Department of Pediatrics, Tokyo Medical and Dental University, 1-5-45 Yushima, Bunkyo-ku, Tokyo, 113-8519, Japan,
Int J Hematol. 2013 Nov;98(5):578-88. doi: 10.1007/s12185-013-1429-2. Epub 2013 Sep 26.
Infants (<1 year old) with acute myeloid leukemia (AML) are particularly vulnerable to intensive cytotoxic therapy. Indeed, the mortality rate was high among infants enrolled in the Japanese Pediatric Leukemia/Lymphoma Study Group AML-05 study, which prompted us to temporarily suspend patient enrollment and amend the protocol. Forty-five infants with AML were enrolled. For patients aged <2 years, drug doses were adjusted for body weight. Following the protocol amendments, doses for infants were reduced by a further 33 % in the initial induction course. Six infants died during the induction phase (including five early deaths), mainly due to pulmonary complications. The 3-year probability of overall survival (pOS) in all 45 infants [55.9 %, 95 % confidence interval (CI) 37.9-70.6 %] was significantly lower than that of patients aged 1 to <2 years (77.0 %, 95 % CI 62.7-86.3 %) and those aged ≥2 years (74.7 %, 95 % CI 69.2-79.4 %) (P = 0.037), mainly due to the higher non-relapse mortality rate in infants. No early deaths occurred after the protocol amendments, and the 3-year pOS of the 17 infants enrolled thereafter was 76.4 % (95 % CI 48.8-90.4 %). In conclusion, appropriate dose reduction is essential to avoid early deaths when treating infants with AML.
婴儿（<1 岁）对强化细胞毒化疗特别敏感。事实上，在日本儿科白血病/淋巴瘤研究组 AML-05 研究中入组的婴儿死亡率很高，这促使我们暂时停止患者入组并修改方案。共有 45 名 AML 婴儿入组。对于<2 岁的患者，药物剂量按体重调整。在方案修订后，初始诱导期内婴儿的剂量进一步减少了 33%。6 名婴儿在诱导期死亡（包括 5 例早期死亡），主要死于肺部并发症。所有 45 名婴儿的 3 年总生存率（pOS）[55.9%，95%置信区间（CI）37.9-70.6%]明显低于 1-<2 岁患者（77.0%，95%CI 62.7-86.3%）和≥2 岁患者（74.7%，95%CI 69.2-79.4%）（P=0.037），主要是由于婴儿的非复发死亡率较高。方案修订后无早期死亡，此后入组的 17 名婴儿的 3 年 pOS 为 76.4%（95%CI 48.8-90.4%）。总之，在治疗 AML 婴儿时，适当减少剂量对于避免早期死亡至关重要。
