Rausch Christian, Hiddemann Wolfgang, von Bergwelt-Baildon Michael, Spiekermann Karsten, Herold Tobias
Medizinische Klinik und Poliklinik III, Klinikum der Universität München, LMU München, München, Deutschland.
Dtsch Med Wochenschr. 2022 Sep;147(17):1108-1114. doi: 10.1055/a-1758-2452. Epub 2022 Aug 28.
The suspicion of acute myeloid leukemia (AML) is a haematological emergency that requires a rapid diagnostic workup. Symptoms are usually caused by cytopenias of all blood cell lines. The differentiation of acute promyelocytic leukemia (APL) is important because of the early death rate caused by thrombembolic and bleeding events. Rapid immunophenotypic and genetic characterization is necessary for risk stratification and therapy selection. For this purpose, a center with appropriate expertise should be contacted. Therapy has become more complex due to numerous new approvals. For certain patients, the established intensive induction therapy with cytarabine and anthracycline is now combined with targeted agents, like the antibody conjugate Gemtuzumab-Ozogamicin or the FLT3 inhibitor Midostaurin. Patients with secondary AML benefit from the liposomal chemotherapy combination CPX-351. Therapy with the hypomethylating agent Azacitidine and the BCL2-inhibitor Venetoclax (Aza/Ven) represents the standard for patients who are not fit for intensive therapy. Here, it is important to consider interactions with CYP3A4-effective drugs.In most cases, APL is treated "chemotherapy-free" with all-trans retinoic acid (ATRA) and arsenic trioxide (ATO). In high-risk patients, the combination of chemotherapy and ATRA is still standard.Moreover, maintenance therapies were (re)established as an important therapeutic element of post-remission therapy. For example, Midostaurin is used in patients with FLT3 mutations, as is the multikinase inhibitor sorafenib after allogeneic stem cell transplantation. In addition, oral azacitidine is available for non-allogeneic transplant eligible patients in first complete remission. These new drugs have improved prognosis and resulted in a more individualized therapy mostly driven by genetic aberrations. This development will continue in the next years and will significantly improve treatment options, especially for older patients.
怀疑患有急性髓系白血病（AML）是一种血液学急症，需要进行快速诊断检查。症状通常由所有血细胞系的血细胞减少引起。急性早幼粒细胞白血病（APL）的鉴别很重要，因为血栓栓塞和出血事件会导致早期死亡率。快速的免疫表型和基因特征分析对于风险分层和治疗选择是必要的。为此，应联系具备适当专业知识的中心。由于众多新药获批，治疗变得更加复杂。对于某些患者，目前已确立的阿糖胞苷和蒽环类药物强化诱导治疗现与靶向药物联合使用，如抗体偶联物吉妥珠单抗-奥唑米星或FLT3抑制剂米哚妥林。继发性AML患者可从脂质体化疗组合CPX-351中获益。使用低甲基化药物阿扎胞苷和BCL2抑制剂维奈克拉（阿扎/维奈）进行治疗是不适合强化治疗患者的标准治疗方法。在此，重要的是要考虑与CYP3A4有效药物的相互作用。在大多数情况下，APL采用全反式维甲酸（ATRA）和三氧化二砷（ATO）进行“无化疗”治疗。在高危患者中，化疗与ATRA联合仍然是标准治疗方法。此外，维持治疗已重新成为缓解后治疗的重要治疗要素。例如，米哚妥林用于FLT3突变患者，异基因干细胞移植后使用多激酶抑制剂索拉非尼也是如此。此外，口服阿扎胞苷可用于首次完全缓解且不符合异基因移植条件的患者。这些新药改善了预后，并导致了更多以基因异常为主导的个体化治疗。这种发展在未来几年将持续，并将显著改善治疗选择，尤其是对老年患者。
