Journal of Pediatric Hematology/Oncology最新文献_第8页

Successful Unmanipulated Haploidentical Hematopoietic Stem Cell Transplant With Post-transplant Cyclophosphamide in a Child With Down Syndrome and Myelodysplastic Syndrome. 移植后环磷酰胺治疗唐氏综合征和骨髓增生异常综合征患儿的单倍体造血干细胞移植成功

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-07-01 Epub Date: 2025-05-12 DOI: 10.1097/MPH.0000000000003051

Chane Choed-Amphai, Yigal Dror, Michaela Cada, Tal Schechter, Joerg Krueger, Muhammad Ali, Yogi Chopra

引用次数: 0

Exploring Maintenance Therapy in Pediatric Embryonal Tumor With Multilayered Rosettes. 探讨儿童胚胎肿瘤多层玫瑰结的维持治疗。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-07-01 Epub Date: 2025-04-07 DOI: 10.1097/MPH.0000000000003033

Mohamed M Ahmed, Fernando Carceller, Leslie R Bridges, Conor Mallucci, Navneet Singh, Sucheta Vaidya

{"title":"Exploring Maintenance Therapy in Pediatric Embryonal Tumor With Multilayered Rosettes.","authors":"Mohamed M Ahmed, Fernando Carceller, Leslie R Bridges, Conor Mallucci, Navneet Singh, Sucheta Vaidya","doi":"10.1097/MPH.0000000000003033","DOIUrl":"10.1097/MPH.0000000000003033","url":null,"abstract":"Embryonal tumors with multilayered rosettes (ETMR) represent a distinct entity characterized by aggressive behavior. Historical retrospective analyses have documented dire overall survival rates ranging from 0% to 14% at 1 year. However, a contemporary report by Khan and colleagues shows overall survival rates reaching 29% at 2 years and 27% at 4 years. We present the case of an 18-month-old girl diagnosed with ETMR, confirmed by chromosome 19 microRNA cluster amplification following initial presentation with focal seizures. The patient underwent a combination of surgical interventions, high-dose chemotherapy with stem cell rescue, and proton therapy, achieving a disease-free status after completing standard treatment. Subsequently, a 12-month maintenance regimen comprising intrathecal topotecan, oral sodium valproate, and oral cis-retinoic acid was administered. The maintenance therapy was well tolerated, with manageable adverse effects. The patient remains progression-free for 32 months postmaintenance therapy (50 months from initial presentation). This study explores the feasibility and safety profile of maintenance therapy in ETMR. Future studies may explore this approach to determine its efficacy in children with ETMR.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"242-245"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143803458","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Ex Vivo Drug Screening: An Emerging Paradigm in the Treatment of Childhood Cancer. 体外药物筛选：儿童癌症治疗的新兴范例。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-07-01 Epub Date: 2025-03-10 DOI: 10.1097/MPH.0000000000003017

Anees Ahmed, Ellen Cox, Louis Lane, Ola Rominiyi, Sarah Danson, Helen E Bryant, Greg Wells, David King

{"title":"Ex Vivo Drug Screening: An Emerging Paradigm in the Treatment of Childhood Cancer.","authors":"Anees Ahmed, Ellen Cox, Louis Lane, Ola Rominiyi, Sarah Danson, Helen E Bryant, Greg Wells, David King","doi":"10.1097/MPH.0000000000003017","DOIUrl":"10.1097/MPH.0000000000003017","url":null,"abstract":"Developing and providing the right therapy for the right patient (or personalized targeted treatments) is key to reducing side-effects and improving survival in childhood cancers. Most efforts aiming to personalize childhood cancer treatment use genomic analysis of malignancies to identify potentially targetable genetic events. But it is becoming clear that not all patients will have an actionable change, and in those that do there is no additional way to determine if treatments will be effective. Ex vivo drug screening is a laboratory technique used to test the effects of various drugs or compounds, on biological tissues or cells that have been removed from an organism. This information is then used to predict which cancer treatments will be most effective based on the therapeutic response in the tissue or cells removed from that individual. Its utility in personalizing treatments in childhood cancer is increasingly recognized. In this review we describe the different methods for ex vivo drug screening and the advantages and disadvantages of each technique. We also present recent evidence that ex vivo screening may have utility in a variety of childhood malignancies including an overview of current clinical trials appraising its use. Finally, we discuss the research questions and hurdles that must be overcome before ex vivo screening can be widely used in pediatric oncology.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e144-e154"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143630576","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Pyrites: A Thigh Mass. 黄铁矿：大腿团块。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-07-01 Epub Date: 2025-04-21 DOI: 10.1097/MPH.0000000000003036

Sam Lyvannak, Has Sotherak, Thy Bunpaov, Bun Sereyleak, Jason Jarzembowski, Bruce Camitta

引用次数: 0

Variability in the Recommendations for Management of Vaso-occlusive Crisis and Acute Chest Syndrome in Sickle Cell Disease: Review of Institutional Algorithms of Pediatric Hospitals Across the United States. 镰状细胞病血管闭塞危象和急性胸综合征管理建议的差异：美国儿科医院机构算法综述

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-07-01 Epub Date: 2025-05-14 DOI: 10.1097/MPH.0000000000003052

Amr Elgehiny, Mariam Z Quraishi, Aravind Yadav, Lakshmi V Srivaths, Olayinka Okeleji, Deborah L Brown, Neethu M Menon

{"title":"Variability in the Recommendations for Management of Vaso-occlusive Crisis and Acute Chest Syndrome in Sickle Cell Disease: Review of Institutional Algorithms of Pediatric Hospitals Across the United States.","authors":"Amr Elgehiny, Mariam Z Quraishi, Aravind Yadav, Lakshmi V Srivaths, Olayinka Okeleji, Deborah L Brown, Neethu M Menon","doi":"10.1097/MPH.0000000000003052","DOIUrl":"10.1097/MPH.0000000000003052","url":null,"abstract":"This study aims to review institutional algorithms for vaso-occlusive crisis (VOC) and acute chest syndrome (ACS) in pediatric sickle cell disease (SCD), comparing them to each other and to national recommendations. Algorithms collected from children's hospitals across the country were compared with recommendations made by the National Heart, Lung, and Blood Institute 2014 and the American Society of Hematology 2020 regarding management of VOC/ACS in SCD. Review of 37 VOC and 17 ACS algorithms from 40 children's hospitals showed that most followed national guidelines for diagnostic evaluation of VOC/ACS. Parenteral opioids and NSAIDs were recommended by all VOC algorithms, with variations in dosing and administration. Intranasal fentanyl was recommended by 31 algorithms. Incentive spirometry was included in 16 of 17 ACS algorithms, but only in 11 of 37 VOC algorithms. Antibiotics were recommended by all ACS algorithms, but 4 used regimens were different from national recommendations. Most ACS algorithms had recommendations regarding transfusion, but with considerable variability. Intravenous fluid management strategies were highly variable, and hypotonic fluids were recommended in 6 VOC and 4 ACS algorithms. Overall, internal algorithms for pediatric SCD showed variability compared with each other and with national guidelines, likely due to the lack of robust evidence supporting specific recommendations.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e155-e160"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144078694","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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 Religious Coping for Burnout in Pediatric Hematology/Oncology. 宗教应对在儿童血液学/肿瘤学职业倦怠中的应用。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-07-01 Epub Date: 2025-04-21 DOI: 10.1097/MPH.0000000000003040

Hüseyin Çaksen

引用次数: 0

In Reply: Time to Resolution of Severe Anemia in Young Children With Iron Deficiency. 答复：解决缺铁儿童严重贫血的时间。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-07-01 Epub Date: 2025-04-21 DOI: 10.1097/MPH.0000000000003030

Hale Ören

引用次数: 0

The Tale of Pediatric Hematopoietic Stem Cell Transplantation in Iran: An Uphill Battle. 伊朗儿童造血干细胞移植的故事：一场艰苦的战斗。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-07-01 Epub Date: 2025-05-08 DOI: 10.1097/MPH.0000000000003050

Maryam Behfar, Pouya Mahdavi Sharif, Amir Ali Hamidieh

{"title":"The Tale of Pediatric Hematopoietic Stem Cell Transplantation in Iran: An Uphill Battle.","authors":"Maryam Behfar, Pouya Mahdavi Sharif, Amir Ali Hamidieh","doi":"10.1097/MPH.0000000000003050","DOIUrl":"10.1097/MPH.0000000000003050","url":null,"abstract":"Hematopoietic stem cell transplantation (HSCT) has revolutionized the treatment of hematologic malignancies, immunodeficiencies, and storage disorders. While this transformative therapy is widely adopted globally, its introduction in Iran faced significant challenges due to geopolitical and economic hardships. In 2007, the first pediatric HSCT department in Iran was established at Tehran University of Medical Sciences. Over the past 18 years, this department has made substantial contributions to the field, including pioneering various HSCT modalities and establishing the first HLA registry in the region, facilitating access to matched unrelated donors. Despite significant challenges, including economic constraints and limited access to advanced therapies, the department and its affiliated research center have persevered in their mission to provide care to pediatric patients and advance in cutting-edge research, including immune cell therapies and gene therapies. With constantly aggravating economic hurdles and geopolitical issues, these milestones could not be achieved without the financial support of health care philanthropies and the sacrifices of dedicated physicians, researchers, and nurses. However, no health system, no matter how resilient, is immune to debacle under perpetuated hurdles, underscoring the need for global collaborations. This letter highlights the journey of pediatric HSCT in Iran and the importance of public advocacy in overcoming obstacles.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"257-259"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144004835","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Effects of Genetic Polymorphisms of WNT Signaling Pathway on the Prognosis of Acute Lymphoblastic Leukemia in Chinese Pediatric Population. WNT信号通路遗传多态性对中国儿童急性淋巴细胞白血病预后的影响

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-07-01 Epub Date: 2025-06-02 DOI: 10.1097/MPH.0000000000003037

Xiaojia Wen, Chao Gao, Weijing Li, Yuanyuan Zhang, Tianlin Xue, Lei Cui, Peijing Qi, Wei Lin, Jun Li, Zhigang Li, Ying Wu, Jiaole Yu, Ruidong Zhang, Huyong Zheng, Shuguang Liu

{"title":"Effects of Genetic Polymorphisms of WNT Signaling Pathway on the Prognosis of Acute Lymphoblastic Leukemia in Chinese Pediatric Population.","authors":"Xiaojia Wen, Chao Gao, Weijing Li, Yuanyuan Zhang, Tianlin Xue, Lei Cui, Peijing Qi, Wei Lin, Jun Li, Zhigang Li, Ying Wu, Jiaole Yu, Ruidong Zhang, Huyong Zheng, Shuguang Liu","doi":"10.1097/MPH.0000000000003037","DOIUrl":"10.1097/MPH.0000000000003037","url":null,"abstract":"The deregulation of WNT signaling has been shown to be important in the development of hematologic malignancies. The genetic variation of key WNT signaling pathway genes may affect the development of leukemia. In the present study, a total of 20 single nucleotide polymorphisms (SNPs) in 6 genes ( CTNNB1, AXIN2, SFRP2, SFRP4, SFRP5 , and DKK3 ) involved in the WNT signaling pathway were selected to investigate the influence of SNPs on the prognosis of 379 Chinese children with acute lymphoblastic leukemia (ALL). Both χ 2 test and Kaplan-Meier survival curve estimation showed that AXIN2 rs7591, rs3923086, and rs11867417 were associated with the risk of relapse with statistical significance. The multifactorial analyses showed that the 3 SNPs of rs7591, rs3923086, and rs11867417 in AXIN2 still had a significant effect on prognosis with statistical significance ( P =0.004, 0.019, and 0.013, respectively). In addition, SFRP4 rs1802073 and rs1802074 were also significantly associated with the risk of relapse after excluding the effect of confounding variables. Taken together, our findings show that polymorphisms in AXIN2 and SFRP4 were independent prognostic predictors for pediatric ALL patients. Further studies are needed to validate these findings and clarify the underlying mechanism.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e168-e176"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144248390","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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

Follow-up Outcomes of Patients Presenting With Ovarian Masses in Childhood: A 15-Year Single-center Experience. 儿童期卵巢肿块患者的随访结果：15年单中心经验。

IF 0.9 4区医学

Journal of Pediatric Hematology/Oncology Pub Date : 2025-07-01 Epub Date: 2025-05-24 DOI: 10.1097/MPH.0000000000003043

Aytül Temuroğlu, Betül B Sevinir

{"title":"Follow-up Outcomes of Patients Presenting With Ovarian Masses in Childhood: A 15-Year Single-center Experience.","authors":"Aytül Temuroğlu, Betül B Sevinir","doi":"10.1097/MPH.0000000000003043","DOIUrl":"10.1097/MPH.0000000000003043","url":null,"abstract":"Objective: Ovarian tumors in childhood are rare. This study aimed to examine the clinical characteristics of pediatric patients presenting with ovarian masses.Materials and methods: A retrospective review of patient data was conducted for patients who presented to our clinic with an ovarian mass between 2009 and 2024.Results: The study included 85 patients treated for ovarian masses in our clinic. The mean age of the patients was 12.2±4.6 (range: 0 to 18) years. The mean size of malignant tumors was 12.1±7.2 cm, and that of benign tumors was 10.74±7 cm ( P =0.43). On the basis of operation type, the mean tumor size in patients who underwent oophorectomy was 12.66±7.2 cm, compared with 8.59±6.15 cm in those with a cystectomy ( P =0.017). Histopathologic examination reported 54.1% (n=46) of tumors as benign, 32.9% (n=31) as malignant, 5.9% (n=5) as borderline, and 3.5% (n=3) as non-neoplastic lesions. Germ cell tumors were the most common, constituting 67.1% (n=57) of cases, followed by epithelial tumors at 22.4% (n=19). Synchronous bilateral ovarian tumors were identified in 4 cases. Chemotherapy was administered to 32.9% (n=28) of the patients. The mean follow-up period was 45 (1 to 204) months. A recurrence was observed in 1 patient, followed up with a diagnosis of immature teratoma. Two patients (2.4%) died, 1 due to acute renal failure and the other due to sepsis.Conclusions: Ovarian tumors in childhood are rare and have high treatment success rates. Further research is needed to improve ovary-preserving surgical approaches and mitigate the side effects of chemotherapy.","PeriodicalId":16693,"journal":{"name":"Journal of Pediatric Hematology/Oncology","volume":" ","pages":"e187-e191"},"PeriodicalIF":0.9,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144159752","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"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