Patient Reported Outcomes and Measures in Children with Rhabdomyosarcoma

SIMPLE SUMMARY: Children with rhabdomyosarcoma often experience difficulties as a result of their disease and treatment, such as pain or low mood. This can have a significant impact on their overall quality of life. It is important to evaluate these outcomes independently and prospectively, to improve the care provided for this population. One approach is to use questionnaires (or patient-reported outcome measures, PROMs) completed by patients. This commentary aims to encourage the use of PROMs by informing professionals in the field. The few available studies suggest that the quality of life of children with rhabdomyosarcoma is impaired. Additionally, children with rhabdomyosarcoma may have problems specifically related to their disease, for example due to their appearance after having surgery and/or radiotherapy. It is therefore important to develop questionnaires that include disease-specific issues. These can be used in addition to the generic quality of life questionnaires which are now more often used for children with cancer. ABSTRACT: 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.