Pediatric Hematology and Oncology最新文献_第3页

Vomiting in children and adolescents receiving intravenous pegaspargase: a retrospective study. 静脉注射培加司琼酶的儿童和青少年的呕吐：一项回顾性研究。

IF 1.2 4区医学

Pediatric Hematology and Oncology Pub Date : 2024-08-01 Epub Date: 2024-03-07 DOI: 10.1080/08880018.2024.2311886

Jamie M Brown, Sherry Mathew, Maria Luisa Sulis, L Lee Dupuis, Jennifer Thackray

引用次数: 0

Challenges in Management of Pediatric Chronic Myeloid Leukemia (pCML) in the Low-Middle Income Countries (LMICs): Insights from an International CML Foundation (iCMLf) Multi-National Survey. 中低收入国家（LMICs）儿科慢性粒细胞白血病（pCML）管理面临的挑战：国际慢性粒细胞白血病基金会（iCMLf）多国调查的启示。

IF 1.7 4区医学

Pediatric Hematology and Oncology Pub Date : 2024-05-01 Epub Date: 2024-01-16 DOI: 10.1080/08880018.2023.2301404

Nirmalya Roy Moulik, Arlene Harriss-Buchan, Giuseppe Saglio, Meinolf Suttorp

引用次数: 0

The Onset of Puberty Presents Unique Management Issues in Penile Chronic Graft-versus-Host Disease Requiring Circumcision in Male Pediatric Patients. 青春期的发病为男性儿科患者需要包皮环切术的阴茎慢性移植物抗宿主疾病提供了独特的管理问题。

IF 1.7 4区医学

Pediatric Hematology and Oncology Pub Date : 2024-05-01 Epub Date: 2023-11-07 DOI: 10.1080/08880018.2023.2277765

Chia Huan Ng, Juliana P Roden, Jefferson Terry, Kirk R Schultz

{"title":"The Onset of Puberty Presents Unique Management Issues in Penile Chronic Graft-versus-Host Disease Requiring Circumcision in Male Pediatric Patients.","authors":"Chia Huan Ng, Juliana P Roden, Jefferson Terry, Kirk R Schultz","doi":"10.1080/08880018.2023.2277765","DOIUrl":"10.1080/08880018.2023.2277765","url":null,"abstract":"Chronic GvHD of the penile tract in male pediatric patients has not been described well in the literature and is often under-diagnosed. We report three cases of penile chronic GvHD in adolescent male patients who received HSCT before the onset of puberty. Their penile cGvHD became symptomatic upon the onset of penile growth associated with puberty in combination with the fibrotic changes in the foreskin. Symptoms did not respond to systemic chronic GvHD medication but require circumcision for alleviation of symptoms. This case series highlights the need for frequent monitoring of the prepubertal pediatric HSCT patient who has the presence of sclerotic cGvHD and enters puberty. This population is particularly reluctant to allow a thorough examination of the genitalia. In addition, optimization of systemic and topical immunosuppression treatment for patients with chronic GvHD of the penile tract potentially with the introduction of novel agents that target the tissue repair and fibrosis pathway is needed to prevent circumcision as the only option in the future.","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":null,"pages":null},"PeriodicalIF":1.7,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"71484664","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

A case of autoimmune lymphoproliferative syndrome with a novel de novo FAS variant. 自身免疫性淋巴细胞增生性综合征伴新发FAS变异1例。

IF 1.7 4区医学

Pediatric Hematology and Oncology Pub Date : 2024-05-01 Epub Date: 2023-12-04 DOI: 10.1080/08880018.2023.2286967

Firat Ozcelik, Kubra Aslan, Veysel Gok, Mucahit Bilgehan Ari, Alper Ozcan, Ahmet Eken, Ekrem Ünal, Yusuf Ozkul, Munis Dundar

引用次数: 0

The significance of surveillance imaging in children with Ewing sarcoma and osteosarcoma. 对尤文肉瘤和骨肉瘤患儿进行监测成像的意义。

IF 1.7 4区医学

Pediatric Hematology and Oncology Pub Date : 2024-05-01 Epub Date: 2024-02-12 DOI: 10.1080/08880018.2024.2311407

Scott Greve Brekke, Arne Lucke, Henrik Hasle, Thomas Baad-Hansen

{"title":"The significance of surveillance imaging in children with Ewing sarcoma and osteosarcoma.","authors":"Scott Greve Brekke, Arne Lucke, Henrik Hasle, Thomas Baad-Hansen","doi":"10.1080/08880018.2024.2311407","DOIUrl":"10.1080/08880018.2024.2311407","url":null,"abstract":"Primary bone tumors in children and adolescents, while rare, pose significant challenges in diagnosis and management. Children treated for Ewing sarcoma and osteosarcoma are offered a 5-year follow-up program after end of treatment, including radiological surveillance of primary location of tumor and the lungs. There is no consensus regarding how often and how the children should be followed with radiological imaging. This retrospective descriptive study of 69 patients (34 with Ewing sarcoma and 35 with osteosarcoma) investigated the consequences of abnormal findings in 1279 follow-up images. Nine relapses were detected, 4 in the Ewing group (3 local and 1 pulmonary) and 5 in the osteosarcoma group (1 local and 4 pulmonary). Of these, only two patients exhibited symptomatic relapses, with the remainder identified through imaging. The positive predictive value for relapse detection was 0.44 in the Ewing group, and 0.5 in the osteosarcoma group. In the Ewing sarcoma patient image follow-up program, the probability of anomaly detection was 12% (95% CI, 10-15). For osteosarcoma patients, the likelihood was 6% (95% CI, 4-8). Our data indicates that abnormal findings on follow-up images rarely represents relapse of tumor. As the surveillance protocol differs between the patient groups, wherein Ewing sarcoma patients primarily are monitored through MRI while osteosarcoma patients are predominantly tracked via X-rays, there is an increased occurrence of incidental findings in the first group. However, it is imperative to interpret imaging data in conjunction with clinical information, avoiding isolated reliance on imaging results when making treatment decisions.","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":null,"pages":null},"PeriodicalIF":1.7,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139723532","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

Hematopoietic stem cell transplantation for B-thalassemia major with alemtuzumab. 使用阿仑妥珠单抗治疗重型 B 型地中海贫血的造血干细胞移植。

IF 1.7 4区医学

Pediatric Hematology and Oncology Pub Date : 2024-05-01 Epub Date: 2023-12-22 DOI: 10.1080/08880018.2023.2296933

Luisanna M Sánchez, Anil George, Brian D Friend, Saleh Bhar, Ghadir Sasa, Erin E Doherty, John Craddock, David Steffin, Baheyeldin Salem, Khaled Yassine, Bilal Omer, Caridad Martinez, Kathryn Leung, Robert A Krance, Tami D John

{"title":"Hematopoietic stem cell transplantation for B-thalassemia major with alemtuzumab.","authors":"Luisanna M Sánchez, Anil George, Brian D Friend, Saleh Bhar, Ghadir Sasa, Erin E Doherty, John Craddock, David Steffin, Baheyeldin Salem, Khaled Yassine, Bilal Omer, Caridad Martinez, Kathryn Leung, Robert A Krance, Tami D John","doi":"10.1080/08880018.2023.2296933","DOIUrl":"10.1080/08880018.2023.2296933","url":null,"abstract":"While matched related donor (MRD) allogeneic hematopoietic stem cell transplantation (HSCT) is a curative option for transfusion-dependent beta-thalassemia (TDT), the use of alternative sources has increased, resulting in the exploration of novel transplant-conditioning regimens to reduce the contribution of graft-versus-host disease (GVHD) and graft failure (GF) to transplant-related morbidity and mortality. Alemtuzumab is a CD52 monoclonal antibody that has been successfully incorporated into myeloablative conditioning regimens for other hematologic conditions, yet there have been limited studies regarding the use of alemtuzumab in HSCT for TDT. The purpose of this study was to evaluate engraftment, incidence of GVHD, and transplant related morbidity and mortality in patients with TDT who received alemtuzumab in addition to standard busulfan-based conditioning. The primary endpoint was severe GVHD-free, event-free survival (GEFS). Our cohort included 24 patients with a median age of 6.8 years (range 1.5-14.9). Eleven patients received a 10/10 MRD HSCT, eleven 10/10 unrelated donor (UD), and two mismatched UD. All patients achieved primary engraftment. For all patients, 5-year GEFS was 77.4% and 5-year overall survival (OS) was 91%. The 5-year cumulative incidence of GF (attributed to poor graft function) without loss of donor chimerism was 13.8% (95% CI: 4.5, 35.3). We report low rates of significant acute GVHD grade II-IV (12.5%) and chronic GVHD (4.4%). Younger age and MRD were associated with significantly improved GEFS, OS and EFS. Our results show that the use of alemtuzumab promotes stable engraftment, may reduce rates of severe GVHD, and results in acceptable GEFS, OS, and EFS.","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":null,"pages":null},"PeriodicalIF":1.7,"publicationDate":"2024-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138830856","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

Successful haploidentical bone marrow transplantation in Griscelli syndrome type 2 with non-busulfan-based regimen and post-transplantation cyclophosphamide: a case report and review of the literature. 非白消安方案和移植后环磷酰胺治疗2型Griscelli综合征的单倍体骨髓移植成功：一例病例报告和文献综述。

IF 1.7 4区医学

Pediatric Hematology and Oncology Pub Date : 2024-05-01 Epub Date: 2023-10-31 DOI: 10.1080/08880018.2023.2273880

Saori Yamada, Yuta Maruyama, Shoji Saito, Kazutoshi Komori, Hirokazu Morokawa, Eri Okura, Koichi Hirabayashi, Yu Furui, Takashi Kurata, Makoto Nishioka, Tetsuhiro Fukuyama, Kazuo Sakashita, Yozo Nakazawa

引用次数: 0

Therapeutic efficacy of RAS inhibitor trametinib using a juvenile myelomonocytic leukemia patient-derived xenograft model. 利用幼年骨髓单核细胞白血病患者衍生异种移植模型研究 RAS 抑制剂曲美替尼的疗效。

IF 1.7 4区医学

Pediatric Hematology and Oncology Pub Date : 2024-04-22 DOI: 10.1080/08880018.2024.2343688

Alex Q. Lee, Hiroaki Konishi, Masami Ijiri, Yueju Li, Arun Panigrahi, Jeremy Chien, Noriko Satake

{"title":"Therapeutic efficacy of RAS inhibitor trametinib using a juvenile myelomonocytic leukemia patient-derived xenograft model.","authors":"Alex Q. Lee, Hiroaki Konishi, Masami Ijiri, Yueju Li, Arun Panigrahi, Jeremy Chien, Noriko Satake","doi":"10.1080/08880018.2024.2343688","DOIUrl":"https://doi.org/10.1080/08880018.2024.2343688","url":null,"abstract":"Juvenile myelomonocytic leukemia (JMML) is an aggressive pediatric leukemia with few effective treatments and poor outcomes even after stem cell transplantation, the only current curative treatment. We developed a JMML patient-derived xenograft (PDX) mouse model and demonstrated the in vivo therapeutic efficacy and confirmed the target of trametinib, a RAS-RAF-MEK-ERK pathway inhibitor, in this model. A PDX model was created through transplantation of patient JMML cells into mice, up to the second generation, and successful engraftment was confirmed using flow cytometry. JMML PDX mice were treated with trametinib versus vehicle control, with a median survival of 194 days in the treatment group versus 124 days in the control group (p = 0.02). Trametinib's target as a RAS pathway inhibitor was verified by showing inhibition of ERK phosphorylation using immunoblot assays. In conclusion, trametinib monotherapy significantly prolongs survival in our JMML PDX model by inhibiting the RAS pathway. Our model can be effectively used for assessment of novel targeted treatments, including potential combination therapies, to improve JMML outcomes.","PeriodicalId":19746,"journal":{"name":"Pediatric Hematology and Oncology","volume":null,"pages":null},"PeriodicalIF":1.7,"publicationDate":"2024-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140675623","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

Characterizing age-related differences in Hodgkin lymphoma in children, adolescents and young adults 儿童、青少年和青年霍奇金淋巴瘤与年龄有关的差异特征

IF 1.7 4区医学

Pediatric Hematology and Oncology Pub Date : 2024-04-08 DOI: 10.1080/08880018.2024.2337627

Nicole E. Kendel, Joseph R. Stanek, Faye K. Willen, Anthony N. Audino

引用次数: 0

Intra-arterial methylprednisolone for pediatric gastrointestinal post-transplant lymphoproliferative disorder. 动脉内甲基强的松龙治疗儿童胃肠道移植后淋巴增生性疾病。

IF 1.7 4区医学

Pediatric Hematology and Oncology Pub Date : 2024-04-01 Epub Date: 2023-10-20 DOI: 10.1080/08880018.2023.2267612

Hoyoung Jung, Ravjot Dhatt, S Rod Rassekh, Manraj K S Heran

引用次数: 0