通过整合功能评估减轻血管闭塞性危象的负担,加强对小儿镰状细胞病的管理

Paul Muteb Boma, Alain Ali Kaponda, Jules Panda, Bruno Bonnechère
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摘要

镰状细胞病(SCD)对健康造成了巨大负担,尤其是在医疗保健专业人员和资源匮乏的中低收入国家。本意见书深入探讨了针对 SCD 儿童患者血管闭塞性危象(VOCs)所采用的管理策略,提倡采用变革性策略。我们探讨了将功能评估方法融入现有程序的问题,强调了技术辅助康复的潜力,包括可穿戴传感器和数字生物标志物,以提高管理和预防 VOC 的有效性。康复组学作为一个综合框架,将康复相关数据与生物标志物相结合,为个性化治疗干预提供了基础。尽管这些方法具有前景广阔的优势,但长期存在的障碍,如康复计划的可用性有限,尤其是在资源有限的环境中,构成了挑战。本文强调了采取合作策略以有效解决 SCD 患者所面临的独特障碍的重要性。这种合作方法包括改善康复服务的可及性、纳入技术支持疗法以及促进重点研究工作。这种综合方法的主要目标是加强对 SCD 患者的整体护理,重点是预防 VOC,以及在资源有限的环境中提供量身定制的(神经)康复服务。通过审视 SCD 管理现状并提出变革性战略,本意见书旨在激励集体行动与合作,以改善全球儿科 SCD 患者的预后。
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Enhancing the Management of Pediatric Sickle Cell Disease by Integrating Functional Evaluation to Mitigate the Burden of Vaso-Occlusive Crises
Sickle cell disease (SCD) imposes a significant health burden, particularly in low- and middle-income countries where healthcare professionals and resources are scarce. This opinion paper delves into the management strategies employed for vaso-occlusive crises (VOCs) in pediatric patients with SCD, advocating for the adoption of a transformative strategy. We explore the integration of functional assessment approaches into existing procedures, highlighting the potential of technology-assisted rehabilitation, including wearable sensors and digital biomarkers, to enhance the effectiveness of managing and preventing VOCs. Rehabilomics, as a comprehensive framework, merges rehabilitation-related data with biomarkers, providing a basis for personalized therapeutic interventions. Despite the promising advantages of these approaches, persistent obstacles such as the limited availability of rehabilitation programs, especially in resource-limited settings, pose challenges. This paper underscores the importance of a collaborative strategy to effectively address the unique obstacles faced by patients with SCD. This collaborative approach involves improving accessibility to rehabilitation services, incorporating technology-supported therapy, and fostering focused research endeavors. The primary objective of this comprehensive approach is to enhance the overall care of SCD patients, with a specific focus on preventing VOCs, as well as providing tailored (neuro)rehabilitation services in resource-limited settings. By examining the current state of SCD management and proposing transformative strategies, this opinion paper seeks to inspire collective action and collaboration to improve outcomes for pediatric SCD patients globally.
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