Pediatric Blood & Cancer最新文献

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Trust in Retinoblastoma Care: Understanding Parent-Physician Relationships in Collectivist, Low-Resource Settings. 视网膜母细胞瘤护理中的信任:理解集体主义、低资源环境下家长与医生的关系。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2026-10-13 DOI: 10.1002/1545-5017.70760
Shraddha Namjoshi, Mahati Chittem, Rajisha Sharma, Lauren Kelada, Justin N Baker, Claire E Wakefield
{"title":"Trust in Retinoblastoma Care: Understanding Parent-Physician Relationships in Collectivist, Low-Resource Settings.","authors":"Shraddha Namjoshi, Mahati Chittem, Rajisha Sharma, Lauren Kelada, Justin N Baker, Claire E Wakefield","doi":"10.1002/1545-5017.70760","DOIUrl":"https://doi.org/10.1002/1545-5017.70760","url":null,"abstract":"<p><strong>Background: </strong>Retinoblastoma is an aggressive intraocular malignancy predominantly affecting young children. In lower- and middle-income countries (LMICs), late presentation and limited resources often lead to poor outcomes and intensify the emotional and decisional burden on families, making parent-physician trust a critical determinant of treatment adherence, decision-making and outcomes. Yet, the mechanisms by which trust is built and sustained in non-Western, collectivist healthcare contexts remain poorly understood.</p><p><strong>Objectives: </strong>Grounded in Sisk and Baker's interpersonal trust model, this qualitative study explored (i) barriers and facilitators to trust in parent-physician communication during retinoblastoma care and (ii) parents' and physicians' experiences of trust across diagnosis, treatment and follow-up.</p><p><strong>Methods: </strong>Six physicians and 15 parents of children with retinoblastoma participated in three separate focus group discussions (FGDs) at a tertiary ocular oncology centre in southern India. Thematic analysis, informed by Sisk and Baker's staged trust model, was applied to the FGD transcripts.</p><p><strong>Results: </strong>We coded data to three stages: (1) Initial trust cues: highly regarded, transparent and approachable physicians establish foundational trust; (2) Demonstrated trustworthiness: multilingual, culturally inclusive communication reinforces trust; and (3) Cyclical trust maintenance: collaborative, interdisciplinary team-based care that honours parental insights sustains trust over time.</p><p><strong>Conclusions: </strong>Trust-building in Indian retinoblastoma care is shaped by collectivist relational norms, linguistic plurality, and systemic inequities that extend beyond existing Western models. These findings highlight the need for culturally adapted communication training, multilingual decision-support resources and language-concordant interdisciplinary staffing as standards of care in LMIC retinoblastoma-care settings. Prospective, multi-site research is needed to validate these findings.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70760"},"PeriodicalIF":2.4,"publicationDate":"2026-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149183755","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
The Added Value of 18F-FDG PET/CT in Children With Langerhans Cell Histiocytosis Presenting as a Clinically Isolated Lesion. 18F-FDG PET/CT对Langerhans细胞组织细胞增多症患儿临床孤立病变的附加价值
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2026-10-13 DOI: 10.1002/1545-5017.70765
Xin Wen, Shuxin Zhang, Xia Lu, Ying Kan, Guanyun Wang, Jigang Yang
{"title":"The Added Value of <sup>18</sup>F-FDG PET/CT in Children With Langerhans Cell Histiocytosis Presenting as a Clinically Isolated Lesion.","authors":"Xin Wen, Shuxin Zhang, Xia Lu, Ying Kan, Guanyun Wang, Jigang Yang","doi":"10.1002/1545-5017.70765","DOIUrl":"https://doi.org/10.1002/1545-5017.70765","url":null,"abstract":"<p><strong>Background: </strong>The clinical utility of <sup>18</sup>F-FDG PET/CT in children diagnosed with Langerhans cell histiocytosis (LCH) who present with a clinically isolated lesion remains unclear. This study aimed to assess the added value of <sup>18</sup>F-FDG PET/CT in identifying occult lesions and assessing its impact on disease classification and clinical outcomes in this patient population.</p><p><strong>Methods: </strong>This retrospective study included children who underwent surgical resection of a clinically isolated lesion, were confirmed to have LCH by histopathology, and received <sup>18</sup>F-FDG PET/CT prior to initiation of systemic therapy. Outcomes including detection of additional lesions, disease reclassification, and progression-free survival were analyzed. Kaplan-Meier analysis with log-rank test was used to compare progression-free survival between groups.</p><p><strong>Results: </strong>A total of 44 children were included (median age 4.29 years; 70.5% male). <sup>18</sup>F-FDG PET/CT identified additional lesions in 24 patients (54.5%), leading to reclassification as single-system multifocal disease in 10 (22.7%) and multisystem disease in 14 (31.8%). Four patients (9.1%) had risk organ involvement. <sup>18</sup>F-FDG PET/CT findings altered the treatment strategy in 54.5% of patients. All 5 relapsed patients belonged to the group with additional lesions (p = 0.044).</p><p><strong>Conclusion: </strong><sup>18</sup>F-FDG PET/CT detected additional lesions in over half of the children diagnosed with LCH presenting as a clinically isolated lesion, resulting in disease reclassification and improved risk stratification. <sup>18</sup>F-FDG PET/CT should be routinely performed in this clinical scenario to ensure accurate staging and appropriate treatment.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70765"},"PeriodicalIF":2.4,"publicationDate":"2026-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149183752","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
Combined Bulk and Single-Cell RNA Sequencing Reveals Alterations in the Immune Microenvironment and Candidate Immune-Related Genes Associated With Diagnosis and Relapse in Childhood B-Cell Acute Lymphoblastic Leukemia. 联合大细胞和单细胞RNA测序揭示了与儿童b细胞急性淋巴细胞白血病诊断和复发相关的免疫微环境和候选免疫相关基因的改变。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2026-10-13 DOI: 10.1002/1545-5017.70741
Lin Han, Haitao Zhao, Xiao Mu, Hongling Zheng, Qingwei Guo
{"title":"Combined Bulk and Single-Cell RNA Sequencing Reveals Alterations in the Immune Microenvironment and Candidate Immune-Related Genes Associated With Diagnosis and Relapse in Childhood B-Cell Acute Lymphoblastic Leukemia.","authors":"Lin Han, Haitao Zhao, Xiao Mu, Hongling Zheng, Qingwei Guo","doi":"10.1002/1545-5017.70741","DOIUrl":"https://doi.org/10.1002/1545-5017.70741","url":null,"abstract":"<p><strong>Background: </strong>The association between relapse and remodeling of the bone marrow immune microenvironment in pediatric B-cell acute lymphoblastic leukemia (B-ALL) remains incompletely understood. This study aims to characterize the transcriptional and cellular alterations associated with B-ALL relapse and identify immune-related genes (IRGs) associated with disease recurrence.</p><p><strong>Methods: </strong>Bulk (GSE3910 and GSE28460) and single-cell RNA sequencing (scRNA-seq) (GSE130116) data were obtained from the GEO database, whereas IRGs were retrieved from ImmPort database. GSE3910 dataset was analyzed to identify differentially expressed genes (DEGs), followed by functional enrichment and immune infiltration analysis. scRNA-seq data were used for cell annotation and cell-cell communication analysis to identify key immune shifts. Candidate genes were identified through the intersection of scRNA-seq DEGs, bulk RNA sequencing (bulk RNA-seq) DEGs, and IRGs. Gene-gene interaction (GGI) and transcription factor analyses, and external validation using GSE28460 were subsequently performed.</p><p><strong>Results: </strong>A total of 544 DEGs were identified from DEG3910. Functional enrichment analysis indicated that relapse was associated with enhanced cell proliferation, immune stress, and inflammatory responses. scRNA-seq analysis revealed substantial microenvironmental heterogeneity. Cell-cell communication analysis identified alternations in signaling networks during relapse, particularly toward T and natural killer cells. Pathways related to prostaglandin and NEGR signaling were notably enriched in relapse samples. Integrative analysis identified seven IRGs (ELANE, S100A8, MNDA, S100A9, S100A12, PSAP, and FCN1) associated with relapse and were predominantly associated with myeloid cells.</p><p><strong>Conclusion: </strong>Relapsed pediatric B-ALL may be associated with heterogeneous alterations in immune microenvironment and intercellular communication. The identified genes may represent relapse-associated molecular features and warrant further functional investigation.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70741"},"PeriodicalIF":2.4,"publicationDate":"2026-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149183764","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
Impact of Initial Treatment Setting on Survival in Relapsed Pediatric and AYA Bone Sarcomas: A Secondary Analysis of a Prospective Clinical Trial. 初始治疗环境对复发儿童和AYA骨肉瘤患者生存的影响:一项前瞻性临床试验的二次分析。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2026-10-12 DOI: 10.1002/1545-5017.70756
Justyna Antoniuk-Majchrzak, Katarzyna Bilska, Tomasz Koziński, Jadwiga Węcławek-Tompol, Dorota Sega-Pondel, Radosław Chaber, Wanda Badowska, Katarzyna Rosłan, Hanna Koseła-Paterczyk, Anna Raciborska
{"title":"Impact of Initial Treatment Setting on Survival in Relapsed Pediatric and AYA Bone Sarcomas: A Secondary Analysis of a Prospective Clinical Trial.","authors":"Justyna Antoniuk-Majchrzak, Katarzyna Bilska, Tomasz Koziński, Jadwiga Węcławek-Tompol, Dorota Sega-Pondel, Radosław Chaber, Wanda Badowska, Katarzyna Rosłan, Hanna Koseła-Paterczyk, Anna Raciborska","doi":"10.1002/1545-5017.70756","DOIUrl":"https://doi.org/10.1002/1545-5017.70756","url":null,"abstract":"<p><strong>Background: </strong>Outcomes in relapsed or refractory pediatric and AYA bone sarcomas remain poor, and the impact of early referral to specialized sarcoma centers on survival is unclear. Early centralization of care may improve local and systemic treatment quality, but evidence in this population is limited.</p><p><strong>Methods: </strong>This secondary analysis of a prospective single-center regorafenib trial in children and adolescents/young adults with relapsed or refractory bone sarcomas compared outcomes between patients managed at a specialized high-volume sarcoma center from initial diagnosis (\"internal\" group) and those referred only at relapse (\"external\" group). Baseline characteristics, prior treatments, and trial exposure were assessed. Overall survival (OS) and event-free survival (EFS) were estimated from trial enrollment using Kaplan-Meier methods and compared between groups.</p><p><strong>Results: </strong>Thirty patients were included (18 internal, 12 external). Baseline clinical features, including histologic subtype, metastatic status at diagnosis, and time from diagnosis to first relapse/progression, were broadly similar between groups. EFS from trial enrollment did not differ significantly between groups. However, 24-month OS was longer in patients managed at the specialized center from diagnosis (77% vs. 42%), suggesting a persistent influence of earlier phases of care despite equal access to protocol-based relapse therapy at study entry.</p><p><strong>Conclusions: </strong>Early centralization of care at a specialized pediatric and AYA sarcoma center may confer a long-term survival advantage in relapsed or refractory bone sarcomas, even when investigational relapse therapy is standardized. These findings support timely referral to expert centers and may inform the organization of care and referral pathways for pediatric and AYA patients with bone sarcomas.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70756"},"PeriodicalIF":2.4,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149168708","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
A Novel VPS45 p.(His331Tyr) Variant Presenting With Severe Hypersplenism and Epstein-Barr Virus Reactivation: A Case Report. 一种新的vps45p .(His331Tyr)变异表现为严重的脾功能亢进和爱泼斯坦-巴尔病毒再激活:一例报告。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2026-10-12 DOI: 10.1002/1545-5017.70761
Hamza Haj Mohamad, Zainab Suhail, Maysa Saleh, Lemis Yavuz
{"title":"A Novel VPS45 p.(His331Tyr) Variant Presenting With Severe Hypersplenism and Epstein-Barr Virus Reactivation: A Case Report.","authors":"Hamza Haj Mohamad, Zainab Suhail, Maysa Saleh, Lemis Yavuz","doi":"10.1002/1545-5017.70761","DOIUrl":"https://doi.org/10.1002/1545-5017.70761","url":null,"abstract":"<p><p>VPS45 deficiency is an ultra-rare autosomal recessive disorder causing severe congenital neutropenia, progressive bone marrow fibrosis, and multiorgan involvement. We report a 4-year-old patient with a novel homozygous VPS45 c.991C>T, p.(His331Tyr) variant, presenting with transfusion-refractory hypersplenism and horizontal nystagmus, whose course was further complicated by a laboratory-confirmed Epstein-Barr virus reactivation that subsequently resolved on repeat testing. An older sibling with the same diagnosis had died after failed hematopoietic stem cell transplantation. Management required double-dose blood product transfusion and multidisciplinary evaluation for splenectomy prior to transplantation, illustrating practical challenges in advanced disease management.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70761"},"PeriodicalIF":2.4,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149170208","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
Bone and Soft Tissue Neoplasms in Infants Versus Older Children: A Report From the Cancer in Young People in Canada Program. 婴儿与大龄儿童的骨骼和软组织肿瘤:来自加拿大年轻人癌症项目的报告。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2026-10-12 DOI: 10.1002/1545-5017.70753
Samuel Sassine, Hallie Coltin, Maria Kondyli, Monia Marzouki, Nicolas Prud'homme, Nida Usmani, Rebecca J Deyell, Lesleigh Abbott, Tony H Truong, Sapna Oberoi, Ketan Kulkarni, Josée Brossard, Lynette Bowes, Paul Gibson, Sarah McKillop, Roona Sinha, Lillian Sung, Catherine Vezina, Laura Wheaton, Alexandra P Zorzi, Marie-Claude Pelland-Marcotte, Thai Hoa Tran
{"title":"Bone and Soft Tissue Neoplasms in Infants Versus Older Children: A Report From the Cancer in Young People in Canada Program.","authors":"Samuel Sassine, Hallie Coltin, Maria Kondyli, Monia Marzouki, Nicolas Prud'homme, Nida Usmani, Rebecca J Deyell, Lesleigh Abbott, Tony H Truong, Sapna Oberoi, Ketan Kulkarni, Josée Brossard, Lynette Bowes, Paul Gibson, Sarah McKillop, Roona Sinha, Lillian Sung, Catherine Vezina, Laura Wheaton, Alexandra P Zorzi, Marie-Claude Pelland-Marcotte, Thai Hoa Tran","doi":"10.1002/1545-5017.70753","DOIUrl":"https://doi.org/10.1002/1545-5017.70753","url":null,"abstract":"<p><strong>Background and aims: </strong>Infant bone and soft tissue neoplasms present distinct clinical challenges and lack large-scale data to inform treatment decisions. This study aims to characterize bone and soft tissue neoplasms in infants in Canada and to compare their outcomes with those of older pediatric patients.</p><p><strong>Methods: </strong>We conducted a retrospective population-based cohort study using the Cancer in Young People in Canada (CYP-C) database, including all children <10 years with extracranial bone and soft tissue neoplasms diagnosed between 2001 and 2020. Kaplan-Meier and Cox regression analyses compared treatment-related mortality (TRM), cumulative incidence of relapse (CIR), event-free survival (EFS), and overall survival (OS) between infants (<1 year) and older children (1 to ≤10 years).</p><p><strong>Results: </strong>A total of 1102 children with bone and soft tissue neoplasms were included, including 146 infants (13.2%). The most common infant tumor subtypes were fibrosarcoma (28.8%), malignant rhabdoid tumors (26.7%), rhabdomyosarcoma (25.3%), clear cell and undifferentiated sarcoma (8.9%), and Ewing sarcoma (4.1%). Compared with older children, infants had a higher risk of TRM (4.8% vs. 1.5%; HR (hazard ratio) 4.03; p = 0.003), inferior 1-year (63.7% vs. 81.5%; p < 0.001) and 5-year OS (56.9% vs. 62.0%; p < 0.001), and a lower but non-significant 5-year CIR (26.3% vs. 38.4%; p = 0.19). After adjustment for tumor subtype and metastatic status, infancy remained independently associated with poorer OS (adjusted HR 1.61, 95% confidence interval [CI] 1.13-2.31; p = 0.009).</p><p><strong>Conclusion: </strong>Despite no increased risk of relapse, infants with bone and soft tissue neoplasms have significantly poorer survival than older children, primarily due to a substantially higher risk of TRM.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70753"},"PeriodicalIF":2.4,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149169139","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
Evaluation of Pediatric Oncology Education Interventions for Physicians in Low- and Middle-Income Countries: A Scoping Review. 对低收入和中等收入国家医生儿科肿瘤学教育干预措施的评估:一项范围审查。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2026-10-12 DOI: 10.1002/1545-5017.70757
Fair Berg, Allyson Andujar, Leeanna Fox Irwin, Jordan Horner, Edith Salazar, Saman K Hashmi, Margit K Mikkelsen, Federico Antillon-Klussmann, Guillermo Chantada, Alan Davidson, Laila Hessissen, Daniel C Moreira
{"title":"Evaluation of Pediatric Oncology Education Interventions for Physicians in Low- and Middle-Income Countries: A Scoping Review.","authors":"Fair Berg, Allyson Andujar, Leeanna Fox Irwin, Jordan Horner, Edith Salazar, Saman K Hashmi, Margit K Mikkelsen, Federico Antillon-Klussmann, Guillermo Chantada, Alan Davidson, Laila Hessissen, Daniel C Moreira","doi":"10.1002/1545-5017.70757","DOIUrl":"https://doi.org/10.1002/1545-5017.70757","url":null,"abstract":"<p><strong>Introduction: </strong>Pediatric oncology remains a global health challenge, as children in low- and middle-income countries are less likely than children in high-income countries to survive. Outcomes-focused pediatric oncology education evaluation frameworks remain essential, as high-quality clinician education remains a positive contributor to workforce development and physician competency development.</p><p><strong>Methods: </strong>Following PRISMA-ScR guidelines, a scoping review was conducted with a search of PubMed, PsycINFO, Web of Science, and ERIC for peer-reviewed studies published between January 1, 2005, and May 31, 2026. The goal of the review was to identify published pediatric oncology education interventions for physicians in LMICs and map their evaluation approaches.</p><p><strong>Results: </strong>After duplicate removal, 10,580 unique records were screened, and 27 met criteria for inclusion. Most of the educational interventions described continuing education for practicing physicians and integrated online learning formats in some capacity. Educational evaluation frameworks or models were infrequently cited (18.5%, n = 5) as the basis for evaluation approaches. Reach evaluation levels (e.g., enrollment numbers) were reported by all studies (100%, n = 27), followed by reaction-level (74.1%, n = 20), learning-level (66.7%, n = 18), and outcomes-level (70.4%, n = 19) evaluations.</p><p><strong>Conclusion: </strong>Published pediatric oncology educational interventions and their evaluations remain limited. Evaluations at higher levels, such as institutional outcomes and patient care, remain less common, and few studies described theoretical framing for educational evaluation. Future research should consider the development of more transparent, standardized, and continuum-based evaluation approaches as a key contributor to workforce strengthening initiatives in pediatric oncology.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70757"},"PeriodicalIF":2.4,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149169547","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
Proton Radiotherapy Decreases Incidence of Radiation-Induced Second Malignant Neoplasms Compared to Photon Radiotherapy in Patients With Retinoblastoma. 与光子放疗相比,质子放疗降低了视网膜母细胞瘤患者放射诱导的第二恶性肿瘤的发生率。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2026-10-12 DOI: 10.1002/1545-5017.70754
Sayeh Fattahi, Daniel G Packard, Beow Y Yeap, Roshan V Sethi, Helen A Shih, Myrsini Ioakeim-Ioannidou, Efren Gonzalez, Eric F Grabowski, Laura Wiltsie, Junne Kamihara, Torunn I Yock, Nancy J Tarbell, Karen J Marcus, Shizuo Mukai, Shannon M MacDonald, Kevin X Liu
{"title":"Proton Radiotherapy Decreases Incidence of Radiation-Induced Second Malignant Neoplasms Compared to Photon Radiotherapy in Patients With Retinoblastoma.","authors":"Sayeh Fattahi, Daniel G Packard, Beow Y Yeap, Roshan V Sethi, Helen A Shih, Myrsini Ioakeim-Ioannidou, Efren Gonzalez, Eric F Grabowski, Laura Wiltsie, Junne Kamihara, Torunn I Yock, Nancy J Tarbell, Karen J Marcus, Shizuo Mukai, Shannon M MacDonald, Kevin X Liu","doi":"10.1002/1545-5017.70754","DOIUrl":"https://doi.org/10.1002/1545-5017.70754","url":null,"abstract":"<p><p>Retinoblastoma is the most common primary intraocular neoplasm in children. Use of radiotherapy has decreased due to the incidence of second malignant neoplasms (SMNs). In our study, photon (n = 42) and proton (n = 66) radiotherapy cohorts had median follow-up of 28.7 and 18.2 years, respectively. The majority of patients (74%) had bilateral disease at diagnosis. Proton radiotherapy is associated with significantly lower cumulative incidence of in-field SMNs (0% vs. 11.1% at 15 years, p = 0.005), with three additional cases occurring > 20 years after photon radiotherapy. These findings suggest that when radiotherapy is indicated for patients with bilateral retinoblastoma, proton radiotherapy should be considered when feasible.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70754"},"PeriodicalIF":2.4,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149164911","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
Successful Use of Sutimlimab in two Pediatric Patients With Refractory Cold Antibody-Mediated Autoimmune Hemolytic Anemia. 舒替单抗在两例难治性寒抗体介导的自身免疫性溶血性贫血患儿中的成功应用
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2026-10-12 DOI: 10.1002/1545-5017.70751
Sibgha Zaheer, Rachel Friedmann, Kimberly Cheng, Guy Young, Christopher Denton, Taylor Kim
{"title":"Successful Use of Sutimlimab in two Pediatric Patients With Refractory Cold Antibody-Mediated Autoimmune Hemolytic Anemia.","authors":"Sibgha Zaheer, Rachel Friedmann, Kimberly Cheng, Guy Young, Christopher Denton, Taylor Kim","doi":"10.1002/1545-5017.70751","DOIUrl":"https://doi.org/10.1002/1545-5017.70751","url":null,"abstract":"<p><p>Cold antibody-mediated autoimmune hemolytic anemia, or cold agglutinin syndrome (CAS), in pediatrics is typically transient, although it can often be severe and require transfusion support. Recurrence in children is rare, but those with underlying immune dysfunction can have a refractory course with limited options available. We report a 4-year-old girl with Evans syndrome and life-threatening CAS refractory to transfusions, plasmapheresis, steroids, and immunosuppression as well as a 12-month-old boy status post liver transplant with CAS refractory to steroids, rituximab, and tacrolimus cessation with transition to sirolimus, both of whom received sutimlimab, a C1s inhibitor, with rapid improvement in hemolysis, transfusion independence, and a sustained remission. To our knowledge, these are the first reported uses of sutimlimab in the pediatric population.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70751"},"PeriodicalIF":2.4,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149166784","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
Pseudoexon Activation as a Novel Molecular Mechanism for Chediak-Higashi Syndrome. 假外显子激活作为Chediak-Higashi综合征的新分子机制。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2026-10-12 DOI: 10.1002/1545-5017.70762
Irem Eldem, David B Wilson, Alexa Dickson, Yang Cao, Sebastian Boluarte, Fan Yang, Regina A Clemens, Marwan S Shinawi, Jeffrey J Bednarski
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