EJC paediatric oncology最新文献

{"title":"Dexamethasone/vincristine pulses significantly reduce administered doses of 6MP/MTX during maintenance therapy in childhood acute lymphoblastic leukemia","authors":"Valérie de Haas,&nbsp;Esmee Slot ,&nbsp;Laura van den Berg ,&nbsp;Rana Dandis,&nbsp;Hester de Groot-Kruseman,&nbsp;Rob Pieters","doi":"10.1016/j.ejcped.2025.100243","DOIUrl":"10.1016/j.ejcped.2025.100243","url":null,"abstract":"<div><h3>Background</h3><div>Maintenance therapy with 6-Mercaptopurine (6MP) and Methotrexate (MTX) with or without dexamethasone (DEX) and vincristine (VCR) pulses, is crucial in childhood acute lymphoblastic leukaemia (ALL). Dosing adjustments are based on white blood cell (WBC) and neutrophil counts (ANC) to balance relapse and infection risks. We investigated if clinicians adjust doses for high versus low WBC/ANC and how pulses affect WBC/ANC and dose intensity.</div></div><div><h3>Methods</h3><div>Analyzing WBC/ANC and 6MP/MTX doses in DCOG-ALL11 (ALL11) and ALLtogether (A2G) patients, we assessed clinical compliance with dose-adjustment guidelines.</div></div><div><h3>Results</h3><div>Across 2901 WBC checkpoints in ALL11 and 3806 in A2G (validation cohort), clinicians were non-compliant with dose-adjustment guidelines in 44 % (ALL11) and 31 % (A2G) of cases.</div><div>At checkpoints where no-compliance was observed, WBC/ANC levels were most frequently above target (85.1 % in ALL11 and 87.7 % in A2G) and rarely below (6.4 % in ALL11 and 6.7 % in A2G, p &lt; 0.001).Median WBC/ANC levels often exceeded recommendations (3.1 and 1.6 ×109/L, respectively) and patients receiving DEX/VCR pulses had a lower median dose 6MP / MTX (60 %) than those without pulses (100 % ALL11, 75 % A2G; P &lt; 0.001), resulting in lower absolute doses.</div></div><div><h3>Conclusions</h3><div>In conclusion, clinicians are less likely to adjust doses properly when WBC/ANC levels exceed targets, reducing 6MP/MTX dose intensity. DEX/VCR pulses significantly lower 6MP/MTX doses, possibly influencing their benefit in ALL maintenance.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100243"},"PeriodicalIF":0.0,"publicationDate":"2025-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144178757","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"SSPedi and SPARK: Promise for the future of electronic patient reported outcomes in pediatric oncology","authors":"Adam P. Yan ,&nbsp;Deborah Tomlinson ,&nbsp;L. Lee Dupuis ,&nbsp;Lillian Sung","doi":"10.1016/j.ejcped.2025.100238","DOIUrl":"10.1016/j.ejcped.2025.100238","url":null,"abstract":"<div><h3>Objective</h3><div>Progress toward incorporating patient-reported outcomes (PROs) into the routine clinical care of pediatric cancer patients has accelerated over the last decade. Objective was to review general PRO considerations for pediatric cancer patients, describe the impact of PROs from adult cancer trials, summarize Symptom Screening in Pediatrics Tool (SSPedi) suite development and validation, and highlight recent clinical trials evaluating SSPedi implementation.</div></div><div><h3>Methods</h3><div>This review addressed the following topics: instrument choice, respondent type, lessons learned from adult oncology trials, SSPedi tools, recent randomized controlled trials (RCTs) of symptoms screening using SSPedi, and future directions.</div></div><div><h3>Results</h3><div>Instrument choice should be guided by the purpose of the assessment, where reasons include monitoring for adverse effects during cancer treatment clinical trials, choice as primary or secondary outcomes for supportive care clinical trials, and enabling routine symptom screening to enhance clinical care. While pediatric patient self-report of subjective symptoms is ideal, it may not always be feasible and careful consideration of respondent type is required. Instruments developed for use by different respondent types will likely be required for clinical implementation. Both adult and pediatric trials have demonstrated the value of symptom screening and support its adoption into routine clinical care. Two recent RCTs have shown that administration of SSPedi can improve symptom control in pediatric cancer patients.</div></div><div><h3>Conclusions</h3><div>There have been many advancements in PROs for pediatric cancer patients. Identifying approaches to feasibly implement symptom screening into routine clinical care is required. In the future, routine monitoring of symptoms should be a measure of quality of care.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100238"},"PeriodicalIF":0.0,"publicationDate":"2025-05-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143941259","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of COVID-19 pandemic on childhood cancer incidence and stage in France – A national registry-based study","authors":"Maud Gédor ,&nbsp;Brigitte Lacour ,&nbsp;Sandra Guissou ,&nbsp;Laure Faure ,&nbsp;Claire Poulalhon ,&nbsp;François Doz ,&nbsp;Arnaud Petit ,&nbsp;Jérémie Rouger ,&nbsp;Virginie Gandemer ,&nbsp;Emmanuel Désandes ,&nbsp;Jacqueline Clavel","doi":"10.1016/j.ejcped.2025.100240","DOIUrl":"10.1016/j.ejcped.2025.100240","url":null,"abstract":"<div><div>COVID-19 pandemic has considerably affected access to healthcare resources with global decline in cancer care activities in 2020. This national study aimed to assess a possible impact of the pandemic on the management of pediatric cancer cases.</div></div><div><h3>Methods</h3><div>The study was based on the French National Childhood Cancer Registry and included all cases of cancer under 18 years of age diagnosed between January 1, 2016 and December 31, 2020. We estimated incidence rates, the proportion of advanced/metastatic stages (Toronto Pediatric Cancer Stage Guidelines) and the distribution of treatment initiation time during the 2020 pandemic period compared to the 2016–2019 reference period.</div></div><div><h3>Results</h3><div>Age-standardized incidence rates of overall pediatric cancer were similar in 2020 (161.4 cases per million person-years; 2250 cases) and in 2016–2019 (162.4 cases per million person-years; 9208 cases). They were also similar by sex, age group, region, and cancer type. We did not observe any significant differences in stage at diagnosis or median time to treatment.</div></div><div><h3>Conclusion</h3><div>Our nationwide population-based study suggests that pediatric cancer management was not substantially altered in 2020, despite the challenges induced by the COVID-19 pandemic and associated lockdown.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100240"},"PeriodicalIF":0.0,"publicationDate":"2025-05-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143941187","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A structured approach for data collection and visualization of the International Childhood Cancer Core Outcomes","authors":"R.J. van Kalsbeek ,&nbsp;L.H. Grundeken ,&nbsp;R.L. Mulder ,&nbsp;M.M. Hudson ,&nbsp;M.J. Ehrhardt ,&nbsp;J.G. den Hartogh ,&nbsp;R.J. Riezebos ,&nbsp;H. van Tinteren ,&nbsp;W.J.W. Kollen ,&nbsp;M.A. Grootenhuis ,&nbsp;R. Pieters ,&nbsp;L.C.M. Kremer ,&nbsp;M. van der Heiden-van der Loo","doi":"10.1016/j.ejcped.2025.100239","DOIUrl":"10.1016/j.ejcped.2025.100239","url":null,"abstract":"<div><h3>Background</h3><div>Insight into clinically relevant outcomes for children with cancer is crucial for improving treatment and follow-up strategies. The International Childhood Cancer Core Outcome Project developed a harmonized core outcome set, involving patients and providers across all types of childhood cancer.</div></div><div><h3>Objective</h3><div>This paper highlights the strategy used at the Princess Máxima Center for pediatric oncology for the collection and visualization of the International Childhood Cancer Core Outcomes.</div></div><div><h3>Project phases</h3><div>In phase 1, we created an overview of data elements related to patient data, cancer diagnosis, and outcomes, and mapped them to openEHR archetypes to optimize data processing. To classify cancer diagnosis according to the predefined 17 tumor subgroups, the International Classification of Childhood Cancer, Third Edition (ICCC-3) was used. In phase 2, we defined a data processing plan to extract these data elements from Electronic Health Records (EHR) and additional existing data sources. In phase 3, we designed a reporting dashboard to visualize results for healthcare professionals.</div></div><div><h3>Future perspectives and considerations</h3><div>Local plans include further automation of data processing, unlocking missing data sources, and adding treatment data. Having a dedicated team and using openEHR with other data (coding) standards facilitates the process and future benchmarking opportunities.</div></div><div><h3>Conclusion</h3><div>Developing a structured approach for data collection and visualization of the International Childhood Cancer Core Outcomes is an extensive process involving multiple steps. We describe our strategy to facilitate the monitoring of improvements in quality of care in childhood cancer centers and allow future benchmarking of the quality of care.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100239"},"PeriodicalIF":0.0,"publicationDate":"2025-05-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144115609","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Global consensus from the PHITT consortium surrounding an interim chemotherapy guidance for the treatment of children with hepatoblastoma, hepatocellular neoplasm – not otherwise specified (HCN-NOS), hepatocellular carcinoma and fibrolamellar carcinoma after trial closure","authors":"Irene Schmid ,&nbsp;Allison F. O’Neill ,&nbsp;Kenichiro Watanabe ,&nbsp;Isabelle Aerts ,&nbsp;Sophie Branchereau ,&nbsp;Penelope R. Brock ,&nbsp;Madhumita Dandapani ,&nbsp;Brice Fresneau ,&nbsp;Jim Geller ,&nbsp;Tomoro Hishiki ,&nbsp;Danielle Ingham ,&nbsp;Kathelijne Kraal ,&nbsp;Milind Ronghe ,&nbsp;Greg Tiao ,&nbsp;Angela Trobaugh-Lotrario ,&nbsp;Arun Rangaswami ,&nbsp;Michael J. Sullivan ,&nbsp;Jozsef Zsiros ,&nbsp;Eiso Hiyama ,&nbsp;Marcio Malogolowkin ,&nbsp;Marc Ansari","doi":"10.1016/j.ejcped.2025.100235","DOIUrl":"10.1016/j.ejcped.2025.100235","url":null,"abstract":"<div><div>Malignant liver tumors affecting children and adolescents are rare. In order to adequately study liver tumors occurring in this patient population and establish a more uniform approach to therapy, the three primary liver tumor consortia, SIOPEL (mainly European), COG (mainly North American) and JCCG (Japan), in concert, designed an international prospective clinical trial [PHITT (Paediatric Hepatic International Tumour Trial)/COGAHEP1531/JPLT4 – to be referred to as PHITT from this point forth] for the study of hepatoblastoma and hepatocellular carcinoma in children and adolescents. Recruitment to PHITT has recently concluded. The major results from this trial will be published in the next 2–3 years, therefore in the interim, we sought to provide a consensus statement on evidence-based chemotherapy guidance for hepatoblastoma and hepatocellular carcinoma of all stages.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100235"},"PeriodicalIF":0.0,"publicationDate":"2025-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143941188","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Near-infrared photoimmunotherapy using antiGD2 antibody for neuroblastoma and osteosarcoma","authors":"Jimei Zhao ,&nbsp;Kohei Nakajima ,&nbsp;Masahiro Ueki ,&nbsp;Yukayo Terashita ,&nbsp;Shinsuke Hirabayashi ,&nbsp;Yuko Cho ,&nbsp;Mikako Ogawa ,&nbsp;Atsushi Manabe","doi":"10.1016/j.ejcped.2025.100233","DOIUrl":"10.1016/j.ejcped.2025.100233","url":null,"abstract":"<div><h3>Introduction</h3><div>Near-infrared photoimmunotherapy (NIR-PIT) is a novel cancer therapy that specifically targets cancer cells. In NIR-PIT, antibody-photo-absorber-conjugates (APCs), specific for cancer antigens, target cancer cells. APCs that bind to antigens and are irradiated with NIR-light kill targeted cells. NIR-PIT has been clinically applied to head and neck squamous cell carcinoma in Japan, but not to pediatric cancers. Generally, neuroblastoma and osteosarcoma express disialoganglioside (GD2), making NIR-PIT that targets GD2 an additional treatment option.</div></div><div><h3>Methods</h3><div>Antihuman GD2 monoclonal antibody (clone 3F8) was conjugated with photo-absorber, IRDye^Ⓡ 700DX (IR700). A human neuroblastoma cell (SK-N-SH) was used for <em>in vitro</em> and <em>in vivo</em> experiments, whereas human osteosarcoma cells (NOS-10, MG-63) were utilized for <em>in vitro</em> experiments. Microscopy and flow cytometry were used to evaluate cell death after NIR-PIT, and longitudinal measurement of tumor size and histological observation were utilized to assess the effect of NIR-PIT on the xenograft model.</div></div><div><h3>Results</h3><div>EthD-1 staining after NIR-PIT confirmed the morphological changes (swelling and bleb formation) observed by microscopy and cell death. Flow cytometry verified the significant increase in dead cells after NIR-PIT. Tumor growth in the mice model was significantly inhibited at day 10 of the experiment and destroyed tumor tissue was histologically observed in the PIT-treated mice.</div></div><div><h3>Conclusion</h3><div>The efficacy of NIR-PIT with antiGD2 antibody for neuroblastoma and osteosarcoma was indicated. Additionally, NIR-PIT efficacy against neuroblastoma was indicated by the mice model <em>in vivo</em>. Therefore, further experiments and more effective protocols should be considered for clinical application.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100233"},"PeriodicalIF":0.0,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143941189","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cognitive and adaptive functioning outcomes after cancer diagnosis in early childhood: A systematic review","authors":"Jessica Beamish,&nbsp;Josephine Drijver,&nbsp;Marita Partanen","doi":"10.1016/j.ejcped.2025.100237","DOIUrl":"10.1016/j.ejcped.2025.100237","url":null,"abstract":"<div><h3>Objectives</h3><div>About one quarter of children who are diagnosed with cancer are under four years old, which is a uniquely critical period in neurodevelopment. Younger age at diagnosis is a known risk factor for poorer long-term cognitive and functional outcomes, indicating that the combined impact of cancer and treatment may disproportionally affect this age group. To date, synthesis of the literature in this area has been limited by heterogeneous findings and methodologies. To help guide clinical practice and future research, this review aimed to describe the cognitive and adaptive functioning outcomes of young children with cancer, as well as summarise the impact of risk factors.</div></div><div><h3>Methods</h3><div>We conducted a systematic review, according to PRISMA guidelines, of cognitive and adaptive functioning outcomes in cancer patients who were younger than four years at diagnosis. We searched PubMed, EMBASE and PsycINFO and included relevant articles. This review was registered in PROSPERO [CRD42024502629].</div></div><div><h3>Results</h3><div>There were 5747 records retrieved, and 54 papers were included after full-text review, representing 8083 participants. Long-term impairments in the domains of processing speed, attention, executive and adaptive functioning were reported in brain tumour, haematological and solid tumour groups. Brain tumour survivors also show deficits in IQ. Cranial radiotherapy was the most consistently associated risk factor with poorer cognitive and adaptive functioning. Risk of bias analysis showed most studies were classified as ‘strong’ quality, but there was variability in study sample size and design.</div></div><div><h3>Conclusion</h3><div>Children diagnosed with cancer in early childhood are at high risk of cognitive and adaptive functioning deficits, potentially reflecting diffuse neurodevelopmental injury. However, outcomes are highly variable, with limited understanding of risk factors beyond medical treatment. The results support the recommended guidelines for early neuropsychological monitoring for all diagnostic groups to identify vulnerable patients and to implement timely interventions.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100237"},"PeriodicalIF":0.0,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143927981","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Dabrafenib and trametinib either in combination or as monotherapy in pediatric patients harboring BRAF alterations in gliomas or other rare tumors: Findings from a managed access program","authors":"Mark Russo ,&nbsp;AnaRita Ferreira ,&nbsp;Michael Roughton ,&nbsp;Sviatlana Rizk ,&nbsp;Naveen Chhabra ,&nbsp;Manar Aoun","doi":"10.1016/j.ejcped.2025.100236","DOIUrl":"10.1016/j.ejcped.2025.100236","url":null,"abstract":"<div><h3>Background</h3><div>To enable global access to therapies in countries where suitable formulations and/or indications are not yet approved, managed access programs (MAPs) are established. Herein, we report patient data from a MAP for pediatric patients with <em>BRAF-</em>altered gliomas or other rare tumors, seeking clinical benefit from dabrafenib and/or trametinib.</div></div><div><h3>Methods</h3><div>We analyzed data from global MAP, where pediatric patients with <em>BRAF</em> alterations in gliomas or other rare tumors received dabrafenib and/or trametinib. Key eligibility criteria were age &lt; 18 years, absence of approved alternative therapies, presence of <em>BRAF</em> alterations, and exhausted standard therapies. Requests analyzed in this study were collected from April 2015 to December 2023 (cutoff date). The main objectives were to describe patients’ characteristics and treatment duration.</div></div><div><h3>Results</h3><div>Overall, 863 patients with solid malignancies were included, of which 30.4 %, 23.4 %, and 46.2 % received combination therapy, dabrafenib monotherapy and trametinib monotherapy, respectively. Median age was 6.4 years, and 51 % were male. Most patients had low-grade (80.9 %) or high-grade (5.6 %) gliomas, followed by Langerhans' cell histiocytosis (13.6 %). The median treatment duration (mDoT) was &gt; 12 months (ranged between 12.4 and 45.9 months) irrespective of the treatment groups, disease types, and formulations. At the data cutoff, treatment was ongoing for approximately 47 % of patients.</div></div><div><h3>Conclusions</h3><div>MAP data are a valuable real-world resource for understanding patient characteristics in rare tumors such as pediatric gliomas. A significant number of treatment requests indicate substantial unmet need, and observed mDoT across subgroups suggests potential treatment benefit in these patients. No new safety findings were identified for dabrafenib/trametinib.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100236"},"PeriodicalIF":0.0,"publicationDate":"2025-05-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143912176","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Diagnosis of a congenital nodule labelled pigmented epithelioid melanocytoma unconfirmed by recent molecular biology tools","authors":"Caroline Donzé,&nbsp;Nathalie Degardin,&nbsp;Nicolas Ortonne,&nbsp;Nicolas Macagno,&nbsp;Arnauld Verschuur","doi":"10.1016/j.ejcped.2025.100234","DOIUrl":"10.1016/j.ejcped.2025.100234","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100234"},"PeriodicalIF":0.0,"publicationDate":"2025-05-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143927982","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Assessing patient-reported outcomes (PROs) in paediatric oncology research: Which PRO would a pro pick, if a pro was picking PROs?","authors":"Ameeta Retzer ,&nbsp;Sarah Al-Jilaihawi ,&nbsp;Shivani Bailey ,&nbsp;Vickie Buenger ,&nbsp;Sarah Damery ,&nbsp;Anne-Sophie Darlington ,&nbsp;Manuel Diezi ,&nbsp;John Devin Peipert ,&nbsp;Jessica Roydhouse ,&nbsp;Bryce B. Reeve","doi":"10.1016/j.ejcped.2025.100232","DOIUrl":"10.1016/j.ejcped.2025.100232","url":null,"abstract":"<div><div>Patient-reported outcomes (PROs) enable the report of experiences that are only known to the patient, such as how an individual’s symptoms, functioning, and quality of life are impacted by a health condition or treatment. The choice of PRO depends on the intended application and associated research questions, and structuring a rationale for the use of PRO data is key to deciding upon a PRO strategy. Rates of PRO use in paediatric oncology research remain low though the general use of PROs in clinical trials has been gradually increasing. This is likely to have risen due to increased emphasis by regulatory agencies to capture outcomes meaningful to patients. Despite increasing interest in PROs, a range of barriers to their use have been identified by researchers exploring their implementation and a pattern of under-reporting of PRO data has also been observed in general oncology trials. Research with children entails specific challenges, including the need for developmentally-appropriate outcome measures, the complexities of family involvement, and the adaptations required in research procedures and settings to accommodate children's physical, cognitive, and emotional development. With the nascent use of PROs in paediatric oncology, there are opportunities to benefit from learning from trials involving adult participants. The current paper outlines considerations for PRO selection for use in paediatric oncology research, describing five steps to determine the PRO study goals, the study outcomes, the reporter(s), measures, and study implementation. We discuss the breadth of applications of PROs in paediatric oncology research and potential for further learning and harmonisation with the aim of centering children’s experiences.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100232"},"PeriodicalIF":0.0,"publicationDate":"2025-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143891842","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}