Abstract：
Hematological malignancies represent defying clinical conditions, with high levels of morbidity and mortality, particularly considering patients who manifest multiple refractory diseases. Recently, chimeric antigen receptor (CAR)-T cell therapy has emerged as a potential treatment option for relapsed/refractory B cell malignancies, which have motivated the Food and Drug Administration approval of a series of products based on this technique. The objective of this systematic review was to assess the efficacy and safety of CAR-T cell therapy for patients with hematological malignancies. A comprehensive literature search was conducted in the electronic databases (CENTRAL, Embase, LILACS, and MEDLINE), clinical trials register platforms (Clinicaltrials.gov and WHO-ICTRP), and grey literature (OpenGrey). The Cochrane Handbook for Reviews of Interventions was used for developing the review and the PRISMA Statement for manuscript reporting. The protocol was prospectively published in PROSPERO database (CRD42020181047). After the selection process, seven RCTs were included, three of which with available outcome results. The available results are from studies assessing axicabtagene, lisocabtagene, and tisagenlecleucel for patients with B cell lymphoma, and the certainty of evidence ranged from very low to low for survival and progression-related outcome and for safety outcomes. Additionally, four randomized controlled trials comparing CAR-T cell therapy to the standard treatment for various types of relapsed/refractory B cell non-Hodgkin lymphomas and multiple myeloma included in this systematic review still did not have available outcome data. The results of this review may be used to guide clinical practice but evidence concerning the safety and efficacy of CAR-T Cell therapy for hematological malignancies is still immature to recommend its application outside of clinical trials or compassionate use context for advanced and terminal cases. It is expected the results of the referred comparative studies will provide further elements to subsidize the broader application of this immunotherapy.
摘要：
恶性血液病代表不符合临床条件，发病率和死亡率都很高，特别是考虑表现出多种难治性疾病的患者。最近,嵌合抗原受体（CAR）-T细胞疗法已成为复发性/难治性B细胞恶性肿瘤的潜在治疗选择，这促使食品和药物管理局批准了一系列基于这种技术的产品。本系统综述的目的是评估CAR-T细胞疗法对血液系统恶性肿瘤患者的疗效和安全性。在电子数据库(CENTRAL，Embase,LILACS,和MEDLINE)，临床试验注册平台（Clinicaltrials.gov和WHO-ICTRP），和灰色文学（OpenGrey）。《Cochrane干预措施审查手册》用于开发审查和PRISMA声明用于手稿报告。该方案前瞻性地发表在PROSPERO数据库(CRD42020181047)中。在选择过程之后，包括七个RCT，其中三个有可用的结果。可用的结果来自评估axicabtagene的研究，lioscabtagene,和tisagenlecleucel治疗B细胞淋巴瘤，对于生存和进展相关结局以及安全性结局,证据的确定性从非常低到低不等.此外,本系统综述中纳入的4项将CAR-T细胞疗法与标准疗法治疗各种类型的复发/难治性B细胞非霍奇金淋巴瘤和多发性骨髓瘤进行比较的随机对照试验仍没有可用的结局数据.本综述的结果可用于指导临床实践，但有关CAR-T细胞疗法治疗血液恶性肿瘤的安全性和有效性的证据仍不成熟，无法推荐其在临床试验或对晚期和晚期病例的同情使用背景之外的应用。预计所提及的比较研究的结果将提供进一步的要素来补贴这种免疫疗法的更广泛的应用。
