Pub Date : 2025-12-01DOI: 10.1016/j.ejcped.2025.100480
Ekaterina Aleshchenko , Pietro Trocchi , Christian Apfelbacher , Katja Baust , Gabriele Calaminus , Patrik Droege , Dirk Horenkamp-Sonntag , Peter Ihle , Melanie Klein , Jutta Kuepper-Nybelen , Thorsten Langer , Christian Luepkes , Ursula Marschall , Hiltrud Merzenich , Thomas Ruhnke , Claudia Spix , Enno Swart , Cecile Ronckers
Background
As survival rates for childhood cancer improve, understanding long-term outcomes and healthcare needs of childhood cancer survivors (CCS) is essential. In Germany, such data have been limited.
Objective
This study aimed to describe demographic and clinical characteristics of a long-term CCS cohort and assess healthcare utilisation patterns based on cancer registry and statutory health insurance (SHI) claims data.
Methods
The VersKiK study linked GCCR data with claims from 13 SHI providers, covering 69 % of the German statutorily insured population. The analysis of healthcare utilisation included 11,863 CCS diagnosed since 1991–2011, known to be alive in 01/2017 and maintained uninterrupted statutory health insurance coverage during 2017–2021.Healthcare utilisation was compared to a 1:3-matched comparison group (matching factors insurance duration, sex, birth year, and region).
Results
Long-term CCS had significantly higher healthcare utilisation across all metrics. They experienced 1.1–5.1 times more outpatient visits, hospital stays, medication prescriptions, and episodes of work incapacity than the control group. Utilisation varied considerably by cancer type, age at diagnosis, sex, and region. Survivors of CNS tumours, bone tumours, and soft tissue sarcomas showed the highest service use. Differences in utilisation between survivors and controls were greater among younger than older survivors.
Conclusions
This large, population-based record linkage study highlights significant differences in healthcare use between CCS and the general population, emphasising the need for evidence-based policies and tailored clinical care models. Further detailed analyses of claims data in this context can guide improvements in long-term care access, coordination, and outcomes for survivors.
{"title":"Characterising childhood cancer survivors in Germany: A retrospective observational study on demographic, clinical, and healthcare utilisation patterns","authors":"Ekaterina Aleshchenko , Pietro Trocchi , Christian Apfelbacher , Katja Baust , Gabriele Calaminus , Patrik Droege , Dirk Horenkamp-Sonntag , Peter Ihle , Melanie Klein , Jutta Kuepper-Nybelen , Thorsten Langer , Christian Luepkes , Ursula Marschall , Hiltrud Merzenich , Thomas Ruhnke , Claudia Spix , Enno Swart , Cecile Ronckers","doi":"10.1016/j.ejcped.2025.100480","DOIUrl":"10.1016/j.ejcped.2025.100480","url":null,"abstract":"<div><h3>Background</h3><div>As survival rates for childhood cancer improve, understanding long-term outcomes and healthcare needs of childhood cancer survivors (CCS) is essential. In Germany, such data have been limited.</div></div><div><h3>Objective</h3><div>This study aimed to describe demographic and clinical characteristics of a long-term CCS cohort and assess healthcare utilisation patterns based on cancer registry and statutory health insurance (SHI) claims data.</div></div><div><h3>Methods</h3><div>The VersKiK study linked GCCR data with claims from 13 SHI providers, covering 69 % of the German statutorily insured population. The analysis of healthcare utilisation included 11,863 CCS diagnosed since 1991–2011, known to be alive in 01/2017 and maintained uninterrupted statutory health insurance coverage during 2017–2021.Healthcare utilisation was compared to a 1:3-matched comparison group (matching factors insurance duration, sex, birth year, and region).</div></div><div><h3>Results</h3><div>Long-term CCS had significantly higher healthcare utilisation across all metrics. They experienced 1.1–5.1 times more outpatient visits, hospital stays, medication prescriptions, and episodes of work incapacity than the control group. Utilisation varied considerably by cancer type, age at diagnosis, sex, and region. Survivors of CNS tumours, bone tumours, and soft tissue sarcomas showed the highest service use. Differences in utilisation between survivors and controls were greater among younger than older survivors.</div></div><div><h3>Conclusions</h3><div>This large, population-based record linkage study highlights significant differences in healthcare use between CCS and the general population, emphasising the need for evidence-based policies and tailored clinical care models. Further detailed analyses of claims data in this context can guide improvements in long-term care access, coordination, and outcomes for survivors.</div></div>","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100480"},"PeriodicalIF":0.0,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145623297","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 : 2025-11-08DOI: 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":"2025-11-08","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 : 2025-11-04DOI: 10.1016/j.ejcped.2025.100331
Patrycja Fryzik, Li-Ting Chen, Evelyn S. Hanemaaijer, Ireen J. Kal, Tito Candelli, Brigit M. te Pas, Nina Epskamp, Valerie de Haas, Marijn Scheijde-Vermeulen, Wim J. de Jonge, Thanasis Margaritis, Mirjam Belderbos
{"title":"SINGLE-CELL AND SPATIAL TRANSCRIPTOMICS OF PEDIATRIC MYELODYSPLASTIC SYNDROME WITH REFRACTORY CYTOPENIA OF CHILDHOOD REVEAL A DISRUPTED BONE MARROW NICHE DRIVING IMPAIRED HEMATOPOIESIS","authors":"Patrycja Fryzik, Li-Ting Chen, Evelyn S. Hanemaaijer, Ireen J. Kal, Tito Candelli, Brigit M. te Pas, Nina Epskamp, Valerie de Haas, Marijn Scheijde-Vermeulen, Wim J. de Jonge, Thanasis Margaritis, Mirjam Belderbos","doi":"10.1016/j.ejcped.2025.100331","DOIUrl":"10.1016/j.ejcped.2025.100331","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100331"},"PeriodicalIF":0.0,"publicationDate":"2025-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428507","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 : 2025-11-04DOI: 10.1016/j.ejcped.2025.100343
Jolien De Waele , Mattias Hofmans , Alexandra Fischer , Albert Catala , Michael Dworzak , Miriam Erlacher , Henrik Hasle , Riccardo Masetti , Markus Schmugge , Marek Ussowicz , Steven Keogh , Ozlem Tufekci , Mehmet Akif Yesilipek , Ayami Yoshimi , Dirk Lebrecht , Maximilian Schönung , Peter Nöllke , Barbara De Moerloose , Charlotte M. Niemeyer
{"title":"LONG-TERM OUTCOME OF AN OBSERVATIONAL APPROACH IN PATIENTS WITH NRAS-MUTATED JUVENILE MYELOMONOCYTIC LEUKEMIA AND ABSENCE OF HIGH RISK FEATURES","authors":"Jolien De Waele , Mattias Hofmans , Alexandra Fischer , Albert Catala , Michael Dworzak , Miriam Erlacher , Henrik Hasle , Riccardo Masetti , Markus Schmugge , Marek Ussowicz , Steven Keogh , Ozlem Tufekci , Mehmet Akif Yesilipek , Ayami Yoshimi , Dirk Lebrecht , Maximilian Schönung , Peter Nöllke , Barbara De Moerloose , Charlotte M. Niemeyer","doi":"10.1016/j.ejcped.2025.100343","DOIUrl":"10.1016/j.ejcped.2025.100343","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100343"},"PeriodicalIF":0.0,"publicationDate":"2025-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428516","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 : 2025-11-04DOI: 10.1016/j.ejcped.2025.100350
Davide Leardini , Miriam Erlacher , Luca Vinci , Barbara De Moerloose , Henrik Hasle , Katja Hetnik , Marko Kavcic , Kirsten Thus , Dominik Turkiewicz , Riccardo Masetti , Michael Dworzak , Yvonne L. Behrens , Martina Pigazzi , Peter Nöllke , Senthilkumar Ramamoorthy , Dirk Lebrecht , for the MDS-EB Working Group of EWOG-MDS and EWOG-MDS National Coordinators
{"title":"GENETIC LANDSCAPE OF PRIMARY MYELODYSPLASTIC SYNDROMES WITH EXCESS OF BLASTS (MDS-EB) IN CHILDREN AND ADOLESCENTS","authors":"Davide Leardini , Miriam Erlacher , Luca Vinci , Barbara De Moerloose , Henrik Hasle , Katja Hetnik , Marko Kavcic , Kirsten Thus , Dominik Turkiewicz , Riccardo Masetti , Michael Dworzak , Yvonne L. Behrens , Martina Pigazzi , Peter Nöllke , Senthilkumar Ramamoorthy , Dirk Lebrecht , for the MDS-EB Working Group of EWOG-MDS and EWOG-MDS National Coordinators","doi":"10.1016/j.ejcped.2025.100350","DOIUrl":"10.1016/j.ejcped.2025.100350","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100350"},"PeriodicalIF":0.0,"publicationDate":"2025-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428523","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 : 2025-11-04DOI: 10.1016/j.ejcped.2025.100374
Passant Shaker, Nathan Gray, Michelle Boals, Kelsey Ray, Melvanique Hale, Sara Lewis, Alyssa Kennedy, Marcin Wlodarski
{"title":"THE ROLE OF GENETIC COUNSELORS IN BONE MARROW FAILURE: A CASE-BASED APPROACH TO SRP72 AND DHX34 VARIANTS OF UNCLEAR SIGNIFICANCE","authors":"Passant Shaker, Nathan Gray, Michelle Boals, Kelsey Ray, Melvanique Hale, Sara Lewis, Alyssa Kennedy, Marcin Wlodarski","doi":"10.1016/j.ejcped.2025.100374","DOIUrl":"10.1016/j.ejcped.2025.100374","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100374"},"PeriodicalIF":0.0,"publicationDate":"2025-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428560","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 : 2025-11-04DOI: 10.1016/j.ejcped.2025.100405
Sara Mostafa Makkeyah , Marwa Waheed Tolba , Elshahhat Ahmed , Menna Allah Zakaria Abou Elwafa , Nihal Hussien Aly
{"title":"CLINICAL SPECTRUM OF AN EGYPTIAN COHORT OF CHILDREN WITH MYELODYSPLASTIC SYNDROME","authors":"Sara Mostafa Makkeyah , Marwa Waheed Tolba , Elshahhat Ahmed , Menna Allah Zakaria Abou Elwafa , Nihal Hussien Aly","doi":"10.1016/j.ejcped.2025.100405","DOIUrl":"10.1016/j.ejcped.2025.100405","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100405"},"PeriodicalIF":0.0,"publicationDate":"2025-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428430","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 : 2025-11-04DOI: 10.1016/j.ejcped.2025.100341
Edoardo Muratore , Valérie de Haas , Dorine Bresters , Christian Flotho , Pierre Goncalves , Mattias Hofmans , Marko Kavcic , Tim Lammens , Daniel B. Lipka , Loizos Petrikkos , Charlotte M. Niemeyer , for the EWOG-MDS JMML Working Group Peter Nöllke , Senthilkumar Ramamoorthy , Dirk Lebrecht , Riccardo Masetti , Maximilian Schönung , EWOG-MDS National Coordinators for EWOG-MDS
{"title":"COMPREHENSIVE CHARACTERIZATION OF SOMATIC PTPN11-MUTATED JMML","authors":"Edoardo Muratore , Valérie de Haas , Dorine Bresters , Christian Flotho , Pierre Goncalves , Mattias Hofmans , Marko Kavcic , Tim Lammens , Daniel B. Lipka , Loizos Petrikkos , Charlotte M. Niemeyer , for the EWOG-MDS JMML Working Group Peter Nöllke , Senthilkumar Ramamoorthy , Dirk Lebrecht , Riccardo Masetti , Maximilian Schönung , EWOG-MDS National Coordinators for EWOG-MDS","doi":"10.1016/j.ejcped.2025.100341","DOIUrl":"10.1016/j.ejcped.2025.100341","url":null,"abstract":"","PeriodicalId":94314,"journal":{"name":"EJC paediatric oncology","volume":"6 ","pages":"Article 100341"},"PeriodicalIF":0.0,"publicationDate":"2025-11-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145428514","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}
扫码关注我们
求助内容：
应助结果提醒方式：
京公网安备 11010802042870号