Merli Pietro, Algeri Mattia, Del Bufalo Francesca, Locatelli Franco
Department of Pediatric Hematology and Oncology, Bambino Gesù Children's Hospital, Piazza Sant'Onofrio, 4, 00165, Rome, Italy.
Sapienza University of Rome, Rome, Italy.
Curr Hematol Malig Rep. 2019 Apr;14(2):94-105. doi: 10.1007/s11899-019-00502-2.
The remarkable improvement in the prognosis of children with acute lymphoblastic leukemia (ALL) has been mainly achieved through the administration of risk-adapted therapy, including allogeneic hematopoietic stem cell transplantation (HSCT). This paper reviews the current indications to HSCT in ALL children, as well as the type of donor and conditioning regimens commonly used. Finally, it will focus on future challenges in immunotherapy.
As our comprehension of disease-specific risk factors improves, indications to HSCT continue to evolve. Future studies will answer the year-old question on the best conditioning regimen to be used in this setting, while a recent randomized controlled study fixed the optimal anti-thymocyte globulin dose in unrelated donor HSCT. HSCT, the oldest immunotherapy used in clinical practice, still represents the gold standard consolidation treatment for a number of pediatric patients with high-risk/relapsed ALL. New immunotherapies hold the promise of further improving outcomes in this setting.
急性淋巴细胞白血病(ALL)患儿预后的显著改善主要通过实施风险适应性治疗实现,其中包括异基因造血干细胞移植(HSCT)。本文综述了目前ALL患儿HSCT的适应证,以及常用的供体类型和预处理方案。最后,将聚焦免疫治疗的未来挑战。
随着我们对疾病特异性危险因素理解的提高,HSCT的适应证不断演变。未来的研究将回答这个由来已久的问题,即在这种情况下使用的最佳预处理方案是什么,而最近一项随机对照研究确定了无关供体HSCT中抗胸腺细胞球蛋白的最佳剂量。HSCT是临床实践中使用最久的免疫疗法,对于许多高危/复发ALL的儿科患者,它仍然是巩固治疗的金标准。新的免疫疗法有望在这种情况下进一步改善治疗效果。