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Indications for Hematopoietic Cell Transplantation and Immune Effector Cell Therapy: Guidelines from the American Society for Transplantation and Cellular Therapy.
Biology of Blood and Marrow Transplantation ( IF 5.609 ) Pub Date : 2020-03-09 , DOI: 10.1016/j.bbmt.2020.03.002
Abraham S Kanate 1 , Navneet S Majhail 2 , Bipin N Savani 3 , Christopher Bredeson 4 , Richard E Champlin 5 , Stephen Crawford 6 , Sergio A Giralt 7 , Charles F LeMaistre 8 , David I Marks 9 , James L Omel 10 , Paul J Orchard 11 , Jeanne Palmer 12 , Wael Saber 13 , Paul A Veys 14 , Paul A Carpenter 15 , Mehdi Hamadani 13
Affiliation  

The American Society for Transplantation and Cellular Therapy (ASTCT) published its first white paper on indications for autologous and allogeneic hematopoietic cell transplantation (HCT) in 2015. It was identified at the time that periodic updates of indications would be required to stay abreast with state of the art and emerging indications and therapy. In recent years the field has not only seen an improvement in transplantation technology, thus widening the therapeutic scope of HCT, but additionally a whole new treatment strategy using modified immune effector cells, including chimeric antigen receptor T cells and engineered T-cell receptors, has emerged. The guidelines review committee of the ASTCT deemed it optimal to update the ASTCT recommendations for indications for HCT to include new data and to incorporate indications for immune effector cell therapy (IECT) where appropriate. The guidelines committee established a multiple stakeholder task force consisting of transplant experts, payer representatives, and a patient advocate to provide guidance on indications for HCT and IECT. This article presents the updated recommendations from the ASTCT on indications for HCT and IECT. Indications for HCT/IECT were categorized as (1) Standard of care, where indication is well defined and supported by evidence; (2) Standard of care, clinical evidence available, where large clinical trials and observational studies are not available but have been shown to be effective therapy; (3) Standard of care, rare indication, for rare diseases where demonstrated effectiveness exists but large clinical trials and observational studies are not feasible; (4) Developmental, for diseases where preclinical and/or early-phase clinical studies show HCT/IECT to be a promising treatment option; and (5) Not generally recommended, where available evidence does not support the routine use of HCT/IECT. The ASTCT will continue to periodically review these guidelines and update them as new evidence becomes available.



中文翻译:

造血细胞移植和免疫效应细胞疗法的适应症:美国移植和细胞疗法学会的指南。

美国移植和细胞治疗学会(ASTCT)于2015年发布了其第一份有关自体和异基因造血细胞移植适应症(HCT)的白皮书。当时已确定需要定期更新适应症以与状态保持同步。和新兴的适应症和疗法。近年来,该领域不仅看到了移植技术的进步,从而扩大了HCT的治疗范围,而且使用修饰的免疫效应细胞(包括嵌合抗原受体T细胞和工程化T细胞受体)的全新治疗策略也有了新的进展出现了。ASTCT的指南审查委员会认为,最佳的方法是更新ASTCT关于HCT适应症的建议,以包括新数据,并在适当的情况下纳入免疫效应细胞治疗(IECT)的适应症。指导委员会成立了一个由利益相关者组成的多方工作组,由移植专家,付款人代表和患者倡导者组成,为HCT和IECT的适应症提供指导。本文介绍了ASTCT关于HCT和IECT适应症的更新建议。HCT / IECT的适应证分类为(1)护理标准,其中适应证明确定义并有证据支持;(2)护理标准,可用的临床证据,尚无大型临床试验和观察性研究但已被证明是有效的治疗方法;(3)护理标准,稀有适应症,适用于已证明有效但罕见的大型临床试验和观察性研究不可行的疾病;(4)对于临床前和/或早期临床研究显示HCT / IECT是有前途的治疗选择的疾病,是发展中的;(5)如果现有证据不支持HCT / IECT的常规使用,则通常不建议这样做。ASTCT将继续定期审查这些指南,并在有新证据时对其进行更新。如果有证据不支持常规使用HCT / IECT。ASTCT将继续定期审查这些指南,并在有新证据时对其进行更新。如果有证据不支持常规使用HCT / IECT。ASTCT将继续定期审查这些指南,并在有新证据时对其进行更新。

更新日期:2020-03-09
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