Pediatric Blood & Cancer最新文献

{"title":"Life-Threatening Infantile Fibrosarcoma in an Infant.","authors":"Katharina Daugs, Ehrenfried Schindler, Christian Vokuhl, Andreas Müller, Arndt Borkhardt, Florian Babor","doi":"10.1002/pbc.31596","DOIUrl":"https://doi.org/10.1002/pbc.31596","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31596"},"PeriodicalIF":2.4,"publicationDate":"2025-02-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143390481","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":"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}

{"title":"Temporal Relationships Between General Self-Efficacy, Social Support and Health-Related Quality of Life Among Adult Survivors of Childhood Acute Lymphoblastic Leukemia: A 9-Year Follow-Up Study","authors":"Katarina Aili,&nbsp;Marianne Jarfelt,&nbsp;Andreas Ivarsson,&nbsp;Susann Arvidsson,&nbsp;Maria Olsson,&nbsp;Jens M Nygren","doi":"10.1002/pbc.31578","DOIUrl":"10.1002/pbc.31578","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Acute lymphoblastic leukemia (ALL) is the most prevalent childhood malignancy. To improve long-term health-related quality of life (HRQOL) in adult survivors of childhood ALL, more longitudinal studies are needed to assess outcomes and risk factors throughout treatment and survivorship. The aim of this study was to examine the long-term changes in HRQOL, self-efficacy, and social support among adult survivors of childhood ALL and to explore the temporal relationship between HRQOL, self-efficacy, and social support.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>The study includes 148 adult childhood ALL survivors who responded to a questionnaire assessing HRQOL (SF36), self-efficacy (General Self-Efficacy Scale, GSE), and quantitative and qualitative social support (AVSI and AVAT in SS13) in 2012 and 2021. Changes in the HRQOL, GSE, and social support were calculated using paired <i>t</i>-tests. Bayesian path models were specified, and separate models were estimated for each relationship between GSE and AVSI, and AVAT and HRQOL. Cross-sectional associations, autoregressive effects within constructs over time, and cross-lagged effects between two variables over time were specified within each model.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The mean of six of the eight HRQOL dimensions, as well as quantitative and qualitative social support, deteriorated during the 9-year follow-up. Self-efficacy was unchanged. Temporal positive relationships were found between baseline GSE and the HRQOL dimension of social functioning, as well as between social support and the HRQOL dimensions of physical functioning, vitality, and mental health at follow-up.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>The findings highlight the importance of self-efficacy and social support as potential buffering factors for HRQOL in adult survivors of childhood ALL over time.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-02-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.31578","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143256270","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":"Anti-Tr/DNER Antibody-Associated Ataxia in a Pediatric Hodgkin Lymphoma Survivor: Successful Treatment With Plasmapheresis and IVIG","authors":"Armin Adibi,&nbsp;Iman Adibi,&nbsp;Hamidreza Danaei","doi":"10.1002/pbc.31594","DOIUrl":"10.1002/pbc.31594","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-02-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143256314","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":"Cytoreductive Surgery and Hyperthermic Intraperitoneal Chemotherapy for Pediatric Abdominal Rhabdomyosarcoma: Experience of a Reference Pediatric Oncosurgical Center","authors":"Cristian Urla,&nbsp;Benedikt Wagner,&nbsp;Andreas Schmidt,&nbsp;Frank Fideler,&nbsp;Jürgen Schäfer,&nbsp;Felix Neunhoeffer,&nbsp;Martin Ebinger,&nbsp;Guido Seitz,&nbsp;Jörg Fuchs","doi":"10.1002/pbc.31573","DOIUrl":"10.1002/pbc.31573","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Pediatric abdominal rhabdomyosarcoma (RMS) represents an oncosurgical challenge. For adults with peritoneal carcinomatosis and for children/adolescents with desmoplastic small round cell tumors (DSRCT), cytoreductive surgery (CRS) combined with hyperthermic intraperitoneal chemotherapy (HIPEC) has been shown to be an effective treatment. Thereby, we present the largest series on CRS and HIPEC in patients with abdominal RMS.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Patients and Methods</h3>\u0000 \u0000 <p>Between August 2013 and January 2024, 18 patients (9 female and 9 male) with abdominal RMS underwent CRS and HIPEC at our institution. All patients were treated according to an institutionalized protocol. The indication for surgery was established by a multidisciplinary national tumor board. HIPEC was performed using cisplatin (37.5–100 mg/m²) and doxorubicin (15–30 mg/m²) for 60 min at 42°C. A retrospective review of patient's records was performed.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The median age at operation was 3.8 years. A complete cytoreduction was achieved in 17/18 patients. Median length of hospital stay was 11 days. Mean length of ICU stay was 2.5 days. No procedure-associated complications and no major short-/long-term toxicities were recorded. The median follow-up was 20 months. The 5-year overall survival (OS) and event-free survival (EFS) were 72% and 53%, respectively. Patients &gt;10 years and those with alveolar histology had the worst prognosis (OS 0%). Patients with relapse after HIPEC (6/18) had a significantly poorer survival compared with those without recurrence (OS 33% vs. 100%, <i>p</i> = 0.001).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>CRS and HIPEC are effective in patients with abdominal RMS. Patients &gt;10 years, those with alveolar RMS and those with relapse after HIPEC had the worst outcome.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143189432","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":"Assessment of Caregivers and Their Children With Cancer Regarding Information Provided by Healthcare Teams: Advancing Stages Toward the Creation of a Structured Education Program at the National Level","authors":"Verónica De la Maza,&nbsp;Virginia Fierro,&nbsp;Carolina Facusse,&nbsp;Daniela Carvajal,&nbsp;Tiare Pavez,&nbsp;Karen Méndez,&nbsp;María Barra,&nbsp;Juan P. Torres,&nbsp;Daniela Torres,&nbsp;Paz Moscoso,&nbsp;María Santolaya,&nbsp;Claudia Paris","doi":"10.1002/pbc.31577","DOIUrl":"10.1002/pbc.31577","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>The dissemination of information to caregivers of pediatric cancer patients in Chilean hospitals is now sporadic and contingent upon available time, underscoring the need for a systematic education program. This study assesses caregivers’ perceptions of the information provided by healthcare teams to support the development of a national education program tailored to their needs.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A descriptive, prospective, multicenter study was conducted from June 2021 to March 2022 across six public hospitals in Chile. The study included caregivers of children undergoing cancer treatment. Caregivers completed a survey assessing the education received and their preferred educational methods. Data were analyzed using STATA 18 and Graph Pad 6.0, with qualitative responses analyzed through an iterative coding process.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Of 173 respondents, 94% rated the education received as very good or good. While 51% felt well-informed at hospital discharge, 28% desired more information. Caregivers preferred information from healthcare teams (88%), websites (55%), and written materials (51%). Significant differences were found in preferred educational content based on cancer type and the time elapses since diagnosis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Caregivers generally rated the education provided positively but expressed a need for more structured and targeted information. The findings inform the design of a national education program, emphasizing the need for tailored content and improved communication strategies to enhance caregiver support.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143188886","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":"Differential Expression of BCL2 and IGFBP2 in Childhood Immune Thrombocytopenic Purpura Clinical Subtypes: Implications for Predicting Disease Progression and Apoptotic Regulation","authors":"Salwa Bakr,&nbsp;Hanaa El Dash,&nbsp;Asmaa Ahmed Ali Youssef,&nbsp;Rehab Galal Abd El-Hamid","doi":"10.1002/pbc.31586","DOIUrl":"10.1002/pbc.31586","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Immune thrombocytopenic purpura (ITP), which poses challenges in treatment response, is an autoimmune-mediated bleeding disorder with an extremely complex pathogenesis and unpredictable clinical progression. Dysregulation of apoptotic pathways may influence both the pathogenesis and prognosis of ITP. This study aimed to evaluate the expression patterns of the apoptotic protein insulin-like growth factor-binding protein 2 (IGFBP2) and the anti-apoptotic protein B-cell lymphoma 2 (BCL2) as potential predictive or prognostic biomarkers for disease progression in childhood ITP.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Patients and Methods</h3>\u0000 \u0000 <p>The expression levels of BCL2 and IGFBP2 were assessed in peripheral blood samples from 40 pediatric ITP patients and 30 age- and sex-matched healthy controls using enzyme-linked immunosorbent assays.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Plasma levels of BCL2 and IGFBP2 were higher in ITP patients than in control subjects. Although the difference in IGFBP2 expression was not statistically significant (<i>p</i> = 0.910), BCL2 expression was significantly elevated (<i>p</i> &lt; 0.001). Notably, chronic ITP patients had significantly lower levels of both IGFBP2 and BCL2 markers compared to patients who achieved spontaneous recovery (<i>p</i> &lt; 0.001).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>BCL2 and IGFBP2 appear to be promising noninvasive biomarkers for predicting disease outcomes in newly diagnosed ITP, emphasizing the need for validation in large-scale, multicenter longitudinal studies.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143189673","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":"Hematological Manifestations of Copper Deficiency in a Child on Ketogenic Diet","authors":"Raquel da Costa Neves,&nbsp;João Ferreira Simões,&nbsp;Sandra Jacinto,&nbsp;Ana Paula Rocha,&nbsp;Paula Kjöllerström","doi":"10.1002/pbc.31597","DOIUrl":"10.1002/pbc.31597","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143189674","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":"Improving Time to Bleeding Disorder Treatment in the Emergency Department","authors":"Lisa Littner,&nbsp;Evan Thomas,&nbsp;Laura Mooney,&nbsp;Julie Doyle,&nbsp;Julie Hendrickson,&nbsp;Cristina Tarango","doi":"10.1002/pbc.31570","DOIUrl":"10.1002/pbc.31570","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Knowledge of the guidelines for treating people with bleeding disorders (PwBD) is vital for emergency department (ED) staff, as PwBD rely on the ED for treatment of acute bleeding episodes or injuries. Unfamiliarity with these guidelines can delay treatment. We aimed to improve the timely treatment of PwBD seeking emergency care by reducing the mean time-to-factor administration in the ED by 20%, from the baseline of 110 to 88 minutes.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Our quality improvement (QI) initiative (2019–2024) used Plan-Do-Study-Analyze cycles. Weekly electronic health record (EHR) reports tracked PwBD's time of arrival, admission, clotting factor concentrate ordering, and administration. Interventions targeted delays identified in the process.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Baseline data showed a 110-mean time-time-to-factor. Our aim of reducing the time-to-factor by 20% from the baseline of 110 minutes was reached in May 2020. This improvement was exceeded and sustained throughout the project. At the conclusion, we achieved a 62% reduction in time-to-factor and a mean time-to-factor of 42 minutes.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>By using an EHR tool, we were able to identify when during the ED visit process delays occurred, which helped us better target our interventions. Our educational-based interventions were adapted to reach our intended audiences while working around barriers related to the COVID-19 pandemic, such as high patient volumes and staff shortages. Our results show a decrease in time to factor in the ED and an increase in the PwBD receiving factor within 60 minutes of arrival time. This has been a sustainable improvement.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143189688","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":"Image-Guided Biopsy for the Diagnosis and Molecular Profiling of Hepatoblastoma","authors":"Sean Schoeman,&nbsp;Fernando Escobar,&nbsp;Portia Kreiger,&nbsp;Suzanne MacFarland,&nbsp;Peter Mattei,&nbsp;Phassawan Rungsiprakarn,&nbsp;Abhay Srinivasan,&nbsp;Michael Acord","doi":"10.1002/pbc.31575","DOIUrl":"10.1002/pbc.31575","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Introduction</h3>\u0000 \u0000 <p>Studies on the use of image-guided percutaneous biopsy for hepatoblastoma (HB), and recommendations put forth by the pediatric hepatic international tumor trial (PHITT), are limited. It is unknown if sufficient tissue can be obtained for trial enrollment as well as molecular profiling, which will likely play a key role in informing future treatment strategies.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods and Materials</h3>\u0000 \u0000 <p>Patients with HB who underwent percutaneous biopsy at initial diagnosis in interventional radiology (IR) over a 12-year period at a single center were included. Patient demographics, pretreatment extent of disease (PRETEXT) stage, tumor size, and procedure details were collected. Pathology reports and tumor genomic analysis, when performed, were assessed for specimen adequacy. Post-procedure records were assessed for hemoperitoneum.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>A total of 33 percutaneous biopsies were performed on 32 patients [17 female; median age 1.3 years (IQR: 0.7–2.5 years); median weight 10.5 kg (IQR: 7.4–12.7 kg)]. Most (<i>n</i> = 27) had a single liver lesion, and most (<i>n</i> = 18) were PRETEXT II. A total of 15 were positive for at least one annotation factor. Median longest tumor axis was 9.3 cm (IQR: 5.0–13.5 cm). A total of 16 patients had concurrent non-targeted liver biopsy, per PHITT recommendations. An 18-gauge instrument was most commonly used (<i>n</i> = 24, 73%) with a median of 8 cores (IQR: 6–12) obtained. There were no instances of hemoperitoneum.</p>\u0000 \u0000 <p>Tissue was adequate for histologic diagnosis in 97% (<i>n</i> = 32), with histologic subtyping obtained in 94% (30/32). When available (<i>n</i> = 29), comparison with the subsequent surgical resection specimen showed subtype concordance in 15 (52%) patients and minor variations secondary to sampling or treatment effect in 14 patients. Molecular profiling was completed on 21/21 specimens (100%), with 19/21 (90%) showing potentially clinically significant variants, most commonly in <i>CTNNB1</i> (16/21).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>In this single-center study, percutaneous biopsy resulted in no serious adverse events, a high rate of diagnosis, and successful subtyping and molecular characterization of HB.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 4","pages":""},"PeriodicalIF":2.4,"publicationDate":"2025-02-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143189756","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}