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Vigabatrin (VGB) is approved as monotherapy for pediatric patients with Infantile Spasms (IS). Duration of VGB use should be limited because of the risk of retinal and neurotoxicity, but the optimal length of treatment is unknown. Our study aimed to determine the risk of spasms relapse after 6 months of VGB as first-line therapy in IS patients deemed VGB good responders. The participants were 44 infants with IS who demonstrated both absence of clinical spasms and hypsarrhythmia four weeks after starting VGB, obtained from two cohorts: 29 patients from a multicenter prospective cohort and 15 patients from a retrospective single-center cohort. We divided them post hoc into two groups according to the duration of VGB treatment: 6-month group (n=34) and >6-month group (n=10) and compared outcome between the two groups. No patient in either group had a relapse of spasms. For patients with non-identified etiology (NIE) in the 6 months treatment group, no other seizure types were observed. Late epilepsy, in the form of focal seizures, emerged in only 5/37 patients (3/30 in the 6-month treatment group; 2/7 in the extended treatment group); all within the first 6-9 months after VGB initiation. Our study provides substantial evidence that a shortened VGB course of 6 months could be sufficient to treat and prevent relapse of spasms in children with IS, particularly those with NIE., (Copyright © 2021 British Epilepsy Association. Published by Elsevier Ltd. All rights reserved.)

Introduction: While most paediatric urologists consider patients' quality of life (QOL) important, few actually measure this outcome. Our goal was to assess instruments used in the pediatric urology QOL literature, specifically looking at whether they captured QOL., Methods: We searched MEDLINE and EMBASE for articles with a self-described primary outcome of measuring QOL. All validated QOL instruments in the papers were analyzed by QOL instrument content experts. Instruments were classified as focusing on: Functioning or QOL (Table). The term Functioning focuses on performing activities. QOL captures person's perceptions about their position in life, informed by circumstances, functioning and conditions. QOL instruments were further subdivided into generic QOL, health-related QOL (HRQOL) and disease-specific HRQOL. Only direct patient self-reported QOL instruments were then assessed, since they are the most clinically useful, reliably assessing patients' own perception of their QOL., Results: Forty-three publications met inclusion criteria (published 1999-2019). Most common conditions included urinary incontinence (16, 37.2%) and kidney transplantation (12, 27.9%). Overall, 22 unique instruments purporting to measure QOL were identified. Looking at the concepts measured by each instrument, nine instruments (40.9%) assessed Functioning. Nine instruments (40.9%) measured a combination of Functioning and QOL. Only the remaining 4 instruments (18.2%) assessed strictly QOL. The 13 instruments assessing any QOL focused on generic QOL (n = 4), HRQOL (n = 3) and disease-specific HRQOL (n = 6). Of the subset of four instruments assessing strictly QOL, and not Functioning, all had patient self-reported versions available: two generic QOL instruments (KINDL, KIDSCREEN), one generic HRQOL (DISABKIDS), and one disease-specific HRQOL (QUALAS). Thirteen of 43 studies (30.2%) employed more than one instrument. Thirty-eight studies (88.4%) used an instrument measuring Functioning, with 19 (44.1%) measuring only Functioning, not QOL at all. Twenty-four studies (55.8%) used an instrument measuring actual QOL, although 17 (39.5%) used a combined Functioning/QOL instrument. Only nine (20.9%) used a strictly QOL instrument (strictly HRQOL instruments: 4.7%)., Discussion: We present encouraging evidence of sustained interest in QOL research in pediatric urology and identify areas needing improvement. Selecting appropriate QOL tools requires a working knowledge of their various underlying meanings and purposes. Whether it adequately assess QOL must be considered. We discuss strengths and weaknesses of instruments and a practical approach to QOL instrument selection., Conclusion: Much of pediatric urology is grounded in improving QOL. Unfortunately, most studies published to date focus on Functioning, rather than young people's perception-based QOL. Future QOL studies should ideally employ validated instruments capturing patient-reported QOL., Competing Interests: Conflict of interest The authors have no conflicts of interest relevant to this article to disclose., (Copyright © 2021 Journal of Pediatric Urology Company. Published by Elsevier Ltd. All rights reserved.)

Aim: To evaluate the long-term developmental trajectory of children with infantile spasms (IS) and identify the clinical protective and risk factors associated with their cognitive outcome., Methods: We analyzed the five-year follow-up results of 41 children (13 female) from the previously published cohort (n = 68) recruited in a multicenter randomized controlled trial for 2-years, examining the effect of an adjunctive therapy (Flunarizine) on standardized IS treatment. The children were subsequently monitored in an open-label study for additional 3 years.  The Vineland Adaptive Behavior Scale, second edition, and either the Stanford-Binet Intelligence Scale, Fifth Edition (SB5) or the Bayley Scales of Infant Development, second edition (BSID-II) were used as cognitive outcome measures., Results: Etiology was the strongest predictor of outcome. Children with no identified etiology (NIE) showed a progressive improvement of cognitive functions, mostly occurring between 2 and 5 years post-diagnosis.  Conversely, symptomatic etiology was predictive of poorer cognitive outcome. Developmental delay, other seizure types (before and after IS diagnosis), and persistent electroencephalographic abnormalities following treatment were predictive of poor cognitive outcome., Interpretation: Given the 5-year cognitive improvement, children with IS should undergo a developmental assessment before school entry. Factors influencing their cognitive outcome emphasize the importance of thorough investigation and evidence-based treatment., (Copyright © 2021 British Epilepsy Association. Published by Elsevier Ltd. All rights reserved.)

Objectives: Patient-reported outcomes are increasingly recommended to guide patient care, develop and evaluate interventions, and modify health systems. However, not enough is known about whether and how children and adolescents, as "experts" in their own health and quality of life (QoL), are being engaged in the development of instruments. Our goals in this review were (1) to identify all QoL-related instruments that have included children and/or adolescents in the development of questionnaire content, including identification of themes and items; and (2) to report how this was done; and (3) to highlight those that used qualitative methods., Methods: MEDLINE and Embase were searched for child- or adolescent-completed QoL-related instruments, supplemented by hand-searching of relevant reviews until 2020. Original development papers were identified and retrieved when possible, from which instrument characteristics and details of qualitative development methods were extracted., Results: We identified 445 instruments, of which 88 used qualitative methods for content development. Interviews and focus groups were the most common methods. A variety of play techniques were used to engage the child and adolescent participants. The specific criteria for the inclusion of children and adolescents (age, developmental stage, duration, and nonclinical location) varied considerably., Conclusions: Researchers frequently involve children and adolescents in qualitative methods when developing QoL-related measures; however, there is little information about the methods used. Better reporting of methodology, improved dissemination of methods guidelines, and research into optimal ways of including children and adolescents in the process of instrument development would be useful., (Copyright © 2020 ISPOR–The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

Objectives: To study the extent to which parents are able to serve as true proxies for their children with epilepsy using a more granular approach than has been found in any study to date., Methods: Proxy resemblance to the child was based on discrepancy in z-centered child minus parent scores of matching quality-of-life (QOL) domains for 477 dyads. Latent class mixed models (LCMMs) were built, with child's age as the independent variable for epilepsy-specific and generic QOL. Data were obtained from the QUALITÉ Canadian cohort, which recruited children with epilepsy aged 8 to 14 years at baseline and their parents., Results: Both epilepsy-specific and generic LCMMs produced latent classes representing proxies that were overly positive, overly negative, or in agreement relative to their children with posterior probabilities of 79% to 84%. The "agreement" classes had N = 411 and N = 349 in the epilepsy-specific and generic LCMMs, respectively. The epilepsy-specific LCMM had a small unique class of N = 5 with a posterior probability of 88% called "growing discrepancy.", Conclusions: Most parents of children with epilepsy can serve as valid proxies for their children on QOL scales. Poorer parental adaptation is more related to overly negative proxies, whereas low peer support from the child's perspective is more related to overly positive proxies., (Copyright © 2019 ISPOR–The Professional Society for Health Economics and Outcomes Research. Published by Elsevier Inc. All rights reserved.)

In this chapter we argue that modern thinking about chronic neurological and developmental conditions requires that we recognize all aspects of functioning and quality of life in addition to the biomedical dimensions of these disorders. We find the International Classification of Functioning, Disability, and Health (ICF) a useful framework by which to think about and understand the many dimensions of health; and we appreciate its heuristic value as a stimulus to consider a range of outcomes of these individuals. Quality of Life (QOL) and Health-Related Quality of Life (HRQL) cover the additional perceived dimensions that are so important to patients' personal valuations, aspirations, and satisfaction about health throughout their lifespan, of which we see QOL being the broader construct. In order to measure outcomes at either the individual clinical or research level it is essential to ask clear and specific questions as a prelude to selecting measures that are appropriate in terms of both their content and their measurement properties. We provide some brief guidelines that we hope will be helpful to readers who wish to expand their activities in measuring clinical outcomes., (Copyright © 2013 Elsevier B.V. All rights reserved.)

The practice of pediatric neurology demands a high level of responsibility at multiple levels. These include listening carefully to people's stories in order to assess each situation, planning and implementing investigations and therapies, individual and family counseling, longitudinal follow-up from fetal life throughout childhood and adolescence, organization of transition to adult care, and societal advocacy. In the 21st century these activities must be carried out in the context of major societal and technological changes which have brought about many new challenges for pediatric neurologists. In this chapter, we address ethical and moral issues that may help guide pediatric neurologists with regard to a number of specific challenges. These include physician-patient relationships that are based on benign paternalism with respect for autonomy and promoting quality of life, practicing evidence-based medicine, and the technological imperative. In addition we discuss the tension between clinical practice and research, relationships between physicians and industry, and the public role of pediatric neurologists to advocate for children with neurological and developmental conditions. We also illustrate some challenges in selected situations such as prenatal counseling (fetal neurology), neonatal encephalopathy, and persistent vegetative state., (Copyright © 2013 Elsevier B.V. All rights reserved.)