Sophie Thomas, Emily Morley, Timothy Ritzmann, Christopher Clayton, Kathyrn Powers, Jac Airdrie, Louise Robinson, Kate Fifield, Anna Packham, Maria Chiara Oprandi, Jo-Fen Lui, Gillian Whitfield, Nicola Thorp, Jennifer Limond, Richard Grundy
It is crucial to understand the morbidity associated with treatments for young children with ependymoma given this is a high incidence age group also known to be at risk of poorer cognitive outcomes. This review aimed to identify the quality of existing evidence describing cognitive outcomes in children treated for ependymoma under 36 months of age with a particular focus on the impact of radiotherapy. Eight studies were identified. Given the quality and heterogeneity of methodology, studies were only suitable for qualitative synthesis, as the majority included small numbers of participants with multiple confounding factors. Whilst some studies reported poor cognitive outcomes, the only large study reporting planned irradiation reported outcomes below the population mean but still broadly in the average range. This was consistent with a further study of interest that did not meet inclusion criteria but reported outcomes for children treated under five years old, many of whom were likely in the target population age for this review. Overall, the length of follow-up was often limited, and further research to monitor long-term impact, including photon and proton irradiation protocols on cognitive development, is required. Importantly, there is an urgent need to agree homogeneous methodology and achieve international consensus for cognitive assessment protocols to interrogate cognitive outcomes in this vulnerable population.
{"title":"Cognitive Outcomes in Children Treated for Ependymoma Diagnosed Under 36 Months: A Systematic Review.","authors":"Sophie Thomas, Emily Morley, Timothy Ritzmann, Christopher Clayton, Kathyrn Powers, Jac Airdrie, Louise Robinson, Kate Fifield, Anna Packham, Maria Chiara Oprandi, Jo-Fen Lui, Gillian Whitfield, Nicola Thorp, Jennifer Limond, Richard Grundy","doi":"10.1002/pbc.31588","DOIUrl":"https://doi.org/10.1002/pbc.31588","url":null,"abstract":"<p><p>It is crucial to understand the morbidity associated with treatments for young children with ependymoma given this is a high incidence age group also known to be at risk of poorer cognitive outcomes. This review aimed to identify the quality of existing evidence describing cognitive outcomes in children treated for ependymoma under 36 months of age with a particular focus on the impact of radiotherapy. Eight studies were identified. Given the quality and heterogeneity of methodology, studies were only suitable for qualitative synthesis, as the majority included small numbers of participants with multiple confounding factors. Whilst some studies reported poor cognitive outcomes, the only large study reporting planned irradiation reported outcomes below the population mean but still broadly in the average range. This was consistent with a further study of interest that did not meet inclusion criteria but reported outcomes for children treated under five years old, many of whom were likely in the target population age for this review. Overall, the length of follow-up was often limited, and further research to monitor long-term impact, including photon and proton irradiation protocols on cognitive development, is required. Importantly, there is an urgent need to agree homogeneous methodology and achieve international consensus for cognitive assessment protocols to interrogate cognitive outcomes in this vulnerable population.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31588"},"PeriodicalIF":2.4,"publicationDate":"2025-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143399675","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jordana K McLoone, Claire E Wakefield, N Glasson, Lori Wiener, R Ortiz, Andre Ilbawi, Clarissa E Schilstra, Ruth Hoffman, Julie Cayrol
Introduction: Lifelong follow-up care for childhood cancer survivors (CCS) is recommended and ideally involves both medical and psychosocial care. It is important for CCS and their families to be adequately informed about what to expect after cancer treatment completion to ensure they receive appropriate care. This study aimed to describe patterns of access to survivorship care among a multi-national sample, as well as examine unmet information and support needs, for CCS and their parents.
Method: An online survey, developed by pediatric psycho-oncology experts and people with lived experience of pediatric cancer, was distributed by the World Health Organization. This study presents a subanalysis from these data.
Results: Participants included 102 parents of CCS (94 females, mean age 45 years, mean time since child's diagnosis 9 years), and 43 CCS (28 females, mean age 31 years, mean time since diagnosis 21 years) from 17 countries. Thirty-five percent of CCS (13/37) were not accessing survivorship care. Most parents (95%; 97/102) and CCS (76%; 31/41) reported a desire for discussion of emotional impacts following cancer treatment completion; however, this did not occur for 69% (70/102) of parents and 46% (19/41) of CCS. Additionally, 92% (93/102) of parents and 83% (33/41) of CCS reported an unmet need for more information about what to expect after cancer treatment. Most CCS (54%; 22/41) reported feeling "somewhat-not at all" supported by healthcare professionals in the period after cancer treatment.
Conclusion: Discussions regarding emotional well-being and ongoing needs post treatment are lacking in cancer survivorship care worldwide.
{"title":"The Lived Experience of Childhood Cancer Survivors and Their Parents: A Multi-National Study of Access to Survivorship Care and Information and Support Needs.","authors":"Jordana K McLoone, Claire E Wakefield, N Glasson, Lori Wiener, R Ortiz, Andre Ilbawi, Clarissa E Schilstra, Ruth Hoffman, Julie Cayrol","doi":"10.1002/pbc.31593","DOIUrl":"https://doi.org/10.1002/pbc.31593","url":null,"abstract":"<p><strong>Introduction: </strong>Lifelong follow-up care for childhood cancer survivors (CCS) is recommended and ideally involves both medical and psychosocial care. It is important for CCS and their families to be adequately informed about what to expect after cancer treatment completion to ensure they receive appropriate care. This study aimed to describe patterns of access to survivorship care among a multi-national sample, as well as examine unmet information and support needs, for CCS and their parents.</p><p><strong>Method: </strong>An online survey, developed by pediatric psycho-oncology experts and people with lived experience of pediatric cancer, was distributed by the World Health Organization. This study presents a subanalysis from these data.</p><p><strong>Results: </strong>Participants included 102 parents of CCS (94 females, mean age 45 years, mean time since child's diagnosis 9 years), and 43 CCS (28 females, mean age 31 years, mean time since diagnosis 21 years) from 17 countries. Thirty-five percent of CCS (13/37) were not accessing survivorship care. Most parents (95%; 97/102) and CCS (76%; 31/41) reported a desire for discussion of emotional impacts following cancer treatment completion; however, this did not occur for 69% (70/102) of parents and 46% (19/41) of CCS. Additionally, 92% (93/102) of parents and 83% (33/41) of CCS reported an unmet need for more information about what to expect after cancer treatment. Most CCS (54%; 22/41) reported feeling \"somewhat-not at all\" supported by healthcare professionals in the period after cancer treatment.</p><p><strong>Conclusion: </strong>Discussions regarding emotional well-being and ongoing needs post treatment are lacking in cancer survivorship care worldwide.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31593"},"PeriodicalIF":2.4,"publicationDate":"2025-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143399677","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Nirali N Shah, Eric S Schafer, Yueh-Yun Chi, Jemily Malvar, Kenneth M Heym, Andrew E Place, Melissa Burns, Bill H Chang, Tamra Slone, Anupam Verma, Nathan Gossai, Peter H Shaw, Michael J Burke, Michelle Hermiston, Reuven J Schore, Todd Cooper, Melinda Pauly, Teresa Rushing, Paul Jarosinski, Ellynore Florendo, Bonnie Yates, Brigitte C Widemann, Cody J Peer, William D Figg, Lewis B Silverman, Deepa Bhojwani, Alan S Wayne
Introduction: Vincristine sulfate liposome injection (VSLI), a liposomal formulation of vincristine, may be better tolerated than standard aqueous vincristine and enable dose intensification.
Procedures: Based on single-agent tolerability, activity, and FDA approval in adults with acute lymphoblastic leukemia (ALL), we tested the safety and feasibility of VSLI as replacement for standard vincristine in the UK ALL R3 mitoxantrone-based four-drug induction (Cohort A), a three-drug anthracycline-free induction (Cohort B), and maintenance chemotherapy (Cohort C) in children and young adults with relapsed/refractory B-cell ALL.
Results: Among 29 participants with a median age of 12.4 years (range: 1.8-19.6 years), 16 received Cohort A, eight received Cohort B, and five received Cohort C therapy. Dose level 1 (DL1): 1.5 mg/m2 and dose level 2 (DL2): 2 mg/m2 of VSLI, each without a dose cap, were tested. Collectively, the median VSLI dose administered was 1.9 mg (range: 0.71-4.06 mg), and 13 (44.8%) received a dose above the standard 2 mg vincristine dose cap. Dose-limiting toxicities (DLTs) at DL2 were seen in three patients, two in Cohort A and one in Cohort B, prompting further evaluation at DL1 for both cohorts. No DLTs were experienced at DL1. Only DL2 was tested in Cohort C-without DLT. Complete remissions were seen in 14 of 16 (87.5%) participants in Cohort A; three of eight (37.5%) in Cohort B; and one (20%) in Cohort C. VSLI with combination chemotherapy at DL1 was generally well tolerated.
Conclusion: Based on the promising response signal in this heavily pretreated population, further study of VSLI is warranted. (ClinicalTrials.gov NCT02879643).
{"title":"Vincristine Sulfate Liposome Injection with Combination Chemotherapy for Children, Adolescents, and Young Adults with Relapsed Acute Lymphoblastic Leukemia: A Therapeutic Advances in Childhood Leukemia and Lymphoma Consortium Trial.","authors":"Nirali N Shah, Eric S Schafer, Yueh-Yun Chi, Jemily Malvar, Kenneth M Heym, Andrew E Place, Melissa Burns, Bill H Chang, Tamra Slone, Anupam Verma, Nathan Gossai, Peter H Shaw, Michael J Burke, Michelle Hermiston, Reuven J Schore, Todd Cooper, Melinda Pauly, Teresa Rushing, Paul Jarosinski, Ellynore Florendo, Bonnie Yates, Brigitte C Widemann, Cody J Peer, William D Figg, Lewis B Silverman, Deepa Bhojwani, Alan S Wayne","doi":"10.1002/pbc.31584","DOIUrl":"https://doi.org/10.1002/pbc.31584","url":null,"abstract":"<p><strong>Introduction: </strong>Vincristine sulfate liposome injection (VSLI), a liposomal formulation of vincristine, may be better tolerated than standard aqueous vincristine and enable dose intensification.</p><p><strong>Procedures: </strong>Based on single-agent tolerability, activity, and FDA approval in adults with acute lymphoblastic leukemia (ALL), we tested the safety and feasibility of VSLI as replacement for standard vincristine in the UK ALL R3 mitoxantrone-based four-drug induction (Cohort A), a three-drug anthracycline-free induction (Cohort B), and maintenance chemotherapy (Cohort C) in children and young adults with relapsed/refractory B-cell ALL.</p><p><strong>Results: </strong>Among 29 participants with a median age of 12.4 years (range: 1.8-19.6 years), 16 received Cohort A, eight received Cohort B, and five received Cohort C therapy. Dose level 1 (DL1): 1.5 mg/m<sup>2</sup> and dose level 2 (DL2): 2 mg/m<sup>2</sup> of VSLI, each without a dose cap, were tested. Collectively, the median VSLI dose administered was 1.9 mg (range: 0.71-4.06 mg), and 13 (44.8%) received a dose above the standard 2 mg vincristine dose cap. Dose-limiting toxicities (DLTs) at DL2 were seen in three patients, two in Cohort A and one in Cohort B, prompting further evaluation at DL1 for both cohorts. No DLTs were experienced at DL1. Only DL2 was tested in Cohort C-without DLT. Complete remissions were seen in 14 of 16 (87.5%) participants in Cohort A; three of eight (37.5%) in Cohort B; and one (20%) in Cohort C. VSLI with combination chemotherapy at DL1 was generally well tolerated.</p><p><strong>Conclusion: </strong>Based on the promising response signal in this heavily pretreated population, further study of VSLI is warranted. (ClinicalTrials.gov NCT02879643).</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31584"},"PeriodicalIF":2.4,"publicationDate":"2025-02-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143399678","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Angidi Mauree, Khumo Myezo, Neil Ranasinghe, Julia Challinor, Rossella Bandini, Kathryn Burns, Katherine Eyal, Julia Downing, Kathy Pritchard-Jones, Eric Bouffet, Jennifer Geel
Background: Paediatric palliative care (PPC) is considered an essential component of the management of children and adolescents with cancer. The International Society of Paediatric Oncology Global Mapping Programme (SIOP GMP) surveyed hospital-based paediatric oncology facilities across Africa from 2018 to 2020 to document PPC and provision of PPC services. We aimed to assess possible correlations between existing PPC services across Africa with economic indicators.
Procedure: An electronic and paper survey was widely distributed to elicit the presence of components of PPC: PPC teams, bereavement counselling services, patient support groups, and spiritual and religious support. Results were correlated with the countries' Gini coefficient, World Bank income status indicators and Human Development Index.
Results: Hospital-based paediatric oncology facilities in 16/54 African countries reported having all four PPC services, while those in 12 countries reported having none of the four PPC services. No clear correlations were found between provision of such services and selected economic factors.
Conclusions: This study assesses components of PPC through four binary questions and demonstrates that hospital-based paediatric oncology facilities with limited resources caring for children and adolescents can provide PPC. Adoption of the World Health Organization's conceptual framework for palliative care and knowledge transfer between African facilities on the integration of PPC into paediatric oncology care, would benefit the increasing numbers of children and adolescents with cancer across the continent.
{"title":"African Hospital-Based Paediatric Palliative Oncology Care Independent of Economic Indicators: An International Society of Paediatric Oncology (SIOP) Global Mapping Programme Survey.","authors":"Angidi Mauree, Khumo Myezo, Neil Ranasinghe, Julia Challinor, Rossella Bandini, Kathryn Burns, Katherine Eyal, Julia Downing, Kathy Pritchard-Jones, Eric Bouffet, Jennifer Geel","doi":"10.1002/pbc.31598","DOIUrl":"https://doi.org/10.1002/pbc.31598","url":null,"abstract":"<p><strong>Background: </strong>Paediatric palliative care (PPC) is considered an essential component of the management of children and adolescents with cancer. The International Society of Paediatric Oncology Global Mapping Programme (SIOP GMP) surveyed hospital-based paediatric oncology facilities across Africa from 2018 to 2020 to document PPC and provision of PPC services. We aimed to assess possible correlations between existing PPC services across Africa with economic indicators.</p><p><strong>Procedure: </strong>An electronic and paper survey was widely distributed to elicit the presence of components of PPC: PPC teams, bereavement counselling services, patient support groups, and spiritual and religious support. Results were correlated with the countries' Gini coefficient, World Bank income status indicators and Human Development Index.</p><p><strong>Results: </strong>Hospital-based paediatric oncology facilities in 16/54 African countries reported having all four PPC services, while those in 12 countries reported having none of the four PPC services. No clear correlations were found between provision of such services and selected economic factors.</p><p><strong>Conclusions: </strong>This study assesses components of PPC through four binary questions and demonstrates that hospital-based paediatric oncology facilities with limited resources caring for children and adolescents can provide PPC. Adoption of the World Health Organization's conceptual framework for palliative care and knowledge transfer between African facilities on the integration of PPC into paediatric oncology care, would benefit the increasing numbers of children and adolescents with cancer across the continent.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31598"},"PeriodicalIF":2.4,"publicationDate":"2025-02-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143399711","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Daniel A Morgenstern, Sarah Cohen-Gogo, Denise Connolly, Meredith Irwin, Paul Nathan, Jordan Staunton, Jonathan Wasserman
{"title":"Comment on: Incidence of Adrenal Insufficiency in Patients with High-Risk Neuroblastoma: A Single-Institution Analysis.","authors":"Daniel A Morgenstern, Sarah Cohen-Gogo, Denise Connolly, Meredith Irwin, Paul Nathan, Jordan Staunton, Jonathan Wasserman","doi":"10.1002/pbc.31600","DOIUrl":"https://doi.org/10.1002/pbc.31600","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31600"},"PeriodicalIF":2.4,"publicationDate":"2025-02-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143365619","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}