Pediatric Blood & Cancer最新文献

{"title":"Correction to “Long-term efficacy and safety of thalidomide treatment in children with β-thalassemia major”","authors":"","doi":"10.1002/pbc.31411","DOIUrl":"10.1002/pbc.31411","url":null,"abstract":"<p>Jian X, Liu X, Peng W, Li L, Hua F, Chen K, Zhang J, Luo S, Yang K, Wu Y. Long-term efficacy and safety of thalidomide treatment in children with β-thalassemia major. Pediatr Blood Cancer. 2023 Apr 28:e30391. https://doi.org/10.1002/pbc.30391.</p><p>The affiliation “Department of Hematology, West China Hospital, Chengdu, China” should be corrected to “Department of Hematology, West China Hospital, Sichuan University, Chengdu, China.”</p><p>Due to an oversight, the affiliation is incomplete for author Yu Wu.</p><p>The author byline and correspondence sections should read:</p><p>Jian Xiao^1,2, Xiaodong Liu², Wei Peng², Lan Li², Fang Hua², Kun Chen², Jin Zhang², Shan Luo², Kun Yang^2*, Yu Wu^1*</p><p>¹Department of Hematology, West China Hospital, Sichuan University, Chengdu, China;</p><p>²Department of Hematology, Zigong First People's Hospital, Zigong, China</p><p>^* Correspondence to:</p><p>Kun Yang, Department of Hematology, Zigong First People's Hospital, Zigong, 643000, China; E-mail: [email protected]</p><p>Yu Wu, Department of Hematology, West China Hospital, Sichuan University, Chengdu, 610041, China, E-mail: [email protected]</p><p>We apologize for this error.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31411","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625644","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}

{"title":"Disparities in Global Authorship and Data Source in the Pediatric Blood and Cancer Journal 2011-2021: Realities and Strategies for Improvement.","authors":"Kathryn Burns, Alexandra Martiniuk, Md Irteja Islam, Dominic Agyemang Opoku, Yuanhui Luo, Dorcas Malahlela, Anita Pickard, Elizabeth Sniderman, Julia Challinor","doi":"10.1002/pbc.31413","DOIUrl":"https://doi.org/10.1002/pbc.31413","url":null,"abstract":"<p><strong>Background: </strong>Research expands knowledge and improves outcomes. Research is needed in all settings, but most often occurs in high-income countries (HIC) compared to low- and middle-income countries (LMICs). Publication in scientific peer-reviewed journals and authorship position are important for academic/clinical advancement. We explored the current state of global authorship and data source distribution for publications in the Pediatric Blood and Cancer (PBC) journal.</p><p><strong>Procedure: </strong>LMIC-affiliated author inclusion and position in selected article categories of the PBC (2011-2021) were recorded. Articles with at least one LMIC-affiliated author (first-listed affiliation) and 5% of exclusively HIC-authored articles were verified. Descriptive statistical analysis was performed.</p><p><strong>Results: </strong>Of 4504 articles reviewed, 593 (13%) included at least one LMIC-affiliated author (517/593 [87%] as first author and 488/593 [82%]) as senior author. In a subset of articles with LMIC-sourced data, 148/675 (22%) included exclusively HIC authors. Within the LMIC-sourced data subset, 81/675 (12%) articles were mixed HIC/LMIC affiliation and 446/675 (66%) were exclusively LMIC-affiliated. The frequency of LMIC-affiliated authors as first or senior author within HIC/LMIC-affiliated collaborations was 31/81 (38%) and 9/81 (11%), respectively.</p><p><strong>Conclusion: </strong>As more than 80% of children live in LMICs and the WHO Global Initiative for Childhood Cancer is increasingly engaged across LMICs, all researchers/clinicians must justly be given an opportunity to conduct, write, publish, and be recognized for their research. PBC is uniquely poised to promote equitable publishing practices and opportunities for professional recognition by drawing on emerging best practices for equitable authorship, including potentially restructuring authorship guidelines and requirements.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31413"},"PeriodicalIF":2.4,"publicationDate":"2024-11-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142605964","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":"Clinicopathological Analysis of a European Cohort of MYOD1 Mutant Rhabdomyosarcomas in Children and Young Adults","authors":"Julia C. Chisholm,&nbsp;Joanna L. Selfe,&nbsp;Rita Alaggio,&nbsp;Edmund Cheesman,&nbsp;Angelica Zin,&nbsp;Lucia Tombolan,&nbsp;Antonina Parafioriti,&nbsp;Giuseppe Maria Milano,&nbsp;Madeleine Adams,&nbsp;Sergey Popov,&nbsp;Maja Česen,&nbsp;Svetlana Tafjord,&nbsp;Meriel Jenney,&nbsp;Paula Z. Proszek,&nbsp;Helene Schlecht,&nbsp;Daniela Di Carlo,&nbsp;Janet Shipley,&nbsp;Anna Kelsey","doi":"10.1002/pbc.31428","DOIUrl":"10.1002/pbc.31428","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Patients with PAX3/7-FOXO1 fusion-negative rhabdomyosarcomas (fnRMS) harbouring the rare L122R <i>MYOD1</i> mutation have significantly poorer prognosis than other fnRMS. We undertook a detailed clinicopathological evaluation of a cohort of patients with <i>MYOD1 mutat</i>ed fnRMS in order to improve risk stratification and treatment options.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>Histological, mutational and clinical data from a cohort of patients with <i>MYOD1</i> mutant RMS treated in Europe were analysed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Thirty-two cases with mutant <i>MYOD1</i> RMS were identified from patients enrolled in sequential European rhabdomyosarcoma clinical trials from 1992 to 2022 (<i>n</i> = 22) and non-trial cohorts (<i>n</i> = 10). Thirty cases had the recurrent L122R missense mutation, one case harboured a K124E mutation and one case had a truncating mutation (S63X). Increased MyoD1 and reduced MYF4 immunostaining were consistent features of <i>MYOD1</i>^L122R-mutated RMS. Applying the risk stratification of the European paediatric Soft tissue sarcoma Study Group (E<i>p</i>SSG) RMS2005 trial, among 20 localised RMS cases that could be assigned a risk category, one was Very High Risk, 13 were High Risk and six were Standard Risk. Eight patients had distant metastases at diagnosis. Of the 25 patients with adequate clinical follow-up data, 15/25 (60%) patients had an event at a median time of 9 months (12/15 included failure of local control) and 13/25 (52%) died of disease.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>This <i>MYOD1</i> mutant cohort demonstrates increased MYOD and reduced MYF4 immunostaining, high risk of local failure and poor survival in agreement with other studies. Increased treatment intensity and improved local control should be considered for these patients.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31428","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142605963","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}

{"title":"Late Events Occurring After 5 Years in Pediatric Rhabdomyosarcoma: A Report From the Children's Oncology Group","authors":"Sara Kreimer,&nbsp;Wei Xue,&nbsp;Amira Qumseya,&nbsp;Timothy B. Lautz,&nbsp;Archana Shenoy,&nbsp;Susan Hiniker,&nbsp;Dana Casey,&nbsp;Rajkumar Venkatramani,&nbsp;Carola Arndt","doi":"10.1002/pbc.31421","DOIUrl":"10.1002/pbc.31421","url":null,"abstract":"<div>\u0000 \u0000 <p>Rhabdomyosarcoma is the most common pediatric soft tissue sarcoma, and 5-year overall survival exceeds 70%. With more long-term survivors, it is critical to understand the frequency of late events, including recurrence, second malignant neoplasm, and death, occurring 5 years after diagnosis, and the variables associated with these events. We report late events in patients enrolled on Intergroup Rhabdomyosarcoma Study Group and Children's Oncology Group trials from 1997 to 2013 including D9602, D9803, D9802, ARST0331, ARST0431, ARST0531, and ARST08P1. A late event occurred in 2.9% of 5-year event-free survivors supporting guidelines to limit surveillance for these events to 5 years from diagnosis.</p>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583777","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":"Soft Tissue Undifferentiated Sarcoma Carrying a Novel Onecut1::Nutm1 Fusion","authors":"Maria Filippidou,&nbsp;Stavros Glentis,&nbsp;Efthymia Rigatou,&nbsp;Philipp Sievers,&nbsp;Florian Selt,&nbsp;Efthymios Dimitriadis,&nbsp;Panagiota Perari,&nbsp;Ilona Binenbaum,&nbsp;Georgia Avgerinou,&nbsp;Cornelis M. van Tilburg,&nbsp;David T. W. Jones,&nbsp;Felix Sahm,&nbsp;Till Milde,&nbsp;Olaf Witt,&nbsp;Stefan M. Pfister,&nbsp;Thomas G. P. Grünewald,&nbsp;Kalliopi Stefanaki,&nbsp;Antonis Kattamis","doi":"10.1002/pbc.31427","DOIUrl":"10.1002/pbc.31427","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583803","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":"Impact of the COVID-19 Pandemic Onset on the Early Careers of Pediatric Oncology Health Professionals and Researchers: A Report From the Children's Oncology Group Young Investigators Committee, Young SIOP Network, and Young SIOPE","authors":"Gemma Bryan,&nbsp;Louise Guolla,&nbsp;Gabriela Inés Villanueva,&nbsp;Sarah Cohen-Gogo,&nbsp;Alejandra Casanovas,&nbsp;Rina Medina,&nbsp;Gabriel Revon-Riviere,&nbsp;Hallie Coltin,&nbsp;Lisa S. Kahalley,&nbsp;Janice S. Withycombe,&nbsp;Roelof van Ewijk,&nbsp;Reineke A. Schoot,&nbsp;Thomas Cash,&nbsp;Reto M. Baertschiger,&nbsp;Mary Frances McAleer,&nbsp;Daniel J. Benedetti,&nbsp;Emily Greengard,&nbsp;Carrie L. Kitko,&nbsp;Adam L. Green,&nbsp;Girish Dhall,&nbsp;Adam J. Esbenshade","doi":"10.1002/pbc.31419","DOIUrl":"10.1002/pbc.31419","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>The COVID-19 pandemic onset had a global debilitating impact on individuals and on burgeoning careers. In 2021, the Children's Oncology Group Young Investigators Committee, Young SIOP (International Society of Paediatric Oncology) Network, and Young SIOPE (European Society for Paediatric Oncology) co-sponsored a survey to explore the impacts of the first year of the pandemic on early-career pediatric oncology professionals with respect to working practices, productivity, professional and career development, personal wellbeing, and changing childcare needs.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The survey comprised demographic, multiple-choice, and free-text questions, and was distributed via email and social media with English, French, and Spanish versions available. Descriptive statistics and chi-square tests were used to compare quantitative data by self-designated gender and country of origin. Qualitative data were described using content analysis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Professionals (<i>N</i> = 499, 26.3% male, 77.2% MDs) in 48 countries (77.6% high income) responded in English (79.4%), Spanish (12.4%), and French (8.2%). Respondents had difficulty obtaining and keeping jobs (26.9%), worsened overall academic productivity (50.7%, with higher rates among bench scientists, <i>p</i> &lt; 0.01), and decreased career opportunities (40.9%). Childcare challenges impacted 56.7% of respondents and was felt more negatively among women (<i>p</i> = 0.008) and in high-income settings (<i>p</i> &lt; 0.0001). Qualitative data (<i>n</i> = 300) highlighted these differences were often attributable to diminished professional/personal boundaries and impacted their personal wellbeing.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The COVID-19 pandemic significantly impacted early-career academic and clinical professionals working in pediatric oncology, with unique challenges noted among those with childcare responsibilities. Career disruptions that resulted from the pandemic should be considered and mitigated by governing bodies and hiring institutions.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31419","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583734","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}

{"title":"Support Experiences and Wishes of Bereaved Parents After the Loss of Their Child to Cancer","authors":"Anna Katharina Vokinger,&nbsp;Eddy Carolina Pedraza,&nbsp;Eva Maria Tinner,&nbsp;André Oscar von Bueren,&nbsp;Katrin Scheinemann,&nbsp;Eva Bergstraesser,&nbsp;Gisela Michel,&nbsp;Eva De Clercq","doi":"10.1002/pbc.31426","DOIUrl":"10.1002/pbc.31426","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>The death of a child has a tremendous impact on parents’ lives. The experience of parents who have lost a child to cancer may differ from other bereavement experiences, including other childhood and adulthood causes of death, because of the uncertainty of the prognosis, the aggressive treatment, and the potential for regret about treatment decisions. Bereavement care remains scarce, and effective interventions to meet the diverse needs of parents have not been defined.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Objective</h3>\u0000 \u0000 <p>To provide insights on bereaved parents’ experiences, their needs, and wishes of support following the loss of their child to cancer.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>We conducted 18 qualitative, in-depth, semi-structured interviews with 23 bereaved parents (seven fathers, 16 mothers), and used reflexive thematic analysis to analyze the data.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Parents received both informal and professional support. Regarding informal support, parents expressed a high level of ambivalence rooted in grief illiteracy. Parents also recognized their own struggles to express what forms of informal support they would have liked to receive. Support provided by healthcare professionals, institutions, and organizations involved in the children's care or in bereavement was inconsistent due to personnel time constraints, interpersonal relationships, or disruptions due to the COVID-19 pandemic. These factors could result in parents not having access to certain forms of support or not receiving long-term support.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Improving grief literacy may strengthen informal support and make discussions of grief and death less taboo. Institutional policies, training, and networking may help to ensure that support provided by healthcare professionals, institutions, and organizations is less vulnerable to inconsistencies.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31426","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583827","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}

{"title":"Osteopathic Manipulative Treatment: A Safe and Feasible Adjunct in Sickle Cell Disease Pain Management","authors":"Amber Brown,&nbsp;Derrick Goubeaux,&nbsp;Seethal A. Jacob,&nbsp;Jennifer A. Belsky","doi":"10.1002/pbc.31433","DOIUrl":"10.1002/pbc.31433","url":null,"abstract":"<div>\u0000 \u0000 <p>Children and adolescents and young adults (CAYAs) with sickle cell disease (SCD) suffer from recurrent acute pain episodes that result in hospitalization and confer substantial morbidity. Pharmacologic pain control is often inadequate, thus non-pharmacologic modalities are needed. Osteopathic manipulative treatment (OMT) is a potentially valuable non-pharmacologic addition to pain management. We describe eight patients hospitalized with SCD pain who received OMT. OMT was safe, feasible, and a promising adjunctive option for management of SCD pain.</p>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142590947","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":"Retrospective Review of Spinal Cord Compression Occurrences and Management in Children With Relapsed, Progressing, or Refractory Malignancies","authors":"Adi Nitzan-Luques,&nbsp;Gabriel Revon-Rivière,&nbsp;Sheila Gandhi,&nbsp;Abdullah AlMutawa,&nbsp;Ronald Grant,&nbsp;Sameera Ahmed,&nbsp;Dana M. Keilty,&nbsp;David C. Hodgson,&nbsp;Derek S. Tsang","doi":"10.1002/pbc.31414","DOIUrl":"10.1002/pbc.31414","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Spinal cord compression (SCC) is a severe complication in pediatric patients with relapsed, progressing, or refractory solid malignancies. This study aims to report the presentation, treatment, and role of proactive radiation therapy in these patients.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>This retrospective study reviewed 28 patients with relapsed, progressing, or refractory solid tumors who were referred for radiation therapy consultation due to potential (impending) or actual spinal cord compression (pSCC and aSCC, respectively) over a 12-year period. Collected data included disease characteristics, details of SCC events, management approaches, and patient outcomes.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Among the 28 patients, 34 SCC events were identified (18 pSCC, 16 aSCC), with neuroblastoma being the most frequent diagnosis (46.4%). No significant differences were noted between pSCC and aSCC groups in pre-event imaging follow-up, age distribution, or malignancy status at SCC presentation. However, aSCC patients exhibited significantly more symptoms at diagnosis. Both groups received SCC-targeted therapy, with no significant differences in functional outcomes, event-free survival (EFS), or overall survival (OS). Pain assessments post treatment showed comparable improvements in both groups.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>Proactive radiotherapy for pSCC did not yield superior outcomes compared to reactive treatment for aSCC. Given the limited benefits observed, proactive RT should be considered on an individual basis, balancing potential QoL improvements against treatment burdens. Further research in larger cohorts is necessary to refine therapeutic strategies for SCC in pediatric oncology.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31414","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583799","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}

{"title":"Outcomes of patients with intermediate-risk neuroblastoma presenting with motor deficits relating to intraspinal tumor extension: A report from the Children's Oncology Group study ANBL0531","authors":"Julie Voeller,&nbsp;Howard M. Katzenstein,&nbsp;Arlene Naranjo,&nbsp;Sheena C. Tenney,&nbsp;Lulu Chen,&nbsp;Wendy B. London,&nbsp;Michael H. Handler,&nbsp;Mary Lou Schmidt,&nbsp;Hiroyuki Shimada,&nbsp;Michael D. Hogarty,&nbsp;Julie Gastier-Foster,&nbsp;Julie R. Park,&nbsp;Susan L. Cohn,&nbsp;John M. Maris,&nbsp;Rochelle Bagatell,&nbsp;Clare J. Twist","doi":"10.1002/pbc.31407","DOIUrl":"10.1002/pbc.31407","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Tumor invasion of the spinal canal is detected radiographically in approximately 15% of patients with newly diagnosed neuroblastoma (NB). The optimal clinical approach to maintain excellent survival outcomes while minimizing long-term sequelae is yet to be defined.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Patients with intermediate-risk neuroblastoma (IR-NB) and radiographically identified intraspinal tumors who were treated on the Children's Oncology Group study ANBL0531 were studied prospectively to evaluate neurologic outcomes related to cord compression. Patients were defined as being symptomatic versus asymptomatic based on reporting of neurologic motor deficits at diagnosis. Patient characteristics, tumor biology, chemotherapy treatment, surgical interventions, and neurologic and disease outcomes are reported.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Of the 92 patients with intraspinal tumors, 42 (46%) were symptomatic and most (73%) had complete resolution of symptoms. Age, degree of motor deficit, and duration of symptoms at diagnosis were not associated with complete resolution. While symptomatic patients were more likely to undergo upfront laminectomy, laminectomy was not associated with improvement of motor symptoms. Administration of additional chemotherapy beyond initial treatment assigned per protocol to achieve the treatment end point was not associated with achieving symptom resolution.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Patients presenting with motor deficits due to intraspinal tumor had excellent survival and favorable neurologic outcomes, with the majority reporting complete resolution of motor symptoms regardless of severity and duration of symptoms at diagnosis or neurosurgical intervention. Prompt diagnosis and initiation of first-line chemotherapy treatment remain priority, while neurosurgical intervention should be reserved for patients with rapid neurologic deterioration. Biology-based therapy and tumor response should continue to be used to maintain favorable outcomes.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 1","pages":""},"PeriodicalIF":2.4,"publicationDate":"2024-11-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142583791","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}