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Future, Volume 2, Issue 2 (June 2024) – 2 articles

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11 pages, 258 KiB  
Review
Outcome Measures of Clinical Trials in Pediatric Chronic Kidney Disease
by Ziyun Liang, Guohua He, Liyuan Tao, Xuhui Zhong, Tianxin Lin, Xiaoyun Jiang and Jie Ding
Future 2024, 2(2), 56-66; https://doi.org/10.3390/future2020005 - 6 May 2024
Viewed by 559
Abstract
Clinical trials of chronic kidney disease (CKD) in children have important implications for the early identification and management of CKD. The selection of clinical trial outcomes is critical for assessing the effectiveness of interventions in pediatric CKD clinical trials. This review systematically examines [...] Read more.
Clinical trials of chronic kidney disease (CKD) in children have important implications for the early identification and management of CKD. The selection of clinical trial outcomes is critical for assessing the effectiveness of interventions in pediatric CKD clinical trials. This review systematically examines the spectrum of outcome measures deployed in pediatric CKD clinical trials, which includes clinical and alternative outcomes, patient-reported outcome measures (PROMs), and safety indicators. Alternative outcome measures were stratified into four levels of evidence strength: convincing, probable, suggestive, and inconclusive. Consequently, the selection of outcome measures for pediatric CKD clinical trials mandates careful consideration of both their methodological feasibility and the robustness of their evidence base. Moreover, the burgeoning field of PROMs warrants integration into the design of future pediatric clinical trials to enrich the relevance and impact of research findings. Full article
10 pages, 785 KiB  
Case Report
Supporting Functional Goals in Spinal Muscular Atrophy: A Case Report of The Cognitive Orientation to Daily Occupational Performance (CO-OP) Approach
by Stephanie Taylor, Iona Novak and Michelle Jackman
Future 2024, 2(2), 46-55; https://doi.org/10.3390/future2020004 - 18 Apr 2024
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Abstract
Children with spinal muscular atrophy (SMA) are now living longer as a result of advancements in pharmaceutical and medical interventions. There is a paucity of research regarding therapeutic interventions to support this population to be independent and participate in life activities that are [...] Read more.
Children with spinal muscular atrophy (SMA) are now living longer as a result of advancements in pharmaceutical and medical interventions. There is a paucity of research regarding therapeutic interventions to support this population to be independent and participate in life activities that are most important to them. The aim of this case report is to explore the use of the Cognitive Orientation to daily Occupational Performance (CO-OP) approach to support a child with SMA type 1 to achieve their functional and participation goals. This is a retrospective case study. A 7-year-old girl with SMA type 1 received ten 1 h sessions of CO-OP, weekly in the home and community settings with a physiotherapist. Clinically meaningful improvements were found in goal performance and satisfaction on the Canadian Occupational Performance Measure (COPM) and Performance Quality Rating Scale (PQRS). Despite the progressive nature of SMA, the CO-OP approach was able to support goal attainment. Given medical advances are leading to a longer life span for children with neuromuscular conditions, further research is needed to investigate the efficacy of functional and participation-based interventions, including impact on quality of life and self-efficacy. Full article
(This article belongs to the Special Issue Child Physical Activity and Health)
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