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当代儿童血液恶性肿瘤的单倍体相合干细胞移植策略。

Contemporary haploidentical stem cell transplant strategies in children with hematological malignancies.

机构信息

Section of Oncology and BMT, Alberta Children's Hospital, University of Calgary, Calgary, AB, Canada.

出版信息

Bone Marrow Transplant. 2021 Jul;56(7):1518-1534. doi: 10.1038/s41409-021-01246-5. Epub 2021 Mar 5.

Abstract

The barriers to HLA-mismatched or haploidentical hematopoietic stem cell transplantation (HSCT), namely GvHD and graft failure, have been overcome with novel transplant platforms. Post-transplant Cyclophosphamide (PTCy) is widely available, feasible and easy to implement. TCRαβ T and B cell depletion comes with consistent GvHD preventive benefits irrespective of age and indication. Naive T-cell depletion helps prevention of severe viral reactivations. The Beijing protocol shows promising outcomes in patients with poor remission status at the time of transplantation. For children, the toxicities and late outcomes related to these transplants are truly relevant as they suffer the most in the long run from transplant-related toxicities, especially chronic GvHD. While comparing the outcomes of different Haplo-HSCT approaches, one must understand the transplant immunobiology and factors affecting the transplant outcomes. Leukemia remission status at the time of conditioning is a consistent factor affecting the transplant outcomes using any of these platforms. Prospective comparison of these platforms lacks in a homogenous population; however, the evidence is growing, and this review highlights the areas of research gaps.

摘要

新型移植平台克服了 HLA mismatched 或半相合造血干细胞移植(HSCT)的障碍,即移植物抗宿主病(GvHD)和移植物衰竭。移植后环磷酰胺(PTCy)广泛可用、可行且易于实施。TCRαβ T 和 B 细胞耗竭具有一致的 GvHD 预防益处,与年龄和适应证无关。幼稚 T 细胞耗竭有助于预防严重的病毒再激活。北京方案在移植时缓解状态较差的患者中显示出有前景的结果。对于儿童来说,与这些移植相关的毒性和晚期结果是非常重要的,因为从长远来看,他们受到移植相关毒性的影响最大,尤其是慢性 GvHD。在比较不同半相合 HSCT 方法的结果时,必须了解移植免疫生物学和影响移植结果的因素。在使用任何这些平台时,预处理时的白血病缓解状态是影响移植结果的一致因素。这些平台的前瞻性比较缺乏同质人群;然而,证据正在增加,本综述强调了研究空白领域。

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