Pediatric Hematology Oncology Journal最新文献

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A rare case report of hereditary hyperferritinemia cataract syndrome 遗传性高铁蛋白血症白内障综合征1例报告
Pediatric Hematology Oncology Journal Pub Date : 2025-03-01 DOI: 10.1016/j.phoj.2024.11.107
Ariadni Neofytou, Anthie Damianaki, Lydia Kossiva
{"title":"A rare case report of hereditary hyperferritinemia cataract syndrome","authors":"Ariadni Neofytou,&nbsp;Anthie Damianaki,&nbsp;Lydia Kossiva","doi":"10.1016/j.phoj.2024.11.107","DOIUrl":"10.1016/j.phoj.2024.11.107","url":null,"abstract":"<div><h3>Background</h3><div>Hereditary hyperferritinemia cataract syndrome (HHCS) is a rare genetic disease caused by a mutation in the ferritin light chain gene (<em>FTL</em> gene). It is characterized by hyperferritinemia without tissue iron overload and bilateral early-onset cataracts.</div></div><div><h3>Case report</h3><div>An otherwise healthy 13-year-old girl was evaluated for persistent hyperferritinemia (1437 ng/ml). Her medical history and clinical examination were normal. The transferrin saturation was normal (28.5 %, normal range: 20–40 %), and secondary causes of hyperferritinemia were excluded. An ophthalmological assessment revealed mild opacity of both lenses. Targeted molecular testing revealed a c.-161C &gt; G mutation of the FTL gene in heterozygosity, confirming the diagnosis of HHCS. Considering the patient's negative family history, this may be a <em>de novo</em> mutation.</div></div><div><h3>Conclusion</h3><div>In patients with early onset cataracts, ferritin levels should be checked and vice versa. An ophthalmological evaluation should be done for unexplained hyperferritinemia. Timely diagnosis prevents unnecessary tests and inappropriate therapeutic intervention.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Pages 17-19"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143683015","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Outcome and recurrence patterns of low-risk gonadal germ cell tumors in children and adolescents 儿童和青少年低风险性腺生殖细胞肿瘤的预后和复发模式
Pediatric Hematology Oncology Journal Pub Date : 2025-03-01 DOI: 10.1016/j.phoj.2025.02.002
Tiphaine Courtel , Sylvie Chabaud , Daniel Orbach , Hélène Sudour-Bonnange , Cecile Verité , Angelique Rome , Cecile Dumesnil , Brice Fresneau , Estelle Thebaud , Marleen Renard , Aissatou Barry , Frederic Hameury , Frederique Dijoud , Cecile Faure Conter
{"title":"Outcome and recurrence patterns of low-risk gonadal germ cell tumors in children and adolescents","authors":"Tiphaine Courtel ,&nbsp;Sylvie Chabaud ,&nbsp;Daniel Orbach ,&nbsp;Hélène Sudour-Bonnange ,&nbsp;Cecile Verité ,&nbsp;Angelique Rome ,&nbsp;Cecile Dumesnil ,&nbsp;Brice Fresneau ,&nbsp;Estelle Thebaud ,&nbsp;Marleen Renard ,&nbsp;Aissatou Barry ,&nbsp;Frederic Hameury ,&nbsp;Frederique Dijoud ,&nbsp;Cecile Faure Conter","doi":"10.1016/j.phoj.2025.02.002","DOIUrl":"10.1016/j.phoj.2025.02.002","url":null,"abstract":"<div><div>Pediatric low-risk gonadal germ cell tumors (LR-GCTs) are typically managed with close surveillance following surgery alone. However, the patterns of relapse and prognostic factors for relapse remain insufficiently understood. We reviewed 72 patients under 19 years old diagnosed with low-risk ovarian (OGCT) or testicular (TGCT) GCT between May 27, 2014, and December 31, 2022, from the TGM13-observatory (EudraCT 2013-004039-60). Among 30 OGCT (median age 13 years), 15 were dysgerminomas and 15 were non-dysgerminomatous OGCT (NDOGCT), with 10 patients having incomplete abdominal staging. Eleven relapses were observed, nine of which were locoregional. The three-year progression-free survival (PFS) was 73 % (95 % CI: 44–89) for dysgerminomas and 53 % (95 % CI: 26–74) for NDOGCT (p = 0.22). Among the 42 testicular tumors (median age 3 years), all but one were NSGCTs. Ten relapses occurred, resulting in one death. The three-year PFS for testicular GCTs was 76 % (95 % CI:59–86). Relapse patterns differed by age: in toddlers, relapses predominantly occurred in the lungs, while in adolescents, relapses were primarily in the para-aortic lymph nodes. The median time to relapse was six months. No significant prognostic factors for relapse were identified, including older age (&gt;11 years), incomplete staging for OGCT, and the presence of lymphovascular invasion or embryonal carcinoma for TGCT. Survival after relapse remains excellent, and further research with a larger sample of children is needed to better identify risk factors for relapse in pediatric LR-GCTs.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Pages 51-56"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143869655","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Corrigendum to “Peg-asparaginase associated toxicities in children with acute lymphoblastic leukemia: A single-center cross-sectional study” [J Pediatr Hematol Oncol 9 (1) (2024) 54–62 ISSN 2468-1245https://doi.org/10.1016/j.phoj.2024.03.001] “peg -天冬酰胺酶在儿童急性淋巴细胞白血病中的相关毒性:一项单中心横切性研究”[J] .儿科血液学杂志,9 (1)(2024)54-62 ISSN 2468-1245https://doi.org/10.1016/j.phoj.2024.03.001]
Pediatric Hematology Oncology Journal Pub Date : 2025-03-01 DOI: 10.1016/j.phoj.2024.12.001
Sameh Awwad , Rawan AbuAlnasr , Fahad AlManjomi , Murtada Al Sultan , Jude Howaidi , Mohammed Almotairi , Issam AlFayyad
{"title":"Corrigendum to “Peg-asparaginase associated toxicities in children with acute lymphoblastic leukemia: A single-center cross-sectional study” [J Pediatr Hematol Oncol 9 (1) (2024) 54–62 ISSN 2468-1245https://doi.org/10.1016/j.phoj.2024.03.001]","authors":"Sameh Awwad ,&nbsp;Rawan AbuAlnasr ,&nbsp;Fahad AlManjomi ,&nbsp;Murtada Al Sultan ,&nbsp;Jude Howaidi ,&nbsp;Mohammed Almotairi ,&nbsp;Issam AlFayyad","doi":"10.1016/j.phoj.2024.12.001","DOIUrl":"10.1016/j.phoj.2024.12.001","url":null,"abstract":"","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Page 41"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143704654","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Editorial Board Information 编委会信息
Pediatric Hematology Oncology Journal Pub Date : 2025-03-01 DOI: 10.1016/S2468-1245(25)00012-9
{"title":"Editorial Board Information","authors":"","doi":"10.1016/S2468-1245(25)00012-9","DOIUrl":"10.1016/S2468-1245(25)00012-9","url":null,"abstract":"","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Page ii"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143869654","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Pediatric mature B-cell non-hodgkin lymphoma in India: A retrospective multicenter pooled analysis of treatment approaches and outcomes 印度儿童成熟b细胞非霍奇金淋巴瘤:治疗方法和结果的回顾性多中心汇总分析
Pediatric Hematology Oncology Journal Pub Date : 2025-03-01 DOI: 10.1016/j.phoj.2024.11.104
Nirmalya Roy Moulik , Sameer Bakhshi , Shripad Banavali , Venkatraman Radhakrishnan , Amita Trehan , Anshul Gupta , Niharendu Ghara , Rachna Seth , Ramandeep Singh Arora
{"title":"Pediatric mature B-cell non-hodgkin lymphoma in India: A retrospective multicenter pooled analysis of treatment approaches and outcomes","authors":"Nirmalya Roy Moulik ,&nbsp;Sameer Bakhshi ,&nbsp;Shripad Banavali ,&nbsp;Venkatraman Radhakrishnan ,&nbsp;Amita Trehan ,&nbsp;Anshul Gupta ,&nbsp;Niharendu Ghara ,&nbsp;Rachna Seth ,&nbsp;Ramandeep Singh Arora","doi":"10.1016/j.phoj.2024.11.104","DOIUrl":"10.1016/j.phoj.2024.11.104","url":null,"abstract":"<div><h3>Background</h3><div>Published data on outcomes of pediatric mature B-cell non-Hodgkin lymphoma (B-NHL) from India is limited and difficult to interpret due to small sample size and non-uniform treatment protocols. This study aims to do a pooled analysis of published patient data from multiple centers across India to provide a clearer understanding of survival rates and treatment-related toxicities with respect to the treatment protocols in this population.</div></div><div><h3>Methods</h3><div>A pooled analysis was conducted of patient data from 505 children with mature B-NHL, including Burkitt lymphoma (n = 395), diffuse large B-cell lymphoma (DLBCL, n = 52), and other subtypes (n = 58), treated from 2000 to 2022 at seven major cancer centers in India. Outcomes assessed were grade 3/4 toxicities, toxic deaths, relapse/progression, and survival rates.</div></div><div><h3>Results</h3><div>Most patients (401/505) presented with advanced disease; bone marrow and CNS involvement were observed in 13.9 % and 6.9 % of cases, respectively. Treatment protocols primarily included LMB (n = 208), BFM (n = 191), and MCP (n = 61). Grade 3/4 toxicities were reported in 79.2 % of patients, with higher rates observed with LMB protocol (92.1 %) compared to BFM (70.8 %) and MCP (70.1 %) (p &lt; 0.001). Toxic death rates were similar across protocols. Overall survival (OS) and event-free survival (EFS) at a median follow-up of 17 months were 69.4 ± 2.2 % and 64.9 ± 2.2 %, respectively, with no significant differences in relapse/progression rates or stage-specific OS between protocols (p = 0.28 and 0.51).</div></div><div><h3>Conclusions</h3><div>This pooled analysis shows that although treatment-related toxicities differ by protocol, overall survival outcomes were similar across the LMB, BFM, and MCP regimens, despite being much lower than those reported from high income countries. Uniform standardized protocols may further improve outcomes for pediatric B-NHL in India.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Pages 33-40"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143697553","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Ewing sarcoma of the hands and feet: Outcome and prognostic factors of a rare subsite in a low-middle income country 手和脚的尤文氏肉瘤:在中低收入国家的一种罕见亚位点的结局和预后因素
Pediatric Hematology Oncology Journal Pub Date : 2025-03-01 DOI: 10.1016/j.phoj.2025.02.004
Shuvadeep Ganguly , Archana Sasi , Chitrakshi Nagpal , Bivas Biswas , Sandeep Agarwala , Deepam Pushpam , Ahitagni Biswas , Venkatesan Sampath Kumar , Love Kapoor , Shah Alam Khan , Vishesh Jain , Sameer Bakhshi
{"title":"Ewing sarcoma of the hands and feet: Outcome and prognostic factors of a rare subsite in a low-middle income country","authors":"Shuvadeep Ganguly ,&nbsp;Archana Sasi ,&nbsp;Chitrakshi Nagpal ,&nbsp;Bivas Biswas ,&nbsp;Sandeep Agarwala ,&nbsp;Deepam Pushpam ,&nbsp;Ahitagni Biswas ,&nbsp;Venkatesan Sampath Kumar ,&nbsp;Love Kapoor ,&nbsp;Shah Alam Khan ,&nbsp;Vishesh Jain ,&nbsp;Sameer Bakhshi","doi":"10.1016/j.phoj.2025.02.004","DOIUrl":"10.1016/j.phoj.2025.02.004","url":null,"abstract":"<div><h3>Background</h3><div>The small bones of the hand and feet represent a rare site of Ewing sarcoma (ES) origin. This study presents a real-world dataset describing the clinical presentation, survival outcomes, and their determinants in this subsite.</div></div><div><h3>Methods</h3><div>This is a single-institutional retrospective study of patients with ES originating from the hands/feet (ES-HF), treated between 2003 and 2018. Clinical/demographic details and survival outcomes were retrieved from medical records. Descriptive statistics were used to summarize baseline characteristics. Prognostic factors for event-free survival (EFS) and overall survival (OS) were identified by Cox regression. The clinical features and outcomes were compared between ES-HF and ES-others (ES from other sites) in the cohort.</div></div><div><h3>Results</h3><div>Of 859 ES cases, 28 (3.2 %) patients had ES-HF including four ES-hands (0.5 %) and 24 ES-feet (2.8 %). The calcaneum was the most common site [11 of 28 cases; 39.3 %]. More than half of the patients (53.6 %) had metastatic disease at presentation. In comparison with ES-others, ES-HF had longer median symptom duration [12 versus 4 months; p &lt; 0.001] and smaller tumor diameter [5.5 versus 9 cm; p &lt; 0.001]. The median EFS and OS of the cohort were 30.5 and 39.13 months respectively. Only local therapy receipt was associated with improved EFS (multivariable HR:0.013; 95%CI:0.001–0.158; p &lt; 0.001) and OS (multivariable HR:0.028; 95 % CI:0.003–0.272; p = 0.002). Patients receiving radiotherapy alone had inferior OS compared to those receiving surgery alone. (HR: 9.22; 95 % CI: 1.12–76.31; p = 0.039)</div></div><div><h3>Conclusion</h3><div>ES-HF is a rare ES subsite. Although indolent, metastases are common at presentation. Meticulous local control can improve survival in both localized and metastatic disease for this subsite.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Pages 24-32"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143683014","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Cefiderocol for severe bloodstream infection by multidrug-resistant Klebsiella pneumoniae in a pediatric patient with acute myeloid leukemia 头孢地罗治疗急性髓性白血病患儿多重耐药肺炎克雷伯菌严重血流感染
Pediatric Hematology Oncology Journal Pub Date : 2025-03-01 DOI: 10.1016/j.phoj.2025.02.003
Francesco De Leonardis , Francesca Marasciulo , Roberta Koronica , Mariachiara Servedio , Enza Pentassuglia , Vittorio Greco Miani , Lidia Dalfino , Stefania Stolfa , Nicola Santoro
{"title":"Cefiderocol for severe bloodstream infection by multidrug-resistant Klebsiella pneumoniae in a pediatric patient with acute myeloid leukemia","authors":"Francesco De Leonardis ,&nbsp;Francesca Marasciulo ,&nbsp;Roberta Koronica ,&nbsp;Mariachiara Servedio ,&nbsp;Enza Pentassuglia ,&nbsp;Vittorio Greco Miani ,&nbsp;Lidia Dalfino ,&nbsp;Stefania Stolfa ,&nbsp;Nicola Santoro","doi":"10.1016/j.phoj.2025.02.003","DOIUrl":"10.1016/j.phoj.2025.02.003","url":null,"abstract":"","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Pages 57-58"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143869653","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Challenges faced in screening for thalassemia and other hemoglobinopathies in a North Indian population 北印度人口地中海贫血和其他血红蛋白病筛查面临的挑战
Pediatric Hematology Oncology Journal Pub Date : 2025-03-01 DOI: 10.1016/j.phoj.2025.02.005
Manisha Kumar, Vani Kargwal, Rajeev Goel, Ekta Debnath, Seema Malhotra, Kirti Balyan, Mahrukh Zaidi, Reena Yadav
{"title":"Challenges faced in screening for thalassemia and other hemoglobinopathies in a North Indian population","authors":"Manisha Kumar,&nbsp;Vani Kargwal,&nbsp;Rajeev Goel,&nbsp;Ekta Debnath,&nbsp;Seema Malhotra,&nbsp;Kirti Balyan,&nbsp;Mahrukh Zaidi,&nbsp;Reena Yadav","doi":"10.1016/j.phoj.2025.02.005","DOIUrl":"10.1016/j.phoj.2025.02.005","url":null,"abstract":"<div><h3>Background</h3><div>Hemoglobinopathies are a common group of inherited disorders contributing significantly to the global healthcare burden, particularly in low and middle-income countries (LMICs) such as India.</div></div><div><h3>Objective</h3><div>This prospective cross-sectional study aimed to investigate the epidemiological, clinical, and genetic aspects of hemoglobinopathies in the antenatal population attending a tertiary care hospital in Delhi.</div></div><div><h3>Material and method</h3><div>A total of 7077 antenatal women were investigated over four years, revealing an overall prevalence of Beta Thalassemia Trait (BTT) of 4.55%. The screening algorithm involved a complete blood count (CBC) and High Performance Liquid Chromatography (HPLC) for all the women, followed by mutation testing using Multiplex amplification refractory mutation system - polymerase chain reaction (ARMS PCR) for four common mutations: IVS1-5 (G &gt; C), CD41/42 -TTCT, CD8/9 +G, and Del 619 bp. Multiplex ligation dependent probe amplification assay (MLPA), ARMS PCR and Sanger sequencing were used to identify other mutations. In cases where abnormal High Peformance Liquid Chromatography (HPLC) results were found, further testing of the husband was conducted to guide counselling and decisions regarding prenatal testing.</div></div><div><h3>Results</h3><div>IVS1-5 (G &gt; C) was identified as the most common mutation. Hemoglobin E (HbE) with CD26 (G &gt; A) exhibited the highest hemoglobin and HbA2 values amongst all mutations screened for. The study underscores the challenges of using RBC indices to diagnose BTT, due to marked overlap with Nutritional Deficiency Anemia (NDA). Similar issues were observed with HbD and HbE due to their relatively high MCV and MCH values. An algorithm for antenatal screening and diagnosis for LMIC is proposed but, given these caveats, its value remains to be proven.</div></div><div><h3>Conclusion</h3><div>The study provides data on the relative distribution of hemoglobinopathy mutations in the antenatal population in North India, and revealed the problems of detecting BTT or hemoglobinopathy in this population with a CBC due to the widespread prevalence of NDA. It emphasized the challenges of targeted screening strategies and genetic counselling to effectively reduce the prevalence of thalassemia in North India.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Pages 59-65"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143869651","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Current management of pulmonary relapse in Ewing sarcoma: A report from the Pediatric Surgical Oncology Research Collaborative 目前尤文氏肉瘤肺部复发的处理:一份来自儿科外科肿瘤研究协进会的报告
Pediatric Hematology Oncology Journal Pub Date : 2025-03-01 DOI: 10.1016/j.phoj.2024.11.105
Audra J. Reiter , Lynn Huang , Jennifer H. Aldrink , Brian T. Craig , Andrew M. Davidoff , Lindsay J. Talbot , Jordan Coggins , Jasmine Smith , Katherine C. Bergus , Taleen A. MacArthur , Stephanie F. Polites , Roshni Dasgupta , Chloe Boehmer , Joseph Brungardt , Marcus M. Malek , Hannah N. Rinehardt , Zachary J. Kastenberg , Cameron M. Arkin , Antoine Gourmel , Nelson Piche , Timothy B. Lautz
{"title":"Current management of pulmonary relapse in Ewing sarcoma: A report from the Pediatric Surgical Oncology Research Collaborative","authors":"Audra J. Reiter ,&nbsp;Lynn Huang ,&nbsp;Jennifer H. Aldrink ,&nbsp;Brian T. Craig ,&nbsp;Andrew M. Davidoff ,&nbsp;Lindsay J. Talbot ,&nbsp;Jordan Coggins ,&nbsp;Jasmine Smith ,&nbsp;Katherine C. Bergus ,&nbsp;Taleen A. MacArthur ,&nbsp;Stephanie F. Polites ,&nbsp;Roshni Dasgupta ,&nbsp;Chloe Boehmer ,&nbsp;Joseph Brungardt ,&nbsp;Marcus M. Malek ,&nbsp;Hannah N. Rinehardt ,&nbsp;Zachary J. Kastenberg ,&nbsp;Cameron M. Arkin ,&nbsp;Antoine Gourmel ,&nbsp;Nelson Piche ,&nbsp;Timothy B. Lautz","doi":"10.1016/j.phoj.2024.11.105","DOIUrl":"10.1016/j.phoj.2024.11.105","url":null,"abstract":"<div><h3>Background</h3><div>Relapse occurs in 30–40 % of patients with localized Ewing sarcoma (EWS). Our objective was to describe the current management and outcomes of patients with initially localized EWS who experience first pulmonary relapse.</div></div><div><h3>Methods</h3><div>This multi-center retrospective cohort study included patients ≤22 years old with initially localized EWS treated from 2007 to 2020 at 19 Pediatric Surgical Oncology Research Collaborative institutions, who developed pulmonary relapse. Kaplan-Meier analysis was performed.</div></div><div><h3>Results</h3><div>Thirty-three patients with initially localized EWS developed pulmonary relapse at a median age of 17 (IQR 14; 20) years. Eleven (33 %) patients also had extra-pulmonary metastases (EPM) at relapse. Among the 22 (67 %) patients with pulmonary-only relapse, 10 (45 %) had solitary pulmonary nodules. Pulmonary metastasectomy was performed in 8/10 (80 %) patients with solitary pulmonary-only metastases, 5/12 (42 %) patients with multiple pulmonary-only metastases, and 2/11 (18 %) patients who also had EPM. Whole lung irradiation was administered in 7/10 (70 %) with solitary pulmonary-only metastases, 7/12 (58 %) with multiple pulmonary-only metastases, and 2/11 (18 %) with EPM. Rates of further pulmonary relapse/progression were similar between groups (p = 0.97). In Kaplan-Meier analysis, 3-year overall survival was 73 % with solitary pulmonary-only metastases, 40 % with multiple pulmonary-only metastases, and 23 % with EPM (p = 0.097).</div></div><div><h3>Conclusions</h3><div>While survival for patients with relapsed EWS is poor, the subset of patients with solitary relapse confined to the lung are often good candidates for pulmonary metastasectomy and have a non-statistically significant trend towards improved survival outcomes.</div></div>","PeriodicalId":101004,"journal":{"name":"Pediatric Hematology Oncology Journal","volume":"10 1","pages":"Pages 20-23"},"PeriodicalIF":0.0,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143683013","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Prevalence of vitamin D deficiency in pediatric patients with sickle cell disease: A systematic review 儿童镰状细胞病患者维生素D缺乏症的患病率:一项系统综述
Pediatric Hematology Oncology Journal Pub Date : 2025-03-01 DOI: 10.1016/j.phoj.2025.02.001
Thiago de Souza Vilela , Mauro Fisberg , Gerson Ferrari , Josefina Aparecida Pellegrini Braga
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