Pediatric Blood & Cancer最新文献

英文中文

Cardiovascular Complications of Immune Effector Cell Therapies in Pediatric Hematological and Solid Tumors 免疫效应细胞治疗在儿童血液病和实体瘤中的心血管并发症。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2025-01-20 DOI: 10.1002/pbc.31557

Aaron B. Ross, Kriti Puri, Thomas L. Johnson, Lilliam D. Correa, Tami D. John, Brian Friend, Shoba A. Navai, Nabil Ahmed, Meenakshi G. Hegde, Bilal Omer, Andras Heczey, Baheyeldin M. Salem, Hossein Tcharmtchi, Rayne H. Rouce, David H. Steffin, Robert A Krance, Nino C. Rainusso, Saleh Bhar

Background

Immune effector cell (IEC) therapies, including chimeric antigen receptor (CAR)-modified T-cell therapy, have shown efficacy in pediatric B-cell acute lymphoblastic leukemia (B-ALL) and are being investigated for other malignancies. A common toxicity associated with IEC therapy is cytokine release syndrome (CRS), which can lead to cardiovascular decompensation due to systemic inflammation. Data are limited regarding cardiovascular adverse effects in children. This study aims to describe the cardiovascular adverse effect profile of IEC therapies in pediatric patients with hematologic and solid tumor malignancies.

Methods

We retrospectively reviewed patients who received IECs directed towards various targets in patients with hematologic, solid, and brain tumor malignancies from January 2014 to June 2023 at Texas Children's Hospital. The primary end point was hypotension requiring vasoactive support and/or heart failure within 30 days of infusion.

Results

A total of 203 patients met inclusion criteria. Pretreatment echocardiogram was available for 142 (70%) pediatric patients, of whom 140 (96%) had normal baseline systolic function. Hypotension requiring vasoactive support occurred in 26 (13%) patients. Hematologic malignancy indications (p = 0.002), total body irradiation (TBI) (p = 0.002), and allogenic hematopoietic stem cell transplants (HCT) (p = 0.035) were associated with increased risk of hypotension requiring vasoactive support. Follow-up echocardiograms were available for 14 patients who met the primary end point, and all showed return to baseline within 6 months.

Conclusions

Significant hemodynamic compromise occurred in a minority of patients treated with IEC therapies. All experiencing cardiac dysfunction had recovery of function, and there was no cardiovascular-related mortality.

背景：免疫效应细胞（IEC）疗法，包括嵌合抗原受体（CAR）修饰的t细胞疗法，已经显示出对儿童b细胞急性淋巴细胞白血病（B-ALL）的疗效，并且正在研究其他恶性肿瘤的疗效。与IEC治疗相关的常见毒性是细胞因子释放综合征（CRS），它可导致全身炎症引起的心血管失代偿。关于儿童心血管不良反应的数据有限。本研究旨在描述儿童血液学和实体肿瘤恶性患者中IEC治疗的心血管不良反应概况。方法：我们回顾性回顾了2014年1月至2023年6月在德克萨斯儿童医院接受针对不同靶点的血液、实体和脑肿瘤恶性肿瘤患者的IECs。主要终点为输注30天内需要血管活性支持的低血压和/或心力衰竭。结果：203例患者符合纳入标准。142例（70%）儿童患者可获得预处理超声心动图，其中140例（96%）基线收缩功能正常。26例（13%）患者出现需要血管活性支持的低血压。血液恶性适应症（p = 0.002）、全身照射（p = 0.002）和同种异体造血干细胞移植（p = 0.035）与低血压需要血管活性支持的风险增加相关。随访的超声心动图显示，14例达到主要终点的患者均在6个月内恢复到基线水平。结论：在接受IEC治疗的少数患者中发生了明显的血流动力学损害。所有经历心功能障碍的患者都恢复了功能，并且没有心血管相关的死亡。

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引用次数: 0

Impact of Platelet Transfusion at Different Doses in Oncohematology Pediatric Inpatients and Outpatients: A Retrospective Study 不同剂量血小板输注对儿科血液学住院和门诊患者的影响：一项回顾性研究。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2025-01-20 DOI: 10.1002/pbc.31550

Pierpaolo Berti, Mauro Montanari, Massimo La Raja, Renato Giua, Irene Terrenato, Marco Becilli, Valeria Paganelli, Valentina Di Ruscio, Daniele Deriu, Eleonora D'Agostino, Ilaria Ferruzzi, Marta Conte, Ottavia Porzio, Michele Vacca

Background

Platelet (PLT) transfusion is an essential strategy to prevent bleeding in children with thrombocytopenia associated to cancer treatment. However, data on optimal pediatric dosing and transfusion thresholds are limited.

Methods

This retrospective study analyzed data from 607 pediatric patients with hematologic malignancies, nonmalignant disorders, and solid tumors who developed hypoproliferative thrombocytopenia during therapy. In the first phase (Objective 1), the effective response to transfusion (ERTR) was assessed following International Collaboration for Transfusion Medicine guidelines (ICTMG), comparing low-dose (1.1 × 10¹¹ PLT/m² body surface area) transfusions for inpatients and medium dose (2.2 × 10¹¹) for outpatients. Transfusion thresholds were set at less than 10,000/µL versus 10,000–20,000/µL, and overall PLT concentrate consumption was analyzed. The second phase (Objective 2) examined the total number of transfusions per patient, incidence of major bleeding events, and bleeding-related mortality rates across dosing groups.

Results

ERTR ranged from 65% to 82%, with significantly higher rates in outpatients compared to inpatients. In outpatients with PLT less than 10,000/µL, the medium dose showed no significant advantage over the lower inpatient dose. Similar efficacy was observed between low (<10,000/µL) and high (10,000–20,000/µL) transfusion triggers, with no statistically significant difference in the incidence of major bleeding events across groups. The low-dose strategy was significantly associated with a reduction in PLT volume transfused compared to the standard expected dose.

Conclusions

These findings support the application of ICTMG in pediatric settings. The lower PLT dose for inpatients is safe and effective, providing benefits in resource utilization, while a higher transfusion trigger (20,000/µL) does not improve outcomes.

背景：血小板（PLT）输血是预防与癌症治疗相关的血小板减少症儿童出血的必要策略。然而，关于最佳儿科剂量和输血阈值的数据有限。方法：本回顾性研究分析了607例在治疗期间出现低增殖性血小板减少症的儿童血液恶性肿瘤、非恶性疾病和实体肿瘤患者的数据。在第一阶段（目的1），根据国际输血医学合作指南（ICTMG）评估输血的有效反应（ERTR），比较住院患者的低剂量（1.1 × 1011 PLT/m2体表面积）输血和门诊患者的中剂量（2.2 × 1011）输血。输血阈值设置为低于10,000/µL和10,000-20,000/µL，并分析整体PLT浓缩物消耗。第二阶段（目标2）检查了每个患者的输血总数、大出血事件的发生率以及各给药组的出血相关死亡率。结果：ERTR范围为65%至82%，门诊患者的ERTR明显高于住院患者。在PLT低于10,000/µL的门诊患者中，中剂量没有明显优于低剂量住院患者。结论：这些发现支持ICTMG在儿科环境中的应用。住院患者较低的PLT剂量是安全有效的，有利于资源利用，而较高的输血触发量（20,000/µL）并不能改善预后。

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引用次数: 0

Impacts of Ethical Dilemmas and Moral Distress in Pediatric Hematology Oncology Nurses 儿科血液肿瘤科护士伦理困境与道德困境的影响。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2025-01-17 DOI: 10.1002/pbc.31546

Arshia Madni, Deena R. Levine, Kimberly E. Sawyer, Yasmine Azzi, Belinda N. Mandrell, Yimei Li, Emily Ashcraft, Jacklyn Boggs, Rushil Acharya, Mary Caples, Jami S. Gattuso, Laurie A. Shoulders, Liza-Marie Johnson

Purpose

To assess the level of moral distress (MD) and perceptions of ethical climate among pediatric hematology/oncology (PHO) nurses and to identify bioethics topics where increased education was desired.

Methods

In this cross-sectional study, we administered the 26-item Swedish Moral Distress Scale-Revised (sMDS-R), specifically revised and validated for pediatric oncology, in conjunction with the Clinical Ethics Needs Assessment Survey (CENAS). Electronic surveys were sent to inpatient and outpatient PHO nurses. Analysis included descriptive statistics, Spearman correlation, and the Mann–Whitney U test.

Results

Of 123 nurse respondents, the overall mean MDS-R score was 2.75 (range: 0–16). Distressing encounters occurred infrequently (frequency mean 0.96, range: 0–4), resulting in relatively low distress (mean 2.24, range: 0–4). The scenarios resulting in the highest overall MD included performing painful procedures on children (2.46), lack of meaningful conversations due to time constraints (2.41), and poor team communication (2.68). Inpatient nurses reported higher levels of MD compared to outpatient nurses (p = 0.0002). The overall CENAS score was greater than 3 (range: 1–4), suggesting a positive ethical climate. There was a moderate negative relationship between sMDS-R combined scores and CENAS scores (p < 0.0001). Inpatient nurses reported a less positive ethical climate compared to outpatient nurses (p = 0.01). Nurses expressed a need for additional ethics education around withholding/withdrawing of life-sustaining treatments, providing end-of-life care, and working with challenging patients/families.

Conclusions

Although the ethical climate was overall positive, several clinical situations appear to result in higher moral distress despite their infrequency, especially on inpatient units. Additional resources and education in navigating ethical dilemmas were requested by the nurses.

目的：评估儿科血液学/肿瘤学（PHO）护士的道德困扰（MD）水平和对伦理气候的看法，并确定需要加强教育的生物伦理主题。方法：在这项横断面研究中，我们使用了26项瑞典道德困扰量表修订版（sMDS-R），该量表专门针对儿科肿瘤学进行了修订和验证，并结合临床伦理需求评估调查（CENAS）。电子调查发送给住院和门诊PHO护士。分析包括描述性统计、Spearman相关和Mann-Whitney U检验。结果：123名受访护士的总体平均MDS-R评分为2.75分（范围：0-16分）。痛苦遭遇发生的频率较低（平均频率0.96，范围0-4），导致相对较低的痛苦（平均2.24，范围0-4）。导致最高总体MD的情况包括对儿童进行痛苦的手术（2.46），由于时间限制而缺乏有意义的对话（2.41），以及糟糕的团队沟通（2.68）。住院护士报告的MD水平高于门诊护士（p = 0.0002）。总体的CENAS得分大于3（范围：1-4），表明一个积极的道德氛围。sMDS-R综合评分与CENAS评分之间存在中度负相关(p)结论：尽管伦理氛围总体上是积极的，但几种临床情况似乎导致更高的道德困扰，尽管这种情况很少发生，特别是在住院病房。护士们要求提供更多的资源和教育，以应对道德困境。

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引用次数: 0

Management of accidental live attenuated virus vaccination during treatment of a child with acute lymphoblastic leukemia 急性淋巴细胞白血病患儿治疗期间意外减毒活疫苗接种的处理。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2025-01-16 DOI: 10.1002/pbc.31398

Marc Bienias, Svenja Feldmann, Angela Wawer, Andreas Osterman, Stephan Böhm, Oliver T. Keppler, Julia Hauer

引用次数: 0

Towards Targeted Therapies in Childhood Autoimmune Cytopenias: A Report on Leniolisib Use 儿童自身免疫性细胞减少症的靶向治疗：lenolisib使用的报告。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2025-01-16 DOI: 10.1002/pbc.31547

Aurélia Alimi, Julie Maitre, Jérôme Hadjadj, Frédéric Rieux Laucat, Michaela Semeraro, Capucine Picard, Stéphane Perdereau, Guy Leverger, Sebastien Héritier

引用次数: 0

Multicenter Retrospective Analysis of Pediatric Differentiated Thyroid Carcinoma: Treatment Practices and Outcomes Prior to Pediatric American Thyroid Association Guidelines Implementation 儿童分化型甲状腺癌的多中心回顾性分析：美国儿童甲状腺协会指南实施前的治疗实践和结果。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2025-01-16 DOI: 10.1002/pbc.31545

Priya Mahajan, Sarah Beth May, Meghan Shekar, David Werny, Briana C. Patterson, Christine M. Chan, Jennifer H. Aldrink, Kyle Z. Horvath, Douglas S. Hawkins, Thomas A. Olson, Rajkumar Venkatramani

Background

Differentiated thyroid carcinoma (DTC) is the most common pediatric thyroid malignancy, with papillary thyroid carcinoma (PTC) representing 90% of the cases. In 2015, the American Thyroid Association (ATA) developed management guidelines for pediatric DTC.

Procedure

Patients less than 21 years of age diagnosed with DTC between 2000 and 2015 at Texas Children's Hospital, Seattle Children's Hospital, Children's Healthcare of Atlanta, Children's Hospital Colorado, and Nationwide Children's Hospital were retrospectively analyzed to evaluate treatment practices before the implementation of the ATA guidelines.

Results

We included 216 pediatric patients with DTC. A majority were female (76%) with a median age at diagnosis of 14.8 years. Neck swelling (56%) and palpable nodules (49%) were common presenting symptoms. Diagnostic evaluations typically included thyroid ultrasound, with common features being microcalcifications (47.7%) and hypoechogenicity (39.0%). Fine-needle aspiration cytology confirmed diagnosis in 50.5%. The majority of patients underwent total thyroidectomy (78.7%) and lymph node dissection (75.5%). ATA risk stratification was low, intermediate, and high risk in 111 (51.4%), 29 (13.4%), and 76 patients (35.2%), respectively. Upfront radioactive iodine (RAI) was administered in 83%. At 1-year follow-up, 46% were disease-free (66% low risk, 59% intermediate risk, and 13% high risk). The 5-year progression-free survival for low, intermediate, and high risk were 86%, 53%, and 43%, respectively. The disease-related overall survival was 100%.

Conclusions

Prior to the introduction of the ATA guidelines, a majority of patients underwent total thyroidectomy with lymph node dissection followed by RAI. Selective use of RAI for low-risk disease as recommended in the current guidelines may be appropriate.

背景：分化型甲状腺癌（DTC）是最常见的儿童甲状腺恶性肿瘤，其中乳头状甲状腺癌（PTC）占90%。2015年，美国甲状腺协会（ATA）制定了儿科DTC的管理指南。方法：回顾性分析2000年至2015年间在德克萨斯儿童医院、西雅图儿童医院、亚特兰大儿童保健中心、科罗拉多州儿童医院和全国儿童医院诊断为DTC的21岁以下患者，以评估实施ATA指南之前的治疗实践。结果：我们纳入了216例DTC患儿。大多数为女性（76%），诊断时的中位年龄为14.8岁。颈部肿胀（56%）和可触及的结节（49%）是常见的症状。诊断评估通常包括甲状腺超声，常见特征是微钙化（47.7%）和低回声（39.0%）。细针穿刺细胞学确诊50.5%。多数患者行甲状腺全切除术（78.7%）和淋巴结清扫（75.5%）。ATA风险分层分别为低、中、高风险111例（51.4%）、29例（13.4%）和76例（35.2%）。83%的患者接受放射性碘（RAI）治疗。在1年的随访中，46%的患者无疾病（66%为低危，59%为中危，13%为高危）。低、中、高风险患者的5年无进展生存率分别为86%、53%和43%。与疾病相关的总生存率为100%。结论：在ATA指南引入之前，大多数患者接受甲状腺全切除术并淋巴结清扫，然后进行RAI。根据现行指南的建议，对低风险疾病选择性使用RAI可能是合适的。

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引用次数: 0

Posterior Mediastinal Tumor Presenting with Compressive Myelopathy: Uncommon Site for a Rare Tumor 后纵隔肿瘤表现为压迫性脊髓病：罕见肿瘤的罕见部位。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2025-01-16 DOI: 10.1002/pbc.31556

Mohanaraj Ramachandran, Padmalatha Kadamba, Namita Ravikumar, Komal Chippalkatti, Rama Mohan Reddy, Harisha Karibasappa, Dinesh Raju, Arjun Srivatsa, Sai Prasad T R, Prasad Narayanan, Harish Pathalingappa

引用次数: 0

Familial History in Ovarian Teratomas Is a Frequent Event: 22 Years’ Experience at a Single Center 卵巢畸胎瘤的家族史是一个常见的事件：在一个中心22年的经验。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2025-01-14 DOI: 10.1002/pbc.31548

Cécile Olivia Muller, Fanny Delehaye, Christopher T. Gordon, Aurore Pire, Alaa Cheikhelard, Carmen Capito, Véronique Rousseau, Jeanne Amiel, Sabine Sarnacki

Background

Ovarian mature teratoma represents the most common benign neoplasm among pediatric germ cell tumors. This study reports the prevalence and characteristics of familial forms identified in a single center over 22 years in order to better understand possible familial predispositions to ovarian teratoma.

Methods

The records of all patients who were surgically treated for ovarian teratoma between 2000 and 2022 were retrospectively reviewed. Families were systematically asked about a possible family history of ovarian tumors (benign or malignant) or testicular germ cell tumors.

Results

Among the 136 patients operated on for ovarian teratoma, 24 cases of familial form (18%) were identified. Patients with familial history of ovarian mature teratoma were more likely to have multifocal tumors at diagnosis and metachronous lesions over time (p = 0.032 and p = 0.018, respectively) compared with patients without familial history. The family history concerned first-degree relatives in 12/24 cases (50%) including one case of twin monozygotic sisters, and affected two consecutive generations in three cases.

Conclusion

A systematic examination made it possible to find up to 18% of familial forms in mature teratomas of the ovary, which in these cases seem to be more likely multifocal. The underlying genetic mechanisms are likely to be heterogeneous. Further work seems to be necessary to provide a better understanding of the genesis of germ cell tumors.

背景：卵巢成熟畸胎瘤是儿童生殖细胞肿瘤中最常见的良性肿瘤。为了更好地了解卵巢畸胎瘤可能的家族性易感性，本研究报告了在单个中心鉴定的家族性畸胎瘤的患病率和特征。方法：回顾性分析2000 ~ 2022年所有卵巢畸胎瘤手术治疗患者的临床资料。系统地询问家庭是否有卵巢肿瘤（良性或恶性）或睾丸生殖细胞肿瘤的家族史。结果：136例卵巢畸胎瘤患者中，发现家族性畸胎瘤24例（18%）。有卵巢成熟畸胎瘤家族史的患者在诊断时多灶性肿瘤和随时间推移发生异时性病变的可能性较无家族史的患者高（p = 0.032和p = 0.018）。12/24例（50%）有一级亲属家族史，其中1例为同卵双胞胎姐妹，3例为连续两代。结论：系统的检查可以在卵巢成熟畸胎瘤中发现高达18%的家族性畸胎瘤，在这些病例中，似乎更可能是多灶性畸胎瘤。潜在的遗传机制可能是异质的。进一步的工作似乎是必要的，以提供更好的理解生殖细胞肿瘤的发生。

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引用次数: 0

Achieving Language Justice in Pediatric Hematology-Oncology: A Multinational Perspective for Language-Concordant Equitable Patient- and Family-Centered Care and Research Inclusion 在儿童血液肿瘤学中实现语言公正：以患者和家庭为中心的语言和谐、公平的护理和研究包容的多国视角。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2025-01-14 DOI: 10.1002/pbc.31521

Jenny Ruiz, Joanna M. Robles, Luisanna M. Sánchez, Alisa Khan, Amy D. Lu, Stacey Marjerrison, Andres Morales La Madrid, Paula Aristizabal

Language-discordant healthcare encounters—when the patient/caregiver and clinician are not able to communicate directly in the patient's/caregiver's preferred language—are associated with worse quality of care, increased adverse events, and research exclusion. Here, we describe the current state of language justice in clinical practice and research in the United States, Canada, and Spain, discuss the role of social determinants of health and language in patient safety and health outcomes, and review an example of culturally and linguistically concordant interventions to increase research participation. We close with practical and global strategies to increase multilingual research participation and to provide equitable patient- and family-centered care in pediatric hematology-oncology.

当患者/护理人员和临床医生不能用患者/护理人员首选的语言直接沟通时，语言不协调的医疗保健遭遇与更差的护理质量、更多的不良事件和研究排斥有关。在这里，我们描述了美国、加拿大和西班牙临床实践和研究中语言公正的现状，讨论了健康和语言的社会决定因素在患者安全和健康结果中的作用，并回顾了一个文化和语言协调的干预措施以增加研究参与的例子。我们以实际和全球战略结束，以增加多语言研究参与，并提供公平的儿童血液学肿瘤学以患者和家庭为中心的护理。

引用次数: 0

Rhabdomyosarcoma Surgical Update. 横纹肌肉瘤外科进展。

IF 2.4 3区医学 Q2 HEMATOLOGY

Pediatric Blood & Cancer

Pub Date : 2025-01-14 DOI: 10.1002/pbc.31496

Timothy Rogers, Andreas Schmidt, Amanda F Buchanan, Thomas Scharschmidt, Roshni Dasgupta, Ekene A Onwuka, David A Rodeberg

Rhabdomyosarcoma (RMS) tumors arise from mesenchymal tissue and represent half of pediatric sarcomas, which in turn make up 7% of pediatric tumors. Advances in local control therapy of RMS have improved outcomes after surgical resection of the primary tumor, either before or after induction chemotherapy, even in the setting of metastatic disease. The utilization of diagnostic core needle and sentinel node biopsy techniques for lymph node staging are becoming more widely used. Over the past several years, refinement of prognostic factors with adoption of fusion status instead of histology, and optimized risk stratification schemas have been developed to assure appropriate therapy. There have been efforts between North American and European surgical oncology cooperative groups to standardize the care we provide, and yet there are still some philosophical differences to overcome.

横纹肌肉瘤（Rhabdomyosarcoma， RMS）肿瘤起源于间质组织，占小儿肉瘤的一半，而小儿肉瘤又占小儿肿瘤的7%。RMS局部控制治疗的进展改善了原发肿瘤手术切除后的预后，无论是在诱导化疗之前还是之后，甚至在转移性疾病的情况下。诊断核心针和前哨淋巴结活检技术在淋巴结分期中的应用越来越广泛。在过去的几年中，采用融合状态代替组织学来改进预后因素，并优化风险分层方案，以确保适当的治疗。北美和欧洲的外科肿瘤合作组织一直在努力使我们提供的护理标准化，但仍有一些哲学上的差异需要克服。

引用次数: 0

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