1,720,994 research outputs found
Effect of neo-adjuvant chemotherapy on long-term health state and behaviour in the PNET3 RCT of treatment for primitive neuroectodermal tumour (PNET)
No full textThe addition of chemotherapy to craniospinal irradiation (CSI) for treatment of PNET is currently considered ‘the standard of care’ to improve both rate and quality of survival (QoS), but its effect on QoS has not been systematically assessed. We assessed QoS in UK children previously enrolled in the SIOP PNET3 RCT of CSI (35 Gy plus 20 Gy boost to posterior fossa) vs. CSI plus neo-adjuvant chemotherapy (Vincristine, Carboplatin, Etoposide, and Cyclophosphamide) using a cross-sectional multi-informant questionnaire design. Total scores on the two core measures, the Health Utilities Index mark 3 (HUI3) and the Strengths and Difficulties Questionnaire (SDQ), were compared between the two treatment arms using the Mann-Whitney U test. Information was obtained from patients, parents, and health professionals on 103/169 (61%) of survivors at a mean (SD) of 15.4 (4.0) years of age and 7.0 (2.25) years from diagnosis. Median {interquartile range} HUI scores and mean (SD) SDQ total difficulties scores were: HUI3 SDQ Respondent Parent Self* Parent Self** n 44 vs. 55 38 vs. 44 33 vs. 43 27 vs. 35 CSI 0.71 {0.52} 0.92 {0.37} 11.4 (5.21) 10.6 (4.59) CSI + Chemo 0.72 {0.48} 0.65 {0.45} 13.7 (5.90) 11.4 (5.90) *: aged over 11 years; **: aged 11–17 years Self-complete HUI3 scores were lower (Z = -2.263, p = 0.024, two-tailed) and parent-complete SDQ scores tended to be higher (Z = -1.559, p = 0.119, two-tailed) in the ‘CSI plus chemotherapy’ group. Parent-/child-complete SDQ scores were abnormally high in 14.0/2.9% and 33.3/7.4% of children in the ‘CSI’ and the ‘CSI plus chemotherapy’ arms, respectively. Patients in the two treatment arms will also be compared with respect to subscale scores on these measures, responses to quality of life questionnaires, and information from health professionals. These findings suggest that differences between self- and proxy-assessment are important and that the addition of chemotherapy to ‘standard dose’ CSI may adversely affect health status and behaviour. The effect on these measures of reducing the dose of CSI and simultaneously adding chemotherapy requires further investigation
A longitudinal study of health related quality of life in children treated for cerebellar tumours compared with a non-tumour group
No full textThis thesis investigated health related quality of life (HRQoL) measured annually at three time points (T1, T2, T3) in children treated for medulloblastoma (SRM) or low grade cerebellar astrocytoma (LGCA) compared with a typically developing group of children. Four research questions were addressed. These were, first, whether HRQoL and other aspects of quality of survival differ between children treated for cerebellar tumours and a representative sample of children in the general population. Second, whether there are differences between HRQoL in children treated for SRM and LGCA. Third, whether HRQoL and the factors that impacted on it changed over time. Fourth whether there were any early modifiable predictors of subsequent HRQoL.Children treated for cerebellar tumours had a significantly poorer HRQoL and IQ than the Comparison group. In addition, those in the SRM group had significantly poorer health status, and behavioural and executive functioning, the latter by teacher-report only. Children in the SRM group had a significantly poorer HRQoL, health status (by parent-report only), and behavioural functioning (by teacher-report only) than children in the LGCA group. IQ and executive functioning were similar.Longitudinally, in the SRM group, HRQoL and health status improved but remained very poor. Behaviour and IQ did not improve, and executive functioning declined (by teacher-report only). In the LGCA group HRQoL and IQ did not improve and remained poor. Specific help at school increased in the SRM group from 40% at T1 to 57% at T3. In the LGCA group the percentages were 11 and 24 compared with a consistent 3% in the Comparison group, indicating increasing need for educational support in both tumour groups. Motor and sensory functioning, emotional functioning (except by parent-report at T3), and cognitive functioning (by child-report at T3 only) were consistent predictors of HRQoL over time. At T1, emotion and cognition (by child-and parent-report), child’s age (by child-report), and motor and sensory functioning (by parent-report) predicted subsequent HRQoL two years later.These findings show that impairment is evident early on in children treated for cerebellar tumours and persists over time. HRQoL remains poor particularly in the LGCA group where no improvement was observed. These children need to be assessed regularly and monitored as early intervention to mitigate cognitive and emotional difficulties especially in older children, may help to improve subsequent HRQoL. Future research should focus on early interventions.<br/
Does a Bevacizumab-based regime have a role in the treatment of children with diffuse intrinsic pontine glioma?: a systematic review
No full textBackground: There are no effective treatments for diffuse intrinsic pontine glioma (DIPG); median survival is 11.2 months. Bevacizumab has the potential to improve quality of life (QOL) and survival in DIPG but has never been evaluated systematically. The aim of this review was to assess Bevacizumab's role in the treatment of DIPG.Methods: MEDLINE, EMBASE, Scopus, and Web of Science were searched for relevant studies using terms developed from alternatives for Bevacizumab and DIPG. One reviewer screened titles and abstracts, then two reviewers screened full texts. Data were extracted into tables and quality assessed using methodological index for non-randomized studies and JBI tools.Results: Searching revealed 1001 papers; after deduplication 851 remained. After screening of titles and abstracts, then 28 full texts, 11 studies were included. Four studies reported a median overall survival longer than historical data, however, two found no significant impact of Bevacizumab. Five studies reported a radiological response in a proportion of participants and two reported no response. Three studies, evaluating clinical response, reported improvement in a proportion of patients. Three studies, evaluating QOL, reported stability or improvement. Four studies, evaluating steroid use, reported reductions in the proportion of patients receiving steroids. In radiation necrosis treatment, Bevacizumab led to clinical improvement in 6/12 patients in 2 studies and permitted a reduction in steroid use in most patients.Conclusions: Insufficient evidence means the role of Bevacizumab in the treatment of DIPG is unclear. However, Bevacizumab may be beneficial to some patients. The review highlights the need for further research in this area
Video analysis of parent-child interactions in behavioural sleep disorders: development of a scoring algorithm
No full textIntroduction: Behavioural sleep disorders, including chronic insomnia (CI), are generally assessed by subjective parent interview. However, evidence suggests that parental report of children’s overnight behaviours is unreliable, perhaps due to recall bias or confusion due to sleep deprivation. Video technology has been used clinically to capture complex behavioural disorders in children during the day. However, there is no standardised means of analysing child and parent behaviour at bedtime or during the night. We aimed to create an algorithm for this purpose.Methods: Child brain tumour survivors (a population previously shown to have a high prevalence of CI) were screened for difficulties initiating and maintaining sleep using sub-scales from the Sleep Disturbance Scale for Children. Those who screened positive (n=3) then completed a detailed parent interview to confirm a clinical diagnosis of CI. One night of home video footage was obtained from initial settling period to morning waking (SOMNOmedics camera). Footage was imported into BORIS© software and a coding system for parent and child behaviour was developed over multiple iterations until agreeable inter-rater reliability (>70%) was achieved between two independent coders.Results: The final coding categories were: 1) Time domains, 2) Physical environment, 3) Child global status, 4) Location, 5) Activity and 6) Physical interaction. This achieved 74% inter-reliability in its last iteration.Discussion: A statistically acceptable behaviour scoring algorithm was achieved. With further development, this tool could be applied clinically to investigate behavioural insomnia and in research to provide more objective outcome measurement
A novel home video behaviour analysis algorithm to diagnose childhood chronic insomnia
No full textObjectives/Introduction: Chronic insomnia (CI) is the most common sleep disorder in childhood, categorised as limit setting or sleep onset association type. In both, parent‐child interaction is significant in the development and persistence of the problem. CI is currently diagnosed by clinical interview with the parent, however parental recall of their own behaviour may be inaccurate. Objective observation may aid diagnosis and treatment. We aimed to trial home video technology in childhood brain tumour survivors who have a high prevalence of CI. Specifically, we aimed to develop a novel scoring algorithm of behavioural observations for future clinical applications.Methods: Participants were recruited from neuro‐oncology clinic sat Southampton Children's Hospital, UK. Inclusion criteria were: aged 3–12 years, completed brain tumour treatment at least 6 months previously, and at risk of CI according to a screening questionnaire (Sleep Disturbance Scale for Children: Disorders of Initiating and Maintaining Sleep subscale). Families completed a home video sleep study using an infra‐red camera with actigraphy (SOMNO medics), followed by a clinical sleep history taken from the parent (audio recorded to allow peer review). Video analysis was undertaken using BORIS v6.1, a software programme designed by researchers at University of Turin for animal behaviour observation. An analysis algorithm was designed and iteratively developed which categorised behaviours into the following domains: sleep schedule, bedtime routine,lights out, night wakings, parent and child speech and behaviour(sleep promoting and sleep inhibiting). These domains could both be visually plotted and quantitatively computed (e.g. total and mean duration of activities) to aid interpretation by the clinician.Results: Five children have been recruited. Inter‐rater reliability for scoring of sleep‐related behaviours will be presented. Preliminary video analysis has indicated that parents of childhood brain tumour survivors commonly demonstrate overly soothing behaviour at bedtime and night wakings (e.g. co‐sleeping and extended close physical contact), which is likely to contribute to CI in the child.Conclusions: Video behavioural analysis has the potential to be a useful tool in aiding diagnosis of CI and, particularly with the further development of our scoring algorithm, could be used to give parents specific feedback regarding their overnight interactions
DIPG-02. Role of a Bevacizumab-based regime in the treatment of children with diffuse intrinsic pontine glioma (DIPG): a systematic review
No full textBackground: diffuse intrinsic pontine glioma (DIPG) is an aggressive paediatric brainstem tumour. There are no effective treatments and median survival is 11.2 months, therefore maintaining quality of life (QOL) is a priority for many families. Bevacizumab, an anti-VEGF IgG antibody, has the potential to improve QOL and survival in DIPG, but has never been evaluated systematically. Aim: to collate the evidence and assess the role of this drug, in the treatment of DIPG. Methods: MEDLINE, EMBASE, Scopus and Web of Science were searched for relevant studies using terms developed from PICO criteria, including alternatives for Bevacizumab and DIPG. One reviewer screened titles and abstracts, then two reviewers screened full texts. Data were extracted into tables and study quality assessed using MINORS and JBI tools. RESULTS: Searching revealed 1,001 papers; after deduplication 851 remained. After screening of titles and abstracts, 28 full texts were screened, resulting in the inclusion of 11 studies. All studies evaluated more than one outcome. Four studies reported a median overall survival longer than historical data, however, two determined no significant impact. Five studies reported a radiological response in a proportion of participants and two reported no response. Three studies evaluating clinical response, reported improvement in a proportion of patients. Three studies evaluating QOL reported stability or improvement. Four studies evaluating steroid use reported reductions in the proportion of patients receiving steroids. In the treatment of radiation necrosis, Bevacizumab led to clinical improvement in 6/12 patients in 2 studies and permitted a reduction in steroid use in the majority of patients. Conclusion: insufficient evidence means the role of Bevacizumab in the treatment of DIPG is unclear. However, Bevacizumab may be beneficial to some patients. The review highlights the need for further research in this area, particularly randomised controlled trials. More effective therapies are desperately needed
QL-008. An exploration of associations between health related quality of life and medulloblastoma molecular subgroup status in survivors from the SIOP UKCCSG PNET3 trial
No full textOBJECTIVES: To explore the possibility of combining retrospective biological and quality of survival (QoS) data, to investigate whether health-related quality of life (HRQoL) may be related to the underlying biology of medulloblastoma. METHODS: Using available data from our previous studies of SIOP UKCCSG PNET3 survivors whose tumours had been assigned to disease molecular subgroup (SHH, WNT and non-SHH/WNT (Group 3 and Group 4) tumours) using DNA methylation and immuno-histochemical methods, and for whom HRQoL data were also available, we conducted univariate analyses to assess any differences in child- and parent-reported health status (HUI), behavioural functioning (SDQ), and HRQoL (PedsQL) between subgroups. This was followed by a three-step hierarchical forward multiple regression analysis; tumour subgroup was entered at step one followed by gender, age at diagnosis and interval from diagnosis (step two) and treatment and cerebellar mutism (step three) as predictors. Predictors were retained in the model if p < 0.1. RESULTS: There was a significant overall inter-group difference in parent-reported PedsQL (p = 0.018), due to significantly better PedsQL scores in the SHH group (median = 95.3) compared with the WNT group (median = 67.1, p = 0.015) and the non-SHH/WNT group (median = 74.0, p = 0.015). SHH trended to better functioning in all other indices used. At each step in the regression modelling, SHH remained the only significant predictor of parent-reported HRQoL, even after controlling for factors previously associated with worse HRQoL outcomes (treatment and cerebellar mutism) in the final model (B = 17.1, R2 = 0.25, p = 0.043). CONCLUSION: These initial investigations indicate combined analyses of biological and QoS data could provide new insights on HRQoL outcome, and should be incorporated into the planning of expanded studies of medulloblastoma survivors, aimed at fully establishing any basis to inform the future management of patients according to their molecular subgroup status
1185 Paediatric quality of life in congenital CMV: current knowledge and future directions
No full textBackground: congenital cytomegalovirus (cCMV) is the most common congenital infection. Around 25% of infants with cCMV develop permanent sequelae. However, there is a paucity of research evaluating the quality of life of children with cCMV.Objectives: this study evaluates current evidence regarding long-term effects of cCMV on affected children, and considers methodology used in quality of life studies that could be applied to cCMV and possible impacts on updated health economic evaluations of cCMV.Methods: we reviewed studies that reported long-term effects of cCMV on affected children, studies that measured quality of life in children with cCMV and similar paediatric populations, and studies that reported psychometric properties of quality of life measures.Results: the high prevalence of long-term sequelae amongst children with cCMV is likely to significantly impact quality of life of affected children and their families. Only one existing study assessed quality of life in children with cCMV and their parents, which was subject to bias and only incorporated one quality of life measure. We identified multiple quality of life measures that could be suitable for use in future studies of quality of life in cCMV, with varying psychometric properties and assessing different constructs.Conclusions: further research evaluating quality of life in cCMV is needed. To assess quality of life in cCMV, quality of life measures should be selected based on their relevance to sequelae of cCMV (for example, inclusion of a hearing-specific measure to assess the effect of sensorineural hearing loss), but generic quality of life measures are also important for comparison to the general population. Researchers should consider the importance of spillover effects on quality of life of family members as well as effects on the child’s own quality of life. A national multi-informant cross-sectional questionnaire-based study in the UK is being undertaken, aiming to describe quality of life of children with cCMV and their families. Quality of life data could be used to inform health economic analyses and decision-making regarding cost-effectiveness of interventions for cCMV prevention and treatment
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