</a>
Rett syndrome: establishing a novel outcome measure for walking activity in an era of clinical trials for rare disorders
Rehabilitation; validity; injury; Rett syndrome; walking; disease; sample; accelerometry; accuracy; activity monitors; Outcome measure; physical activity; physical-activity; step; test-retest reliability; tone and motor problems; tool development; scale development; StepWatch
Background: Rett syndrome is a pervasive neurological disorder with impaired gait as one criterion. This study investigated the capacity of three accelerometer-type devices to measure walking activity in Rett syndrome. Methods: Twenty-six participants (mean 18 years, SD 8) wore an Actigraph, ActivPAL and StepWatch Activity Monitor (SAM) during a video-taped session of activities. Agreement was determined between step-counts derived from each accelerometer and observation. Repeatability of SAM-derived step counts was determined using pairs of one-minute epochs during which the same participant was observed to walk with the same cadence. Results: The mean difference (limit of agreement) for the Actigraph, ActivPAL and SAM were -41 (SD 33), -16 (SD 21) and -1 (SD 16) steps/min, respectively. Agreement was influenced by a device/cadence interaction (p < 0.001) with greater under-recording at higher cadences. For SAM data, repeatability of step-count pairs was excellent (intraclass correlation coefficient 0.91, 95% CI 0.79-0.96). The standard error of measurement was 6 steps/min and we would be 95% confident that a change >= 17 steps/min would be greater than within-subject measurement error. Conclusions: The capacity of the SAM to measure physical activity in Rett syndrome allows focus on participation-based activities in clinical practice and clinical trials.
Downs J; Leonard H; Jacoby P; Brisco L; Baikie G; Hill K
Disability and Rehabilitation
2015
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<a href="http://doi.org/10.3109/09638288.2014.993436" target="_blank" rel="noreferrer noopener">10.3109/09638288.2014.993436</a>
Tuberous sclerosis associated neuropsychiatric disorders (TAND) and the TAND Checklist
behavior; feeding difficulties; sleep disturbance; tuberous sclerosis; tool development; scale development; TAND checklist
BACKGROUND: Tuberous sclerosis complex is a multisystem genetic disorder with a range of physical manifestations that require evaluation, surveillance, and management. Individuals with tuberous sclerosis complex also have a range of behavioral, psychiatric, intellectual, academic, neuropsychologic, and psychosocial difficulties. These may represent the greatest burden of the disease. Around 90% of individuals with tuberous sclerosis complex will have some of these difficulties during their lifetime, yet only about 20% ever receive evaluation and treatment. The Neuropsychiatry Panel at the 2012 Tuberous Sclerosis Complex International Consensus Conference expressed concern about the significant "treatment gap" and about confusion regarding terminology relating to the biopsychosocial difficulties associated with tuberous sclerosis complex. METHODS: The Tuberous Sclerosis Complex Neuropsychiatry Panel coined the term TAND-tuberous sclerosis complex-associated neuropsychiatric disorders-to bring together these multidimensional manifestations of the disorder, and recommended annual screening for TAND. In addition, the Panel agreed to develop a TAND Checklist as a guide for screening. RESULTS: Here, we present an outline of the conceptualization of TAND, rationale for the structure of the TAND Checklist, and include the full US English version of the TAND Checklist. CONCLUSION: We hope that the unified term TAND and the TAND Checklist will raise awareness of the importance of tuberous sclerosis complex-associated neuropsychiatric disorders and of the major burden of disease associated with it, provide a shared language and a simple tool to describe and evaluate the different levels of TAND, alert clinical teams and families or individuals of the importance of screening, assessment, and treatment of TAND, and provide a shared framework for future studies of tuberous sclerosis complex-associated neuropsychiatric disorders.Copyright © 2015 Elsevier Inc. All rights reserved.
de Vries P J; Whittemore V H; Leclezio L; Byars A W; Dunn D; Ess K C; Hook D; King B H; Sahin M; J ansen A
Pediatric Neurology
2015
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1016/j.pediatrneurol.2014.10.004" target="_blank" rel="noreferrer noopener">10.1016/j.pediatrneurol.2014.10.004</a>
A national survey of Rett syndrome: age, clinical characteristics, current abilities, and health
breathing difficulties; constipation; feeding difficulties; tone and motor problems; Rett syndrome; trajectory; characteristics; epilepsy; weight; gastrointestinal problems; bowel problems
As part of a wider study to investigate the behavioral phenotype of a national sample of girls and women with Rett syndrome (RTT) in comparison to a well-chosen contrast group and its relationship to parental well-being, the development, clinical severity, current abilities and health of 91 participants were analyzed in relation to diagnostic, clinical and genetic mutation categories. Early truncating mutations or large deletions were associated with greater severity. Early regression was also associated with greater severity. All three were associated with lower current abilities. Epilepsy and weight, gastrointestinal and bowel problems were common co-morbidities. Participants with classic RTT had greater health problems than those with atypical RTT. A substantial minority of respondents reported fairly frequent signs of possible pain experienced by their relative with RTT. Overall, the study provides new data on the current abilities and general health of people with RTT and adds to the evidence that the severity of the condition and variation of subsequent disability, albeit generally within the profound range, may be related to gene mutation. The presence of certain co-morbidities represents a substantial ongoing need for better health. The experience of pain requires further investigation.
Cianfaglione R; Clarke A; Kerr M; Hastings R P; Oliver C; Felce D
American Journal of Medical Genetics Part A
2015
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<a href="http://doi.org/10.1002/ajmg.a.37027" target="_blank" rel="noreferrer noopener">10.1002/ajmg.a.37027</a>
A national survey of Rett syndrome: behavioural characteristics
behavior; breathing difficulties; tone and motor problems; Rett syndrome; trajectory; characteristics; hand stereotypies; stereotypy; breathing problems; sleep problems; anxiety; inappropriate fear
BACKGROUND: The aim was to gain a UK national sample of people with Rett syndrome (RTT) across the age range and compare their characteristics using a variety of relevant behavioural measures with a well-chosen contrast group. METHODS: The achieved sample was 91 girls and women, aged from 4 to 47 years, of whom 71 were known to be MECP2 positive. The contrast group (n = 66), matched for age, gender, language and self-help skills, comprised individuals with six other syndromes associated with intellectual disability. Parental questionnaire measures of RTT specific characteristics, impulsivity, overactivity, mood, interest and pleasure, repetitive behaviour and self-injury were administered. RESULTS: Hand stereotypies, breathing irregularities, night-time unrest and anxiety or inappropriate fear were commonly reported among the RTT sample. Problems of low mood were also reported as common. However, mood and interest and pleasure were no lower than found in the contrast group. In addition, self-injury was lower than in the contrast group and was associated with factors found to predict self-injury in other groups of people with severe intellectual disabilities. CONCLUSIONS: There is variability in the manifestation of problem behaviours potentially associated with the syndrome across individuals, with some more severely affected in most areas than others. Some of this variability appears to be underpinned by genetic mutation.
Cianfaglione R; Clarke A; Kerr M; Hastings R P; Oliver C; Moss J; Heald M; Felce D
Journal of Neurodevelopmental Disorders
2015
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<a href="http://doi.org/10.1186/s11689-015-9104-y" target="_blank" rel="noreferrer noopener">10.1186/s11689-015-9104-y</a>
A cross-sectional controlled developmental study of neuropsychological functions in patients with glutaric aciduria type i
adolescent; cognition; cross sectional study; Dystonia; metabolic disorder; human; article; child; female; male; controlled study; adult; clinical article; dystonia/di [Diagnosis]; Barry Albright Dystonia Scale; cognitive development; Cognitive development; continuous performance test; enzyme deficiency; glutaric aciduria type I; Glutaric aciduria type I; Information processing; motor dysfunction; motor performance; neurologic disease assessment; newborn screening; response time; visual memory; visuomotor coordination; working memory; tone and motor problems; Glutaric acidemia type I; trajectory; characteristics
Background: Glutaric aciduria type I (GA-I) is an inherited metabolic disease due to deficiency of glutaryl-CoA dehydrogenase (GCDH). Cognitive functions are generally thought to be spared, but have not yet been studied in detail. Methods: Thirty patients detected by newborn screening (n = 13), high-risk screening (n = 3) or targeted metabolic testing (n = 14) were studied for simple reaction time (SRT), continuous performance (CP), visual working memory (VWM), visual-motor coordination (Tracking) and visual search (VS). Dystonia (n = 13 patients) was categorized using the Barry-Albright-Dystonia Scale (BADS). Patients were compared with 196 healthy controls. Developmental functions of cognitive performances were analysed using a negative exponential function model. Results: BADS scores correlated with speed tests but not with tests measuring stability or higher cognitive functions without time constraints. Developmental functions of GA-I patients significantly differed from controls for SRT and VS but not for VWM and showed obvious trends for CP and Tracking. Dystonic patients were slower in SRT and CP but reached their asymptote of performance similar to asymptomatic patients and controls in all tests. Asymptomatic patients did not differ from controls, except showing significantly better results in Tracking and a trend for slower reactions in visual search. Data across all age groups of patients and controls fitted well to a model of negative exponential development. Conclusions: Dystonic patients predominantly showed motor speed impairment, whereas performance improved with higher cognitive load. Patients without motor symptoms did not differ from controls. Developmental functions of cognitive performances were similar in patients and controls. Performance in tests with higher cognitive demand might be preserved in GA-I, even in patients with striatal degeneration. Copyright © 2015 Boy et al.
Boy N; Heringer J; Haege G; Glahn E M; Hoffmann G F; Garbade S F; Kolker S; Burgard P
Orphanet Journal of Rare Diseases
2015
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1186/s13023-015-0379-6" target="_blank" rel="noreferrer noopener">10.1186/s13023-015-0379-6</a>
Sleep problems and their management in Rett syndrome
sleep disturbance/disorders; Rett syndrome; trajectory; characteristics
Rett syndrome (RTT), a rare but severe neurological disorder is associated with a mutation in the methyl CpG binding protein 2 (MECP2) gene on the X chromosome occurring in 1:9000 live female births. Apparently normal development is followed by a regression in hand and communication skills, and subsequent development of hand stereotypies and abnormal gait. These characteristics are usually accompanied with comorbidities such as sleep problems and scoliosis. Over 80% of RTT individuals have specific sleep problems including night waking and difficulty falling asleep. Using a cross-sectional survey design, this study explored types of sleeping problems observed and relationships with variables such as age and genotype; and finally investigated specific management strategies. Participants mainly comprised families registered in the International Rett Syndrome Phenotype Database (InterRett) with a child of any age who has a confirmed RTT diagnosis and/or a MECP2 mutation. New families were invited via InterRett Facebook page and parent Listserve RettNet. A web-based questionnaire using freely available software, “REDCap” was developed to collect data. The independent variables tested were age group, mutation type, epilepsy, scoliosis, mobility and uses of sleep hygiene strategies while dependent variables tested include the presence, nature and frequency of sleep problems. Descriptive statistics were used to examine each variables and regression models including simple logistic regression and multinomial logistic regression models were employed to investigate the relationships between phenotype, treatments and sleep problems. Findings from this study showed sleep problems were more pronounced in RTT cases than in the general population. The prevalence of most sleep problems were higher in younger children and those with a p.Arg294* mutation. Other covariates including severe seizure activity was associated with poor sleep by taking into account the effect of age, mutation type, scoliosis and mobility. Non-pharmacological interventions were more perceived by families as effective in comparison to pharmacological medications.
Boban S
2015
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/" target="_blank" rel="noreferrer noopener"></a>
Anxiety-like behavior in Rett syndrome: characteristics and assessment by anxiety scales
children; Anxiety; Intellectual disabilities; Neurosciences & Neurology; Rett syndrome; mecp2; mental-retardation; fragile-x-syndrome; checklist; autism spectrum disorder; down-syndrome; mouse model; Problematic behavior; quality-of-life; severity; Social avoidance; behavior; breathing difficulties; sleep disturbance; trajectory; characteristics
Background: Rett syndrome (RTT) is a severe neurodevelopmental disorder characterized by regression of language and motor skills, cognitive impairment, and frequent seizures. Although the diagnostic criteria focus on communication, motor impairments, and hand stereotypies, behavioral abnormalities are a prevalent and disabling component of the RTT phenotype. Among these problematic behaviors, anxiety is a prominent symptom. While the introduction of the Rett Syndrome Behavioral Questionnaire (RSBQ) represented a major advancement in the field, no systematic characterization of anxious behavior using the RSBQ or other standardized measures has been reported. Methods: This study examined the profiles of anxious behavior in a sample of 74 girls with RTT, with a focus on identifying the instrument with the best psychometric properties in this population. The parent-rated RSBQ, Anxiety, Depression, and Mood Scale (ADAMS), and Aberrant Behavior Checklist-Community (ABC-C), two instruments previously employed in children with neurodevelopmental disorders, were analyzed in terms of score profiles, relationship with age and clinical severity, reliability, concurrent validity, and functional implications. The latter were determined by regression analyses with the Vineland Adaptive Behavior Scales-Second Edition (Vineland-II) and the Child Health Questionnaire (CHQ), a quality of life measure validated in RTT. Results: We found that scores on anxiety subscales were intermediate in range with respect to other behavioral constructs measured by the RSBQ, ADAMS, and ABC-C. Age did not affect scores, and severity of general anxiety was inversely correlated with clinical severity. We demonstrated that the internal consistency of the anxiety-related subscales were among the highest. Test-retest and intra-rater reliability was superior for the ADAMS subscales. Convergent and discriminant validity were measured by inter-scale correlations, which showed the best profile for the social anxiety subscales. Of these, only the ADAMS Social Avoidance showed correlation with quality of life. Conclusions: We conclude that anxiety-like behavior is a prominent component of RTT's behavioral phenotype, which affects predominantly children with less severe neurologic impairment and has functional consequences. Based on available data on standardized instruments, the ADAMS and in particular its Social Avoidance subscale has the best psychometric properties and functional correlates that make it suitable for clinical and research
Barnes K V; Coughlin F R; O'Leary H M; Bruck N; Bazin G A; Beinecke E B; Walco A C; Cantwell N G; Kaufmann W E
Journal of Neurodevelopmental Disorders
2015
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1186/s11689-015-9127-4" target="_blank" rel="noreferrer noopener">10.1186/s11689-015-9127-4</a>
Co-occurrence of Dystonic and Dyskinetic Tongue Movements with Oral Apraxia in Post-regression Dysphagia in Classical Rett Syndrome Years of Life 1 Through 5
Deglutition; disorders; girls; phenotype; dysfunction; mecp2 mutations; Classical Rett syndrome; Dysphagia; Early Pseudostationary stage; Fluoroscopy; genotype; Otorhinolaryngology; Swallowing; Videofluoroscopic study of swallowing; women; feeding difficulties; tone and motor; Rett syndrome; trajectory; characteristics; dysmotility; dystonia; dyskinesia
We do not know the natural history of dysphagia in classical Rett syndrome (RTT) by stage or age. This study investigated swallowing physiology in 23 females ages 1:7 to 5:8 (years, months) with classical Rett syndrome to determine common and distinguishing features of dysphagia in post-regression early Pseudostationary Stage III. In-depth analysis of videofluoroscopic swallowing studies (VFSS) found dysmotility of oral stage events across subjects implicating oral apraxia. Impaired motility was further compromised by recurrent dystonic and dyskinetic movements that co-occurred with oral apraxia during oral ingestion in 78 % (n = 18) of the subjects with RTT. Of this group, 44 % displayed rocking and/or rolling lingual pattern, 56 % had recurrent oral tongue retroflexions, and/or elevated posturing of the tongue tip, and, 72 % displayed multi-wave oropharyngeal transfer pattern. The proportion of subjects whose swallowing motility was disrupted by aberrant involuntary tongue movements did not differ significantly between bolus types (liquid, puree, and solid) trialed. Liquid ingestion was significantly more efficient in subjects using bottles with nipples than their counterparts who used spouted or straw cups. Dystonic and dyskinetic tongue movements disrupted liquid ingestion in subjects using cups with spouts or straws significantly more than those using bottles. Analysis of food ingestion revealed that significantly more subjects were able to orally form, transport, and transfer a puree bolus into the pharynx than they were a solid bolus. A significantly larger number of subjects aspirated and penetrated liquid than they did puree or solid. No significant relationship was found between subjects with airway contamination and those with dystonic and dyskinetic tongue movements. Subjects' rocking and rolling lingual patterns were consistent with those evidenced in adults with Parkinson's disease. Subjects' tongue retroflexions were classified as provisionally unique to RTT. VFSS pre-planning, fluoroscopic procedures, and therapeutic strategies specific to this specialty population were derived.
Abraham S S; Taragin B; Djukic A
Dysphagia
2015
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<a href="http://doi.org/10.1007/s00455-014-9587-9" target="_blank" rel="noreferrer noopener">10.1007/s00455-014-9587-9</a>
Elements of optimal paediatric palliative care for children and young people: An integrative review using a systematic approach
Palliative Care/og [Organization & Administration]; Social Support; Child; Humans; Adolescent; Information Storage and Retrieval; Respite Care; Siblings
BACKGROUND: Models of palliative care need to address the unmet needs of children, young people and families. OBJECTIVE: To undertake an integrative review to identify the key elements of optimal paediatric palliative care from the perspectives of children and young people with palliative care needs and their parents. DATA SOURCES: Electronic databases including CINAHL, Medline, PsycINFO and AMED searched using combined terms for palliative care, service models and children along with reference lists of included studies. STUDY SELECTION: Peer reviewed empirical studies reporting on evaluation of paediatric palliative care by children and young people with palliative care needs (0-19 years), or their families, published in English, between 2000 and 2013. The views of health professionals and grey literature were excluded. Quality appraisal completed by two researchers, consensus reached following discussion. DATA EXTRACTION AND SYNTHESIS: Data extracted by two researchers, entered into an electronic proforma and synthesised using a narrative approach. RESULTS: Seven studies were identified of which two were quantitative, one was qualitative and four were mixed methods. Synthesis highlighted the need for tailored support enabling flexibility in care, with specific reference to location of care and access to psychosocial support, 24h specialist support, respite care and sibling support. CONCLUSIONS: Paediatric palliative care should be flexible, responsive and tailored to the needs of children and their families. Robust evaluation of models of care that incorporate these elements is required to inform optimal care.
Virdun C; Brown N; Phillips J; Luckett T;Agar M; Green A; Davidson PM
Collegian: Journal of the Royal College of Nursing, Australia
2015
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1016/j.colegn.2014.07.001" target="_blank" rel="noreferrer noopener">10.1016/j.colegn.2014.07.001</a>
Paediatric palliative care: recommendations for treatment of symptoms in the Netherlands
Netherlands; Palliative Care/st [Standards]; Humans; Palliative Care/mt [Methods]; Pediatrics/st [Standards]; Pediatrics/mt [Methods]; Health Planning Guidelines; Consensus; Caregivers
BACKGROUND: Children dying of a life threatening disease suffer a great deal at the end of life. Symptom control is often unsatisfactory, partly because many caregivers are simply not familiar with paediatric palliative care. To ensure that a child with a life-threatening condition receives high quality palliative care, clinical practice guidelines are needed. The aim of this study is to improve palliative care for children by making high quality care recommendations to recognize and relieve symptoms in paediatric palliative care. METHODS: An extensive search was performed for guidelines and systematic reviews on paediatric palliative care up to year 2011. An expert panel combined the evidence with consensus to form recommendations on the treatment of symptoms in paediatric palliative care. RESULTS: We appraised 21 guidelines and identified 693 potentially eligible articles of which four met our inclusion criteria. None gave recommendations on the treatment of symptoms in paediatric palliative care. Two textbooks and an adult palliative care website were eventually our main sources of evidence. CONCLUSION: Hardly any evidence is available for the treatment of symptoms in paediatric palliative care. By combining evidence for adult palliative care and the sparse evidence for paediatric palliative care with expert opinion we defined a unique set of high quality care recommendations to relieve symptoms and lessen the suffering of children in palliative care. These results are an important tool to educate caregivers on how to relieve symptoms in children in paediatric palliative care.
Knops RR; Kremer L C; Verhagen AA; Dutch Paediatric Palliative Care Guideline Group for Symptoms
BMC Palliative Care
2015
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1186/s12904-015-0054-7" target="_blank" rel="noreferrer noopener">10.1186/s12904-015-0054-7</a>
Research Priorities in Pediatric Palliative Care
Palliative Care; Parents/px [Psychology]; Humans; United States; Pediatrics; Delphi Technique; Attitude of Health Personnel; Research
OBJECTIVE: To synthesize the perspectives of a broad range of pediatric palliative care (PPC) clinicians and parents, to formulate a consensus on prioritization of the PPC research agenda. STUDY DESIGN: A 4-round modified Delphi online survey was administered to PPC experts and to parents of children who had received PPC. In round 1, research priorities were generated spontaneously. Rounds 2 and 3 then served as convergence rounds to synthesize priorities. In round 4, participants were asked to rank the research priorities that had reached at least 80% consensus. RESULTS: A total of 3093 concepts were spontaneously generated by 170 experts and 72 parents in round 1 (65.8% response rate [RR]). These concepts were thematically organized into 78 priorities and recirculated for round 2 ratings (n = 130; 53.7% RR). Round 3 achieved response stability, with 31 consensus priorities oscillating within 10% of the mode (n = 98; 75.4% RR). Round 4 resulted in consensus recognition of 20 research priorities, which were thematically grouped as decision making, care coordination, symptom management, quality improvement, and education. CONCLUSIONS: This modified Delphi survey used professional and parental consensus to identify preeminent PPC research priorities. Attentiveness to these priorities may help direct resources and efforts toward building a formative evidence base. Investigating PPC implementation approaches and outcomes can help improve the quality of care services for children and families.
Baker JN; Levine D R; Hinds PS; Weaver MS; Cunningham MJ; Johnson L; Anghelescu D; Mandrell B; Gibson DV; Jones B; Wolfe J; Feudtner C; Friebert S; Carter B; Kane J R
Journal of Pediatrics
2015
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
<a href="http://doi.org/10.1016/j.jpeds.2015.05.002" target="_blank" rel="noreferrer noopener">10.1016/j.jpeds.2015.05.002</a>
Opening end-of-life discussions: how to introduce Voicing My CHOiCESTM, an advance care planning guide for adolescents and young adults
OBJECTIVE: Each year, more than 11,000 adolescents and young adults (AYAs), aged 15-34, die from cancer and other life-threatening conditions. In order to facilitate the transition from curative to end-of-life (EoL) care, it is recommended that EoL discussions be routine, begin close to the time of diagnosis, and continue throughout the illness trajectory. However, due largely to discomfort with the topic of EoL and how to approach the conversation, healthcare providers have largely avoided these discussions. METHOD: We conducted a two-phase study through the National Cancer Institute with AYAs living with cancer or pediatric HIV to assess AYA interest in EoL planning and to determine in which aspects of EoL planning AYAs wanted to participate. These results provided insight regarding what EoL concepts were important to AYAs, as well as preferences in terms of content, design, format, and style. The findings from this research led to the development of an age-appropriate advance care planning guide, Voicing My CHOiCESTM. RESULTS: Voicing My CHOiCESTM: An Advanced Care Planning Guide for AYA became available in November 2012. This manuscript provides guidelines on how to introduce and utilize an advance care planning guide for AYAs and discusses potential barriers. SIGNIFICANCE OF RESULTS: Successful use of Voicing My CHOiCESTM will depend on the comfort and skills of the healthcare provider. The present paper is intended to introduce the guide to providers who may utilize it as a resource in their practice, including physicians, nurses, social workers, chaplains, psychiatrists, and psychologists. We suggest guidelines on how to: incorporate EoL planning into the practice setting, identify timepoints at which a patient's goals of care are discussed, and address how to empower the patient and incorporate the family in EoL planning. Recommendations for introducing Voicing My CHOiCESTM and on how to work through each section alongside the patient are provided
Zadeh S; Pao M; Wiener L
Palliative & Supportive Care
2015
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<a href="http://doi.org/10.1017/S1478951514000054" target="_blank" rel="noreferrer noopener">10.1017/S1478951514000054</a>
Emotional nursing labour in the childcare at the end-of-life and their family: A sistematic review
We intend to present a systematization of scientiic evidence about the emotional labour of nurses in the process of childcare in the end-of-life and their family. It was performed a literature search in the databases CINAHL, Medline, Psychology and Behavioral Sciences Collection, from which we obtained nine studies for analysis and responses to the research questions: "How nurses perform the emotional labour inherent to nursing care provided to children in the end-of-life and their family?" and "What are the nursing sensitive outcomes?". The indings highlights the focus of the emotional labour, simultaneously focused in the child and family, extolling the philosophy of familycentered care and also focused on the nurses themselves, because they are also affected by the emotional responses of clients and their need to manage these emotions in their care practice. The emotional labour is featured as part of the care process, as key competence in caring, as a stressful experience and as a regulation of their own emotions, and presents nursing sensitive outcomes. Caring for children at the end-of-life is one of the biggest emotional challenges for parents and nurses. Nurses play an emotional labour enrolled in the philosophy of holism and care relationship.
Article information provided for research and reference use only. PedPalASCNET does not hold any rights over the resource listed here. All rights are retained by the journal listed under publisher and/or the creator(s).
Diogo P; Vilelas J; Rodrigues L; Almeida T
Atencion Primaria
2015
<a href="http://doi.org/10.15640/ijn.v1n2a6" target="_blank" rel="noreferrer noopener">10.15640/ijn.v1n2a6</a>
Healthcare users' experiences of communicating with healthcare professionals about children who have life-limiting conditions: a qualitative systematic review protocol
Communication; Critical Illness/px [Psychology]; Palliative Care/px [Psychology]; Parents/px [Psychology]; Professional-Family Relations; adolescent; Adult; Child; Female; Health Personnel/px [Psychology]; Humans; infant; Male; Newborn; Preschool; Qualitative Research; Young Adult
REVIEW QUESTION/OBJECTIVE: The objective of this review is to identify and synthesize the best international qualitative evidence on healthcare users' experiences of communication with healthcare professionals about children who have life-limiting conditions. For the purposes of this review, "healthcare users" will be taken to include children who have life-limiting conditions and their families. The question to be addressed is:What are healthcare users' experiences of communicating with healthcare professionals about children who have life-limiting conditions? BACKGROUND: The prospect of the death of a child from an incurable medical condition is harrowing, yet finding a way to discuss this prospect is crucial to maximize the quality of life for such children and their families. High-quality communication is well recognized as a core skill health care professionals need to maximize the quality of care they provide. This skill is valued by service users, who consistently rate it as one of the highest priorities for the care they receive. Evidence suggests, however, that healthcare professionals can feel ill-equipped or uncomfortable communicating with and about such children. Therefore, it is important to understand what represents high-quality communication and what is involved in accomplishing this within pediatric palliative care.In recent decades there has been an increased focus on providing palliative care for children who have life-limiting conditions. These are conditions for which no cure is available and for which the probable outcome is premature death. Palliative care may also be appropriate for children who have life-threatening conditions; these are conditions where there is not only a high probability of premature death but also a chance of long-term survival into adulthood Although pediatric palliative care is underpinned by the same philosophy as adult palliative care, children who have life-limiting conditions and their families have particular needs that distinguish them from users of adult palliative care. For example, at a physical level children are more likely than adults to have non-malignant conditions that follow trajectories in which children oscillate between feeling relatively well and acutely unwell. The social dynamic of their care is also radically different, particularly given the role of parents or guardians in making surrogate decisions about their child's care. Such factors warrant considering pediatric palliative care as distinct from palliative care more generally.Although the particular circumstances of children who have life-limiting conditions have led to development of pediatric palliative care, the particular provisions of this care differs among countries. One aspect of variation is the age range of patients. Pediatric palliative care is usually provided to neonates, infants, children, adolescents and young adults, but international variations in the definitions of these age ranges, particularly for adolescents and young adults, means pediatric palliative care is provided to different age groups in different countries. This review therefore adopts a pragmatic rather than an age-based definition of a pediatric palliative care, considering all studies relating to service users who are being cared for by pediatric rather than adult healthcare services.In catering for the unique needs of children who have life-limiting conditions and their families, pediatric palliative care aims to achieve pain and symptom management, enhanced dignity and quality of life, and psychosocial and spiritual care. It also seeks to incorporate care for patients' broader families and facilitating access to appropriate services and support. High-quality communication is crucial for achieving these aims. It enables healthcare users and providers to make decisions that underpin the care that is provided and the quality of the life that is possible for patients and their families.Although both users and providers recognize the value of high-quality communication with and about children who have life-limiting conditions, this does not mean that these stakeholders necessarily share the same perspective of what constitutes high-quality communication and the best way of accomplishing this. Focusing on healthcare users' experiences of communication with healthcare professionals about children who have life-limiting conditions, the present review will explore the subjective impact of professionals' communication on the people for whom they provide care. It may be necessary to consider a range of contextual factors to understand healthcare users' experiences of communicating with healthcare professionals about children who have life-limiting conditions. For instance, age, developmental stage, cognitive capacity, emotional and social strengths, and family dynamics can influence a child's level of involvement in discussions about their condition and care.13,28,29,35 Although there are factors that appear more consistent across the range of pediatric palliative care users, such as parents' preferences for being treated by healthcare professionals as partners in making decisions about the care of their child, there is not always such consistency.29 Nor is it clear whether such findings can be generalized across different cultural contexts.13,36 In appraising existing research, this systematic review will therefore consider the relationship between the context of individual studies and their reported findings. Background The primary aim of this review is to identify, appraise and synthesize existing qualitative evidence of healthcare users' experiences of communicating with healthcare professionals about children who have life-limiting conditions. The review will consider relevant details of these findings, particularly whether factors like age are relevant for understanding particular experiences of communication. An outcome of this review will be the identification of best available qualitative evidence that can be used to inform professional practice, as well as an identification of priorities for future research in pediatric palliative care. Background A preliminary search in MEDLINE and CINAHL found primary studies exploring healthcare users' experiences of aspects of communicating with healthcare professionals about children who have life-limiting conditions. A search was also conducted for existing systematic reviews in PubMed, CINAHL, EMBASE, PsycINFO, the Cochrane Database of Systematic Reviews, the JBI Database of Systematic Reviews and Implementation Reports, and PROSPERO. No systematic reviews on this topic were found.
2015-11
Ekberg S; Bradford N; Herbert A; Danby S; Yates P
Jbi Database Of Systematic Reviews And Implementation Reports
2015
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<a href="http://doi.org/10.11124/jbisrir-2015-2413" target="_blank" rel="noreferrer">10.11124/jbisrir-2015-2413</a>
Palliative care in neonatal neurology: robust support for infants, families and clinicians
Infant; Nervous System Diseases; Palliative Care; Parents/px [Psychology]; Clinical Decision-Making/es [Ethics]; Diseases; Diseases/px [Psychology]; Diseases/th [Therapy]; Humans; infant; Intensive Care Units; Neonatal/og [Organization & Administration]; Nervous System Diseases/px [Psychology]; Nervous System Diseases/th [Therapy]; Neurology/mt [Methods]; Newborn; Palliative Care/es [Ethics]; Palliative Care/mt [Methods]; Palliative Care/px [Psychology]; social support
Infants with neurological injury and their families face unique challenges in the neonatal intensive care unit. As specialty palliative care support becomes increasingly available, we must consider how to intentionally incorporate palliative care principles into the care of infants with neurological injury. Here, we review data regarding neonatal symptom management, prognostic uncertainty, decision making, communication and parental support for neonatal neurology patients and their families.
2015
Lemmon ME; Bidegain M; Boss RD
Journal Of Perinatology
2015
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<a href="http://doi.org/10.1038/jp.2015.188" target="_blank" rel="noreferrer">10.1038/jp.2015.188</a>
Good-parent beliefs of parents of seriously ill children
adolescent; Child; Cross-Sectional Studies; Female; Humans; infant; Male; Parent-Child Relations; Parents; Questionnaires; Chronic disease; Child welfare; Acute Disease; Stress; Practice; Preschool; Adaptation; Psychological; Attitudes; Newborn; Health Knowledge; Philadelphia
IMPORTANCE: Parents' beliefs about what they need to do to be a good parent when their children are seriously ill influence their medical decisions, and better understanding of these beliefs may improve decision support. OBJECTIVE: To assess parents' perceptions regarding the relative importance of 12 good-parent attributes. DESIGN, SETTING, AND PARTICIPANTS: A cross-sectional, discrete-choice experiment was conducted at a children's hospital. Participants included 200 parents of children with serious illness. MAIN OUTCOMES AND MEASURES: Ratings of 12 good-parent attributes, with subsequent use of latent class analysis to identify groups of parents with similar ratings of attributes, and ascertainment of whether membership in a particular group was associated with demographic or clinical characteristics. RESULTS: The highest-ranked good-parent attribute was making sure that my child feels loved, followed by focusing on my child's health, making informed medical care decisions, and advocating for my child with medical staff. We identified 4 groups of parents with similar patterns of good-parent-attribute ratings, which we labeled as: child feels loved (n=68), child's health (n=56), advocacy and informed (n=55), and spiritual well-being (n=21). Compared with the other groups, the child's health group reported more financial difficulties, was less educated, and had a higher proportion of children with new complex, chronic conditions. CONCLUSIONS AND RELEVANCE: Parents endorse a broad range of beliefs that represent what they perceive they should do to be a good parent for their seriously ill child. Common patterns of how parents prioritize these attributes exist, suggesting future research to better understand the origins and development of good-parent beliefs among these parents. More important, engaging parents individually regarding what they perceive to be the core duties they must fulfill to be a good parent may enable more customized and effective decision support.
2015-01
Feudtner C; Walter JK; Faerber JA; Hill DL; Carroll KW; Mollen CJ; Miller VA; Morrison WE; Munson DA; Kang T; Hinds PS
Jama Pediatrics
2015
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Journal Article
<a href="http://doi.org/10.1001/jamapediatrics.2014.2341" target="_blank" rel="noreferrer">10.1001/jamapediatrics.2014.2341</a>
Hypoxic-ischemic encephalopathy: a review for the clinician
IMPORTANCE: Hypoxic-ischemic encephalopathy (HIE) occurs in 1 to 8 per 1000 live births in developed countries. Historically, the clinician has had little to offer neonates with HIE other than systemic supportive care. Recently, the neuroprotective therapy of hypothermia has emerged as the standard of care, and other complementary therapies are rapidly transitioning from the basic science to clinical care. OBJECTIVE: To examine the pathophysiology of HIE and the state of the art for the clinical care of neonates with HIE. EVIDENCE REVIEW: We performed a literature review using the PubMed database. Results focused on reviews and articles published from January 1, 2004, through December 31, 2014. Articles published earlier than 2004 were included when appropriate for historical perspective. Our review emphasized evidence-based management practices for the clinician. FINDINGS: A total of 102 articles for critical review were selected based on their relevance to the incidence of HIE, pathophysiology, neuroimaging, placental pathology, biomarkers, current systemic supportive care, hypothermia, and emerging therapies for HIE and were reviewed by both of us. Seventy-five publications were selected for inclusion in this article based on their relevance to these topics. The publications highlight the emergence of serum-based biomarkers, placental pathology, and magnetic resonance imaging as useful tools to predict long-term outcomes. Hypothermia and systemic supportive care form the cornerstone of therapy for HIE. CONCLUSIONS AND RELEVANCE: The pathophysiology of HIE is now better understood, and treatment with hypothermia has become the foundation of therapy. Several neuroprotective agents offer promise when combined with hypothermia and are entering clinical trials.
2015-04
Douglas-Escobar M; Weiss MD
Jama Pediatrics
2015
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Journal Article
<a href="http://doi.org/10.1001/jamapediatrics.2014.3269" target="_blank" rel="noreferrer">10.1001/jamapediatrics.2014.3269</a>
Close to home
Humans; Professional-Family Relations; Physicians; social support; Leukemia; Community Networks; Grief
2015-03
Rosenberg AR
Jama Pediatrics
2015
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Journal Article
<a href="http://doi.org/10.1001/jamapediatrics.2014.3449" target="_blank" rel="noreferrer">10.1001/jamapediatrics.2014.3449</a>
Hearing others' perspectives when we hear, "do everything!"
2015-05
Hirni K; Carter B
Jama Pediatrics
2015
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Journal Article
<a href="http://doi.org/10.1001/jamapediatrics.2014.3699" target="_blank" rel="noreferrer">10.1001/jamapediatrics.2014.3699</a>
Insurance status and risk of cancer mortality among adolescents and young adults
BACKGROUND: Adolescents and young adults with cancer have inferior survival outcomes compared with younger pediatric patients and older adult patients. Lack of insurance may partly explain this disparity. The objective of this study was to identify associations between insurance status and both advanced-stage cancer and cancer-specific mortality. METHODS: Using the Surveillance, Epidemiology, and End Results (SEER) 18 registries, 57,981 patients ages 15 to 39 years were identified who were diagnosed between 2007 and 2010 and had complete insurance and staging information. Multinomial logistic regression models were used to identify associations between insurance type and disease stage, with the models adjusted for sex, age, and race. Cox proportional hazards models were used to estimate cancer-specific mortality. RESULTS: Overall, 84% of patients were aged ≥25 years, 64% were women, and 79% were privately insured. Compared with patients who had private insurance, those who had nonprivate insurance tended to present with more advanced-stage disease and to die more quickly and more commonly from their cancer. Patients ages 25 to 39 years who had Medicaid coverage or no insurance had 3.2 times and 2.4 times higher odds of having stage IV disease, respectively, than privately insured patients (95% confidence interval [CI], 3.0-3.5 times higher odds and 2.1-2.6 times higher odds, respectively). Among those with stage I/II and III/IV cancers, the risk of death was 2.9 times greater (95% CI, 2.2-3.9 times greater) and 1.7 times greater (95% CI, 1.5-1.9 times greater), respectively, than the risk for privately insured patients. Patients who died from stage III/IV cancers survived at least 2 months longer if they had private insurance. CONCLUSIONS: Among young adults, insurance status is independently associated with advanced-stage cancer and the risk of death from cancer, even for patients who have low-stage disease. Broader insurance coverage and access to health care may improve some of the disparate outcomes of adolescents and young adults with cancer. Cancer 2015;121:1279-1286. © 2014 American Cancer Society.
2015-04
Rosenberg AR; Kroon L; Chen Lu; Li CI; Jones BL
Cancer
2015
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Journal Article
<a href="http://doi.org/10.1002/cncr.29187" target="_blank" rel="noreferrer">10.1002/cncr.29187</a>
Does phase 1 trial enrollment preclude quality end-of-life care? Phase 1 trial enrollment and end-of-life care characteristics in children with cancer
Oncology at EOL
BACKGROUND: End-of-life care (EOLC) discussions and treatment-related decisions, including phase 1 trial enrollment, in patients with incurable disease are complex and can influence the quality of EOLC received. The current study was conducted in pediatric oncology patients to determine whether end-of-life characteristics differed between those who were and were not enrolled in a phase 1 trial. METHODS: The authors reviewed the medical records of 380 pediatric oncology patients (aged <22 years at the time of death) who died during a 3.5-year period. Of these, 103 patients with hematologic malignancies were excluded. A total of 277 patients with a diagnosis of a brain tumor or other solid tumor malignancy were divided into 2 groups based on phase 1 trial enrollment: a phase 1 cohort (PIC; 120 patients) and a non-phase 1 cohort (NPIC; 157 patients). The EOLC characteristics of these 2 cohorts were compared using regression analysis and chi-square testing. RESULTS: A comparison of patients in the PIC and NPIC revealed no significant differences in either demographic characteristics (including sex, race, religious affiliation, referral origin, diagnosis, or age at diagnosis, with the exception of age at the time of death [P =.03]) or in EOLC indices (such as use or timing of do not attempt resuscitation orders, hospice use or length of stay, forgoing life-sustaining therapies, location of death, time from first EOLC discussion to death, and total number of EOLC discussions). CONCLUSIONS: The results of the current study of a large cohort of deceased pediatric cancer patients indicate that enrollment on a phase 1 trial does not affect EOLC characteristics, suggesting that quality EOLC can be delivered regardless of phase 1 trial participation. Cancer 2015;121:1508-1512. © 2014 American Cancer Society.
2015-05
Levine DR; Johnson LM; Mandrell BN; Yang J; West NK; Hinds PS; Baker JN
Cancer
2015
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Journal Article
<a href="http://doi.org/10.1002/cncr.29230" target="_blank" rel="noreferrer">10.1002/cncr.29230</a>
Practical communication guidance to improve phase 1 informed consent conversations and decision-making in pediatric oncology
Ethics; patient perspectives; Pediatric oncology; physician communication
BACKGROUND: It can be difficult to explain pediatric phase 1 oncology trials to families of children with refractory cancer. Parents may misunderstand the information presented to them, and physicians may assume that certain topics are covered in the informed consent document and need not be discussed. Communication models can help to ensure effective discussions. METHODS: Suggestions for improving the informed consent process were first solicited from phase 1 study clinicians via questionnaire. Eight parents who had enrolled their child on a phase 1 pediatric oncology trial were recruited for an advisory group designed to assess the clinicians' suggestions and make additional recommendations for improving informed consent for pediatric phase 1 trials. RESULTS: A phase 1 communication model was designed to incorporate the suggestions of clinicians and families. It focused on educating parents/families about phase 1 trials at specific time points during a child's illness, but specifically at the point of disease recurrence. An informative phase 1 fact sheet that can be distributed to families was also presented. CONCLUSIONS: Families who will be offered information regarding phase 1 clinical trials can first receive a standardized fact sheet explaining the general purpose of these early-phase clinical trials. Parental understanding may be enhanced further when oncologists address key themes, beginning at the time of diagnosis and continuing through important decision points during the child's illness. This model should be prospectively evaluated. Cancer 2015. © 2015 American Cancer Society.
2015-04
Johnson LM; Leek AC; Drotar D; Noll RB; Rheingold SR; Kodish ED; Baker JN
Cancer
2015
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Journal Article
<a href="http://doi.org/10.1002/cncr.29354" target="_blank" rel="noreferrer">10.1002/cncr.29354</a>
Adolescent end of life preferences and congruence with their parents' preferences: results of a survey of adolescents with cancer
BACKGROUND: Little is known about how well family members accurately represent adolescents when making EOL decisions on their behalf. This study reports on surveys given to adolescents with cancer and their parents as part of a larger study facilitating advanced care discussions, as well as the results of a survey for health care providers. PROCEDURE: Trained facilitators administered surveys orally to adolescents and families in the intervention arm of the FAmily CEntered Advance Care Planning (ACP) for Teens with Cancer (FACE-TC) study. In addition, a post-hoc survey was sent to oncology providers. RESULTS: Seventeen adolescent/family dyads completed this survey. Seventy five percent of adolescents believed it was appropriate to discuss EOL decisions early and only 12% were not comfortable discussing death. Most preferred to be at home if dying. There were substantial areas of congruence between adolescents and their surrogates, but lower agreement on the importance of dying a natural death, dying at home and "wanting to know if I were dying." Among providers, 83% felt their patients' participation in the study was helpful to the patients and 78% felt it was helpful to them as providers. CONCLUSIONS: Adolescents with cancer were comfortable discussing EOL, and the majority preferred to talk about EOL issues before they are facing EOL. There were substantive areas of agreement between adolescents and their surrogates, but important facets of adolescents' EOL wishes were not known by their families, reinforcing the importance of eliciting individual preferences and engaging dyads so parents can understand their children's wishes.
2015-04
Jacobs S; Perez J; Cheng YI; Sill A; Wang J; Lyon ME
Pediatric Blood & Cancer
2015
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Journal Article
<a href="http://doi.org/10.1002/pbc.25358" target="_blank" rel="noreferrer">10.1002/pbc.25358</a>
Differences in end-of-life communication for children with advanced cancer who were referred to a palliative care team
BACKGROUND: There is a general consensus that involving a specialized palliative care team in the care of children with advanced cancer can help optimize end-of-life communication; however, how this compares to standard oncology care is still unknown. We aimed to determine whether there was an association between specialist palliative care involvement and improved end-of-life communication for children with advanced cancer and their families. PROCEDURE: We administered questionnaires to 75 bereaved parents (response rate 54%). Outcome measures were presence or absence of 11 elements related to end-of-life communication. RESULTS: Parents were significantly more likely to receive five communication elements if their child was referred to a palliative care team. These elements are: discussion of death and dying with parents by the healthcare team (P < 0.01); discussion of death and dying with child by the healthcare team when appropriate (P < 0.01); providing parents with guidance on how to talk to their child about death and dying when appropriate (P < 0.01); preparing parents for medical aspects surrounding death (P = 0.02) and sibling support (P = 0.02). Children were less likely to be referred to a palliative care team if they had a hematologic malignancy. CONCLUSIONS: Children who receive standard oncology care are at higher risk of not receiving critical communication elements at end of life. Strategies to optimize end-of-life communication for children who are not referred to a palliative care team are needed. Pediatr Blood Cancer © 2015 Wiley Periodicals, Inc.
2015-04
Kassam A; Skiadaresis J; Alexander S; Wolfe J
Pediatric Blood & Cancer
2015
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Journal Article
<a href="http://doi.org/10.1002/pbc.25530" target="_blank" rel="noreferrer">10.1002/pbc.25530</a>
Patterns of End-of-Life Care in Children with Advanced Solid Tumor Malignancies Enrolled on a Palliative Care Service
CONTEXT: Pediatric patients with solid tumors can have a significant symptom burden that impacts quality of life (QoL) and end-of-life care needs. OBJECTIVES: We evaluated outcomes and symptoms in children with solid tumors and compared patterns of end-of-life care after implementation of a dedicated institutional pediatric palliative care (PC) service. METHODS: We performed a retrospective cohort study of children with solid tumors treated at St. Jude Children's Research Hospital, before and after implementation of the institutional QoL/PC service in January 2007. Patients who died between July 2001 and February 2005 (historical cohort; n = 134) were compared with those who died between January 2007 and January 2012 (QoL/PC cohort; n = 57). RESULTS: Median time to first QoL/PC consultation was 17.2 months (range 9-33). At consultation, 60% of children were not receiving or discontinued cancer-directed therapy. Within the QoL/PC cohort, 54 patients had documented symptoms, 94% required intervention for ≥3 symptoms, and 76% received intervention for ≥5 symptoms. Eighty-three percent achieved their preferred place of death. Compared with the historical cohort, the QoL/PC cohort had more end-of-life discussions per patient (median 12 vs. 3; P < 0.001), earlier end-of-life discussions, with longer times before do-not-resuscitate orders (median 195 vs. 2 days; P < 0.001), and greater hospice enrollment (71% vs. 46%, P = 0.002). CONCLUSION: Although children with solid tumor malignancies may have significant symptom burden toward the end of life, positive changes were documented in communication and in places of care and death after implementation of a pediatric PC service.
2015-04
Vern-Gross Tamara Z; Lam CG; Graff Z; Singhal S; Levine DR; Gibson D; Sykes AD; Anghelescu DL; Yuan Y; Baker JN
Journal Of Pain And Symptom Management
2015
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Journal Article
<a href="http://doi.org/10.1016/j.jpainsymman.2015.03.008" target="_blank" rel="noreferrer">10.1016/j.jpainsymman.2015.03.008</a>
Placebo effects in psychiatry: mediators and moderators
2015-03
Weimer K; Colloca L; Enck P
The Lancet Psychiatry
2015
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Journal Article
<a href="http://doi.org/10.1016/S2215-0366(14)00092-3" target="_blank" rel="noreferrer">10.1016/S2215-0366(14)00092-3</a>
Communication intervention in the neonatal intensive care unit: can it backfire?
BACKGROUND: For parents of a critically ill infant, good communication may help alleviate stress and anxiety. To improve communication, physicians must be responsive to families' needs and values surrounding the care of their hospitalized infant. OBJECTIVE: We adapted a Decision-Making Tool for the Neonatal Intensive Care Unit (N-DMT) to encourage consideration of family concerns and preferences in daily care planning. DESIGN: This was a randomized controlled design. SETTING/SUBJECTS: Parents and providers of critically ill neonates were eligible. Parents were randomized to an intervention group (using the N-DMT) or standard of care. N-DMT information was shared through the electronic medical record and communicated directly to the primary provider. MEASUREMENTS: Daily rounds on all infants were audio recorded. Parents completed the State-Trait Anxiety Inventory at the first interview and 2 weeks later. Parents completed the Family Inventory of Needs-Pediatrics (FIN-PED) survey and an N-DMT-specific survey 2 weeks postenrollment. RESULTS: Complete data were obtained on 10 control and 9 intervention families. Groups did not differ on demographics or mean infant Score of Neonatal Acute Physiology (SNAP) scores (36 versus 37). FIN-PED scores were similar for both groups. The control group showed decreased anxiety over time. The content of rounds did not differ between groups. The intervention group reported lower satisfaction with care, specifically in questions regarding communication. CONCLUSIONS: In this pilot study, we found that families in the intervention group were less satisfied with communication. Families who are primed to expect better communication, such as those participating in a communication intervention, may be less satisfied with standard care.
2015-02
Clarke-Pounder JP; Boss RD; Roter DL; Hutton N; Larson S; Donohue PK
Journal Of Palliative Medicine
2015
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Journal Article
<a href="http://doi.org/10.1089/jpm.2014.0037" target="_blank" rel="noreferrer">10.1089/jpm.2014.0037</a>
Parents' Faith and Hope during the Pediatric Palliative Phase and the Association with Long-Term Parental Adjustment
BACKGROUND: The loss of a child is associated with an increased risk for developing psychological problems. However, studies investigating the impact of parents' faith and hope for a cure during the palliative phase on long-term parental psychological functioning are limited. OBJECTIVE: The study's objective was to explore the role of faith and hope as a source of coping and indicator of long-term parental adjustment. METHODS: Eighty-nine parents of 57 children who died of cancer completed questionnaires retrospectively, exploring faith, hope, and sources of coping, and measuring parents' current level of grief and depression. RESULTS: For 19 parents (21%) faith was very important during the palliative phase. The majority of parents remained hopeful for a meaningful time with their child (n=68, 76%); a pain-free death (n=58, 65%); and a cure (n=30, 34%). Their child (n=70, 79%) was parents' main source of coping. Twelve parents (14%) suffered from traumatic grief, and 22 parents (25%) showed symptoms of depression. Parents' faith was not associated with less long-term traumatic grief (OR=0.86, p=0.51) or symptoms of depression (OR=0.95, p=0.74), and parents' hope for a cure was not related to more long-term traumatic grief (OR=1.07, p=0.71) or symptoms of depression (OR=1.12, p=0.47). CONCLUSIONS: Faith was important for a minority of parents and was not associated with less long-term traumatic grief or symptoms of depression. The majority of parents remained hopeful. Hope for a cure was not associated with more long-term traumatic grief or symptoms of depression.
2015-05
van der Geest Ivana MM; van den Heuvel-Eibrink MM; Falkenburg N; Michiels EMC; van Vliet L; Pieters R; Darlington Anne-Sophie E
Journal Of Palliative Medicine
2015
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Journal Article
<a href="http://doi.org/10.1089/jpm.2014.0287" target="_blank" rel="noreferrer">10.1089/jpm.2014.0287</a>
From goals of care to improved family outcomes in the neonatal intensive care unit: determining the intervention
2015-02
Collura C; Wolfe J
Journal Of Palliative Medicine
2015
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Journal Article
<a href="http://doi.org/10.1089/jpm.2015.1009" target="_blank" rel="noreferrer">10.1089/jpm.2015.1009</a>
Introducing a Lexicon of Terms for Pediatric Palliative Care
PEDI Study
2015
Spicer S; Macdonald ME; Davies D; Vadeboncoeur C; Siden H
Paediatrics & Child Health
2015
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Journal Article
<a href="http://doi.org/10.1093/pch/20.3.155" target="_blank" rel="noreferrer">10.1093/pch/20.3.155</a>
Pediatric delirium and associated risk factors: a single-center prospective observational study
OBJECTIVE: To describe a single-institution pilot study regarding prevalence and risk factors for delirium in critically ill children. DESIGN: A prospective observational study, with secondary analysis of data collected during the validation of a pediatric delirium screening tool, the Cornell Assessment of Pediatric Delirium. SETTING: This study took place in the PICU at an urban academic medical center. PATIENTS: Ninety-nine consecutive patients, ages newborn to 21 years. INTERVENTION: Subjects underwent a psychiatric evaluation for delirium based on the Diagnostic and Statistical Manual IV criteria. MEASUREMENTS AND MAIN RESULTS: Prevalence of delirium in this sample was 21%. In multivariate analysis, risk factors associated with the diagnosis of delirium were presence of developmental delay, need for mechanical ventilation, and age 2-5 years. CONCLUSIONS: In our institution, pediatric delirium is a prevalent problem, with identifiable risk factors. Further large-scale prospective studies are required to explore multi-institutional prevalence, modifiable risk factors, therapeutic interventions, and effect on long-term outcomes.
2015-05
Silver G; Traube C; Gerber LM; Sun X; Kearney J; Patel A; Greenwald B
Pediatric Critical Care Medicine
2015
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Journal Article
<a href="http://doi.org/10.1097/PCC.0000000000000356" target="_blank" rel="noreferrer">10.1097/PCC.0000000000000356</a>
Continuing education needs of pediatricians across diverse specialties caring for children with medical complexity
OBJECTIVE: Care for children with medical complexity (CMC) relies on pediatricians who often are ill equipped, but striving to provide high quality care. We performed a needs assessment of pediatricians across diverse subspecialties at a tertiary academic US children's hospital about their continuing education needs regarding the care of CMC. METHODS: Eighteen pediatricians from diverse subspecialties were asked to complete an online anonymous open-ended survey. Data were analyzed using modified grounded theory. RESULTS: The response rate was 89% (n = 16). Of participants, 31.2% (n = 5) were general pediatricians, 18.7% (n = 3) were hospitalists, and 50% (n = 8) were pediatric subspecialists. Pediatricians recognized the need for skills in care coordination, giving bad news, working in interprofessional teams, and setting goals of care with patients. CONCLUSIONS: Practicing pediatricians need skills to improve care for CMC. Strategically incorporating basic palliative care education may fill an important training need across diverse pediatric specialties.
2015-03
Bogetz JF; Bogetz AL; Gabhart JM; Bergman DA; Blankenburg RL; Rassbach CE
Clinical Pediatrics
2015
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Journal Article
<a href="http://doi.org/10.1177/0009922814564049" target="_blank" rel="noreferrer">10.1177/0009922814564049</a>
Why does it happen like this?' Consulting with users and providers prior to an evaluation of services for children with life limiting conditions and their families
adolescent; Child; Humans; Palliative Care; referral and consultation; Family; Interviews as Topic; disabled children; Health Services Needs and Demand; Health Services Research; Child Health Services; Focus Groups; Children’s participation; quality of care
Children with life limiting conditions and their families have complex needs. Evaluations must consider their views and perspectives to ensure care is relevant, appropriate and acceptable. We consulted with children, young people, their parents and local professionals to gain a more informed picture of issues affecting them prior to preparing a bid to evaluate services in the area. Multiple methods included focus groups, face-to-face and telephone interviews and participatory activities. Recordings and products from activities were analysed for content to identify areas of relevance and concern. An overarching theme from parents was 'Why does it happen like this?' Services did not seem designed to meet their needs. Whilst children and young people expressed ideas related to quality of environment, services and social life, professionals focused on ways of meeting the families' needs. The theme that linked families' concerns with those of professionals was 'assessing individual needs'. Two questions to be addressed by the evaluation are (1) to what extent are services designed to meet the needs of children and families and (2) to what extent are children, young people and their families consulted about what they need? Consultations with families and service providers encouraged us to continue their involvement as partners in the evaluation.
2015-09
Hunt A; Brown E; Coad J; Staniszewska S; Hacking S; Chesworth B; Chambers L
Journal Of Child Health Care
2015
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Journal Article
<a href="http://doi.org/10.1177/1367493513510630" target="_blank" rel="noreferrer">10.1177/1367493513510630</a>
Initial development and psychometric testing of an instrument to measure the quality of children's end-of-life care
BACKGROUND: The field of pediatric palliative care is hindered by the lack of a well-defined, reliable, and valid method for measuring the quality of end-of-life care. METHODS: The study purpose was to develop and test an instrument to measure mothers' perspectives on the quality of care received before, at the time of, and following a child's death. In Phase 1, key components of quality end-of-life care for children were synthesized through a comprehensive review of research literature. These key components were validated in Phase 2 and then extended through focus groups with bereaved parents. In Phase 3, items were developed to assess structures, processes, and outcomes of quality end-of-life care then tested for content and face validity with health professionals. Cognitive testing was conducted through interviews with bereaved parents. In Phase 4, bereaved mothers were recruited through 10 children's hospitals/hospices in Canada to complete the instrument, and psychometric testing was conducted. RESULTS: Following review of 67 manuscripts and 3 focus groups with 10 parents, 141 items were initially developed. The overall content validity index for these items was 0.84 as rated by 7 health professionals. Based on feedback from health professionals and cognitive testing with 6 parents, a 144-item instrument was finalized for further testing. In Phase 4, 128 mothers completed the instrument, 31 of whom completed it twice. Test-retest reliability, internal consistency, and construct validity were demonstrated for six subscales: Connect With Families, Involve Parents, Share Information With Parents, Share Information Among Health Professionals, Support Parents, and Provide Care at Death. Additional items with content validity were grouped in four domains: Support the Child, Support Siblings, Provide Bereavement Follow-up, and Structures of Care. Forty-eight items were deleted through psychometric testing, leaving a 95-item instrument. CONCLUSIONS: There is good initial evidence for the reliability and validity of this new quality of end-of-life care instrument as a mechanism for evaluative feedback to health professionals, health systems, and policy makers to improve children's end-of-life care.
2015
Widger K; Tourangeau AE; Steele R; Streiner DL
Bmc Palliative Care
2015
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Journal Article
<a href="http://doi.org/10.1186/1472-684X-14-1" target="_blank" rel="noreferrer">10.1186/1472-684X-14-1</a>