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Patient Reported Outcomes and Measures in Children with Rhabdomyosarcoma
Marloes van Gorp1, Martha A Grootenhuis1, Anne-Sophie Darlington2
1Princes Máxima Center for Pediatric Oncology, 3584 CS Utrecht, The Netherlands.
Insights
Improving quality of life (QOL) for children with rhabdomyosarcoma (RMS) is crucial. This commentary highlights the need for patient-reported outcome measures (PROMs) to assess QOL and symptoms in pediatric RMS research and care.
Area of Science:
- Pediatric Oncology
- Quality of Life Research
- Patient-Reported Outcomes
Background:
- Optimizing survival for children with rhabdomyosarcoma (RMS) remains a priority.
- Increasing focus on improving quality of life (QOL) and symptom management during and after treatment for pediatric RMS patients.
- Patient-reported outcomes (PROs) and patient-reported outcome measures (PROMs) are essential for assessing QOL and symptoms.
Purpose of the Study:
- To encourage the use of PROMs in pediatric RMS by informing professionals about available measures.
- To provide considerations for the future implementation of PROMs in RMS research and clinical practice.
- To address the scarcity of PROM utilization in the current pediatric RMS literature.
Main Methods:
- Literature review of existing PROMs applicable to pediatric RMS.
- Analysis of current reporting of PROMs in pediatric RMS studies.
- Discussion of challenges and opportunities for PROM implementation.
Main Results:
- Existing literature indicates lower QOL in children with RMS compared to general populations.
- Disease-specific symptoms are frequently reported in pediatric RMS patients.
- There is a notable lack of a rhabdomyosarcoma-specific PROM.
Conclusions:
- There is a critical need to increase the use of PROMs in pediatric RMS research and clinical practice.
- Ongoing developments include creating RMS-specific PROMs and integrating PROM evaluation into clinical trials.
- Utilizing PROMs will enhance the understanding and management of QOL and symptoms in children with RMS.
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.
