Emelia Eagling-Every, Shu Ki Tsoi, Hannah Walker, Gabrielle M. Haeusler
Chronic disseminated candidiasis (CDC) is a rare complication of immunosuppression. This review describes the presentation, management, and outcomes of CDC in pediatric patients with cancer or following hematopoietic cell transplant (HCT). PubMed, Embase, and Medline were searched identifying 32 studies, describing 95 cases of CDC. CDC occurred almost exclusively in patients with leukemia (91%), with only 5% occurring in lymphoma, 1% post HCT, and 3% in solid tumor. The most frequent presenting symptoms were fever (97%) and abdominal pain (45%), with lesions in liver in 63% and spleen in 54% (less common in kidney, lungs and skin/soft tissue). Of the 67 (71%) episodes with microbiological confirmation, Candida tropicalis (28%) was the most common causative species. Antifungal treatment durations varied from 14 days to 28 months. Additionally, 31 (33%) patients received an adjuvant therapy, the most common being corticosteroids. Mortality, directly attributable to CDC, occurred in nine (9%). There remains insufficient data to guide a unified approach to management.
{"title":"Systematic Review of the Presentation, Treatment, and Outcome of Chronic Disseminated Candidiasis in Children With Cancer or Following Hematopoietic Cell Transplant","authors":"Emelia Eagling-Every, Shu Ki Tsoi, Hannah Walker, Gabrielle M. Haeusler","doi":"10.1002/pbc.31560","DOIUrl":"10.1002/pbc.31560","url":null,"abstract":"<div>\u0000 \u0000 <p>Chronic disseminated candidiasis (CDC) is a rare complication of immunosuppression. This review describes the presentation, management, and outcomes of CDC in pediatric patients with cancer or following hematopoietic cell transplant (HCT). PubMed, Embase, and Medline were searched identifying 32 studies, describing 95 cases of CDC. CDC occurred almost exclusively in patients with leukemia (91%), with only 5% occurring in lymphoma, 1% post HCT, and 3% in solid tumor. The most frequent presenting symptoms were fever (97%) and abdominal pain (45%), with lesions in liver in 63% and spleen in 54% (less common in kidney, lungs and skin/soft tissue). Of the 67 (71%) episodes with microbiological confirmation, <i>Candida tropicalis</i> (28%) was the most common causative species. Antifungal treatment durations varied from 14 days to 28 months. Additionally, 31 (33%) patients received an adjuvant therapy, the most common being corticosteroids. Mortality, directly attributable to CDC, occurred in nine (9%). There remains insufficient data to guide a unified approach to management.</p>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143046523","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}
Scott Moerdler, Yufei Yan, Stavroula Chrysanthopoulou, Maura Barry, Elizabeth Henry, Tiffany Lucas, Ariela Marshall, Don S. Dizon
Purpose
To explore the potential relationship between social media (SoMe) and burnout or overall wellbeing within the field of oncology.
Design
A cross-sectional study of adult and pediatric oncology professionals conducted using an anonymous electronic survey. The survey was disseminated through the Children's Oncology Group (COG) and the SWOG Cancer Research Network (SWOG) member listservs.
Results
The majority of pediatric and adult oncology professionals are not engaging on, with only 873/3000 (29%) using SoMe professionally. Use of SoMe was associated with statistically significant higher incidence of self-reported burnout and poorer self-reported work‒life integration (WLI). However, both groups reported the same degree of career satisfaction and choosing the same career/job again. SoMe users and non-users reported similar overall psychological distress, although the use of SoMe was associated with less severe psychological distress.
Conclusion
While SoMe users reported higher rates of burnout and poorer WLI compared to non-users, it was not accompanied by higher levels of psychological distress. Furthermore, there were no differences in career satisfaction. These misalignments require further study.
{"title":"Likes and Hashtags: Exploring the Potential Relationship Between Social Media Use and the Emotional Wellbeing of Oncology Professionals","authors":"Scott Moerdler, Yufei Yan, Stavroula Chrysanthopoulou, Maura Barry, Elizabeth Henry, Tiffany Lucas, Ariela Marshall, Don S. Dizon","doi":"10.1002/pbc.31568","DOIUrl":"10.1002/pbc.31568","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Purpose</h3>\u0000 \u0000 <p>To explore the potential relationship between social media (SoMe) and burnout or overall wellbeing within the field of oncology.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Design</h3>\u0000 \u0000 <p>A cross-sectional study of adult and pediatric oncology professionals conducted using an anonymous electronic survey. The survey was disseminated through the Children's Oncology Group (COG) and the SWOG Cancer Research Network (SWOG) member listservs.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The majority of pediatric and adult oncology professionals are not engaging on, with only 873/3000 (29%) using SoMe professionally. Use of SoMe was associated with statistically significant higher incidence of self-reported burnout and poorer self-reported work‒life integration (WLI). However, both groups reported the same degree of career satisfaction and choosing the same career/job again. SoMe users and non-users reported similar overall psychological distress, although the use of SoMe was associated with less severe psychological distress.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>While SoMe users reported higher rates of burnout and poorer WLI compared to non-users, it was not accompanied by higher levels of psychological distress. Furthermore, there were no differences in career satisfaction. These misalignments require further study.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31568","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143047625","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Nicolas André, Caroline Donzé, Gabriel Revon-Rivière
{"title":"Striped Hair Colour Change on Tovorafenib in an 18-Year-Old Boy With a Brainstem Low-Grade Glioma","authors":"Nicolas André, Caroline Donzé, Gabriel Revon-Rivière","doi":"10.1002/pbc.31567","DOIUrl":"10.1002/pbc.31567","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143040703","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}
{"title":"Ghosal Type Hematodiaphyseal Dysplasia—A Rare and Unusual Cause of Cytopenias","authors":"Sirisha Rani Siddaiahgari, Veena Akkineni","doi":"10.1002/pbc.31533","DOIUrl":"10.1002/pbc.31533","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143047624","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}
Sheila Terwisscha van Scheltinga, Reineke A. Schoot, Jonathan C. Routh, Guido Seitz, Simon C. Kao, Bart de Keizer, Barry Shulkin, Roelof van Ewijk, Beth McCarville, Dana Casey, Wendy Allen-Rhoades, Federico Mercolini, Hans Merks, Daniel Orbach, Tejas Kapadia, David O. Walterhouse, Raquel Davila Fajardo, Laura Hiemcke-Jiwa, Christiane Franzius, Federica De Corti, Vivian Tang, Jonathan Metts, Saphna Oberoi, Christian Vokuhl, Roshni Dasgupta, Suzi Birz, David Rodeberg
Accurate staging of nodal involvement in pediatric sarcoma patients is important to determine correct systemic and local therapy, with the goal to reduce subsequent recurrences. However, differences in lymph node staging strategies, definitions, and treatment protocols between the Children's Oncology Group (COG), European paediatric Soft tissue sarcoma Study Group (EpSSG), and the Cooperative Weichteilsarkom Studiengruppe (CWS) complicate comparisons. In this article, we aim to establish internationally recognized recommendations for lymph node assessment and treatment of children and adolescents diagnosed with rhabdomyosarcoma (RMS) and non-rhabdomyosarcoma soft tissue sarcoma (NRSTS) according to the Consensus Conference Standard Operating Procedure methodology.
{"title":"Lymph Node Staging and Treatment in Pediatric Patients With Soft Tissue Sarcomas: A Consensus Opinion From the Children's Oncology Group, European paediatric Soft Tissue Sarcoma Study Group, and the Cooperative Weichteilsarkom Studiengruppe","authors":"Sheila Terwisscha van Scheltinga, Reineke A. Schoot, Jonathan C. Routh, Guido Seitz, Simon C. Kao, Bart de Keizer, Barry Shulkin, Roelof van Ewijk, Beth McCarville, Dana Casey, Wendy Allen-Rhoades, Federico Mercolini, Hans Merks, Daniel Orbach, Tejas Kapadia, David O. Walterhouse, Raquel Davila Fajardo, Laura Hiemcke-Jiwa, Christiane Franzius, Federica De Corti, Vivian Tang, Jonathan Metts, Saphna Oberoi, Christian Vokuhl, Roshni Dasgupta, Suzi Birz, David Rodeberg","doi":"10.1002/pbc.31538","DOIUrl":"10.1002/pbc.31538","url":null,"abstract":"<div>\u0000 \u0000 <p>Accurate staging of nodal involvement in pediatric sarcoma patients is important to determine correct systemic and local therapy, with the goal to reduce subsequent recurrences. However, differences in lymph node staging strategies, definitions, and treatment protocols between the Children's Oncology Group (COG), European <i>paediatric</i> Soft tissue sarcoma Study Group (E<i>p</i>SSG), and the Cooperative Weichteilsarkom Studiengruppe (CWS) complicate comparisons. In this article, we aim to establish internationally recognized recommendations for lymph node assessment and treatment of children and adolescents diagnosed with rhabdomyosarcoma (RMS) and non-rhabdomyosarcoma soft tissue sarcoma (NRSTS) according to the Consensus Conference Standard Operating Procedure methodology.</p>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143024309","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}
Angela Steineck, Karen Chao, Anurekha G. Hall, Elad Jacoby, Allison Barz Leahy, John A. Ligon, Katia Luciani, Liora M. Schultz, Corinne N. Summers, Lisa Ward, Lena E. Winestone, Nirali N. Shah
Chimeric antigen receptor (CAR) T-cell therapy is a potentially life-saving treatment for children with relapsed/refractory B-cell hematologic malignancies, and remains an important investigational therapy for other childhood cancers. Yet, access to this class of therapies remains suboptimal through both commercial use and clinical trials, especially in children, adolescents, and young adults. Using a series of case-based discussions, we outline guidance on real-world medical decision-making, and offer potential solutions to enhancing access to CAR T-cell therapy as a treatment modality.
{"title":"Access to CARe: A Narrative of Real-World Medical Decision-Making to Access Chimeric Antigen Receptor (CAR) T-Cell Therapy in Children, Adolescents, and Young Adults","authors":"Angela Steineck, Karen Chao, Anurekha G. Hall, Elad Jacoby, Allison Barz Leahy, John A. Ligon, Katia Luciani, Liora M. Schultz, Corinne N. Summers, Lisa Ward, Lena E. Winestone, Nirali N. Shah","doi":"10.1002/pbc.31516","DOIUrl":"10.1002/pbc.31516","url":null,"abstract":"<div>\u0000 \u0000 <p>Chimeric antigen receptor (CAR) T-cell therapy is a potentially life-saving treatment for children with relapsed/refractory B-cell hematologic malignancies, and remains an important investigational therapy for other childhood cancers. Yet, access to this class of therapies remains suboptimal through both commercial use and clinical trials, especially in children, adolescents, and young adults. Using a series of case-based discussions, we outline guidance on real-world medical decision-making, and offer potential solutions to enhancing access to CAR T-cell therapy as a treatment modality.</p>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143024307","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}
Lauro José Gregianin, Adriana Rose, Milena Villarroel, Maria Tereza Almeida, Laurice Siqueira, Carmen Salgado, Gildene Alves da Costa, Luis Castillo, Julie Francine Cerutti Santos Pestilho, Algemir Lunardi Brunetto, GALOP Latin American Pediatric Oncology Group Ewing Sarcoma Investigators
Background
GALOP investigators developed a prospective cooperative protocol for localized Ewing sarcoma (ES) incorporating interval-compressed chemotherapy (VDC/IE, vincristine, doxorubicin, cyclophosphamide/ifosfamide and etoposide). After completing conventional treatment, patients were randomized to 1 year of metronomic chemotherapy (vinblastine and cyclophosphamide).
Methods
Phase III randomized prospective trial. Induction consisted of six alternating cycles of VDC/IE every 14 days, followed by local control, and eight cycles of consolidation every 21 days. After consolidation, patients were randomized 1:1 to metronomic chemotherapy or stop treatment, balanced by age (>/< 14 years-old), sex (M/F), site (pelvic/non-pelvic), and size (>/< 8 cm). The results of randomization will be published elsewhere with longer follow-up.
Results
Between 2011 and 2019, 315 patients (59.7% male, median age 11.0 years) were recruited across 34 centers in Argentina, Brazil, Chile, and Uruguay. The most frequent localizations were axial (45.1%), extremity (38.1%), and pelvic (16.8%). The median time interval between cycles was 19 and 22 days at induction and consolidation, respectively. There were no unexpected toxicity or toxic deaths related to interval compression. The overall response rate post-induction was 81.6%. Local treatment with surgery (50.8%), radiotherapy (19.7%), or a combination (26%) was performed in 304 (96.5%) patients. With a median follow-up of 50 months (range: 1.67–121.7), the 5-year overall and event-free survivals were 68.6% (SE: 0.030) and 63.7% (SE: 0.029), respectively.
Conclusion
Implementation of a multi-institutional protocol with the strategy of interval-compressed induction for ES in South America was feasible with favorable results. This success is attributed to rigorous protocol adherence, extensive educational efforts, and a strong emphasis on data quality maintenance, demonstrating a reproducible model for countries with similar resource limitations.
{"title":"Results of the Latin American Pediatric Oncology Group (GALOP-2011) Trial for Patients With Localized Ewing Sarcoma: A Multicentric Study of Interval-Compressed Multiagent Chemotherapy","authors":"Lauro José Gregianin, Adriana Rose, Milena Villarroel, Maria Tereza Almeida, Laurice Siqueira, Carmen Salgado, Gildene Alves da Costa, Luis Castillo, Julie Francine Cerutti Santos Pestilho, Algemir Lunardi Brunetto, GALOP Latin American Pediatric Oncology Group Ewing Sarcoma Investigators","doi":"10.1002/pbc.31554","DOIUrl":"10.1002/pbc.31554","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>GALOP investigators developed a prospective cooperative protocol for localized Ewing sarcoma (ES) incorporating interval-compressed chemotherapy (VDC/IE, vincristine, doxorubicin, cyclophosphamide/ifosfamide and etoposide). After completing conventional treatment, patients were randomized to 1 year of metronomic chemotherapy (vinblastine and cyclophosphamide).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Phase III randomized prospective trial. Induction consisted of six alternating cycles of VDC/IE every 14 days, followed by local control, and eight cycles of consolidation every 21 days. After consolidation, patients were randomized 1:1 to metronomic chemotherapy or stop treatment, balanced by age (>/< 14 years-old), sex (M/F), site (pelvic/non-pelvic), and size (>/< 8 cm). The results of randomization will be published elsewhere with longer follow-up.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Between 2011 and 2019, 315 patients (59.7% male, median age 11.0 years) were recruited across 34 centers in Argentina, Brazil, Chile, and Uruguay. The most frequent localizations were axial (45.1%), extremity (38.1%), and pelvic (16.8%). The median time interval between cycles was 19 and 22 days at induction and consolidation, respectively. There were no unexpected toxicity or toxic deaths related to interval compression. The overall response rate post-induction was 81.6%. Local treatment with surgery (50.8%), radiotherapy (19.7%), or a combination (26%) was performed in 304 (96.5%) patients. With a median follow-up of 50 months (range: 1.67–121.7), the 5-year overall and event-free survivals were 68.6% (SE: 0.030) and 63.7% (SE: 0.029), respectively.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Implementation of a multi-institutional protocol with the strategy of interval-compressed induction for ES in South America was feasible with favorable results. This success is attributed to rigorous protocol adherence, extensive educational efforts, and a strong emphasis on data quality maintenance, demonstrating a reproducible model for countries with similar resource limitations.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009325","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}
{"title":"Expanding the Concept of Iron Deficiency Using a Mitochondrial Metabolic Approach: Explaining and Preventing Sudden Death","authors":"Christineil Thompson","doi":"10.1002/pbc.31561","DOIUrl":"10.1002/pbc.31561","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009597","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}
Shachi Srivatsa, Greg Metzger, Kyle Horvath, Kelli Patterson, Megan Scruggs, Tran Bourgeois, Joseph Stanek, Peter C. Minneci, Jennifer H. Aldrink, Keri A. Streby
Background
Neuroblastoma is the most common extracranial solid tumor in children, with about half of cases classified as high risk. Treatment varies by risk level, with high-risk patients undergoing aggressive multimodal therapy. While long-term survival has improved, survivors face significant risks of late treatment effects, including adrenal insufficiency. This study investigates the incidence of adrenal insufficiency among neuroblastoma patients of varying disease risk.
Procedure
This retrospective cohort study at a single tertiary children's hospital reviewed records from 1998 to 2021, identifying 370 neuroblastoma patients, with 137 having complete risk stratification. The primary outcome was the incidence of adrenal insufficiency, diagnosed via clinical evaluation, and compared by risk group.
Results
Among 137 patients with neuroblastoma, nine (12.0%) high-risk and three (4.9%) non-high-risk patients were diagnosed with adrenal insufficiency. The cumulative incidence of adrenal insufficiency at 3 years after neuroblastoma diagnosis was 9.9% in high-risk and 3.5% in non-high-risk patients. Among high-risk neuroblastoma patients with adrenal insufficiency, median time to adrenal insufficiency diagnosis was 10.2 months from neuroblastoma diagnosis, with cases more commonly occurring in patients with adrenal primary tumors (p = 0.0234). There were no other significant differences in demographic or clinical characteristics between high-risk patients with and without adrenal insufficiency.
Conclusions
Adrenal insufficiency occurs more commonly in children with high-risk neuroblastoma than in non-high-risk cases, particularly those with adrenal primary tumors. These findings underscore the need for vigilant monitoring and screening for adrenal insufficiency during and after treatment. Future research should include larger, multi-institutional cohorts to better understand risk factors and optimize screening protocols.
{"title":"Incidence of Adrenal Insufficiency in Patients With High-Risk Neuroblastoma: A Single-Institution Analysis","authors":"Shachi Srivatsa, Greg Metzger, Kyle Horvath, Kelli Patterson, Megan Scruggs, Tran Bourgeois, Joseph Stanek, Peter C. Minneci, Jennifer H. Aldrink, Keri A. Streby","doi":"10.1002/pbc.31543","DOIUrl":"10.1002/pbc.31543","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Neuroblastoma is the most common extracranial solid tumor in children, with about half of cases classified as high risk. Treatment varies by risk level, with high-risk patients undergoing aggressive multimodal therapy. While long-term survival has improved, survivors face significant risks of late treatment effects, including adrenal insufficiency. This study investigates the incidence of adrenal insufficiency among neuroblastoma patients of varying disease risk.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>This retrospective cohort study at a single tertiary children's hospital reviewed records from 1998 to 2021, identifying 370 neuroblastoma patients, with 137 having complete risk stratification. The primary outcome was the incidence of adrenal insufficiency, diagnosed via clinical evaluation, and compared by risk group.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Among 137 patients with neuroblastoma, nine (12.0%) high-risk and three (4.9%) non-high-risk patients were diagnosed with adrenal insufficiency. The cumulative incidence of adrenal insufficiency at 3 years after neuroblastoma diagnosis was 9.9% in high-risk and 3.5% in non-high-risk patients. Among high-risk neuroblastoma patients with adrenal insufficiency, median time to adrenal insufficiency diagnosis was 10.2 months from neuroblastoma diagnosis, with cases more commonly occurring in patients with adrenal primary tumors (<i>p</i> = 0.0234). There were no other significant differences in demographic or clinical characteristics between high-risk patients with and without adrenal insufficiency.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Adrenal insufficiency occurs more commonly in children with high-risk neuroblastoma than in non-high-risk cases, particularly those with adrenal primary tumors. These findings underscore the need for vigilant monitoring and screening for adrenal insufficiency during and after treatment. Future research should include larger, multi-institutional cohorts to better understand risk factors and optimize screening protocols.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31543","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009646","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Naureen Mushtaq, Farrah Bashir, Soha Zahid, Quratulain Riaz, Gohar Javed, Maria Tariq, Bilal Mazhar Qureshi, Kiran Hilal, Vijay Ramaswamy, Cynthia Hawkins, Khurram Minhas, Eric Bouffet
Introduction
Medulloblastoma (MB) is the most common malignant childhood brain tumor. Molecular subgrouping of MB has become a major determinant of management in high-income countries. Subgrouping is still very limited in low- and middle-income countries (LMICs), and its relevance to management with the incorporation of risk stratification (low risk, standard risk, high risk, and very high risk) has yet to be evaluated in this setting. We describe molecular findings from a tertiary care center in Pakistan and their implications for outcome.
Methods
Children aged between 3 and 18 years diagnosed with MB from April 2014 to December 2020 at Aga Khan University Hospital (AKUH) were included. Subgrouping was performed by NanoString through a collaboration with The Hospital for Sick Children, Toronto.
Results
Thirty-seven patients (30 males) were included in this study; median age was 9 years. Twenty patients (54.1%) were high-risk, including 12 with metastatic disease. In 30 children, there was a clear molecular subgroup: 4 wingless (WNT) (10.8%), 6 sonic hedgehog (SHH) (16.2%), 3 Group 3 (8.1%), and 17 Group 4 (45.9%) MBs. Molecular subgrouping was inconclusive for three patients (8.1%) and not done in four patients (10.8%). All patients underwent surgery; 26 patients received radiation therapy at AKUH, and 9 were referred outside for radiotherapy; 24 patients received chemotherapy at AKUH (10 outside AKUH). Overall survival (OS) at 5 years was 100%, 66.7%, 66.7%, and 88.2% for WNT, SHH, Group 3, and Group 4 patients, respectively (p = 0.668). Low- and standard-risk patients had a 5-year OS of 100%, whereas very high-risk patients exhibited a significantly lower OS of 0% (p < 0.001).
Conclusion
WNT and Group 4 patients had excellent results despite one WNT patient having metastatic disease and eight Group 4 patients being high risk. Our study depicts that molecular subgrouping aids in accurately predicting survival, suggesting the potential benefit of tailored testing and treatment in the LMIC setting.
{"title":"Medulloblastoma Molecular Subgrouping and Outcomes Data of a Single Center From a Low- and Middle-Income Country","authors":"Naureen Mushtaq, Farrah Bashir, Soha Zahid, Quratulain Riaz, Gohar Javed, Maria Tariq, Bilal Mazhar Qureshi, Kiran Hilal, Vijay Ramaswamy, Cynthia Hawkins, Khurram Minhas, Eric Bouffet","doi":"10.1002/pbc.31555","DOIUrl":"10.1002/pbc.31555","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Medulloblastoma (MB) is the most common malignant childhood brain tumor. Molecular subgrouping of MB has become a major determinant of management in high-income countries. Subgrouping is still very limited in low- and middle-income countries (LMICs), and its relevance to management with the incorporation of risk stratification (low risk, standard risk, high risk, and very high risk) has yet to be evaluated in this setting. We describe molecular findings from a tertiary care center in Pakistan and their implications for outcome.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Children aged between 3 and 18 years diagnosed with MB from April 2014 to December 2020 at Aga Khan University Hospital (AKUH) were included. Subgrouping was performed by NanoString through a collaboration with The Hospital for Sick Children, Toronto.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Thirty-seven patients (30 males) were included in this study; median age was 9 years. Twenty patients (54.1%) were high-risk, including 12 with metastatic disease. In 30 children, there was a clear molecular subgroup: 4 wingless (WNT) (10.8%), 6 sonic hedgehog (SHH) (16.2%), 3 Group 3 (8.1%), and 17 Group 4 (45.9%) MBs. Molecular subgrouping was inconclusive for three patients (8.1%) and not done in four patients (10.8%). All patients underwent surgery; 26 patients received radiation therapy at AKUH, and 9 were referred outside for radiotherapy; 24 patients received chemotherapy at AKUH (10 outside AKUH). Overall survival (OS) at 5 years was 100%, 66.7%, 66.7%, and 88.2% for WNT, SHH, Group 3, and Group 4 patients, respectively (<i>p</i> = 0.668). Low- and standard-risk patients had a 5-year OS of 100%, whereas very high-risk patients exhibited a significantly lower OS of 0% (<i>p</i> < 0.001).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>WNT and Group 4 patients had excellent results despite one WNT patient having metastatic disease and eight Group 4 patients being high risk. Our study depicts that molecular subgrouping aids in accurately predicting survival, suggesting the potential benefit of tailored testing and treatment in the LMIC setting.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009658","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}
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