Department of Pediatric Hemato-Oncology and Cell and Gene Therapy, Istituto di Ricovero e Cura a Carattere Scientifico Bambino Gesù Children's Hospital, 00165 Rome, Italy.
Department of Biomedicine and Prevention, University of Rome Tor Vergata, 00133 Rome, Italy.
Int J Mol Sci. 2021 Jan 11;22(2):642. doi: 10.3390/ijms22020642.
Acute promyelocytic leukemia (APL) represents a paradigm of precision medicine. Indeed, in the last decades, the introduction of all-trans retinoic acid (ATRA) and arsenic trioxide (ATO) completely revolutionized the therapeutic approach to this previously highly fatal disorder. This entirely chemotherapy-free treatment, which provided excellent survival rates, has been initially validated in adults and, recently, translated in the pediatric setting. This review summarizes currently available data on the use of ATRA and ATO combination in pediatric APL, providing a particular focus on peculiar issues and challenges, such as the occurrence of and death during induction (early death), as well as the advantage offered by the ATO/ATRA combination in sparing long-term .
急性早幼粒细胞白血病 (APL) 是精准医学的典范。事实上，在过去的几十年中，全反式维甲酸 (ATRA) 和三氧化二砷 (ATO) 的引入彻底改变了这种以前高度致命疾病的治疗方法。这种完全无化疗的治疗方法提供了极好的生存率，最初在成人中得到验证，最近在儿科中得到了应用。这篇综述总结了目前关于 ATRA 和 ATO 联合治疗儿科 APL 的可用数据，特别关注了一些特殊问题和挑战，例如诱导期间的发生和死亡（早期死亡），以及 ATO/ATRA 联合治疗在减少长期毒性方面的优势。
