Pediatric Blood & Cancer最新文献

{"title":"Outcomes for Patients Aged 12–18 Months With Metastatic MYCN Non-Amplified Neuroblastoma and Unfavorable Biologic Features (“Mixed Biology Toddlers”): A Report From the International Neuroblastoma Risk Group (INRG) Project","authors":"Mallory R. Taylor,&nbsp;James R. Anderson,&nbsp;Julie R. Park,&nbsp;Meredith S. Irwin,&nbsp;Mark A. Applebaum,&nbsp;Navin R. Pinto,&nbsp;Keri Streby,&nbsp;Sara M. Federico,&nbsp;Carolina Rosswog,&nbsp;Miki Ohira,&nbsp;Pei-Chi Kao,&nbsp;Wendy B. London,&nbsp;Thomas Cash","doi":"10.1002/pbc.31968","DOIUrl":"10.1002/pbc.31968","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Toddlers (age 365–&lt;547 days) with <i>MYCN</i> non-amplified (<i>MYCN</i>-NA) metastatic neuroblastoma (NBL) that have at least one unfavorable biologic feature (“mixed biology toddlers”) are a rare NBL subgroup in need of further study.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>Using the International Neuroblastoma Risk Group (INRG) Data Commons, we identified all toddlers with newly diagnosed metastatic, <i>MYCN</i>-NA NBL. Unfavorable biologic features included (1) unfavorable histology, (2) DNA index ≤1, (3) 1p loss of heterozygosity (LOH), and (4) 11q LOH. Kaplan–Meier curves of event-free survival (EFS) and overall survival (OS) were generated and compared using a log rank test; Cox proportional hazard regression models were used for multivariable analysis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Of the 450 patients identified, 157 had ≥1 unfavorable biologic feature and 26 had all favorable features. After adjusting for unfavorable biologic features, only high ferritin and high lactate dehydrogenase (LDH) remained significantly associated with worse EFS and OS. A total of 5-year EFS for mixed biology toddlers was 74.3% versus 88.5% for those with all favorable biologic features; 5-year OS was 79.3% versus 100%, respectively.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Mixed biology toddlers have lower EFS and OS compared to those whose tumors have favorable biologic features. Inclusion in high-risk NBL trials should be considered for this rare subgroup.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964430","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":"A Misdiagnosed Primary CNS-PTLD Concurrent With Invasive Aspergillosis in a Child Following Allogeneic Stem Cell Transplantation","authors":"Camilla Cereda,&nbsp;Francesca Vendemini,&nbsp;Sonia Bonanomi,&nbsp;Marta Adavastro,&nbsp;Pietro Casartelli,&nbsp;Giovanna Lucchini,&nbsp;Sara Napolitano,&nbsp;Giulia Prunotto,&nbsp;Marta Verna,&nbsp;Giovanni Palumbo,&nbsp;Guglielmo M. Migliorino,&nbsp;Sergio M. I. Malandrin,&nbsp;Alice Parisi,&nbsp;Alice Arduini,&nbsp;Simone Cesaro,&nbsp;Adriana Balduzzi,&nbsp;Giorgio Ottaviano","doi":"10.1002/pbc.32028","DOIUrl":"10.1002/pbc.32028","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964315","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":"Two Cases of Juvenile Myelomonocytic Leukemia With Identical Somatic PTPN11 Mutations in Children After In Utero Exposure to Thiopurine-Containing Chemotherapy","authors":"Marek Ussowicz,&nbsp;Monika Rosa,&nbsp;Monika Biedroń,&nbsp;Renata Tomaszewska,&nbsp;Tomasz Szczepański,&nbsp;Charlotte Niemeyer","doi":"10.1002/pbc.32003","DOIUrl":"10.1002/pbc.32003","url":null,"abstract":"<div>\u0000 \u0000 <p>Juvenile myelomonocytic leukemia (JMML) is a rare pediatric malignancy without defined environmental triggers. We report two cases of JMML with identical somatic <i>PTPN11</i> mutations in children exposed in utero to thiopurine-containing chemotherapy. One mother underwent antileukemic treatment including thioguanine during early pregnancy; the other received 6-mercaptopurine for Crohn's disease throughout gestation. Both children were diagnosed with JMML at 34 and 37 months, respectively, with pathogenic <i>PTPN11</i> exon 3 (c.226G&gt;A, p.E76K) mutation. Each underwent allogeneic hematopoietic stem cell transplantation and is currently in remission. These findings suggest a possible link between prenatal thiopurine exposure and leukemogenesis.</p>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964369","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":"Ras-Associated Autoimmune Leukoproliferative Disorder With Cutaneous and Ocular Involvement","authors":"Florent Amatore,&nbsp;Agathe Beaufrere,&nbsp;Aurore Aziz,&nbsp;Aurélie Haffner,&nbsp;Nicolas Macagno,&nbsp;Vincent Barlogis,&nbsp;Audrey Petit,&nbsp;Hélène Cave,&nbsp;Gauthier Decool,&nbsp;Philippe Minodier,&nbsp;Marie Loosveld,&nbsp;Laurene Fenwarth,&nbsp;Anne-Laure Jurquet,&nbsp;Quentin Berthier,&nbsp;Lionel Fourcade,&nbsp;Olympe Gaudray,&nbsp;Marie-Aleth Richard,&nbsp;Thierry David,&nbsp;Paul Saultier,&nbsp;Stéphanie Mallet","doi":"10.1002/pbc.32019","DOIUrl":"10.1002/pbc.32019","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964428","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":"Introductory Course in Pediatric Oncology for Registrars/Residents and Medical Officers in Sub-Saharan Africa: A Pilot Study","authors":"Saman K. Hashmi,&nbsp;Inam Chitsike,&nbsp;Justin Makasa Mulindwa,&nbsp;Gita Naidu,&nbsp;Leeanna Fox Irwin,&nbsp;Fair Berg,&nbsp;Georgia Chatman,&nbsp;Allyson Andujar,&nbsp;Carlos Rodriguez-Galindo,&nbsp;Nickhill Bhakta,&nbsp;Uma Athale,&nbsp;Miguel Bonilla,&nbsp;Daniel C. Moreira","doi":"10.1002/pbc.32017","DOIUrl":"10.1002/pbc.32017","url":null,"abstract":"<div>\u0000 \u0000 <p>A shortage of pediatric oncologists limits teaching and supervision of registrars/residents and medical officers working in pediatric oncology units. Our aim was to develop and pilot a blended, introductory course in pediatric oncology in Zimbabwe and Zambia comprising self-paced online modules and case-based discussions. The self-paced course was subsequently adopted in South Africa. From May 2021 until August 2024, 25 learners completed the pre-test, and eight completed the post-test, with higher average post-test scores (<i>p</i> = 0.0253). Learners found the content relevant, with knowledge gain noted by local pediatric oncologists. Future courses will include strategies to reduce learner attrition.</p>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964436","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":"Association Between Caregiver Health Literacy, Social Determinants of Health, and the Pediatric Oncology New Diagnosis Educational Experience","authors":"K. Elizabeth Skipper,&nbsp;Aman Wadhwa,&nbsp;Peng Li,&nbsp;Marti Rice,&nbsp;Nataliya V. Ivankova,&nbsp;Paula D. Campos González,&nbsp;Adelynn J. Salem,&nbsp;Smita Bhatia,&nbsp;Wendy Landier","doi":"10.1002/pbc.32014","DOIUrl":"10.1002/pbc.32014","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Caregiver new diagnosis education is understudied in pediatric oncology. Caregiver health literacy (HL) and social determinants of health (SDoH) may affect the caregiver-reported educational experience (CREE) of receiving new diagnosis education. Our purpose was to determine relationships between HL, SDoH, and CREE by surveying caregivers who received new diagnosis education for their children undergoing cancer treatment.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Procedure</h3>\u0000 \u0000 <p>HL was assessed using the Newest Vital Sign (limited HL: 0–3; adequate HL: 4–6). Five SDoH domains (economic stability; education access/quality; healthcare access/quality; neighborhood/built environment; social/community context) were assessed (adverse SDoH: ≥1 adverse factor). CREE was assessed using items adapted from the Agency for Healthcare Research and Quality (AHRQ) Health Literacy Supplemental Items (possible range: 6–36; unfavorable: &lt;33). Multivariable logistic regression was used to identify associations between unfavorable CREE score (outcome), SDoH, and HL, adjusting for caregiver sociodemographic and child clinical factors.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Of 67 caregivers, 77.6% were mothers, 65.7% non-Hispanic White, median [range] age was 34 [20–69] years. Among the patients (<i>N</i> = 67): males were 56.7%, leukemia 55.2%, and median [range] age 4.4 [0.2–17.5] years. Over one-third (35.8%) of caregivers had limited HL, 71.6% had one or more adverse SDoH, and 40.3% reported unfavorable CREE. In multivariable logistic regression, adverse SDoH was associated with 6.3-fold higher odds of unfavorable CREE (adjusted odds ratio [aOR] = 6.3, 95% confidence interval [CI] = 1.4–28.7, <i>p</i> = 0.017); adequate HL was associated with 8.0-fold higher odds of unfavorable CREE (aOR = 8.0, 95% CI = 1.9–34.4, <i>p</i> = 0.005).</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>SDoH and HL are important factors to consider when providing new diagnosis education to pediatric oncology caregivers.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964352","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":"Rapid Donor-Specific Antibodies Clearance With Short-Course Blinatumomab Prior to Haploidentical HSCT: A Case of Successful Engraftment","authors":"Zebin Luo,&nbsp;Miner Gu,&nbsp;Xiaoping Guo,&nbsp;Fenying Zhao,&nbsp;Xiaojun Xu","doi":"10.1002/pbc.32018","DOIUrl":"10.1002/pbc.32018","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964388","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":"Sustainability of a Cash Transfer Program in Malawi: A CANCaRe Africa “Zero Abandonment” Pilot","authors":"Harriet Khofi,&nbsp;Beatrice Chikaphonya,&nbsp;Asya Agulnik,&nbsp;Lillian Sung,&nbsp;Cecilia Mdoka,&nbsp;Elizabeth Molyneux,&nbsp;Ross C. Brownson,&nbsp;George Chagaluka,&nbsp;Trijn Israels","doi":"10.1002/pbc.32021","DOIUrl":"10.1002/pbc.32021","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Background</h3>\u0000 \u0000 <p>Sustainability—the continued delivery of an intervention's intended benefits after external donor support ends—is essential to ensure long-term impact and success. In 2019, a cash transfer program in Blantyre, Malawi, provided full transport reimbursement (mean ∼200 Euros/family), counseling, and patient tracking for caregivers of children with common and curable cancers. This reduced treatment abandonment from 19% to 7% (<i>p</i> &lt; 0.001). We evaluated the program's sustainability over a 4-year period post-implementation.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>The intervention was implemented from June 2019 to June 2020. We conducted a mixed-methods study to assess sustainment and sustainability. We evaluated the continuation of cash transfers and treatment abandonment rates among children (&lt;16 years) newly diagnosed with common and curable cancers from 2022 to 2024. Exploratory stakeholder interviews identified perceived facilitators and barriers to sustainability.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>The program continued beyond 2020 with modifications—transport reimbursement was limited to the home district. A new donor assumed funding. Reduced abandonment rates were sustained: 9% (10/110) in 2022, 10% (13/127) in 2023, and 3% (2/70) in 2024 (with 40% still on treatment) (<i>p</i> = 0.28). Reported facilitators of sustainability included high acceptability, local ownership, demonstrated effectiveness, and availability of alternative donor support. Barriers included resource constraints, competing health priorities, and concerns about misuse of funds.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusion</h3>\u0000 \u0000 <p>Four years after initial funding ended, the cash transfer intervention remained active with sustained reductions in treatment abandonment. These findings highlight the potential for sustained impact of financial support programs in low-resource settings. Further research is needed to identify the key determinants of sustainability, informing future scale-up efforts.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964401","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":"Severe Hearing Loss in Children With Central Nervous System Tumors: A Population-Based Cancer in Young People in Canada (CYP-C) Report","authors":"Hallie Coltin,&nbsp;Ofélie Trudeau-Ferrin,&nbsp;Sébastien Perreault,&nbsp;Lucie Lafay-Cousin,&nbsp;Derek S. Tsang,&nbsp;Samuele Renzi,&nbsp;Valérie Larouche,&nbsp;Craig Erker,&nbsp;Juliette Hukin,&nbsp;Randy Barber,&nbsp;Paul C. Nathan,&nbsp;Annie Huang,&nbsp;Eric Bouffet,&nbsp;Vijay Ramaswamy","doi":"10.1002/pbc.32024","DOIUrl":"10.1002/pbc.32024","url":null,"abstract":"<div>\u0000 \u0000 \u0000 <section>\u0000 \u0000 <h3> Purpose</h3>\u0000 \u0000 <p>Children with central nervous system (CNS) tumors are prone to treatment-related hearing loss (HL) and subsequent functional impairment. This study reports a dedicated population-based analysis of CNS tumor-specific rates and predictors of early severe HL.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Methods</h3>\u0000 \u0000 <p>A cohort study of children ≤15 years diagnosed with CNS tumors between 2001 and 2019 through the Cancer in Young People in Canada (CYP-C) program. The primary outcome was Grade 3 and 4 severe HL within 5 years following diagnosis.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Results</h3>\u0000 \u0000 <p>Among 3201 children with CNS tumors, 5.1% experienced early severe HL. Children with medulloblastoma (<i>N</i> = 570) and ATRT/other embryonal tumors (<i>N</i> = 269) had higher rates of early HL (16.1%, 15.2%, respectively). Cisplatin was administered to 80.1% of children with embryonal tumors, and 67.3% received radiotherapy. In children with medulloblastoma, age less than 6 years at diagnosis (OR 2.4, 1.5–3.8; vs. ≥6 years), radiation (OR 3.5, 1.6–7.6), and cisplatin (OR 20.4, 1.3–329.7) predicted early severe HL. Younger age at diagnosis doubled the probability of early severe HL (10.6% in &lt;6 years vs. 4.8% in ≥6 years), while radiation exposure tripled the probability across age groups (29.8% and 10.6% if &lt;6 years; 15.3% and 4.8% in ≥6 years). In children with ATRT/other embryonal tumors, cisplatin (OR 31.6, 1.9–521.9) was the sole predictor of early severe HL.</p>\u0000 </section>\u0000 \u0000 <section>\u0000 \u0000 <h3> Conclusions</h3>\u0000 \u0000 <p>High rates of early HL were observed in children with embryonal tumors. Younger children who received radiotherapy had higher probabilities of early HL, suggesting an additive interaction between age and radiation. Standardized otoprotection and research on cisplatin avoidance and therapy de-escalation in young children with embryonal tumors are urgently needed.</p>\u0000 </section>\u0000 </div>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://onlinelibrary.wiley.com/doi/epdf/10.1002/pbc.32024","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964367","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":"Paired Tumor/Normal Sequencing Identifies a SEPT9::ABL1 Fusion in a Child With T-Cell Lymphoblastic Lymphoma","authors":"Miyoung Lee,&nbsp;Dailia B. Francis,&nbsp;Melinda G. Pauly,&nbsp;Ryan J. Summers,&nbsp;Bojana Pencheva,&nbsp;Ayjha C. Brown,&nbsp;Jodi Dougan,&nbsp;Christopher C. Porter","doi":"10.1002/pbc.32023","DOIUrl":"10.1002/pbc.32023","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":"72 11","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-08-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144964395","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}