Mengxuan Shang, Fen Zhou, Runming Jin
Department of Pediatrics, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Front Pediatr. 2022 Jun 23;10:923419. doi: 10.3389/fped.2022.923419. eCollection 2022.
With the markedly increased cure rate for children with newly diagnosed pediatric B-cell acute lymphoblastic leukemia (B-ALL), relapse and refractory B-ALL (R/R B-ALL) remain the primary cause of death worldwide due to the limitations of multidrug chemotherapy. As we now have a more profound understanding of R/R ALL, including the mechanism of recurrence and drug resistance, prognostic indicators, genotypic changes and so on, we can use newly emerging technologies to identify operational molecular targets and find sensitive drugs for individualized treatment. In addition, more promising and innovative immunotherapies and molecular targeted drugs that are expected to kill leukemic cells more effectively while maintaining low toxicity to achieve minimal residual disease (MRD) negativity and better bridge hematopoietic stem cell transplantation (HSCT) have also been widely developed. To date, the prognosis of pediatric patients with R/R B-ALL has been enhanced markedly thanks to the development of novel drugs. This article reviews the new advancements of several promising strategies for pediatric R/R B-ALL.
随着新诊断的儿童B细胞急性淋巴细胞白血病(B-ALL)治愈率显著提高,由于多药化疗的局限性,复发和难治性B-ALL(R/R B-ALL)仍是全球范围内主要的死亡原因。随着我们现在对R/R ALL有了更深入的了解,包括复发机制、耐药性、预后指标、基因型变化等,我们可以利用新兴技术来确定可操作的分子靶点,并找到敏感药物进行个体化治疗。此外,更有前景和创新性的免疫疗法以及分子靶向药物也得到了广泛发展,这些疗法和药物有望在保持低毒性的同时更有效地杀死白血病细胞,以实现微小残留病(MRD)阴性,并更好地衔接造血干细胞移植(HSCT)。迄今为止,由于新型药物的研发,R/R B-ALL儿科患者的预后已得到显著改善。本文综述了几种治疗儿科R/R B-ALL的有前景策略的新进展。