AML 患者的 5 年生存率大约为 25%。[98]Howlader N, Noone AM, Krapcho M, et al, eds. Table 13.16: Acute myeloid leukemia - 5-year relative and period survival by race, sex, diagnosis year and age. In: SEER Cancer Statistics Review, 1975-2011. Bethesda, MD: National Cancer Institute; 2014. Based on November 2013 SEER data submission, posted to the SEER web site, April 2014.http://seer.cancer.gov/csr/1975_2011/browse_csr.php?sectionSEL=13&pageSEL=sect_13_table.16.html
较为年轻(年龄<60岁)的健康患者
总的来说,在 18 - 60 岁的 AML 患者中,只有约 1/3 的患者将被治愈。然而,医生可使用 2 个参数将患者分为不同的风险组,这两个参数是非常重要的复发预后指标:诊断时的细胞遗传学以及第一个化疗疗程后骨髓中原始细胞的百分比。[66]Breems DA, Van Putten WL, Huijgens PC, et al. Prognostic index for adult patients with acute myeloid leukemia in first relapse. J Clin Oncol. 2005;23:1969-1978.http://jco.ascopubs.org/content/23/9/1969.fullhttp://www.ncbi.nlm.nih.gov/pubmed/15632409?tool=bestpractice.com[95]Milligan DW, Grimwade D, Cullis JO, et al; British Committee for Standards in Haematology. Guidelines on the management of acute myeloid leukaemia in adults. Br J Haematol. 2006;135:450-474.http://onlinelibrary.wiley.com/doi/10.1111/j.1365-2141.2006.06314.x/fullhttp://www.ncbi.nlm.nih.gov/pubmed/17054678?tool=bestpractice.com
对于细胞遗传学有利的低风险组患者 [即t (8;21) 或 inv16] ,不论首疗程治疗后的骨髓结果如何,5 年生存率和复发风险分别为 76% 和 23%。[95]Milligan DW, Grimwade D, Cullis JO, et al; British Committee for Standards in Haematology. Guidelines on the management of acute myeloid leukaemia in adults. Br J Haematol. 2006;135:450-474.http://onlinelibrary.wiley.com/doi/10.1111/j.1365-2141.2006.06314.x/fullhttp://www.ncbi.nlm.nih.gov/pubmed/17054678?tool=bestpractice.com
对于标准风险组患者(细胞遗传学既不好也不差,治疗第一个疗程后骨髓中原始细胞的百分比不大于 15%),5 年生存率和复发风险分别为 48% 和 52%。[46]Schlenk RF, Döhner K, Krauter J, et al. Mutations and treatment outcome in cytogenetically normal acute myeloid leukemia. N Engl J Med. 2008;358:1909-1918.http://www.nejm.org/doi/full/10.1056/NEJMoa074306#t=articlehttp://www.ncbi.nlm.nih.gov/pubmed/18450602?tool=bestpractice.com[95]Milligan DW, Grimwade D, Cullis JO, et al; British Committee for Standards in Haematology. Guidelines on the management of acute myeloid leukaemia in adults. Br J Haematol. 2006;135:450-474.http://onlinelibrary.wiley.com/doi/10.1111/j.1365-2141.2006.06314.x/fullhttp://www.ncbi.nlm.nih.gov/pubmed/17054678?tool=bestpractice.com
对于细胞遗传学不利 [即 -5、-7、del (5q) 、异常 3q、t (9,22) ] 和/或第一个化疗疗程后骨髓中原始细胞的百分比>15% 的高危组患者,5 年生存率和复发风险分别为 21% 和 73%。[95]Milligan DW, Grimwade D, Cullis JO, et al; British Committee for Standards in Haematology. Guidelines on the management of acute myeloid leukaemia in adults. Br J Haematol. 2006;135:450-474.http://onlinelibrary.wiley.com/doi/10.1111/j.1365-2141.2006.06314.x/fullhttp://www.ncbi.nlm.nih.gov/pubmed/17054678?tool=bestpractice.com
年龄较长的(年龄 ≥60 岁)患者
老年患者的总体前景较年轻患者差。其原因包括不良细胞遗传学的发生率较高、老年患者中的先前骨髓增生异常、多药耐药的发生率较高以及影响强化治疗耐受能力的共存疾患的发生率升高。[95]Milligan DW, Grimwade D, Cullis JO, et al; British Committee for Standards in Haematology. Guidelines on the management of acute myeloid leukaemia in adults. Br J Haematol. 2006;135:450-474.http://onlinelibrary.wiley.com/doi/10.1111/j.1365-2141.2006.06314.x/fullhttp://www.ncbi.nlm.nih.gov/pubmed/17054678?tool=bestpractice.com
虽然经强化化疗治疗的年龄≥60 岁的患者经标准诱导化疗几乎可达到 60% 的完全缓解率,但 3 年内的复发率约为 90%。只有约 5% 至 15% 的这些患者可达到长期无病生存。[95]Milligan DW, Grimwade D, Cullis JO, et al; British Committee for Standards in Haematology. Guidelines on the management of acute myeloid leukaemia in adults. Br J Haematol. 2006;135:450-474.http://onlinelibrary.wiley.com/doi/10.1111/j.1365-2141.2006.06314.x/fullhttp://www.ncbi.nlm.nih.gov/pubmed/17054678?tool=bestpractice.com一些临床试验表明,与治疗强度较弱或姑息治疗方法相比,强化化疗只有极少的生存获益。许多临床试验对健康情况较好的患者给予试验性药物和低强度预处理异体干细胞移植治疗,并对其治疗结局进行了研究。[99]Burnett AK, Milligan D, Goldstone A, et al. The impact of dose escalation and resistance modulation in older patients with acute myeloid leukaemia and high risk myelodysplastic syndrome: the results of the LRF AML14 trial. Br J Haematol. 2009;145:318-332.http://www.ncbi.nlm.nih.gov/pubmed/19291085?tool=bestpractice.com
急性早幼粒细胞白血病 (APML)
采用目前的治疗方案可使 APML 治愈率超过 80%。[100]Kelaidi C, Chevret S, De Botton S, et al. Improved outcome of acute promyelocytic leukemia with high WBC counts over the last 15 years: the European APL Group experience. J Clin Oncol. 2009;27:2668-2676.http://www.ncbi.nlm.nih.gov/pubmed/19414681?tool=bestpractice.com当前试验的目的是确定可提供最大治愈率以及最小毒性的策略。