EJC paediatric oncology最新文献

{"title":"Osteosarcomas in retinoblastoma-survivors. A report of 28 affected patients from the Cooperative Osteosarcoma Study Group (COSS)","authors":"Stefan S. Bielack ,&nbsp;Daniel Baumhoer ,&nbsp;Stefanie Hecker-Nolting ,&nbsp;Simone Hettmer ,&nbsp;Leo Kager ,&nbsp;Petra Ketteler ,&nbsp;Matthias Kevric ,&nbsp;Christian P. Kratz ,&nbsp;Thomas Kühne ,&nbsp;Vanessa Mettmann ,&nbsp;Markus Metzler ,&nbsp;Dirk Reinhardt ,&nbsp;Benjamin Sorg ,&nbsp;Claudia Blattmann","doi":"10.1016/j.ejcped.2024.100158","DOIUrl":"https://doi.org/10.1016/j.ejcped.2024.100158","url":null,"abstract":"<div><h3>Purpose</h3><p>Many publications address the epidemiology of secondary osteosarcomas following retinoblastoma. Treatment- and outcome-related information is, however, scarce. We used a large cooperative group’s database to study this issue.</p></div><div><h3>Patients and methods</h3><p>The database Cooperative Osteosarcoma Study Group COSS was searched for patients with osteosarcoma following a previous retinoblastoma. Patients were then analyzed for demographic factors, local/systemic treatments received, and outcomes.</p></div><div><h3>Results</h3><p>28 eligible patients were identified. Median age at retinoblastoma was .5 years, 89% occurred bilaterally. Retinoblastoma-therapy was by surgery in 26/27, radiotherapy in 26/27, chemotherapy in 10/26 (rest unknown). Osteosarcoma was diagnosed after 13.7 (3.1 – 36.1) years, extremities and head/neck affected in 14/28, each. Four/28 patients had primary metastases. Osteosarcoma treatment included chemotherapy in all cases, local therapy surgery in 27/28. Histological response was good in 9/16 evaluable cases. Surgery of all sites was macroscopically complete in 23/27 operated tumors and microscopically complete in 17/26 (1 unknown). Radiotherapy was administered to 3 craniofacial tumors. Median follow-up was 3.5 (.4 – 30.1) years from osteosarcoma diagnosis, 8/28 patients remaining event-free. Altogether, five patients suffered further secondary malignancies. Actuarial overall and event-free survival at 2 and 5 years from osteosarcoma were 73% (standard error: 8%) / 50% (10%) and 47% (10%) / 22% (9%), respectively.</p></div><div><h3>Conclusion</h3><p>This comparatively large cohort of osteosarcomas after retinoblastoma proves that the latter may be treated curatively. While their prognosis is far worse than that of primary osteosarcomas, partly due to a predilection for craniofacial involvement, selected patients may still become long-term survivors with appropriate therapies.</p></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-03-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2772610X24000175/pdfft?md5=f181efe2921dcefac8343255b80f04b8&pid=1-s2.0-S2772610X24000175-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140342197","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":"CAR T cells redirected to B7-H3 for pediatric solid tumors: Current status and future perspectives","authors":"Rebecca Epperly,&nbsp;Stephen Gottschalk,&nbsp;Christopher DeRenzo","doi":"10.1016/j.ejcped.2024.100160","DOIUrl":"https://doi.org/10.1016/j.ejcped.2024.100160","url":null,"abstract":"<div><p>Despite intensive therapies, pediatric patients with relapsed or refractory solid tumors have poor outcomes and need novel treatments. Immune therapies offer an alternative to conventional treatment options but require the identification of differentially expressed antigens to direct antitumor activity to sites of disease. B7-H3 (CD276) is an immune regulatory protein that is expressed in a range of malignancies and has limited expression in normal tissues. B7-H3 is highly expressed in pediatric solid tumors including osteosarcoma, rhabdomyosarcoma, Ewing sarcoma, Wilms tumor, neuroblastoma, and many rare tumors. In this article we review B7-H3-targeted chimeric antigen receptor (B7-H3-CAR) T cell therapies for pediatric solid tumors, reporting preclinical development strategies and outlining the landscape of active pediatric clinical trials. We identify challenges to the success of CAR T cell therapy for solid tumors including localizing to and penetrating solid tumor sites, evading the hostile tumor microenvironment, supporting T cell expansion and persistence, and avoiding intrinsic tumor resistance. We highlight strategies to overcome these challenges and enhance the effect of B7-H3-CAR T cells, including advanced CAR T cell design and incorporation of combination therapies.</p></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2772610X24000199/pdfft?md5=c747012f8719471c331ca17f416f2eef&pid=1-s2.0-S2772610X24000199-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140188205","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":"A narrative review of 35 years of meta-[131I]iodobenzylguanidine therapy in neuroblastoma","authors":"Atia Samim ,&nbsp;Gitta Bleeker ,&nbsp;Kathelijne C.J.M. Kraal ,&nbsp;Max M. van Noesel ,&nbsp;Bart de Keizer ,&nbsp;Godelieve A.M. Tytgat","doi":"10.1016/j.ejcped.2024.100159","DOIUrl":"https://doi.org/10.1016/j.ejcped.2024.100159","url":null,"abstract":"<div><p>Neuroblastoma is the most common extracranial solid malignancy of childhood. Approximately half of the patients have high-risk neuroblastoma (HR-NBL), typically presenting as widespread metastatic disease at diagnosis. Despite aggressive multimodality treatment, patients with HR-NBL have a long-term survival rate of below 50%. This is primarily due to frequent progression and relapse, which often proves to be therapy resistant. To overcome therapy resistance in HR-NBL, researchers are exploring diverse treatment strategies, including radionuclide therapy. Radiolabelled meta-iodobenzylguanidine (mIBG) has served as a theranostic (<u>thera</u>peutic and diag<u>nostic</u>) radiopharmaceutical in the field of neuroblastoma for several decades. [¹²³I]mIBG scintigraphy is recognized as the international standard to evaluate disease dissemination at diagnosis and to monitor treatment response. In contrast, the role of [¹³¹I]mIBG therapy in the management of neuroblastoma is less clear. Over the past 35 years, [¹³¹I]mIBG therapy has been studied in more than 1500 patients with neuroblastoma. In initial studies, [¹³¹I]mIBG monotherapy was applied as a second-line treatment in patients who failed first-line treatment. In current applications, [¹³¹I]mIBG therapy is combined with chemotherapy, radiosensitizers, and/or immunotherapy, and is increasingly integrated in the first-line treatment of HR-NBL. This narrative review provides an overview of the literature on [¹³¹I]mIBG therapy in HR-NBL. Studies show that [¹³¹I]mIBG therapy can be an effective treatment in one-third of patients with acceptable toxicity. Further investigations, particularly randomized controlled trials, are needed to determine the efficacy and optimal use of [¹³¹I]mIBG therapy in HR-NBL.</p></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-03-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2772610X24000187/pdfft?md5=3bf5965d3b5582c858f1f73cd517bf67&pid=1-s2.0-S2772610X24000187-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140190891","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":"Japan’s contribution to an ongoing global pediatric cancer clinical trial: The experience of the National Cancer Center Hospital (NCCH) in Tokyo","authors":"Miho Nakajima,&nbsp;Ayumu Arakawa,&nbsp;Chitose Ogawa","doi":"10.1016/j.ejcped.2024.100157","DOIUrl":"https://doi.org/10.1016/j.ejcped.2024.100157","url":null,"abstract":"<div><p>Compared to Europe and the United States, where the development of pediatric oncology drugs is mandated by law, Japan hosts fewer clinical trials. This can lead to a significant drug lag, especially in the case of treatments for pediatric solid tumors. Notably, regulatory authorities and the government have initiated discussions on strategies to mitigate Japan’s drug lag problem in pediatric oncology. Over the past decade, we have actively sought opportunities to participate in international collaborative clinical trials through Japan’s involvement in ACCELERATE and its predecessor organizations. This approach has aimed to address the issue above, coinciding with the ongoing centralization of medical care and advancements in genomic medicine, among other systems that support pediatric cancer care in Japan. Inspired by the ACCELERATE initiatives and in response to patient advocacy groups’ requests for new pediatric oncology drugs, Japan established its inaugural pediatric oncology support platform in 2021, the National Childhood Cancer Consortium (N3C). N3C comprises patient advocacy groups, industries, and academia. This work outlines the current status of pediatric drug development in Japan and highlights the initial successful experience of NCCH’s participation in a global clinical trial through ACCELERATE resulting in rapid preparation and patient recruitment. Participation of Asian countries including Japan in global collaborative clinical trials may benefit both pharmaceutical companies and the participating countries partnering to advance the development of pediatric oncology drugs.</p></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2772610X24000163/pdfft?md5=2f7099c73d2c72937b2ee48f61f1ec60&pid=1-s2.0-S2772610X24000163-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140161006","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":"Telomere maintenance mechanisms in neuroblastoma: New insights and translational implications","authors":"Lisa Werr ,&nbsp;Carolina Rosswog ,&nbsp;Christoph Bartenhagen ,&nbsp;Sally L. George ,&nbsp;Matthias Fischer","doi":"10.1016/j.ejcped.2024.100156","DOIUrl":"https://doi.org/10.1016/j.ejcped.2024.100156","url":null,"abstract":"<div><p>The clinical behaviour of neuroblastoma ranges from spontaneous tumour regression or maturation into benign ganglioneuroma to fatal progression despite intensive treatment [1,2]. The mechanisms underlying the distinct phenotypes have remained uncertain yet. A number of recent studies noticed that activation of telomere maintenance mechanisms (TMM) in the malignant neuroblasts is strongly associated with high-risk disease and poor outcome, whereas TMM are absent in spontaneously regressing low-risk tumours, suggesting that telomere maintenance is essential to drive neuroblasts into the fully malignant state. Stabilization of the telomeres above a critical threshold is essential for cancer cells to gain replicative immortality and has thus been considered a hallmark of cancer [3]. In most high-risk neuroblastomas, TMM is conferred by induction of telomerase, either through genomic rearrangements of the <em>TERT</em> locus, encoding for the catalytic subunit of telomerase, or amplification of the <em>MYCN</em> proto-oncogene, whereas the alternative lengthening of telomeres (ALT) pathway is activated in approximately one-quarter of high-risk tumours [4–8]. We here review recent advances on our understanding of the mechanistic role of telomere maintenance in neuroblastoma pathogenesis, and discuss potential implications on neuroblastoma risk assessment and on the development of novel therapies directed against TMM.</p></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2772610X24000151/pdfft?md5=de1f29ad7bc35889849f4f9efb14f365&pid=1-s2.0-S2772610X24000151-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140138799","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":"Prevalence of childhood cancer survivors in Europe: a scoping review","authors":"Neimar de Paula Silva ,&nbsp;Andrea Gini ,&nbsp;Anastasia Dolya ,&nbsp;Murielle Colombet ,&nbsp;Isabelle Soerjomataram ,&nbsp;Danny Youlden ,&nbsp;Charles Stiller ,&nbsp;Eva Steliarova-Foucher ,&nbsp;the CRICCS consortium,&nbsp;Joanne Aitken ,&nbsp;Freddie Bray ,&nbsp;Murielle Colombet ,&nbsp;Neimar de Paula Silva ,&nbsp;Anastasia Dolya ,&nbsp;Friederike Erdmann ,&nbsp;Jeanette Falck Winther ,&nbsp;Andrea Gini ,&nbsp;Delphine Heenen ,&nbsp;Lars Hjorth ,&nbsp;Claudia E. Kuehni ,&nbsp;Danny Youlden","doi":"10.1016/j.ejcped.2024.100155","DOIUrl":"https://doi.org/10.1016/j.ejcped.2024.100155","url":null,"abstract":"<div><p>Childhood cancer survivors (CCS) require specialized follow-up throughout their lifespan to prevent or manage late effects of cancer treatment. Knowing the size and structure of the population of CCS is crucial to plan interventions. In this scoping review we reviewed studies that reported prevalence of CCS in Europe. We searched Medline, Web of Science, and Embase using permutations of terms referring to childhood, cancer, survivors, prevalence, registries, and Europe. We followed PRISMA-ScR guidelines to select studies and The Joanna Briggs Institute Prevalence Critical Appraisal Tool to evaluate their quality. From 979 unique studies published between 1989 and 2022, 12 were included. Limited-duration prevalence (LDP) for all childhood cancers, assessed in three studies using counting method, varied between 450 and 1240 persons per million. Complete prevalence (CP) of survivors of any childhood cancer except skin carcinomas, reported in three studies using observed data complemented with modelled data for the unobserved period, varied between 730 and 1110 persons per million. CP of survivors of an embryonal tumour was estimated by completeness index method in six studies. In four of them CP ranged from 48 to 95 persons per million for all embryonal tumours, while CP for those occurring in central nervous system was 43 per million in one study and CP for rhabdomyosarcoma was 17 per million in another. Information on prevalence of CCS in Europe is fragmented and inconsistent. The large variations in LDP and CP estimates were linked to differences in data availability, the selection of populations, prevalence measure, statistical method, incidence period, index date, age at diagnosis and prevalence, cancer types, sex, and, for LDP, also the length of follow-up. Standardisation of methodology and reporting are needed to systematically monitor and compare CCS prevalence in Europe and provide data to help address survivors’ needs.</p></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-03-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2772610X2400014X/pdfft?md5=4115c91bca18701bb5c80c478a2fce0c&pid=1-s2.0-S2772610X2400014X-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140138798","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":"Twinning to reduce research and innovation inequalities in paediatric solid tumours across Europe","authors":"Jelena Rascon ,&nbsp;Renata Blackute ,&nbsp;Alma Cerkauskiene ,&nbsp;Sabine Taschner-Mandl ,&nbsp;Nuno Andrade ,&nbsp;Adriana Planinic ,&nbsp;Stefan Rutkowski ,&nbsp;Ulrich Schuller ,&nbsp;Karsten Nysom ,&nbsp;Ruta Tuckuviene ,&nbsp;Jesper Brok ,&nbsp;Kjeld Schmiegelow ,&nbsp;Marry M. van den Heuvel-Eibrink ,&nbsp;M.E. Madeleine van der Perk ,&nbsp;Riccardo Haupt ,&nbsp;Monica Muraca ,&nbsp;Davide Saraceno ,&nbsp;Birgit Geoerger ,&nbsp;Giorgia Manuzi ,&nbsp;Ruth Ladenstein","doi":"10.1016/j.ejcped.2024.100153","DOIUrl":"https://doi.org/10.1016/j.ejcped.2024.100153","url":null,"abstract":"<div><p>Inequalities in research and innovations affect childhood cancer survival across Europe. Vilnius University Hospital Santaros Klinikos (VULSK, the coordinator) and eight research-intensive institutions from seven European countries implemented the TREL project (Twinning in Research and Education to improve survival in childhood solid tumours in Lithuania) supported by the Horizon 2020 Widening programme. TREL aimed to enhance translational, clinical, and survivorship research in paediatric CNS, neuroblastoma, and renal tumours to improve future treatment outcomes in Lithuania. From January 2021 to December 2023, 49 VULSK professionals and 55 peers from partner institutions collaborated in this twinning program. Achievements after three years were: nine educational events, the initiation of basic and clinical research on fertility preservation, ten VULSK researchers joining international research groups, six signed agreements to participate in international academic clinical trials and the implementation of the European Survivorship Passport. Thirty patients received individual treatment recommendations following multidisciplinary discussions with experts from partner institutions. Twenty-five rare genetic variants were classified by the twinning bioinformatician teams with direct consequences on patient management. In conclusion, coordination of the Horizon 2020 project enhanced VULKS’s research capacities, networking channels and attractiveness for industry and academia-initiated innovative actions that will improve survival rates in the long run.</p></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2772610X24000126/pdfft?md5=c32781086be65a384542762fae4be4dc&pid=1-s2.0-S2772610X24000126-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140031155","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":"131-I-MIBG therapy in combination with PARP inhibitors for young adult patient with relapsed neuroblastoma and DNA repair pathway alterations","authors":"Sarah Cohen-Gogo ,&nbsp;Amer Shammas ,&nbsp;Adam Shlien ,&nbsp;Meredith Irwin ,&nbsp;Daniel Morgenstern","doi":"10.1016/j.ejcped.2024.100152","DOIUrl":"https://doi.org/10.1016/j.ejcped.2024.100152","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2772610X24000114/pdfft?md5=d13c8be54bd68674569537c0f8ff2605&pid=1-s2.0-S2772610X24000114-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140016033","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":"Malignant extracranial germ cell tumors in the Netherlands between 1990 and 2018: Stable incidence and improved survival","authors":"Caroline C.C. Hulsker ,&nbsp;Maya Schulpen ,&nbsp;Annelies M.C. Mavinkurve-Groothuis ,&nbsp;Otto Visser ,&nbsp;József Zsiros ,&nbsp;Marc H.W. Wijnen ,&nbsp;Ronald R. de Krijger ,&nbsp;Annette H. Bruggink ,&nbsp;Leendert H.J. Looijenga ,&nbsp;Henrike E. Karim-Kos ,&nbsp;Alida F.W. van der Steeg","doi":"10.1016/j.ejcped.2024.100148","DOIUrl":"https://doi.org/10.1016/j.ejcped.2024.100148","url":null,"abstract":"<div><h3>Background</h3><p>Population-based studies assessing long-term patterns of incidence and disease characteristics of germ cell tumors (GCTs) in children are scarce. We investigated incidence and survival trends of malignant extracranial GCTs in children using population-based nationwide data from the Netherlands.</p></div><div><h3>Methods</h3><p>All malignant extracranial GCTs diagnosed in patients aged 0–18 years between 1990 and 2018 were selected from the Netherlands Cancer Registry. Incidence rates were calculated as the average annual number of cases per 1 million person-years. Five-year overall survival (OS) was calculated.</p></div><div><h3>Results</h3><p>A total of 815 cases were identified. Gonadal GCTs (n=665, testis n=485, ovarian n=180) were more common than extragonadal GCTs (n=149). Stage distribution for testicular and extragonadal GCTs shifted between 1990 and 2004 and 2005–2018 towards more localized disease. The overall incidence remained stable over time, but a significant increase was noted for extragonadal GCTs in the 0–9 years age group. Survival of extragonadal GCTs (5-year OS 84.1%, 95% CI 77.1–89.1), in particular mediastinal GCTs (5-year OS 66.7%, 95% CI 45.7–81.1), was lower than that of gonadal GCTs (5-year OS testis 95.0%, 95% CI 92.7–96.7;ovary 97.8%, 95% CI 94.2–99.2). The 5-year OS of our entire cohort was 93.6% (95% CI 91.7–95.1). Five-year OS significantly increased from 89.5% (95% CI 86.1–92.2) in 1990–2004–97.4% (95% CI 95.3–98.5) in 2005–2018.</p></div><div><h3>Conclusions</h3><p>Although the incidence of all malignant pediatric extracranial GCTs remained stable during 1990–2018, an increase was observed for extragonadal GCTs in younger children (0–9 years). There was a shift towards more localized disease for testicular and extragonadal GCTs. Five-year OS increased over time exceeding 90% (91.4%, 95% CI 82.7–95.8) in the most recent diagnostic period. Mediastinal GCTs had the lowest OS, supporting the need for future research.</p></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2772610X24000072/pdfft?md5=503469431d7200fe24642a6af61277f3&pid=1-s2.0-S2772610X24000072-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139999308","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":"How we approach early phase clinical trial and off-label therapy consults in pediatric oncology: The New Agents and Innovative Therapy (NAIT) team experience","authors":"Gabriel Revon-Rivière,&nbsp;Pauline Tibout,&nbsp;Jennifer Cabral,&nbsp;Aiman Siddiqi,&nbsp;Ashley Doka,&nbsp;Denise Mills,&nbsp;Karen Fung,&nbsp;Sandra Judd,&nbsp;Daniel A. Morgenstern,&nbsp;Sarah Cohen-Gogo","doi":"10.1016/j.ejcped.2024.100154","DOIUrl":"https://doi.org/10.1016/j.ejcped.2024.100154","url":null,"abstract":"<div><p>In the context of hard-to-cure disease, pediatric oncologists may have to explore novel therapy options and explain their rationale, risks and constraints to patients and caregivers. The New Agents and Innovative Therapy (NAIT) program at Hospital for Sick Children in Toronto facilitates patient enrollment in clinical trials as well as access to innovative therapies outside of clinical trials. Here, we summarize our experience with helping patients, caregivers, and their primary oncology team navigate information and access to new therapeutic options through enrollment in clinical trials but also off-label and compassionate use. We expose our approach to exploring clinical trial and other therapy options. We share lessons learned from clinical practice regarding the specific role of NAIT consultant, as opposed to the primary oncologist or the disease expert. We expand on ways to communicate regarding the objectives of early phase clinical trials, their methods and the important commitment asked from participants. We describe our views on equipoise, uncertainty and hope in this very specific practice. We support a model of shared decision making and empowerment of patients and caregivers. We also detail the use, benefits and challenges of virtual care applied to NAIT consults. Overall, we hope to contribute and facilitate the NAIT practice not only for trained trialists but also less-specialized teams.</p></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":null,"pages":null},"PeriodicalIF":0.0,"publicationDate":"2024-02-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S2772610X24000138/pdfft?md5=a358b55bee4d9dda2b38d12d51888a4b&pid=1-s2.0-S2772610X24000138-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140016032","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}