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Mitapivat for Acquired Pyruvate Kinase Deficiency. 治疗获得性丙酮酸激酶缺乏症的 Mitapivat。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-02-01 Epub Date: 2024-11-13 DOI: 10.1002/pbc.31440
Hanny Al-Samkari
{"title":"Mitapivat for Acquired Pyruvate Kinase Deficiency.","authors":"Hanny Al-Samkari","doi":"10.1002/pbc.31440","DOIUrl":"10.1002/pbc.31440","url":null,"abstract":"<p><p>Pyruvate kinase (PK) activation is emerging as a promising treatment modality for numerous congenital hemolytic anemias of diverse pathophysiology, and one agent, mitapivat, is already licensed to treat patients with congenital PK deficiency. However, PK deficiency may also be acquired in the setting of clonal myeloid disorders and other pathologies, where it may result in severe hemolytic anemia and remains without known therapies. This case report describes the novel application of mitapivat therapy in a patient with acquired PK deficiency causing red cell transfusion dependence, liberating the patient from transfusions and resulting in marked improvement in symptoms and quality of life.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31440"},"PeriodicalIF":2.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11666393/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625671","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}
引用次数: 0
Quality of Life Priorities of Childhood Acute Lymphoblastic Leukemia Survivors Enrolled in EORTC Studies, and a Comparison of Instruments. 参加EORTC研究的儿童急性淋巴细胞白血病幸存者的生活质量优先级,以及仪器的比较。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-02-01 Epub Date: 2024-11-30 DOI: 10.1002/pbc.31446
Jammbe Musoro, Charlotte Sleurs, Ali Rowsell, Stefan Suciu, Michal Kicinski, Sofia Chantziara, Corneel Coens, Madeline Pe, Pierre Missotten, Els Vandecruys, Anne Uyttebroeck, Marie-Françoise Dresse, Claire Pluchart, Alina Ferster, Claire Freycon, Jutte Van Der Werff Ten Bosch, Pierre Rohrlich, Yves Benoit, Caroline Piette, Anne-Sophie Darlington
{"title":"Quality of Life Priorities of Childhood Acute Lymphoblastic Leukemia Survivors Enrolled in EORTC Studies, and a Comparison of Instruments.","authors":"Jammbe Musoro, Charlotte Sleurs, Ali Rowsell, Stefan Suciu, Michal Kicinski, Sofia Chantziara, Corneel Coens, Madeline Pe, Pierre Missotten, Els Vandecruys, Anne Uyttebroeck, Marie-Françoise Dresse, Claire Pluchart, Alina Ferster, Claire Freycon, Jutte Van Der Werff Ten Bosch, Pierre Rohrlich, Yves Benoit, Caroline Piette, Anne-Sophie Darlington","doi":"10.1002/pbc.31446","DOIUrl":"10.1002/pbc.31446","url":null,"abstract":"<p><strong>Purpose: </strong>Survivors of childhood cancer can suffer from long-term sequelae or decline in quality of life (QoL), for which careful and standardized selection of outcome measures become more important. This study aims to assess different QoL-related outcomes using three distinct questionnaires in an international study, identify the priorities of childhood ALL survivors via the administered questionnaires, and investigate potential interrelationships among QoL domains across the questionnaires.</p><p><strong>Methods: </strong>Childhood ALL survivors treated according to the EORTC CLG treatment protocols 58741, 58831/2, and 58881 were recruited in Belgium and France and answered self-report QoL questionnaires, including the Short-Form Health Survey 12 (SF12), the Quality-of-Life Systemic Inventory (QLSI), and the Impact of Cancer for Childhood Cancer Survivors (IOC-CS). To explore which scales overlapped or were novel, Pearson correlations were used to explore associations. In addition, based on the QLSI, we checked whether each of the top priorities of childhood ALL survivors were covered by the SF12 or IOC-CS, by mapping their scales quantitatively and qualitatively.</p><p><strong>Results: </strong>QoL data for 186 survivors were provided. Priority areas, as assessed by the QLSI, were vitality, physical abilities, memory, overall physical health, sleep, interaction with friends, love life. Love life was an important source of happiness (for 42%), and for some reported as the domain they were unhappiest in (13%). Quantitative mapping shows moderate correlations between the SF12 scales and IOC-CS scales: life challenges, body and health, thinking and memory, and socializing. Qualitative mapping highlighted additional important domains, specifically family, romantic and friendship relationships, and sleep and memory.</p><p><strong>Conclusions: </strong>Our findings suggest that the measures complement each other, but are less valuable in isolation for ALL survivors. Using a cancer survivorship measure, combined with some additional items covering priorities might provide a more holistic picture.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31446"},"PeriodicalIF":2.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142771216","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
Association Between Perinatal Factors and Childhood Lymphoma-A Pooled Analysis of the ESCALE and ESTELLE Studies (SFCE). 围产期因素与儿童淋巴瘤之间的关系--ESCALE 和 ESTELLE 研究的汇总分析 (SFCE)。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-02-01 Epub Date: 2024-11-23 DOI: 10.1002/pbc.31439
Elissa Msallem, Hélène Pacquement, Laura Olivier, Laurence Brugières, Judith Landman Parker, Nathalie Garnier, Anne Lambilliotte, Laure Faure, Jacqueline Clavel, Audrey Bonaventure
{"title":"Association Between Perinatal Factors and Childhood Lymphoma-A Pooled Analysis of the ESCALE and ESTELLE Studies (SFCE).","authors":"Elissa Msallem, Hélène Pacquement, Laura Olivier, Laurence Brugières, Judith Landman Parker, Nathalie Garnier, Anne Lambilliotte, Laure Faure, Jacqueline Clavel, Audrey Bonaventure","doi":"10.1002/pbc.31439","DOIUrl":"10.1002/pbc.31439","url":null,"abstract":"<p><strong>Context: </strong>There is much interest in the perinatal period in relation to childhood cancer aetiology, with most studies focussing on childhood leukaemia. This work aimed to investigate the associations between pregnancy-related and perinatal factors and childhood lymphoma.</p><p><strong>Methods: </strong>We conducted a pooled analysis of two French nationwide population-based case-control studies. Data on sociodemographic, perinatal and lifestyle factors were collected through maternal interviews. Odds ratios (OR) and 95% confidence intervals (CIs) were computed using adjusted logistic regression models, separately for non-Hodgkin lymphoma (NHL) and Hodgkin lymphoma (HL). Specific analyses also investigated Burkitt NHL and nodular sclerosis HL, two most common histological types in children.</p><p><strong>Results: </strong>We included 305 NHL, 328 HL and 2415 controls in this study. No associations were observed with gestational age, foetal growth indicators, folic acid supplementation, factors related to maternal fertility and reproductive history, or maternal smoking during pregnancy. Maternal coffee consumption during pregnancy was associated with NHL (>2 cups/day, OR = 1.5 [95% CI: 1.1-2.1]), with a dose-response relationship; while maternal alcohol consumption was associated with Burkitt NHL (OR = 1.5 [1.1-2.2]). Paternal smoking during preconception/pregnancy was associated with NHL (OR = 1.4 [1.1-1.8]). Breastfeeding (ever/never) was not significantly associated with NHL and HL, but an inverse log-linear trend was observed with the duration of breastfeeding for NHL (p = 0.04).</p><p><strong>Conclusion: </strong>Maternal coffee and alcohol consumptions during pregnancy and paternal smoking during preconception/pregnancy might increase the risk of childhood NHL. While warranting replication, these findings could help us better understand the aetiology of childhood lymphoma.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31439"},"PeriodicalIF":2.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142695725","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
Effective Use of ALK Inhibitors in EML4::ALK-Positive Lymphatic Malformations. 在 EML4::ALK 阳性淋巴畸形中有效使用 ALK 抑制剂。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-02-01 Epub Date: 2024-11-11 DOI: 10.1002/pbc.31441
Beth Apsel Winger, Christopher F Dowd, Kristin A Shimano, W Patrick Devine, Erin Mathes, Ilona Frieden, Carrie Schaefer, Alok Kothari
{"title":"Effective Use of ALK Inhibitors in EML4::ALK-Positive Lymphatic Malformations.","authors":"Beth Apsel Winger, Christopher F Dowd, Kristin A Shimano, W Patrick Devine, Erin Mathes, Ilona Frieden, Carrie Schaefer, Alok Kothari","doi":"10.1002/pbc.31441","DOIUrl":"10.1002/pbc.31441","url":null,"abstract":"<p><p>Genetically targeted medications are emerging as important therapies for lymphatic malformations (LMs) unresponsive to sirolimus. We describe two patients with EML4::ALK-positive LMs, one with Gorham Stout disease and one with a large genitourinary (GU) LM, who were successfully treated with ALK inhibitors. This report adds ALK inhibitors to the growing toolbox of molecularly targeted therapies for LMs.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31441"},"PeriodicalIF":2.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625646","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 Persistent Alopecia on Quality of Life in Childhood Cancer and Transplant Survivors. 顽固性脱发对儿童癌症和器官移植幸存者生活质量的影响
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-02-01 Epub Date: 2024-11-11 DOI: 10.1002/pbc.31432
Eun Jae Kim, Madeline Hlobik, Laura Berbert, Jill Brace-O'Neill, Lynda Vrooman, Jennifer T Huang
{"title":"Impact of Persistent Alopecia on Quality of Life in Childhood Cancer and Transplant Survivors.","authors":"Eun Jae Kim, Madeline Hlobik, Laura Berbert, Jill Brace-O'Neill, Lynda Vrooman, Jennifer T Huang","doi":"10.1002/pbc.31432","DOIUrl":"10.1002/pbc.31432","url":null,"abstract":"<p><strong>Background: </strong>While alopecia associated with chemotherapy, radiation, or hematopoietic stem-cell transplant (C/R/HSCT) is transient in most children, prior reports indicate nearly one in seven childhood cancer survivors suffer from persistent alopecia after their treatment is completed. The objective of our study was to better characterize the impact of C/R/HSCT-associated persistent alopecia on patient quality of life.</p><p><strong>Procedure: </strong>A cross-sectional cohort study of patients with a history of C/R/HSCT who were seen at Dana Farber Cancer Institute/Boston Children's Hospital Dermatology from August 2023 to February 2024 for any indication was conducted. Patients who completed their C/R/HSCT treatment regimen >6 months prior to visit were invited to fill out a survey on patient experience with persistent alopecia, including a modified Children's Dermatology Life Quality Index (CDLQI). Participants also underwent a full scalp examination.</p><p><strong>Results: </strong>Twenty one out of 47 (44.7%) patients in our cohort self-reported persistent alopecia. For nine additional patients, alopecia was not self-reported but noted by a dermatologist on exam. Median self-reported alopecia severity was 3 (interquartile range [IQR] 2-5.25) on a visual analog scale of 1-10. The most common pattern of alopecia was diffuse thinning. Median CDLQI score was 5 (IQR 2-7) for those with persistent alopecia, indicating a small negative effect of disease on patient quality of life. Fifteen (31.9%) patients report receiving information about persistent alopecia prior to their C/R/HSCT.</p><p><strong>Conclusions: </strong>Nearly half of childhood cancer and transplant survivors evaluated by dermatology suffered from persistent alopecia, which negatively impacted their quality of life. Better counseling on persistent alopecia should be provided to childhood cancer patients.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31432"},"PeriodicalIF":2.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625664","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 Global Prevalence of Iron Deficiency in Collegiate Athletes: A Systematic Review and Meta-Analysis. 大学生运动员铁缺乏症的全球患病率:系统回顾与元分析》。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-02-01 Epub Date: 2024-11-12 DOI: 10.1002/pbc.31415
Christineil Thompson, Darci Block, Zhen Wang, Nathan Foster, Leslie C Hassett, Dana Steien, Paul Galardy, Ahmad Al-Huniti
{"title":"The Global Prevalence of Iron Deficiency in Collegiate Athletes: A Systematic Review and Meta-Analysis.","authors":"Christineil Thompson, Darci Block, Zhen Wang, Nathan Foster, Leslie C Hassett, Dana Steien, Paul Galardy, Ahmad Al-Huniti","doi":"10.1002/pbc.31415","DOIUrl":"10.1002/pbc.31415","url":null,"abstract":"<p><p>Athletes are at high risk of iron deficiency even without anemia. In this systematic review and meta-analysis that included 17,519 athletes in 122 references from 23 countries, participating in 62 unique sports over a 41-year timeframe, approximately 53.9% of student-athletes were found to be hypoferritinemic (ferritin concentration below 50 mcg/L) with 23.0% percent showing absolute iron deficiency based on current definitions (ferritin concentration below 20 mcg/L). These findings strongly suggest that changes in clinical practice guidelines are warranted to enable routine screening for iron deficiency among adolescent and collegiate athletes.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31415"},"PeriodicalIF":2.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625686","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
Use of Palliative Site-Directed Ablation in Pediatric Oncology: A Retrospective Study From the Pediatric Surgical Oncology Research Collaborative. 在儿科肿瘤中使用姑息性定点消融术:儿科肿瘤外科研究合作组织的回顾性研究。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-02-01 Epub Date: 2024-11-24 DOI: 10.1002/pbc.31453
Lindsay J Talbot, Ann Schechter, Suraj Sarvode Mothi, Mecklin Ragan, Jennifer H Aldrink, Joseph Brungardt, Roshni Dasgupta, Timothy B Lautz, Shankar Rajeswaran, Mary E Moya-Mendez, Elisabeth T Tracy, Michael Temple, Andrew M Davidoff
{"title":"Use of Palliative Site-Directed Ablation in Pediatric Oncology: A Retrospective Study From the Pediatric Surgical Oncology Research Collaborative.","authors":"Lindsay J Talbot, Ann Schechter, Suraj Sarvode Mothi, Mecklin Ragan, Jennifer H Aldrink, Joseph Brungardt, Roshni Dasgupta, Timothy B Lautz, Shankar Rajeswaran, Mary E Moya-Mendez, Elisabeth T Tracy, Michael Temple, Andrew M Davidoff","doi":"10.1002/pbc.31453","DOIUrl":"10.1002/pbc.31453","url":null,"abstract":"<p><strong>Background: </strong>Children with end-stage solid tumors would benefit from established nonsurgical options for palliative site-directed local control with the intent of improving quality of life (QOL) and extending survival. We investigated the practice and tolerability of palliative ablation strategies in patients with pediatric solid tumors.</p><p><strong>Methods: </strong>Children and young adults with solid tumors ineligible for surgical site-directed control were treated with palliative ablative strategies between 2010 and 2020 at five institutions participating in the Pediatric Surgical Oncology Research Collaborative. Demographic, histologic, and clinical data were retrospectively evaluated, and assessment of patient reasons for choosing ablative strategies was qualitatively assessed.</p><p><strong>Results: </strong>A total of 69 patients underwent 89 ablative procedures for 132 lesions. More than 10 histologies were represented, the most common of which were Ewing sarcoma (n = 15 patients), osteosarcoma (n = 13 patients), and rhabdomyosarcoma (n = 9 patients). Ablation techniques included cryoablation (n = 9 procedures/16 lesions), radiofrequency ablation (RFA) (n = 4 procedures/5 lesions), microwave ablation (MWA) (n = 5 procedures/12 lesions), and stereotactic body radiotherapy (SBRT) (n = 68 procedures/99 lesions). Grade 3 or higher adverse events (AEs) included pain requiring IV medication in cryoablation (three out of 12 procedures), RFA (one out of four procedures), and MWA (two out of five procedures), bleeding in MWA (two out of five procedures), and skin burns requiring surgical intervention in SBRT (one out of 68 procedures). Patients were generally hospitalized after percutaneous ablation (19 out of 21 cryoablation, RFA, and MWA procedures) but the hospital stay was short (mean/SD 1 ± 4 days). Only four patients who were already hospitalized experienced hospitalization extension due to undergoing an ablative procedure. The primary reason for pursuing ablative measures could be broadly categorized into four groups: treatment of unresectable disease either due to surgical inaccessibility or metastasis (n = 31), symptom relief, either pain or mass effect (n = 25), specific patient QOL concerns unrelated to pain or life prolongation (n = 5), and physiologic instability for otherwise resectable disease (n = 4). All patients undergoing treatment for symptom relief received SBRT.</p><p><strong>Conclusion: </strong>In this cohort of patients undergoing palliative treatment of incurable or unresectable pediatric solid tumors, ablation strategies were well tolerated. Further research is needed to determine patient reported outcomes after ablation strategies are pursued and patient and family satisfaction with the choice for ablative strategies. An understanding of institutional availability and ability to employ these strategies would also be informative.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31453"},"PeriodicalIF":2.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142710688","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
Socioeconomic Factors Associated With Participation in Postmortem Tissue Donation in Pediatric Central Nervous System Tumors. 与参与小儿中枢神经系统肿瘤死后组织捐献有关的社会经济因素。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-02-01 Epub Date: 2024-11-12 DOI: 10.1002/pbc.31417
Judith M Kabat, Lauren K Boland, Melissa Williams, Ian Michelson, Alicia Lenzen, Ashley S Plant-Fox, Nitin Wadhwani, Angela J Waanders
{"title":"Socioeconomic Factors Associated With Participation in Postmortem Tissue Donation in Pediatric Central Nervous System Tumors.","authors":"Judith M Kabat, Lauren K Boland, Melissa Williams, Ian Michelson, Alicia Lenzen, Ashley S Plant-Fox, Nitin Wadhwani, Angela J Waanders","doi":"10.1002/pbc.31417","DOIUrl":"10.1002/pbc.31417","url":null,"abstract":"<p><strong>Background: </strong>The gift of postmortem tissue donation is critical to pediatric central nervous system (CNS) research. As a \"Center of Excellence\" for the Gift from a Child Program, our institution has a well-established postmortem tissue donation program. Our objective was to determine if differences exist between patients who participated in and those that declined research-based autopsy.</p><p><strong>Procedure: </strong>We performed a single-institution retrospective chart review of pediatric patients with CNS malignancies who died from their disease between January 1, 2021 and December 31, 2022. Individual clinical, demographic, and socioeconomic data were assessed. Population-level data were estimated using Zip Code Tabulation Areas. Descriptive statistics were used to compare categorical data.</p><p><strong>Results: </strong>Among the 23 patient families approached during the study time frame, 8/23 (35%) consented to participation. In the consented (C) versus declined (D) group, there was a higher percentage of White, non-Hispanic/Latino patients by self-reported race (C: 88% vs. D: 55%), and no patients who identified as Asian or Hispanic/Latino (C: 0% and 0% vs. D: 13% and 27%). Of all patients approached, two required interpreters (9%), and both families declined participation. The rate of private insurance was higher in the consented group (C: 75% vs. D: 47%) compared to Medicaid as primary insurance in the declined group (C: 13% vs. D: 53%).  CONCLUSIONS: Future research should aim to understand and improve identified disparities to ensure research advancements benefit all children with CNS malignancies. One area we plan to address is improved communication with non-English-speaking families by partnering with our interpreter services.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31417"},"PeriodicalIF":2.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625675","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
Pediatric Neuroendocrine Tumors in Denmark: Incidence, Management, and Outcome From 1995 to 2020. 丹麦小儿神经内分泌肿瘤:1995年至2020年的发病率、管理和结果。
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-02-01 Epub Date: 2024-11-23 DOI: 10.1002/pbc.31420
Mona P Ankerstjerne, Sara Giovannoni, Lene G Christensen, Sören Möller, Pernille Holmager, Ulrich Knigge, Mark B Ellebaek, Mathias Rathe
{"title":"Pediatric Neuroendocrine Tumors in Denmark: Incidence, Management, and Outcome From 1995 to 2020.","authors":"Mona P Ankerstjerne, Sara Giovannoni, Lene G Christensen, Sören Möller, Pernille Holmager, Ulrich Knigge, Mark B Ellebaek, Mathias Rathe","doi":"10.1002/pbc.31420","DOIUrl":"10.1002/pbc.31420","url":null,"abstract":"<p><strong>Background: </strong>Neuroendocrine tumors (NETs), although rare, are considered one of the most common gastrointestinal and bronchopulmonary pediatric neoplasms. We aimed to determine the incidence, tumor characteristics, management, and outcome of NETs and explore the role of genetic predisposition, focusing on low and intermediate grade tumors.</p><p><strong>Methods: </strong>Using the Danish National Pathology Registry, we conducted a nationwide retrospective study including all Danish children aged ≤18 years diagnosed with a pathology-proven NET between 1995 and 2020.</p><p><strong>Results: </strong>We identified 220 patients, with a 1.89:1 female to male ratio. The yearly incidence was 6.84 per 1 million children, with no significant change in incidence throughout the observation period. NETs were located in the appendix (93.2%), the pulmonary system (4.5%), and pancreas (2.3%). One recurrence was noted in the pancreas in a genetically predisposed patient with multiple neuroendocrine neoplasia type 1 (MEN1), resulting in an overall recurrence rate of 0.5% (0% in appendiceal NETs; 0% in bronchopulmonary NETs; 20.0% in pancreatic NETs). No NET-related mortality was registered. Four patients had a known predisposing genetic condition, one appendiceal NET associated with neurofibromatosis type 1, and three pancreatic NETs associated with MEN1. Postsurgical surveillance regimes, choice of tumor markers, and imaging modality varied throughout the study period.</p><p><strong>Conclusions: </strong>We confirmed a stable incidence of pediatric NETs during the study period. The overall recurrence rate was 0.5% and no NET-related mortality was observed. Known genetic predisposition was present in 1.8% of patients. Future guidelines should consider the apparent indolent nature and excellent prognosis of these tumors.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31420"},"PeriodicalIF":2.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142695749","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 Non-Pharmacological Cognitive Interventions on Real-World Daily Function in Children With Cancer: A Systematic Review. 非药物认知干预对癌症儿童实际日常生活功能的影响:系统回顾
IF 2.4 3区 医学
Pediatric Blood & Cancer Pub Date : 2025-02-01 Epub Date: 2024-11-12 DOI: 10.1002/pbc.31429
Allison J L'Hotta, Bailey Martin-Giacalone, Jennifer Zink, Anna Fung, Andrew Myers, Kim Lipsey, Rachelle Brick
{"title":"Impact of Non-Pharmacological Cognitive Interventions on Real-World Daily Function in Children With Cancer: A Systematic Review.","authors":"Allison J L'Hotta, Bailey Martin-Giacalone, Jennifer Zink, Anna Fung, Andrew Myers, Kim Lipsey, Rachelle Brick","doi":"10.1002/pbc.31429","DOIUrl":"10.1002/pbc.31429","url":null,"abstract":"<p><p>Childhood cancer survivors (CCS) frequently experience cognitive challenges, which impact their ability to participate in functional activities. This systematic review examines the effects of nonpharmacological cognitive interventions on functional outcomes (e.g., activities of daily living). We systematically searched eight databases (e.g., PubMed, EMBASE) from 2012 to 2023. Two team members independently screened articles and extracted article, intervention, sample characteristics, and outcome data. We assessed intervention reporting with the Template for Intervention Description and Replication. Fourteen studies met inclusion criteria, representing 12 unique trials. Four of the six trials evaluating Cogmed, a computerized working memory training program, demonstrated functional benefits; small to large effects were observed for academic achievement (Cohen's d = 0.28-0.87) and decreased inattention in daily life (d = -0.36 and -0.98). Other interventions included exergaming, math or reading interventions, occupational therapy, and neurofeedback. Less than half of studies reported on intervention personalization (n = 5), fidelity assessment (n = 4), or cost (n = 1). Study heterogeneity limited our ability to meta-analyze results for functional outcomes. Limited evidence and gaps in quality of intervention reporting are barriers to addressing the cognitive challenges of CCS. Standardizing functional outcome measurement, identifying effective interventions, and improving the quality of intervention reporting could accelerate the translation of intervention research to clinical practice.</p>","PeriodicalId":19822,"journal":{"name":"Pediatric Blood & Cancer","volume":" ","pages":"e31429"},"PeriodicalIF":2.4,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142625652","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}
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