EJC paediatric oncology最新文献

{"title":"Artificial intelligence in paediatric cancer: Insights from innovation experts in the UNICA4EU project","authors":"Pilar Gangas ,&nbsp;Norbert Graf ,&nbsp;Shuping Wen ,&nbsp;Carlotta Cattaneo ,&nbsp;Marilena Bicchieri ,&nbsp;Niamh Lennox-Chhugani","doi":"10.1016/j.ejcped.2024.100208","DOIUrl":"10.1016/j.ejcped.2024.100208","url":null,"abstract":"<div><div>This article presents the results of interviews with AI development and innovation experts, focusing on the potential contribution of AI to paediatric cancer treatment, its barriers and facilitators. AI-based technologies are expanding in health care provision and potentially to paediatric oncology, particular imageology. However, no AI based technology specifically developed for paediatric cancer has been identified. Interviews identified key barriers, including legal, regulatory and ethical challenges; validation and evaluation standards; integration with public healthcare systems; acceptance, explainability and trust of AI technologies; digital literacy and skills development; data management and privacy Protection; and promoting multidisciplinary collaboration. Facilitators largely coincide, including legal, regulatory and ethical considerations; data management and storage; building trust and ensuring privacy; engaging key stakeholders; promoting multidisciplinary collaboration through AI technologies; and education, training and innovation programmes. Barriers can be turned into facilitators if properly managed. These interviews were conducted under the EU funded project UNICA4EU (Towards a UNIque approach for artificial intelligence data-driven solutions to fight Childhood cAncer FOR EUrope)(1), which was implemented between 2022 and 2024. The goal of the overall project was to analyse the current landscape of Artificial Intelligence (AI) and to map applications to Childhood Cancer. This work further develops and confirms the results of a previous EU-funded pilot project, A crowdsourced ecosystem to fight childhood cancer (EU4CHILD)(1).</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100208"},"PeriodicalIF":0.0,"publicationDate":"2024-12-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143104302","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Superior outcome but high incidence of pseudoprogression and unfavorable neurocognitive outcome in children with embryonal tumor with multylayered rosettes treated with radiation and high-dose chemotherapy with tandem autologous stem cell rescue","authors":"Elena Carceller ,&nbsp;Felisa Vázquez-Gómez ,&nbsp;Sara Sirvent ,&nbsp;José Luis Moreno ,&nbsp;Marta González-Vicent ,&nbsp;Borja Esteso ,&nbsp;Luis Madero ,&nbsp;Alvaro Lassaletta","doi":"10.1016/j.ejcped.2024.100207","DOIUrl":"10.1016/j.ejcped.2024.100207","url":null,"abstract":"<div><h3>Objective</h3><div>Embryonal tumor with multilayered rosettes (ETMR) is a rare and highly aggressive embryonal brain tumor in young children. Treatment of this tumor often includes maximal safe resection, radiotherapy, and high-dose chemotherapy (HDCT) with autologous stem cell transplantation (ASCT). Early radiation has been recommended according to recent outcome results. The combination of this multimodal treatment may provoke radiographic changes that can mimic tumor progression. This phenomenon is known as pseudoprogression.</div></div><div><h3>Methods</h3><div>We report four consecutive patients under 5 years old with ETMR, treated according to CCG 99703 protocol (which includes conventional chemotherapy and HDCT with ASCT). Off protocol, radiation was given before (three patients) or after (one patient) high-dose chemotherapy.</div></div><div><h3>Results</h3><div>All patients developed pseudoprogression. All patients but one presented symptoms such as irritability, hypotonia, headache and vomiting. Severe neurocognitive impairment was evident in all patients. At a median follow-up of 50.5 months (range, 37–64), three of the patients are alive. Patients who are alive, continue to require significant multidisciplinary support to address treatment sequelae.</div></div><div><h3>Conclusion</h3><div>This case series demonstrates that, while survival rates appear encouraging, combining radiation therapy with high-dose chemotherapy and autologous stem cell transplantation (ASCT) in the treatment of embryonal tumors with multilayered rosettes may carry a substantial risk of pseudoprogression and notable neurological damage.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100207"},"PeriodicalIF":0.0,"publicationDate":"2024-12-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143104360","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"KMT2A-rearranged acute lymphoblastic leukaemia","authors":"Rishi S. Kotecha ,&nbsp;Rob Pieters ,&nbsp;Janine Stutterheim","doi":"10.1016/j.ejcped.2024.100204","DOIUrl":"10.1016/j.ejcped.2024.100204","url":null,"abstract":"<div><div><em>KMT2A</em>-rearranged acute lymphoblastic leukaemia (ALL) represents a high risk subtype of childhood ALL. Historical treatment strategies have comprised of intensification with conventional chemotherapy. However, outcomes have remained consistently poor compared to the advances that have been seen for other ALL subtypes, particularly for infants diagnosed before their first birthday. The advent of novel immunotherapeutic approaches has led to a change in the treatment paradigm, with the integration of blinatumomab to the current suite of clinical trials for <em>KMT2A</em>-rearranged ALL expected to result in marked improvements. Furthermore, significant progress has been made to understand the unique biology of <em>KMT2A</em>-rearranged ALL, which has led to the development of novel agents that directly target the <em>KMT2A</em> complex or dysregulated proteins/pathways. Clinical trials are currently poised to evaluate therapies such as venetoclax and menin inhibitors, offering further hope for achieving a cure. In this review, we discuss the remarkable progress that has been made for <em>KMT2A</em>-rearranged ALL, leading to much optimism for improved outcomes in the future.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"4 ","pages":"Article 100204"},"PeriodicalIF":0.0,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143181358","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Validation of the stratification for newly diagnosed hepatoblastoma: An analysis from the Children’s Hepatic tumors International Collaboration (CHIC) database","authors":"Arun A. Rangaswami ,&nbsp;Angela D. Trobaugh-Lotrario ,&nbsp;Rudolf Maibach ,&nbsp;Allison F. O’Neill ,&nbsp;Daniel C. Aronson ,&nbsp;Rebecka L. Meyers ,&nbsp;Mark D. Krailo ,&nbsp;Jin Piao ,&nbsp;Eiso Hiyama ,&nbsp;Tomoro Hishiki ,&nbsp;Marc Ansari ,&nbsp;Dolores Lopez-Terrada ,&nbsp;Piotr Czauderna ,&nbsp;Marcio Malogolowkin ,&nbsp;Beate Häberle","doi":"10.1016/j.ejcped.2024.100206","DOIUrl":"10.1016/j.ejcped.2024.100206","url":null,"abstract":"<div><h3>Introduction</h3><div>The Children’s Hepatic Tumors International Collaboration (CHIC) previously developed the CHIC hepatoblastoma risk stratification (CHIC-HS) model by analyzing the original CHIC dataset (development set). A validation of the results was planned with data from contemporary trials that were ongoing at the time of the initial analysis.</div></div><div><h3>Material and methods</h3><div>Subsequent analysis was performed on the contemporary trials (validation set) SIOPEL 4, SIOPEL 6, COG AHEP0731, and JPLT 2 (2011–2013 cohort) utilizing the same methodology as in the CHIC-HS development.</div></div><div><h3>Results</h3><div>403 patients were available for analysis for validation of the CHIC-HS model. Clinical characteristics were comparable between the validation and development sets. Event free survival (EFS) was similar in the very low and low-risk CHIC-HS strata in both the development and validation sets. EFS was higher in the intermediate and high-risk strata in the validation set as compared with the development set consistent with improved survival of intermediate-risk patients on AHEP0731 and high-risk patients on SIOPEL 4.</div></div><div><h3>Conclusions</h3><div>The four risk groups in the CHIC-HS retain their relevance for risk stratification in this analysis, notwithstanding improved outcomes in patients with higher risk hepatoblastoma; therefore, the CHIC-HS remains a valid model which differentiates patients into statistically distinct risk strata. The model will be further evaluated with data from the current pediatric international hepatic tumors trial (PHITT).</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100206"},"PeriodicalIF":0.0,"publicationDate":"2024-11-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143171739","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Medulloblastoma therapy: Consensus treatment recommendations from SIOP-Europe and the European Reference Network","authors":"S. Bailey ,&nbsp;S. Jacobs ,&nbsp;M. Kourti ,&nbsp;M. Massimino ,&nbsp;N. Andre ,&nbsp;F. Doz ,&nbsp;C. Dufour ,&nbsp;S. Vennarini ,&nbsp;L. Padovani ,&nbsp;K. Aquilina ,&nbsp;U. Thomale ,&nbsp;A. Joshi ,&nbsp;T. Pietsch ,&nbsp;S. Avula ,&nbsp;G. Morana ,&nbsp;S. Rutkowski ,&nbsp;B. Pizer ,&nbsp;SC Clifford","doi":"10.1016/j.ejcped.2024.100205","DOIUrl":"10.1016/j.ejcped.2024.100205","url":null,"abstract":"<div><div>The treatment of medulloblastoma has evolved considerably over the last 10 years. Treatment intensity is now stratified within clinical trials, using risk-associated clinical features and molecular biomarkers, aimed at maximising cure rates whilst minimising long-term disease and therapy-associated side-effects. In Europe, we have developed a long-term strategy of using randomised trials to test stratified treatments across all medulloblastoma disease demographics, and to investigate further stratification using biological analysis of the samples collected. Importantly, care must be taken not to adopt experimental arms from trial protocols into routine practice, prior to trials’ results being available. Moreover, there are time periods when trials are not open to recruitment for all the various risk-groups. These guidelines, developed under the auspices of SIOP-Europe and the European Reference Network for Paediatric Oncology, ERN PaedCan, review recent and current trials, alongside the literature, to provide evidence-based guidance for the contemporary therapy of medulloblastoma.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100205"},"PeriodicalIF":0.0,"publicationDate":"2024-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143172278","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Potential of non-FDG PET radiotracers for paediatric patients with solid tumours","authors":"Leonor Teles ,&nbsp;Nelleke Tolboom ,&nbsp;Sabine L.A. Plasschaert ,&nbsp;Alex J. Poot ,&nbsp;Arthur J.A.T. Braat ,&nbsp;Max M. van Noesel","doi":"10.1016/j.ejcped.2024.100203","DOIUrl":"10.1016/j.ejcped.2024.100203","url":null,"abstract":"<div><div>Molecular imaging with positron emission tomography (PET) offers significant potential for improving diagnostic accuracy, staging and treatment monitoring in paediatric solid tumours, by using radiopharmaceuticals more specific than [¹⁸F]fluorodeoxyglucose ([¹⁸F]FDG). While non-[¹⁸F]FDG tracers have already improved diagnostic abilities in adult solid cancers such as prostate and neuroendocrine tumours, their use in the paediatric population has been underexplored. This narrative review summarises clinical evidence regarding the use, advantages, and limitations of these more specific PET tracers in paediatric patients. In neuroblastoma, [¹⁸F]mFBG, [¹⁸F]F-DOPA, and SSTR-targeting peptides stand out as the most evolved and promising tracers for the clinical setting, with encouraging results regarding feasibility, safety and detection rates. SSTR-targeting peptides have also consistently outperformed other imaging methods (both conventional and functional) and carry the benefits of theragnostic applications. For brain tumours, amino acid-based tracers in general stand out due to their ability to surpass the blood-brain barrier/blood-tumour barrier (BBB/BTB) and their specific accumulation in malignant tissue. Other paediatric solid tumours, such as sarcoma and bone tumours, suffer from a clear lack of clinical evidence that should be addressed in the near future. The studies performed to date show high accuracy, evident prognostic value, and significant clinical impact of non-[¹⁸F]FDG tracers in paediatric patients with solid tumours. However, prospective studies with longer follow-up times are warranted to provide high-level evidence regarding the impact of these tracers on patient management and prognosis, to consolidate the encouraging results obtained so far. Further research and international collaboration will be essential to overcome current challenges related to low incidence of paediatric solid tumours, logistical barriers and concerns about radiation exposure.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"4 ","pages":"Article 100203"},"PeriodicalIF":0.0,"publicationDate":"2024-11-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142721418","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Eight-year national multicenter experience on the use of glucarpidase as effective rescue therapy for delayed methotrexate elimination after high-dose methotrexate cycles administered in children with hemato-oncological diseases","authors":"Nicolò Peccatori ,&nbsp;Marta Coppola ,&nbsp;Antonella Colombini ,&nbsp;Daniela Silvestri ,&nbsp;Nicoletta Bertorello ,&nbsp;Valentina Kiren ,&nbsp;Fraia Melchionda ,&nbsp;Rosamaria Mura ,&nbsp;Daniela Onofrillo ,&nbsp;Simona Gobbi ,&nbsp;Raffaele Mattera ,&nbsp;Luciana Vinti ,&nbsp;Tommaso Casini ,&nbsp;Nicola Santoro ,&nbsp;Domenico Sperlì ,&nbsp;Carmelita D’Ippolito ,&nbsp;Valentino Conter ,&nbsp;Andrea Biondi ,&nbsp;Carmelo Rizzari","doi":"10.1016/j.ejcped.2024.100202","DOIUrl":"10.1016/j.ejcped.2024.100202","url":null,"abstract":"<div><h3>Background</h3><div>High-dose methotrexate (HDMTX) for cancer treatment can be complicated by delayed methotrexate elimination (DME) and associated acute kidney injury (AKI). This study evaluated glucarpidase for the treatment of DME and HDMTX-AKI in pediatric hemato-oncology patients.</div></div><div><h3>Methods</h3><div>This multicenter, retrospective study reviewed the medical records of pediatric patients (1–18 years) with ALL or NHL who were given glucarpidase as rescue therapy for DME or HDMTX-AKI at 13 Italian AIEOP centers between January 1, 2015, and June 30, 2023. Patients also received uniform supportive therapy, per study protocols and guidelines, to prevent and treat AKI.</div></div><div><h3>Results</h3><div>Data were available for 42/44 patients given glucarpidase, following i.v. HDMTX as monotherapy (non-high risk ALL [non-HR ALL], n=24), or within combined intensive chemotherapy blocks (HR-ALL, n=13; NHL, n=5). Median time to glucarpidase infusion was 53 hours (range 32–72). Most patients required glucarpidase during the first cycle of HDMTX. Glucarpidase led to a rapid decrease in plasma MTX levels (median 72.53 %; range 12.62–94.57 %). Median time for complete elimination of MTX and its metabolites was 216 hours (range 120–672). Recovery of renal function (return of serum creatinine to ≤1.5 times baseline value) took a median 18 days (range 4–72). Of the 22/42 patients (52 %) re-challenged with HDMTX (at 40–100 % of recommended dose), none experienced DME and all completed per-protocol number of HDMTX cycles.</div></div><div><h3>Conclusions</h3><div>Our experience shows that glucarpidase is effective and well tolerated for the treatment of DME and HDMTX-AKI in pediatric patients with hemato-oncologic diseases.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"5 ","pages":"Article 100202"},"PeriodicalIF":0.0,"publicationDate":"2024-11-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143104359","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"New insights into hematopoietic cell transplantation in ALL: Who should be transplanted, when, and how","authors":"Daisuke Tomizawa ,&nbsp;Evgenios Goussetis","doi":"10.1016/j.ejcped.2024.100200","DOIUrl":"10.1016/j.ejcped.2024.100200","url":null,"abstract":"<div><div>Advancements in multi-agent chemotherapy through clinical trials conducted by multi-institutional collaborative groups worldwide, along with risk stratification using molecular genetic features and treatment responses, including minimal residual disease, have significantly improved outcomes for children and adolescents with acute lymphoblastic leukemia (ALL) over the past half-century. However, for a certain number of high-risk patients, including those with relapsed or refractory disease, for whom existing chemotherapy alone is insufficient for cure, allogeneic hematopoietic cell transplantation (HCT) has provided a potential opportunity for leukemia cure. For these patients, the appropriate selection of donor and stem cell source, conditioning regimen, timing of transplantation, and comprehensive supportive care, including effective graft-versus-host disease prophylaxis, are prerequisites for successful HCT. While HCT from a human leukocyte antigen (HLA)-matched sibling has traditionally been the preferred option, less than 25 % of patients currently have such a donor in developed countries. Consequently, alternative donor HCT options, such as those from matched unrelated donors identified through high-resolution HLA typing, unrelated cord blood donors, and more recently, haploidentical donors using post-transplant cyclophosphamide or TCRαβ+/CD19+ cell-depleted grafts, are providing broader access to HCT for patients lacking matched sibling donors. Nonetheless, HCT carries the risk of various acute and late toxicities. In particular, the use of myeloablative conditioning with total body irradiation, a standard in pediatric ALL, is associated with significant long-term sequelae. As our understanding of the pathophysiology of the disease improves and novel molecular targeted therapies and immunotherapies are developed, the indication for HCT in pediatric ALL is becoming more selective, leading to a gradual decrease in the number of transplants performed. However, further optimization and evolution of allogeneic HCT are needed to both maximize its anti-leukemia effects and minimize transplant-related complications, as there remain cases that undoubtedly require HCT for the cure of leukemia.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"4 ","pages":"Article 100200"},"PeriodicalIF":0.0,"publicationDate":"2024-10-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142571256","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Patients’, parents’, and survivors’ perspective about AI applications in pediatric oncology","authors":"Hyseni Bocolli Albina,&nbsp;Schneider Carina,&nbsp;Bastos Pais Teresa,&nbsp;Willi Michaela,&nbsp;Brunmair Mattias","doi":"10.1016/j.ejcped.2024.100201","DOIUrl":"10.1016/j.ejcped.2024.100201","url":null,"abstract":"<div><div>Patients, parents and survivors of childhood cancer are those who represent the critical link between the clinical and technical research spheres. Thus, UNICA4EU worked towards a patient- centric approach to integrate AI in the care pathways for childhood cancer, with evidence-based patient advocacy at its core to build trust while protecting and guaranteeing patients’ fundamental rights <span><span>[1]</span></span>.</div><div>The task “Increase Knowledge and Transparency about AI among patients, parents and survivors” was led by CCI Europe as the biggest pan-European childhood cancer parents’ and survivors’ organization which represents childhood cancer parents´ and survivors´ groups and other childhood cancer organizations and reunites 63 member-organizations in 34 countries <span><span>[2]</span></span>.</div><div>To investigate the knowledge base of AI application in pediatric oncology among those who are affected, a survey was conducted. The survey translated into nine European languages, gathered responses from 332 individuals. To delve deeper into the survey findings, discussions were held with a diverse focus group, including four parents of childhood cancer former patients (survivors), three childhood cancer survivors, and one bereaved parent, each representing different backgrounds, age groups, and countries.</div><div>Insights and outcomes of this study produced a report for guiding the multi-stakeholder board of the project when defining the governance structures reg. data sharing, ownership, protection, access and usage.</div><div>Perspective of parents, patients and survivors of pediatric cancer regarding AI applications in Pediatric Oncology focused in six areas of interest including: data anonymization and data protection, data ownership, data withdrawal, ethical concerns of use of data, data types and, additionally, informed consents. This paper summarizes the respective results, along concluding policy recommendations.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"4 ","pages":"Article 100201"},"PeriodicalIF":0.0,"publicationDate":"2024-10-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142586831","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Enhancing pediatric oncology clinical trials through patient reported outcomes (PROs)","authors":"David Riedl ,&nbsp;Niclas Hubel ,&nbsp;Annalena Endres ,&nbsp;Chiara Vetrano ,&nbsp;Andreas Meryk ,&nbsp;Roman Crazzolara","doi":"10.1016/j.ejcped.2024.100199","DOIUrl":"10.1016/j.ejcped.2024.100199","url":null,"abstract":"<div><div>Survival rates of children with cancer have significantly increased over the last decades in high income countries. However, cancer survivors often face significant long-term adverse effects on physical, psychosocial, and neurocognitive health. The use of Patient-Reported Outcomes (PROs) in clinical trials may help to further optimize treatment regimens to minimize acute and late adverse events. Despite clear recommendations of regulatory agencies, the use of PROs in pediatric clinical trials remains limited. In this article, we discuss the rationale to assess PROs in pediatric clinical trials with a specific highlight on electronic PRO (ePRO) assessment. In addition, we underscore the importance of recent international guideline developments such as the <em>Setting International Standards in Analyzing Patient-Reported Outcomes and Quality of Life Endpoints Data</em> (SISAQOL-IMI) and the <em>CONsolidated Standards of Reporting Trials – Patient-Reported Outcomes</em> (CONSORT-PRO) to improve the robustness of PRO data assessment in pediatric oncology, thus facilitating better patient care and more accurate assessments of treatment effects. Best practice examples for the use of modern technologies in ePRO assessments are presented and potential barriers and challenges are discussed. We conclude that – despite some challenges and barriers – the routine assessment of (e)PROs in pediatric clinical trials has the potential to substantially improve the quality of the trials and facilitate the usability of outcome data. We call for a joint effort to take proactive steps to incorporate PROs into clinical trials in pediatric oncology, thus giving children a voice.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"4 ","pages":"Article 100199"},"PeriodicalIF":0.0,"publicationDate":"2024-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142533856","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}