Tigelaar Leonie G, Haveman Lianne M, Bekkering Willem P, Oude Lansink Irene L B, Rohrich Christel D, Van der Hoek Hinke, Beek Laura R, Van Dijk Jennifer, Langemeijer Marjolein E M, Slooff-Lentink Relinde W, Van der Aa-Van Delden Alied M, Maurice-Stam Heleen, Peek Annemarie M L, Van der Pal Helena J H, Koopman Maria M W, Kremer Leontien C M, Westerbos Stijn J, Van Tinteren Harm, Bramer Jos A M, Van de Sande Michiel A J, Grootenhuis Martha A, Schreuder Hendrik W B, Merks Johannes H M
Princess Máxima Center for Pediatric Oncology, Utrecht, Netherlands.
Department of Pediatric Rehabilitation, Wilhelmina Children's Hospital, University Medical Center Utrecht, Utrecht, Netherlands.
Front Pediatr. 2025 Apr 15;13:1534153. doi: 10.3389/fped.2025.1534153. eCollection 2025.
Bone sarcoma patients face intensive treatment, including life-changing local therapy, which impacts both short- and long-term functioning. Moreover, bone sarcoma survivors experience the highest burden of adverse events of all childhood cancer survivors. To address these issues, we set up a structured multidisciplinary outpatient follow-up clinic for patients who completed treatment and integrated this clinic into the standard of care. This study protocol describes the methodology of a cross-sectional study that aims to systematically report the functional outcomes, adverse events, psychosocial outcomes and health-related quality of life of the cohort seen at this clinic.
Participants are recruited at the multidisciplinary follow-up clinic and their consent is obtained. Standard of care clinical assessments serve as the primary data source for this study. Furthermore, additional research assessments are performed to further expand our knowledge. Assessments are structured by standardized assessment sets that we developed based on literature review and joint national expertise in bone sarcoma care. The sets comply with international guidelines such as the World Health Organization's International Classification of Functioning, disability and health, and include a combination of patient-reported, clinician-reported and performance-based outcome measures for comprehensive representation of outcomes.
This study will generate valuable knowledge on the functional outcomes, adverse events, psychosocial outcomes and quality of life of a national cohort of pediatric bone sarcoma patients in follow-up care. By aligning additional research assessments with standardized patient care, a comprehensive range of outcomes will be obtained while minimizing the patient's burden. Moreover, this protocol may serve as a template for clinics and research internationally, allowing for the merging of standardized outcome data in such rare disease. This will facilitate the optimization of current patient care and inform the important shared decision-making process for local treatment in future patients.
骨肉瘤患者面临强化治疗，包括改变生活的局部治疗，这会影响短期和长期功能。此外，骨肉瘤幸存者在所有儿童癌症幸存者中经历的不良事件负担最高。为了解决这些问题，我们为完成治疗的患者设立了一个结构化的多学科门诊随访诊所，并将该诊所纳入标准治疗流程。本研究方案描述了一项横断面研究的方法，该研究旨在系统报告在该诊所接受治疗的队列患者的功能结局、不良事件、心理社会结局以及与健康相关的生活质量。
在多学科随访诊所招募参与者并获得他们的同意。标准治疗临床评估作为本研究的主要数据源。此外，还进行了额外的研究评估以进一步拓展我们的认知。评估由我们基于文献综述和骨肉瘤护理方面的国家联合专业知识制定的标准化评估集构建。这些评估集符合国际指南，如世界卫生组织的《国际功能、残疾和健康分类》，并包括患者报告、临床医生报告和基于表现的结局测量相结合的方式，以全面呈现结局。
本研究将产生关于全国儿科骨肉瘤患者随访护理队列的功能结局、不良事件、心理社会结局和生活质量的宝贵知识。通过将额外的研究评估与标准化患者护理相结合，在最小化患者负担的同时将获得全面的结局范围。此外，本方案可作为国际诊所和研究的模板，允许在这种罕见疾病中合并标准化结局数据。这将有助于优化当前的患者护理，并为未来患者的局部治疗重要的共同决策过程提供信息。
