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DICER1 syndrome with hepatoblastoma and pleuropulmonary blastoma 伴有肝母细胞瘤和胸膜肺泡瘤的 DICER1 综合征
IF 3.2 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-17 DOI: 10.1002/pbc.31285
Melda Berber Hamamci, Şule Yeşil, Pınar Bıçakçıoğlu, Tuğba Ramaslı Gürsoy, Yetkin Ağaçkıran, Burçak Kurucu, Azize Ceren Kılcı, Şeyma Ünüvar Gök, Ali Fettah, Gürses Şahin
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引用次数: 0
Successful surgical monotherapy for a patient with low‐risk head and neck nonparameningeal rhabdomyosarcoma with genetic profiling 利用基因图谱成功为一名低风险头颈部非脑膜旁横纹肌肉瘤患者提供单一手术疗法
IF 3.2 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-17 DOI: 10.1002/pbc.31323
Yusuke Tsumura, Yuko Kakuda, Takashi Mukaigawa, Masakuni Serizawa, Koiku Asakura, Mitsuko Akaihata, Rieko Taniguchi, Ikuko Takahashi, Yuji Ishida
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引用次数: 0
Outcomes of proton therapy to infradiaphragmatic sites in pediatric patients with Hodgkin lymphoma 对霍奇金淋巴瘤儿科患者膈下部位进行质子治疗的结果
IF 3.2 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-13 DOI: 10.1002/pbc.31290
Cecilia Jiang, Michele Kim, Xiaoyan Han, Monica Chelius, Travis Hoover, Leslie Kersun, Anne F. Reilly, Harper Hubbeling, Elizabeth Cummings, Goldie Kurtz, Christine Hill‐Kayser, John P. Plastaras, Michael J. LaRiviere
BackgroundProton therapy (PT) has potential advantages in pediatric Hodgkin lymphoma (pHL). However, there are limited data on PT, specifically to infradiaphragmatic targets. We report on PT planning details, doses achieved to organs at risk (OARs), and clinical and toxicity outcomes for patients with pHL who received PT to infradiaphragmatic regions.MethodsThis is a retrospective study including patients treated between 2011 and 2022. Demographic and clinical factors were collected, and toxicity was reported using Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. Dosimetric and clinical factors associated with key outcomes were assessed via Cox regression. Photon plans were generated for all patients, and the paired t‐tests or Wilcoxon signed rank sum tests were used for dosimetric comparisons.ResultsTwenty‐one patients comprising 22 PT courses were included. Median follow‐up was 5.0 years, and mean age was 14.2 years. Median dose was 21 Gray equivalent (GyE) over 14 fractions. Top acute grade 1 (G1) toxicities included fatigue (59%) and anorexia (36%). Rates of acute G2 and G3+ toxicity were 18% and 0%, respectively. After PT, no local or marginal failures occurred. Five percent experienced disease progression, who were all successfully salvaged, and all patients were alive and disease‐free at last follow‐up. No secondary malignancies developed. Compared to photon radiotherapy, PT achieved significantly lower doses to the bowels, stomach, spleen, pancreatic tail, liver, kidneys, and pelvic bones.ConclusionsPT is well‐tolerated and leads to excellent oncologic and toxicity outcomes with long‐term follow‐up. PT confers dosimetric advantages when compared to photons.
背景质子疗法(PT)在小儿霍奇金淋巴瘤(pHL)中具有潜在优势。然而,有关质子治疗的数据有限,尤其是针对膈下靶点的治疗。我们报告了接受膈下靶区质子治疗的pHL患者的质子治疗计划细节、危险器官(OARs)所达到的剂量以及临床和毒性结果。研究收集了人口统计学和临床因素,并使用不良事件通用术语标准(CTCAE)5.0版报告了毒性。通过 Cox 回归评估了与主要结果相关的剂量和临床因素。所有患者的光子计划均已生成,剂量比较采用配对t检验或Wilcoxon符号秩和检验。中位随访时间为 5.0 年,平均年龄为 14.2 岁。中位剂量为21格雷当量(GyE),共14个分次。最常见的急性1级(G1)毒性包括疲劳(59%)和厌食(36%)。G2 和 G3+ 急性毒性的发生率分别为 18% 和 0%。PT 治疗后,没有出现局部或边缘失败。有5%的患者病情恶化,但他们都被成功救治,所有患者在最后一次随访时都健在且无病。无继发性恶性肿瘤发生。与光子放疗相比,PT对肠、胃、脾、胰尾、肝、肾和盆腔骨骼的剂量明显较低。与光子相比,PT 具有剂量学优势。
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引用次数: 0
Pylorus‐preserving pancreaticoduodenectomy with superior mesenteric vein resection and reconstruction in a child with recurrent hepatoblastoma after liver transplantation 一名肝移植后复发肝母细胞瘤患儿的保留幽门胰十二指肠切除术和肠系膜上静脉切除与重建术
IF 3.2 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-13 DOI: 10.1002/pbc.31330
Ioannis A. Ziogas, Ankush Gosain, Richard D. Schulick, Jonathan P. Roach, Marco Del Chiaro
Pancreaticoduodenectomy with vascular reconstruction is rarely performed in children. We present a 3‐year‐old male with stage IV hepatoblastoma and pre‐treatment extent of disease (PRETEXT) stage III with tumor into the portal vein and superior mesenteric vein (SMV), and with brain and lung metastases status post chemotherapy and stereotactic radiosurgery to left frontal brain lesion. He then underwent deceased donor liver transplant with Roux‐en‐Y hepaticojejunostomy complicated by two recurrences to bilateral lungs treated with wedge resections. His course lastly involved a third hepatoblastoma recurrence to the SMV that was managed with pylorus‐preserving pancreaticoduodenectomy with SMV resection and reconstruction.
儿童很少接受胰十二指肠切除术和血管重建术。我们为您介绍一名 3 岁男性患者,肝母细胞瘤 IV 期,治疗前疾病程度(PRETEXT)III 期,肿瘤进入门静脉和肠系膜上静脉(SMV),化疗后出现脑和肺转移,左额叶脑部病变接受了立体定向放射外科手术。随后,他接受了Roux-en-Y肝空肠吻合术的死亡供体肝脏移植手术,术后并发双肺楔形切除术治疗的两次复发。最后,他的第三次肝母细胞瘤复发至SMV,经保留幽门的胰十二指肠切除术和SMV切除与重建术治疗。
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引用次数: 0
Outcomes in children with first‐relapsed acute lymphoblastic leukemia in Japan: Results from JCCG Study JPLSG‐ALL‐R08 日本首次复发急性淋巴细胞白血病患儿的治疗结果:JCCG研究JPLSG-ALL-R08的结果
IF 3.2 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-13 DOI: 10.1002/pbc.31319
Junko Yamanaka, Chitose Ogawa, Ayumu Arakawa, Takao Deguchi, Toshinori Hori, Nobutaka Kiyokawa, Hideaki Ueki, Masanori Nishi, Shinji Mochizuki, Takuro Nishikawa, Tadashi Kumamoto, Ritsuo Nishiuchi, Atsushi Kikuta, Shohei Yamamoto, Katsuyoshi Koh, Daisuke Hasegawa, Atsushi Ogawa, Kenichiro Watanabe, Atsushi Sato, Akiko M. Saito, Tomoyuki Watanabe, Atsushi Manabe, Keizo Horibe, Hiroaki Goto, Hidemi Toyoda
Background/objectivesThe Berlin–Frankfurt–Münster (BFM)‐S classification is a crucial prognostic indicator in children experiencing first‐relapsed acute lymphoblastic leukemia (ALL). Early molecular response to therapy, evaluated by measurable/minimal residual disease (MRD), has a significant impact on the survival of patients with childhood ALL. Applying risk stratification based on the BFM‐S classification and MRD response after induction, the first nationwide prospective multicenter study, ALL‐R08, was conducted in children with first‐relapsed ALL in Japan.MethodsThe ALL‐R08 study comprised two parts: ALL‐R08‐I, an observational study aimed at obtaining an overall picture of outcomes in first‐relapsed childhood ALL, and ALL‐R08‐II, a clinical trial for the non‐T‐ALL S2 risk group. In ALL‐R08‐II, patients with an MRD level of ≥10−3 at the end of induction therapy were assigned to undergo allogeneic hematopoietic stem cell transplantation (allo‐HCT), whereas those with an MRD level less than 10−3 and isolated extramedullary relapse continued to receive chemotherapy.ResultsIn total, 163 patients were enrolled in the ALL‐R08 study, and 82 and 81 patients were enrolled in the ALL‐R08‐I and the ALL‐R08‐II, respectively. In ALL‐R08‐I, the probability of 3‐year event‐free survival (EFS) for patients with S1, S2, S3, S4, and post‐HCT groups was 83% ± 15%, 37% ± 11%, 28% ± 8%, 14% ± 7%, and 0%, respectively. In the ALL‐R08‐II trial, 3‐year EFS in patients with post‐induction MRD less than 10−3 and ≥10−3 was 70% ± 9% (n = 27) and 68% ± 8% (n = 31) (p = .591), respectively.ConclusionsALL‐REZ BFM‐type treatment is equally effective for children with first‐relapsed ALL treated according to the Japanese frontline protocols and for children with first‐relapsed ALL treated according to the BFM‐type frontline protocols.
背景/目的柏林-法兰克福-明斯特(BFM)-S分类是首次复发急性淋巴细胞白血病(ALL)患儿的重要预后指标。根据可测量/最小残留病(MRD)评估的早期分子治疗反应对儿童 ALL 患者的生存有重要影响。根据BFM-S分类和诱导治疗后的MRD反应进行风险分层,日本首次在全国范围内对首次复发的ALL儿童进行了前瞻性多中心研究ALL-R08:ALL-R08-I是一项观察性研究,旨在全面了解首次复发儿童ALL的治疗效果;ALL-R08-II是一项临床试验,针对非T-ALL S2风险组。在ALL-R08-II中,诱导治疗结束时MRD水平≥10-3的患者被分配接受异基因造血干细胞移植(allo-HCT),而MRD水平小于10-3和孤立髓外复发的患者则继续接受化疗。结果ALL-R08研究共招募了163名患者,ALL-R08-I和ALL-R08-II分别招募了82名和81名患者。在ALL-R08-I试验中,S1、S2、S3、S4组和HCT后组患者的3年无事件生存(EFS)概率分别为83%±15%、37%±11%、28%±8%、14%±7%和0%。在ALL-R08-II试验中,诱导后MRD小于10-3和≥10-3的患者的3年EFS分别为70%±9%(n = 27)和68%±8%(n = 31)(p = .591)。结论ALL-REZ BFM型治疗对按照日本一线方案治疗的首次复发ALL儿童和按照BFM型一线方案治疗的首次复发ALL儿童同样有效。
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引用次数: 0
Role, education, policies and competencies for advanced practice in paediatric haematology‐oncology nursing in Europe: A scoping review 欧洲儿科血液肿瘤学护理高级实践的角色、教育、政策和能力:范围审查
IF 3.2 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-13 DOI: 10.1002/pbc.31325
Matteo Amicucci, Eugenia Trigoso, Maria Grazia Nori, Sara Colomer‐Lahiguera, Elena Rostagno, Valentina Biagioli, Vincenza Sansone, Andrea Zibaldo, Andrea Mastria, Moreno Crotti Partel, Marta Canesi, Andreea Cristina Schiopu, Immacolata Dall'Oglio
The aim of this scoping review is to describe the role, education, policies/regulation, skills and competencies required for advanced practice in paediatric haematology‐oncology nursing in Europe, highlighting the differences in development between the different European countries. A scoping review was conducted following the methodological framework of guidelines by Arksey and O'Malley and the recommendations for advancing the methodology by Levac et al. We searched MEDLINE/PubMed, EMBASE, CINAHL, Cochrane Library, Scopus, grey literature, webpages, reference lists and performed a manual search, without any restrictions on language or time. The intersection between databases, grey literature and evidence documents traced from the sites of the most authoritative European organisations in the field made it possible to identify the regulatory and training differences between the various countries that were examined. This scoping review highlights how advanced knowledge and competences are used in the care of paediatric haematology‐oncology patients, which are strictly necessary for implementing quality care. At present these competences are not recognised in policies and regulation in most of the countries that were examined. It is desirable that all EU member states work to implement a radical change and allow these more competent figures to assist patients in the best possible way.
本范围界定综述旨在描述欧洲儿科血液学-肿瘤学护理高级实践所需的角色、教育、政策/法规、技能和能力,突出不同欧洲国家之间的发展差异。我们检索了 MEDLINE/PubMed、EMBASE、CINAHL、Cochrane 图书馆、Scopus、灰色文献、网页、参考文献列表,并进行了手动检索,没有任何语言或时间限制。数据库、灰色文献和从该领域最权威的欧洲组织网站上搜索到的证据文件之间的交叉使我们有可能确定被审查国家之间在监管和培训方面的差异。本范围界定综述强调了儿科血液肿瘤学患者护理中如何使用先进的知识和能力,这些知识和能力是实施优质护理的严格必要条件。目前,大多数国家的政策和法规都没有承认这些能力。欧盟所有成员国都应努力实施彻底变革,让这些能力更强的人员以最佳方式为患者提供帮助。
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引用次数: 0
Successful ibrutinib treatment for pulmonary involvement in a post‐transplant patient with inherited bone marrow failure syndrome and very short telomeres 伊布替尼治疗一名患有遗传性骨髓衰竭综合征和极短端粒的移植后患者的肺部受累获得成功
IF 3.2 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-10 DOI: 10.1002/pbc.31314
Yu Furui, Shoji Saito, Yuta Maruyama, Eri Okura, Koichi Hirabayashi, Miyuki Tanaka, Yozo Nakazawa
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引用次数: 0
Feasibility of a prospective pediatric melanocytic tumor clinical trial: A report of multidisciplinary clinician survey data from the Children's Oncology Group Rare Tumor Committee 前瞻性儿科黑色素细胞肿瘤临床试验的可行性:儿童肿瘤学组罕见肿瘤委员会多学科临床医生调查数据分析报告
IF 3.2 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-10 DOI: 10.1002/pbc.31312
Sarah G. Mitchell, Emily Christison‐Lagay, Jennie Aldrink, Michael R. Sargen, Theodore W. Laetsch, Mary Austin, Melinda Jen, Robyn Gartrell, Arivarasan Karunamurthy, John M. Kirkwood, Alberto S. Pappo, Brittani K. N. Seynnaeve
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引用次数: 0
NRAS melanoma response to trametinib and azacitidine NRAS 黑色素瘤对曲美替尼和阿扎胞苷的反应
IF 3.2 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-10 DOI: 10.1002/pbc.31316
Shannon Wessel, Daniela Russi, Harper Price, Francis Eshun
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引用次数: 0
Multilevel challenges to equitable inclusion of children in trials when parents use languages other than English: A qualitative report from Children's Oncology Group's Diversity and Health Disparities Committee Language Equity Working Group 当父母使用英语以外的语言时,儿童公平参与试验所面临的多层面挑战:儿童肿瘤学集团多样性与健康差异委员会语言公平工作组的定性报告。
IF 2.4 3区 医学 Q2 HEMATOLOGY Pub Date : 2024-09-05 DOI: 10.1002/pbc.31321
Sheila Judge Santacroce, Melissa P. Beauchemin, Wendy Pelletier, Joanna M. Robles, Jenny Ruiz, Lindsay J. Blazin, Paula Aristizabal, Manuela Orjuela-Grimm, Anurekha G. Hall, Justine Kahn, Cassie Kline, Alix E. Seif, Maria C. Velez, Lena E. Winestone

Background

Increasing representation in clinical trials is a priority for the National Cancer Institute and Children's Oncology Group (COG). Our survey of COG-affiliated institutions revealed that many sites have insufficient processes and resources to enroll children whose parents use languages other than English (LOE). We describe reported barriers and facilitators to enrolling children in clinical trials when parents use LOE and propose opportunities for improvement.

Procedures

We sent a 20-item survey to COG-affiliated institutions. Five items allowed respondents to expand on replies to questions about (a) local institutional review board (IRB) requirements regarding translation of consent documents, (b) contributors to provider discomfort consenting parents who use LOE, (c) available language services and resources, and (d) barriers to enrolling children whose parents use LOE or offer ideas about approaches to improvements. Two pairs of researchers independently coded free-text responses and compared results for concordance.

Results

A total of 139 (N = 230; 60%) institutions returned the survey. Respondents were mainly physician principal investigators (n = 79/139; 57%) at the United States sites (n = 118/139; 85%) serving less than 100 newly diagnosed children per year (n = 99/139, 71%). They described challenges at multiple levels. Proposed approaches to improvements included centralized provision of translated materials and video educational materials in various languages, and collaborating with IRBs on regulatory processes that protect families and facilitate equitable clinical trial access.

Conclusions

Clinical trial consortia, such as COG, face challenges in enrolling representative samples. Further research is required to design and implement multilevel interventions to ensure equitable access for all, regardless of language used, and mitigate disparate research participation.

背景:提高临床试验的代表性是美国国立癌症研究所和儿童肿瘤组织(COG)的一项优先工作。我们对 COG 附属机构的调查显示,许多研究机构没有足够的流程和资源让父母使用非英语语言(LOE)的儿童参与临床试验。我们描述了所报告的父母使用 LOE 时儿童参与临床试验的障碍和促进因素,并提出了改进机会:我们向 COG 附属机构发送了一份包含 20 个项目的调查问卷。其中五个项目允许受访者对以下问题的回答进行扩展:(a) 当地机构审查委员会(IRB)关于同意书翻译的要求;(b) 使用LOE的父母同意提供者不适的促成因素;(c) 可用的语言服务和资源;(d) 父母使用LOE的儿童入组的障碍或提供改进方法的想法。两对研究人员独立对自由文本回答进行编码,并比较结果是否一致:共有 139 家(N = 230;60%)机构返回了调查问卷。受访者主要是美国医疗机构(118/139;85%)的医生首席研究员(79/139;57%),他们每年为不到 100 名新确诊儿童提供服务(99/139,71%)。他们描述了多个层面的挑战。建议的改进方法包括集中提供各种语言的翻译材料和视频教育材料,以及与IRB合作制定保护家庭和促进公平获得临床试验机会的监管流程:临床试验联盟(如 COG)在招募代表性样本方面面临挑战。需要进一步开展研究,设计并实施多层次干预措施,以确保所有人(无论使用何种语言)都能公平参与,并减少研究参与的差异。
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引用次数: 0
期刊
Pediatric Blood & Cancer
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