Understanding Clinical Effectiveness and Safety Implications of Botulinum Toxin in Children: A Narrative Review of the Literature.

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Abstract

[Indexed for MEDLINE] Conflict of interest statement: GT has served, over the last 3 years, on advisory boards/seminars funded by Sanofi, MSD, Eli Lilly, Sobi, Celgene, Daichii Sankyo, Novo Nordisk, Gilead, and Amgen; he is also a scientific coordinator of the academic spin-off “INSPIRE srl”, which has received funding from several pharmaceutical companies (i.e., PTC Pharmaceuticals, Kiowa Kirin, Shonogi, Shire, Novo Nordisk, and Daichii Sankyo) for conducting observational studies. Additionally, he is currently a consultant for Viatris in a legal case concerning an adverse reaction to sertraline. None of these listed activities are related to the topic of the article. The other authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in, or financial conflict with, the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties. 14. J Patient Rep Outcomes. 2022 Jun 2;6(1):58. doi: 10.1186/s41687-022-00469-4. Quality of patient- and proxy-reported outcomes for children with impairment of the upper extremity: a systematic review using the COSMIN methodology. Kalle JPR(1), Saris TFF(2), Sierevelt IN(3), Eygendaal D(4), van Bergen CJA(2). Author information: (1)Utrecht University, Heidelberglaan 8, 3584CS, Utrecht, The Netherlands. jprkallepub@gmail.com. (2)Amphia Hospital, Molengracht 21, 4818CK, Breda, The Netherlands. (3)Stichting SCORE, Laarderhoogtweg 12, 1101AE, Amsterdam, The Netherlands. (4)Erasmus MC, Dr. Molewaterplein 40, 3015GD, Rotterdam, The Netherlands. BACKGROUND: As patient-reported outcome measures (PROMs) have become of significant importance in patient evaluation, adequately selecting the appropriate instrument is an integral part of pediatric orthopedic research and clinical practice. This systematic review provides a comprehensive overview of PROMs targeted at children with impairment of the upper limb, and critically appraises and summarizes the quality of their measurement properties by applying the COnsensus-based Standards for selection of health Measurement INstruments (COSMIN) methodology. METHODS: A systematic search of the MEDLINE and EMBASE databases was performed to identify relevant publications reporting on the development and/or validation of PROMs used for evaluating children with impairment of the upper extremity. Data extraction and quality assessment (including a risk of bias evaluation) of the included studies was undertaken by two reviewers independently and in accordance with COSMIN guidelines. RESULTS: Out of 6423 screened publications, 32 original articles were eligible for inclusion in this review, reporting evidence on the measurement properties of 22 self- and/or proxy-reported questionnaires (including seven cultural adaptations) for various pediatric orthopedic conditions, including cerebral palsy (CP) and obstetric brachial plexus palsy (OBPP). The measurement property most frequently evaluated was construct validity. No studies evaluating content validity and only four PROM development studies were included. The methodological quality of these development studies was either 'doubtful' or 'inadequate'. The quantity and quality of the evidence on the other measurement properties of the included questionnaires varied substantially with insufficient sample sizes and/or poor methodological quality resulting in significant downgrading of evidence quality. CONCLUSION: This review provides a comprehensive overview of currently available PROMs for evaluation of the pediatric upper limb. Based on our findings, none of the PROMs demonstrated sufficient evidence on their measurement properties to justify recommending the use of these instruments. These findings provide room for validation studies on existing pediatric orthopedic upper limb PROMs (especially on content validity), and/or the development of new instruments. © 2022. The Author(s). DOI: 10.1186/s41687-022-00469-4 PMCID: PMC9163282