Riley L C, Hann I M, Wheatley K, Stevens R F
Department of Paediatrics, Middlesex Hospital, London, UK.
Br J Haematol. 1999 Aug;106(2):436-44. doi: 10.1046/j.1365-2141.1999.01550.x.
Between 1988 and 1995, 341 children with acute myeloid leukaemia (AML) were treated on the Medical Research Council Acute Myeloid Leukaemia Trial (MRC AML10). The 5-year overall survival was 57%, much improved on previous trials. However, there were 47 deaths (13. 8%), 11 of which were associated with bone marrow transplantation (BMT). The treatment-related mortality was significant at 13.8%, but decreased in the latter half of the trial from 17.8% in 1998-91 to 9. 6% in 1992-95 (P = 0.03%). The main causes of death were infection (65.9%), haemorrhage (19.1%) and cardiac failure (19.1%). Fungal infection was a significant problem, causing 23% of all infective deaths. Haemorrhage occurred early in treatment, in children with initial white cell counts >100 x 109/l (P = 0.001), and was more common in those with M4 and M5 morphology. Cardiac failure only occurred from the third course of chemotherapy onwards, with 78% (7/9) in conjunction with sepsis as a terminal event. Some deaths could be prevented by identifying those most at risk, and with prompt recognition and aggressive management of complications of treatment. Future options include the prophylactic use of antifungal agents, and the use of cardioprotectants or alternatives to conventional anthracyclines to decrease cardiac toxicity.
1988年至1995年间，341名急性髓系白血病（AML）患儿参加了医学研究委员会急性髓系白血病试验（MRC AML10）。5年总生存率为57%，比之前的试验有了显著提高。然而，有47例死亡（13.8%），其中11例与骨髓移植（BMT）有关。治疗相关死亡率为13.8%，具有统计学意义，但在试验后半期有所下降，从1990 - 1991年的17.8%降至1992 - 1995年的9.6%（P = 0.03%）。主要死亡原因是感染（65.9%）、出血（19.1%）和心力衰竭（19.1%）。真菌感染是一个严重问题，占所有感染性死亡的23%。出血发生在治疗早期，初始白细胞计数>100×10⁹/L的儿童中更为常见（P = 0.001），并且在具有M4和M5形态的患儿中更常见。心力衰竭仅在化疗第三疗程及以后出现，78%（7/9）与败血症作为终末事件同时发生。通过识别高危人群以及及时识别和积极处理治疗并发症，可以预防一些死亡。未来的选择包括预防性使用抗真菌药物，以及使用心脏保护剂或替代传统蒽环类药物以降低心脏毒性。
