Pediatric Blood & Cancer最新文献

{"title":"T-Cell Lymphoblastic Lymphoma in a Child With Trisomy 21: A Unique Case Demonstrates the Need for Malleability in Therapy.","authors":"Sydney Tape, Stuart Winter, Lane Miller, Megan Hilgers, Johann Hitzler, Karen R Rabin, Nathan Gossai","doi":"10.1002/1545-5017.70256","DOIUrl":"10.1002/1545-5017.70256","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70256"},"PeriodicalIF":2.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147474814","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":"Treatment Decision-Making Roles and Preferences Among Adolescents and Young Adults With Cancer.","authors":"Amanda M Gutierrez, Jill O Robinson, Wendy A Allen-Rhoades, Hadley S Smith, Isabel Canfield, Rebecca L Hsu, Sarah R Scollon, Lauren R Desrosiers-Battu, Nicole M Schneider, Frank Y Lin, Donald Williams Parsons, Sharon E Plon, Mary A Majumder, Janet Malek, Amy L McGuire","doi":"10.1002/1545-5017.70235","DOIUrl":"10.1002/1545-5017.70235","url":null,"abstract":"<p><strong>Background: </strong>Decision-making (DM) dynamics between adolescents and young adults (AYAs) with cancer, parents, and oncologists remain underexplored in diverse populations. We examined cancer treatment DM preferences among an ethnically and socioeconomically diverse group of AYAs and their parents.</p><p><strong>Procedure: </strong>We surveyed AYAs with cancer (n = 75) and their parents (n = 52) participating in the Texas KidsCanSeq study. Among AYA-parent pairs, we compared preferred and actual treatment DM roles. We explored associations among DM roles, having pediatric-to-adult care transition discussions, and clinical and sociodemographic characteristics. We report AYAs' and parents' preferences for involvement in shared decision-making (SDM) with oncologists about cancer treatment.</p><p><strong>Results: </strong>AYAs' DM role preferences varied: 44% preferred to equally share DM with parents, 31% preferred AYA-led DM, and 25% preferred parent-led DM. Parents who preferred parent-led DM had younger AYAs than parents who preferred equally shared DM (p = 0.011). For actual DM, 48% of AYAs reported DM was equally shared, 24% reported DM was AYA-led, and 28% reported DM was parent-led. Most AYAs had their preferred role (73%). AYAs' actual role did not significantly differ from their own or their parents' preferences (both p > 0.05). Approximately half of AYAs reported recent pediatric-to-adult care transition discussions. AYAs' DM preferences and participation in transition discussions did not significantly differ along clinical or sociodemographic characteristics (all p > 0.05). AYAs and parents perceived high importance across all aspects of SDM with oncologists.</p><p><strong>Conclusion: </strong>In this diverse study population, AYAs desired engagement in treatment DM and SDM. Although specific role preferences varied, opportunities existed to increase discussion around pediatric-to-adult care transition.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70235"},"PeriodicalIF":2.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147481153","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 PRSZT Registry Analysis of Prognostic Factors Influencing Survival and Relapse Rates After Second Allogeneic Hematopoietic Stem Cell Transplantation in Pediatric Acute Lymphoblastic Leukemia.","authors":"Ava Momm, Imme Haubitz, Rita Beier, Martin Zimmermann, Andrej Lissat, Peter Bader, Matthias Eyrich, Jan-Henning Klusmann, Matthias Wölfl, Paul-Gerhardt Schlegel, Verena Wiegering","doi":"10.1002/1545-5017.70263","DOIUrl":"10.1002/1545-5017.70263","url":null,"abstract":"<p><p>A second allogeneic (allo-)hematopoietic stem cell transplantation (HSCT2) is a potential curative option for pediatric patients with acute lymphoblastic leukemia (ALL) following relapse after first allogeneic transplantation (HSCT1), but its efficacy is limited by high relapse rates and transplant-related toxicity in highly pretreated patients. This retrospective multicenter study analyzed 75 pediatric patients enrolled in the PRSZT registry who underwent HSCT2 between 2011 and 2020. Indications for HSCT2 were relapse in 55 patients (73%) and graft failure in 16 patients (21%). In 4 patients (6%), the indication could not be clearly determined due to incomplete documentation. At 2 and 5 years, overall survival (OS) was 48% and 40%, while relapse-free survival (RFS) was 35.2% and 29.9%. Relapse was the leading cause of mortality, occurring in 36% of patients post-HSCT2, with a median time to relapse of 8.2 months. Univariate analysis identified a longer duration of remission after HSCT1 (>6 months) as a predictor of improved OS (p = 0.013) and chronic graft-versus-host disease (cGVHD) after HSCT2 as beneficial for OS (p = 0.034) and RFS (p = 0.024). In multivariate analysis, older age (>10 years) (HR: 2.778, p < 0.01) and precursor T-cell ALL (HR: 6.62, p = 0.001) were associated with inferior OS, as well as with inferior RFS (HR: 2.2, p = 0.03; HR: 3.89, p < 0.005), while male sex (HR: 0.354, p = 0.02) and ATG administration (HR: 0.35, p = 0.019) predicted improved OS. Despite high relapse rates, HSCT2 remains a potentially curative treatment in a subgroup of patients. These findings highlight the need for better patient selection and the integration of novel immunotherapeutic strategies to further improve outcomes.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70263"},"PeriodicalIF":2.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147486903","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":"Poorly Differentiated Signet-Ring Cell Gastroesophageal Carcinoma in a Child With No Detectable Genetic Predisposition: Report of a Rare and Highly Aggressive Tumor.","authors":"Mohamad Bakkar, Madhvi Rajpurkar, Jeffrey W Taub, Scott Langenburg, Anthony F Shields, Ali G Saad, Avanti Gupte","doi":"10.1002/1545-5017.70277","DOIUrl":"10.1002/1545-5017.70277","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70277"},"PeriodicalIF":2.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147521529","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":"Patient Activation in Childhood, Adolescent, and Young Adult Cancer Survivors: Current Insights and Implications for Survivorship Care-A Systematic Review From the e-QuoL Project.","authors":"Charlotte Demoor-Goldschmidt, Anne Maas, Gisela Michel, Amandine Bertrand, Kristen E T Thornton, Martine Bellanger, Anna-Liesa Filbert, Desiree Grabow, Monica Muraca, Katharina Roser, Sara Oberti, Jelena Roganovic, Hanne Cathrine Lie","doi":"10.1002/1545-5017.70174","DOIUrl":"10.1002/1545-5017.70174","url":null,"abstract":"<p><strong>Purpose: </strong>Patient activation-encompassing knowledge, confidence, and skills in managing individual's health-is a cornerstone of person-centered care. However, its significance among childhood, adolescent, and young adult cancer survivors (CAYACS) remains unexplored. This article examines the application of the 13-item Patient Activation Measure (PAM-13) in CAYACS, highlights factors influencing activation levels, and draws insights from adult oncology.</p><p><strong>Methods: </strong>A systematic review was conducted to identify studies assessing patient activation using PAM-13 in CAYACS populations.</p><p><strong>Results: </strong>Only two relevant studies were identified. Reduced activation levels were associated with psychological distress, fatigue, neurocognitive impairment, and complex late effects. Activation levels fluctuated over time, with lower levels during active treatment and higher levels during disease-free survivorship. Evidence from adult oncology suggests that structured education, psychosocial support, and hybrid care models (e.g., Oncokompas and SMARTCare) may enhance health ownership. However, limitations, such as PAM-13 ceiling effects and potential psychometric variability in oncology populations, have been reported.</p><p><strong>Conclusions: </strong>Activation-based approaches hold promise for improving self-management in CAYACS. Future interventions should be developmentally tailored, acknowledge caregiver roles, and address transition-related challenges and disparities in healthcare access. Recognizing patient engagement as a core outcome in survivorship research may strengthen personalized care and support the development of scalable, evidence-based interventions.</p><p><strong>Implications for cancer survivors: </strong>Despite some measurement biases, activation assessment in CAYACS facilitates identification of vulnerable survivors, enables tailored interventions, and fosters empowerment and self-management to improve personalized survivorship care and long-term outcomes.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70174"},"PeriodicalIF":2.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147521567","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":"When Neuromuscular Weakness Is Not Chemotherapy‑Related: Spinal Muscular Atrophy Type II Diagnosed After Acute Lymphoblastic Leukemia Onset in a 1-Year-Old Child: A Case Report and Review of the Literature.","authors":"Francesca Romani, Francesca Cattaneo, Emanuele Villa, Giacomo Gotti, Antonella Colombini, Claudia Dosi, Cinzia Peruzzi, Francesca Lanfranconi, Adriana Balduzzi, Carmelo Rizzari, Laura Rachele Bettini","doi":"10.1002/1545-5017.70278","DOIUrl":"10.1002/1545-5017.70278","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70278"},"PeriodicalIF":2.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147521576","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":"Dermatofibrosarcoma Protuberans in Infancy: Targeted Therapy Facilitating Complete Resection.","authors":"Aditi Tulsiyan, Anuj Singh, Neel Aggerwal, Devajit Nath, Nita Radhakrishnan","doi":"10.1002/1545-5017.70281","DOIUrl":"10.1002/1545-5017.70281","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70281"},"PeriodicalIF":2.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147521579","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":"MYCN Amplification in RB1-Inactivated Retinoblastoma: Association With High-Risk Features.","authors":"Kyriaki Papaioannou, Regina Kubica, Karen Fischhuber, Jasmin Beygo, Madlen Reschke, Petra Ritter-Sovinz, Eva Biewald, Petra Ketteler, Dietmar Lohmann, Deniz Kanber","doi":"10.1002/1545-5017.70297","DOIUrl":"10.1002/1545-5017.70297","url":null,"abstract":"<p><strong>Background: </strong>MYCN amplification occurs in a subset of retinoblastoma cases, both with and without RB1 inactivation. It has been suggested that retinoblastomas with MYCN amplification represent a distinct entity with more aggressive clinical behavior.</p><p><strong>Methods: </strong>We examined the incidence of MYCN gain/amplification and RB1 inactivation in 192 unilateral retinoblastoma samples from children enucleated between 2011 and 2018 at the German reference center. MYCN copy number was assessed using quantitative PCR and confirmed by single nucleotide polymorphism microarray analysis. Clinical characteristics, RB1 mutation status, and histopathological features were compared between MYCN-amplified and nonamplified retinoblastomas.</p><p><strong>Results: </strong>MYCN gain/amplification was found in 10 of 139 retinoblastomas included in the final analysis (7.2%). All 10 tumors exhibited alterations in at least one RB1 allele (RB1^-/- or RB1^+/-). The RB1 mutation spectrum and overall genomic copy number changes were similar between MYCN-amplified and MYCN-nonamplified retinoblastomas. Age at diagnosis did not differ significantly between the two groups (p = 0.21); however, secondary glaucoma, massive choroidal, and scleral invasion occurred more often in MYCN-amplified retinoblastomas (p = 0.038, p = 0.03, and p = 0.04, respectively). No cases of extraocular retinoblastoma or distant metastasis were observed during a median follow-up of 50 months.</p><p><strong>Conclusion: </strong>MYCN gain/amplification was identified in 7.2% of enucleated unilateral retinoblastomas, all of which showed RB1 inactivation. MYCN amplification was associated with more advanced disease and more aggressive clinical and histopathological features.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70297"},"PeriodicalIF":2.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147593721","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":"Clinical Characteristics and Specific Management Considerations in Children With Cystic Fibrosis and Childhood Cancer: Case Series and Review of the Literature.","authors":"Jaques van Heerden, Bénédicte Brichard, Dries Ruttens, Stijn Verhulst, Katleen Balliauw, Nicolette Moes, Hilde Dotremont, Anne Uyttebroeck, Joris Verlooy, Toon Van Genechten, Liesbeth Vanheeswijck, Kim Van Hoorenbeeck, Machiel van den Akker","doi":"10.1002/1545-5017.70295","DOIUrl":"10.1002/1545-5017.70295","url":null,"abstract":"<p><strong>Background: </strong>Cystic fibrosis (CF) is an autosomal recessive inherited chronic disorder that predisposes adults to gastrointestinal malignancies. The latter has not been observed in children, perhaps due to the limited documentation of the copresentation of childhood cancer in CF. The following review will provide a comprehensive overview of the relevant literature on this subject.</p><p><strong>Methodology: </strong>The objective of this study was to identify cases in Belgium over a 25-year period and to review the literature for similar cases. Our secondary objective was to identify areas of attention during oncological management based on the clinical cases and extant literature.</p><p><strong>Results: </strong>Four cases were diagnosed in Belgium and a further 10 cases were identified in the literature. These fourteen cases exhibited a 1:1 male-to-female ratio. Four cases were hematological malignancies, and 10 cases were solid tumors, predominantly neuroblastomas and osteosarcomas. No specific age group predominated at the time of diagnosis of the malignancy. As the literature indicated that organs were at risk in all systems, at increased risk for treatment toxicities, and a need for increased nutritional and antimicrobial support. The threshold for intervention with supportive care should be substantially lower than in non-CF patients. These patients are predisposed to an increased risk for premature morbidity and mortality.</p><p><strong>Conclusion: </strong>Short and long-term CF-specific survivorship care plans should be developed based on both disease entities in anticipation of compounded risks for early cardio-respiratory disease and an increased awareness of secondary cancer screening. Research into the copresentation of these diseases should be promoted.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70295"},"PeriodicalIF":2.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147623488","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":"Nivolumab-Associated Autoimmune Encephalopathy in a Child With Multiply Relapsed Anaplastic Ependymoma.","authors":"Çiğdem Bulut, Eray Akay, Özge Dedeoğlu, İkbal Ok Bozkaya, Neriman Sarı","doi":"10.1002/1545-5017.70262","DOIUrl":"10.1002/1545-5017.70262","url":null,"abstract":"","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e70262"},"PeriodicalIF":2.3,"publicationDate":"2026-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147474782","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}