The Use of Patient-Reported Outcome Measures in Paediatric Haematopoietic Stem Cell Transplant: A Systematic Review

Children and adolescents undergoing Haematopoietic Stem Cell Transplantation (HSCT) experience complex symptoms, often under-reported by patients and undetected by clinicians, which cause distress. Patient-Reported Outcome Measures (PROMs) offer a way to capture symptom experiences directly from patients, with the potential of supporting effective symptom assessment and management, yet their routine use in paediatric HSCT remains unclear. This systematic review synthesises evidence on PROMs used during inpatient paediatric HSCT care, examining their role in symptom monitoring and clinical decision-making, and identifying gaps to strengthen person-centred, developmentally appropriate care.

We searched the MEDLINE, CINAHL, Embase, APA PsychINFO, and Cochrane Library in October 2024 for studies published in English between 2014 and 2025 describing the use of PROMs during inpatient paediatric (0-18 years) HSCT admission (up to Day +100 post HSCT). In March 2025, prior to data extraction, we added additional studies published by authors of included studies. Two-stage independent screening and data extraction were conducted, and the Quality Assessment with Diverse Studies (QuADS) tool was used to appraise each study. Narrative syntheses informed by Symptom Management Theory were used to compare PROM use, clinical integration, and reported impacts.

Seventeen studies met inclusion criteria, describing 20 PROMs used during paediatric HSCT hospitalisation. PROMs captured a wide range of physical and psychological symptoms, with pain and nausea most frequently reported. While PROMs reportedly improve symptom detection and communication, integration into routine paediatric HSCT clinical care was rare; and only two studies systematically used PROMs data to guide symptom management. Evidence of PROMs-driven improvements in HSCT clinical outcomes was scarce, and longitudinal data on symptom trajectories were limited.

PROMs are not routinely used to inform clinical practice in paediatric HSCT, and current evidence provides only a partial understanding of symptom trajectories and lived symptom experiences during the paediatric acute transplant admission. To realise the full potential of PROMs in enhancing symptom assessment and management, systematic PROMs integration into clinical workflows is required, supported by electronic health record integration, clinician training, and longitudinal research designs that capture symptom evolution across the transplant continuum.