Tosi Manuela, Spinelli Orietta, Leoncin Matteo, Cavagna Roberta, Pavoni Chiara, Lussana Federico, Intermesoli Tamara, Frison Luca, Perali Giulia, Carobolante Francesca, Viero Piera, Skert Cristina, Rambaldi Alessandro, Bassan Renato
Hematology Unit, Azienda Socio Sanitaria Territoriale (ASST), Ospedale Papa Giovanni XXIII, 24127 Bergamo, Italy.
Hematology Unit, Azienda Ulss3 Serenissima, Ospedale dell'Angelo, 30174 Venezia-Mestre, Italy.
Cancers (Basel). 2021 Apr 27;13(9):2108. doi: 10.3390/cancers13092108.
In many clinical studies published over the past 20 years, adolescents and young adults (AYA) with Philadelphia chromosome negative acute lymphoblastic leukemia (Ph- ALL) were considered as a rather homogeneous clinico-prognostic group of patients suitable to receive intensive pediatric-like regimens with an improved outcome compared with the use of traditional adult ALL protocols. The AYA group was defined in most studies by an age range of 18-40 years, with some exceptions (up to 45 years). The experience collected in pediatric ALL with the study of post-induction minimal residual disease (MRD) was rapidly duplicated in AYA ALL, making MRD a widely accepted key factor for risk stratification and risk-oriented therapy with or without allogeneic stem cell transplantation and experimental new drugs for patients with MRD detectable after highly intensive chemotherapy. This combined strategy has resulted in long-term survival rates of AYA patients of 60-80%. The present review examines the evidence for MRD-guided therapies in AYA's Ph- ALL, provides a critical appraisal of current treatment pitfalls and illustrates the ways of achieving further therapeutic improvement according to the massive knowledge recently generated in the field of ALL biology and MRD/risk/subset-specific therapy.
在过去20年发表的许多临床研究中,费城染色体阴性急性淋巴细胞白血病(Ph-ALL)的青少年和青年(AYA)被视为一个临床预后相当同质的患者群体,适合接受类似儿科的强化治疗方案,与使用传统成人ALL方案相比,预后有所改善。在大多数研究中,AYA组的定义年龄范围为18至40岁,也有一些例外(最高至45岁)。儿科ALL中关于诱导后微小残留病(MRD)研究积累的经验很快在AYA ALL中得到复制,使得MRD成为广泛接受的风险分层和风险导向治疗的关键因素,无论是否进行异基因干细胞移植以及对于高强度化疗后可检测到MRD的患者使用实验性新药。这种联合策略使AYA患者的长期生存率达到了60%-80%。本综述探讨了AYA的Ph-ALL中MRD指导治疗的证据,对当前治疗缺陷进行了批判性评估,并根据ALL生物学以及MRD/风险/亚组特异性治疗领域最近产生的大量知识,阐述了实现进一步治疗改善的方法。