Pub Date : 2025-12-01Epub Date: 2025-07-26DOI: 10.1016/j.ejcped.2025.100312
Madeleine Adams , Rachel M. Taylor
Patient reported outcomes measures (PROM) are the gold standard for evaluating patient experience and quality of life (QOL), both in research studies and clinical practice. In paediatric oncology, the wide spectrum of disease and treatment strategies means that correct choice of PROM and robust methodology when developing new PROMs is vital to ensuring that the desired outcomes (e.g. pain, functional ability or quality of life) are accurately assessed. Children differ from adults both medically and developmentally meaning that specific factors should be considered including age and developmental ability, use of proxy/observer reports and mode of administration. Best practice guidelines outline the methodology for developing the content of a new PROM as well as assessing psychometric properties. This paper provides a review of the background, current guidelines, and methodology for developing and choosing PROMs for children with cancer.
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Pub Date : 2025-12-01Epub Date: 2025-11-08DOI: 10.1016/j.ejcped.2025.100478
Caroline Atkinson , Alvin Kamili , Carole M. Tactacan , Nisitha Jayatilleke , Sebastian P.A. Rosser , Federica Saletta , Christine C. Gana , Philipp Graber , Amanda Wanninayaka , Rosa Mistica C. Ignacio , Maria Kavallaris , Andrew J. Gifford , Richard B. Lock , Murray D. Norris , Michelle Haber , Chelsea Mayoh , Christa E. Nath , Toby N. Trahair , Jamie I. Fletcher
Background
Half of all children with high-risk neuroblastoma progress or relapse. We identified ABCB1, encoding for the ATP binding cassette transporter P-glycoprotein (P-gp), as a candidate drug resistance mechanism based on differential expression in survivors and non-survivors and investigated whether P-gp expression limits the effectiveness of chemotherapy.
Methods
P-gp/ABCB1 expression and regulation were assessed in tumours, PDX models in NSG mice, and cell lines by RNA-sequencing, immunohistochemistry, western blotting and through publicly available ChIP-Seq and RNA-Seq data. Response to standard-of-care induction therapies and targeted agents was assessed in vitro and in vivo using cell line and PDX models with genetic and pharmacological P-gp inhibition.
Results
P-gp expression is common in high-risk neuroblastoma and elevated compared to other cancers. High relative expression at diagnosis is associated with poorer outcome, consistent with drug efflux. Pharmacological or genetic targeting of P-gp partially restores sensitivity of neuroblastoma cells to vincristine, doxorubicin, etoposide, and some targeted agents, but not to the ALK inhibitors crizotinib, ceritinib, alectinib and lorlatinib and sensitizes neuroblastoma xenografts to vincristine, extending survival.
Conclusions
P-gp is a clinically relevant drug resistance mechanism for high-risk neuroblastoma. Tumour P-gp levels could inform treatment options for individual patients to avoid ineffective treatments and unnecessary toxicities.
{"title":"P-glycoprotein as a chemotherapy resistance mechanism and biomarker of poor response in high-risk neuroblastoma","authors":"Caroline Atkinson , Alvin Kamili , Carole M. Tactacan , Nisitha Jayatilleke , Sebastian P.A. Rosser , Federica Saletta , Christine C. Gana , Philipp Graber , Amanda Wanninayaka , Rosa Mistica C. Ignacio , Maria Kavallaris , Andrew J. Gifford , Richard B. Lock , Murray D. Norris , Michelle Haber , Chelsea Mayoh , Christa E. Nath , Toby N. Trahair , Jamie I. Fletcher","doi":"10.1016/j.ejcped.2025.100478","DOIUrl":"10.1016/j.ejcped.2025.100478","url":null,"abstract":"<div><h3>Background</h3><div>Half of all children with high-risk neuroblastoma progress or relapse. We identified <em>ABCB1,</em> encoding for the ATP binding cassette transporter P-glycoprotein (P-gp), as a candidate drug resistance mechanism based on differential expression in survivors and non-survivors and investigated whether P-gp expression limits the effectiveness of chemotherapy.</div></div><div><h3>Methods</h3><div>P-gp/<em>ABCB1</em> expression and regulation were assessed in tumours, PDX models in NSG mice, and cell lines by RNA-sequencing, immunohistochemistry, western blotting and through publicly available ChIP-Seq and RNA-Seq data. Response to standard-of-care induction therapies and targeted agents was assessed <em>in vitro</em> and <em>in vivo</em> using cell line and PDX models with genetic and pharmacological P-gp inhibition.</div></div><div><h3>Results</h3><div>P-gp expression is common in high-risk neuroblastoma and elevated compared to other cancers. High relative expression at diagnosis is associated with poorer outcome, consistent with drug efflux. Pharmacological or genetic targeting of P-gp partially restores sensitivity of neuroblastoma cells to vincristine, doxorubicin, etoposide, and some targeted agents, but not to the ALK inhibitors crizotinib, ceritinib, alectinib and lorlatinib and sensitizes neuroblastoma xenografts to vincristine, extending survival.</div></div><div><h3>Conclusions</h3><div>P-gp is a clinically relevant drug resistance mechanism for high-risk neuroblastoma. Tumour P-gp levels could inform treatment options for individual patients to avoid ineffective treatments and unnecessary toxicities.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100478"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145527989","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}
Pub Date : 2025-12-01Epub Date: 2025-11-04DOI: 10.1016/j.ejcped.2025.100324
Caitlin W. Elgarten , Joseph H. Oved , Lisa Wray , Kimberly Venella , Peter Nicholas , Stephan Kadauke , Yongping Wang , Stephan Grupp , Timothy S. Olson
{"title":"ALTERNATIVE DONOR PERIPHERAL STEM CELL TRANSPLANTATION WITH TCRΑΒ/CD19 DEPLETION FOR PEDIATRIC PATIENTS WITH BONE MARROW FAILURE","authors":"Caitlin W. Elgarten , Joseph H. Oved , Lisa Wray , Kimberly Venella , Peter Nicholas , Stephan Kadauke , Yongping Wang , Stephan Grupp , Timothy S. Olson","doi":"10.1016/j.ejcped.2025.100324","DOIUrl":"10.1016/j.ejcped.2025.100324","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100324"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428305","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}
Pub Date : 2025-12-01Epub Date: 2025-11-04DOI: 10.1016/j.ejcped.2025.100375
Victor Pastor Loyola , Enrico Attardi , Jamie Maciaszek , Sara Lewis , Taylor Cain , Passant Shaker , Nathan Gray , Michelle Boals , Jennifer Neary , Mark Wilkinson , Michael Rusch , Lu Wang , Jeffery Klco , Marcin Wlodarski
{"title":"INTEGRATION OF A BONE MARROW FAILURE GERMLINE PANEL INTO THE COMPREHENSIVE CLINICAL GENOMICS PIPELINE AT ST. JUDE CHILDREN’S RESEARCH HOSPITAL: INSIGHTS FROM 22 MONTHS’ EXPERIENCE","authors":"Victor Pastor Loyola , Enrico Attardi , Jamie Maciaszek , Sara Lewis , Taylor Cain , Passant Shaker , Nathan Gray , Michelle Boals , Jennifer Neary , Mark Wilkinson , Michael Rusch , Lu Wang , Jeffery Klco , Marcin Wlodarski","doi":"10.1016/j.ejcped.2025.100375","DOIUrl":"10.1016/j.ejcped.2025.100375","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100375"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428561","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}
Pub Date : 2025-12-01Epub Date: 2025-11-04DOI: 10.1016/j.ejcped.2025.100385
Rafael Balceiro , Neysimelia Costa Villela , Silva Eduardo Caetano Albino da , Glaucia Regina Costa Murra , Juliana Costa Gaspar , Silva Samia Frahia Bento da , Aline Tansini , Thais Regina Toledo de Santis , Luiz Fernando Lopes , Anita Frisanco Oliveira
{"title":"AZACITIDINE FOR PEDIATRIC PATIENTS WITH ADVANCED MYELODYSPLASTIC SYNDROME: EXPERIENCE FROM BRAZILIAN COOPERATIVE GROUP OF PEDIATRIC MYELODYSPLASTIC SYNDROME (GCB-SMD-PED)","authors":"Rafael Balceiro , Neysimelia Costa Villela , Silva Eduardo Caetano Albino da , Glaucia Regina Costa Murra , Juliana Costa Gaspar , Silva Samia Frahia Bento da , Aline Tansini , Thais Regina Toledo de Santis , Luiz Fernando Lopes , Anita Frisanco Oliveira","doi":"10.1016/j.ejcped.2025.100385","DOIUrl":"10.1016/j.ejcped.2025.100385","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100385"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428571","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}
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