van Gorp Marloes, Grootenhuis Martha A, Darlington Anne-Sophie, Wakeling Sara, Jenney Meriel, Merks Johannes H M, Hjalgrim Lisa Lyngsie, Adams Madeleine
Princes Máxima Center for Pediatric Oncology, 3584 CS Utrecht, The Netherlands.
School of Health Sciences, University of Southampton, Southampton SO17 1BJ, UK.
Cancers (Basel). 2023 Jan 9;15(2):420. doi: 10.3390/cancers15020420.
In addition to optimising survival of children with rhabdomyosarcoma (RMS), more attention is now focused on improving their quality of life (QOL) and reducing symptoms during treatment, palliative care or into long-term survivorship. QOL and ongoing symptoms related to the disease and its treatment are outcomes that should ideally be patient-reported (patient-reported outcomes, PROs) and can be assessed using patient-reported outcome measures (PROMS). This commentary aims to encourage PRO and PROM use in RMS by informing professionals in the field of available PROMs for utilisation in paediatric RMS and provide considerations for future use in research and clinical practice. Despite the importance of using PROMs in research and practice, PROMs have been reported scarcely in paediatric RMS literature so far. Available literature suggests lower QOL of children with RMS compared to general populations and occurrence of disease-specific symptoms, but a lack of an RMS-specific PROM. Ongoing developments in the field include the development of PROMs targeted at children with RMS specifically and expansion of PROM evaluation within clinical trials.
除了优化横纹肌肉瘤（RMS）患儿的生存率外，现在更多的注意力集中在改善他们的生活质量（QOL）以及减少治疗期间、姑息治疗期间或长期存活期的症状。生活质量以及与疾病及其治疗相关的持续症状是理想情况下应由患者报告的结果（患者报告结果，PROs），并且可以使用患者报告结果测量工具（PROMS）进行评估。本评论旨在通过向该领域的专业人员介绍可用于儿科RMS的PROMS，鼓励在RMS中使用PRO和PROM，并为未来在研究和临床实践中的使用提供考虑因素。尽管在研究和实践中使用PROM很重要，但到目前为止，儿科RMS文献中几乎没有关于PROM的报道。现有文献表明，与一般人群相比，RMS患儿的生活质量较低，且存在特定疾病的症状，但缺乏针对RMS的PROM。该领域正在进行的发展包括专门针对RMS患儿开发PROM，以及在临床试验中扩大PROM评估。
