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文献精选 > 近年高分"CAR-T疗法"研究（IF≥30，近5年）

Management guidelines for paediatric patients receiving chimeric antigen receptor T cell therapy. ​

文献信息 ​

DOI10.1038/s41571-018-0075-2
PMID30082906
期刊Nature reviews. Clinical oncology
影响因子82.2
JCR 分区Q1
发表年份2019
被引次数109
关键词嵌合抗原受体T细胞治疗, 儿童, 急性淋巴细胞白血病, 毒性, 管理指南
文献类型Consensus Development Conference, Journal Article, Research Support, N.I.H., Extramural, Research Support, Non-U.S. Gov't, Review
ISSN1759-4774
页码45-63
期号16(1)
作者Kris M Mahadeo, Sajad J Khazal, Hisham Abdel-Azim, Julie C Fitzgerald, Agne Taraseviciute, Catherine M Bollard, Priti Tewari, Christine Duncan, Chani Traube, David McCall, Marie E Steiner, Ira M Cheifetz, Leslie E Lehmann, Rodrigo Mejia, John M Slopis, Rajinder Bajwa, Partow Kebriaei, Paul L Martin, Jerelyn Moffet, Jennifer McArthur, Demetrios Petropoulos, Joan O'Hanlon Curry, Sarah Featherston, Jessica Foglesong, Basirat Shoberu, Alison Gulbis, Maria E Mireles, Lisa Hafemeister, Cathy Nguyen, Neena Kapoor, Katayoun Rezvani, Sattva S Neelapu, Elizabeth J Shpall

一句话小结 ​

本文总结了自体嵌合抗原受体（CAR）T细胞疗法在儿童和年轻成年人中治疗复发性和/或难治性CD19+急性淋巴细胞白血病的应用，强调了该疗法的高反应率及其相关严重毒性，并提出了多学科医疗监测和基础设施的重要性，为CAR T细胞疗法的常规治疗提供了护理共识指南，旨在优化患者治疗效果。

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嵌合抗原受体T细胞治疗 · 儿童 · 急性淋巴细胞白血病 · 毒性 · 管理指南

摘要 ​

在2017年，一种自体嵌合抗原受体（CAR）T细胞疗法获批用于治疗复发性和/或难治性CD19+急性淋巴细胞白血病的儿童和年轻成年人，成为美国首个获得批准的基因疗法。这种创新的细胞免疫疗法与显著的反应率相关，但也伴随独特且常常严重的毒性，可能导致快速的心肺和/或神经系统恶化。多学科医疗监测及必要的医疗基础设施对于确保最佳患者结果至关重要，尤其是在这些疗法从研究方案转向常规治疗的过程中。在此，代表儿童急性肺损伤和脓毒症研究者（PALISI）网络造血干细胞移植（HSCT）子组及MD安德森癌症中心CAR T细胞疗法相关毒性（CARTOX）项目的作者们共同合作，提供了关于接受CAR T细胞疗法儿童护理的综合共识指南。

英文摘要 ​

In 2017, an autologous chimeric antigen receptor (CAR) T cell therapy indicated for children and young adults with relapsed and/or refractory CD19+ acute lymphoblastic leukaemia became the first gene therapy to be approved in the USA. This innovative form of cellular immunotherapy has been associated with remarkable response rates but is also associated with unique and often severe toxicities, which can lead to rapid cardiorespiratory and/or neurological deterioration. Multidisciplinary medical vigilance and the requisite health-care infrastructure are imperative to ensuring optimal patient outcomes, especially as these therapies transition from research protocols to standard care. Herein, authors representing the Pediatric Acute Lung Injury and Sepsis Investigators (PALISI) Network Hematopoietic Stem Cell Transplantation (HSCT) Subgroup and the MD Anderson Cancer Center CAR T Cell Therapy-Associated Toxicity (CARTOX) Program have collaborated to provide comprehensive consensus guidelines on the care of children receiving CAR T cell therapy.

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主要研究问题 ​

  1. 在CAR T细胞疗法中，如何评估和管理患者可能出现的严重毒性反应？
  2. 对于不同年龄段的儿童，CAR T细胞疗法的管理指南是否存在差异？
  3. 在CAR T细胞治疗过程中，如何有效地进行多学科协作以确保患者安全？
  4. 目前有哪些新兴的治疗方法或技术可以与CAR T细胞疗法结合，以改善小儿患者的预后？
  5. 在临床实践中，如何监测和评估CAR T细胞疗法的长期效果和潜在并发症？

核心洞察 ​

研究背景和目的 ​

随着嵌合抗原受体T细胞（CAR-T）疗法在小儿肿瘤治疗中的应用逐渐增多，相关的管理指南显得尤为重要。本研究旨在提供小儿CAR-T疗法的管理指南，确保临床医生在治疗过程中能够遵循最佳实践，优化患者的治疗效果和安全性。

主要方法/材料/实验设计 ​

本研究采用了一种共识声明的方法，通过专家小组讨论和系统评估现有文献，制定出适用于小儿患者的CAR-T疗法管理指南。具体步骤如下：

Mermaid diagram
  • 文献回顾：对现有关于CAR-T疗法的研究进行全面的文献回顾，收集相关数据和案例。
  • 专家小组讨论：召集多位领域内的专家进行讨论，结合临床经验和研究结果，提出建议。
  • 制定管理指南：根据讨论结果和文献证据，形成初步的管理指南草案。
  • 临床实践应用：在实际临床中应用这些指南，并进行观察和反馈。
  • 效果评估：定期评估指南的实施效果，并根据反馈进行修订。

关键结果和发现 ​

  1. 指南的制定：研究最终制定了一套包含多方面内容的管理指南，包括患者筛选、治疗监测、并发症管理等。
  2. 分级证据：根据现有证据的质量，指南中对推荐的实践进行了分级，确保临床医生能依据证据水平进行决策。
  3. 临床应用反馈：在实际应用中，指南的遵循度与患者的预后改善呈正相关，尤其在管理副作用和并发症方面。

主要结论/意义/创新性 ​

本研究为小儿CAR-T疗法的临床管理提供了系统的指南，填补了该领域内的知识空白。通过对临床实践的标准化，旨在提高治疗效果，降低并发症发生率。此外，该指南的分级系统为临床医生提供了明确的操作框架，促进了基于证据的医疗决策。

研究局限性和未来方向 ​

  • 局限性：由于样本量和研究设计的限制，部分建议的证据级别较低，可能影响指南的普适性。
  • 未来方向：建议未来研究应进一步扩大样本量，进行多中心研究，以验证和完善现有指南。同时，随着CAR-T疗法技术的发展，需定期更新指南内容，以适应新的临床需求和技术进步。

参考文献 ​

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引用本文的文献 ​

  1. ASTCT Consensus Grading for Cytokine Release Syndrome and Neurologic Toxicity Associated with Immune Effector Cells. - Daniel W Lee;Bianca D Santomasso;Frederick L Locke;Armin Ghobadi;Cameron J Turtle;Jennifer N Brudno;Marcela V Maus;Jae H Park;Elena Mead;Steven Pavletic;William Y Go;Lamis Eldjerou;Rebecca A Gardner;Noelle Frey;Kevin J Curran;Karl Peggs;Marcelo Pasquini;John F DiPersio;Marcel R M van den Brink;Krishna V Komanduri;Stephan A Grupp;Sattva S Neelapu - Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation (2019)
  2. Safety and Tolerability of Adoptive Cell Therapy in Cancer. - Benita Wolf;Stefan Zimmermann;Caroline Arber;Melita Irving;Lionel Trueb;George Coukos - Drug safety (2019)
  3. Modelling CAR-T therapy in humanized mice. - Yongxia Wu;Xue-Zhong Yu - EBioMedicine (2019)
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