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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
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-23 DOI: 10.1002/pbc.31538
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
{"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,&nbsp;Reineke A. Schoot,&nbsp;Jonathan C. Routh,&nbsp;Guido Seitz,&nbsp;Simon C. Kao,&nbsp;Bart de Keizer,&nbsp;Barry Shulkin,&nbsp;Roelof van Ewijk,&nbsp;Beth McCarville,&nbsp;Dana Casey,&nbsp;Wendy Allen-Rhoades,&nbsp;Federico Mercolini,&nbsp;Hans Merks,&nbsp;Daniel Orbach,&nbsp;Tejas Kapadia,&nbsp;David O. Walterhouse,&nbsp;Raquel Davila Fajardo,&nbsp;Laura Hiemcke-Jiwa,&nbsp;Christiane Franzius,&nbsp;Federica De Corti,&nbsp;Vivian Tang,&nbsp;Jonathan Metts,&nbsp;Saphna Oberoi,&nbsp;Christian Vokuhl,&nbsp;Roshni Dasgupta,&nbsp;Suzi Birz,&nbsp;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}
引用次数: 0
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
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-23 DOI: 10.1002/pbc.31516
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
{"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,&nbsp;Karen Chao,&nbsp;Anurekha G. Hall,&nbsp;Elad Jacoby,&nbsp;Allison Barz Leahy,&nbsp;John A. Ligon,&nbsp;Katia Luciani,&nbsp;Liora M. Schultz,&nbsp;Corinne N. Summers,&nbsp;Lisa Ward,&nbsp;Lena E. Winestone,&nbsp;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}
引用次数: 0
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 拉丁美洲儿童肿瘤组(GALOP-2011)局部尤文氏肉瘤患者试验的结果:一项间隔压缩多药化疗的多中心研究。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-21 DOI: 10.1002/pbc.31554
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
{"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,&nbsp;Adriana Rose,&nbsp;Milena Villarroel,&nbsp;Maria Tereza Almeida,&nbsp;Laurice Siqueira,&nbsp;Carmen Salgado,&nbsp;Gildene Alves da Costa,&nbsp;Luis Castillo,&nbsp;Julie Francine Cerutti Santos Pestilho,&nbsp;Algemir Lunardi Brunetto,&nbsp;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 (&gt;/&lt; 14 years-old), sex (M/F), site (pelvic/non-pelvic), and size (&gt;/&lt; 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}
引用次数: 0
Expanding the Concept of Iron Deficiency Using a Mitochondrial Metabolic Approach: Explaining and Preventing Sudden Death 利用线粒体代谢方法扩展铁缺乏的概念:解释和预防猝死。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-21 DOI: 10.1002/pbc.31561
Christineil Thompson
{"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}
引用次数: 0
Incidence of Adrenal Insufficiency in Patients With High-Risk Neuroblastoma: A Single-Institution Analysis 高危神经母细胞瘤患者肾上腺功能不全的发生率:单机构分析。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-21 DOI: 10.1002/pbc.31543
Shachi Srivatsa, Greg Metzger, Kyle Horvath, Kelli Patterson, Megan Scruggs, Tran Bourgeois, Joseph Stanek, Peter C. Minneci, Jennifer H. Aldrink, Keri A. Streby
{"title":"Incidence of Adrenal Insufficiency in Patients With High-Risk Neuroblastoma: A Single-Institution Analysis","authors":"Shachi Srivatsa,&nbsp;Greg Metzger,&nbsp;Kyle Horvath,&nbsp;Kelli Patterson,&nbsp;Megan Scruggs,&nbsp;Tran Bourgeois,&nbsp;Joseph Stanek,&nbsp;Peter C. Minneci,&nbsp;Jennifer H. Aldrink,&nbsp;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}
引用次数: 0
Medulloblastoma Molecular Subgrouping and Outcomes Data of a Single Center From a Low- and Middle-Income Country 来自中低收入国家单一中心的成神经管细胞瘤分子亚群和结局数据
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-21 DOI: 10.1002/pbc.31555
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
{"title":"Medulloblastoma Molecular Subgrouping and Outcomes Data of a Single Center From a Low- and Middle-Income Country","authors":"Naureen Mushtaq,&nbsp;Farrah Bashir,&nbsp;Soha Zahid,&nbsp;Quratulain Riaz,&nbsp;Gohar Javed,&nbsp;Maria Tariq,&nbsp;Bilal Mazhar Qureshi,&nbsp;Kiran Hilal,&nbsp;Vijay Ramaswamy,&nbsp;Cynthia Hawkins,&nbsp;Khurram Minhas,&nbsp;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> &lt; 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}
引用次数: 0
Addressing Disparities in Global Authorship in Pediatric Oncology Research: A Call for Equitable Practices 解决儿童肿瘤学研究中全球作者的差异:呼吁公平实践。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-20 DOI: 10.1002/pbc.31553
Muheem Khan
{"title":"Addressing Disparities in Global Authorship in Pediatric Oncology Research: A Call for Equitable Practices","authors":"Muheem Khan","doi":"10.1002/pbc.31553","DOIUrl":"10.1002/pbc.31553","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009573","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}
引用次数: 0
Improving Completion Rates of Transcranial Doppler Ultrasounds in Children With Sickle Cell Disease Using Quality Improvement Efforts: In-Clinic Versus Population-Based Assessments 提高镰状细胞病儿童经颅多普勒超声检查的完成率:临床与基于人群的评估
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-20 DOI: 10.1002/pbc.31549
Madhav Vissa, Priya Parikh, Chuck McCulloch, Neha Bhasin, Michael D. Cabana, Jong Chung, Anne Marsh, Trisha Wong, Marsha Treadwell, Naomi S. Bardach
{"title":"Improving Completion Rates of Transcranial Doppler Ultrasounds in Children With Sickle Cell Disease Using Quality Improvement Efforts: In-Clinic Versus Population-Based Assessments","authors":"Madhav Vissa,&nbsp;Priya Parikh,&nbsp;Chuck McCulloch,&nbsp;Neha Bhasin,&nbsp;Michael D. Cabana,&nbsp;Jong Chung,&nbsp;Anne Marsh,&nbsp;Trisha Wong,&nbsp;Marsha Treadwell,&nbsp;Naomi S. Bardach","doi":"10.1002/pbc.31549","DOIUrl":"10.1002/pbc.31549","url":null,"abstract":"<div>\u0000 \u0000 <p>Transcranial Doppler (TCD) screening is essential for stroke prevention in children with sickle cell disease (SCD), but screening rates are low nationally. We use Medicaid administrative claims to assess TCD completion rates before and after a 12-month QI initiative to improve rates in the Pacific Sickle Cell Regional Collaborative (PSCRC). We found an increase in TCD screening rates for QI participant sites (52.4% to 58.6%), but not for the nonparticipant SCD population (48.3% to 38.3%). The increase in QI participant sites was not sustained 1 year after the initiative. Local clinic-level QI initiatives may need to be supplemented with health system partnerships to sustain higher TCD screening rates, thereby improving SCD outcomes.</p>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009634","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}
引用次数: 0
Intensified Induction Therapy for Newly Diagnosed, Localized Skeletal Ewing Sarcoma (ISG/AIEOP EW-1): A Randomized, Open-Label, Phase 3, Non-Inferiority Trial 强化诱导治疗新诊断的局限性骨尤文氏肉瘤(ISG/AIEOP EW-1):一项随机、开放标签、3期、非劣效性试验
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-20 DOI: 10.1002/pbc.31551
Roberto Luksch, Emanuela Palmerini, Giuseppe Maria Milano, Anna Paioli, Sebastian Asaftei, Francesco Barretta, Nadia Puma, Marilena Cesari, Elisa Tirtei, Marta Podda, Marta Pierobon, Carla Manzitti, Virginia Ferraresi, Angela Tamburini, Rossella Bertulli, Daniela Di Pinto, Maurizio Mascarin, Giovanni Grignani, Luca Coccoli, Marco Rabusin, Francesco De Leonardis, Marco Gambarotti, Antonina Parafioriti, Silvia Cammelli, Sabina Vennarini, Stefano Ferrari, Davide Maria Donati, Stefano Bastoni, Maura Massimino, Franca Fagioli, Toni Ibrahim
{"title":"Intensified Induction Therapy for Newly Diagnosed, Localized Skeletal Ewing Sarcoma (ISG/AIEOP EW-1): A Randomized, Open-Label, Phase 3, Non-Inferiority Trial","authors":"Roberto Luksch,&nbsp;Emanuela Palmerini,&nbsp;Giuseppe Maria Milano,&nbsp;Anna Paioli,&nbsp;Sebastian Asaftei,&nbsp;Francesco Barretta,&nbsp;Nadia Puma,&nbsp;Marilena Cesari,&nbsp;Elisa Tirtei,&nbsp;Marta Podda,&nbsp;Marta Pierobon,&nbsp;Carla Manzitti,&nbsp;Virginia Ferraresi,&nbsp;Angela Tamburini,&nbsp;Rossella Bertulli,&nbsp;Daniela Di Pinto,&nbsp;Maurizio Mascarin,&nbsp;Giovanni Grignani,&nbsp;Luca Coccoli,&nbsp;Marco Rabusin,&nbsp;Francesco De Leonardis,&nbsp;Marco Gambarotti,&nbsp;Antonina Parafioriti,&nbsp;Silvia Cammelli,&nbsp;Sabina Vennarini,&nbsp;Stefano Ferrari,&nbsp;Davide Maria Donati,&nbsp;Stefano Bastoni,&nbsp;Maura Massimino,&nbsp;Franca Fagioli,&nbsp;Toni Ibrahim","doi":"10.1002/pbc.31551","DOIUrl":"10.1002/pbc.31551","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Several studies have shown that the intensity of treatment in Ewing sarcoma has an impact on outcome. The present trial tested the non-inferiority of intensive, shorter, induction chemotherapy (25 weeks total treatment time) compared to the standard treatment (37 weeks) in non-metastatic Ewing sarcoma (ES) at onset.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>This national, multicenter, parallel, randomized, controlled, open-label, non-inferiority, phase III trial was conducted in 14 specialized hospitals in Italy. Patients aged 2-40 years with newly diagnosed localized ES were randomized to receive four courses of induction therapy (one every 21 days) either with a standard arm (Arm A) or with an intensive arm (Arm B). For consolidation therapy, good responders (GRs) in Arm A received nine courses (37 weeks), while Arm B patients received five courses (25 weeks). Poor responders for both arms received four courses followed by high-dose busulfan/melphalan + autologous stem cell rescue. Follow-up was 5 years.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>In the study period 2009–2018, 274 patients with ES at onset were screened, 248 were eligible, 15 refused randomization, and 233 were randomized (Arm A: 113; Arm B: 120). Median age was 14 years. Arm B was not inferior to Arm A: 5-year EFS was 77.5% and 71.6%, respectively (HR vs. Arm A: 0.74, 90% CI: 0.49–1.14). GRs were 54.9% in Arm A and 62.5% in Arm B. Hematological, gastrointestinal, and cardiovascular Grade ≥3 toxicities had higher frequencies in Arm B.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Intensive induction therapy showed non-inferiority in 5-year EFS when compared with the standard induction therapy. Higher toxicity was reported in Arm B with similar outcome, counterbalanced in GRs with a shorter treatment plan. ClinicalTrials.gov Identifier: NCT02063022.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-01-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31551","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143009656","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}
引用次数: 0
The Utility and Impact of Telemedicine in Childhood Cancer Care: A Mixed Methods Study 远程医疗在儿童癌症护理中的效用和影响:一项混合方法研究。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-01-20 DOI: 10.1002/pbc.31552
Shuvadeep Ganguly, Archana Sasi, Manraj Singh Sra, Swetambri Sharma, Shubhangi Sharma, Mamta Kumari, Deepam Pushpam, Sameer Bakhshi
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